Ocugen Announces First Patient Dosed in Phase 1/2 Trial of OCU410 Modifier Gene Therapy for GA
Ocugen announced that the first patient has been dosed in the ArMaDa phase 1/2 clinical trial of OCU410 (AAV-RORA), a modifier gene therapy product candidate being developed for dry AMD (AMD).
This phase 1/2 trial will assess the safety and efficacy of OCU410 for geographic atrophy (GA) secondary to dry AMD and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose-ranging study. Phase 2 is a randomized expansion phase in which subjects will be randomized in a 1:1:1 ratio to either one of two OCU410 dose groups or to an untreated control group.
“OCU410, our first-in-class modifier gene therapy for dry AMD, addresses gaps among other therapies available and in development for dry AMD as a potential one-time treatment for life,” Shankar Musunuri, PhD, Chairman, CEO and Co-Founder of Ocugen, said in a company news release. “We are very pleased to continue advancing our ophthalmic gene therapy pipeline, which remains the company’s primary focus.”
OCU410 is a potential curative therapy with a single sub-retinal injection that targets multiple pathways causing dry AMD, including lipid metabolism, inflammation, oxidative stress, and complement activation. Currently, the other therapeutic options available target only complement activation and require approximately 6-12 intravitreal injections annually.
“Breaking new ground in the pursuit of vision restoration, our pioneering modifier gene therapy candidate, OCU410, achieves another major milestone by dosing a GA patient in a phase 1/2 clinical trial,” said Arun Upadhyay, PhD, Chief Scientific Officer, Head of R&D at Ocugen. “OCU410 offers hope for those battling GA that are faced with limited treatment options and the real prospect of ultimately losing their vision.”
“There remains a great unmet need for novel durable and effective treatments for GA, which remains one of the most common causes of vision loss globally,” said Benjamin Bakall, MD, PhD, director of clinical research at Associated Retina Consultants (ARC) and clinical assistant professor at University of Arizona, College of Medicine–Phoenix. “I am excited that we performed the first surgery with this novel therapeutic approach—designed to restore homeostasis and slow disease progression following a single treatment—at ARC in Phoenix, AZ, with the surgical team led by Dr. Mark Kwong, medical director of ARC.”
The first surgery was successful in delivering the new gene underneath the retina.