Ocugen Completes Dosing in Phase 1/2 ArMaDa Trial of OCU410 for Geographic Atrophy Due to dAMD

Ocugen, Inc., a biotechnology firm specializing in innovative gene and cell therapies and vaccines, has announced the completion of dosing in the third cohort of its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA). This therapy is being developed to treat geographic atrophy (GA), an advanced form of dry age-related macular degeneration (dAMD), which affects about a million individuals in the U.S.

GA is primarily addressed by two FDA-approved anti-complement therapies, which target a single pathway in a multifactorial disease. Dr. Majda Hadziahmetovic, Associate Professor of Ophthalmology at Duke University Eye Center and the trial's lead investigator, indicated that current treatments, though significant, offer limited benefits and require continuous intravitreal injections over several years. The potential for a transformative approach to GA treatment through modifier gene therapy was emphasized.

In the trial, three subjects received a single subretinal administration of the highest tested dose (200 µL of 1.5x10^11 vg/mL), marking the completion of the Phase 1 stage. The ArMaDa clinical trial for OCU410 is being conducted at 14 prominent retinal surgery centers across the U.S.

Dr. Huma Qamar, Chief Medical Officer at Ocugen, expressed optimism about OCU410's potential as a one-time treatment for GA. Ocugen has now initiated a Phase 2 clinical trial to evaluate the safety and efficacy of OCU410 in a larger patient group. Participants will be randomized into two treatment groups (medium- or high-dose) or a control group. Ocugen plans to provide regular clinical updates.

The Phase 1/2 ArMaDa clinical trial focuses on the safety of unilateral subretinal administration of OCU410 in GA patients. The trial is divided into two phases: Phase 1, a multicenter, open-label, dose-escalation study with three dose levels (low, medium, and high), and Phase 2, a randomized, outcome assessor-blinded, dose-expansion study. In Phase 2, subjects will be randomized in a 1:1:1 ratio to one of the two OCU410 treatment groups or a control group.

Dry age-related macular degeneration (dAMD) affects around 10 million Americans and over 266 million people globally. It is marked by the thinning of the macula, leading to central vision impairment. dAMD results in the gradual deterioration of the retina with submacular drusen, atrophy, and loss of macular function. It accounts for 85-90% of all age-related macular degeneration cases.

OCU410 leverages an adeno-associated virus (AAV) platform to deliver the RORA gene to the retina. The RORA protein is crucial in lipid metabolism, reducing lipofuscin deposits and oxidative stress. It also shows anti-inflammatory properties and inhibits the complement system in both in vitro and animal model studies. These findings highlight OCU410's capacity to target multiple pathways involved in dAMD pathophysiology. Ocugen aims to develop AAV-RORA as a one-time gene therapy for GA treatment.

Ocugen, Inc. is committed to discovering, developing, and commercializing novel gene and cell therapies and vaccines to improve health and offer hope to patients worldwide. The company’s innovative approaches aim to treat multiple retinal diseases with a single product and advance research in infectious and orthopedic diseases to address unmet medical needs.