MALVERN, Pa. — January 15, 2026 — Leads & Copy — Ocugen, Inc. (NASDAQ: OCGN), a biotechnology company specializing in gene therapies for blindness diseases, has announced positive preliminary 12-month data from its Phase 2 ArMaDa clinical trial, evaluating OCU410 (AAV5-RORA) for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD).
The Phase 2 trial data, covering approximately 50% of patients evaluated, demonstrated a 46% reduction in lesion growth compared to the control group. The company reported no OCU410-related serious adverse events across both Phase 1 and Phase 2 clinical trials.
According to Ocugen, the global prevalence of dAMD is 266 million, with GA affecting roughly 2-3 million people in the U.S. and Europe. Current treatments in the U.S. involve frequent injections and carry unwanted side effects. Outside the U.S., approximately 2 million patients in Europe lack approved treatment options.
Key findings from the Phase 2 trial include a 46% lesion growth reduction at 12 months in the medium and high dose groups versus the control (p=0.015; N=23). The medium dose achieved a 54% lesion reduction (p=0.02; N=10), while the high dose showed a 36% reduction (p=0.05; N=8) compared to the control. The responder rate was 50%, with patients achieving greater than 50% lesion size reduction compared to the control. A subgroup of patients (N=14) with larger lesions at baseline showed a 57% greater reduction in lesion size compared to the control.
New Phase 1 findings (N=9) indicate that in evaluable subjects (N=7), ellipsoid zone (EZ) loss was 60% slower in OCU410-treated eyes compared to untreated fellow eyes at 12 months. The company also noted that EZ-RPE complex loss was reduced in treated eyes versus fellow eyes, demonstrating photoreceptor and RPE preservation.
Ocugen reported no OCU410-related serious adverse events in either the Phase 1 or Phase 2 clinical trials. No cases of endophthalmitis, retinal detachment, vasculitis, choroidal neovascularization, or optic ischemic neuropathy have been reported.
Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen, stated that the Phase 1 and Phase 2 results mark a pivotal moment for Ocugen’s modifier gene therapy platform. Dr. Musunuri added that the company anticipates reporting full data from the OCU410 Phase 2 clinical trial later this quarter and initiating Phase 3 in 2026.
Dr. Huma Qamar, Chief Medical Officer of Ocugen, noted the Phase 2 trial delivered statistically significant efficacy across multiple analyses, with safety data showing no drug-related serious adverse events, inflammation signals, or injection complications.
Lejla Vajzovic, MD, FASRS, Director, Duke Surgical Vitreoretinal Fellowship Program, Professor of Ophthalmology with Tenure, Adult and Pediatric Vitreoretinal Surgery and Disease, Duke University Eye Center, and Retina Scientific Advisory Board Chair of Ocugen, believes OCU410 has the potential to set a new standard of care with a single treatment for life.
The Phase 2 study assessed the safety and efficacy of OCU410 in patients with GA secondary to dAMD, with 51 patients randomized into treatment groups (medium or high dose) or a control group. The company anticipates a Biologics License Application (BLA) filing for OCU410 in 2028.
OCU410 is an investigational, AAV5-based gene therapy that delivers RORA (retinoid-related orphan receptor alpha) and has received Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency.
dAMD affects approximately 10 million Americans and more than 266 million people worldwide.
Tiffany Hamilton
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com
Source: Ocugen, Inc.
