Ocugen

Ocugen treated the first patient in ArMaDa3, a global phase‑3 trial of 237 participants in the US, Canada, Europe and Latin America. Patients are randomized 2:1 and receive a single subretinal injection of OCU410 or no treatment; the primary endpoint is change in lesion area at 4, 8 and 12 months. OCU410 received FDA RMAT designation, phase‑2 data showed a 31% reduction in lesion growth rate in the mid‑dose group with no serious adverse events; Ocugen is targeting a BLA submission in 2028. » Więcej na de.investing.com

Ocugen presented a late-stage gene therapy strategy at the Canaccord Growth Conference with three programs targeting retinitis pigmentosa (OCU400), Stargardt disease and geographic atrophy (OCU410). A $130 million convertible note is intended to fund the company through 2028 and cover key clinical and regulatory milestones. The company reports solid safety data in more than 325 treated patients, with no therapy-related serious adverse events. Top-line data for OCU400 are expected in Q1 2025 and for OCU410 in Stargardt in Q2 2025; two BLA filings are planned for 2025, and approval for geographic atrophy is targeted for 2028. Ocugen is exploring partnerships outside the U.S. to commercialize and minimize dilutive financing. » Więcej na de.investing.com

The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to Ocugen’s gene therapy candidate OCU410 based on Phase 2 data on efficacy and safety without serious drug‑related adverse events. OCU410 is a one‑time subretinal AAV5‑based therapy delivering RORA and is being developed to treat geographic atrophy due to dry age‑related macular degeneration. Ocugen expects to start the pivotal Phase 3 trial in Q3 2026 and plans to submit a Biologics License Application in 2028; the RMAT designation enables more intensive FDA interaction, a rolling‑BLA and potential accelerated review pathways. » Więcej na de.investing.com