Ocugen, Inc. announced that the Data and Safety Monitoring Board for the Phase 1/2 ArMaDa clinical trial for OCU410 recently convened and approved to proceed dosing with the medium dose of OCU410 in the dose-escalation phase of the study. Three subjects with geographic atrophy (GA) were dosed in the Phase 1/2 clinical trial to date. An additional three subjects will be dosed with the medium dose (Cohort 2) and three patients with the high dose (Cohort 3) of OCU410 in the dose-escalation phase.

Currently approved products to treat GA have significant limitations, as they require multiple injections per year and only target one pathway contributing to GA. OCU410 regulates multiple pathways involved with the disease including: lipid metabolism, inflammation, oxidative stress, and membrane attack complex (complement). The ArMaDa clinical trial will assess the safety of unilateral subretinal administration of OCU410 in subjects 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 subjects will be randomized in a 1:1:1 ratio to either one of two OCU410 treatment groups or to an untreated control group. Ocugen is committed to finding solutions for inherited retinal diseases as well as blindness diseases affecting millions.

GA is an advanced form of dry age-related macular degeneration and affects approximately 1 million people in the United States. dAMD affects approximately 10 million Americans and more than 266 million people worldwide. It is characterized by the thinning of the macula.

The macula is the part of the retina responsible for clear vision in one?s direct line of sight. dAMD involves the slow deterioration of the retina with submacular drusen (small white or yellow dots on the retina), atrophy, loss of macular function and central vision impairment. dAMD accounts for 85-90% of the total AMD population.

OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (ROR Related Orphan Receptor A) gene. The RORA protein plays an important role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role as well as inhibiting the complement system in in-vitro and in-vivo (animal model) studies. These results demonstrate the ability for OCU410 to target multiple pathways linked with dAMD pathophysiology.

Ocugen is developing AAV-RORA as a one-time gene therapy for the treatment of GA.