Ocugen, Inc. announced that the first patient has been dosed in its Phase 2/3 GARDian3 clinical trial for OCU410ST (AAV5-hRORA)--a modifier gene therapy candidate being developed for all Stargardt disease (ABCA4-associated retinopathies). The Phase 2/3 clinical trial for OCU 410ST builds upon encouraging results and positive data from the Phase 1 GARDian trial, which demonstrated 48% slower lesion growth at 12-month follow up in evaluable treated eyes compared to untreated eyes. Additionally, evaluable treated eyes showed a statistically significant (p=0.031) and clinically meaningful improvement of nearly 2-line gain in best corrected visual acuity (BCVA) at 12-month follow-up when compared to untreated eyes.
Key secondary endpoints include improvements inBCVA and low luminance visual acuity (LLVA), compared to controls. Data from the one-year follow-up will be used to support the company's planned Biologics License Application (BLA). The OCU410ST Phase 2/3 pivotal confirmatory trial represents Ocugen's second late-stage clinical program.
Ocugen plans to submit a BLA for OCU410ST in 2027 in alignment with its strategic goal of filing three BLAs over the next three years. OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR-Related Orphan Receptor A) gene. It represents Ocugen's modifier gene therapy approach, which is based on Nuclear Hormone Receptor (NHR) RORA that regulates pathophysiological pathways linked to Stargardt disease, such as lipofuscin formation,oxidative stress, complement formation, inflammation, and cell survival networks.
The breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through gene-agnostic approach.
