Ocugen disclosed that its Phase 2 ArMaDa trial of the OCU410 modifier gene therapy has produced encouraging 12‑month data from roughly half of the 51 randomized patients. The interim analysis shows a 46 % reduction in geographic atrophy lesion growth compared with the untreated control group, a 60 % slower loss of the ellipsoid zone in the Phase 1 cohort, and no OCU410‑related serious adverse events.
The results are significant because geographic atrophy affects an estimated 2–3 million patients in the United States and Europe, and current approved therapies require frequent intravitreal injections. OCU410’s once‑off, multi‑pathway approach could provide a durable, single‑treatment option that differentiates it from the complement‑inhibitor drugs Syfovre and Izervay.
Ocugen plans to report the full Phase 2 data later this quarter and to initiate a Phase 3 study in 2026, with a Biologics License Application filing targeted for 2028. The company’s roadmap positions OCU410 as a potential first‑in‑class therapy for a large, unmet market.
Management emphasized the clinical promise of the platform. CEO and co‑founder Dr. Shankar Musunuri said the 46 % lesion‑growth reduction and 60 % ellipsoid‑zone preservation “demonstrate the capability of our multi‑pathway RORA approach.” Chief Medical Officer Dr. Huma Qamar noted that the safety profile—no drug‑related serious adverse events, no inflammation signals, and no injection complications—supports a favorable risk‑benefit profile for a single‑dose therapy.
The announcement was well received by investors, with Ocugen’s shares trading higher in pre‑market trading. Market participants highlighted the robust efficacy data, the absence of safety concerns, and the potential for a once‑off treatment as key drivers of the positive reaction.
The positive interim data strengthen Ocugen’s pipeline and reinforce confidence in its modifier gene‑therapy platform. While the company continues to face financial challenges, the clinical success of OCU410 provides a critical milestone that could unlock future revenue streams and improve the company’s long‑term prospects.
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