MALVERN, Pa., Sept. 1, 2026

Ocugen, Inc. has announced that the first patient has been dosed in the global Phase 3 registrational trial of OCU410, its investigational modifier gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). The milestone marks the transition of OCU410 into pivotal-stage clinical development and follows recent regulatory alignment with the U.S. Food and Drug Administration (FDA). The single global Phase 3 study, known as ArMaDa3, is designed to evaluate OCU410 as a potential one-time subretinal gene therapy for GA. Ocugen said the Phase 3 program is fully aligned with FDA feedback following a Type B End-of-Phase 2 meeting completed in July 2026. The company anticipates that the pivotal study could support a Biologics License Application (BLA) filing in 2028.

Global Phase 3 Study Targets Geographic Atrophy

Ocugen’s ArMaDa3 Phase 3 trial is a global, multicenter, randomized and controlled study planned to enroll 237 patients with GA secondary to dAMD. Participants will be randomized in a 2:1 ratio to receive either a single subretinal injection of OCU410 or an untreated control. The trial is being conducted across sites in the United States, Canada, Europe and Latin America, expanding the geographic reach of the program as Ocugen moves toward a potential registration package. The primary endpoint will assess the rate of change in GA lesion area using fundus autofluorescence measurements through Month 12. Secondary endpoints will evaluate functional vision through low-luminance visual acuity and structural retinal changes involving the ellipsoid zone. Ocugen said the trial design, including its endpoints, dose and adaptive elements, was aligned with FDA during the company’s End-of-Phase 2 discussions, providing a defined clinical development pathway for the pivotal program.

OCU410 Receives FDA RMAT Designation

The Phase 3 launch follows the FDA’s Regenerative Medicine Advanced Therapy (RMAT) designation for OCU410, granted on July 29, 2026. The designation provides opportunities for enhanced interaction with the FDA during development and can provide eligibility for accelerated approval and priority review when applicable requirements are met. Ocugen said the designation was supported by Phase 2 clinical evidence indicating clinically meaningful efficacy and a favorable safety profile. The regulatory designation is particularly relevant to the company’s development strategy because OCU410 uses a modifier gene therapy approach intended to influence multiple biological pathways associated with GA. Ocugen is positioning the program as a potential alternative to treatments requiring repeated administration, although the Phase 3 trial will ultimately need to establish the therapy’s efficacy and safety to support regulatory approval.

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Phase 2 Data Support OCU410 Development

Ocugen’s Phase 3 program is supported by 12-month results from the Phase 2 ArMaDa study, which enrolled 51 patients with GA secondary to dAMD. In the medium-dose group, the company reported a 31% reduction in GA lesion growth rate compared with control at 12 months, with the difference reaching statistical significance. The company also reported a 27% reduction in ellipsoid zone area loss, while approximately 20% of treated subjects in the medium-dose group showed no disease progression and 75% demonstrated more than a 30% reduction in lesion growth. Ocugen reported no OCU410-related serious adverse events or adverse events of special interest to date. OCU410 delivers the human RORA gene through an AAV5 vector in a single subretinal injection and is designed to influence complement activation, neuroinflammation, oxidative stress and lipid metabolism. The company is now advancing the program through the global pivotal trial, with a potential BLA submission targeted for 2028.

Source: Ocugen, press relese