Ocugen, Inc. Announces U.S. FDA Clearance of Investigational New Drug Amendment to Initiate Phase 2/3 Pivotal Confirmatory Clinical Trial of OCU410ST—Modifier Gene Therapy Candidate for Stargardt Disease

Core Viewpoint - Ocugen, Inc. has received FDA clearance to initiate a Phase 2/3 pivotal confirmatory trial for OCU410ST, a gene therapy candidate for Stargardt disease, highlighting the urgency for treatment options for patients with this condition [1][2]. Company Overview - Ocugen, Inc. is a biotechnology company focused on developing gene therapies for blindness diseases, utilizing a unique modifier gene therapy platform [10]. Clinical Trial Details - The Phase 2/3 trial will enroll 51 participants with Stargardt disease, with 34 receiving a subretinal injection of OCU410ST and 17 in a control group [3]. - The primary objective is to evaluate the reduction in atrophic lesion size, with secondary endpoints including improvements in best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA) [3]. Regulatory Designations - OCU410ST has received Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation from the FDA, emphasizing the need for innovative treatments for ABCA4-associated retinopathies [1][4]. Patient Population - Approximately 100,000 patients in the U.S. and Europe, and 1 million globally, are affected by Stargardt disease, which is characterized by complex genetic mutations [4]. Clinical Data - Positive results from the Phase 1 GARDian trial indicated a 48% slower lesion growth at 12 months in treated eyes compared to untreated eyes, and a statistically significant improvement in visual function [6][7]. Future Plans - Ocugen plans to submit a Biologics License Application (BLA) for OCU410ST in 2027, aiming to file three BLAs over the next three years [5].