Ocugen, Inc announced positive preliminary 12-month data (~50% of patients evaluated to date) from the second stage of the phase 1/2 ArMaDa clinical trial evaluating OCU410 (AAV5-RORA), its novel modifier gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD).
In the phase 2 study, 51 patients were randomized 1:1:1 into either of 2 treatment groups (medium or high dose) or a control group. In the treatment groups, subjects received a single subretinal 200-µL administration of 5 x 1010 vector genomes (vg)/mL (medium dose) or 1.5 x 1011 vg/mL (high dose), while the control group remained untreated.
Key findings from the phase 2 trial, include a 46% lesion growth reduction (medium + high dose vs control; P=0.015; n=23) at 12 months; a medium dose that achieved 54% lesion reduction (P=0.02; n=10) vs high dose 36% (P=0.05; n=8) compared to control; and a 50% responder rate with patients achieving >50% lesion size reduction vs control, the company reported. In addition, the subgroup (n=14, subjects with ≥7.5 mm2 at baseline) showed 57% greater reduction in lesion size compared to control.
In new findings from the first stage of the trial, 7 evaluable subjects showed ellipsoid zone (EZ) loss was 60% slower in OCU410-treated eyes compared to untreated fellow eyes at 12 months. In addition, EZ-RPE complex loss reduced in treated eyes vs fellow eyes, demonstrating photoreceptor + RPE preservation, the company noted.
According to Ocugen, 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 in ArMaDa to date.
The company said it remains on track for a biologics license application filing for OCU410 in 2028.