Ocugen announced that enrollment for its Phase 3 liMeliGhT trial of OCU400, a modifier gene‑therapy candidate for retinitis pigmentosa (RP), is now complete. The study enrolled 140 patients and randomized them 2:1 to receive a 2.5×10^10 viral genomes per eye dose or an untreated control, covering a broad spectrum of RP mutations—including a dedicated RHO arm and a gene‑agnostic cohort.
The trial is a one‑year, randomized, controlled study designed to evaluate the safety and efficacy of OCU400 across the widest possible RP patient population. By enrolling 140 participants, the company has reached the majority of its target enrollment, positioning the study to deliver topline data in the first quarter of 2027. The gene‑agnostic approach allows OCU400 to potentially treat 98‑99 % of RP patients, a group largely excluded from current gene‑specific therapies.
Ocugen plans to file a rolling Biologics License Application (BLA) for OCU400 in 2026, with the expectation that regulatory approval could follow the Q1 2027 data readout. The company estimates the addressable market for a single‑dose therapy to exceed $2 billion, reflecting the large unmet need among RP patients who are not candidates for existing gene‑replacement products.
"With enrollment complete for OCU400, we enter into a very significant time as a Company. 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," said Dr. Shankar Musunuri, Chairman, CEO, and Co‑founder of Ocugen. Dr. Huma Qamar, Chief Medical Officer, added, "The Phase 3 liMeliGhT clinical trial includes representation of a wide range of gene mutations associated with early to advanced stages of RP and we believe the patient response will support the gene‑agnostic mechanism of action of our novel modifier gene therapy platform."
Ocugen also has an expanded access program approved by the FDA in August 2024, allowing eligible patients to receive OCU400 before regulatory approval. In addition to OCU400, the company is advancing OCU410 for geographic atrophy and OCU410ST for Stargardt disease, underscoring its broader modifier‑gene‑therapy platform. The completion of enrollment for OCU400 de‑risks the program’s clinical trajectory, strengthens the company’s regulatory strategy, and could unlock a substantial market opportunity for a single‑dose therapy that addresses the majority of RP patients.
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