Ocugen advances phase 2/3 Stargardt disease trial for modifier gene therapy

Ocugen has aligned with the U.S. FDA on a plan to move forward with a phase 2/3 pivotal confirmatory clinical trial for the company’ modifier gene therapy in Stargardt disease, representing the first time this therapeutic approach has been explored in clinical trials for the genetic eye disorder.

The Pennsylvania-based biotech plans to use the results of the trial for OCU410ST, if positive, as the basis of its BLA submission, anticipated by 2027.

Stargardt disease, the most common form of inherited macular degeneration, causes retinal degeneration and progressive vision loss. OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR-Related Orphan Receptor A) gene. It utilizes 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.

In the phase 1/2 GARDian trial for Stargardt disease, OCU410ST demonstrated a favorable safety and tolerability profile with no serious adverse events related to the treatment. At a six-month follow-up, patients reported considerably slower lesion growth (52%) from baseline in treated eyes versus untreated.

The upcoming phase 2/3 clinical trial will randomize 51 subjects, 34 of whom will receive a single, subretinal, 200-μL injection of OCU410ST in the eye with worse visual acuity, and 17 of whom will serve as untreated controls. The primary endpoint in the clinical trial is change in atrophic lesion size. Secondary endpoints include visual acuity as measured by best corrected visual acuity and low luminance visual acuity compared to untreated controls. One-year data will be utilized for the BLA filing.