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

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

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

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

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

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

About AGTC


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

Forward-Looking Statements


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

PR Contact:

Kerry Sinclair

Spectrum Science Communications

[email protected]

Corporate Contact:

Jon Lieber

Chief Financial Officer

Applied Genetic Technologies Corporation

T: (617) 843-5778

[email protected]