Ocugen, Inc. Announces First Patient Dosed in Phase 3 liMeliGhT Clinical Trial for OCU400—First Gene Therapy in Phase 3 with a Broad Retinitis Pigmentosa Indication

Core Viewpoint - Ocugen, Inc. has initiated the Phase 3 liMeliGhT clinical trial for OCU400, a gene therapy product aimed at treating retinitis pigmentosa (RP), with the first patient dosed, marking a significant milestone in the development of potential treatments for this condition [3][11]. Company Overview - Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines to improve health and offer hope to patients globally [9]. - The company is advancing research in various areas, including retinal diseases, infectious diseases, and orthopedic diseases, with a unique modifier gene therapy platform that has the potential to treat multiple retinal diseases with a single product [9]. Clinical Trial Details - The Phase 3 liMeliGhT study will evaluate the efficacy of OCU400, focusing on the proportion of responders achieving an improvement of at least 2 Lux levels from baseline in treated and untreated groups [1]. - The study will include 150 participants, divided into two arms: one with RHO gene mutations and the other gene-agnostic, with a randomization of 2:1 to treatment and control groups [11]. - The trial duration is set for one year, targeting patients aged eight years and older with varying stages of RP [11]. Previous Clinical Data - Positive results from Phase 1/2 trials indicated that 89% of RP subjects showed preservation or improvement in visual acuity or mobility testing scores [5]. - Specifically, 80% of subjects with RHO mutations experienced preservation or improvement in mobility testing scores [5]. Therapeutic Potential - OCU400 is positioned as a novel modifier gene therapy that could significantly change the treatment landscape for RP, providing long-term benefits to patients who currently have no therapeutic options [4]. - The therapy aims to restore homeostasis in the retina, potentially improving retinal health and function for patients with RP and other retinal diseases [7][8]. Regulatory Status - OCU400 has received orphan drug and RMAT designations from the FDA, and the EMA has accepted the U.S.-based trial for submission of a Marketing Authorization Application (MAA) [6].