Ocugen Reports Positive Phase 2 Data for OCU410 Gene Therapy In Geographic Atrophy, Plans Phase 3 Trial Advancing Toward Registration

Ocugen, Inc. reported positive 12-month results from its Phase 2 ArMaDa trial evaluating OCU410 (AAV5-RORA), an investigational modifier gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration. Dry AMD affects an estimated 266 million people worldwide, with GA impacting roughly 2–3 million patients across the U.S. and Europe, a burden expected to rise as populations age.

“There remains a considerable unmet need in treating patients with GA and I am encouraged by the various analyses of the Phase 2 OCU410 data,” said Lejla Vajzovic, MD, FASRS, Director, Duke Surgical Vitreoretinal Fellowship Program, Associate Professor of Ophthalmology with Tenure, Adult and Pediatric Vitreoretinal Surgery and Disease, Duke University Eye Center, and Chair, Ocugen Retina Scientific Advisory Board. “In addition to the strong efficacy and safety data, OCU410 has the potential to eliminate the chronic treatment burden associated with monthly or every-other-month intravitreal injections and to reduce treatment attrition driven by patient fatigue.​”

Patients with dry AMD have limited treatment options, and currently available U.S. therapies require frequent lifelong injections, creating significant treatment burden and high real-world discontinuation rates. No approved treatments are available in many regions outside the U.S. In this context, OCU410 demonstrated meaningful clinical benefit in the Phase 2 study, including a 31% reduction in lesion growth in the optimal dose group versus control and a 27% slower rate of ellipsoid zone loss, suggesting preservation of photoreceptor structure linked to visual function. More than half of treated patients achieved at least a 30% reduction in lesion size, with subgroup analyses showing consistent benefits across subgroups.

“Our Phase 2 data consistently demonstrates statistically significant reduction of GA lesion growth after treatment with OCU410 optimal dose, and we continue to benchmark these results against natural history data to contextualise the magnitude of effect,” said Huma Qamar, MD, MPH, CMI, Chief Medical Officer of Ocugen. “We are incorporating these learnings into an anticipated Phase 3 pivotal confirmatory trial with up to 300 subjects and an adaptive design powered at over 95%.​”

The therapy also maintained a favourable safety profile, building on Phase 1 findings, with no treatment-related serious adverse events reported and no cases of major ocular complications to date. GA is driven by multiple biological pathways, while existing U.S. treatments focus only on complement inhibition. OCU410 is designed as a first-in-class RORA-based gene therapy targeting multiple disease mechanisms simultaneously, including drusen formation, inflammation, oxidative stress, and complement activation, to protect the central retinal structure.

“We have confirmed a robust treatment effect from a well-controlled Phase 2 trial of a genetic medicine for GA. Now we can move on to Phase 3 with a high degree of confidence,” said Dr Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “This moves us one step closer to bringing a transformative one-time treatment to GA patients globally who are desperately seeking rescue from vision loss.”

The randomised study enrolled 51 patients aged 50 and older who received a one-time subretinal injection of either a medium or high dose of OCU410 or were assigned to a control group. Researchers measured changes in lesion size using fundus autofluorescence imaging, an accepted regulatory endpoint, and evaluated photoreceptor integrity using OCT imaging. Based on the encouraging results, Ocugen plans to begin a Phase 3 registrational trial in the third quarter of 2026 as part of its strategy to pursue three biologics license applications within three years.