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Ocugen Announces Topline 12-month Data from Phase 2 ArMaDa Clinical Trial Evaluating OCU410 Modifier Gene Therapy for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration
Optimal dose intended for Phase 3 demonstrates statistically significant reduction in lesion growth (31%) versus control at 12 months (pPotential 2X treatment
About this update from Ocugen, Inc.
[{"type":"text","content":"Optimal dose intended for Phase 3 demonstrates statistically significant reduction in lesion growth (31%) versus control at 12 months (pPotential 2X treatment benefit compared to 15% and 22% reductions reported for currently approved therapies at 12 and 24 months, respectivelyNo serious adverse events and no adverse events of special interest related to OCU410 reported to date MALVERN, Pa., March 24, 2026 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced positive 12-month data from the Phase 2 ArMaDa clinical trial evaluating OCU410 (AAV5-RORA), its novel modifier gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). The global prevalence of dAMD is 266 million worldwide, and GA affects approximately 2-3 million people in the United States (U.S.) and Europe. Importantly, this number is expected to increase significantly as populations age. There are limited options for patients with dAMD in the U.S. and current therapies require 6–12 injections per year indefinitely, leading to substantial burden and significant dropout rates in real-world practice. Outside of the U.S., there are no approved products available. Key findings from Phase 2 include: 31% reduction in lesion growth in the optimal dose (medium) group compared to control (p27% slower rate of ellipsoid zone (EZ) loss compared to control, indicating structural preservation of photoreceptors, which correlates with visual function55% of treated patients demonstrated ≥30% lesion size reduction vs. controlSubgroup analysis (subjects with baseline GA lesions ≥5 mm2 and ≤17.5 mm2) showed 33% reduction in lesion growth compared to control in medium dose OCU410 with similar reductions in the high dose group The Phase 2 clinical trial builds directly on the clean safety profile observed in Phase 1 with no OCU410-related serious adverse events observed and no cases of endophthalmitis, retinal detachment, vasculitis, choroidal neovascularization, or ischemic optic neuropathy reported to date. GA is a multifactorial disease with a complex etiology that involves genetic and environmental factors. The current treatment options for GA in the U.S. are limited to those targeting a single mechanism—the complement pathway. By contras...