Ocugen, Inc. Starts Phase 3 Dosing for OCU410; DMC Recommends Continue

What Happened

• Ocugen, Inc. announced that the first patient was dosed on September 1, 2026 in the global Phase 3 registrational trial of OCU410 (AAV5-hRORA) for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD).
• The dosing follows a July 2026 Type B End-of-Phase 2 meeting with the FDA’s CBER where the agency and Ocugen aligned on key Phase 3 design elements (endpoints, dose, adaptive design, and a single pivotal trial pathway to support a BLA). The FDA also granted OCU410 Regenerative Medicine Advanced Therapy (RMAT) designation in July 2026.
• On September 3, 2026, the independent Data Monitoring Committee (DMC) completed a pre-specified interim analysis of 26 subjects (16 treated, 10 control) and recommended continuing the study per protocol to obtain 8-month follow-up for the entire study population. The DMC noted the interim sample showed a negative direction of treatment effect and a baseline lesion size imbalance, and therefore recommended collecting the full 8-month dataset before further judgment.

Key Details

• First patient dosed: September 1, 2026.
• FDA interactions: Type B EOP2 meeting and RMAT designation both in July 2026.
• Interim analysis population: 26 subjects (16 treated, 10 control); DMC reviewed atrophic lesion size at ~8 months.
• DMC action: Recommended modification to continue per protocol to gather 8-month follow-up for the entire trial population; Ocugen intends to follow this recommendation.

Why It Matters

• The Phase 3 dosing start is a major clinical milestone for Ocugen’s lead modifier gene therapy OCU410 and advances the registrational program toward a potential Biologics License Application (BLA).
• RMAT designation and FDA alignment on trial design may facilitate regulatory interactions, though designation does not guarantee approval.
• The DMC’s recommendation to continue (despite a negative direction in a small interim sample) preserves the trial’s ability to produce a fuller, less-biased 8-month dataset, which investors should watch for future readouts.
• Ocugen attached a company presentation (Exhibit 99.1) it will post to its website on September 8, 2026 with additional information.