Ocugen Completes 140-Patient Phase 3 RP Gene Therapy Enrollment; 2-Line Vision Gain Data

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Ocugen completed enrollment of 140 patients in its Phase 3 liMeliGhT trial, randomizing subjects 2:1 to assess 12-month visual function. Three-year Phase 1/2 data showed approximately two-line LLVA gains in 88% of treated eyes, supporting a potential BLA filing and 2027 approval timeline.

1. Phase 3 Enrollment Completion

Ocugen finalized enrollment of 140 retinitis pigmentosa patients in the liMeliGhT Phase 3 trial, randomizing them 2:1 to receive OCU400 (2.5×10^10 vg per eye) or control. The primary endpoint is a 12-month change in luminance dependent navigation assessment (LDNA) to measure visual function improvements.

2. Durable Phase 1/2 Efficacy Data

Long-term results from the three-year Phase 1/2 study showed sustained clinically meaningful gains of approximately two lines in low-luminance visual acuity for 88% of treated eyes, with no new treatment-related serious adverse events or tolerability issues.

3. Regulatory Timeline and Approval Path

Topline data from the Phase 3 trial are expected in the first quarter of 2027, with Ocugen targeting a rolling BLA submission in the third quarter of 2026. The EMA has accepted the U.S.-based data for a future MAA, positioning OCU400 for potential approval in 2027.

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