Ocugen, Inc. 8-KCurrent Reports (8-K / ad hoc)

Financial Statements

Item 8.01 Other Events. On September 1, 2026, the Company announced that the first patient was dosed in the global Phase 3 registrational trial of OCU410 (AAV5-hRORA), its first-in-class modifier gene therapy candidate for Geographic Atrophy (“GA”) secondary to dry age-related macular degeneration (“dAMD”). The initiation of dosing follows the successful completion of a Type B End-of-Phase 2 (EOP2) meeting with the Center for Biologics Evaluation and Research (CBER) of the U.S. Food and Drug Administration (the “FDA”) in July 2026, resulting in alignment on all critical Phase 3 design elements, including primary and secondary endpoints, dose, adaptive design, and a single pivotal trial pathway to support a Biologics License Application (BLA). In July 2026, the Company also announced that the FDA granted OCU410 Regenerative Medicine Advanced Therapy (“RMAT”) designation for the treatment of GA, secondary to dAMD. On September 3, 2026, the independent Data Monitoring Committee (the “DMC”) for the Phase 2/3 clinical trial of OCU410ST completed a pre-specified interim analysis and provided its recommendation to modify and continue the clinical study as described below. This interim analysis examined atrophic lesion size in 26 subjects (16 treated and 10 control subjects) after they had completed their 8-month clinical assessments. The DMC recommended that the study be continued per the existing protocol in order to obtain 8 months follow-up of the entire study population. The DMC noted that one could consider futility based on the negative direction of treatment effect on the interim sample and other interim results, but the DMC recommended the modification to obtain the entire dataset at 8 months in order to observe the results without the baseline lesion size imbalance that existed between the treatment and control arms in the small interim analysis population and that does not exist in the entire dataset at 8 months. The Company intends to follow the DMC’s recommendation to obtain 8 months follow-up of the entire study population.