rAAV2tYF-PR1.7-hCNGA3(Applied Genetic Technologies Corp.)

July 7, 2022 at 8:00 AM EDTGAINESVILLE, Fla., and CAMBRIDGE, Mass., July 07, 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 announced two encore presentations on the interim safety results in three studies. One of the presentations will focus on interim safety results in two Phase 1/2 dose-escalation clinical trials of AGTC-401 and AGTC-402 in patients with achromatopsia (ACHM) caused by mutations in the CNGB3 and CNGBA3 genes respectively. The other presentation will focus on interim safety results in the 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). Each presentation will be shared during the American Society of Retina Specialists (ASRS) Annual Meeting, taking place July 13-16 in New York City. “We are honored to join our colleagues in the ophthalmology community, during this important retina meeting, to present data from the exciting gene therapies we are developing at AGTC as potential treatments for both ACHM and XLRP,” said Dr. Susan Schneider, Chief Medical Officer of AGTC. “We look forward to sharing future updates as we continue to progress these assets through their respective clinical development paths.” Presentation details are as follows: Interim Subretinal Gene Therapy Safety Results in Two Phase 1/2 Open-label, Dose-escalation Clinical Trials to Treat AchromatopsiaPresenter: Lejla Vajzovic, MD, FASRS, Assistant Professor of Ophthalmology, Adult and Pediatric Vitreoretinal Surgery and Diseases, Duke University Eye Center, Durham, NC Session Date/Time: July 16, 2022; 3:35 PM – 4:15 PM ET Presentation Date/Time: July 16, 2022; 3:57 PM – 4:03 PM ET Sub-retinal Gene Therapy Drug AGTC-501 for X-Linked Retinitis Pigmentosa Phase 2 Randomized, Controlled, Masked Multicenter Clinical Trial (Skyline) Interim Safety Results Presenter: Rajiv Anand, MD, FRCS, FASRS, Texas Retina Associates, Dallas, Texas Presentation Date/Time: Paper on Demand, recorded presentation available to attendees during and after the ASRS meeting Additional information can be found on the 40th Annual Scientific Meeting schedule. About X-Linked Retinitis Pigmentosa (XLRP) XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th or 5th decade of life. About Achromatopsia (ACHM) ACHM is an inherited condition associated with extremely poor visual acuity (most affected individuals are legally blind), extreme light sensitivity resulting in daytime blindness, and complete loss of color discrimination. 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 XLRP and ACHM. 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 safety of AGTC-401, AGTC-402, and AGTC 501, the potential for the clinical development of AGTC-401 as a treatment of ACHM and AGTC-501 as a treatment for XLRP, the continued safety development profile of AGTC-401 and AGTC-501, the ability to use the interim results clinical study results for AGTC-401 and AGTC-501 as a predictor of success, and whether these results will support future regulatory filings for AGTC-401 and 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, including but not limited to XLRP, 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. Corporate Contact: Jonathan Lieber Chief Financial Officer Applied Genetic Technologies Corporation T: (617) 843-5778 jlieber@agtc.com

Applied Genetic Technologies Corporation (Nasdaq: AGTC), a clinical stage biotechnology company focused on the development and commercialization of adeno-associated virus (AAV)-based gene therapies for the treatment of rare and debilitating diseases with an initial focus on inherited retinal diseases, today reported updated interim three-month pediatric results and additional adult safety results out to as long as 24 months from its ongoing Phase 1/2 dose escalation study of AGTC-401. AGTC-401 is a recombinant AAV viral vector-based gene therapy targeting mutations in the CNGB3 gene in patients with achromatopsia (ACHMB3). Data from seven pediatric patients support previously reported findings from adults treated with AGTC-401, which demonstrated encouraging biologic activity and a favorable safety profile. There were no new Suspected Unexpected Serious Adverse Reactions (SUSARs) and for the three previously reported SUSARs, the inflammation continues to improve with steroid treatment. Based on the totality of the ACHMB3 data generated to date from 31 patients over as long as 24 months, AGTC plans to advance the clinical development of AGTC-401 subject to consultation with the U.S. Food and Drug Administration (FDA) at an End-of-Phase 2 (EOP2) meeting in the first half of 2022. The Company also reported updated results from a parallel study of AGTC-402 targeting CNGA3 mutations in patients with achromatopsia that are consistent with previously reported results in adults, provide no indication of clinical improvements, and do not support further clinical development. Most patients with CNGA3 mutations express a mutant protein that is not typically found in patients with CNGB3 mutations, which the Company believes may have impacted results seen in patients that received AGTC-402. AGTC will continue to follow the ACHMA3 patients for long-term safety observations. “The three-month pediatric findings provide further evidence for the strong potential of our product candidate for patients with ACHMB3, and we look forward to continuing our discussions with the regulatory agencies to determine the best path forward to bring this important therapy to patients,” said Sue Washer, President and Chief Executive Officer of AGTC. “We understand the challenges that patients with achromatopsia face in their daily lives and our team remains dedicated to advancing this program toward commercialization. These additional data provide us the opportunity to help inform the next phase of clinical development and we thank the patients and investigators for their participation in this important clinical trial.” In the Phase 1/2 dose escalation study of AGTC-401 in ACHMB3 patients, a total of 21 adults were treated over a 100-fold dose range in five groups and a total of ten pediatric patients were treated at the three highest dose groups. The primary purpose of any Phase1/2 clinical trial is to identify a well-tolerated dose that provides clinical benefit to patients. The Company believes that the data to date support that the 1.1E+12vg/mL dose is well tolerated and provides clinical benefit in both adult and pediatric patients. “We’re pleased to have identified a generally safe and well tolerated dose of AGTC-401 for both adults and pediatric patients when inflammation is controlled,” said Dr. Robert Sisk, MD, Director of Pediatric Vitreoretinal Surgery and Director of Ophthalmic Genetics – Cincinnati Children’s Hospital and the Cincinnati Eye Institute and an investigator in the ongoing AGTC achromatopsia Phase 1/2 trials. “Based on the available pediatric data at three months, which are consistent with previously reported results in adults with ACHMB3, we are encouraged by the biologic activity observed in patients treated with AGTC-401. The achromatopsia community currently faces a significant unmet need and the totality of this data reinforces the promise of AGTC-401 as a potential treatment option if confirmed in future studies.” As previously reported, in both the ACHMB3 and ACHMA3 trials, treatment with the highest doses of AGTC-401 and AGTC-402, respectively, led to three cases of severe ocular inflammation in pediatric patients, which were reported as SUSARs. No new additional SUSARs have been reported and the inflammation in all previously reported SUSARs improved with an adjusted steroid regimen. Two SUSARs (one in ACHMA3 and one in ACHMB3) have since fully resolved and one (ACHMA3) continues to resolve, with all three patients’ best corrected visual acuity returning to baseline. Secondary outcome measures evaluating efficacy were assessed by standard visual function tests such as perimetry. We defined two pediatric patients (17 and 7 years old) in the 1.1E+12 vg/mL dose group as responders based on improvements in visual sensitivity. Therefore, of the three adults and four children (total n=7) in the 1.1E+12 vg/mL dose group, four (>50%) are visual sensitivity responders. These patients also had improvements in quality of life as measured by a patient reported outcome survey developed specifically for patients with achromatopsia. The two other pediatric patients in the 1.1E+12 vg/mL dose group and three pediatric patients ages 7 years and younger in the 3E+12vg/mL dose group (total n=5) could not sufficiently concentrate and consistently complete the visual sensitivity testing. Similar to other trials where endpoints are adapted for young children, AGTC plans to work closely with clinicians and regulators to develop potential adaptations for younger patients for visual sensitivity testing. The Company is currently working on an EOP2 meeting package for the FDA and expects to receive feedback in the first half of 2022. Subject to these discussions with FDA, the Company plans to initiate the next phase of AGTC-401 clinical development. The clinical program will be based on the favorable risk benefit pro the 1.1E+12 vg/mL dose, which had a generally safe and well tolerated safety pro all patients and signs of biologic response based on improvements in visual sensitivity and other measures of visual function. Final data and analysis of both ACHMA3 and ACHMB3 studies will be presented at relevant conferences and published for the ophthalmology community. About Achromatopsia (ACHM) Achromatopsia (ACHM) is an inherited condition caused by mutations in one of several genes, with the two most common being mutations in either the CNGB3 or CNGA3 genes. ACHM is associated with extremely poor visual acuity (most affected individuals are legally blind), extreme light sensitivity resulting in daytime blindness, and complete loss of color discrimination. AGTC is currently developing two separate AAV gene therapy product candidates for the two most prevalent forms of ACHM, caused by either a genetic mutation in the CNGB3 or CNGA3 genes. Together, these two genetic mutations account for up to 75% of the ACHM patient population. 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 and ACHM CNGA3). Its preclinical programs build on the company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical needs in optogenetics, otology and CNS disorders, and has entered strategic collaborations with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.