Ocugen Inc

Ocugen has dosed the first patient in the global phase III ArMaDa3 study evaluating its modifier gene therapy candidate OCU410 for geographic atrophy secondary to dry age-related macular degeneration. The study follows a Type B End-of-phase II meeting with the FDA in July 2026, which aligned on key design elements including endpoints, dose, and a single pivotal study pathway to support a biologics license application. This randomized registrational study, the first pivotal gene therapy study in GA, will enroll 237 patients randomized 2:1 to receive a single subretinal injection of OCU410 at 5×10^10 vg/mL or remain untreated. The primary endpoint measures the rate of change in square root-transformed GA lesion area through month 12, with secondary endpoints evaluating functional vision. Ocugen expects to file a BLA for OCU410 in 2028 and is in discussions with the European Medicines Agency for potential alignment.

Ocugen reported second quarter 2026 financial results and provided updates on its three late-stage gene therapy programs. The company's cash, cash equivalents, and restricted cash totaled $100.4 million as of June 30, 2026, up from $32.2 million as of March 31, 2026, following the closing of a $130 million convertible notes financing that extended its cash runway into 2028. Net loss per common share was $0.07 for the three months ended June 30, 2026, compared to $0.05 for the prior-year period. Ocugen plans to initiate a Phase III trial for OCU410 in geographic atrophy by September 2026, with a Biologics License Application targeted for 2028, and expects top-line Phase III data for OCU400 in retinitis pigmentosa in the first quarter of 2027.

Ocugen announced that the U.S. FDA has granted Regenerative Medicine Advanced Therapy designation to its gene therapy candidate OCU410 for the treatment of geographic atrophy secondary to dry age-related macular degeneration. The decision was supported by Phase 2 ArMaDa clinical data showing clinically meaningful efficacy and a favorable safety profile for the one-time modifier gene therapy. OCU410 delivers the RORA gene via an AAV-5 vector to regulate multiple retinal disease pathways and has also received Advanced Therapy Medicinal Product status from the European Medicines Agency. The FDA noted that preliminary clinical evidence indicates OCU410 has the potential to address a serious condition with significant unmet medical need, qualifying it for an expedited development pathway. Ocugen shares closed Thursday down 4.03% at $1.19 and were trading up 3.36% at $1.23 in the pre-market.

Ocugen has signed a binding term sheet with Roots Pharmaceutical and its strategic partner Al-Dhow International Holding to license OCU400, its modifier gene therapy for retinitis pigmentosa, in the Middle East and North Africa region. Under the expected license agreement, Ocugen would receive upfront license fees and near-term development milestone payments totaling up to $4 million, plus sales milestone payments up to $255 million and a 22% royalty on net sales. Ocugen would also manufacture and supply OCU400 commercially. The definitive agreement is expected within 90 days, while Ocugen continues its Phase 3 liMeliGhT trial with topline data anticipated in the first quarter of 2027.

Ocugen announced it will present on its innovative modifier gene therapy platform at several investor and industry conferences in July 2026. The company will participate in a fireside chat at the Piper Sandler Virtual Ophthalmology Day on July 10, and its executives will join a panel on regenerative approaches in retinal disease at the OIS Retina Innovation Summit in Montreal on July 14. At the ASRS meeting on July 17, one-year results from the Phase 2 ArMaDa trial of OCU410 will be presented, along with a separate analysis of quantitative FAF/SD-OCT and safety outcomes from the Phase 1/2 study of OCU410. A webcast of the Piper Sandler presentation will be available on the company's investor relations website for 30 days.