OCU410 is a modifier gene therapy product candidate being developed by Ocugen for dry AMD.
Ocugen announced 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 age-related macular degeneration (dAMD).
Shankar Musunuri, PhD, MBA, chairman, CEO and co-founder of Ocugen, pointed out in a news release OCU410 is the company’s first-in-class modifier gene therapy for dAMD that addresses gaps among other therapies available and in development for dAMD as a potential one-time treatment for life.
“We are very pleased to continue advancing our ophthalmic gene therapy pipeline, which remains the company’s primary focus,” Musunuri said in the news release.
According to the company, the Phase 1/2 trial will assess the safety and efficacy of OCU410 for geographic atrophy (GA) secondary to dAMD and will be conducted in two phases. The first phase is a multicenter, open-label, dose-ranging study. Phase 2 is a randomly assigned expansion phase in which subjects will be randomized in a 1:1:1 ratio to either 1 of 2 OCU410 dose groups or to an untreated control group.
Moreover, the company noted OCU410 is a potential curative therapy with a single sub-retinal injection that targets multiple pathways causing dAMD, 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.
Arun Upadhyay, PhD, chief scientific officer and the head of R&D at Ocugen, noted this is another key step for OCU410.
“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,” Upadhyay said in the news release. “OCU410 offers hope for those battling GA that are faced with limited treatment options and the real prospect of ultimately losing their vision.”
Benjamin Bakall, MD, PhD, director of clinical research at Associated Retina Consultants (ARC) and clinical assistant professor at University of Arizona, College of Medicine in Phoenix, Arizona, noted in the news release that the candidate fills an unmet need for the treatment of GA.
“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,” Bakall said. “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, Arizona, 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; the light-sensitive nerve tissue lining the inside of the eye.
Ocugen noted in its news release OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR 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 in-vitro and in-vivo (animal model) studies.
The company noted that these results demonstrate the ability for OCU410 to target multiple pathways linked with dAMD pathophysiology.
Further, the company also is developing AAV-RORA as a one-time gene therapy for the treatment of GA. Currently, the other therapeutic options available target only complement activation and require approximately 6-12 intravitreal injections annually.
According to the news release, 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.
The company also noted 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.
Moreover, GA, an advanced form of dry age-related macular degeneration, affects approximately 1 million people in the United States alone.