Ocugen Announces Dosing Completion of Subjects with Stargardt Disease in Cohort 2 of Phase 1/2 GARDian Clinical Trial of OCU410ST—A Modifier Gene Therapy

MALVERN, Pa., May 15, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that dosing is complete in the second cohort of its Phase 1/2 GARDian clinical trial for OCU410ST (AAV-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease as a one-time treatment for life.

"The completion of dosing for Cohort 2 participants signifies an important clinical milestone for our pioneering modifier gene therapy,” said Huma Qamar, MD, MPH, Chief Medical Officer of Ocugen. “We are encouraged by the ongoing positive safety and tolerability profile demonstrated by OCU410ST, enabling us to consider higher doses in patients as we progress with the dose-escalation study. We look forward to sharing preliminary safety and efficacy data from Phase 1 of the clinical trial.”

Six patients with Stargardt disease have been dosed in the Phase 1/2 clinical trial to date. An additional three patients will be dosed with the high dose (Cohort 3) of OCU410ST in the dose-escalation phase.

“There remains a great unmet medical need for patients with Stargardt disease, which is the most common inherited retinal disease affecting the center of the vision and does not have any FDA-approved treatment options. OCU410ST is a novel modifier gene therapy that provides hope to these patients,” said Benjamin Bakall, MD, PhD, Director of Clinical Research at Associated Retina Consultants and Clinical Assistant Professor at University of Arizona, College of Medicine – Phoenix. “I am excited that we completed dosing of the last patient in Cohort 2, who received medium dose of this novel therapeutic leveraging a gene-agnostic approach, at Associated Retina Consultants (ARC) in Phoenix, AZ with the surgical team led by Dr. Mark Kwong, Medical Director of ARC.”

A Data and Safety Monitoring Board meeting will convene next month to review the 4-week safety data of the medium dose cohort before proceeding with Cohort 3 (high dose), which is the final dose in the Phase 1 dose-escalation study.

The GARDian clinical trial will assess the safety and efficacy of unilateral subretinal administration of OCU410ST in subjects with Stargardt disease and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose-ranging study consisting of three dose levels [low dose (3.75×10¹⁰ vg/mL), medium dose (7.5×10¹⁰ vg/mL), and high dose (2.25×10¹¹ vg/mL)]. Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study in which adult and pediatric subjects will be randomized in a 1:1:1 ratio to either one of two OCU410ST dose groups or to an untreated group.