Ocugen, Inc. Announces FDA Alignment on Phase 2/3 Pivotal Confirmatory Clinical Trial for Modifier Gene Therapy Candidate OCU410ST for Stargardt Disease

Core Viewpoint - Ocugen, Inc. has reached alignment with the FDA to proceed with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST, a gene therapy for Stargardt disease, which could lead to a biologics license application (BLA) submission if successful [1][2][5] Company Developments - The GARDian trial for OCU410ST has shown promise, with the FDA recognizing its potential to address a critical unmet medical need for approximately 44,000 Stargardt patients in the U.S. [3] - The company plans to initiate the pivotal trial in the coming months and aims for a potential BLA filing by 2027 [2] - OCU410ST has received orphan drug designations from both the FDA and the European Medicines Agency (EMA) in 2023 and 2024, respectively [2] Clinical Trial Details - The Phase 2/3 trial will involve 51 subjects, with 34 receiving a single subretinal injection of OCU410ST and 17 serving as untreated controls [4] - The primary endpoint is the change in atrophic lesion size, while secondary endpoints include visual acuity measurements [4] - One-year data from the trial will be utilized for the BLA filing [4] Safety and Efficacy - OCU410ST has demonstrated a favorable safety profile with no serious adverse events reported, including no cases of ischemic optic neuropathy or endophthalmitis [7] - The Phase 1 study indicated a 52% slower lesion growth from baseline in treated eyes compared to untreated eyes at the 6-month follow-up [7] - There was a statistically significant 2-line (10-letter) improvement in visual function at the 6-month follow-up in treated eyes [7] About Stargardt Disease - Stargardt disease is a genetic eye disorder leading to retinal degeneration and vision loss, affecting around 100,000 people in the U.S. and Europe combined [2][8] - The disease primarily causes loss of central vision due to the degeneration of photoreceptor cells in the macula [9] About Ocugen, Inc. - Ocugen, Inc. focuses on developing novel gene and cell therapies, biologics, and vaccines aimed at improving health and addressing unmet medical needs [10] - The company is advancing research in multiple areas, including retinal diseases and infectious diseases [10]