– 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]

Source: Applied Genetic Technologies Corporation

– 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]

Source: Applied Genetic Technologies Corporation

Read the story on WCVB

BOSTON —

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.

“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.

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

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:

• Provide 12-month data for Phase 1/2 dose groups 5 and 6 in 2Q 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 3Q 2022.

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:

• Provide 12-month data from the adult patients in both trials in 2Q 2021; and
• Provide preliminary 3-month data from the pediatric patients in both trials in 4Q 2021, AGTC currently plans to focus on completing enrollment depending on any effects of the COVID pandemic.

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 4^th or 5^th 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]

Source: Applied Genetic Technologies Corporation

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]

Source: Applied Genetic Technologies Corporation

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]

Source: Applied Genetic Technologies Corporation

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]

Source: Applied Genetic Technologies Corporation

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

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]

Source: Applied Genetic Technologies Corporation

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 Loss: The 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]

Source: Applied Genetic Technologies Corporation

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:

• Measurable improvements were observed in visual sensitivity for two of the evaluable eight patients, while a third patient identified as a responder at 6-months fell just below the cut-off. The Company believes this represents an encouraging sign of a durable biologic effect.
  • Patients are defined as responders when at least 5 loci within the central 36 loci of the perimetry grid increase by at least 7 decibels.
• Eight of nine patients treated centrally also had stable or improving visual acuity, a result not reported in other XLRP trials.

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

• Measurable improvements were observed in visual sensitivity for five of the 11 patients.
  • Patients are defined as responders when at least 5 loci within the central 36 loci of the perimetry grid increase by at least 7 decibels.
  • Three of the 11 patients in these groups, who were not responders, would not meet the inclusion criteria for future trials meaning that five of eight patients, or 62%, would be considered responders.
• Nine of the 11 patients treated centrally also had stable or improving visual acuity, a result not reported in other XLRP trials.

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 4^th or 5^th 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 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]

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:

• Alliance for Regenerative Medicine Cell & Gene Meeting on the Mesa (October 12–16, 2020)
  A company presentation by Sue Washer, President & Chief Executive Officer, will be available for registered attendees to view on-demand throughout the entirety of the conference. Please visit www.meetingonthemesa.com for full information including registration.
• Dry AMD Therapeutic Development Summit (October 28–29, 2020)
  Mark Shearman, Chief Scientific Officer, will participate in a panel discussion titled “Defining the Therapeutic Journey for Dry AMD Patients”  at 9:15 a.m. ET on Wednesday, October 28, 2020. Please visit www.dry-amd-therapeutics.com for full information including registration.

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]

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 gene–specific and gene variant–specific 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]

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.

• Analysis of visual sensitivity data from patients in the ongoing Phase 1/2 XLRP trial shows that 7 of 15 patients in Groups 2, 4 and 5 are responders at month 6 (month 3 in one case) based on the responder criteria defined above.  Focusing on the Phase 1/2 Group 5 dose group, a dose level that the Company is planning to use as the high dose in the Phase 2/3 trial, 4 of 7 patients met the response criteria. Of note, using the planned inclusion/exclusion criteria for the Phase 2/3 trial, one Phase 1/2 Group 5 patient would be removed such that the responder rate would be 4 of 6, or 67%.
• The Company also plans to expand its ongoing Phase 1/2 trial to include approximately 12 additional patients who will be masked and randomized to doses of 1.2E+11 vg/mL (Group 2 in original trial plan) and 1.1E+12 vg/mL (Group 5 in original trial plan). The Company expects to begin enrolling these additional patients in 4Q 2020 and to provide results from a 3-month interim analysis in 4Q 2021, dependent on future effects of the COVID-19 pandemic on clinical trial enrollment.
• AGTC remains on track to provide 12-month data from the ongoing Phase 1/2 trial by 4Q 2020 for Groups 1-4 to evaluate durability of effect and continued safety, as well as full 6-month data analysis for Groups 5 and 6 to evaluate safety and efficacy at higher doses.             
• The Company remains on-track to have clinical trial material produced in time for the initiation of the Phase 2/3 trial with its advanced manufacturing process that provides improved yields, purity and potency. 

Achromatopsia (ACHM)

• In January 2020, AGTC announced encouraging interim data from the dose-escalation cohorts of its ongoing Phase 1/2 clinical programs in patients with ACHM due to mutations in the ACHM CNGB3 or ACHM CNGA3 genes. In March, AGTC completed the planned enrollment in all dose groups for adult patients (age 18 years or older), including the two higher dose groups, of both ACHM trials, bringing the total number of adults dosed to 15 in the ACHM A3 trial and 22 in the ACHM B3 trial.  Pediatric dosing is ongoing with four pediatric patients dosed in each trial to date. The Company expects that pediatric enrollment may continue to be challenging.
  
• The Company plans to release additional data for the adult dose groups in 4Q 2020, which will be used to inform decision-making regarding readiness to move the product candidates to pivotal trials.

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)

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:

• 2020 Wells Fargo Virtual Healthcare Conference (September 9-10, 2020)
  Sue Washer, President & Chief Executive Officer, will participate in a fireside chat at 9:20 a.m. ET on Thursday, September 10, 2020.
  
• H.C. Wainwright 22nd Annual Global Investment Virtual Conference (September 14-16, 2020)
  AGTC management will participate in investor 1×1 meetings on Monday, September 14, 2020 and Tuesday, September 15, 2020.
  
• Cantor Fitzgerald Virtual Global Healthcare Conference 2020 (September 15-17, 2020)
  Ms. Washer will participate in a fireside chat at 2:40 p.m. ET on Wednesday, September 16, 2020.

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]

Source: Applied Genetic Technologies Corporation

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]

Source: Applied Genetic Technologies Corporation

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]

Source: Applied Genetic Technologies Corporation

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 Company’s 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:

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]

Source: Applied Genetic Technologies Corporation

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]

Source: Applied Genetic Technologies Corporation

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]

Source: Applied Genetic Technologies Corporation

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)

• In January, AGTC announced positive interim six-month data from its ongoing Phase 1/2 clinical program in XLRP. The results show that four of eight evaluable patients treated centrally with the company’s product candidate demonstrated durable improvement in visual sensitivity six months after dosing. All patients demonstrated a favorable safety profile for the XLRP candidate, with no dose-limiting inflammatory responses observed and no secondary inflammatory responses requiring re-administration of any steroids in any patients. Preliminary data also showed that all nine centrally dosed patients had stable or improving visual acuity at the six-month time point, a result that has not been reported by others.
• In February, AGTC completed enrollment in the two highest dose groups of its XLRP trial, bringing the total number of patients dosed to 28.

• An End of Phase 2 meeting with the FDA is planned for the second quarter of 2020.
   
• The company plans to release additional data in the second half of 2020 and initiate a pivotal trial by the end of 2020.

Achromatopsia (ACHM)

• In January, AGTC announced encouraging interim data from the dose-escalation cohorts of its ongoing Phase 1/2 clinical programs in patients with ACHM due to mutations in the ACHM CNGB3 or ACHM CNGA3 genes. The interim three-month results from both studies demonstrated encouraging signs of biologic activity based on improvements in light discomfort. Interim six-month data from both trials continue to demonstrate a favorable safety profile with no dose-limiting inflammatory responses.

• In March, AGTC completed the planned enrollment in all dose groups for adult patients (age 18 years or older), including the two higher dose groups, of both ACHM trials, bringing the total number of adults dosed to 15 in the ACHM A3 trial and 22 in the ACHM B3 trial.  Pediatric dosing is ongoing with three pediatric patients dosed in each trial to date. The company expects that pediatric enrollment will continue to be challenging.
  
• The company plans to release additional data for the adult dose groups in the second half of 2020, which will be used to inform decision-making regarding readiness to move the product candidates to pivotal trials.

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)

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]

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]

Source: Applied Genetic Technologies Corporation

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:

• Measurable improvements were observed in visual sensitivity for four of the evaluable eight patients. These are the same four patients discussed in September and indicate encouraging signs of a durable biologic effect.
  º  Patients are defined as responders with improvement in visual sensitivity within the treatment area that is beyond the testing variability on at least two different test dates.
• All nine patients treated centrally also had stable or improving visual acuity, 78% saw a 5 letter or more improvement, a result not reported in other XLRP trials.
• Patients who improved in either visual sensitivity or BCVA also anecdotally report noticeable improvement in visual function including greater clarity and reduced night blindness.

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]

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

• In September, AGTC announced positive data from the ongoing Phase 1/2 trial in patients with X-linked retinitis pigmentosa (XLRP), demonstrating stabilization of decline in the peripheral vision, improvement in central vision, and a favorable safety profile. Based on its ongoing discussion with physicians and advocacy groups, the company expects these results will be highly meaningful to XLRP patients, who today have no treatment options that address the underlying cause of their disease.
  
  
• In September, AGTC announced positive data demonstrating biological activity in its ongoing Phase 1/2 trials in patients with achromatopsia (ACHM) due to mutations in the ACHM B3 or A3 genes.
  
  
• AGTC entered into a strategic collaboration with Otonomy, Inc., to co-develop and co-commercialize an AAV-based gene therapy to restore hearing in patients with sensorineural hearing loss caused by a mutation in the gap junction protein beta 2 gene (GJB2) — the most common cause of congenital hearing loss.
  
  
• In a poster at the European Society of Gene and Cell Therapy annual meeting, AGTC presented 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 showed 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 support the safety and efficiency of the company’s AAV vectors independent of Nab levels, which should allow use of these vectors in larger patient populations, including those with pre-existing antibodies against AAV.
  
  
• AGTC identified Stargardt disease, an inherited form of macular dystrophy, as the company’s second preclinical ophthalmology program, and reported preclinical data supporting continued development of its Stargardt disease product candidate.

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 
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]

Source: Applied Genetic Technologies Corporation

• Simultaneous delivery of two optimized AAV vectors expands the therapeutic and commercial potential of AGTC’s industry-leading AAV platform by enabling delivery of DNA encoding larger proteins –
  GAINESVILLE, Fla., and CAMBRIDGE, Mass., Nov. 5, 2019 – 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 identified Stargardt disease as the second ophthalmology program in its previously announced preclinical pipeline expansion, which also includes a program targeting the dry form of age-related macular degeneration (AMD). The company is also reporting proof-of-concept expression data for its Stargardt disease gene therapy candidate in non-human primates (NHPs).
  Stargardt disease, the most common form of inherited macular degeneration, occurs in 1 in 8,000-10,000 individuals and most often results from mutations in the ABCA4 gene. Loss of functional ABCA4 protein leads to the accumulation of toxic substances in photoreceptors, resulting in photoreceptor death and progressive vision loss. A key challenge in developing an AAV-based gene therapy for the treatment of Stargardt disease is that the DNA sequence encoding the ABCA4 protein exceeds the payload capacity of AAV vectors. AGTC’s Stargardt disease program effectively addresses this challenge by dividing the coding sequence into two separate AAV vectors. Once inside the cell, the two DNA fragments recombine to form the complete coding sequence, which produces full length, functional ABCA4 protein.
  “The expansion of our preclinical pipeline to include Stargardt disease and dry AMD demonstrates the broad therapeutic and commercial utility of our proprietary AAV gene delivery platform,” said Sue Washer, President and CEO of AGTC. “Our Stargardt disease program underscores our ability to leverage our expertise in AAV vector and genome engineering to expand AAV gene therapy into new indications that require delivery of larger DNA payloads. Dry AMD represents a compellingly large market opportunity given that the disease affects over 24 million people globally. Each of these preclinical programs builds on our industry-leading capabilities in gene therapy for retinal diseases and will leverage our expanding expertise in designing and implementing preclinical and clinical studies that are optimized for success.”
  William Hauswirth, PhD, a leading innovator in AAV gene therapy at the University of Florida and a long-term academic collaborator with AGTC, and his colleagues at the University of British Columbia, recently published data demonstrating that a hybrid ABCA4 dual AAV vector system was safe and provided therapeutic benefit in a mouse model of Stargardt disease.1 Researchers at AGTC optimized the vectors
  2
  used in Dr. Hauswirth’s study for administration to NHPs.
  In a 13-week study, subretinal injection of AAVs prepared at AGTC encoding the N-terminal fragment and C-terminal fragment of human ABCA4, which was tagged to distinguish it from the naturally occurring non-human primate ABCA4 protein, resulted in clear detection of full length recombinant human ABCA4 protein. The protein was detected in tissue punches harvested from inside the subretinal bleb, but not in the untreated surrounding tissue, confirming the specificity and effectiveness of the vector administration. These results in NHPs are an important technical advance for the use of dual AAV vector expression systems in retinal gene therapy. Based on the results of this study, AGTC is continuing therapeutic development of its optimized dual vector system for the treatment of Stargardt disease.
  “Our recent publication provided important proof-of-concept data supporting the safety and therapeutic utility of using a dual AAV vector system to deliver and express the ABCA4 protein,” said William W. Hauswirth, PhD, Professor of Ophthalmology and the Maida and Morris Rybaczki Eminent Scholar Chair in Ophthalmic Sciences in the Department of Ophthalmology at the University of Florida. “AGTC’s demonstration that this approach is safe and results in ABCA4 protein expression in NHPs following subretinal injection of their optimized vectors is an important milestone in advancing gene therapy for Stargardt disease toward the clinic.”
  Reference
  1 Dyka FM, Molday LL, Chiodo VA, Molday RS and Hauswirth WW. Dual ABCA4-AAV vector treatment reduces pathogenic retinal A2E accumulation in a mouse model of autosomal recessive Stargardt disease. Hum Gene Ther. 2019 Sep 30. doi: 10.1089/hum.2019.132.
  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. 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.
  3
  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, 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)
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]

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:

• The presence of preexisting anti-AAV antibodies did not result in increased inflammation and did not correlate to inflammation in the dosed eye.
• Very high serum anti-AAV Nab titers were not sufficient to block or impact efficiency of AAV transduction in the retina. This indicates that systemic anti-AAV Nab titers are not the sole predictor of transduction efficiency after AAV dosing to the eye.
• There was good correlation between increases in anti-AAV Nabs in the serum and the treated eye following ocular administration of an AAV vector.  
• Ocular anti-AAV NAb levels in the aqueous and vitreous humor were similar to each other, demonstrating that aqueous humor sampling can be used to represent ocular anti-AAV NAb levels.
• Levels of anti-AAV NAbs in serum and the eye and expression of the marker gene in the retina did not appear to be impacted by the presence of preexisting anti-AAV NAbs.

“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]

Source: Applied Genetic Technologies Corporation

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. 

• Measurable improvements were observed in visual sensitivity for four of the eight (50%) patients (one centrally dosed patient was not evaluable for visual sensitivity).
• Patients with improvement in visual sensitivity within the treatment area that is beyond the testing variability on at least two different test dates are defined as responders.
• Three of eight (38%) patients had greater than five loci in the treated area with at least a 7 decibel increase from baseline, at three months compared with 33% reported by others.
• All nine patients treated centrally also had stable or improving visual acuity, a result which has not been reported by others.

“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]

Source: Applied Genetic Technologies Corporation

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:

• AGTC is a sponsor of the trip as part of its ongoing commitment to patients worldwide as the company endeavors in the future to expand its clinical program on a global level. AGTC is conducting two Phase 1/2 clinical trials of an experimental gene-based therapy for the treatment of achromatopsia. To learn more about these studies, please visit https://achromatopsiatrials.com or www.agtc.com. Additional information may be found on www.ClinicalTrials.gov (CNGA3 trial number NCT02935517 and CNGB3 NCT02599922).  
   
• Straudi SpA is a landmark for a wide range of artisans, among which carpenters, furniture makers, painters, coachmen and others in the North of Italy and especially among the Provinces of Bolzano, Trento, Verona, Vicenza, Padova, Mantova and Modena. Passion, professionalism and a wide range of high quality products, make our company a trustworthy partner for thousands of artisans.
  
  “We believed in this project at first sight, since color is our passion and we aim to bring it everywhere in the world,” says Stefano Straudi, CEO at Straudi SpA.

• The mission of the BCM Families Foundation is to eradicate Blue Cone Monochromacy by supporting the most promising biomedical and scientific research in an effort to find a cure. BCM Families Foundation is a United States non-profit organization incorporated in May 2014 by families affected by Blue Cone Monochromacy. Learn more by visiting http://www.blueconemonochromacy.org/.
   
• The Oliver Sacks Foundation is a nonprofit organization devoted to increasing understanding of the human brain and mind through the power of narrative nonfiction and case histories. The Foundation’s goals include working to reduce the stigma of mental and neurological illness, and supporting a humane approach to neurology and psychiatry. Learn more at www.oliversacks.com/oliver-sacks-foundation/.

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 Hat, Musicophilia, and An Anthropologist on Mars. Awakenings, 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   

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]

Source: Applied Genetic Technologies Corporation