Ocugen announces positive 2-year safety and efficacy update of Phase 1/2 OCU400 for treatment of retinitis pigmentosa

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Key Takeaways

OCU400 showed significant 2-line gain in LLVA in treated eyes, validating its gene-agnostic mechanism for retinitis pigmentosa.
The therapy's effectiveness across multiple mutations highlights its potential for broader retinal disease applications.
Long-term safety and efficacy of OCU400 are consistent with earlier findings, demonstrating its durability.
The Phase 3 OCU400 liMeliGhT trial is progressing towards a BLA submission in the first half of 2026.

OCU400 demonstrated meaningful improvement of 2-line gain (10 letters on ETDRS chart) in low-luminance visual acuity (LLVA) in treated eyes when compared to untreated fellow eyes.

(Image Credit: AdobeStock/pakbung)

Ocugen, Inc. announced the positive 2-year safety and efficacy update for the Phase 1/2 OCU400 clinical trial for the treatment of early to advanced retinitis pigmentosa (RP) in pediatric and adult populations.

According to the company, OCU400 demonstrated meaningful improvement of 2-line gain (10 letters on ETDRS chart) in low-luminance visual acuity (LLVA) in treated eyes when compared to untreated fellow eyes. This was statistically significant (p=0.01) in all subjects, regardless of mutation at 2 years, validating the gene-agnostic mechanism of action for OCU400.¹

Syed M. Shah, MD, FACS, Vice Chair for Research and Digital Medicine, Director of Retina Service at Gundersen Health System, La Crosse, Wisconsin is quoted in the press release¹, as follows, “The broad spectrum of genes and mutations causing RP presents a unique challenge in developing treatments for this unmet need. This is where the promise of mutation-agnostic therapies becomes particularly compelling. OCU400’s demonstrated effectiveness across multiple mutations not only offers hope to RP patients but also opens new possibilities for treating other retinal diseases."

Huma Qamar, MD, MPH, Chief Medical Officer at Ocugen spoke to the long-term potential of this treatments, saying, “Establishing the long-term safety and efficacy of OCU400 demonstrates the durability of this novel gene therapy. These 2-year low light visual acuity (LLVA) findings, which are the most sensitive measure of visual function, are consistent with the results observed at one year.”¹

The Phase 3 OCU400 liMeliGhT clinical trial is ongoing and on target for BLA submission in the first half of 2026.¹

Reference:

Ocugen, Inc. Announces Positive 2-Year Data Across Multiple Mutations from Phase 1/2 Clinical Trial of OCU400 —A Novel Modifier Gene Therapy for Retinitis Pigmentosa. Ocugen, Inc. January 13, 2025. Accessed January 13, 2025. https://www.globenewswire.com/news-release/2025/01/13/3008252/0/en/Ocugen-Inc-Announces-Positive-2-Year-Data-Across-Multiple-Mutations-from-Phase-1-2-Clinical-Trial-of-OCU400-A-Novel-Modifier-Gene-Therapy-for-Retinitis-Pigmentosa.html