Ocugen Announces Phase 3 liMeliGhT Enrollment Completion for OCU400, a Novel Modifier Gene Therapy for Broad Retinitis Pigmentosa

• Enrollment for liMeliGhT, the first and largest gene therapy registrational trial for broad retinitis pigmentosa (RP) patients, was completed, reflecting strong interest from investigators and patients
• Topline Phase 3 data expected in 1Q 2027, advancing OCU400 towards potential approval in 2027 as a treatment option for early- to late-stage RP
• Positive long-term, 3-year Phase 1/2 durable, safety and tolerability data for OCU400 demonstrates sustained clinically meaningful, approximately 2-line LLVA gain, reinforcing durable gene-agnostic benefit

Excerpt from the Press Release:

MALVERN, Pa., March 02, 2026 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that enrollment is now complete for the OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP). As a one-year clinical trial, topline data will be available in the first quarter of 2027. These data are anticipated to support the Biologics License Application (BLA) filing for OCU400 and potential approval in 2027. The European Medicines Agency (EMA) has also provided acceptability of the U.S.-based trial for submission of a Marketing Authorization Application (MAA).

“With enrollment complete for OCU400, we enter into a very significant time as a Company,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “This milestone brings us even closer to potentially delivering our first novel modifier gene therapy candidate to market and providing a one-time treatment for life to hundreds of thousands of RP patients across the globe with unmet medical need.”

The liMeliGhT clinical trial enrolled 140 patients who were randomized 2:1 into the treatment group (2.5×¹⁰ vg per eye 250 µL) and untreated control group across mutations (RHO and gene-agnostic arms). The target population included patients with early- to late-stage disease among a broad RP population, including pediatrics (3+ years).

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