MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) said preliminary 12-month data from its Phase 2 ArMaDa clinical trial showed meaningful reductions in disease progression for patients with geographic atrophy, a late-stage form of dry age-related macular degeneration, reinforcing the company’s push toward late-stage development of its investigational gene therapy, OCU410.
The Phase 2 analysis, based on approximately half of enrolled patients evaluated to date, showed a 46% reduction in lesion growth for patients receiving OCU410 compared with untreated controls. Medium-dose patients experienced a 54% reduction, while high-dose patients showed a 36% reduction, both reaching statistical significance. The company reported no OCU410-related serious adverse events across its Phase 1 and Phase 2 studies.
OCU410 is a modifier gene therapy designed to target multiple disease pathways involved in geographic atrophy, including oxidative stress, inflammation, and complement regulation. Current U.S. treatment options largely focus on a single mechanism and require frequent intravitreal injections, while no approved therapies are available in Europe.
Additional Phase 2 findings showed that 50% of treated patients achieved more than a 50% reduction in lesion size versus control. A subgroup of patients with larger baseline lesions showed a 57% greater reduction compared with untreated patients. Phase 1 data also indicated slower deterioration of retinal structure, with ellipsoid zone loss progressing 60% more slowly in treated eyes than in untreated fellow eyes at 12 months.
Chairman and Chief Executive Officer Shankar Musunuri said the results demonstrate the potential of the company’s multi-pathway gene therapy approach. He said Ocugen expects to report full Phase 2 data later this quarter and plans to initiate a Phase 3 trial in 2026.
Chief Medical Officer Huma Qamar said the randomized Phase 2 trial delivered statistically significant anatomic efficacy across multiple analyses, while safety data across 60 patients showed no inflammation signals or injection-related complications to date.
The Phase 2 study enrolled 51 patients randomized evenly into medium-dose, high-dose, or control groups. Treated patients received a single subretinal administration, while control patients received no treatment. Ocugen said it remains on track for a biologics license application filing for OCU410 in 2028.
Geographic atrophy affects an estimated 2 million to 3 million people in the United States and Europe and represents an advanced stage of dry AMD, which impacts roughly 10 million Americans and more than 266 million people worldwide.
OCU410 has received Advanced Therapy Medicinal Product classification from the European Medicines Agency, a designation intended to accelerate the development of innovative therapies for serious conditions.
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