Ocugen (NASDAQ:OCGN) today announced positive 12-month results from its Phase 2 ArMaDa clinical trial evaluating OCU410 (AAV-hRORA), a modifier gene therapy for the treatment of Geographic Atrophy (GA) secondary to dry Age-Related Macular Degeneration (dAMD).
The 51-patient trial, which randomized subjects 1:1:1 into medium-dose, high-dose, and untreated control groups, demonstrated that a single subretinal injection of the medium (optimal) dose resulted in a 31% reduction in lesion growth compared to the control group (p<0.05).
Beyond the primary lesion growth metric, the trial reported a 27% slower loss of the ellipsoid zone (EZ), a critical structural biomarker for photoreceptor health and visual function.
These findings are particularly significant as OCU410 utilizes a "modifier" approach—targeting the RORA gene to regulate multiple pathways involved in dAMD (oxidative stress, inflammation, and lipid metabolism)—rather than the single-pathway inhibition seen in currently approved complement-based therapies.
Safety remains a standout feature of the OCU410 profile.
Ocugen reported that there were no OCU410-related serious adverse events (SAEs), including no cases of intraocular inflammation or vasculitis, which have been a concern for other GA treatments.
The high-dose group also showed activity, though the medium dose was identified as the optimal balance of efficacy and safety for future development.
Buoyed by these results, Ocugen has finalized the design for its Phase 3 registrational program.
The company plans to initiate the trial in Q3 2026, enrolling up to 300 subjects.
The study will utilize an adaptive design and is statistically powered at greater than 95% to detect a meaningful difference in GA lesion growth.