Ocugen (NASDAQ:OCGN), a Malvern, Pennsylvania-based clinical-stage biotechnology company developing modifier gene therapies for ophthalmic and other diseases, on Wednesday reported financial results for the fourth quarter and full year ended December 31, 2025, alongside a business update highlighting progress across its three lead modifier gene therapy programs.
The company completed enrollment of 140 patients in the Phase 3 liMeliGhT trial evaluating OCU400, its lead modifier gene therapy candidate for retinitis pigmentosa (RP) associated with mutations in multiple genes including RHO, NR2E3 and CEP290.
Topline data from the study are expected in the first quarter of 2027, with Ocugen planning to initiate a rolling submission of a biologics license application (BLA) to the U.S. Food and Drug Administration in the third quarter of 2026, potentially positioning OCU400 as one of the first gene-agnostic therapies for RP.
In the OCU410 program for geographic atrophy (GA) secondary to dry age-related macular degeneration (dry AMD), Ocugen reported positive 12-month interim data from the Phase 2 ArMaDa trial.
Treatment with OCU410 resulted in a 46% reduction in the growth rate of atrophic lesion area compared with untreated eyes (p=0.015, N=23), demonstrating statistically significant slowing of disease progression and supporting further advancement toward pivotal studies.
Ocugen also secured an exclusive license agreement in Korea for OCU400, expanding its global footprint and providing potential near-term milestone and royalty revenue as development progresses.
Financially, the company ended 2025 with cash, cash equivalents and restricted cash of $18.9 million.
In January 2026, Ocugen completed a $22.5 million financing transaction, extending its operational runway into the fourth quarter of 2026 based on current plans and excluding potential additional milestone payments or business development activities.
Full-year 2025 revenue was $4.4 million, primarily from collaboration agreements and grant funding, while the net loss was $67.8 million, or 23 cents per share.