Arun Upadhyay, PhD, on Results from IRD Gene Therapy OCU400’s Phase 1/2 Trial

“I would like to highlight that currently we are enrolling patients in our OCU400 phase 3 study... It is for a broader indication, and this is the first of its own kind of trial in this space. No other company to our knowledge is running a broader RP designation trial.”

Currently, substantial unmet need remains for patients with inherited retinal diseases (IRDs) such as retinitis pigmentosa (RP). Because mutations in a wide range of genes can cause IRDs like RP, developing targeted gene therapy products with the traditional “1 therapy for 1 gene” approach is impractical. As such, Ocugen has decided upon pursuing a gene-agnostic approach to treating IRDs with its Modifier Gene Therapy Platform. OCU400, a gene therapy product based on this platform, is currently being evaluated in a phase 1/2 clinical trial (NCT05203939) for the treatment of RP and Leber congenital amaurosis (LCA), as well as a more recently-initiated phase 3 clinical trial (liMeliGhT; NCT06388200), which dosed its first patient in June of this year. Notably, data from the earlier phase 1/2 trial was recently presented at the 24th EURETINA Congress held September 19 to 22, 2024, in Barcelona, Spain.

Shortly after the conference, CGTLive® spoke with Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen, to learn more about the updated data. Upadhyay pointed out that across 18 patients treated in the study, OCU400 was generally safe and well-tolerated. In terms of efficacy, he emphasized that close to 60% of the intent-to-treat population responded to OCU400 with regard to a luminance dependent navigation assessment, which constitutes a primary end point in the phase 3 trial. Upadhyay also noted that OCU400 has received orphan drug designation from both the FDA and the European Medicines Agency for RP and LCA, and that it has received regenerative medicines advanced therapy designation from the FDA, as well as an expanded access protocol from the FDA in RP.

REFERENCE
1. 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. June 20, 2024. Accessed September 30, 2024. https://www.globenewswire.com/news-release/2024/06/20/2901513/0/en/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.html