Ocugen Reports Positive Phase 2 Interim Data; OCU410 Gene Therapy Shows Reduced Lesion Growth And Favourable Safety In dAMD

Ocugen announced encouraging preliminary 12-month data from its Phase 2 ArMaDa trial evaluating OCU410, a modifier gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). The interim analysis, covering around half of the enrolled patients, showed meaningful reductions in GA lesion growth, highlighting the therapy’s potential in an area with significant unmet medical need. Globally, dAMD affects hundreds of millions of people, with GA impacting millions across the U.S. and Europe.

"The OCU410 Phase 1 and Phase 2 results mark a pivotal moment for Ocugen's modifier gene therapy platform and GA patients worldwide," said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. "Delivering 60% slower EZ loss in Phase 1 and 46% lesion growth reduction in the Phase 2 preliminary analysis demonstrates the capability of our multi-pathway RORA approach. We look forward to reporting full data from the OCU410 Phase 2 clinical trial later this quarter and initiating Phase 3 in 2026.”

At 12 months, OCU410 achieved a 46% reduction in lesion growth compared with control across the combined medium- and high-dose groups, with the medium dose showing the strongest effect. Half of treated patients were classified as responders, achieving more than a 50% reduction in lesion size, and a subgroup of patients with larger baseline lesions demonstrated even greater benefit compared with untreated controls.

"The clinical development journey of OCU410 has been remarkable," said Dr. Huma Qamar, Chief Medical Officer of Ocugen. "Our Phase 2 randomized trial delivered robust anatomic efficacy that was statistically significant across multiple analyses. Critically, our safety data across 60 patients has shown no drug-related serious adverse events, no inflammation signals, and no injection complications to date, supporting a favorable risk-benefit profile."

Additional supportive findings from the Phase 1 study showed slower degeneration of key retinal structures in treated eyes compared with untreated fellow eyes, suggesting preservation of photoreceptors and retinal pigment epithelium. Importantly, across both Phase 1 and Phase 2 studies, OCU410 has shown a favorable safety profile, with no treatment-related serious adverse events reported to date.

"As a practicing retinal specialist, OCU410's clinical profile is genuinely exciting for geographic atrophy patients, including a reduction in ellipsoid zone loss observed in Phase 1, which may serve as a potential marker of retinal health, and a reduction in lesion growth seen in Phase 2," said Lejla Vajzovic, MD, FASRS, Director, Duke Surgical Vitreoretinal Fellowship Program, Professor of Ophthalmology with Tenure, Adult and Pediatric Vitreoretinal Surgery and Disease, Duke University Eye Center, and Retina Scientific Advisory Board Chair of Ocugen. "With these promising results, I believe OCU410 has the potential to set a new standard of care with a single treatment for life."

Unlike currently available GA therapies that target a single pathway and require frequent injections, OCU410 is designed as a one-time, multifunctional gene therapy addressing multiple disease pathways. Ocugen plans to continue advancing the program and remains on track to submit a Biologics License Application for OCU410 in 2028 as part of its broader regulatory strategy.