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AGTC Announces Positive Three-Month Interim Results from Skyline Trial of AGTC-501 for the Treatment of X-Linked Retinitis Pigmentosa

– 62.5% of Patients in Dose Group B Were Responders for Improvements in Visual Sensitivity, the Study’s Primary Efficacy Endpoint –

– Continued Strong Safety Data –

– Robust Safety and Efficacy Data Consistent with Previously Reported Results from the Phase 1/2 Trial in XLRP –

– Company to Host Conference Call to Review Data on May 17 at 8:00 a.m. ET –

GAINESVILLE, Fla. and CAMBRIDGE, Mass., May 16, 2022 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a clinical stage biotechnology company focused on the development of adeno-associated virus (AAV)-based gene therapies for the treatment of rare and debilitating diseases with an initial focus on inherited retinal diseases, today reported positive three-month interim data from its ongoing Phase 2 Skyline trial of AGTC-501, a recombinant AAV viral vector-based gene therapy targeting mutations in the RPGR gene in patients with X-linked retinitis pigmentosa (XLRP).

There were robust improvements in visual sensitivity, the trial’s primary efficacy endpoint, in multiple patients three months after dosing, with a 62.5% response rate in dose group B and a 25% response rate in dose group A. This is well above the statistically significant 50% response rate the Vista Phase 2/3 trial for XLRP is powered to detect. The Company considers a responder to be a patient who has an improvement in visual sensitivity as assessed by microperimetry of at least 7 decibels (dB) in at least 5 loci. Importantly, for responders in this trial, the number of loci that improved by at least 7 dB was between 9 and 17. It is also notable that the mean visual sensitivity of the entire macula increased and the area of the macula with visual sensitivity also increased in the patients who were responders. Although these results exceeded the current standard set by the United States Food and Drug Administration (FDA) that at least 5 loci increase by at least 7 dB, the improved loci were not pre-specified, as also required in the FDA standard.

There was a significant difference in visual sensitivity as assessed by MAIA microperimetry in the treated eyes compared with the untreated eyes of all Skyline responders at 3 months. In the Phase 1/2 trial, patients who were responders at 3 months remained responders at 12 months. Based on the durability of the Phase 1/2 improvements in visual sensitivity, we believe the results observed at 3 months in the Skyline trial will be consistent at 12 months. Additional data related to the Skyline trial is available on the AGTC website or by clicking here.

Consistent with the previously reported Phase 1/2 results, AGTC-501 was generally well-tolerated in this trial with no clinically relevant safety concerns attributed to the study agent. The majority of adverse events were non-serious and mild to moderate in severity. The Company believes that the favorable safety profile is largely the result of a strong focus on product design, extensive pre-clinical testing, enhanced inclusion and exclusion criteria, and surgical consistency.

“The meaningful response rate in visual sensitivity seen in this interim Skyline data is very encouraging, as are the favorable safety data,” said Dr. Robert Sisk, MD, FACS, FASRS, Director of Pediatric Vitreoretinal Surgery and Director of Ophthalmic Genetics – Cincinnati Children’s Hospital and the Cincinnati Eye Institute and an investigator in the trial. “Patients with XLRP have no FDA-approved options to treat this devastating disease and we believe the results presented today show great promise to provide outcomes that are truly meaningful and potentially life-changing to patients.”

Skyline is a 14 patient Phase 2 expansion of the Company’s Phase 1/2 clinical trial, in which patients are randomized to either a high or low dose of AGTC-501 with the main objective to evaluate the efficacy, safety and tolerability of AGTC-501. The primary endpoint is the proportion of treated eyes that demonstrate improvement from baseline in visual sensitivity at 12 months with secondary endpoints (also at 12 months) that include improvements in best-corrected visual acuity (BCVA) and the patient’s ability to navigate a mobility maze more successfully under varying light and challenge conditions. The interim analysis includes 13 male pediatric and adult patients that were treated over a 10-fold dose range in two groups. The fourteenth patient had not yet reached the three-month time point at the cut-off date for the interim analysis. Patients in the Skyline trial were younger than the patients in the comparable dose groups from the Phase 1/2 clinical trial with better BCVA and better mean visual sensitivity at baseline than in the previously reported Phase 1/2 study. Given the higher baseline BCVA, improvements in BCVA at three months were less pronounced than in the Phase 1/2 clinical trial. More than half of the patients, including four of the six responders in visual sensitivity, showed improvement in the mobility maze based on light levels passed, improved speed and/or reduced errors.

“We are incredibly excited by the compelling interim results seen in the Skyline trial, including strong safety data and robust improvements in visual sensitivity with a clear difference between the two dose groups. These results add to the growing body of evidence supporting the best-in-class potential of AGTC-501 for the treatment of XLRP,” said Sue Washer, President and Chief Executive Officer of AGTC. “We believe that if we achieve similar results in the Vista Phase 2/3 clinical trial, we will have a broad and compelling body of data to support the submission of a BLA to the FDA, and to enable a differentiated and highly competitive product profile.”

Conference Call and Webcast
AGTC will host a conference call and webcast to review the updated and previously reported interim study results tomorrow, May 17, at 8:00 a.m. ET. To access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US). A live webcast will be available in the Events and Presentations section of AGTC’s Investor Relations site at http://ir.agtc.com/events-and-presentations.

Please log in approximately 10 minutes prior to the scheduled start time. The archived webcast will be available in the Events and Presentations section of the Company’s website.

About X-Linked Retinitis Pigmentosa (XLRP)
X-linked Retinitis Pigmentosa (XLRP) is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th or 5th decade of life.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies with the potential to address unmet patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with inherited retinal diseases. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3). Its preclinical programs build on the company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical needs in optogenetics, otology and CNS disorders, and has entered strategic collaborations with companies including Bionic Sight, an innovator in the emerging field of optogenetics, and retinal coding and Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology. For more information, please visit https://agtc.com/.

Forward-Looking Statements 
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements about the potential of the company’s gene therapy platform and the strength of interim results from the Skyline Trial in XLRP, the potential of AGTC-501 as a treatment for XLRP, the ability to use the interim Skyline results as a predictor of the success of the final Skyline and Vista clinical trial results and whether these results will support future regulatory filings for AGTC-501. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; risks and uncertainties related to funding sources for our development programs; the direct and indirect impacts of the ongoing COVID-19 pandemic on the Company’s business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in the company’s most recent annual report on Form 10-K, as it may be supplemented by subsequent periodic reports filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future. 

PR Contact:
Kerry Sinclair
Spectrum Science Communications
[email protected]

Corporate Contact:
Jonathan Lieber
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5778
[email protected]


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Source: Applied Genetic Technologies Corporation

AGTC Hosts Topping Off Ceremony for Gene Therapy Manufacturing Facility to Celebrate the Building’s Structural Completion

 Facility in Alachua, Florida Is Expected to be Completed in the Fourth Quarter of 2022 –

GAINESVILLE, Fla. and CAMBRIDGE, Mass., May 10, 2022 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a clinical stage biotechnology company focused on the development of adeno-associated virus (AAV)-based gene therapies for the treatment of rare and debilitating diseases with an initial focus on inherited retinal diseases, recently hosted a topping off ceremony for its state-of-the-art gene therapy manufacturing facility during the BioFlorida Celebration of Biotechnology, signaling the completion of the building’s exterior structure.

“Leasing our own manufacturing facility is an essential part of our strategy to support the near term production of materials for upcoming clinical trials and eventually the potential production of commercial supply for any approved products,” said President and CEO of AGTC, Sue Washer. “We were thrilled to host this event as part of BioFlorida’s Celebration of Biotechnology and contribute to a growing biotech presence in the state. We look forward to the grand opening later this year and welcoming everyone to the facility.”

The new manufacturing building is part of the Company’s strategy to exert more control over its manufacturing needs and enable a more rapid filing of a Biologics Licensing Application and, if approved by the FDA, support the commercial launch of its XLRP candidate. The facility is also expected to support more rapid advancement of the Company’s product pipeline, providing improved quality assurance and supply chain redundancy while reducing manufacturing risk.

The event, held on May 5, began with remarks from BioFlorida’s President & CEO, Nancy Bryan, Matt Cason, President of Concept Companies, the project developer, and Sue Washer, President and CEO of AGTC. The event was attended by many in the local biotech community as well as the vendors and sub-contractors supporting the project. To commemorate the event, key members of the project team signed the last beam to be put in place this week.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies with the potential to address unmet patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with inherited retinal diseases. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3). Its preclinical programs build on the company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical needs in optogenetics, otology and CNS disorders, and has entered strategic collaborations with companies including Bionic Sight, an innovator in the emerging field of optogenetics, and retinal coding and Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology. For more information, please visit https://agtc.com/.

Forward-Looking Statements 
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements about the potential of the company’s gene therapy platform, the strength of its XLRP clinical program, whether clinical trial results will support future regulatory filings, the expected timing for completion of the manufacturing facility and the potential for the new manufacturing facility to support AGTC’s pipeline programs. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; risks and uncertainties related to funding sources for our development programs; the direct and indirect impacts of the ongoing COVID-19 pandemic on the Company’s business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in the company’s most recent annual report on Form 10-K, as it may be supplemented by subsequent periodic reports filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future. 

PR Contact:

Kerry Sinclair

Spectrum Science Communications

[email protected]

Corporate Contact:

Jonathan Lieber

Chief Financial Officer

Applied Genetic Technologies Corporation

T: (617) 843-5778

[email protected]


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Source: Applied Genetic Technologies Corporation

Mobile eye care clinic offering free exams, glasses to Boston students

Read the story on WCVB

Many people have put off medical care during the pandemic and that includes eye exams for children. But on Tuesday, the doctor went to the students at one Boston school.

At Blackstone Elementary in Roxbury, some students took a quick test outside the classroom, including Heaven, a fourth grader who sometimes has trouble seeing the board.

“My eyes — they are used to being shut like this — not open,” she said.

“Eighty percent of learning is visual, and 20% of students have a visual deficiency where they can’t see the board, and a simple pair of glasses can fix that,” said Alexis McLaughlin, CEO of 20/20 Onsite.

20/20 Onsite brings eye exams right to companies and schools with their mobile office.

“A lot of people don’t prioritize their eye health or they are in situations where they don’t have access to care,” McLaughlin said. “We just try to make it easy to get access to care and during the pandemic. We facilitate clinical research.”

Their visit Tuesday was funded by AGTC, a biotechnology company that decided to honor participants in their clinical trials by donating eye exams through Greater Gift.

“That really enforces for the patients involved in clinical trials, all involved in eye clinical trials, the importance of the impact of their contribution as clinical trial participants,” said Lilly Skok Bunch of Greater Gift.

Heaven does need a prescription and she picked out the perfect pair of free frames.

Any student who needs glasses will have them delivered to the school in two weeks.

AGTC Exceeds Enrollment Target in SKYLINE Trial of AGTC-501 for the Treatment of X-linked Retinitis Pigmentosa (XLRP)

January 11, 2022 at 8:00 AM EST
  • Company on track to report three-month interim study results, including visual acuity, visual sensitivity, mobility and safety data, in Q2 2022
  • Company providing further updated guidance on other milestones

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Jan. 11, 2022 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a clinical stage biotechnology company focused on the development and commercialization of adeno-associated virus (AAV)-based gene therapies for the treatment of rare and debilitating diseases with an initial focus on inherited retinal diseases (IRDs), today announced it had exceeded the enrollment target in the Company’s SKYLINE trial of AGTC-501, a recombinant AAV vector-based gene therapy developed for the treatment of X-linked retinitis pigmentosa (XLRP). A total of 14 patients have been enrolled across the high and low dose groups, exceeding the planned target enrollment of 12.

“We are pleased to have reached this important milestone in the clinical development of AGTC-501, which positions us to announce interim three-month trial results in the second quarter of 2022,” said Sue Washer, President and Chief Executive Officer of AGTC. “We’re looking forward to adding to the growing body of evidence that supports the potential of our product candidate and further validates the meaningful and durable benefits seen to date in treated patients. Specifically, we believe the mobility data, in conjunction with the visual acuity and visual sensitivity data from the high dose group in the SKYLINE trial, will potentially serve as a strong indicator of success for not only the final 12-month endpoint for the SKYLINE trial, but also for the Company’s VISTA trial. We are extremely grateful to the patients and investigators participating in our clinical trials for their help completing enrollment with a goal of bringing this potential therapeutic option to patients suffering from the progressive vision loss resulting from XLRP.”

SKYLINE is a multi-site expansion of the ongoing Phase 1/2 study in which patients are randomized to either a high or low dose of AGTC-501 with the primary objective to identify the proportion of treated eyes that demonstrate improvement from baseline in measures of visual sensitivity and visual acuity as well as the patient’s ability to navigate a mobility maze more successfully under varying light and challenge conditions. Importantly, this is the first trial that has the potential to demonstrate a correlation of visual sensitivity and visual acuity with the maze outcome, a key functional endpoint in the VISTA trial.

Earlier this year, AGTC released updated data from the ongoing Phase1/2 clinical trial of AGTC-501, which demonstrated that at 12 months, 50% of patients in the high dose groups were considered responders to treatment based on visual sensitivity and that treatment resulted in a statistically significant improvement in best-corrected visual acuity (BCVA) across all treatment groups in patients where the macula was treated. Additionally, key findings on a patient’s baseline characteristics, combined with a novel application of machine learning techniques, are being applied to the SKYLINE and VISTA trials to potentially further enhance the ability to identify likely responders. Importantly, at 12 months post treatment, primary outcome measures of safety continued to indicate AGTC-501 to be generally safe and well-tolerated at all doses. The SKYLINE and VISTA trials are intended to support registration if they are able to show continued safety and comparable response rates.

The Company anticipates releasing the following, subject to any continuing impact of COVID-19:

  • 24-month results from the ongoing Phase 1/2 clinical trial of AGTC-501 in the third quarter of calendar year 2022;
  • 12-month results from the SKYLINE trial in the first quarter of calendar year 2023; and
  • interim results from the VISTA trial in the first half of calendar year 2023.

About X-linked Retinitis Pigmentosa (XLRP)
X-linked Retinitis Pigmentosa (XLRP) is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th or 5th decade of life.

About AGTC-501
AGTC-501 (laruparetigene zosaparvovec), the Company’s lead gene therapy development program, uses an engineered AAV vector to insert a stabilized and functional copy of the Retinitis Pigmentosa GTSase Regulator (RPGR) gene into a patient’s photoreceptor cells. AGTC-501 is comprised of that stabilized RPGR gene and a promoter that was specifically selected due to its ability to drive efficient gene expression in rods and cones, maintain photoreceptor function and delay disease progression in large animal, naturally occurring preclinical models of XLRP. In addition, published NHP studies have demonstrated that AGTC’s proprietary AAV capsid has as much as twice the transfection efficiency in photoreceptors compared to capsids used in competing programs. AGTC was granted U.S. Food and Drug Administration (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies with the potential to address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders, and has entered into strategic collaborations with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.

Forward-Looking Statements This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements about the potential of the Company’s late-stage development programs in X-Linked Retinitis Pigmentosa (XLRP), including the expected timing for the release of data in its SKYLINE and VISTA trials and the likelihood that these results further support the potential of AGTC-501. Forward-looking statements also include information concerning preclinical and clinical product development and regulatory progress, potential growth opportunities, and potential market opportunities. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition, including impacts on supply chain and availability of clinical trial material; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in our most recent annual report on Form 10-K and subsequent periodic reports filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

PR Contact:

Kerry Sinclair

Spectrum Science Communications

[email protected]

Corporate Contact:

Jonathan Lieber

Chief Financial Officer

Applied Genetic Technologies Corporation

T: (617) 843-5778

[email protected]

 


Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC Appoints Biopharmaceutical Industry Leader James Robinson to its Board of Directors

December 6, 2021 at 8:00 AM EST

Robinson’s expertise in development and commercialization adds to the clinical and commercial experience of newly appointed management team
GAINESVILLE, Fla. and CAMBRIDGE, Mass., Dec. 06, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a clinical stage biotechnology company focused on the development and commercialization of adeno-associated virus (AAV)-based gene therapies for the treatment of rare and debilitating diseases with an initial focus on inherited retinal diseases (IRDs), today announced the appointment of James Robinson, President and Chief Executive Officer (CEO) of Urovant Sciences, to its Board of Directors.

“We are honored to have Jim join our Board of Directors, where his exemplary track record of execution as an executive at leading biotech and pharma companies will be invaluable to AGTC as we seek to deliver on the clinical and commercial promise of our robust pipeline including our late stage XLRP and ACHM programs,” said Sue Washer, President and CEO of AGTC. “Jim’s appointment, along with the recent appointments to our executive leadership team, usher in a new, exciting chapter for AGTC as we close a successful 2021 and focus on our many opportunities for success in 2022 including key milestones in XLRP and ACHM.”

Mr. Robinson is an expert in building, deploying and leading successful organizations from their earliest stages of development through their emergence as commercial enterprises, and has successfully led multiple global businesses, operations and commercial ventures over the past 28 years. A proven industry leader, Mr. Robinson served as a member of the Urovant Board of Directors prior to being appointed Urovant’s CEO in 2020. Prior to his role at Urovant, Mr. Robinson held the position of President and Chief Operating Officer at Paragon Biosciences. He came to this role after serving as the President and Chief Operating Officer of Alkermes, where he was responsible for global commercial operations, new product planning, corporate planning, manufacturing, quality, human resources and business development.

“I am excited to join AGTC’s Board of Directors at a time when the organization has already made impressive progress developing its pipeline of gene therapies, particularly in ophthalmology,” said Mr. Robinson. “I look forward to contributing my operational and commercial experience and working closely with Sue and the executive leadership team.”

Prior to Alkermes, Mr. Robinson spent more than a decade at Astellas U.S. – most recently as President, Americas Operations and previously as President of Astellas Pharma US, where he was responsible for leading the U.S. commercial organization. Prior to Astellas, Mr. Robinson spent thirteen years at Schering-Plough Pharmaceuticals where his last role was Vice President, Hepatitis Sales and Managed Care.

Mr. Robinson serves on the Board of Directors for Urovant Sciences and is an advisor to BridgeBio Pharma. Mr. Robinson previously served on the Board of Directors of the Pharmaceutical Research and Manufacturers of America (PhRMA) and served as Chairman of PhRMA’s State Committee. He is a founding member of MATTER, the Commercial Club of Chicago and member of the Chicago Chapter of the Young Presidents Organization. Mr. Robinson received a Bachelor of Science degree from DePaul University.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies with the potential to address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders, including entering into strategic collaborations with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding. For more information, please visit www.agtc.com/.

Forward-Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements about the potential of the Company’s AGTC-501 product candidate, the ongoing late-stage development programs in XLRP and ACHM and the potential for the Company’s gene therapy platform. Forward-looking statements include information concerning preclinical and clinical product development and regulatory progress, potential growth opportunities, and potential market opportunities. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process, including that interim results are not necessarily indicative of final results; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in our most recent annual report on Form 10-K and subsequent periodic reports filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

PR Contact:

Kerry Sinclair

Spectrum Science Communications

[email protected]

Corporate Contact:

Jon Lieber

Chief Financial Officer

Applied Genetic Technologies Corporation

T: (617) 843-5778

[email protected]

 


Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC Appoints Abraham Scaria, Ph.D., as Chief Science Officer

November 16, 2021 at 8:00 AM EST
  • With 25+ years of experience, Dr. Scaria has led numerous gene therapy programs from early research into clinical development 
  • Dr. Scaria completes a newly expanded management team in place to continue development of late-stage programs in XLRP and Achromatopsia and advance AGTC’s preclinical pipeline

GAINESVILLE, Fla., and CAMBRIDGE, Mass., Nov. 16, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare inherited retinal diseases (IRDs), today announced the appointment of Abraham Scaria, Ph.D., as Chief Science Officer.

“A gene therapy pioneer with deep knowledge of retinal and rare diseases, Dr. Scaria will bring invaluable insights and expertise as we advance and seek to expand our pipeline of gene therapy programs,” said Sue Washer, President and Chief Executive Officer of AGTC. “We are privileged to have an accomplished scientist and researcher join us in making transformational gene therapies a reality for patients.”

Dr. Scaria has more than 25 years of experience in the biotech and pharmaceutical industries, with deep knowledge in discovery research, preclinical studies and phase 1/2 clinical trials, with a focus on gene therapies to treat rare diseases. Most recently, Dr. Scaria was the Senior Vice President and Chief Science Officer at IVERIC Bio, where he was responsible for preclinical research and development for retinal disease therapies.

“I am excited to become part of the dedicated and talented team at AGTC and to help lead the way in providing breakthrough therapies for patients suffering from inherited retinal diseases and other debilitating indications, many of which have no treatment options available,” said Abraham Scaria, Ph.D. “My research and development experience with a wide variety of gene therapies will be beneficial as the team at AGTC enters a new phase of growth and advances both their clinical and pre-clinical product candidates.”

Before his tenure at IVERIC Bio, Dr. Scaria served as Vice President and Head of Ophthalmology at Casebia Therapeutics, where he built out the ophthalmology research and development group from the ground-up. Prior to Casebia, Dr. Scaria served as the Head of Gene Therapy Research at Sanofi Genzyme, where he was responsible for all aspects of gene therapy research, including AAV vector mediated gene delivery optimization. Previously, he was the Senior Scientific Director for Gene Therapy and Ophthalmology Research at Genzyme Corporation.

Dr. Scaria earned his Ph.D. at the Indiana University School of Medicine and his M.Sc. at the University of Bombay in India.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies with the potential to address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders, and has entered into strategic collaborations with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.

Forward-Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements about the potential of the Company’s late-stage development programs in X-Linked Retinitis Pigmentosa (XLRP) and Achromatopsia (ACHM) and its ability to expand its existing pipeline of gene therapy programs. Forward-looking statements include information concerning preclinical and clinical product development and regulatory progress, potential growth opportunities, and potential market opportunities. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in our most recent annual report on Form 10-K and subsequent periodic reports filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

PR Contact:

Kerry Sinclair

Spectrum Science Communications

T: (202) 587-1507

[email protected]

Corporate Contact:

Jonathan Lieber

Chief Financial Officer

Applied Genetic Technologies Corporation

T: (617) 843-5778

[email protected]

 

Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC Presents Data from Ongoing Phase 1/2 Trial of AGTC-501 at Upcoming American Academy of Ophthalmology 2021 Annual Meeting

November 12, 2021 at 8:00 AM EST

– Poster presentation highlights safety and efficacy potential of AGTC-501 as a treatment for X-linked retinitis pigmentosa (XLRP) –
GAINESVILLE, Fla. and CAMBRIDGE, Mass., Nov. 12, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare inherited retinal diseases (IRDs), today announced the presentation of additional analyses and conclusions from previously reported data from the Company’s ongoing Phase 1/2 study of AGTC-501, a recombinant AAV2 vector developed for the treatment of X-linked retinitis pigmentosa (XLRP), at the American Academy of Ophthalmology (AAO) 2021 Annual Meeting being held today in New Orleans, Louisiana by Dr. Robert Sisk, Director of Pediatric Vitreoretinal Surgery and Director of Ophthalmic Genetics – Cincinnati Children’s Hospital and the Cincinnati Eye Institute.

In the non-randomized, open-label study, secondary outcome measures of clinical activity were observed through functional improvements in treated versus untreated control eyes. Treatment with AGTC-501 resulted in a statistically significant improvement in best-corrected visual acuity (BCVA) across all treatment groups (n=20) in patients where the macula was treated. At 12 months, 50% of patients were considered responders, meeting the strict criteria, as discussed with the FDA, of at least a 7 decibels (dB) improvement in at least 5 loci, as measured by macular integrity assessment (MAIA) microperimetry.

“I am encouraged by the responses we have seen in this trial to date, including the safety and ability of AGTC-501 to produce clinically meaningful improvements in macular sensitivity and statistically significant improvements in visual acuity,” said Dr. Sisk. “These data bring us one step closer to improving outcomes for patients losing their vision as a result of XLRP, a condition for which there are currently no treatment options.”

At 12 months post treatment, primary outcome measures of safety demonstrated AGTC-501 to be well-tolerated at all doses. No serious adverse events related to AGTC-501 were reported. All adverse events were Grade 1-2 in centrally treated patients, including those related to the subretinal injection procedure and importantly, immunological assessments did not indicate safety concerns. An important biomarker of efficacy was demonstrated with restoration of macular ellipsoid zone (EZ) as measured by OCT in patients with measurable EZ and microperimetry at the baseline visit.

“We continue to be very excited about these 12-month findings for our lead candidate AGTC-501, as they represent a powerful indicator of the anticipated promise of both AGTC-501 and our gene therapy platform,” said Sue Washer, President and Chief Executive Officer of AGTC. “These data, coupled with our ongoing clinical trials, gives us confidence that we are on a course to make gene therapies for rare retinal diseases a reality for patients.”

Dr. Sisk’s presentation, Clinically Meaningful Visual Improvements Demonstrated 12 Months After AGTC-501 Gene Therapy for X-linked Retinitis Pigmentosa, will be available on demand November 12-15, 2021 during AAO 2021.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies with the potential to address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders, including entering into strategic partnerships with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.

Forward-Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements about the potential of the Company’s AGTC-501 product candidate, the ongoing late-stage development program in XLRP and the potential for the Company’s gene therapy platform. Forward-looking statements include information concerning preclinical and clinical product development and regulatory progress, potential growth opportunities, and potential market opportunities. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process, including that interim results are not necessarily indicative of final results; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in our most recent annual report on Form 10-K and subsequent periodic reports filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

PR Contact:

Kerry Sinclair

Spectrum Science Communications

[email protected]

Corporate Contact:

Jon Lieber

Chief Financial Officer

Applied Genetic Technologies Corporation

T: (617) 843-5778

[email protected]


Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC Appoints Hope D’Oyley-Gay as General Counsel

November 11, 2021 at 8:00 AM EST
  • Ms. D’Oyley-Gay brings significant legal experience as well as business development and commercial operations expertise from both biotech and pharmaceutical companies

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Nov. 11, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare inherited retinal diseases (IRDs), today announced the appointment of Hope D’Oyley-Gay as General Counsel. In this role Ms. D’Oyley-Gay will oversee legal, IP and compliance for the Company.

“We are excited to welcome Hope D’Oyley-Gay to AGTC, and to have the benefit of her tremendous legal expertise. Additionally, with her extensive business development and commercial experience, she will play an important role in our efforts to advance our pipeline of valuable gene therapies towards potential approval and commercialization,” said Sue Washer, President and Chief Executive Officer of AGTC. “Hope is another high-profile executive that has chosen to join our talented leadership team and we are looking forward to the contributions that she will make in our efforts towards bringing transformational genetic therapies for patients with rare and debilitating diseases.”

Ms. D’Oyley-Gay has more than 25 years of legal experience, with the majority of her career spent in the healthcare industry, and deep knowledge of pharmaceutical, gene therapy and life sciences companies. Most recently, Ms. D’Oyley-Gay was General Counsel and Vice President of Administration at Spirovant Sciences where she was a member of the company’s executive leadership team, helping to develop strategy for, and lead operations of, the company.

“I am thrilled to join the team at AGTC, whose decades of experience in gene therapy have established a strong foundation for growth and success,” said Hope D’Oyley-Gay, General Counsel of AGTC. “I’m looking forward to bringing my experience both as in-house counsel and at leading law firms to deliver on the promise of AGTC’s scientific vision and bring gene therapies to patients with rare diseases.”

Before her tenure at Spirovant Sciences, Ms. D’Oyley-Gay previously served as Counsel at Reed Smith, LLC, where she represented biotechnology and pharmaceutical companies in a variety of strategic collaborations. Ms. D’Oyley-Gay also served as an Executive-In-Residence at Militia Hill Ventures, where she supported the building and growth of successful life sciences companies. Previously, she was Vice President and Associate General Counsel at GlaxoSmithKline, where she supported worldwide business development on numerous strategic collaborations, including licenses, divestments, joint ventures and mergers and acquisitions.

Ms. D’Oyley-Gay earned her J.D. at the University of Pennsylvania Law School and her B.S. in Economics from the University of Delaware.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies with the potential to address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders, and has entered into strategic collaborations with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.

Forward-Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements about the Company’s development programs and gene therapy platform. Forward-looking statements include information concerning preclinical and clinical product development and regulatory progress, potential growth opportunities, and potential market opportunities. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” promise”, “”should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process, including that interim results are not necessarily indicative of final results; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in our most recent annual report on Form 10-K and subsequent periodic reports filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

PR Contact:

Kerry Sinclair
Spectrum Science Communications
[email protected]

Corporate Contact:
Jon Lieber
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5778
[email protected]

 


Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC Announces Financial Results and Business Update for the Quarter Ended September 30, 2021

November 9, 2021 at 7:00 AM EST

-Company on track to provide multiple data readouts for XLRP and ACHM clinical programs in 2021 and 2022-

-Company to host management update and webcast today at 8:00 a.m. ET-

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Nov. 09, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare inherited retinal diseases (IRDs), today announced financial results for the quarter ended September 30, 2021.

“We have significant momentum toward achieving multiple clinical and regulatory milestones in the year ahead. We believe that the data we have generated to date in the Phase 1/2 XLRP clinical trial point to a higher likelihood of success in achieving similar results in both the Skyline and Vista trials and look forward to sharing that data in 2022,” said Sue Washer, President and CEO of AGTC. “In order to execute on these opportunities, we have strengthened our management team with individuals who we believe will provide additional expertise to navigate the clinical development and regulatory pathways for both our clinical programs and the exciting preclinical product candidates we are advancing towards potential IND filings. We are confident that we will be able to generate value for patients, clinicians and stockholders.”

Recent Highlights

X-linked Retinitis Pigmentosa (XLRP)

The Company is currently conducting the Skyline expanded Phase 1/2 XLRP and Vista Phase 2/3 XLRP clinical trials to support a potential filing of a Biologics License Application (BLA). Earlier in the year, the Company reported positive 12-month data from the highest dose groups in the ongoing XLRP Phase 1/2 clinical trial, including 12-month data from Groups 5 and 6 reflecting a 50% response rate among patients who meet the inclusion criteria for the Skyline and Vista trials. In addition, the Company reported 24-month data from two Group 4 patients that provides preliminary evidence of response durability. These data together with the statistically significant improvements in visual acuity and data presented in September 2021 indicating a correlation between improvements in macular structure and visual function as well as continued evidence of a favorable safety profile add to the growing body of evidence suggesting the significant benefit possible for patients receiving AGTC’s XLRP product candidate.

Over the next 12 months, the Company expects to achieve the following milestones in the XLRP development program:

  • Presentation of 12-month trial results from the ongoing Phase 1/2 clinical trial at the American Academy of Ophthalmology Annual Meeting being held from November 12 – 15, 2021;
  • Release of 3-month interim Skyline trial results in 1H 2022;
  • Release of 24-month results from the ongoing Phase 1/2 clinical trial in 3Q 2022;
  • Release of 12-month Skyline trial results in 4Q 2022; and
  • Release of 6-month interim Vista trial results in 4Q 2022.

 

Achromatopsia (ACHM)

In June, the Company reported 12-month data from its ongoing Phase 1/2 ACHM clinical trials, including data from all adult and low-dose pediatric patients. In the ACHMB3 trial, the product candidate demonstrated biologic activity based on improvements in visual sensitivity and light discomfort, both of which were supported by anecdotal patient reports. In addition, the safety profile of the product candidate remained favorable. Based on these data, AGTC intends to advance the ACHMB3 program to the next stage of clinical development.

AGTC recently enrolled six pediatric ACHMB3 patients and five pediatric ACHMA3 patients in higher dose groups 5a and 6a and has achieved a key objective of the trial – the identification of a maximum tolerated dose. To address the previously reported Suspected Unexpected Serious Adverse Reactions, or SUSARs, in three pediatric patients in the highest dose group 6a, systemic and local steroid doses were increased. In addition, patient monitoring also increased and the investigators are now tapering these patients to lower doses of steroids and all patients are improving.

Over the next 12 months, the Company expects to achieve the following milestones in the ACHM development program:

  • Release of 3-month data from the pediatric patients in both trials in 4Q 2021; and
  • Submission of an End-of-Phase 2 briefing packet to the U.S. Food and Drug Administration (FDA) and receipt of feedback in 1H 2022.

Bionic Sight Collaboration

Earlier in 2021 the Company’s collaborator, Bionic Sight, announced promising initial Phase 1/2 data in a retinitis pigmentosa optogenetics study that showed that treated patients, all of whom have complete or near-complete blindness, can now see light and motion, and, in two cases, can detect the direction of motion.

Manufacturing Facility

In May, the Company announced an expansion of its manufacturing and analytics capabilities by leasing a 21,250 square foot build-to-suit current Good Manufacturing Practice (cGMP) manufacturing facility adjacent to the Company’s Florida facility. The company expects the new facility to be operational in the fourth quarter of 2022 and has already made several key hires to support the design and construction of the building. The facility is a key part of the Company’s strategy to expedite the potential BLA filing and commercial launch, subject to FDA approval, of its candidate for the treatment of XLRP, support the manufacture and supply of materials for the ACHM and early pipeline programs, and to provide overall supply chain security and redundancy.

Preclinical Programs

AGTC has a broad and diversified preclinical pipeline addressing patient populations with significant unmet needs, including an additional ophthalmology program targeting the dry form of age-related macular degeneration (dAMD), two programs targeting central nervous system (CNS) disorders – one in Frontotemporal Dementia (FTD) and the second in Amyotrophic Lateral Sclerosis (ALS) – and a program targeting non-syndromic hearing loss through its strategic collaboration with Otonomy, Inc. (Otonomy). The dAMD and FTD programs are in advanced preclinical development with expected toxicology and biodistribution studies to begin in 2022. The program with Otonomy is expected to move forward with an Investigational New Drug (IND) application in the first half of 2023.

Leadership Appointments

AGTC recently announced the appointments of Susan Schneider, M.D. as Chief Medical Officer and Sarah C. DiSalvatore, M.P.H. as Vice President of Clinical Operations.

Dr. Schneider is a biopharmaceutical leader with a proven track record of delivering high quality clinical results, globally, across multiple ophthalmology drug development programs including LUCENTIS®. An ophthalmologist by training, her expertise in developing high performing cross-functional teams and building strong therapeutic development programs will further strengthen AGTC’s leadership in advancing the Company’s clinical stage assets.

Ms. DiSalvatore is an experienced and an accomplished pharmaceutical professional who brings deep expertise in research and development to AGTC’s clinical operations team, including laboratory bench work, unique strategies to expedite clinical trial enrollment as well as the preparation of IND and New Drug Applications / BLA filings.

Financial Results for the Quarter Ended September 30, 2021

R&D Expenses: Research and development expenses were $12.3 million for the quarter ended September 30, 2021 compared to $11.6 million during the same period in the prior year. The increase of $0.7 million was primarily due to increased external XLRP spending to support planned manufacturing, clinical site preparation and other activities related to the Skyline and Vista trials and higher employee-related costs that were primarily attributable to increased headcount. These increases were partially offset by decreased external spending on the ACHM development program.

G&A Expenses: General and administrative expenses were $4.1 million for the quarter ended September 30, 2021 compared to $3.4 million during the same period in the prior year. The increase of $0.7 million was primarily due to higher operating and business development costs pertaining to our recurring operations, partially offset by lower legal fees.

Interest Expense: Interest expense for the quarter ended September 30, 2021 increased by $0.3 million compared to the same period in the prior year due to supplemental borrowing by the Company in May 2021 under its amended loan agreement with Hercules Capital, Inc.

Net Loss: The Company’s net loss was $17.1 million and $15.4 million for the quarters ended September 30, 2021 and 2020, respectively.

Financial Guidance: As of September 30, 2021, the Company’s cash and cash equivalents totaled $90.5 million. Management believes that these funds will be sufficient to allow the Company to generate data from its ongoing and planned clinical programs and fund currently planned research and discovery programs into calendar year 2023.

Conference Call and Webcast

AGTC will host a conference call and webcast to review business operations and discuss financial results for the quarter ended September 30, 2021 today at 8:00 a.m. ET. To access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US). A live webcast will be available in the Events and Presentations section of AGTC’s Investor Relations site at http://ir.agtc.com/events-and-presentations.

Please log in approximately 10 minutes prior to the scheduled start time. The archived webcast will be available in the Events and Presentations section of the company’s website.

About AGTC

AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies with the potential to address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical needs in optogenetics, otology and CNS disorders, including entering into strategic partnerships with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.

Forward-Looking Statements

This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements about the potential and expected timeline for the Company’s late-stage development programs in X-Linked Retinitis Pigmentosa (XLRP) and Achromatopsia (ACHM), the timing and potential of the Company’s pre-clinical programs, partnered programs and collaborations, and the timing for an impact of its planned manufacturing facilities. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic, including the impact on its ability to obtain the raw materials necessary to conduct its clinical trials. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in our most recent annual report on Form 10-K, as updated by subsequent quarterly reports on Form 10-Q and in other reports we file with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

APPLIED GENETIC TECHNOLOGIES CORPORATION
CONDENSED BALANCE SHEETS
(Unaudited)

In thousands September 30, 2021 June 30, 2021
ASSETS
Current assets:
Cash and cash equivalents $ 90,515 $ 105,052
Investments 2,000
Prepaid and other current assets 2,871 2,655
Total current assets 93,386 109,707
Property and equipment, net 4,550 4,658
Intangible assets, net 1,303 1,287
Investment in Bionic Sight, LLC 7,969 8,000
Right-of-use assets – operating leases 3,059 3,167
Right-of-use asset – financing lease 23 34
Other assets 121 113
Total assets $ 110,411 $ 126,966
LIABILITIES AND STOCKHOLDERS’ EQUITY
Current liabilities:
Accounts payable $ 2,083 $ 1,879
Accrued and other liabilities 14,061 14,500
Lease liabilities – operating 1,121 1,116
Lease liability – finance 26 38
Current portion of long-term debt 4,418 2,181
Total current liabilities 21,709 19,714
Lease liabilities – operating, net of current portion 3,218 3,418
Long-term debt, net of debt discounts and deferred financing fees 15,659 17,727
Other liabilities 300 299
Total liabilities 40,886 41,158
Stockholders’ equity:
Preferred stock
Common stock 43 43
Additional paid-in capital 326,126 325,245
Treasury stock (256 ) (211 )
Accumulated deficit (256,388 ) (239,269 )
Total stockholders’ equity 69,525 85,808
Total liabilities and stockholders’ equity $ 110,411 $ 126,966

APPLIED GENETIC TECHNOLOGIES CORPORATION
CONDENSED STATEMENTS OF OPERATIONS
(Unaudited)

Three Months
Ended September 30,
In thousands, except per share data 2021 2020
Revenue $ $
Operating expenses:
Research and development 12,325 11,626
General and administrative and other 4,100 3,436
Total operating expenses 16,425 15,062
Loss from operations (16,425 ) (15,062 )
Other income (expense), net:
Investment income, net 6 64
Interest expense (669 ) (332 )
Total other income (expense), net (663 ) (268 )
Loss before provision for income taxes (17,088 ) (15,330 )
Provision for income taxes 21
Loss before equity in net losses of an affiliate (17,088 ) (15,351 )
Equity in net losses of an affiliate (31 ) (29 )
Net loss $ (17,119 ) $ (15,380 )
Weighted average shares outstanding:
Basic 42,823 25,818
Diluted 42,823 25,818
Net loss per common share:
Basic $ (0.40 ) $ (0.60 )
Diluted $ (0.40 ) $ (0.60 )

IR Contact: 
David Carey
Lazar FINN Partners
T: (212) 867-1768
[email protected]

Corporate Contact:
Jonathan Lieber
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5778
[email protected]


Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC Clinical Trial Investigators to Present Data from Ongoing XLRP and Achromatopsia Phase 1/2 Trials at the Fourteenth International Symposium on Retinal Degeneration

September 28, 2021 at 8:00 AM EDT

Dr. Rachel Huckfeldt and Dr. Paul Yang to Present 12-Month Findings and Analysis from AGTC’s Clinical Trials
GAINESVILLE, Fla. and CAMBRIDGE, Mass., Sept. 28, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare inherited retinal diseases, today announced a presentation of the Company’s ongoing clinical trials in achromatopsia (ACHM) and X-linked retinitis pigmentosa (XLRP) at the Fourteenth International Symposium on Retinal Degeneration (RD2021).

Rachel Huckfeldt, MD, PhD, Assistant Professor of Ophthalmology at Harvard Medical School will present Twelve-month Findings from Two Phase 1/2 Clinical Trials of Subretinal Gene Therapy for Achromatopsia in adults and low-dose pediatrics on September 29, 2021, at 9:35 AM ET.

Paul Yang, MD, PhD, Assistant Professor of Ophthalmology at the Casey Eye Institute, Oregon Health & Science University in Portland, will present Twelve-Month Analysis of Macular Structure using Optical Coherence Tomography (OCT) from a Phase 1/2 Clinical Study of Subretinal Gene Therapy Drug AGTC-501 for X-Linked Retinitis Pigmentosa, on September 29, 2021, at 10:35 AM ET.

“We look forward to our investigators having the opportunity to share analysis of these results with members of the eye health community,” said Sue Washer, President and CEO of AGTC. “We believe the data provide important validation for the broad application of our AAV technology platform, including the best-in-class potential for our XLRP therapy candidate and further clinical investigation of our therapy to treat ACHM.”

“ACHM is an inherited condition caused by mutations in one of several genes, including the CNGB3 or CNGA3 genes, resulting in nonfunctioning “cone” photoreceptors responsible for color vision, and is associated with poor visual acuity, extreme light sensitivity leading to daytime blindness, and partial or complete loss of color discrimination,” said Dr. Huckfeldt. “These results are very encouraging, and I look forward to sharing them with my fellow retinal specialists.”

“Patients with XLRP experience damage to photoreceptors in the retina (both rods and cones) leading to declining vision in dim light conditions and progressive peripheral vision loss that can eventually result in legal blindness,” said Dr. Yang. “The sustained durability of improved visual function over 12 months is compelling evidence of biological activity for this XLRP gene therapy.”

AGTC plans to advance its ACHM program to the next stage of clinical development and is moving forward on an End-of-Phase 2 (EOP2) briefing packet to submit to the U.S. Food and Drug Administration, developing assays for pivotal ready testing, and planning production of clinical trial material. Along with the 12-month results in XLRP and other key XLRP data previously released by the company, AGTC is currently executing the Skyline and Vista Phase 2/3 trials that will expand safety and efficacy analyses, including microperimetry and luminance mobility maze outcomes.

Learn more and register here for the virtual/digital event to attend these important presentations: https://bit.ly/3z9uHre.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHMB3 and ACHMA3). Its preclinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders. In recent years AGTC has entered into strategic partnerships with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.

Forward-Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements about the potential of the Company’s late-stage development programs in X-Linked Retinitis Pigmentosa (XLRP) and Achromatopsia (ACHM). Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic, including the impact on its ability to obtain the raw materials necessary to conduct its clinical trials. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in our most recent annual report on Form 10-K and subsequent periodic reports filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

PR Contact:
Bryan Blatstein
Spectrum Science Communications
T: (212) 468-5379 or (917) 714-2609
[email protected]

Corporate Contact:
Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

 


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Source: Applied Genetic Technologies Corporation

AGTC to Host Fourth Quarter and 2021 Fiscal Year End Financial Results Conference Call and Webcast on September 23, 2021

September 16, 2021 at 8:00 AM EDT

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Sept. 16, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare inherited retinal diseases (IRDs), today announced that it will report financial results for the quarter and year ended June 30, 2021 after the market closes on Thursday, September 23, 2021. AGTC management will host a conference call beginning at 4:30 PM Eastern Time on the same date to review results and provide a corporate update.

To access the call, dial (844) 646-2697 (US) or (918) 922-6902 (outside of the US). A live webcast will be available in the Events and Presentations section of the Investor Relations page at http://ir.agtc.com/events-and-presentations. Please log in approximately 10 minutes prior to the scheduled start time. The archived webcast will be available in the Events and Presentations section of the company’s website.

About AGTC

AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies with the potential to address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is also advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders, including entering into strategic partnerships with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.

IR CONTACT:

Jonathan Nugent

Stern IR

T: 212-698-8698

[email protected]

Corporate Contacts:

Stephen Potter

Chief Business Officer

Applied Genetic Technologies Corporation

T: (617) 413-2754

[email protected]

 


Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC to Participate in Clinical Trials in Rare Diseases Conference Panel and Case Study

September 9, 2021 at 8:00 AM EDT

AGTC patient advocacy executive director and director of patient enrollment to speak at Clinical Trials in Rare Diseases Conference

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Sept. 09, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare inherited retinal diseases (IRDs), today announced that Jill Dolgin, PharmD, Executive Director of Global Patient Advocacy, and Halley Losekamp, Director of Patient Enrollment, will participate as speakers at the Clinical Trials in Rare Diseases Conference, September 14, 2021 starting at 9 AM ET.

Dr. Dolgin will join Stephanie Kalley of Crinetics Pharmaceuticals, Wendy Erler of Alexion Pharmaceuticals, and a senior representative of Scout Clinical to discuss how to work effectively with patients and advocacy groups to ensure studies are accessible and of maximum benefit to patients. This panel discussion will be moderated by a senior representative of GlobalData. Ms. Losekamp will present a case study on patient recruitment and retention in a rare disease setting.

“AGTC understands the need for and benefit of accessible and diverse studies that facilitate trial accessibility for all potential participants,” said Dr. Dolgin. “It is an honor to join a panel of industry experts to address how we can work better with patients and advocacy groups to create inclusive, diverse, and accessible studies to maximize the potential for patient benefits.”

“Our clinical trial and patient recruitment work at AGTC has provided us with many perspectives on how to recruit and retain patients in a rare disease setting,” said Ms. Losekamp. “AGTC is excited to share our work and lessons learned in patient recruitment, including virtual recruitment and factors to take into account to improve patient experience, especially during the COVID-19 pandemic.”

Dr. Dolgin’s panel entitled, “Working effectively with patients and advocacy groups to ensure your study is accessible and of maximum benefit to patients,” begins at 11:15 AM ET, and Ms. Losekamp’s case study entitled, “Patient recruitment and retention in a rare disease setting,” begins at 1:30 PM ET. To register for the event, visit https://bit.ly/3ywCb7j.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders. In recent years AGTC has entered into strategic partnerships with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.

Forward-Looking Statements

This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements about the potential of the Company’s late-stage development programs in X-Linked Retinitis Pigmentosa (XLRP) and Achromatopsia (ACHM). Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic, including the impact on its ability to obtain the raw materials necessary to conduct its clinical trials. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in our most recent annual or quarterly report and in other reports we have filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

PR Contact:

Bryan Blatstein
Spectrum Science Communications
T: (212) 468-5379 or (917) 714-2609
[email protected]

Corporate Contact:

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

AGTC is Excited to Announce Seasoned Healthcare Financial and Operations Executive Jonathan I. Lieber as their new Chief Financial Officer

September 8, 2021 at 8:00 AM EDT

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Sept. 08, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare retinal diseases, today announced Jonathan I. Lieber as their new Chief Financial Officer.

“We are thrilled to welcome Jonathan Lieber to the AGTC team,” said Sue Washer, President and CEO of AGTC. “He’s a seasoned financial and operations executive with valuable experience in the healthcare sector, making him a great addition to the AGTC team. We are excited to see his strategic perspective and apply it to our operational planning.”

Mr. Lieber brings 30 years of experience as a both CFO for emerging growth public and private life sciences companies and an investment banker. In his role as a CFO, Mr. Lieber has led numerous capital raises and had responsibility for financial reporting and regulatory compliance. He has developed relationships with investors, and provided strategic counsel to C-suite executives and Boards of Directors to support strategic planning, business development, and investor relations activities.

“I’m excited to become a part of the exciting work that AGTC is doing in the gene therapy world,” said Mr. Lieber. “AGTC’s work is leading the way to meet an unmet medical need in the ophthalmology community, and I’m thrilled that I have the opportunity to help AGTC execute on its future plans.”

About AGTC

AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders. In recent years AGTC has entered into strategic partnerships with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.

Forward-Looking Statements

This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements about the potential of the Company’s late-stage development programs in X-Linked Retinitis Pigmentosa (XLRP) and Achromatopsia (ACHM). Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic, including the impact on its ability to obtain the raw materials necessary to conduct its clinical trials. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in our most recent annual or quarterly report and in other reports we have filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

Corporate Contact:

Stephen Potter

Chief Business Officer

Applied Genetic Technologies Corporation

T: (617) 413-2754

[email protected]

Source: Applied Genetic Technologies Corporation

Greater Gift, AGTC, and 2020 On-site Partner To Honor & Celebrate Clinical Trial Participants and Provide Eye Exams to Underserved Children in the Local Community

August 24, 2021 at 8:00 AM EDT

WINSTON SALEM, N.C., Aug. 24, 2021 (GLOBE NEWSWIRE) — Greater Gift of Winston Salem, a non-profit that celebrates participation in clinical trials today announced a partnership with Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company developing genetic therapies for the treatment of rare inherited retinal diseases (IRDs), and mobile vision provider 2020 On-site, a company involved in IRD clinical research and provides complimentary eye exams and facilitates the delivery of eyeglasses to students in need.

“The pandemic has shone a bright light on the need for clinical trial participation as never before in history,” said Lilly Skok Bunch, Executive Director of Greater Gift. “Greater Gift has been celebrating and acknowledging clinical trial participants for more than 10 years; this is the first time that a program will recognize trial participants in a way that also yields benefits for students with vision challenges.”

Greater Gift’s programs celebrate clinical trial participants by making donations on behalf of those individuals to reinforce the impact of their contribution on the advancement of clinical research. Celebrations of clinical trial participants have provided an additional benefit of 130,000 vaccines and meals to children in need through prior programs.

“AGTC is thrilled to partner with Greater Gift to honor clinical trial participants and their families for their contribution to the clinical research community,” said Halley Losekamp, Director of Patient Enrollment at AGTC. “Through this partnership, our goal is to enhance awareness within the community about the value of clinical trial participation in advancing the science and understanding of gene therapies in IRDs while underscoring AGTC’s commitment to support the underserved communities that have limited access to vision care services.”

Greater Gift partnered with 2020 On-site because of the organization’s commitment to the underserved through their relationship with the Boston Public School System. This donation will allow 2020 On-site to continue the longstanding eye exam program and to double the impact of the gift, as they conduct their annual exam program and delivery of eyeglasses to public school students in need in Boston.

“We are honored to be part of this partnership with AGTC and Greater Gift. Both companies align with our mission to make eye care more accessible and to increase trust in and awareness of clinical trials. Now, because of this relationship, we will be able to support even more Boston area school children to help them succeed in the coming school year and beyond,” said Jessica Mays, Senior Director of Life Sciences for 2020 On-site, which uses their fleet of Mobile Vision Clinics to provide eye care both routinely and as part of ongoing clinical trials.

About Greater Gift (www.greatergift.org): Greater Gift is a non-profit organization, founded in 2010, with a mission to increase awareness of clinical research, especially among underrepresented communities, to improve global health. Greater Gift builds bridges with underrepresented communities to engage them in research, ensure equal representation in clinical research and to increase access to research as an option for medical care. Since its founding, Greater Gift has honored 130,000 clinical trial volunteers by making donations of vaccines and meals to children in need in their honor.

About AGTC: AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM B3 and ACHM A3). Its preclinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders. In recent years AGTC has entered into strategic partnerships with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.

About: 2020 On-site: 2020 On-site has been revolutionizing how vision care benefits are being delivered to companies and patients for 7 years. Its state-of-the-art mobile vision clinics serve over 450 companies in the greater Boston area. The company has also expanded its services to support BioPharma companies, CROs, and clinical sites to develop programs to assess their patients close to home, or even right at their front door. To learn more about how 2020 On-site is supporting clinical research, visit their website.

Contact:
David Harrison
[email protected]
410-804-1728


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AGTC Appoints Ophthalmology and Retinal Disease Specialist and Industry R&D Veteran Yehia Hashad, MD to its Board of Directors

August 18, 2021 at 8:00 AM EDT

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Aug. 18, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare retinal diseases, today announced the addition of Yehia Hashad, MD to its Board of Directors.

“We are honored to welcome Dr. Hashad to our Board of Directors,” said Sue Washer, President and CEO of AGTC. “His experience as an accomplished pharmaceutical executive and physician, combined with his expertise in ophthalmology and retinal disease, will bring AGTC invaluable perspective and guidance as we work to bring groundbreaking gene therapies to patients.”

For more than 25 years, Dr. Hashad has successfully led global development strategies from research to launch to post-launch management for innovations that address high unmet medical needs. After practicing as an ophthalmologist and retina specialist for many years, Dr. Hashad joined industry and has held several leadership roles in Novartis and Allergan, most recently as Senior Vice President & Global Head of Research and Development at Allergen Aesthetics. In this role, Dr. Hashad was responsible for all research and development activities and the overall performance of more than 40 active projects in the Allergan Aesthetics pipeline. He also served as the Vice President & Global Head of Clinical Development for Ophthalmology, Dermatology and Medical Aesthetics where he led the corporate development strategy. Prior to his current position he served as Vice President & Global Head of Ophthalmology Therapeutic Areas where he established a well-diversified ophthalmic portfolio with more than 20 active projects in different therapeutic indications with high unmet medical needs. He has served on the Boards of the Glaucoma Research Foundation and the National Alliance for Eye and Vision Research and currently sits on the Executive Board for the University of California Irvine Research Center and Institutes.

“Gene therapy is showing incredible potential to help patients with rare and debilitating retinal diseases who currently have limited treatment options,” said Dr. Hashad. “AGTC is leading the way to meet this unmet medical need and I am excited to work with the company’s leadership team and Board to bring these innovative therapies to patients.”

About AGTC

AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders. In recent years AGTC has entered into strategic partnerships with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.

Forward-Looking Statements

This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements about the potential of the Company’s late-stage development programs in X-Linked Retinitis Pigmentosa (XLRP) and Achromatopsia (ACHM). Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic, including the impact on its ability to obtain the raw materials necessary to conduct its clinical trials. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in our most recent annual or quarterly report and in other reports we have filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

IR/PR Contacts:

David Carey (IR) or Glenn Silver (PR)

Lazar FINN Partners

T: (212) 867-1768 or (646) 871-8485

[email protected] or [email protected]

Corporate Contact:

Stephen Potter

Chief Business Officer

Applied Genetic Technologies Corporation

T: (617) 413-2754

[email protected]


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Source: Applied Genetic Technologies Corporation

AGTC to Host Virtual R&D Day on July 22, 2021

July 8, 2021 at 7:00 AM EDT

Widely Recognized Experts and AGTC Leadership Will Expand on Recent ACHM Phase 1/2 Clinical Trial Data and Discuss XLRP Milestones

Webcast Scheduled from 10:00 am – 1:00 pm ET

GAINESVILLE, Fla. and CAMBRIDGE, Mass., July 08, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare retinal diseases, today announced that it will host a virtual R&D Day from 10:00 am to 1:00 pm ET on Thursday, July 22, 2021.

AGTC’s R&D Day will include a review of 12-month data from the highest dose groups in the ongoing Phase 1/2 clinical trials in X-linked retinitis pigmentosa (XLRP), which the Company announced in May, and expanded analysis of the recently reported data from the Company’s ongoing Phase 1/2 clinical trial in achromatopsia (ACHM). The event also will include a discussion on light sensitivity and achromatopsia genetics, and an overview of the Company’s manufacturing capabilities, including the 21,000 square foot current Good Manufacturing Practices (cGMP) manufacturing and quality control facility being built in Florida.

“AGTC has seen highly encouraging 12-month data from our XLRP and ACHM clinical trials over the last two months, which gives us confidence in our abilities to execute the clinical and regulatory steps necessary to advance those therapies,” said Sue Washer, President and Chief Executive Officer of AGTC. “We look forward to taking a deeper dive into this data during our R&D Day and discussing preparations for late-stage development of our ACHM and XLRP candidates.”

AGTC R&D Day Agenda:

  • Corporate Overview
    • Sue Washer, President & Chief Executive Officer
  • Pre-Clinical Pipeline
    • Adrian Timmers, Executive Director, In vivo Pharmacology and Toxicology
  • Manufacturing and New Facility Update
    • Dave Knop, PhD, Vice President of Process Development
    • Stephen Potter, Chief Business Officer
  • XLRP Indication, Phase 1/2 Data
    • Robert Sisk, MD – Associate Professor of Ophthalmology, University of Cincinnati and Cincinnati Eye Institute, XLRP Phase 1/2 clinical trial investigator
  • ACHM Indication, Phase 1/2 Data
    • Rachel Huckfeldt, MD, PhD, Assistant Professor of Ophthalmology, Harvard Medical School Department of Ophthalmology, Director, Inherited Retinal Degenerations Fellowship, Massachusetts Eye and Ear Infirmary
  • Light Sensitivity and Achromatopsia Genetics
    • Joseph Carroll, PhD – Richard O. Schultz, MD / Ruth Works Professor of Ophthalmology Professor of Ophthalmology & Visual Sciences, Biophysics, and Cell Biology, Neurobiology and Anatomy Director, Advanced Ocular Imaging Program

A live audio webcast of the presentation with accompanying slides can be accessible by visiting ir.agtc.com/events-and-presentations. Please log in approximately 10 minutes prior to the scheduled start time.

Those interested in attending can RSVP by clicking the RSVP link in the Events & Presentations page of the investor section at AGTC.com.

The archived webcast and slide presentation will be available in the Events and Presentations section of the Company’s website.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM B3 and ACHM A3). Its preclinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders. In recent years AGTC has entered into strategic partnerships with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.

IR/PR CONTACTS:
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contact:
Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC Reports 12-Month Data from its Ongoing Phase 1/2 Achromatopsia Clinical Trials Showing Biologic Activity in Patients with Mutations in the ACHM B3 Gene

June 24, 2021 at 7:00 AM EDT

– Activities to support the next stage of clinical development of ACHM B3 candidate are ongoing-

– Dosing of pediatric patients in ACHM B3 program and ACHM A3 program is expected to be completed in August 2021 –

– Conference call to review data today at 8:00am ET –

GAINESVILLE, Fla. and CAMBRIDGE, Mass., June 24, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare retinal diseases, today reported 12-month data from its ongoing achromatopsia (ACHM) Phase 1/2 clinical trials, including data from all adult patients and low-dose pediatric patients. For its ACHM B3 candidate, results demonstrate biologic activity based on improvements in visual sensitivity in the treated area measured by static perimetry and light discomfort measured by the Ocular Photosensitivity Analyzer (OPA) and are supported by anecdotal patient reports. In addition, the safety profile of the Company’s ophthalmic gene therapy platform remained favorable. Based on these data, AGTC intends to advance the ACHM B3 trial to the next stage of clinical development. The path forward for ACHM A3 will be determined after additional pediatric patient data and pre-clinical studies are available and can be evaluated.

“The results regarding responders in the ACHM B3 trial are encouraging,” said Rachel Huckfeldt, MD, PhD, Assistant Professor of Ophthalmology at Harvard Medical School and an investigator on the ongoing AGTC achromatopsia Phase 1/2 trials. “The data from the ACHM B3 trial support further clinical investigation of this candidate, and data from additional pediatric patients may support the focused development of the ACHM A3 candidate specifically in younger patients.”

“We are incredibly pleased with these data, which further support AGTC as a leader in the field of ophthalmic gene therapy. Our strong capabilities lead to differentiated products based on capsid and vector engineering, robust manufacturing, and rigorous preclinical evaluation of our product candidates in validated models of retinal diseases,” said Sue Washer, President and CEO of AGTC. “We believe that these data provide important validation for the broad potential of our AAV technology platform and build on and extend the favorable safety and efficacy profiles observed to date in the clinical trials of our candidate for X-linked retinitis pigmentosa.”

ACHM B3 12-Month Results
12-month data, which are available for 25 patients, consisting of 21 adult and 4 pediatric (<18 years of age) patients, show continued improvements on perimetry for higher dose and younger patients and include positive patient anecdotes. Improvements in retinal sensitivity as measured by static perimetry were seen in the treated eye compared with the untreated eye in four of 11 patients from the high-dose and pediatric cohorts. Improvements in light discomfort as measured by OPA, were also observed in six of these 11 patients.

Improvements in light discomfort also were observed in the untreated eye of these patients, which we believe may suggest possible cortical adaptation within the brain. There is no evidence that this observation is due to the presence of the ACHM B3 vector in the untreated eye. At the Company’s R&D Day, which is scheduled to take place on Thursday, July 22, 2021, beginning at 10:00 AM ET, an external expert is scheduled to provide a more detailed analysis of the bilateral light discomfort finding and its potential causes. This will be in addition to clinical investigators presenting a detailed patient by patient review of all the data to date.

Based on these data, AGTC plans to advance its ACHM B3 program to the next stage of clinical development. Toward this end, the Company is drafting an End-of-Phase 2 (EOP2) briefing packet to submit to the U.S. Food and Drug Administration, developing assays for pivotal ready testing, and planning production of clinical trial material.

AGTC plans to continue to collect data from younger pediatric patients that may provide additional supportive data for the development of ACHM B3, but this data will not be a gating factor for the Company’s current clinical development activities.

ACHM A3 12-Month Results
12-month data are available for 20 patients, including 16 adults and 4 pediatric patients. The results at this time point do not show consistent evidence of ACHM A3 candidate biologic activity, although we believe that the patient-reported anecdotes continue to be encouraging. In contrast with patients in the B3 trial, the majority of whom have mutations that results in the complete absence of B3 protein, the majority of A3 patients have mutations that result in the production of non-functional protein. The results observed to date in the Phase 1/2 ACHM trials suggest that the presence of these non-functional proteins may interfere with the activity of the vector expressed ACHM A3 protein. There will be an external expert at R&D Day to provide a deeper analysis.

Safety Data
Consistent with earlier data from the Company’s ACHM Phase 1/2 trials and data out to 24 months for its X-linked retinitis pigmentosa candidate, 12-month ACHM data continue to support a favorable safety profile for AGTC’s AAV technology platform. No serious adverse events (SAEs) related to the ACHM candidates were reported; one SAE was related to the surgical procedure and has resolved, two SAEs were related to the use of steroids and one has resolved, and one is improving.

Upcoming ACHM Clinical Milestones
AGTC believes it has a best-in-class ACHM B3 product candidate that may provide significant benefit to patients. The Company expects to report 3-month data from younger pediatric patients in both the ACHM B3 and ACHM A3 trials in the fourth quarter of 2021.

Conference Call and Webcast
AGTC will host a conference call and webcast to discuss the 12-month data from its ongoing Phase 1/2 ACHM clinical trials today at 8:00am ET. The live webcast will be available in the Events and Presentations section of the Investor Relations page at http://ir.agtc.com/events-and-presentations. To access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US). Please log in approximately 10 minutes prior to the scheduled start time.

The archived webcast will be available in the Events and Presentations section of the Company’s website.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM B3 and ACHM A3). Its preclinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders. In recent years AGTC has entered into strategic partnerships with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.

Forward-Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements regarding AGTC’s proposed clinical development of ACHM B3, planned pediatric surgeries for its ACHM clinical programs and the potential that future pediatric data will be supportive of future development of ACHM A3, the potential results of both its ACHM trials, its ability to enroll patients for its clinical trials, regulatory progress, potential growth and market opportunities, and the effects of competition. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic, including the impact on its ability to enroll patients. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in our most recent annual or quarterly report and in other reports we have filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

IR/PR CONTACTS: 
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: 212-867-1768 or 646-871-8485
[email protected] or [email protected]

Corporate Contact:
Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: 617-413-2754
[email protected]

Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC to Host Conference Call to Discuss Achromatopsia 12-Month Data in its Ongoing Phase 1/2 Clinical Trials

June 23, 2021 at 4:03 PM EDT

GAINESVILLE, Fla. and CAMBRIDGE, Mass., June 23, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare retinal diseases, today announced that it plans to release 12-month data for adult patients and low dose pediatric patients in its Phase 1/2 clinical Achromatopsia (ACHM) trials on Thursday, June 24, 2021. AGTC management will host a conference call and webcast with accompanying slides beginning at 8:00 AM Eastern Time on the same date to review the ACHM CNGB3 and CNGA3 data and provide an update on the trials.

The live webcast will be available in the Events and Presentations section of the Investor Relations page at http://ir.agtc.com/events-and-presentations. To access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US). The archived webcast and slide presentation will be available in the Events and Presentations section of the Company’s website.

About AGTC

AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders. In recent years AGTC has entered into strategic partnerships with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.

IR/PR CONTACTS: 
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: 212-867-1768 or 646-871-8485
[email protected] or [email protected]

Corporate Contact:
Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: 617-413-2754
[email protected] 

 

Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC to Present at American Optometric Association’s Optometry’s Meeting

June 22, 2021 at 7:00 AM EDT

-AGTC patient advocacy executive director will highlight the state of gene therapy trial recruitment for IRDs-

GAINESVILLE, Fla. and CAMBRIDGE, Mass., June 22, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare retinal diseases, today announced that Jill Dolgin, PharmD, Executive Director of Global Patient Advocacy, will address optometric graduate students on the topic of inherited retinal diseases (IRDs) at the 124th Annual American Optometric Association Congress & 51st Annual American Optometric Student Association Conference: Optometry’s Meeting®, being held June 24-26 in Denver.

Dr. Dolgin will be joined by Rachelle Lin, OD, MS, FAAO, Assistant Professor at the Southern California College of Optometry at Marshall B. Ketchum University, to present on the future management of IRDs as part of the AOA+ interactive student sessions. Dr. Lin also will also lead three continuing education courses related to gene therapy and IRDs.

“AGTC recognizes the central role that optometrists play as vision healthcare providers, initially identifying patients with symptoms of IRDs such as x-linked retinitis pigmentosa and achromatopsia and navigating them to the latest IRD clinical trials,” said Dr. Dolgin. “AGTC looks forward to the opportunity to educate future optometrists about the importance of rare disease patient registries and gene therapy clinical trials and the critical roles of low vision optometrists and people with IRDs in the development of IRD therapies.”

Dr. Lin and Dr. Dolgin’s presentations are scheduled as follows:

Rachelle Lin, OD

  • Ethical and Legal Considerations of Genetic Testing and Gene Therapy
    Thursday, June 24, 10:30-11:30 a.m. (MT)
  • Gene Therapy for Inherited Retinal Dystrophies – The Present & Future
    Thursday, June 24, 1:30 -2:30 p.m. (MT)
  • Genetic Testing for Inherited Retinal Dystrophies: Step-by-Step Workshop from Diagnosis to Gene Therapy
    Friday, June 25, 1:30-3:30 p.m. (MT)

Rachelle Lin, OD and Jill Dolgin, PharmD

  • Cutting Edge Developments Changing the Future Management of Inherited Retinal Dystrophies
    Saturday, June 26, 9-9:30 a.m., 9:40-10:10 a.m. (MT)

AGTC will also have a presence on the exhibit hall floor at booth #1316.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders. In recent years AGTC has entered into strategic partnerships with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.

IR/PR CONTACTS: 
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contact:
Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

 

Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC Reports Positive 12-Month Data from Highest Dose Groups in its Ongoing XLRP Phase 1/2 Clinica

AGTC Reports Positive 12-Month Data from Highest Dose Groups in its Ongoing XLRP Phase 1/2 Clinical Trial
May 6, 2021 at 7:00 AM EDT

-Groups 5 and 6 had a 50% response rate among patients who met the inclusion criteria for the Skyline and Vista trials-

-Best Corrected Visual Acuity (BCVA) data continue to provide supportive evidence of biological response at 12 months-

-Data from a subset of Group 4 patients available for analysis at 24 months also provide preliminary evidence of continued durable responses and continue to demonstrate a favorable safety profile-

-Conference call to review data today at 8:00 AM EDT-

GAINESVILLE, Fla. and CAMBRIDGE, Mass., May 06, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare retinal diseases, today reported additional positive data from the ongoing X-linked retinitis pigmentosa (XLRP) Phase 1/2 clinical trial, including 12-month data from Groups 5 and 6 reflecting a 50% response rate among patients who met the inclusion criteria for the Skyline and Vista trials and 24-month data from two of three Group 4 patients providing preliminary evidence of response durability. The third patient was not a responder at Month 12 and no early data are currently available for the remaining four Group 4 patients. Taken together, these data add to the body of evidence suggesting that durable improvements in visual sensitivity and visual acuity may be achieved in patients receiving AGTC’s XLRP product candidate while continuing to demonstrate a favorable safety profile.

“The rate and durability of response for improvements in retinal sensitivity are very promising, as are continued trends of increased visual acuity at Month 12 in some patients,” said Robert Sisk, MD, Associate Professor of Ophthalmology, University of Cincinnati and Cincinnati Eye Institute, and an investigator on AGTC’s XLRP Phase 1/2 clinical trial. “Gene therapy holds great promise in improving outcomes for these early and moderate staged XLRP patients, who today have no FDA-approved treatment options. I believe that the data presented today are an important advance for the field of gene therapy for retinitis pigmentosa, and I look forward to presenting the full Month 12 Phase 1/2 data at the American Academy of Ophthalmology annual meeting later this year.”

“A growing body of data supports the best-in-class potential of our XLRP therapy product candidate and we are executing a robust plan to advance this program as rapidly as possible toward commercialization,” said Sue Washer, President and CEO of AGTC. “The 50% response rate observed in Groups 5 and 6, coupled with the 12-month visual sensitivity and visual acuity data from these groups and 24-month data from three Group 4 patients, position the Phase 2/3 Vista trial and expanded Phase 1/2 Skyline trials for success. The clear differences in visual sensitivity and visual acuity between the treated and untreated eyes at 12 months in Groups 5 and 6 demonstrate a biological response to our XLRP candidate.”

Groups 5 and 6 Month 12 Data
Data at 12 months were available from seven patients in Group 5 and four patients in Group 6. One patient in Group 5 and two patients in Group 6 would not meet the inclusion criteria for the Skyline and Vista trials, resulting in a total of eight patients who were included in the responder analysis. Four of these eight patients (50%) were considered responders, all four of whom met the strict criteria of at least a 7 decibel (dB) improvement in at least 5 loci. One additional patient did not meet these criteria but had a statistically significant improvement in retinal sensitivity in the treated compared with the untreated eye at 12 months.

Consistent with previously reported 6-month data from Groups 2, 4, 5 and 6, assessment of BCVA in these groups at 12 months continue to provide supportive evidence of improved visual acuity in these patients; the difference between treated and untreated eyes is statistically significant. The Company believes that these data, together with the favorable safety profile, differentiate its XLRP candidate from competitors.

Group 4 Month 24 Data
Data from three of the seven Group 4 patients were available for analysis at Month 24, including two who were responders at Month 12 (one by the 7dB change in at least 5 loci response criteria and the other based on improved retinal sensitivity in the treated compared with the untreated eye). These two patients are still responders at Month 24 according to the same criteria; the third patient who has reached Month 24 was not a responder at Month 12 or Month 24. To the best of the Company’s knowledge, this is the first XLRP gene therapy clinical trial to demonstrate continued durability of response at this time point.

Safety Data
Data from all 28 patients across six dose groups continue to demonstrate a favorable safety profile with no dose-limiting inflammatory responses observed. This safety profile, which has shown no clinically significant inflammation not manageable with steroids, continues to be observed out to 24 months.

Upcoming XLRP Clinical Milestones
AGTC believes it has a best-in-class XLRP product candidate that may provide significant benefit to patients with XLRP. The Company expects to:

  • Present 12-month trial results from the ongoing Phase 1/2 clinical trial at the American Academy of Ophthalmology Annual Meeting in November 2021;
  • Provide Skyline trial results from the 3-month masked interim analysis in 4Q 2021;
  • Provide Skyline trial results from the 12-month data in 3Q 2022; and
  • Provide Vista trial results from the 6-month masked interim analysis in 4Q 2022.

Conference Call and Webcast
AGTC will host a conference call and webcast to discuss the 12-month data from Groups 5 and 6 and 24-month data from Group 4 from its ongoing Phase 1/2 XLRP clinical trial today at 8:00am ET. To access the call, dial 866-269-4262 (US) or 323-347-3278 (outside of the US) referencing conference ID# 9770776. A live webcast will be available in the Events and Presentations section of AGTC’s Investor Relations site at http://ir.agtc.com/events-and-presentations. Please log in approximately 10 minutes prior to the scheduled start time.

The archived webcast will be available in the Events and Presentations section of the Company’s website.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders. In recent years AGTC has entered into strategic partnerships with companies including Otonomy, a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, an innovator in the emerging field of optogenetics and retinal coding.

Forward-Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements regarding the projected timing for its planned Vista (Phase 2/3 XLRP) and Skyline (Expanded Phase 1/2) clinical trials, the timing for reporting data in both its Skyline and Vista trials. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic, including the impact on its ability to enroll patients. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in our most recent annual or quarterly report and in other reports we have filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

IR/PR CONTACTS: 
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contacts:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC Announces New Initiatives To Enhance The Patient Journey In Its XLRP Trials

March 25, 2021 at 7:00 AM EDT

-AGTC’s deep commitment to the patient voice drives solutions to support patient access to information and to improve the experience of patients participating in clinical trials-

GAINESVILLE, Fla., and CAMBRIDGE, Mass., March 25, 2021 (GLOBE NEWSWIRE) — Applied Genetics Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, which include X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM), announces two important initiatives to help patients with potential interest in its XLRP clinical trials. The first is an enhanced website to improve the experience for patients with low vision, and the second is a dedicated patient information call center to provide support to patients with potential interest in AGTC’s clinical trials for XLRP.

AGTC has long recognized the importance of patients’ views in understanding and serving their needs and launched its Patient Advisory Council (PAC) in August 2020 to incorporate patient and caregiver voices into its culture and clinical and pre-clinical programs. Along with guidance from the global advocacy community on low vision, which included the Foundation Fighting Blindness, Fighting Blindness Canada and Retina International, AGTC has improved its website to make it more accessible and welcoming. These enhancements to the website (www.agtc.com), which are intended to make information easier to find for patients with low vision, include different font sizes and color options to make the pages easier to read and an option for the page to be read aloud. Because AGTC recognizes the increasing use of mobile devices by patients, these enhanced features are designed for mobile compatibility.

In addition to upgrading the AGTC website, AGTC also has developed a website that is dedicated to XLRP information and can be accessed at www.scenictrials.com.

“For those with low vision there is often a feeling of the loss of independence and control, yet they are committed to learning more about their condition, seeking treatment and researching relevant clinical trials,” said Benjamin Yerxa, PhD, Chief Executive Officer of Foundation Fighting Blindness. “I am grateful that AGTC sought feedback not only from their Patient Advisory Council, but also from international advocates, and then incorporated that feedback into their website redesign. This is a major step to help patients gain control of their treatment research.”

To assist those who want to learn more about AGTC’s XLRP clinical trials, AGTC partnered with Serva Health, a nurse-staffed patient engagement center, to assist them in navigating the clinical trial experience. This partnership provides access to nurses who can answer questions to help potential participants better understand the XLRP clinical trials. If a potential patient is interested and qualified for AGTC’s trial, the call center will provide guidance and logistical support to navigate the process.

“Building relationships with the global advocacy community and listening to families and caregivers provides us with valuable insights into patients’ unique unmet medical needs and the challenges they face when interested in learning more about their indication and research into potential treatment options,” said Sue Washer, President and CEO of AGTC. “This dialogue equips us with the information we need to accomplish our goals of being transparent in the clinical trial design process, conducting research, providing education and support to patients, and delivering high-value solutions throughout the patient journey.”

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders. In recent years AGTC has entered into strategic partnerships with companies including Otonomy, a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, an innovator in the emerging field of optogenetics and retinal coding.

IR/PR CONTACTS: 
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contacts:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

 

Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC Announces Financial Results and Business Update for the Quarter Ended December 31, 2020

February 11, 2021 at 7:00 AM ESTPDF Version

– Company on track to provide multiple data readouts for XLRP and ACHM clinical programs in 2021 and 2022 –

– Net proceeds of approximately $69.2 million from a recent public offering provide cash runway into calendar year 2023 –

– Company to host conference call and webcast today at 8:00 a.m. ET –

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Feb. 11, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced financial results for the quarter ended December 31, 2020.

“We believe that the data generated to date in our XLRP trials continue to give us a leading position in this indication and we are eager to move our Phase 1/2 expansion (Skyline) and Phase 2/3 (Vista) trials forward,” said Sue Washer, President and CEO of AGTC. “The latest data from our ongoing Phase 1/2 trials in patients with achromatopsia provide the first reported quantitative evidence of improvements in visual sensitivity and provide a path forward to collect additional data and to fully realize the potential of this treatment.   Continued progress in our clinical and earlier-stage development efforts is expected to create multiple data milestones, including four important data releases for XLRP and two for achromatopsia over the next two years, while bringing us closer to our goal of developing best-in-class therapies that provide meaningful benefit to patients.”

X-linked Retinitis Pigmentosa (XLRP)
In November 2020, the Company reported additional positive data from its Phase 1/2 clinical trial in patients with XLRP that indicated durable improvements observed in visual sensitivity and supportive trends in visual acuity over a wide dose range with a favorable safety profile out to month 12 in two of the dose groups. The Company believes it has a best-in-class product candidate that may provide significant benefit to patients with XLRP. The Company expects to:

Achromatopsia (ACHM)
In January 2021, the Company announced the first reported quantitative evidence of improvement in visual sensitivity, as measured by full field static perimetry, supported in some patients by other endpoints. Seven of the 16 patients in the three highest dose groups in the ACHMB3 trial showed these improvements in visual sensitivity in the treated area. No consistent results were seen in the other dose groups. In a subset of these patients with evaluable multi-focal electroretinograms (mfERG), improvements in electrical signaling were measurable in the same treated area.

For ACHMA3, of the 16 patients in the four highest dose groups, three patients showed improvements in visual sensitivity in the treated area, as measured by static perimetry. No consistent results were seen in other dose groups. None of these three patients with improvements in visual sensitivity had evaluable mfERGs.

AGTC currently plans to focus on completing enrollment of pediatric patients in the two highest dose groups in its ACHMB3 and ACHMA3 trials and to follow all patients through 12 months. The Company expects to:

Financial Results for the Three and Six Months Ended December 31, 2020

Revenue: There was no revenue for the three and six months ended December 31, 2020, as compared to $2.5 million in each of the comparable 2019 periods. Revenue during the three and six months ended December 31, 2019 was primarily due to $2.2 million of non-cash collaboration revenue in connection with in-kind contributions made to Bionic Sight, LLC pursuant to a collaborative agreement.

R&D Expenses: Research and development expenses for the three and six months ended December 31, 2020 were $11.8 million and $23.4 million, respectively, compared to $8.4 million and $17.0 million, respectively, during the comparable 2019 periods. The increase of $6.4 million during the 2020 six-month period was primarily due to increased external XLRP spending for planned manufacturing, clinical site preparation and other activities related to the Skyline and Vista trials, partially offset by decreased external ACHM spending.

G&A Expenses:   General and administrative expenses for the three and six months ended December 31, 2020 were $3.3 million and $6.7 million, respectively, compared to $3.0 million and $6.4 million, respectively, during the comparable 2019 periods. The increase of $0.3 million during the 2020 six-month period was primarily due to higher fees from outside legal counsel, partially offset by lower employee-related expenses and share-based compensation expense.

Investment Income, net: Investment income, net for the three and six months ended December 31, 2020 declined by $0.3 million and $0.7 million, respectively, when compared to the comparable 2019 periods, which was primarily due to lower interest rates in the marketplace.

Interest Expense: Interest expense for the three and six months ended December 31, 2020 increased by $0.3 million and $0.7 million, respectively, when compared to the comparable 2019 periods due to the loan agreement that the Company entered into on June 30, 2020.

Net Loss: The Company’s net loss for the three and six months ended December 31, 2020 was $15.5 million and $30.8 million, respectively, compared to $8.6 million and $20.2 million, respectively, during the comparable 2019 periods.

Financial Guidance: As of December 31, 2020, the Company’s cash, cash equivalents and investments totaled $53.1 million. The Company believes that these funds, along with net proceeds of approximately $69.2 million from an underwritten public offering that closed in February 2021, will be sufficient to allow the Company to generate data from its ongoing and planned clinical programs and fund currently planned research and discovery programs into calendar year 2023.

Conference Call and Webcast
AGTC will host a conference call and webcast to discuss financial results for the fiscal quarter ended December 31, 2020 today at 8:00 a.m. ET. To access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US). A live webcast will be available in the Events and Presentations section of AGTC’s Investor Relations site at http://ir.agtc.com/events-and-presentations. Please log in approximately 10 minutes prior to the scheduled start time.

The archived webcast will be available in the Events and Presentations section of the Company’s website.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the Company’s industry-leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

About XLRP
XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th or 5th decade of life. AGTC was granted U.S. Food and Drug Administration (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

About ACHM
ACHM is an inherited retinal disease, which is present from birth and is characterized by the lack of cone photoreceptor function. The condition results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by ACHM, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind.

Forward-Looking Statements
This press release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements regarding the projected timing for its planned Vista (Phase 2/3 XLRP) and Skyline (Expanded Phase 1/2 XLRP) clinical trials, the timing for reporting data in both its Skyline and Vista trials and the potential of its ACHM clinical programs. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic, including the impact on AGTC’s ability to enroll patients. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on the Company’s business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in AGTC’s most recent annual and subsequently filed quarterly reports filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

IR/PR CONTACTS: 
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contacts:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC Executives Awarded First Place in the BioProcess International Reader’s Choice Awards, Cell & Gene Therapies Category

February 4, 2021 at 12:02 PM ESTPDF Version

Article reflects Company’s leadership and innovation in scalable, reproducible manufacture of adeno-associated virus (AAV)-based gene therapies

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Feb. 04, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company focused on developing adeno-associated virus (AAV) based gene therapies for the treatment of rare inherited diseases, announced that Sue Washer, President & Chief Executive Officer and Dave Knop, Vice President of Process Development, have been awarded first place in the BioProcess International (BPI) magazine inaugural Reader’s Choice Awards program, cell and gene therapies category, for their article, “Viral-Vectored Gene Therapies: Harnessing Their Potential Through Scalable, Reproducible Manufacturing Processes.”

“High-productivity approaches to AAV manufacturing processes, like AGTC’s HSV-helper based platform, will be crucial if we are to address the unmet clinical need growing across a variety of indications,” said AGTC President and CEO, Sue Washer. “There is no question that investing in the manufacturing process is imperative and our early commitment in this area has put AGTC in a strong position with respect to the purity and quality needed for late stage development and commercialization.”

Concentrating on articles published from September 2019 through June 2020, and using rankings based on views, engagement, and download rates, BioProcess International identified the four most popular articles within each of its six pillars of bioprocessing coverage. The AGTC authors’ article received the highest number of votes from BPI readers, who ranked the nominees in terms of their innovativeness, presentability and applicability.

The eBook featuring the first-place article by Washer and Knop, as well as summarized versions of the second- and third-place articles, are available by visiting:
https://bioprocessintl.com/wp-content/uploads/2020/11/18-11-eBook-RCA-CellGeneTherapies.pdf.                        

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Company’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its preclinical programs build on the Company’s industry-leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

IR/PR CONTACTS: 
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC to Host Second Quarter Financial Results Conference Call and Webcast on February 11, 2021

February 4, 2021 at 7:00 AM ESTPDF Version

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Feb. 04, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced that it will report financial results for the fiscal quarter ended December 31, 2020 before the market opens on Thursday, February 11, 2021. AGTC management will host a conference call beginning at 8:00 AM Eastern Time on the same date to review results and provide a corporate update.

To access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US). A live webcast will be available in the Events and Presentations section of the Investor Relations page at http://ir.agtc.com/events-and-presentations. Please log in approximately 10 minutes prior to the scheduled start time. The archived webcast will be available in the Events and Presentations section of the company’s website.

About AGTC

AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its preclinical programs build on the Company’s industry-leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

IR/PR CONTACTS: 
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC Executives Awarded First Place in the BioProcess International Reader’s Choice Awards, Cell & Gene Therapies Category

February 4, 2021 at 12:02 PM ESTPDF Version

Article reflects Company’s leadership and innovation in scalable, reproducible manufacture of adeno-associated virus (AAV)-based gene therapies

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Feb. 04, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company focused on developing adeno-associated virus (AAV) based gene therapies for the treatment of rare inherited diseases, announced that Sue Washer, President & Chief Executive Officer and Dave Knop, Vice President of Process Development, have been awarded first place in the BioProcess International (BPI) magazine inaugural Reader’s Choice Awards program, cell and gene therapies category, for their article, “Viral-Vectored Gene Therapies: Harnessing Their Potential Through Scalable, Reproducible Manufacturing Processes.”

“High-productivity approaches to AAV manufacturing processes, like AGTC’s HSV-helper based platform, will be crucial if we are to address the unmet clinical need growing across a variety of indications,” said AGTC President and CEO, Sue Washer. “There is no question that investing in the manufacturing process is imperative and our early commitment in this area has put AGTC in a strong position with respect to the purity and quality needed for late stage development and commercialization.”

Concentrating on articles published from September 2019 through June 2020, and using rankings based on views, engagement, and download rates, BioProcess International identified the four most popular articles within each of its six pillars of bioprocessing coverage. The AGTC authors’ article received the highest number of votes from BPI readers, who ranked the nominees in terms of their innovativeness, presentability and applicability.

The eBook featuring the first-place article by Washer and Knop, as well as summarized versions of the second- and third-place articles, are available by visiting:
https://bioprocessintl.com/wp-content/uploads/2020/11/18-11-eBook-RCA-CellGeneTherapies.pdf.                        

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Company’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its preclinical programs build on the Company’s industry-leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

IR/PR CONTACTS: 
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC Announces Proposed Public Offering of Common Stock and Warrants

GAINESVILLE, Fla., and CAMBRIDGE, Mass., Jan. 27, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a
biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases,
today announced that it is offering to sell shares of its common stock and pre-funded warrants, together with accompanying warrants to purchase
shares of its common stock in an underwritten public offering. All of the securities in the proposed offering are being sold by AGTC. The offering is
subject to market conditions and there can be no assurance as to whether or when the offering may be completed or as to the actual size or terms of
the offering.
Stifel, Wells Fargo Securities and MTS Health Partners are acting as joint book-running managers of the offering.
AGTC intends to use the net proceeds from this offering, together with other available funds, to fund its ongoing Skyline and Vista clinical trials in its
X-linked retinitis pigmentosa (XLRP) program and its ongoing Phase 1/2 clinical trials in its Achromatopsia (ACHM) program, and for working capital
and other general corporate purposes.
A registration statement relating to the shares of common stock, the pre-funded warrants and accompanying warrants being offered has been filed
with, and declared effective by, the Securities and Exchange Commission (the “SEC”). A preliminary prospectus supplement relating to the offering
has also been filed with the SEC and is available on the SEC’s website at http://www.sec.gov. Copies of the preliminary prospectus supplement and
accompanying prospectus may be obtained from Stifel, Nicolaus & Company, Incorporated, Attention: Syndicate, One Montgomery Street, Suite 3700,
San Francisco, CA 94104, by telephone at (415) 364-2720 or by email at [email protected]; Wells Fargo Securities, LLC, Attention: Equity
Syndicate Department, 500 West 33rd Street, New York, NY 10001, by telephone at (800) 326-5897 or by email at [email protected];
or MTS Securities, LLC, Attention: Syndicate Department, 623 Fifth Avenue, 14th Floor, New York, NY 10022, by telephone at (212) 887-2100 or by
email at [email protected] This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any
sale of, these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or
qualification under the securities laws of any such state or jurisdiction.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including
statements regarding AGTC’s proposed offering of securities, that involve a number of risks and uncertainties. Statements that are not historical facts
are based on AGTC’s current expectations, beliefs and assumptions regarding the market for its common stock. There can be no assurance regarding
the completion, timing or size of the proposed offering. Important factors that could cause actual outcomes to differ materially from those indicated by
these forward-looking statements include risks and uncertainties related to market conditions, the satisfaction of customary closing conditions related
to the proposed public offering and others described in AGTC’s most recent Annual Report on Form 10-K for the year ended June 30, 2020, Quarterly
Report on Form 10-Q for the quarter ended September 30, 2020 and the preliminary prospectus supplement. AGTC cautions investors not to place
undue reliance on the forward-looking statements contained in this release. These statements speak only as of the date of this release, and AGTC
undertakes no obligations to update or revise these statements, except as may be required by law.
About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central
nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to
develop customized therapies that address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform
to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and
achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its preclinical programs build on the Company’s industry-leading AAV manufacturing technology
and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology
and CNS disorders.
IR/PR CONTACTS:
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]
Corporate Contacts:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]
Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]
Source: Applied Genetic Technologies Corporation

AGTC Announces First Reported Improvements in Visual Sensitivity for Achromatopsia (ACHM) patients from its Ongoing Clinical Trials

January 27, 2021 at 4:19 PM ESTPDF Version

-Preliminary results suggest sustained improvements to 12-months in visual sensitivity, as measured by full field static perimetry, supported in some patients by other endpoints-
-Company plans longer term follow-up, dosing of younger pediatric patients, and addition of new brain imaging and color brightness tests-

GAINESVILLE, Fla., and CAMBRIDGE, Mass., Jan. 27, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, is today providing an update on its ongoing clinical trials in patients with achromatopsia (ACHM). The Company believes the additional data reported today provides the first reported quantitative evidence of improvements in visual sensitivity, supports the positive patient reported outcomes and provides a path forward to collect additional data to fully realize the potential of this treatment.

“Encouraging data from a careful patient-by-patient analysis of both the ACHMB3 and ACHMA3 trials provide additional support of our ACHM gene therapies,” said Sue Washer, President and CEO of AGTC. “We currently plan to focus on completing enrollment of pediatric patients in the two highest dose groups of both trials. In addition, we have amended the study protocol for these trials to allow enrollment of patients as young as 4 years of age and to include both functional magnetic resonance imaging of the brain and improved color brightness tests. We are hopeful that these changes, combined with longer follow-up times, will add to the developing body of evidence and supportive anecdotal patient-reported outcomes.”

In January 2020, AGTC provided 3-month ACHM data indicating evidence of biologic activity in the dose escalation portions in both our ACHMB3 and ACHMA3 trials, based on improvements in light discomfort.

In November 2020, the Company provided additional 12- and 6-month data across dose groups in both trials, including one group of pediatric patients. While some patients showed improvements in at least one measure of visual function, no consistent sustained improvements were observed within the dose groups analyzed on a groupwise basis. Anecdotal statements, however, and assessments from patient-reported outcome surveys continued to provide us with confidence that patients were subjectively experiencing improved vision in their treated eye.

AGTC is now reporting preliminary results based on a patient-by-patient analysis of data from both ACHMB3 and ACHMA3 trials. For ACHMB3, this consists of 12month data from 15 patients, 9month data from five patients, 6month data from three patients and 3month data from three patients, for a total of 26 patients across all dose groups. These results reflect a further analysis of the data discussed in November 2020, together with new data that became available in January 2021. Seven of the 16 patients in the three highest dose groups in the ACHMB3 trial showed improvements in visual sensitivity, in the treated area, as measured by static perimetry. No consistent results were seen in the other dose groups. In a subset of these patients with evaluable multi-focal electroretinograms, improvements in electrical signaling were measurable in the same treated area.

For ACHMA3, the new data analysis consists of 12month data from 10 patients, 9month data from four patients, 6month data from one patient and 2 or 3-month data from three patients, for a total of 18 patients across all dose groups. One additional patient did not have evaluable data. In the 16 patients in the four highest dose groups, three patients showed improvements in visual sensitivity, in the treated area, as measured by static perimetry. No consistent results were seen in other dose groups. None of these three patients with improvements in visual sensitivity had evaluable ERGs.

AGTC currently plans to focus on completion of enrollment of pediatric patients in the two highest dose groups in our ACHMB3 and ACHMA3 trials and following all patients through 12 months. The Company has amended the study protocol for these trials to allow enrollment of patients as young as 4 years of age and to include both functional magnetic resonance imaging (fMRI) and improved color brightness tests. AGTC will also work with sites to obtain the best quality multi-focal ERG data possible. The Company is hopeful that these changes, combined with longer follow-up times, will add to the developing body of evidence and supportive anecdotal patient-reported outcomes. The Company expects to report 12month data from the adult patients in both trials in second quarter of calendar year 2021, and preliminary 3-month data from the pediatric patients in both trials is anticipated in the fourth quarter of calendar year 2021, dependent on any effects of the COVID pandemic.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Company’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its preclinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

About ACHM
ACHM (achromatopsia) is an inherited retinal disease, which is present from birth and is characterized by the lack of cone photoreceptor function. The condition results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by ACHM, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind.

Forward-Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements regarding the projected timing for enrollment of patients in and data from, and the potential of, its ACHM clinical programs. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic, including the impact on its ability to enroll patients. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on the Company’s business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in AGTC’s most recent annual and subsequently filed quarterly reports filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

IR/PR CONTACTS: 
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contacts:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Primary Logo

Source: Applied Genetic Technologies Corporation

AGTC Announces Financial Results and Business Update for the Quarter Ended September 30, 2020

November 16, 2020 at 4:05 PM ESTPDF Version

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Nov. 16, 2020 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced financial results for the quarter ended September 30, 2020. The Company is also providing an update on its ongoing clinical trials in patients with X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM).

“The updated XLRP data that we reported last week provide evidence that our XLRP product candidate provides durable improvements in visual sensitivity and visual acuity over a wide dose range and has a favorable safety profile,” said Sue Washer, President and CEO of AGTC. “Data publicly reported by our competitors this weekend at the American Academy of Ophthalmology (AAO) Annual Meeting further increase our confidence that we have the industry-leading XLRP program. We are on track to initiate enrollment in the planned Phase 1/2 expansion trial (Skyline) in 4Q 2020 and the Phase 2/3 trial (Vista) in 1Q 2021. Additionally, new data from our ongoing ACHM trials have provided important insights for the potential development of our ACHM product candidates. We are pleased that we met our goal of reporting out data from all three ongoing clinical trials in 4Q 2020 and are now focused on advancing the Skyline and Vista trials for XLRP as rapidly as possible.”

XLRP
On November 11, 2020, the Company reported additional positive data from its Phase 1/2 clinical trial in patients with XLRP that indicated durable improvements observed in visual sensitivity and visual acuity over a wide dose range with a favorable safety profile out to month 12 in two of the dose groups. Based on comparison to publicly released data reported by competitors this weekend at the AAO Annual Meeting, the Company believes it has a best-in-class product that may provide significant benefit to patients with XLRP.

ACHM
Today the Company is reporting 12-month ACHM data in the original three dose groups (low, medium and high), as well as 6- to 9-month data at two higher dose groups (higher and highest groups in the January 2020 data release), and data for 6 pediatric subjects (three in each study; 12-17 years old) at the first of three planned dose levels. While some patients showed improvements in at least one measure of visual function, no consistent sustained improvements were observed based on current assessments within the dose groups tested. However, anecdotal statements and assessments from patient-reported outcome surveys continue to provide the Company with confidence that patients are subjectively experiencing improved vision. Based on the characteristics of ACHM, the Company continues to believe that longer treatment durations and/or focusing on younger patients may be necessary to fully realize the potential of this treatment. To this end, the Company intends to complete the planned enrollment of pediatric patients in the two highest dose groups of the ongoing ACHM clinical trials and has amended the study protocol to allow enrollment of patients as young as four years of age and to include additional assessments such as functional MRI (fMRI) and improved color brightness tests.

Financial Results for the Three Months Ended September 30, 2020

R&D Expenses: Research and development expenses were $11.6 million for the quarter ended September 30, 2020 compared to $8.6 million during the comparable period in the prior fiscal year. The increase of $3.0 million was primarily due to increased external XLRP spending (primarily related to Skyline and Vista activities) and increased external spending related to ACHM (primarily due to patient enrollment and deployment of the Company’s mobile vision center). These expenses were partially offset by a reduction in employee-related costs and decreased external research and discovery spending on other programs.

G&A Expenses:   General and administrative expenses were $3.4 million for the quarter ended September 30, 2020 compared to $3.3 million during the comparable period in the prior fiscal year. The increase of $0.1 million was primarily due to higher fees from outside legal counsel in the 2020 period, partially offset by lower employee-related expenses and share-based compensation expense.

Interest Expense: Interest expense increased by $0.3 million during the quarter ended September 30, 2020 when compared to the comparable period in the prior fiscal year due to the loan agreement that the Company entered into on June 30, 2020.

Net LossThe Company’s net loss was $15.4 million and $11.6 million for the quarters ended September 30, 2020 and 2019, respectively.

Financial Guidance:   As of September 30, 2020, the Company’s cash, cash equivalents and investments totaled $66.6 million. We believe that these funds will be sufficient to allow the Company to generate data from its ongoing clinical programs, initiate the Skyline and Vista studies and fund currently planned research and discovery programs into the fourth quarter of calendar year 2021.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Company’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its preclinical programs build on the Company’s industry-leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

About XLRP
XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th or 5th decade of life. AGTC was granted U.S. Food and Drug Administration (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

About ACHM
ACHM is an inherited retinal disease, which is present from birth and is characterized by the lack of cone photoreceptor function. The condition results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by ACHM, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind.

Forward-Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements regarding the projected timing for its planned Vista (Phase 2/3 XLRP) and Skyline (Expanded Phase 1/2 XLRP) clinical trials, the timing for reporting data in both its Skyline and Vista trials and the potential of its ACHM clinical programs. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic, including the impact on its ability to enroll patients. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on the Company’s business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in AGTC’s most recent annual and subsequently filed quarterly reports filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

IR/PR CONTACTS:
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contacts:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Applied Genetic Technologies Logo

Source: Applied Genetic Technologies Corporation

AGTC Reports Additional Positive Data from its Phase 1/2 Clinical Trial in Patients with X-Linked Retinitis Pigmentosa

November 11, 2020 at 7:00 AM ESTPDF Version

– Durable improvements observed in visual sensitivity and visual acuity over a wide dose-range with a favorable safety profile –

– Recent FDA interactions clarify clinically meaningful improvement on microperimetry and no further comments or questions regarding the company’s pre-clinical or CMC plans –

– Company plans to initiate enrollment in the planned Phase 1/2 expansion trial (Skyline) in 4Q 2020 and the Phase 2/3 trial (Vista) in 1Q 2021 –

– Company to host management update and webcast with slides today at 8:00am ET –

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Nov. 11, 2020 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today reported positive data from its ongoing Phase 1/2 clinical program in patients with X-linked retinitis pigmentosa (XLRP). Visual sensitivity, visual acuity and safety data were reported for 12-month timepoints for Groups 2 and 4, and 6-month time points for Groups 5 and 6. Eight of the 11 patients in Groups 5 and 6 would meet the eligibility criteria for AGTC’s future XLRP trials, and five of these eight (62%), met the definition for response based on an improvement of at least 7 decibels in at least 5 loci. The Company plans to initiate its Skyline trial by enrolling approximately 12 additional patients total in Group 2 (1.2E+11 vg/mL) and Group 5 (1.1E+12 vg/mL). In addition, the Company remains on track to initiate enrollment in its planned Vista trial in 1Q 2021.

“The high proportion of responders from multiple dose groups with sustained durability of improved visual function over 12 months is compelling evidence of biological activity for this XLRP gene therapy,” said Dr. Paul Yang, Assistant Professor of Ophthalmic Genetics and Immunology at Casey Eye Institute (Oregon Health and Science University). “With further investigation, there is a high likelihood that this gene therapy candidate could become a meaningful treatment for patients with XLRP.”

XLRP Phase 1/2 6-Month and 12-Month Data
Data from all 28 patients across six dose groups continue to demonstrate a favorable safety profile with no dose-limiting inflammatory responses observed.

At the 12-month time-point for the nine centrally dosed patients in Groups 2 and 4:

At the 6-month time-point for the 11 centrally dosed patients in Groups 5 and 6:

Further a combined analysis of visual sensitivity data from all 19 evaluable centrally dosed patients shows that 10 of 15 evaluable patients in Groups 2, 4, 5 and 6 that meet inclusion criteria for the planned Vista Phase 2/3 trial show robust and durable signs of improvements in visual sensitivity through month 6 for Groups 5 and 6 and month 12 for Groups 2 and 4. The Company is basing improvement of visual sensitivity on multiple measures including on a change from baseline in visual sensitivity of at least 7 decibels in at least 5 loci or a statistically meaningful improvement in sensitivity improvement profile between the treated and untreated eyes.

“These updated data provide important evidence that our XLRP product candidate provides durable improvements in multiple endpoints that are also meaningful to patients,” said Sue Washer, President and CEO of AGTC. “We are especially pleased to see that 62% of patients with appropriate baseline characteristics in Groups 5 and 6 show biologic activity of our product across multiple measures of visual sensitivity and that there are encouraging trends in visual acuity. This result and the continued favorable safety profile further increase our confidence in the potential of our XLRP gene therapy to become the industry-leading treatment for this disease, for which there are currently no therapies.”

Planned XLRP Phase 2/3 Vista Trial
The proposed design of the XLRP Phase 2/3 or Vista trial is currently expected to include approximately 60 patients randomized across three arms: a low-dose group (the 1.2E+11 vg/mL, Group 2 dose from the ongoing Phase 1/2 trial), a high-dose group (the 1.1E+12 vg/mL, Group 5 dose from the ongoing Phase 1/2 trial), and an untreated control group. The primary endpoint will be based on visual sensitivity defined having at least a 7 decibel improvement in visual sensitivity in at least 5 pre-specified loci at month 12. This primary endpoint was informed by recent comments that the Company received from the Food and Drug Administration (FDA) on what evidence would help support a showing of a clinically meaningful improvement on microperimetry at the Group 2 and Group 5 doses. Importantly, the Company plans to use this endpoint as one of several measures of visual sensitivity that have the potential to support a clinically meaningful benefit. The Company plans to compare the responder rates in each active arm to responder rates in the control arm of the Vista trial. The Company plans to submit a 6-month interim analysis of the data from the Vista trial, together with complete 12-month data from the Skyline trial to the FDA to obtain feedback on the Company’s development plan to support approval. Based on any FDA feedback, the Company may modify the final trial design, enrollment numbers, and/or statistical analysis plan. The Company also plans to discuss with the FDA dose selection for the treatment of the contralateral eye. The Company expects to initiate enrollment in the Vista trial in 1Q 2021 and to provide results from the 6-month interim analysis in 3Q 2022.

The Company remains on-track to have clinical trial material produced in time for the initiation of the Vista trial through its advanced manufacturing process that provides improved yields, purity and potency.

Conference Call and Webcast Today at 8:00 am ET
AGTC will host a conference call and webcast with accompanying slides to discuss the 12-month and 6-month data from its ongoing Phase 1/2 XLRP clinical trial today at 8:00am ET. To access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US). A live webcast will be available in the Events and Presentations section of AGTC’s Investor Relations site at http://ir.agtc.com/events-and-presentations. Please log in approximately 10 minutes prior to the scheduled start time.

The archived webcast will be available in the Events and Presentations section of the Company’s website.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Company’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its pre-clinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

About X-linked Retinitis Pigmentosa (XLRP)
XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th or 5th decade of life. AGTC was granted U.S. Food and Drug Administration (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

ForwardLooking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements regarding the projected timing for its planned Vista (Phase 2/3 XLRP) and Skyline (Expanded Phase 1/2) clinical trials, the timing for reporting data in both its Skyline and Vista trials and its ACHM clinical programs. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic, including the impact on its ability to enroll patients. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in our most recent annual or quarterly report and in other reports we have filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

IR/PR CONTACTS:
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

AGTC to Present at Upcoming Virtual Conferences

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Oct. 08, 2020 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company focused on developing adeno-associated virus (AAV) based gene therapies for the treatment of rare inherited diseases, today announced that management will participate in the following virtual conferences:

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Company’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its pre-clinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

IR/PR CONTACTS:
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

AGTC Joins the My Retina Tracker® Program as a New Scientific Collaborator

October 7, 2020 at 7:00 AM EDTPDF Version

GAINESVILLE, Fla., and CAMBRIDGE, Mass., Oct. 07, 2020 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (AGTC), Blueprint Genetics, and Foundation Fighting Blindness, announce today that AGTC, a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, will join the My Retina Tracker® Program as a new scientific collaborator to enhance development of its investigational gene therapies for inherited retinal diseases (IRDs). The My Retina Tracker Program, a collaboration between Blueprint Genetics, Foundation Fighting Blindness and InformedDNA, is an open access, no-cost program that offers individuals with IRDs easy access to high-quality genetic diagnostics and genetic counseling. This program has become one of the largest volume genetic testing initiatives for IRDs globally.   

As a scientific collaborator, AGTC will have access to specific deidentified genetic data and expert healthcare providers to help AGTC rapidly identify potential candidates eligible for their clinical trials. AGTC’s initial focus in the collaboration will be X-linked retinitis pigmentosa (XLRP), specifically RPGR-associated disease. Disease-causing variants in RPGR are the third most common cause of IRDs. AGTC has initiated an expansion of its Phase 1/2 gene therapy clinical trial and is planning to initiate a Phase 2/3 clinical trial in early 2021 in males with XLRP with a pathogenic variant in the RPGR gene.

“We are delighted to be among the scientific collaborators in the My Retina Tracker Program who share our mission of improving the vision of patients with IRDs. Patient registries are a rich source of information for sponsors of clinical trials, particularly for rare diseases, where patient identification can be more challenging,” said Sue Washer, President and CEO of AGTC. “The critical genetic data from the My Retina Tracker Program will benefit current and future patients as it is an important step in being able to participate in clinical trials that may lead to effective treatments for these diseases. We thank all the registrant participants for their contribution to a greater understanding of these conditions.”

Patients taking part in the My Retina Tracker Program are offered Blueprint Genetics’ 322-gene panel that includes comprehensive coverage of major IRD genes, noncoding variants, copy number variants and mitochondrial genome analysis. One of the hallmarks of the panel is the high performance of RPGR testing, including the difficult-to-sequence ORF15 region which harbors approximately 80% of disease-causing RPGR variants. Blueprint Genetics has done extensive work to develop high-quality testing capabilities for this gene using a unique NGS-based approach in order to maximize XLRP diagnostic yield. The My Retina Tracker Program has also received excellent feedback regarding the comprehensive genetic counseling provided by InformedDNA. All patients taking part in the program are provided no-cost, post-test genetic counseling by expert genetic counselors from InformedDNA.

“With the very strong focus on genespecific and gene variantspecific therapies dominating the late preclinical and clinical trials pipelines, knowing the genetic basis of disease is an important component of any inherited retinal disease diagnosis,” said Dr. Brian Mansfield, executive vice president research and interim chief scientific officer of the Foundation Fighting Blindness. “The Foundation is delighted to partner with industry partners, like AGTC, and other non-profit foundations, to be able to make genetic testing accessible to people with an inherited retinal disease—at no cost to patients. By partnering with industry sponsors to proactively genotype people with these rare diseases, our partners not only provide a valuable service to affected people but also significantly reduce their overall expense in finding potential clinical trial participants, compared to solo screening programs.”

“Over the past few years, we have invested in improving the quality and performance of genetic analysis of this challenging RPGR gene. It has been a privilege to witness the significance of these efforts to patients and is even more rewarding now as we have opportunities to collaborate with biopharmaceutical companies like AGTC that are developing product candidates with the potential to change the lives of these patients with RPGR-targeted gene therapy,” said Blueprint Genetics Executive Medical Director, Dr. Tero-Pekka Alastalo.

About the My Retina Tracker® Program

The My Retina Tracker® Program offers open access, no-cost genetic testing and genetic counseling for individuals living in the United States with a clinical diagnosis of an inherited retinal disease. The program provides people with these diseases access to the highest quality genetic testing and genetic counseling. InformedDNA provides genetic counseling by certified genetic counselors with inherited retinal disease expertise. Although not required for participation, this program also offers an easy opportunity to join the Foundation Fighting Blindness My Retina Tracker Registry which gives individuals the opportunity to share their deidentified information and contribute to focus groups, patient journey analyses, research studies, and, when available, invitations to apply for enrollment in relevant natural history studies and clinical trials.

For more information on the My Retina Tracker Program, please visit https://blueprintgenetics.com/my-retina-tracker-program/

For more information on My Retina Tracker Registry, please visit: FightingBlindness.org/my-retina-tracker-registry

About AGTC

AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Company’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with inherited retinal diseases. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its pre-clinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in larger ophthalmology indications, optogenetics, otology and CNS disorders. www.agtc.com

About the Foundation Fighting Blindness
Established in 1971, the Foundation Fighting Blindness is the world’s leading private funding source for retinal degenerative disease research. The Foundation has raised more than $800 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the entire spectrum of retinal degenerative diseases including: retinitis pigmentosa, age-related macular degeneration, Usher syndrome, and Stargardt’s disease. Visit FightingBlindness.org for more information.

About Blueprint Genetics
Blueprint Genetics, a Quest Diagnostics company, is a leading specialty genetics and bioinformatics company focused on providing genetic testing for inherited diseases. The company is based in Helsinki with a US hub in Seattle, and a customer base spanning over 70 countries. www.blueprintgenetics.com

Contact information:

AGTC
Jill Dolgin, PharmD., Executive Director, Patient Advocacy, [email protected]

Blueprint Genetics
Tero-Pekka Alastalo, executive medical director, [email protected]Saara Salonoja, communication specialist, [email protected]

Foundation Fighting Blindness
Chris Adams, VP Marketing & Communications, +1 410 423 0585, [email protected]

AGTC Provides Trial Design Update for its Phase 2/3 XLRP Clinical Trial, New Data on XLRP Higher Dose Group and Financial Results for the Fourth Quarter and Year Ended June 30, 2020

September 9, 2020 at 7:00 AM EDTPDF Version

– Planned Phase 2/3 trial expected to include approximately 60 patients and a responder analysis based on at least 7 decibel improvement in visual sensitivity in at least 5 loci across two active dose groups and one control group –

– Data from Group 5 supports use of higher dose level in Phase 2/3 trial –

– Company on track to provide multiple data readouts for XLRP and ACHM clinical programs in 4Q 2020 –

– Company to host management update and webcast with slides today at 8:00am ET –

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today provided additional information about the proposed design of the planned Phase 2/3 trial for its X-linked retinitis pigmentosa (XLRP) clinical program, which is expected to commence in the first quarter of 2021, and new preliminary data on a higher dose Group 5 from the ongoing Phase 1/2 XLRP trial.  The Company also announced financial results for the fourth quarter and fiscal year ended June 30, 2020. 

“We are excited to close out our 2020 fiscal year with a path toward initiating a Phase 2/3 trial for our XLRP program, and to provide new preliminary data for the higher dose Group 5 in our Phase 1/2 trial, which supports inclusion of that dose level in the Phase 2/3. In addition, we remain on track to report additional data readouts from all three of our clinical programs in the fourth quarter of the 2020 calendar year,” said Sue Washer, President and CEO of AGTC. “The advancements we are making in our clinical programs, highlighted by the proposed design of our planned Phase 2/3 XLRP trial, underscore our continued progress.”

Recent Highlights

X-linked Retinitis Pigmentosa (XLRP)

The proposed design of the XLRP Phase 2/3 trial is expected to include approximately 60 patients randomized across three arms: a low-dose group (1.2E+11 vg/mL the Group 2 dose from the ongoing Phase 1/2 trial), a high-dose group (1.1E+12 vg/mL the Group 5 dose from the ongoing Phase 1/2 trial), and an untreated control group. The primary endpoint will be based on visual sensitivity, with a responder defined as having at least 7 decibel improvement in visual sensitivity in at least 5 loci at month 12, which is intended to represent a clinically meaningful benefit. Responder rates in each active arm will be compared to responder rates in the control arm. The Company plans to submit a 6-month interim analysis of the data from the Phase 2/3 to the FDA to obtain feedback on the Company’s development plan to support approval.  Based on any FDA feedback, the Company may modify the final trial design, enrollment numbers, and/or statistical analysis plan.  The Company also will discuss with FDA the possibility of finalizing dose selection for the treatment of the contralateral eye based on this 6-month interim data. The Company expects to begin enrolling patients in 1Q 2021 and to provide results from the 6-month interim analysis in 3Q 2022, dependent on future effects of the COVID-19 pandemic on clinical trial enrollment.

Achromatopsia (ACHM)

Preclinical Programs

AGTC’s preclinical pipeline includes two ophthalmology programs, one of which targets the dry form of age-related macular degeneration (AMD), and three programs targeting central nervous system (CNS) disorders. The CNS programs target adrenoleukodystrophy (ALD) and two additional rare genetic CNS indications—frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS)—that have substantial patient populations and well-defined clinical phenotypes. AGTC also has collaborations with Otonomy and Bionic Sight for genetic forms of hearing loss and optogenetics, respectively.

Mobile Vision Testing Program

In June 2020, AGTC announced that it launched a mobile vision testing program to conduct follow-up assessments during the COVID-19 pandemic for patients enrolled in the Company’s ongoing clinical trials in XLRP and ACHM.  The mobile vision testing program is available to patients enrolled in AGTC’s clinical trials across the United States so that they are able to maintain their follow-up study assessments while COVID-19 restrictions remain in effect. To date, more than 25 patients have been seen in the mobile vision testing program.

Financial Results for the Fourth Quarter and Fiscal Year Ended June 30, 2020

Revenue: There was no revenue for the fourth quarter of 2020 and $2.5 million for the year ended June 30, 2020, compared to $0.4 million and $41.7 million in the comparable periods in fiscal year 2019. Revenue for the year ended June 30, 2020 was primarily $2.2 million of non-cash collaboration revenue in connection with the in-kind contributions made to Bionic Sight.

R&D Expenses:  Research and development expenses were $10.5 million for the fourth quarter of 2020 and $35.8 million for the year ended June 30, 2020, compared to $8.3 million and $33.2 million in the comparable periods in fiscal year 2019. The increase of $2.6 million during the full year period was primarily due to increased external XLRP spending primarily related to Phase 2/3 activities, increased employee-related costs and increased external spending related to ACHM, primarily due to patient enrollment. These expenses were partially offset by decreased external research and discovery spending and share-based compensation expenses. 

G&A Expenses:  General and administrative expenses were $4.1 million for the fourth quarter of 2020 and $13.6 million for the fiscal year ended June 30, 2020, compared to $3.5 million and $12.9 million in the comparable periods in fiscal year 2019. The increase in general and administrative expenses for the full year was primarily driven by increased employee-related expenses partially offset by a decrease in share-based compensation expenses.

Net Income (Loss): Net loss was $14.5 million for the fourth quarter of 2020 and $45.9 million for the year ended June 30, 2020, compared to net loss of $10.5 million and $2.0 million in the comparable periods in 2019.

Financial Guidance:  As of June 30, 2020, the Company’s cash, cash equivalents and investments totaled $80.5 million. The Company believes these funds will be sufficient to allow AGTC to generate data from its ongoing clinical programs, initiate a Phase 2/3 on XLRP trial and to fund currently planned research and discovery programs into the fourth quarter of calendar year 2021. 

Conference Call and Webcast
AGTC will host a conference call and webcast with accompanying slides to review the Phase 2/3 XLRP trial design and discuss financial results for the fourth quarter and fiscal year ended June 30, 2020 today at 8:00am ET. To access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US). A live webcast will be available in the Events and Presentations section of AGTC’s Investor Relations site at http://ir.agtc.com/events-and-presentations. Please log in approximately 10 minutes prior to the scheduled start time.

The archived webcast will be available in the Events and Presentations section of the Company’s website.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Company’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its pre-clinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

About X-linked Retinitis Pigmentosa (XLRP)
XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life. AGTC was granted U.S. Food and Drug Administration (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

About Achromatopsia (ACHM)
Achromatopsia is an inherited retinal disease, which is present from birth and is characterized by the lack of cone photoreceptor function. The condition results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by achromatopsia, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind.

Forward-Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements regarding the timing for and expected expansion of its planned Phase 2/3 XLRP clinical trial, the timing for reporting data in both its XLRP and ACHM clinical programs and the funding needs for these programs. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic, including the impact on its ability to enroll patients. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in our most recent annual or quarterly report and in other reports we have filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

APPLIED GENETIC TECHNOLOGIES CORPORATION 
BALANCE SHEETS
(Unaudited)


IR/PR CONTACTS: 
David Carey
 (IR) or Glenn Silver
 (PR)
Lazar FINN Partners

T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]
Corporate Contact:
Bill Sullivan

Chief Financial Officer
Applied Genetic Technologies Corporation

T: (617) 843-5728
[email protected]
Stephen Potter

Chief Business Officer
Applied Genetic Technologies Corporation

T: (617) 413-2754
[email protected]
 




Source: Applied Genetic Technologies Corporation

AGTC to Participate at Upcoming Virtual Investor Conferences

September 3, 2020 at 4:05 PM EDTPDF Version

GAINESVILLE, Fla., and CAMBRIDGE, Mass., Sept. 03, 2020 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company focused on developing adeno-associated virus (AAV) based gene therapies for the treatment of rare inherited diseases, today announced that management will be participating in the following virtual conferences:

A live audio webcast of the presentation at the Wells Fargo and Cantor Fitzgerald conferences can be accessed by visiting http://ir.agtc.com/events-and-presentations. A replay will be available on the company’s website following the event.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Company’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its pre-clinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

IR/PR CONTACTS: 
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Applied Genetic Technologies Logo

Source: Applied Genetic Technologies Corporation

AGTC to Host Management Update on September 9 at 8:00 am ET to Discuss Planned Phase 2/3 XLRP Trial, Share Additional XLRP Data and Report Fourth Quarter and Fiscal Year End 2020 Financial Results

September 2, 2020 at 4:05 PM EDTPDF Version

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Sept. 02, 2020 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced that it will provide a management update on its planned Phase 2/3 X-Linked Retinitis Pigmentosa (XLRP) clinical trial design, a re-analysis of dose Groups 2 and 4 data, and new preliminary visual sensitivity data from Group 5; data from Group 5 is in advance of the full interim data analysis that the Company expects to provide in 4Q 2020 for both Groups 5 and 6.  The Company will also report financial results for the fiscal quarter and year ended June 30, 2020 before the market opens on Wednesday, September 9, 2020.  AGTC management will host a conference call and webcast with accompanying slides beginning at 8:00am Eastern Time on the same date.

To access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US). A live webcast will be available in the Events and Presentations section of the Investor Relations page at http://ir.agtc.com/events-and-presentations. Please log in approximately 10 minutes prior to the scheduled start time. The archived webcast will be available in the Events and Presentations section of the company’s website.

About AGTC

AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Company’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its pre-clinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in larger ophthalmology indications, optogenetics, otology and CNS disorders.

About X-linked Retinitis Pigmentosa (XLRP)

XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life. AGTC was granted U.S. Food and Drug Administration (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

Forward-Looking Statements

This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements regarding the timing for and expected expansion of its XLRP clinical development program, the timing for reporting data in its XLRP and ACHM clinical programs, and its ability to enroll patients, effectively design and successfully complete its ongoing clinical trials. Forward-looking statements include information concerning possible or assumed preclinical and clinical product development and regulatory progress, future results of operations, financial guidance, business strategies and operations, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on the Company’s business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in the Company’s most recent annual or quarterly report and in other reports AGTC has filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, AGTC assumes no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

IR/PR CONTACTS: 
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Applied Genetic Technologies Logo

Source: Applied Genetic Technologies Corporation

AGTC Announces Publication of Preclinical Data that Support the Ongoing Clinical Development of Its XLRP Gene Therapy Program

August 25, 2020 at 7:00 AM EDTPDF VersionPreclinical studies validate the transgene and dosing used in the ongoing Phase 1/2 clinical trial in patients with XLRP due to mutations in the RPGR gene

GAINESVILLE, Fla., and CAMBRIDGE, Mass., Aug. 25, 2020 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced that preclinical data validating the transgene (hRPGRco) that is being evaluated in the Company’s ongoing Phase 1/2 clinical trial in patients with X-linked retinitis pigmentosa (XLRP) have been published in the July 15, 2020 print issue of Human Gene Therapy. The studies, which evaluated the safety and efficacy of hRPGRco and another XLRP transgene in a canine model of XLRP, demonstrated stronger expression of hRPGRco than the other transgene at all dose levels evaluated. Following subretinal administration in AGTC’s proprietary rAAV2tYF vector, each of the XLRP transgenes corrected rod-cone opsin mislocalization, which are early markers of the disease, but the hRPGRco transgene demonstrated a broader therapeutic index. Study results also helped to guide the initial dosing in ongoing Phase 1/2 trial. As previously announced, AGTC expects to begin a Phase 2/3 clinical trial of its XLRP gene therapy candidate in the first quarter of 2021.

“Gene-based therapies contain multiple elements, and small differences in any one of those elements can have a significant impact on safety and efficacy,” said Mark Shearman, PhD, Chief Scientific Officer of AGTC and an author on the publication. “We have shown our commitment to gene therapy through almost 20 years of experience and multiple clinical programs; this commitment demands that we conduct rigorous preclinical development work to ensure that we advance gene-therapy candidates that are optimized for safety and efficacy. These newly published preclinical data not only further validate the safety of our therapy but our choice of XLRP transgene for our clinical program.”

The publication reports data from studies conducted in the RPGR-mutant canine model (XLRPA2), which has been validated as a model for human XLRP due to mutations in the RPGR gene. AAV vectors contained a “stabilized” version of the human RPGR gene (hRPGRstb), which is shorter than the wildtype DNA sequence, or a full-length version of the RPGR gene that has been codon optimized for improved expression and stability (hRPGRco). Both transgenes have previously been evaluated in the XLRPA2 model using an AAV5 vector. The current studies were conducted using AGTC’s proprietary AAV2tYF vector, which is optimized for delivery to target cells within the retina. Safety was similar for the two transgenes. A key finding from the studies is that the AAV2tYF-hRPGRco vector resulted in higher RPGR gene expression compared with AAV2tYF-hRPGRstb and with AAV5-GRK1-hRPGRco. Additionally, the studies showed that while the mid-dose led to optimal correction of disease phenotypes, structural and functional rescue of photoreceptors was also achieved when treating at mid-stage disease with rAAV2tYFGRK1-hRPGRco at the lowest dose. This significantly expands therapeutic index and guided a starting dose for the ongoing Phase 1/2 clinical trial.

“Our focus on optimizing every aspect of gene therapy – including vector elements, routes of administration and manufacturing – differentiates AGTC from competitors,” said Sue Washer, President and CEO of AGTC. “Our rigorous approach to developing best-in-class therapies is, we believe, the reason that our XLRP trial data to date, based on publicly released information, compares favorably with that reported by our competitors. With more than 100 patients enrolled in our collective clinical trials, studies have shown that each of our therapies have been generally safe and well-tolerated with no serious adverse events reported. We are currently enrolling additional patients into our expanded Phase 1/2 trial and look forward to initiating a Phase 2/3 trial in the first quarter of 2021.”

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Company’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its pre-clinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in larger ophthalmology indications, optogenetics, otology and CNS disorders.

About X-linked Retinitis Pigmentosa (XLRP)
XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life. AGTC was granted U.S. Food and Drug Administration (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

Forward-Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements regarding the expected commencement of its Phase 2/3, the timing for reporting trial data and AGTC’s ability to enroll patients in its ongoing clinical trials, effectively design and successfully complete clinical trials. Forward-looking statements include information concerning possible or assumed preclinical and clinical product development and regulatory progress, future results of operations, financial guidance, business strategies and operations, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic.  Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in our most recent annual or quarterly report and in other reports we have filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

IR/PR CONTACTS:
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Applied Genetic Technologies Logo

Source: Applied Genetic Technologies Corporation

AGTC Formalizes Patient Advisory Council to Provide Insights and Guidance on Patient Perspectives for Pipeline Therapies

August 12, 2020 at 7:00 AM EDTPDF Version

Patient Advisory Council to include advocates from the inherited retinal diseases global community

 New Patient Advisory Council to focus initially on Companys clinical stage programs for X-linked retinitis pigmentosa (XLRP)

GAINESVILLE, Fla., and CAMBRIDGE, Mass., Aug. 12, 2020 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company focused on developing adeno-associated virus (AAV) based gene therapies for the treatment of rare inherited diseases, today announced the formation of a Patient Advisory Council to build on its focus of incorporating the patient and caregiver voice into the Company’s culture and clinical and pre-clinical programs.

Engagement with patients and caregivers has provided AGTC with substantive information that has guided its clinical trial design, enhanced its understanding of retinal disorders, and inspired its patient centric culture. The Council, spearheaded by Jill Dolgin, PharmD, Head of Patient Advocacy at AGTC, is comprised of individuals with inherited retinal diseases (IRDs) and members from the global community of organizations that represent them.

“Patient engagement will continue to be a critical success factor for our programs moving forward, and we are very pleased to formalize the establishment of this distinguished group of patient experts, especially as we move into the next phase of development for our X-linked retinitis pigmentosa gene therapy candidate,” said Sue Washer, President and CEO of AGTC.

In July, AGTC announced next steps in the clinical development of the Company’s potential treatment of XLRP caused by mutations in the RPGR gene following receipt of written feedback from the U.S. Food and Drug Administration. AGTC is expanding the ongoing Phase 1/2 trial to dose additional patients in two masked dosing arms to collect additional functional data. In parallel, a planned Phase 2/3 trial, which is expected to begin in Q1 2021, will be designed to evaluate sustained efficacy across multiple measures of potential benefit in patients with XLRP. 

“We are delighted to be a part of this advisory council established by AGTC to lend our voice and collective experience that spans more than five decades in the search for treatments to address blindness and vision loss,” said Brian Mansfield, PhD, Executive Vice President of Research and Interim Chief Scientific Officer at the Foundation Fighting Blindness. “Having the patient’s perspective at the center of clinical drug development is a crucial component for addressing the unmet needs of patients within the inherited retinal diseases community.”

Organizations and advocate members of the Patient Advisory Council include:

OrganizationNameTitle
Foundation Fighting BlindnessBrian Mansfield, PhDExecutive Vice President of Research, Interim Chief Scientific Officer, and oversees the My Retina Tracker Registry patient database
 Todd Durham, PhDVice President, Clinical Outcomes Research
 Michelle GlazeAssociate Director, Professional Outreach, and an individual affected by retinitis pigmentosa (RP)
 Richard FaubionSenior Director, Development; individual affected by XLRP, and a stem cell transplant clinical trial participant
Fighting Blindness CanadaShari Shaw, MHScHealth Information Officer, and an individual with RP
Retina InternationalAvril DalyCEO; Vice President, Board of Directors of the European Organization for Rare Diseases (Eurordis), and an individual affected by RP
Sofia Sees HopeLaura ManfreePresident and Founder, and parent of a child with Leber Congenital Amaurosis (LCA)
 Alison Lynch, JDDisabilities attorney, individual affected by achromatopsia, and non-gene therapy clinical trial participant

About AGTC

AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Company’s most advanced clinical programs leverage its best-in-class investigational technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its pre-clinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

About X-linked Retinitis Pigmentosa (XLRP)

XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life. AGTC was granted U.S. Food and Drug Administration (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

Forward-Looking Statements

This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements regarding the timing for and expected expansion of its XLRP clinical development program, the timing for reporting data in its XLRP and ACHM clinical programs, and its ability to enroll patients, effectively design and successfully complete its ongoing clinical trials. Forward-looking statements include information concerning possible or assumed preclinical and clinical product development and regulatory progress, future results of operations, financial guidance, business strategies and operations, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on the Company’s business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in the Company’s most recent annual or quarterly report and in other reports AGTC has filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, AGTC assumes no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

IR/PR CONTACTS: 
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Applied Genetic Technologies Logo

Source: Applied Genetic Technologies Corporation

AGTC Announces Updated Development Plan for its X-Linked Retinitis Pigmentosa Clinical Program

AGTC Announces Updated Development Plan for its X-Linked Retinitis Pigmentosa Clinical Program, Including Q1 2021 Start of Planned Phase 2/3 Trial

July 22, 2020 at 7:00 AM EDTPDF Version

FDA feedback allows for forward program development

Company reiterates that its favorable safety profile and its advanced manufacturing and analytics capabilities enable rapid clinical development

GAINESVILLE, Fla. and CAMBRIDGE, Mass., July 22, 2020 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced next steps in the clinical development of the Company’s potential treatment of X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene following receipt of written feedback from the FDA. The revised development plan, which includes immediate expansion of the current trial in parallel with the planned Phase 2/3 trial, will be designed to evaluate sustained efficacy across multiple measures of potential benefit in patients with XLRP. 

In lieu of an in-person meeting likely due to limitations imposed by COVID-19, the FDA provided comprehensive written feedback regarding the design and execution of a registration trial and future regulatory submissions. The Company continues to move forward as planned with manufacturing, clinical site preparation and other activities to enable initiation of the studies as quickly as possible. 

“We are pleased with the productive feedback from the FDA and are modifying our development program based on their recommendations to advance our XLRP gene therapy candidate,” said Sue Washer, President and CEO of AGTC. “Based on data available to date, we believe we have the potential for a best-in-class product when important factors such as visual sensitivity improvements, BCVA and safety are considered, which could provide meaningful benefit to patients with XLRP who today have no treatment options.”

AGTC is expanding its ongoing Phase 1/2 trial immediately and plans to dose approximately 20 patients in two masked dosing arms to collect additional functional data, including a mobility test added as a supplemental endpoint. The Company expects to begin dosing in Q4 2020.   

For late stage studies, the FDA has indicated in its written feedback, which is consistent with how others in the XLRP gene therapy space are analyzing data, that a change in visual sensitivity of 7 decibels or greater in at least 5 loci would be clinically meaningful. AGTC has previously reported visual sensitivity as a mean over an entire treated area, but believes multiple patients already evaluated in the ongoing Phase 1/2 trial would meet the FDA’s definition. The Company’s revised Phase 2/3 trial design is also expected to include two masked active arms in addition to a control group, with visual sensitivity as the primary endpoint and several supplemental endpoints such as the mobility test.  AGTC expects to begin this trial in Q1 2021.

Further information on the protocols for these trials including patient numbers, timelines and corporate cash guidance will be provided in the Company’s 10K filing for the fiscal year ending June 30, 2020. In addition, AGTC remains on track to provide multiple data readouts for both its XLRP and ACHM clinical programs in the second half of 2020.  These readouts will include data from the two higher dose groups in the XLRP Phase 1/2 trial.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. Initially focusing on ophthalmology, our goal is to preserve or, hopefully, be able to improve vision in some cases. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Our pre-clinical programs build on our industry leading AAV manufacturing technology and expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

About X-linked Retinitis Pigmentosa (XLRP)
XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life. AGTC was granted U.S. Food and Drug Administration (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

Forward-Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements regarding the timing for and expected expansion of its XLRP clinical development program, the timing for reporting data in its XLRP and ACHM clinical programs, and its ability to enroll patients, effectively design and successfully complete its ongoing clinical trials. Forward-looking statements include information concerning possible or assumed preclinical and clinical product development and regulatory progress, future results of operations, financial guidance, business strategies and operations, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic.  Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on the Company’s business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in the Company’s most recent annual or quarterly report and in other reports AGTC has filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, AGTC assumes no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

IR/PR CONTACTS: 
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Applied Genetic Technologies Logo

Source: Applied Genetic Technologies Corporation

AGTC Launches Nationwide Mobile Vision Testing Program for Patients Enrolled in Its Ongoing Phase 1/2 Clinical Trials

June 15, 2020 at 7:00 AM EDTPDF Version

Mobile vision testing unit enables X-linked retinitis pigmentosa and achromatopsia clinical trial patients to maintain important scheduled study assessments during COVID-19 pandemic

GAINESVILLE, Fla., and CAMBRIDGE, Mass., June 15, 2020 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced the launch of an innovative mobile vision testing program to conduct follow-up assessments during the COVID-19 pandemic for patients enrolled in the Company’s ongoing clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3).

“We recognize the incredible commitment patients make when they enroll in our trials and our goal is to match that commitment and support them in any way possible,” said Sue Washer, President and CEO of AGTC. “Our highest priority is patients and their safety. During the COVID-19 crisis, patients may be less inclined or find it harder to receive in-person follow up appointments, an important aspect of assessing the safety and efficacy of the therapy. By using a creative patient focused approach to testing via our mobile vision testing program, we “take the test to the patient” to support their needs during this extremely difficult time.”

AGTC’s Mobile Vision Center was launched in partnership with 2020 On-Site, a mobile vision company based in Boston with more than 450 corporate clients, and has been customized to include the necessary testing equipment needed, as well as appropriate safety protocols, to conduct the key follow-up assessments required to determine the potential safety and efficacy of AGTC’s investigational products. The mobile vision testing program is available to patients enrolled in AGTC’s clinical trials across the United States with up-coming scheduled visits for those who want to maintain their follow-up study assessments while restrictions remain in effect due to COVID-19.

About AGTC

AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. Initially focusing on ophthalmology, our goal is to preserve or, hopefully, be able to improve vision in some cases. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Our pre-clinical programs build on our industry leading AAV manufacturing technology and expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

About X-linked Retinitis Pigmentosa (XLRP)

XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life. AGTC was granted U.S. Food and Drug (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

About Achromatopsia (ACHM)

Achromatopsia is an inherited retinal disease, which is present from birth and is characterized by the lack of cone photoreceptor function. The condition results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by achromatopsia, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind.

Forward-Looking Statements

This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in our most recent annual or quarterly report and in other reports we have filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

IR/PR CONTACTS: 
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Applied Genetic Technologies Logo

Source: Applied Genetic Technologies Corporation

AGTC Announces Financial Results and Business Update for the Quarter Ended March 31, 2020

May 13, 2020 at 7:00 AM EDTPDF Version

– Company on track to provide multiple data readouts for XLRP and ACHM clinical programs in 2H 2020 –

– Company on track for End of Phase 2 meeting in Q2 2020 –

– Company sees minimal COVID-19 impact to date –

– Company to host conference call and webcast today at 8:00am ET –

GAINESVILLE, Fla. and CAMBRIDGE, Mass., May 13, 2020 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced financial results for the quarter ended March 31, 2020.

“AGTC started 2020 with significant momentum, announcing sustained improvements in visual function for four of eight centrally dosed patients in our XLRP trial and encouraging preliminary signs of biologic activity in our ACHM trials. The company also completed enrollment in the XLRP trial, completed adult enrollment in both ACHM trials, and strengthened our balance sheet with a successful financing in February,” said Sue Washer, President and CEO of AGTC. “We remain on track to provide multiple data readouts for both our XLRP and ACHM clinical programs in the second half of 2020, including top-line 12-month data from the peripherally and centrally-dosed initial groups and interim data from the two new higher dose groups in the XLRP trial as well as interim data in the new adult groups in both ACHM trials. Our industry-leading XLRP data package has us well-positioned for an End of Phase 2 meeting with the FDA in the second quarter of 2020. The biological activity observed to date in our XLRP clinical trial reinforces our optimism that the program will move into a pivotal trial by the end of 2020.”

Recent Highlights

X-linked Retinitis Pigmentosa (XLRP)

Achromatopsia (ACHM)

Preclinical Programs

As previously announced at AGTC’s R&D Day on January 28, 2020, AGTC’s preclinical pipeline includes two ophthalmology programs, one of which targets the dry form of age-related macular degeneration (AMD), and three programs targeting central nervous system (CNS) disorders, which include the previously announced program in adrenoleukodystrophy (ALD) as well as two additional rare genetic CNS indications, frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS), that have substantial patient populations and well-defined clinical phenotypes. AGTC also has collaborations with Otology and Bionic Sight for genetic forms of hearing loss and optogenetics, respectively.

COVID-19 Business Update

Despite the worldwide impact of the COVID-19 pandemic, AGTC remains on track and expects to report data from its XLRP program and both ACHM programs in the second half of 2020. The company already completed enrollment in the XLRP Phase 1/2 clinical trial and enrollment in all dose groups for adult patients of both ACHM trials. The company also closed a $34.8 million financing in February, providing funding to support its ongoing clinical programs.

AGTC is taking appropriate measures to ensure that the health and safety of its employees and patients are protected. The majority of the company’s personnel have been working from home and those corporate facilities and clinical testing sites that are open are operating with the utmost caution and in-line with government guidelines. These precautions and pandemic-related travel restrictions have created challenges for new patients to meet with clinicians and receive proper evaluations.

Financial Results for the Three and Nine Months Ended March 31, 2020

Revenue: Total revenue for the three and nine months ended March 31, 2020 was nil and $2.5 million, respectively, compared to $21.3 million and $41.3 million, respectively, during the comparable 2019 periods. Revenue for the nine months ended March 31, 2020 was primarily $2.2 million of non-cash consideration collaboration revenue in connection with the in-kind contributions made to Bionic Sight. Thereafter, no additional collaboration revenue will be recognized in connection with the Bionic Sight agreement.

R&D Expenses:  Research and development expenses for the three and nine months ended March 31, 2020 were $8.3 million and $25.3 million, respectively, compared to $7.2 million and $24.9 million, respectively, during the comparable 2019 periods. The increase of $0.4 million during the nine month period was primarily due to increased ACHM Phase 1/2 clinical trial expenses associated with increased patient enrollment and new site activations and increased employee-related costs, partially offset by decreased sublicense expense associated with receiving a milestone payment from Biogen in the previous fiscal year. 

G&A Expenses:  General and administrative expenses for the three and nine months ended March 31, 2020 were $3.1 million and $9.5 million, respectively, compared to $3.1 million and $9.3 million, respectively, during the comparable 2019 periods.

Net Income (Loss): Net loss for the three and nine months ended March 31, 2020 was $11.2 million and $31.4 million, respectively.  Net income for the three and nine months ended March 31, 2019 was $11.5 million and $8.5 million, respectively.

Financial Guidance:  As of March 31, 2020, the company’s cash, cash equivalents and investments totaled $84.5 million. The company believes these funds will be sufficient to allow AGTC to generate data from its ongoing clinical programs, initiate a pivotal trial on XLRP and to fund currently planned research and discovery programs into the second half of 2021.

Conference Call and Webcast
AGTC will host a conference call and webcast to discuss financial results for the third fiscal quarter ended March 31, 2020 today at 8:00am ET. To access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US). A live webcast will be available in the Events and Presentations section of AGTC’s Investor Relations site at http://ir.agtc.com/events-and-presentations. Please log in approximately 10 minutes prior to the scheduled start time.

The archived webcast will be available in the Events and Presentations section of the Company’s website.

About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. Initially focusing on ophthalmology, our goal is to preserve or, hopefully, be able to improve vision in some cases. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Our pre-clinical programs build on our industry leading AAV manufacturing technology and expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

About X-linked Retinitis Pigmentosa (XLRP)
XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life.  AGTC was granted U.S. Food and Drug (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

About Achromatopsia (ACHM)
Achromatopsia is an inherited retinal disease, which is present from birth and is characterized by the lack of cone photoreceptor function. The condition results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by achromatopsia, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind.

Forward-Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements regarding the timing for reporting data and the commencement of pivotal clinical trials. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in our most recent annual or quarterly report and in other reports we have filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

APPLIED GENETIC TECHNOLOGIES CORPORATION
BALANCE SHEETS
(Unaudited)

APPLIED GENETIC TECHNOLOGIES CORPORATION
STATEMENTS OF OPERATIONS
(Unaudited)

AGTC Announces Completion of Enrollment in All Adult Dose Groups of its Ongoing Phase 1/2 Clinical Trials in Patients with Achromatopsia

March 16, 2020 at 7:00 AM EDTPDF Version

Enrollment ongoing in pediatric dose groups

Interim data of all adult dose groups expected in 2H of 2020

GAINESVILLE, Fla. and CAMBRIDGE, Mass., March 16, 2020 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced that it has completed the planned enrollment in all dose groups for adult patients (age 18 years or older), including the two higher dose groups, of its Phase 1/2 clinical programs with achromatopsia due to mutation in the ACHM CNGB3 or ACHM CNGA3 genes. The company continues to enroll pediatric patients at the higher dose groups in both trials.

“We expect the information to be obtained from these adult dose groups, as well as the additional pediatric data, will help to reinforce the encouraging preliminary data generated to date and previously reported in September 2019 and January 2020,” said Sue Washer, president and CEO of AGTC. “We plan to report interim data from all adult dose groups in the second half of 2020 and to use the data to inform decision-making regarding readiness to move the product candidates to pivotal trials. In addition to being on track for our End of Phase 2 submission for  our X-linked retinitis pigmentosa (XLRP) clinical study, we expect to have multiple achromatopsia data read-outs in 2020 that will build on the momentum we created in January.”

AGTC most recently reported interim six-month data from the dose escalation cohorts of its ongoing ACHM Phase 1/2 clinical trials in January 2020. Results from both studies demonstrated encouraging signs of biologic activity as shown by positive changes in light discomfort testing as well as encouraging patient anecdotes describing real-world improvements in visual function. A favorable safety profile with no dose-limiting inflammatory responses was observed. 

About AGTC
AGTC is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, where it has active clinical trial programs in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, otology and adrenoleukodystrophy (ALD), frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS), which are diseases of the central nervous system (CNS), and other ophthalmology indications including Stargardts and dry age-related macular degeneration (dry AMD). The optogenetics program is being developed in collaboration with Bionic Sight. The otology program is being developed in collaboration with Otonomy. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation, manufacture and physical delivery of gene therapy products.

About Achromatopsia (ACHM)
Achromatopsia is an inherited retinal disease, which is present from birth and is characterized by the lack of cone photoreceptor function. The condition results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by achromatopsia, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind.

Forward Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities and the effects of competition. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the fiscal year ended June 30, 2018, filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

IR/PR CONTACTS: David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contacts:Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728  
[email protected]

Stephen PotterChief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Applied Genetic Technologies Logo

AGTC Announces Completion of Enrollment in the Two Highest Dose Groups of its Ongoing Phase 1/2 Clinical Trial in Patients with X-Linked Retinitis Pigmentosa

February 19, 2020 at 7:00 AM ESTPDF Version

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Feb. 19, 2020 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced that it has completed enrollment in the two highest dose groups of its Phase 1/2 clinical trial evaluating the safety and efficacy of sub-retinal injection of AGTC-501 for the treatment of X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene. The patients in these additional groups received a higher or highest dose of AGTC’s XLRP candidate.

“We expect that the information that will be obtained from the two additional dose groups will help to reinforce the data generated to date and previously reported in September 2019 and January 2020,” said Sue Washer, president and CEO of AGTC. “We plan to report interim data from these two new dose groups and to report top-line 12-month data for the first four dose groups in the second half of 2020 and intend to initiate a pivotal trial by the end of the year. Combined with the ongoing progress in our two achromatopsia clinical studies, we expect to have multiple data read-outs in 2020 that will build on the momentum we created in January.”

AGTC most recently reported data from the ongoing XLRP Phase 1/2 clinical trial in January 2020. Results at the six-month time point in patients dosed centrally were indicative of durable and meaningful improvements in central visual sensitivity, encouraging improvements in Best Corrected Visual Acuity and a favorable safety profile. 

About AGTC
AGTC is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, where it has active clinical trial programs in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, otology and adrenoleukodystrophy (ALD), frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS), which are diseases of the central nervous system (CNS), and other ophthalmology indications. The optogenetics program is being developed in collaboration with Bionic Sight. The otology program is being developed in collaboration with Otonomy. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation, manufacture and physical delivery of gene therapy products.

About X-linked Retinitis Pigmentosa (XLRP)
XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life.  AGTC was granted U.S. Food and Drug (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

Forward Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities and the effects of competition. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the fiscal year ended June 30, 2018, filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

For AGTC

David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Applied Genetic Technologies Logo

Source: Applied Genetic Technologies Corporation

AGTC Reports Positive Six-Month Data from its Ongoing Phase 1/2 Clinical Trial in X-Linked Retinitis Pigmentosa

January 9, 2020 at 7:00 AM ESTPDF Version

– Data suggest durable and meaningful improvements in central visual sensitivity
– Secondary data showed encouraging improvements in Best Corrected Visual Acuity
– All patients dosed continue to demonstrate a favorable safety profile
– Company plans to initiate pivotal trial by the end of 2020
– Company to host conference call and webcast with slides today at 8:00 am ET

GAINESVILLE, Fla., and CAMBRIDGE, Mass., Jan. 09, 2020 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today reported positive interim six-month data from its ongoing Phase 1/2 clinical program in X-linked retinitis pigmentosa (XLRP). The results show that patients treated centrally with its product candidate demonstrated durable improvement in visual function six months after dosing. These data reinforce the promising efficacy and safety results reported in September 2019 and will help to design the XLRP pivotal trial that is currently being planned to initiate by the end of 2020. The company also remains on track to report interim six-month data from the dose escalation cohorts of both of its ongoing trials in achromatopsia later this month.

“These promising results further demonstrate that our XLRP candidate has tremendous potential to provide meaningful benefit to XLRP patients who today have no treatment options,” said Sue Washer, President and CEO of AGTC. “The positive results observed to date give us confidence that the data as a whole will support advancement of our XLRP clinical program to a pivotal trial in 2020.”

Data from 17 of the 25 patients have been previously reported in September 2019 and demonstrated a favorable safety profile. Of the 17, eight peripherally treated patients showed stable visually function through six months and nine centrally treated patients showed improvement in visual function as measured by microperimetry and/or BCVA through three months.

At the six-month time-point for these same nine centrally dosed patients:

Preliminary data for additional patients enrolled at a new higher dose group are consistent with previous data.

Safety data from all 25 patients dosed to date continue to demonstrate a favorable profile for the XLRP candidate, with no dose-limiting inflammatory responses observed and no secondary inflammatory responses requiring re-administration of any steroid in any patients.

The company is scheduling additional patients for enrollment during the first quarter of the 2020. These patients will enable AGTC to generate the most robust set of data possible as the company moves forward with planning for a pivotal trial and eventual BLA application.

“The sustained improvement in visual sensitivity in centrally dosed patients are compelling and, if confirmed in a pivotal trial, would be highly meaningful to patients,” said Dr. Paul Yang, MD, PhD, Assistant Professor of Ophthalmology at the Casey Eye Institute, Oregon Health & Science University in Portland. “Additionally, this is the first investigational therapy for XLRP to report on encouraging improvements in visual acuity. The combination of improved visual function across two endpoints in centrally treated patients and the previously reported stabilization of visual function in peripherally dosed patients, suggest that this gene-based therapy has the potential to be an important new approach to treating XLRP.”

Conference Call and Webcast Today at 8:00 am ET
AGTC will host a conference call and webcast with accompanying slides to discuss the interim six-month data from the dose expansion cohort of its Phase 1/2 XLRP clinical trial today 8:00am ET. To access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US). A live webcast will be available in the Events and Presentations section of AGTC’s Investor Relations site at http://ir.agtc.com/events-and-presentations. Please log in approximately 10 minutes prior to the scheduled start time.

The archived webcast will be available in the Events and Presentations section of the Company’s website.

Tuesday January 28, 2020 R&D Day
AGTC plans to review the data from the XLRP and achromatopsia Phase 1/2 clinical programs at an R&D Day on Tuesday, January 28, 2020 beginning at 7:30 am ET in New York City. Members of the financial community interested in attending this event can register on the Events and Presentations page of the AGTC website or by clicking here.

About AGTC
AGTC is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, in which it has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, adrenoleukodystrophy (ALD), which is a disease of the central nervous system (CNS) and other CNS, ophthalmology and indications. The optogenetics program is being developed in collaboration with Bionic Sight. The otology program is being developed in collaboration with Otonomy. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation, manufacture and physical delivery of gene therapy products.

About X-linked Retinitis Pigmentosa (XLRP)
XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life. AGTC was granted U.S. Food and Drug (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

Forward Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities and the effects of competition. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the fiscal year ended June 30, 2019, filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

For AGTC
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

AGTC Announces Financial Results and Business Update for the Quarter Ended September 30, 2019

November 12, 2019 at 4:01 PM ESTPDF Version

Company to host R&D Day on January 28, 2020, in New York

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Nov. 12, 2019 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced financial results for the quarter ended September 30, 2019.

“Presenting promising topline six-month and preliminary three-month data from the dose-escalation cohorts of our ongoing Phase 1/2 clinical programs in X-linked retinitis pigmentosa and both achromatopsia trials, respectively, was a key milestone for AGTC,” said Sue Washer, President and CEO of AGTC. “We are on track to present additional data from all three programs and are advancing our discussions with the FDA such that we can finalize the design and initiate a pivotal trial in X-linked retinitis pigmentosa in 2020. We also announced the expansion of our preclinical pipeline, including naming a potential treatment for Stargardt disease as our next orphan ophthalmology product candidate, and the establishment of a strategic collaboration with Otonomy. All of these programs give us additional opportunities to leverage our industry-leading AAV platform and expertise to create value for patients and shareholders.”

Recent Highlights

AGTC Clinical Program Update

XLRP Phase 1/2 Clinical Trial
As of November 12th, a total of 24 patients have been dosed in the trial. AGTC continues to enroll patients to create a robust data set.

In September 2019, AGTC reported data from 17 patients, which continued to demonstrate a favorable profile for the XLRP candidate, with no dose-limiting inflammatory responses observed and no secondary inflammatory responses requiring re-administration of any steroid in any patients dosed to date. Efficacy analyses showed stable visual function in all eight patients dosed peripherally and for whom six-month data were available, as measured by visual fields and best-corrected visual acuity (BCVA). Evidence of improved visual function was also observed in preliminary data from nine centrally dosed patients at the three-month time point, with all nine patients demonstrating stable or improving visual acuity, a result that has not been reported by others.

ACHM Phase 1/2 Clinical Trials

As of November 12th, a total of 29 patients have been dosed in both trials. AGTC continues to enroll patients to create a robust data set. Safety data from these patients continue to demonstrate a favorable profile for each of the ACHM candidates, and the Data Safety Monitoring Committee has supported continued dose escalation and dosing of pediatric patients.

In September 2019, AGTC reported data from 14 patients dosed across the B3 and A3 Phase 1/2 trials. One of three patients at the middle dose level in each trial and two of three patients at the high dose level in the ACHM B3 trial have shown clinically meaningful improvements in light discomfort, defined as greater than one log lux change from baseline at three months. These early data suggest a potential for meaningful benefit for these patients.

Preclinical Programs

As announced in September 2019, AGTC’s preclinical pipeline includes two ophthalmology programs, one of which targets the dry form of age-related macular degeneration (AMD), and three programs targeting central nervous system (CNS) disorders, which include the previously announced program in adrenoleukodystrophy (ALD) as well as two additional rare genetic CNS indications that have substantial patient populations and well-defined clinical phenotypes. We also have collaborations with Otology and Bionic Sight for genetic forms of hearing loss and optogenetics, respectively.

On November 5, 2019, AGTC identified Stargardt disease as its second preclinical ophthalmology program and reported that its dual AAV vector system effectively expressed full-length ABCA4 protein in the retinal tissue of non-human primates following subretinal injection. Most cases of Stargardt disease result from mutations in the ABCA4 gene, and absence of functional ABCA4 protein results in the accumulation of toxic substances in photoreceptor cells. The non-human primate study provides a solid foundation on which to continue advancing this program to human clinical trials, and AGTC is conducting additional studies in support of a potential Investigational New Drug Application submission to the U.S. Food and Drug Administration.

Financial Results for the Quarter Ended September 30, 2019

R&D Expenses: Research and development expenses were $8.6 million for the three months ended September 30, 2019, compared to $10.1 million for the three months ended September 30, 2018. The decrease of $1.5 million for the quarter was primarily due to decreased XLRP sublicense expenses associated with a milestone payment from Biogen in 2018, decreased discovery spending associated with the company’s pre-clinical ophthalmology programs, and decreased XLRS spending associated with completing enrollment of the XLRS Phase 1/2 trial in April 2018.

G&A Expenses: General and administrative expenses for the three months ended September 30, 2019, were $3.3 million, compared to $3.2 million for the three months ended September 30, 2018, an increase of $0.1 million. The increase was primarily driven by higher employee-related and other G&A expenses partially offset by decreased shared based compensation expenses.    

Net Loss: Net loss was $11.6 million for the three months ended September 30, 2019, compared to net income of $1.2 million for the three months ended September 30, 2018. 

Financial Guidance: As of September 30, 2019, the company’s cash, cash equivalents, and investments amounted to $71.1 million. The company believes these funds will be sufficient to allow AGTC to generate data from its ongoing clinical programs, to initiate activities to ensure efficient transition into pivotal trials and fund the currently planned research and discovery programs into the first half of 2021. The company expects total cash, cash equivalents and investments as of June 30, 2020, to be between $30 and $40 million.

AGTC Hosting New York R&D Day on January 28, 2020

AGTC will host an R&D Day on Tuesday, January 28, 2020, in New York. The R&D Day will feature presentations from AGTC’s management team and key opinion leaders specializing in the field of retinal diseases covering XLRP, ACHM and the company’s preclinical programs. Additional details will be provided in the coming weeks.

Conference Call and Webcast
AGTC will host a conference call and webcast to discuss financial results for the first fiscal quarter ended September 30, 2019 today at 4:30pm ET. To access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US). A live webcast will be available in the Events and Presentations section of AGTC’s Investor Relations site at http://ir.agtc.com/events-and-presentations. Please log in approximately 10 minutes prior to the scheduled start time.

The archived webcast will be available in the Events and Presentations section of the Company’s website.

About AGTC
AGTC is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, in which it has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, adrenoleukodystrophy (ALD), which is a disease of the central nervous system (CNS) and other CNS, ophthalmology and other indications. The optogenetics program is being developed in collaboration with Bionic Sight. The otology program is being developed in collaboration with Otonomy. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation, manufacture and physical delivery of gene therapy products.

About X-linked Retinitis Pigmentosa (XLRP)
XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life.  AGTC was granted U.S. Food and Drug (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

About Achromatopsia (ACHM)
Achromatopsia is an inherited retinal disease, which is present from birth and is characterized by the lack of cone photoreceptor function. The condition results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by achromatopsia, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind.

Forward-Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities and the effects of competition. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the fiscal year ended June 30, 2019, as amended, filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

Financial tables follow

 
APPLIED GENETIC TECHNOLOGIES CORPORATION 
BALANCE SHEETS 
(Unaudited)

APPLIED GENETIC TECHNOLOGIES CORPORATION 
STATEMENTS OF OPERATIONS 
(Unaudited)

IR/PR CONTACTS: 
David Carey (IR) or Tom Vickery (PR)
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8482
[email protected] or [email protected]

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Applied Genetic Technologies Logo

Source: Applied Genetic Technologies Corporation

AGTC Announces Stargardt Disease as its Second Preclinical Ophthalmology Program and Reports Compelling Preclinical Data Supporting the Potential of its Dual-Vector Technology

#

IR/PR CONTACTS:
David Carey (IR) or Glenn Silver (PR)
4
Lazar FINN Partners
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]
Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]
Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

AGTC Presents Data Demonstrating Efficiency of its AAV Vectors for Ocular Gene Therapy Even in the Presence of Anti-AAV Antibodies

October 21, 2019 at 7:00 AM EDTPDF Version

Data support safety and potential clinical utility of AGTC’s ocular gene therapy candidates regardless of patients’ AAV antibody status-

GAINESVILLE, Fla., and CAMBRIDGE, Mass., Oct. 21, 2019 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today reported new data from non-clinical studies evaluating the effect of pre-existing anti-AAV antibodies on the transduction and expression efficiency of AAV vectors. Results show that the presence of neutralizing antibodies (NAbs) to AAV in the serum or in the eye did not affect gene delivery, gene expression, or inflammation following ocular administration of AAV vectors. The data will be presented in a poster at the European Society of Gene and Cell Therapy (ESGCT) taking place in Barcelona, October 22 – 25.

“Preexisting immunity to AAV remains a challenge for many AAV gene therapies due to the high prevalence of AAV in the general population,” said Mark Shearman, Ph.D., Chief Scientific Officer of AGTC and lead author on the poster. “While the eye has partial immune privilege and appears less affected by NAbs, fully understanding the degree of vector neutralization that occurs following ocular administration of AAV-based gene therapies is important for the approval of our clinical-stage product candidates and for their safe and effective use in patients. The data to be presented support the safety and efficiency of our AAV vectors independent of Nab levels, which should allow use of these vectors in larger patient populations.”

Non-human primates with low, intermediate, or high levels of systemic NAbs against AAV-TYF (the vector used in AGTC’s current clinical development programs) received intravitreal injections of an AAV vector carrying a fluorescent marker gene into their right eyes. The treated and untreated eyes were evaluated at multiple time points over the 12-week study. Vitreous humor samples were collected from treated eyes and aqueous humor samples were collected from both eyes at week 12. NAb titers were measured for all collected samples. Key findings include the following:

“AGTC’s commitment to addressing unmet patient needs requires that we fully evaluate and provide robust data that allow patients and physicians to make data-based treatment decisions,” said Sue Washer, President and CEO of AGTC. “As we advance our Phase 1/2 clinical programs in X-linked retinitis pigmentosa and achromatopsia due to mutations in the ACHM A3 or B3 genes, we are conducting parallel non-clinical studies designed to enable the most complete data set possible in support of our future Biologics License Applications. We believe these studies will be essential for regulatory success and for the safe and effective clinical use of our potential products.”

About AGTC
AGTC is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, in which it has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). In addition to its clinical trials, AGTC has preclinical programs in optogenetics; adrenoleukodystrophy (ALD), which is a disease of the central nervous system (CNS), and other CNS, ophthalmology, and otology indications. The optogenetics program is being developed in collaboration with Bionic Sight. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products, including capsids, promoters, and expression cassettes, as well as expertise in the formulation, manufacture and physical delivery of gene therapy products.

About Achromatopsia (ACHM)
Achromatopsia is an inherited retinal disease which is present from birth and is characterized by the lack of cone photoreceptor function. The condition results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by achromatopsia, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind.

About X-linked Retinitis Pigmentosa (XLRP)
XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life.  AGTC was granted U.S. Food and Drug Administration (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

Forward Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities and the effects of competition. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the fiscal year ended June 30, 2019, filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

IR/PR CONTACTS: 
David Carey (IR) or Glenn Silver (PR)
Lazar FINN Partners Ltd.
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Applied Genetic Technologies Logo

Source: Applied Genetic Technologies Corporation

AGTC Announces Financial Results and Business Update for the Fourth Quarter and Fiscal Year Ended June 30, 2019

September 26, 2019 at 4:01 PM EDTPDF Version

Company announces promising topline six-month dose escalation data from its ongoing Phase 1/2 clinical trials for X-linked retinitis pigmentosa (XLRP)

Preliminary three-month XLRP dose expansion data demonstrates improvement in 50% of centrally dosed patients

Early data from both achromatopsia (ACHM) programs indicate biologic activity

Company to host conference call and webcast with slides today at 4:30pm ET

GAINESVILLE, Fla., and CAMBRIDGE, Mass., Sept. 26, 2019 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced financial results for the fourth quarter and fiscal year ended June 30, 2019. The company also provided topline six-month data from the dose escalation groups of the Phase 1/2 XLRP program as planned.  Additionally, the Company shared a preliminary look at three-month data from the XLRP dose expansion group.  Both data sets demonstrated a favorable safety profile with stability of visual function in peripherally dosed patients and improvement of visual function in 50% of centrally dosed patients.  AGTC also provided an early look at data from the achromatopsia B3 and A3 trials showing signs of clinically meaningful improvements in light discomfort.

“The encouraging signs of stable or improved visual function, as measured by visual fields and visual acuity analysis in the XLRP trial and improvements in light discomfort in both ACHM trials, further increase our confidence in the promise of our gene therapies to make a difference to patients in need,” said Sue Washer, President and CEO of AGTC. “Taken together, we believe these data put AGTC in a favorable position to move its current clinical programs forward and bring important therapies to patients who today lack effective treatment options.  AGTC has already engaged in pivotal trial planning as well as assay validation and manufacturing, such that we will be ready to move forward rapidly as soon as the remaining data are available. We are also announcing important new programs in our preclinical pipeline that leverage our unique capabilities in vector design and manufacturing, and provide a significant platform for growth beyond our current clinical programs.”

AGTC Clinical Program Update

X-linked Retinitis Pigmentosa (XLRP) Phase 1/2 Clinical Trial
A total of 22 patients have been dosed to date. Safety data from all 22 patients continue to demonstrate a favorable profile for the XLRP candidate, with no dose-limiting inflammatory responses observed and no secondary inflammatory responses requiring re-administration of any steroid in any patients dosed to date. Data from 17 of the 22 patients, comprising top-line interim efficacy results for ten patients (eight dosed peripherally and two dosed centrally) who have been analyzed at the six-month time point and preliminary data from an additional seven centrally dosed patients who have been analyzed at the three-month time point demonstrated the following results:

• The eight patients from the dose escalation portion of the study who were dosed peripherally showed stable visual function at six months as measured by visual fields and best corrected visual acuity (BCVA).
• Preliminary data from nine centrally dosed patients at the three-month time point (seven who have completed three-month evaluation and two who have completed six-month evaluation) showed evidence of improvement in visual function. 

“Maintenance of peripheral vision and improvements in central vision are important and clinically meaningful.  Reductions in these capabilities are hallmarks of this degenerative disease and negatively impact patients’ ability to engage in daily living and work.” said Dr David Birch, Scientific Director, Retinal Foundation of the Southwest. 

AGTC believes that the biologic activity observed to date in this clinical trial, especially if sustained through the 12-month timepoint, supports our belief that this treatment may offer a stabilization of peripheral vision as well as improvement in central vision with a favorable safety profile that could be of tremendous clinical benefit to patients. 

Achromatopsia (ACHM) Phase 1/2 Clinical Trials
To date, 27 patients have been dosed across the B3 and A3 Phase 1/2 trials. Safety data from these patients continue to demonstrate a favorable profile for each of the ACHM candidates, and the Data Safety Monitoring Committee (DSMC) has supported continued dose escalation and dosing of pediatric patients. Early data suggest meaningful benefit from treatment for the patients. One of three patients at the middle dose level in each trial and two of three patients at the high dose level in the ACHM B3 trial have shown clinically meaningful improvements in light discomfort, defined as greater than one log lux change from baseline at three months.

“The evidence of biological activity observed in both the XLRP and ACHM programs furthers our confidence in the design of these trials and the potential to advance our programs to pivotal trials.” said Theresa Heah, MD, MBA, Chief Medical Officer.  “As we analyze the ACHM data, our belief is that we will see this type of meaningful improvement in light discomfort as well as other measures of visual function for these patients.”

Preclinical Programs
As AGTC’s clinical programs demonstrate the potential of its technology platform, the company continues to advance a diverse portfolio of preclinical programs, including two ophthalmology programs, one of which targets the dry form of age-related macular degeneration (AMD), which accounts for 90 percent of the 24 million AMD cases globally; continued research in the field of otology, and three programs targeting central nervous system (CNS) disorders. The CNS programs include the previously announced program in adrenoleukodystrophy (ALD) as well as two additional rare genetic CNS indications that have substantial patient populations and well-defined clinical phenotypes. During today’s conference call, the company will briefly review these programs, which leverage the company’s deep capabilities in AAV gene therapy manufacturing and technology and represent important opportunities to create unique therapeutic solutions for patients.

Financial Results for the Fourth Quarter and Fiscal Year Ended June 30, 2019

Revenues: Revenue was $0.4 million for the fourth quarter of 2019 and $41.7 million for the year ended June 30, 2019, compared to $5.4 million and $24.2 million in the comparable periods in fiscal year 2018. Revenue primarily consists of license and related services, development services and milestone revenue. The increase of $17.5 million for the year ended June 30, 2019 compared to the same period in 2018 was primarily due to recognizing revenue of $20.4 million as a result of the termination of the Collaboration Agreement with Biogen, and recognizing revenue of $8.3 million associated with the receipt of a $10.0 million milestone payment from Biogen during the first quarter of fiscal year 2019, partially offset by decreased license and related service revenue due to the Company’s revised pattern of revenue recognition under ASC 606, Revenue from Contracts with Customers.

R&D Expenses: Research and development expenses were $8.3 million for the fourth quarter of 2019 and $33.2 million for the year ended June 30, 2019, compared to $8.8 million and $32.2 million in the comparable periods in fiscal year 2018. The increase of $1.0 million for the full year was primarily due to incurring sublicense expense of $2.3 million associated with receiving a milestone payment from Biogen and increased employee-related costs, partially offset by decreased pre-clinical R&D spending.

G&A Expenses: General and administrative expenses were $3.5 million for the fourth quarter of 2019 and $12.9 million for the fiscal year ended June 30, 2019, compared to $3.4 millionand $14.4 million in the comparable periods in fiscal year 2018.  The decrease in general and administrative expenses for the full year was primarily driven by decreased employee-related and share-based compensation expenses.

Net Loss: Net loss was $10.5 million for the fourth quarter of 2019 and $2.0 million for the year ended June 30, 2019, compared to net loss of $6.6 million and $21.3 million in the comparable periods in 2018.

Financial Guidance: As of June 30, 2019, the company’s cash, cash equivalents, and investments amounted to $82.0 million. The company believes these funds will be sufficient to allow AGTC to generate data from its ongoing clinical programs, to move the pre-clinical optogenetic program in collaboration with Bionic Sight into the clinic and fund the currently planned research and discovery programs into the first half of 2021. The company expects total cash, cash equivalents and investments as of June 30, 2020 to be between $30 and $40 million.

Conference Call and Webcast

AGTC will host a conference call and webcast with accompanying slides to review the Phase 1/2 XLRP and ACHM data and discuss financial results for the fourth quarter and fiscal year ended June 30, 2019 today at 4:30pm ET. To access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US). A live webcast will be available in the Events and Presentations section of AGTC’s Investor Relations site at http://ir.agtc.com/events-and-presentations. Please log in approximately 10 minutes prior to the scheduled start time.

The archived webcast and slide presentation will be available in the Events and Presentations section of the Company’s website.

About AGTC

AGTC is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, where it has active clinical trials in X-linked retinitis pigmentosa (XLRP), and achromatopsia (ACHM CNGB3 & ACHM CNGA3). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, adrenoleukodystrophy (ALD), which is a disease of the central nervous system (CNS) and other CNS, ophthalmology and otology indications. The optogenetics program is being developed in collaboration with Bionic Sight. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation, manufacture and physical delivery of gene therapy products.

About Achromatopsia (ACHM)

Achromatopsia is an inherited retinal disease, which is present from birth and is characterized by the lack of cone photoreceptor function. The condition results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by achromatopsia, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind.

About X-linked Retinitis Pigmentosa (XLRP)
XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life.  AGTC was granted U.S. Food and Drug (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

Forward Looking Statements

This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities and the effects of competition. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the fiscal year ended June 30, 2019, filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

APPLIED GENETIC TECHNOLOGIES CORPORATION 
BALANCE SHEETS 
(Unaudited)

APPLIED GENETIC TECHNOLOGIES CORPORATION 
STATEMENTS OF OPERATIONS 
(Unaudited)

IR/PR CONTACTS: 
David Carey (IR) or Tom Vickery (PR)
Lazar Partners Ltd.
T: (212) 867-1768 or (646) 871-8482
[email protected] or [email protected] 

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Applied Genetic Technologies Logo

Source: Applied Genetic Technologies Corporation

Italian Achromatopsia Association Increases Global Awareness of Achromatopsia With an Expedition to the Island of the Color Blind

August 14, 2019 07:30 ET Source: Italian Achromatopsia Association

Italian Achromatopsia Association Increases Global Awareness of Achromatopsia, a Rare Genetic Eye Disease, With an Expedition to Pingelap, the Island of the Color Blind

TRENTO, Italy, Aug. 14, 2019 (GLOBE NEWSWIRE) — The Italian Achromatopsia Association (IAA) today announced that it is organizing a trip to Pingelap, a remote Micronesian atoll in the South Pacific, also known as the “Island of the Color Blind.” While the incidence of achromatopsia, a rare inherited retinal disease (IRD), in the general population is 1 in every 30,000 individuals, the condition is found in between 10 and 30 percent of Pingelap’s population. Achromatopsia, for which there is no cure, causes extreme light sensitivity resulting in daytime blindness, poor visual acuity and a complete loss of color discrimination. 

The trip was made possible in part by a number of sponsors, including Applied Genetic Technologies Corporation (Nasdaq:AGTC), a biotechnology company that is committed to developing transformational genetic therapies for patients suffering from rare diseases, including achromatopsia.

“Our team is incredibly excited to embark on this journey to enhance global understanding of achromatopsia and the differences between this condition and the more common forms of color blindness,” said Elisabetta Luchetta, President of the IAA. “We are so pleased to have AGTC as a sponsor of this initiative, which aims to increase awareness about the challenges of living with achromatopsia and enable the global community to understand the impact this inherited condition has on the daily lives of achromats.“

Twenty-five years ago, Oliver Sacks, MD, the neurologist and author, embarked on a journey to Pingelap to study the island’s culture and the way the Pingelapese have adapted to accommodate this genetic eye condition, which, while rare in most populations, is common within their community. Sacks documented the trip in his 1996 book The Island of the Colorblind and in a BBC film of the same name. Inspired by his trip, the IAA will develop a documentary about Pingelap to provide a unique perspective that compares and contrasts the lives of people with achromatopsia who experience their disease as a rare condition and those on the island, who experience it in a more communal context.

“While there are ways to manage the condition, there is still a significant need to develop more advanced treatment options to improve the lives of patients,” said Dr. Irene Maumenee, Director of Ophthalmic Genetics at Columbia, and one of the original researchers to travel to Pingelap and initiate a long-term analysis of achromatopsia. “Gene therapy has the potential to emerge as an innovative and game-changing treatment for people with achromatopsia and other rare IRDs. We hope that some of the insights gathered on this trip can be translated into meaningful clinical outcomes for people with rare IRDs across the globe.”

Achromatopsia is an inherited retinal condition caused by mutations in one of several genes. The two most commonly affected are the CNGB3 and CNGA3 genes. Over 200 years ago, Pingelap was devastated by a typhoon which spared only a few inhabitants of the island. Some of those who lived are believed to have carried a recessive mutation in the CNGB3 gene. This created a genetic “founder effect” as the increased prevalence of mutation within the tiny population resulted in a higher rate of achromatopic individuals.

“We are thrilled to support the IAA and hope that what they learn from the expedition will help illuminate the unique challenges of achromatopsia,” said Kate Edgar, Executive Director of the Oliver Sacks Foundation. “No one is better equipped to do that than people who live with the condition themselves, including the members of the IAA. Dr. Sacks would have been intrigued and delighted to know that his book The Island of the Colorblind has inspired this new exploration.”

Sponsors Making the Trip a Reality

Sponsors of this trip include the following:

Dr. Sacks, referred to by The New York Times as “the poet laureate of medicine,” was a physician, best-selling author, and professor of neurology. He is best known for his collections of neurological case histories, including The Island of the Colorblind, The Man who Mistook His Wife for a HatMusicophilia, and An Anthropologist on MarsAwakenings, his book about a group of patients who had survived the great encephalitis lethargica epidemic of the early twentieth century, inspired the 1990 Academy Award-nominated feature film starring Robert De Niro and Robin Williams.

About the Italian Achromatopsia Association
The Italian Achromatopsia Association (IAA) is a NGO operating in Italy since 1999 with over 300 direct supporters in Italy – achromats and their families. The Association is committed to raising awareness about achromatopsia and enable achromats to meet all over Europe to share their experience. The Association organizes a yearly European meeting and periodic family gatherings.

Stefania Pesavento
Italian Achromatopsia Association
[email protected] 
+34 682 667 666   

AGTC Completes Enrollment of Third Group in the Dose Escalation Portion of the Achromatopsia CNGA3 Phase 1/2 Clinical Study

July 23, 2019 at 7:00 AM EDT

GAINESVILLE, Fla., and CAMBRIDGE, Mass., July 23, 2019 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced that it has completed enrollment of the third group in the dose escalation portion of its Phase 1/2 clinical trial evaluating the safety and efficacy of subretinal injection of (rAAV2tYF-PR1.7-hCNGA3) for the treatment of achromatopsia (ACHM) caused by mutations in the CNGA3 gene.

“This achievement is another demonstration of our continued progress in the clinical development of our three product candidates for the treatment of X-linked retinitis pigmentosa and achromatopsia due to mutations in the CNGA3 or CNGB3 gene,” said Sue Washer, president and CEO of AGTC. “To date, we have dosed 68 patients across our orphan ophthalmology programs. This reflects the strong enthusiasm and support among patients and physicians for novel approaches to treating diseases that have tremendous unmet medical need.”

AGTC is enrolling patients in two parallel Phase 1/2 clinical trials of its product candidates for ACHM caused by mutations in the two most common ACHM genes, CNGB3 and CNGA3. The primary focus of the ACHM trials is assessing the safety of the vector and subretinal delivery procedure through analysis of focal (ocular) and systemic treatment-emergent adverse events. Safety will be monitored by evaluation of ocular and non-ocular adverse events. In addition, each trial will measure biologic activity by assessing changes in a wide number of measures of visual function, including visual acuity, light sensitivity, color vision, and quality of life. The company expects to provide interim dose-escalation data from both trials in the fourth quarter of 2019.

About AGTC
AGTC is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, where it has active clinical trials in X-linked retinitis pigmentosa (XLRP), achromatopsia (ACHM CNGB3 & ACHM CNGA3) and X-linked retinoschisis (XLRS). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, adrenoleukodystrophy (ALD), which is a disease of the central nervous system (CNS) and other ophthalmology and otology indications. The optogenetics program is being developed in collaboration with Bionic Sight. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation, manufacture and physical delivery of gene therapy products.

About Achromatopsia (ACHM)
Achromatopsia is an inherited retinal disease, which is present from birth and is characterized by the lack of cone photoreceptor function. The condition results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by achromatopsia, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind.

About X-linked Retinitis Pigmentosa (XLRP)
XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life.  AGTC was granted U.S. Food and Drug (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

Forward Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities and the effects of competition. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the fiscal year ended June 30, 2018, filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

IR/PR Contacts: 
David Carey (IR) or Glenn Silver (PR)
Lazar Partners Ltd.
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contacts:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728   
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

Applied Genetic Technologies Logo

Source: Applied Genetic Technologies Corporation

AGTC Announces Reaching Enrollment Milestones in Phase 1/2 Clinical Trials

April 08, 2019 07:00 ET Source: Applied Genetic Technologies Corporation

GAINESVILLE, Fla., and CAMBRIDGE, Mass., April 08, 2019 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced that it has achieved enrollment milestones in two of its Phase 1/2 clinical trials. The company completed enrollment of the dose escalation portion of the achromatopsia (ACHM) CNGB3 trial, which is evaluating the safety and efficacy of its product candidate (rAAV2tYF-PR1.7-hCNGB3) for the treatment of ACHM caused by mutations in the CNGB3 gene. The company also completed enrollment in the expansion group, which includes pediatric patients, of its product candidate (rAAV2tYF-GRK1-RPGR) for the treatment of X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene. Both investigational therapies utilize the company’s proprietary AAV delivery technology and are administered by subretinal injection. 

“Continued success enrolling in all our clinical trials is critical to advancing new and innovative gene therapies for patients with inherited retinal diseases that lack approved treatment options and we are pleased with the progress we have made,” said Sue Washer, president and CEO of AGTC. “Achieving these important milestones is a testament to the dedication of the AGTC clinical teams and our clinical investigators, underscoring the support that our investigational therapies have among the patient communities we seek to serve.”

The company is enrolling patients in two parallel Phase 1/2 clinical trials of its product candidates for ACHM caused by mutations in the two most common ACHM genes, CNGB3 and CNGA3. To date, AGTC has enrolled 12 patients in the ACHM CNGB3 trial and six patients in the ACHM CNGA3 trial. AGTC plans to provide topline interim six-month data for the dose escalation portion of the CNGB3 study in the second half of 2019. To date, AGTC has completed enrollment of 10 and six patients in the dose escalation and expansion portions of the XLRP trial, respectively. The company plans to provide topline interim six-month data for the dose escalation portion of the trial in the third quarter of 2019 and the expansion portion in the fourth quarter.

The primary focus of each trial is assessing the safety of the vector and subretinal delivery procedure through analysis of focal (ocular) and systemic treatment-emergent adverse events. Safety will be monitored by evaluation of ocular and non-ocular adverse events. In addition, each trial will measure biologic activity by assessing changes in a wide number of measures of visual function, including visual fields, visual acuity and quality of life. 

About AGTC
AGTC is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, where it has active clinical trials in X-linked retinitis pigmentosa (XLRP), achromatopsia (ACHM CNGB3 & ACHM CNGA3) and X-linked retinoschisis (XLRS). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, adrenoleukodystrophy (ALD), which is a disease of the central nervous system (CNS) and other ophthalmology and otology indications. The optogenetics program is being developed in collaboration with Bionic Sight. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation, manufacture and physical delivery of gene therapy products.

About Achromatopsia (ACHM)
Achromatopsia is an inherited retinal disease, which is present from birth and is characterized by the lack of cone photoreceptor function. The condition results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by achromatopsia, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind. 

About X-linked Retinitis Pigmentosa (XLRP)
XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life. AGTC was granted U.S. Food and Drug (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

Forward Looking Statements
This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities and the effects of competition. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the fiscal year ended June 30, 2018, filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

IR/PR CONTACTS: 
David Carey (IR) or Tom Vickery (PR)
Lazar Partners Ltd.
T: (212) 867-1768 or (646) 871-8482
[email protected] or [email protected]

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

AGTC Announces Topline Interim Six-Month Data from Phase 1/2 X-Linked Retinoschisis Clinical Study

December 12, 2018 at 4:01 PM ESTPDF Version

– Results from XLRS study support general safety and tolerability of AGTC’s gene delivery platform but did not demonstrate signs of clinical activity at interim six-month time point –
– AGTC to regain rights to five clinical and preclinical development programs; Biogen terminates collaboration agreement –
– AGTC on schedule to advance its Phase 1/2 clinical studies in achromatopsia and X-linked retinitis pigmentosa; three data releases expected in 2019 –
– AGTC to host conference call and webcast today at 5:00 p.m. ET –

GAINESVILLE, Fla., and CAMBRIDGE, Mass., Dec. 12, 2018 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (NASDAQ: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today reported topline interim six-month data from its Phase 1/2 clinical trial of rAAV2tYF-CB-hRS1, an investigational AAV-based gene therapy delivered via intravitreal injection for X-linked retinoschisis (XLRS) due to mutations in the RS1 gene. Results from the study show that rAAV2tYF-CB-hRS1 is generally safe and well-tolerated, but no signs of clinical activity were observed at six-months. As per the study protocol, AGTC will continue to monitor enrolled patients at scheduled visits through the end of the study.

AGTC also announced that the company will regain full rights to the XLRS and X-linked retinitis pigmentosa (XLRP) programs and the three other partnered discovery programs following Biogen’s termination of the collaboration agreement, which will be effective on March 8, 2019. The company will complete patient monitoring activities on the XLRS program according to the clinical protocol but will not further develop the product. AGTC plans to advance the XLRP program as previously disclosed, and will determine next steps for the three discovery programs over the next several months.

“The data from the XLRS trial support the general safety and tolerability of our gene delivery platform and further reinforce our confidence in our ongoing Phase 1/2 trials in achromatopsia (ACHM) and XLRP,” said Sue Washer, President and CEO of AGTC. “We are extremely grateful for the commitment of the patients, families, patient advocacy organizations and our clinical collaborators, which was crucial to completing enrollment of this Phase 1/2 trial. Through this study, we have deepened our understanding of the challenges that patients with inherited ophthalmic diseases face every day. It has also provided us with valuable information and insights in designing and analyzing clinical trials for inherited retinal diseases. We remain dedicated to leveraging our gene delivery platform to improve the care and outcomes for patients with ACHM, XLRP and other ophthalmic diseases.”

Ms. Washer continued: “We appreciate our collaboration with Biogen and the support they have provided in advancing our joint efforts. AGTC will regain full rights to the XLRP Phase 1/2 clinical program, which has completed its dose escalation phase ahead of our previous guidance and is moving directly to an expansion phase in 2019. AGTC’s two ongoing clinical programs in ACHM are proceeding as previously disclosed and the company is expecting data readouts from all three trials in 2019. We will also regain control of the preclinical programs in adrenoleukodystrophy (ALD) and two ophthalmic programs. After reviewing all available data for these programs, we will decide which to independently move forward into IND-enabling studies.” 

Topline XLRS Six-Month Clinical Data 
The Phase 1/2 XLRS trial is an open-label, dose escalation study designed to assess the safety and efficacy of intravitreal administration of the AAV-based gene therapy in patients diagnosed with XLRS caused by mutations in the RS1 gene. Trial participants were enrolled sequentially in four groups. Individuals in Groups 1, 2 and 3 received a low, middle or high dose, respectively, of the investigational study agent. Patients in Group 4 received the maximum tolerated or high dose as determined by the first three groups. In addition, a group of pediatric patients was enrolled at the middle dose. The primary endpoint of this study was designed to evaluate safety and tolerability, whereas secondary efficacy outcomes included changes in visual acuity, visual field, schisis (cystic) cavity size and ERG b-wave amplitude, among others. A total of 27 subjects were treated and all subjects completed study visits through at least month six. AGTC will continue to monitor study participants as required in the study protocol.

Key study findings include:

In summary, Ms. Washer notes: “Based on AGTC’s XLRS data and other recently reported intravitreal injection data, it is clear that the intravitreal injection used in our XLRS program created a challenge compared with our other clinical programs, all of which utilize a subretinal injection. While clinical plans for XLRP and ACHM are supported by robust data from naturally occurring large animal models, such models do not exist for XLRS.  AGTC will benefit from our experience with the XLRS trial data because it strengthens our ability to effectively analyze and interpret a variety of important ophthalmic endpoints that have not commonly been used in inherited retinal diseases. I believe this expertise will give us an advantage in supporting future development of our diverse ophthalmic pipeline across multiple products.”

The company plans to present data from the trial at an upcoming medical or scientific meeting in 2019. For more information on AGTC and its pipeline of AAV-based gene therapy candidates in rare diseases, please visit https://agtc.com/programs/.

Financial Guidance
AGTC’s previous financial guidance, even in light of Biogen’s termination of the collaboration agreement, remains unchanged. As of September 30, 2018, the company’s cash, cash equivalents and investments totaled $105.4 million. The company believes these funds will be sufficient to allow AGTC to generate data from its ongoing clinical programs, to move the preclinical optogenetic program in collaboration with Bionic Sight into the clinic and fund the currently planned research and discovery programs for at least the next two years. The company expects total cash, cash equivalents and investments as of June 30, 2019, to be between $65 and $75 million.

Conference Call and Webcast Information 
AGTC’s management will host a conference call today at 5:00 p.m. ET.  A question and answer session will follow AGTC’s remarks. To access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US). A live webcast will be available in the Events and Presentations section of AGTC’s Investor Relations site at http://ir.agtc.com/events-and-presentations. Please log in approximately 10 minutes prior to the scheduled start time.

The archived webcast will be available in the Events and Presentations section of the company’s website.

About AGTC
AGTC is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, where it has active clinical trials in X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, adrenoleukodystrophy (ALD), which is a disease of the central nervous system (CNS), other ophthalmology indications and otology. The optogenetics program is being developed in collaboration with Bionic Sight. In addition to its product pipeline, AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation, manufacture and physical delivery of gene therapy products.

Forward Looking Statements

This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs.  Forward-looking statements include information concerning possible or assumed future results of operations, business strategies and operations, preclinical and clinical product development, including regarding which programs, if any, will be advanced in the future, and regulatory progress, potential growth opportunities, potential market opportunities and the effects of competition.  Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “anticipates,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “projects,” “should,” “will,” “would” or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the fiscal year ended June 30, 2018, as filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management’s plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

IR/PR CONTACTS: 
David Carey (IR) or Glenn Silver (PR)
Lazar Partners Ltd.
T: (212) 867-1768 or (646) 871-8485
[email protected] or [email protected]

Corporate Contact:
Bill Sullivan
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5728   
[email protected]

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
[email protected]

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Source: Applied Genetic Technologies Corporation

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