Ocugen reported positive 12-month results from its phase 2 ArMaDa trial evaluating OCU410 (AAV5-RORA), an investigational gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). In the medium-dose group, lesion growth was reduced by 31% compared with untreated controls (P<.05). Structural analysis also showed a 27% slower rate of ellipsoid zone loss, a biomarker for photoreceptor integrity.
OCU410 is a first-in-class therapy that delivers the RORA (retinoid related orphan receptor alpha) gene to the retina via a single subretinal injection. The expressed RORA protein is intended to support central retina and photoreceptor integrity by modulating multiple biological pathways implicated in GA, including lipid metabolism, oxidative stress, inflammation, and complement activation.
The therapy was well tolerated in ArMaDa, with no serious adverse events or ocular complications related to OCU410. More than half of treated patients demonstrated at least a 30% reduction in lesion size. Subgroup analysis of patients with baseline lesions between 5 mm2 and 17.5 mm2 showed similar reductions.
In a press release, Ocugen reported plans to launch a phase 3 registrational trial in the third quarter of 2026, anticipating enrollment of up to 300 patients.