Ocugen, Inc. (NASDAQ: OCGN) has announced significant progress in its gene therapy programs for inherited retinal diseases, along with a business update highlighting its third quarter 2024 financial results. The company's lead candidate, OCU400, is advancing through Phase 3 clinical development for retinitis pigmentosa (RP), while OCU410 and OCU410ST are in Phase 1/2 trials for geographic atrophy (GA) and Stargardt disease, respectively.
OCU400: Phase 3 Trial for Retinitis Pigmentosa
The Phase 3 liMeliGhT clinical trial of OCU400 for retinitis pigmentosa is on track to complete enrollment in the first half of 2025. The trial has expanded into Canada, potentially broadening commercialization opportunities in the U.S. and Europe. OCU400 is designed as a one-time treatment for life for patients with RP, a group of inherited genetic eye diseases that leads to progressive vision loss. The expanded access program (EAP) for adult patients with early to advanced RP aims to reach a greater segment of the RP patient population, estimated at 300,000 in the U.S., Canada, and Europe combined.
OCU410: Phase 2 Trial for Geographic Atrophy
Ocugen is actively recruiting patients for Phase 2 of the Phase 1/2 ArMaDa clinical trial of OCU410 for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD). The trial will assess the safety and efficacy of OCU410 in a larger group of patients randomized into either of two treatment groups (medium- or high-dose) or a control group. GA affects approximately 2-3 million people in the U.S. & EU. Current FDA-approved treatments address only the complement system and require approximately 6-12 intravitreal injections per year, whereas OCU410 addresses all four pathways linked with dAMD pathophysiology and is delivered through a single subretinal injection. There remains no approved product to treat GA in the EU.
OCU410ST: Advancing for Stargardt Disease
The Data and Safety Monitoring Board (DSMB) for the OCU410ST GARDian clinical trial has approved enrollment for the second phase of the Phase 1/2 clinical trial for Stargardt disease. Preliminary safety and efficacy data from the Phase 1/2 trials for OCU410 and OCU410ST will be presented at an upcoming clinical showcase on November 12, 2024, along with RP and LCA data updates from the OCU400 Phase 1/2 clinical trial.
Financial Update and Cash Runway
Ocugen reported that recent debt and equity financings have extended its cash runway into the first quarter of 2026. As of September 30, 2024, the Company’s cash and restricted cash totaled $39.0 million, compared to $39.5 million as of December 31, 2023. Total operating expenses for the three months ended September 30, 2024 were $14.4 million and included research and development expenses of $8.1 million and general and administrative expenses of $6.3 million. This compares to total operating expenses for the three months ended September 30, 2023 of $16.1 million that included research and development expenses of $7.0 million and general and administrative expenses of $9.1 million.
"With the recent equity and debt financings, we have sufficient cash-on-hand to continue supporting our robust ophthalmology pipeline and extend our cash runway into 1Q2026," said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen.
