Ocugen, a gene therapy company, received FDA clearance to begin a Phase 2/3 trial for OCU410ST, a modifier gene therapy for Stargardt disease (ABCA4-associated retinopathies). The FDA previously granted OCU410ST Rare Pediatric Disease and Orphan Drug Designations for treating ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3. This pivotal trial follows positive Phase 1 results demonstrating safety, slower lesion growth, and improved visual function.
This advancement is crucial because Stargardt disease, the most common inherited macular degeneration, affects a significant population (approximately 100,000 in the U.S. and Europe and 1 million globally) with limited treatment options. The genetic complexity of the disease, with over 1,200 known mutations, makes traditional gene therapy or editing approaches challenging, highlighting the need for innovative solutions like OCU410ST. The FDA’s decision to clear the IND amendment expedites the clinical development timeline, potentially bringing a much-needed therapy to patients sooner.
The Phase 2/3 trial will involve 51 participants with Stargardt disease. Thirty-four participants will receive a single subretinal injection of OCU410ST, and 17 will be in an untreated control group. Researchers will primarily evaluate the reduction in atrophic lesion size, with secondary endpoints including improvements in best corrected and low luminance visual acuity. One-year follow-up data will support the company’s planned Biologics License Application (BLA) in 2027. This trial is Ocugen’s second late-stage clinical program, aligning with their goal of submitting three BLAs within the next three years.
OCU410ST delivers the RORA gene via an AAV vector to the retina. This modifier gene therapy approach targets the RORA gene, a Nuclear Hormone Receptor regulating pathways linked to Stargardt disease, including lipofuscin formation, oxidative stress, and cell survival. The positive Phase 1 data and the FDA’s clearance for the pivotal trial create momentum for OCU410ST. This progress suggests a potential future treatment option for patients with Stargardt disease, an area of significant unmet medical need. The results of the Phase 2/3 trial will be critical in determining the therapy’s efficacy and potential path to regulatory approval.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
