MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has received clearance from the U.S. Food and Drug Administration (FDA) to begin a Phase 2/3 pivotal confirmatory trial of OCU410ST, an innovative gene therapy for Stargardt disease, a debilitating genetic retinal disorder. This trial marks a crucial step toward addressing the urgent unmet medical needs of patients with ABCA4-associated retinopathies, including children and young adults facing progressive loss of central vision.
The FDA has previously granted Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation to OCU410ST, underscoring its potential as a groundbreaking treatment for Stargardt disease and related retinal conditions.
“We have had a highly productive and collaborative engagement with the FDA’s Center for Biologics Evaluation and Research (CBER) in establishing the pivotal confirmatory trial for OCU410ST,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen. “It’s evident that there is a real sense of urgency by the agency in providing treatment options for patients who currently have nothing available to them. As we initiate the Phase 2/3 registration trial, we are expediting the clinical development of OCU410ST by two to three years and potentially providing an innovative gene therapy to patients desperate for a treatment option.”
Early data from the Phase 1 GARDian trial revealed a favorable safety profile and promising efficacy. Treated eyes showed 48% slower lesion growth at 12 months compared to untreated eyes, along with meaningful improvements in visual function.
The Phase 2/3 trial will enroll 51 participants diagnosed with Stargardt disease. Thirty-four patients will receive a one-time subretinal injection targeting the less visually acute eye, while 17 patients will serve as an untreated control group. The study’s primary goal is to measure reduction in atrophic lesion size, with secondary endpoints including improvements in visual acuity. Results will inform Ocugen’s planned Biologics License Application (BLA) submission in 2027.
“The initiation of this pivotal Phase 2/3 study represents a significant milestone in our commitment to bringing transformative genetic therapies to individuals affected by Stargardt disease,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “The recent RPDD granted by the FDA for this program further underscores the urgent need for innovative treatment options for children living with Stargardt disease. OCU410ST, developed through our proprietary modifier gene therapy platform, is designed to target the underlying biological mechanisms of the disease.”
Stargardt disease, a progressive condition caused by retinal degeneration, affects an estimated one million people globally. Traditional gene therapy approaches have struggled to address the genetic complexity of this condition, which involves more than 1,200 known mutations. Ocugen’s approach leverages its proprietary AAV delivery platform, focusing on the RORA gene to regulate biological pathways associated with degeneration and vision loss.
With this clearance and the launch of the trial, Ocugen continues to demonstrate momentum in advancing late-stage clinical programs, bringing hope to patients and families living with Stargardt disease.
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