Ocugen Completes Dosing of Subjects with Stargardt Disease in Cohort 2 of Phase 1/2 GARDian Clinical Trial of OCU410ST Gene Therapy

Ocugen 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.

"The completion of dosing for Cohort 2 participants signifies an important clinical milestone for our pioneering modifier gene therapy,” Huma Qamar, MD, MPH, Chief Medical Officer of Ocugen, said in a company news release. “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.

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.

The company expects to provide a clinical trial update for OCU410ST in the third quarter of 2024.