Phase 1/2 ArMaDa Trial: OCU410 Gene Therapy Dosing Complete for GA Patients

Biotechnology firm Ocugen, Inc. has announced the completion of dosing in the second group of its Phase 1/2 clinical trial for a gene therapy candidate, OCU410 (AAV-hRORA). This treatment is designed for geographic atrophy (GA), which is a severe form of dry age-related macular degeneration (dAMD) affecting around 1 million people in the United States.

Dr. Huma Qamar, Ocugen's Chief Medical Officer, expressed optimism about OCU410's potential as a single-administration, gene-agnostic therapy for GA. The treatment is designed to regulate various disease-related pathways, such as lipid metabolism, inflammation, oxidative stress, and the complement system, through a single sub-retinal injection.

The second group of the trial involved three participants who received a medium dose of OCU410. The study is being conducted across 13 leading retinal surgery centers in the United States. Syed M. Shah, MD, the lead investigator for the trial, highlighted the current limitations of FDA-approved anti-complement therapies for GA, which target only one aspect of the disease's complex etiology and require multiple intravitreal injections over several years. He believes that OCU410's novel gene therapy approach could significantly change the treatment landscape for GA.

A Data and Safety Monitoring Board is scheduled to meet to review the 4-week safety data of the medium dose group before the trial proceeds to the high dose phase. The Phase 1/2 trial is divided into two parts: Phase 1, an open-label, multicenter dose-ranging study with three levels, and Phase 2, a randomized, outcome accessor-blinded, dose-expansion study with subjects allocated to treatment or control groups.

Ocugen is committed to providing ongoing updates on the clinical trial's progress. The company is focused on developing innovative gene and cell therapies, biologics, and vaccines to improve global health. Its breakthrough gene therapy platform aims to treat multiple retinal diseases with a single product and is also advancing research in infectious and orthopedic diseases.

dAMD, the condition that GA is a part of, affects approximately 10 million Americans and over 266 million people globally. It is characterized by the thinning of the macula, leading to the deterioration of the retina and central vision impairment. GA is a significant cause of vision loss among the elderly population.

OCU410 is based on an adeno-associated virus (AAV) delivery platform for the retinal delivery of the RORA gene. The RORA protein has been shown to play a crucial role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and has demonstrated anti-inflammatory and complement system inhibition capabilities in both in-vitro and in-vivo studies. This suggests that OCU410 could address the multiple pathways associated with dAMD's pathophysiology. Ocugen is developing AAV-RORA as a one-time gene therapy specifically for GA treatment.