Ocugen reports EMA positive advice for acceptability of OCU410ST

Ocugen (OCGN) announced that the Committee for Medicinal Products for Human Use, or CHMP, of the European Medicines Agency, or EMA, reviewed the study design, endpoints and planned statistical analysis of the ongoing pivotal confirmatory OCU410ST Phase 2/3 GARDian3 clinical trial for Stargardt disease and provided acceptability of a single U.S.-based trial for submission of a Marketing Authorization Application, or MAA. EMA provided this opinion based on safety and tolerability that OCU410ST demonstrated in the Phase 1 GARDian trial, including 48% slower lesion growth and statistically significant and clinically meaningful improvement of nearly 2-line/9-letter gain in best corrected visual acuity, or BCVA, at 12-month follow-up in evaluable treated eyes compared to untreated eyes. The Phase 2/3 study will enroll 51 participants diagnosed with Stargardt disease. Of these, 34 will receive a one-time subretinal injection of OCU410ST in the eye with poorer visual acuity, while 17 will be assigned to an untreated control group. The primary objective of the trial is to evaluate the reduction in atrophic lesion size. Key secondary endpoints include improvements in BCVA and low luminance visual acuity, or LLVA, compared to controls. Data from the one-year follow-up will be used to support the Company’s planned Biologics License Application and MAA in the EU. The EMA opinion is an extremely favorable outcome, as it will potentially reduce the time and cost to gain marketing authorization in the EU.