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. » More on 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. » More on 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. » More on de.investing.com

Ocugen signs a binding term sheet with Roots Pharmaceutical and Al-Dhow for exclusive rights to the gene therapy OCU400 in the MENA region. The company expects upfront and near-term milestone payments of up to USD 4 million, sales-related milestones of up to USD 255 million, and a 22% royalty on net sales; Ocugen is to handle commercial manufacturing. OCU400 is in the liMeliGhT phase-3 study; topline data are expected in Q1 2027, followed by a planned BLA submission. Ocugen has a market capitalization of USD 497.63 million, 12-month revenue of USD 4.46 million and EPS of -USD 0.23; InvestingPro currently rates the stock as undervalued. » More on de.investing.com

Ocugen presented clinical data and a regulatory roadmap at the Virtual Ophthalmology Day for three late-stage ophthalmology programs (OCU400, OCU410ST, OCU410). Phase data show promising effects: a 33% reduction in lesion size in GA and a 54% decrease in lesion growth in Stargardt disease; no drug-related serious adverse events reported to date. The company plans BLA filings for retinitis pigmentosa and Stargardt disease in 2025 and for geographic atrophy in 2028, is using adaptive Phase‑3 designs, and also highlights remaining clinical and regulatory risks. » More on de.investing.com