Ocugen Completes Enrollment in Phase 3 Gene Therapy Trial for Retinitis Pigmentosa
Ocugen has completed enrollment of 140 patients in its Phase 3 liMeliGhT trial for OCU400, a gene therapy for retinitis pigmentosa. Topline data expected in Q1 2027 could support potential approval later that year.
Ocugen, Inc. (NASDAQ: OCGN) 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 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). The primary endpoint is 12-month change in visual function assessed by LDNA (luminance dependent navigation assessment) with improvement in Lux Level from baseline to 12 months. LDNA is a more sensitive and specific mobility test, proprietary to Ocugen.
The OCU400 Phase 3 liMeliGhT clinical trial is the only broad RP gene-agnostic trial and the largest known Phase 3 orphan gene therapy trial. The European Medicines Agency (EMA) has also provided acceptability of the U.S.-based trial for submission of a Marketing Authorization Application (MAA).
Positive long-term, 3-year Phase 1/2 data for OCU400 was recently assessed in evaluable subjects and builds on prior 2-year results showing consistent clinically meaningful, approximately 2-line LLVA gain across mutations. OCU400 maintained a favorable durability, safety and tolerability profile with no new treatment-related serious adverse events or adverse events of interest emerged.
Visual function benefits were consistently observed over 3 years, with 88% (7/8) of evaluable treated subjects showing improvement or preservation versus untreated fellow eyes. Approximately 2-line gain (N=8) observed across multiple mutation types in treated eyes compared to untreated eyes at 3 years.
Ocugen remains on track to file the rolling BLA in the third quarter of 2026. OCU400 is the Company's modifier gene therapy candidate based on a nuclear hormone receptor gene called NR2E3. This gene regulates diverse physiological functions within the retina, such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation, and cell survival.
RP is a group of rare genetic disorders that cause a breakdown in the cells of the retina, leading to vision loss and blindness. RP is associated with mutations in more than 100 genes. There are no approved treatment options that slow or stop the progression of multiple forms of RP.