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

Core Viewpoint - Ocugen, Inc. has completed enrollment for the OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP), with topline data expected in Q1 2027, which may support a Biologics License Application (BLA) filing and potential approval in 2027 [1][5] Company Developments - The liMeliGhT clinical trial enrolled 140 patients, randomized into treatment and control groups, targeting early- to late-stage RP patients, including pediatric cases [2][3] - The primary endpoint of the trial is the 12-month change in visual function, assessed by a proprietary test called LDNA [2] - Ocugen is on track to file a rolling BLA in Q3 2026, following positive long-term data from a previous Phase 1/2 study showing approximately 2-line gains in visual acuity [4][5] Clinical Trial Insights - The Phase 3 trial is notable for its broad representation of gene mutations associated with RP, supporting a gene-agnostic therapeutic approach [3] - Positive long-term data from the Phase 1/2 study indicated that 88% of evaluable treated subjects showed improvement or preservation of visual function over three years [6] Treatment Landscape - Currently, there are no approved treatments that effectively address the needs of the approximately 98% of RP patients who are not candidates for existing gene therapies [4][9] - OCU400 aims to reset dysfunctional gene networks in retinal cells, potentially improving vision for patients with RP [7][9] Industry Context - RP is a group of rare genetic disorders leading to vision loss, associated with mutations in over 100 genes, highlighting the need for innovative, gene-agnostic therapies [8][9] - The development of novel therapeutic approaches targeting broader RP disease mechanisms offers hope for patients with multiple and unknown genetic mutations [9]