Ocugen Inc. reported 12-month data from its Phase 2 ArMaDa study of OCU410 modifier gene therapy for geographic atrophy secondary to dry age-related macular degeneration. The company said the trial's optimal dose achieved a statistically significant 31% reduction in lesion growth at the one-year mark compared with the control group.
In addition to the lesion size outcome, Ocugen highlighted structural benefits on retinal imaging. The trial showed a 27% slower rate of ellipsoid zone loss in treated eyes versus control, a finding the company presented as evidence of photoreceptor preservation.
The randomized Phase 2 study enrolled 51 patients aged 50 years and older who had geographic atrophy lesions. Treatment arms included a medium dose of 1 x 1010 vector genomes per eye and a high dose of 3 x 1010 vector genomes per eye, while the control group received no treatment.
On safety, Ocugen reported no serious adverse events and no adverse events of special interest attributed to OCU410 to date. The company specifically noted there were no instances of endophthalmitis, retinal detachment, vasculitis, choroidal neovascularization, or ischemic optic neuropathy in the study population.
The trial's primary endpoint was change in geographic atrophy lesion size at 12 months, measured in square millimeters by fundus autofluorescence imaging. Ocugen reported that 55% of treated patients achieved at least a 30% reduction in lesion size relative to control.
Following the announcement of the 12-month results, Ocugen shares declined 5% on Tuesday. The company said it plans to initiate a Phase 3 registrational trial for OCU410 in the third quarter of 2026 and stated an objective of submitting three biologics license applications within a three-year period.
Ocugen provided a prevalence estimate for geographic atrophy, stating that the condition affects approximately 2 to 3 million people across the United States and Europe. The company also noted that current therapeutic options typically require 6 to 12 injections per year on an ongoing basis.
These results represent a clinical readout that the company is using to support a registrational pathway. The program's next milestone is the planned Phase 3 start in Q3 2026, which will determine whether the observed Phase 2 effects can be confirmed in a larger, registrational population.