Ocugen Completes Dosing in Phase 1/2 Trial of OCU410 for Geographic Atrophy

Ocugen announced dosing is complete in the second cohort of its phase 1/2 ArMaDa trial of OCU410, an adeno-associated viral vector 5 human RORA (AAV5-hRORA) gene therapy being developed for geographic atrophy (GA). Up to 13 retinal surgery centers across the United States are participating in the ArMaDa clinical trial.

The phase 1/2 trial will assess the safety of unilateral subretinal administration of OCU410 in patients with GA and will be conducted in two phases:

• Phase 1 is a multicenter, open-label, dose-ranging study consisting of three dose levels— low dose (2.5×10¹⁰ vg/mL), medium dose (5×10¹⁰ vg/mL), and high dose (1.5 ×10¹¹ vg/mL)
• Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study in which patients will be randomized in a 1:1:1 ratio to either one of two OCU410 treatment groups or to an untreated control group

In the completed second cohort, three patients received 200 mL single subretinal administration of the medium dose (5x10¹⁰ vg/mL) of OCU410.

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 high dose, which is the final dose in the phase 1 dose-escalation study. The company advised it will provide clinical updates on an ongoing basis.

The lead investigator for the OCU410 phase 1/2 trial is Syed M. Shah, Vice Chair of Research and Digital Medicine and Director of Retina Service at Gundersen Health System, La Crosse, Wisconsin.

“Currently we have two FDA approved, anticomplement therapies for GA targeting a single pathway of the disease, which has multifactorial and complex etiology,” Dr. Shah said in a company news release. “The limited benefit comes with the burden of continued multiple intravitreal injections spanning over several years. This novel modifier gene therapy has the potential to transform the therapeutic landscape in GA treatment.”

Huma Qamar, MD, Chief Medical Officer of Ocugen, added, “We are very enthusiastic about the potential of OCU410 as a one-time, gene-agnostic option for the treatment of GA. OCU410 regulates multiple pathways involved with the disease, including lipid metabolism, inflammation, oxidative stress, and membrane attack complex (complement) with a single sub-retinal injection.”