• Optimal dose intended for Phase 3 demonstrates statistically significant reduction in lesion growth (31%) versus control at 12 months (p< 0.05)
  • Potential 2X treatment benefit compared to 15% and 22% reductions reported for currently approved therapies at 12 and 24 months, respectively
  • No serious adverse events and no adverse events of special interest related to OCU410 reported to date

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
  • 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 Phase 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.

GA is a multifactorial disease with a complex etiology that involves genetic and environmental factors. The current treatment options for GA in the U.S. 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 multi-pathway 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 Co-founder 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 demonstrates statistically significant reduction of GA lesion growth after treatment with OCU410 optimal dose, 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%.​"

"There remains a considerable unmet need in treating patients with GA and I am encouraged by the various analyses of the Phase 2 OCU410 data," said Lejla Vajzovic, MD, FASRS, Director, Duke Surgical Vitreoretinal Fellowship Program, Associate Professor of Ophthalmology with Tenure, Adult and Pediatric Vitreoretinal Surgery and Disease, Duke University Eye Center, and Chair, Ocugen Retina Scientific Advisory Board. "In addition to the strong efficacy and safety data, OCU410 has the potential to eliminate the chronic treatment burden associated with monthly or every-other-month intravitreal injections and to reduce treatment attrition driven by patient fatigue.​"

In the Phase 2 study, the safety and efficacy of OCU410 in patients with GA secondary to dAMD are being assessed. Fifty-one (51) patients aged 50 years and older with GA lesions within the foveal or non-foveal 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 three-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 three BLA filings in three years.