Ocugen announced positive 12-month data from the phase 2 ArMaDa trial evaluating OCU410 (AAV5-RORA), its modifier gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). 

Key findings from Phase 2 include:

  • 31% reduction in lesion growth in the optimal dose (medium) group compared to control (P < 0.05);
  • 27% slower rate of ellipsoid zone (EZ) loss compared to control, indicating structural preservation of photoreceptors, which correlates with visual function;
  • 55% of treated patients demonstrated ≥30% lesion size reduction vs. control; and
  • Subgroup analysis (subjects with baseline GA lesions ≥5 mm2 and ≤17.5 mm2) showed 33% reduction in lesion growth compared to control in medium dose OCU410 with similar reductions in the high dose group

The phase 2 clinical trial builds directly on the clean safety profile observed in hase 1 with no OCU410-related serious adverse events observed and no cases of endophthalmitis, retinal detachment, vasculitis, choroidal neovascularization, or ischemic optic neuropathy reported to date. The current treatment options for GA in the United States are limited to those targeting a single mechanism—the complement pathway. By contrast, OCU410 is a first-in-class RORA-based gene therapy designed to support central retina and photoreceptor integrity through a multipathway mechanism—targeting drusen, inflammation, oxidative stress, and complement activation.​

“We have confirmed robust treatment effect from a well-controlled phase 2 trial of a genetic medicine for GA. Now we can move on to phase 3 with a high degree of confidence,” said Dr. Shankar Musunuri, Chairman, CEO, and Cofounder of Ocugen. “This moves us one step closer to bringing a transformative one-time treatment to GA patients globally who are desperately seeking rescue from vision loss.”

“Our phase 2 data consistently demonstrate statistically significant reduction of GA lesion growth after treatment with OCU410, and we continue to benchmark these results against natural history data to contextualize the magnitude of effect,” said Huma Qamar, MD, MPH, CMI, chief medical officer of Ocugen. “We are incorporating these learnings into an anticipated phase 3 pivotal confirmatory trial with up to 300 subjects and an adaptive design powered at over 95%.​”

In the phase 2 study, the safety and efficacy of OCU410 in patients with GA secondary to dry AMD are being assessed. Fifty-one patients aged 50 years and older with GA lesions within the foveal or nonfoveal region were randomized 1:1:1 to receive a single subretinal administration of OCU410 at a medium dose of 1 × 1010 vector genomes per eye, a high dose of 3 × 1010 vector genomes per eye, or no treatment in the control group; each injection volume was 200 microliters. Of note, choroidal neovascularization in the fellow eye was not exclusionary, and patients with prior exposure to pegcetacoplan or avacincaptad pegol were eligible following a 3-month washout.

The primary endpoint was change in GA lesion size at 12 months, measured in square millimeters by fundus autofluorescence, an FDA-accepted structural endpoint used in recent GA registration trials. Exploratory endpoints included EZ preservation on OCT, a key biomarker for photoreceptor integrity, which correlates with visual function.

Ocugen plans to initiate the OCU410 phase 3 registrational trial in the third quarter of 2026 in line with the company’s goal of 3 BLA filings in 3 years.