Announcement • Sep 03

Ocugen Doses First Patient in Phase 3 Registrational Trial of Ocu410 Modifier Gene Therapy for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration

Ocugen dosed the first patient in the global Phase 3 registrational trial of OCU410 (AAV5-hRORA), its first-in-class modifier gene therapy candidate for GA secondary to dry age-related macular degeneration (dAMD). The single global Phase 3 trial, ArMaDa3 (NCT07770828), is the first pivotal gene therapy trial in geographic atrophy (GA). U.S. Food and Drug Administration (FDA) granted OCU410 Regenerative Medicine Advanced Therapy (RMAT) designation, providing enhanced agency engagement throughout development and eligibility for accelerated approval and priority review. Phase 3 design fully aligned with FDA; Biologics License Application (BLA) filing anticipated in 2028. OCU410 is designed as a one-time subretinal gene therapy that addresses multiple disease pathways, offering a differentiated approach from approved complement inhibitors in the U.S. which address individual disease pathways and require ongoing intravitreal injections. The initiation of dosing follows the successful completion of a Type B End-of-Phase 2 (EOP2) meeting with FDA’s Center for Biologics Evaluation and Research (CBER) in July 2026, resulting in alignment on all critical Phase 3 design elements, including primary and secondary endpoints, dose, adaptive design, and a single pivotal trial pathway to support a BLA. The Phase 3 trial is a global, multicenter, randomized, controlled study enrolling 237 subjects with GA secondary to dAMD, randomized 2:1 to a single 200 µL subretinal injection of OCU410 (5×1010 vg/mL) or an untreated control arm, with sites in the United States, Canada, Europe, and Latin America. Primary endpoint: Rate of change of square root-transformed GA lesion area (vmm²/year) by fundus autofluorescence (FAF) at baseline, Month 4, Month 8, and Month 12, analyzed by MMRM. Secondary endpoints: Proportion of subjects with Low-Luminance Visual Acuity (LLVA) loss =15 ETDRS letters at two consecutive visits through Month 12, providing a functional vision anchor to the primary anatomic endpoint; and rate of change of ellipsoid zone (EZ) area loss by SD-OCT. Regulatory path: A single, adequate and well-controlled Phase 3 trial, aligned with FDA feedback, is intended to support a BLA filing anticipated in 2028. Discussions are ongoing with the European Medicines Agency (EMA) regarding alignment to potentially support a marketing authorization application (MAA) in Europe with this single Phase 3 trial. On July 29, 2026, FDA granted RMAT designation to OCU410 based on Phase 2 clinical data demonstrating clinically meaningful efficacy and a favorable safety profile, with no serious adverse events related to OCU410 reported. For the OCU410 program, RMAT designation provides: Eligibility for accelerated approval and priority review, which may compress the time from BLA submission to potential market entry. All benefits of Breakthrough Therapy designation, including intensive FDA guidance on efficient development and organizational commitment involving senior FDA leadership. Early and frequent FDA interactions on the use of surrogate and intermediate clinical endpoints reasonably likely to predict long-term clinical benefit-directly relevant to OCU410’s FAF-based anatomic primary endpoint. Potential flexibility in satisfying post-approval requirements, including through expanded patient registries or real-world evidence. The Phase 3 trial and the RMAT designation are supported by 12-month data from the Phase 2 ArMaDa trial (NCT06018558), a multicenter, randomized, controlled study of 51 subjects with GA secondary to dAMD. Lesion growth (FAF): 31% reduction in GA lesion area growth rate in the medium dose group versus control at 12 months (p < 0.05) in the pivotal phase 3 population (lesion size of =2.5 mm2 and =17.5 mm2), a potential 2× treatment benefit relative to the 15% and 22% reductions reported for currently approved therapies in the U.S. at 12 and 24 months, respectively. EZ preservation (SD-OCT): 27% reduction in ellipsoid zone area loss in the medium dose group versus control, a structural correlate of visual function. Responder analysis: In the medium dose group, approximately 20% of treated subjects showed no disease progression; 75% demonstrated >30% reduction in lesion growth at 12 months. Safety: No OCU410-related serious adverse events (SAEs) or adverse events of special interest (AESIs) reported to date. OCU410 (AAV5-hRORA) is Ocugen’s investigational first-in-class modifier gene therapy, delivering the RORA gene via a single unilateral subretinal injection to regulate complement activation, neuroinflammation, oxidative stress, and lipid metabolism – multiple pathways implicated in the pathogenesis of GA. OCU410 has received RMAT designation from the FDA and Advanced Therapy Medicinal Product classification from the European Medicines Agency's Committee for Advanced Therapies.

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