Benzinga | Jun 24, 2021 07:12AM EDT

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

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."