Ocugen has initiated its global Phase III ArMaDa3 trial of OCU410, an investigational gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD), with the dosing of the first patient. The trial is a significant step in the development of a new treatment for GA, a condition that affects millions of people worldwide.
Trial Design and Objectives
The randomised, controlled, multi-centre study is being conducted across sites in Canada, Latin America, Europe, and the US, and will enrol 237 participants. OCU410 is designed to be administered as a single subretinal injection, delivering the human retinoid-related orphan receptor alpha gene using an AAV5 vector.
This approach differs from available treatments in the US, which are complement inhibitors administered through repeated eye injections and address individual disease pathways. The main goal of the trial is to measure the rate of change in the square root-transformed GA lesion area per year using fundus autofluorescence at several time points over 12 months.
Regulatory Background
The US Food and Drug Administration granted OCU410 regenerative medicine advanced therapy (RMAT) designation in July 2026, following Phase II data. The global Phase III study is randomising participants with GA due to dAMD in a 2:1 ratio to receive either OCU410 or no treatment.
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Secondary outcomes include the proportion of subjects experiencing significant loss in low-luminance visual acuity and the rate of change in ellipsoid zone area loss as measured by SD-OCT. The Phase III trial design aligns with guidance from the FDA, as confirmed in a Type B End-of-Phase II meeting in July 2026.
Ocugen chairman, CEO and co-founder Dr Shankar Musunuri said: “Dosing the first patient in our global Phase III trial, just weeks after receiving RMAT designation, marks a defining moment for the OCU410 programme—and for the millions of people living with geographic atrophy.”
Development and Registration
Ocugen anticipates submission of a biologics license application in 2028, and discussions are underway with the European Medicines Agency (EMA) for possible registration in Europe based on this trial. Support for advancement into Phase III is drawn from 12-month Phase II results showing a 31% reduction in lesion growth and no OCU410-related serious adverse events.
Earlier this year, Ocugen reported positive results for OCU410 in GA secondary to dAMD. The company’s progress with OCU410 is notable, given the limited treatment options currently available for GA. As the trial progresses, it will be important to monitor the safety and efficacy of OCU410 in a larger patient population.
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In the context of other gene therapies, OCU410’s approach is distinct in its use of a single subretinal injection to target multiple disease pathways. This could potentially offer advantages in terms of convenience and reduced risk of adverse events compared to repeated injections. However, the results of the Phase III trial will determine the therapy’s overall benefit and risk profile.
Ocugen’s initiation of the Phase III trial is a significant milestone. The company will likely be closely watched as it moves forward with the development of OCU410. With the trial expected to enrol 237 participants, the results will provide valuable insights into the therapy’s potential to address the needs of patients with GA secondary to dAMD.
Dr Shankar Musunuri’s statement highlights the importance of this trial. The trial’s outcome will have a significant impact on the development of OCU410.
It is a new treatment.
