Ocugen, Inc. announced positive preliminary 12-month data (50% of patients evaluated to date) from the Phase 2 ArMaDa clinical trial evaluating OCU410 (AAV5-RORA), its novel modifier gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). The global prevalence of dAMD is 266 million worldwide, and GA - the late stage of dAMD - affects approximately 2-3 million people in the United States (U.S.) and Europe. There are limited options for patients with dAMD in the U.S. and current therapies involve frequent (monthly or every other month) injections and have unwanted side effects that can affect vision.
Outside of the U.S., there are no approved products available, leaving approximately 2 million patients in Europe without a treatment option. Key findings from Phase 2 include: 46% lesion growth reduction (medium + high dose vs. control; p=0.015; N=23) at 12 months; Medium dose achieved 54% lesion reduction (p=0.02; N=10) vs.
high dose 36% (p=0.05; N=8) compared to control; 50% responder rate with patients achieving >50% lesion size reduction vs. control; Subgroup (N=14, subjects with 7.5 mm2 at baseline) showed 57% greater reduction in lesion size compared to control. New findings from Phase 1 (N=9) include: In evaluable subjects (N=7) ellipsoid zone (EZ) loss was 60% slower in OCU410-treated eyes compared to untreated fellow eyes at 12 months; EZ-RPE complex loss reduced in treated eyes versus fellow eyes, demonstrating photoreceptor + RPE preservation.
In both the Phase 1 and Phase 2 clinical trials no OCU410-related serious adverse events were observed and no cases of endophthalmitis, retinal detachment, vasculitis, choroidal neovascularization, or optic ischemic neuropathy have been reported to date. GA is a multifactorial disease with a complex etiology that involves genetic and environmental factors.
