Eugenia C. Greig, MD
Byers Eye Institute
Stanford
Late Breaking Presentation: Safety and Efficacy of Subretinal OCU410 Gene Therapy for Geographic Atrophy: 12-Month Randomized Phase 2 ArMaDa Results
Thursday afternoon at the Retina Society included a Late Breaking presentation by Dr. Raj Maturi, who presented 12-month data for Ocugen’s Phase 2 ArMaDa trial of subretinal OCU410 gene therapy in geographic atrophy (GA).
OCU410 acts on a nuclear hormone receptor pathway that upregulates complement regulator CD59, reducing membrane attack complex formation, while also dampening lipid dysregulation and broader inflammation. The therapy is delivered subretinally at 3 to 4 sites surrounding the GA lesion, sparing the optic nerve and fovea, so that drug is absorbed locally where it can contain lesion growth.
ArMaDa was a randomized trial comparing medium- and high-dose arms with an untreated control group (which received no surgery). The low-dose arm was dropped after Phase 1. Safety was the primary endpoint for the trial. Each cohort enrolled 17 subjects, and eligibility was broad, permitting lesions up to 8-disc areas in size. Four subjects were lost to follow-up after randomization.
A single subretinal administration of OCU410 was well tolerated, and there were no treatment-related serious adverse events (SAEs). Choroidal neovascularization occurred in one or two subjects per cohort, including controls, suggesting this reflected underlying disease rather than drug effect.
At 12 months, the medium dose produced the clearest efficacy signal, showing a 34% reduction in lesion growth vs control and a 27% reduction in ellipsoid zone (EZ) loss vs control. Dr. Maturi emphasized the EZ data, as these findings suggest that OCU410 might aid in photoreceptor preservation rather than only protecting the retinal pigment epithelium. The high dose group performed comparably.
A Phase 3 trial is currently underway. If the Phase 3 results hold, OCU410 could offer a one-time surgical alternative to the monthly or every-other-month intravitreal injections currently approved to slow GA progression.
