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

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

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

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

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

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

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

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

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

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

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

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


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

Corporate Contact:
Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
[email protected]