Ocugen completes dosing of subjects with geographic atrophy in Phase 1/2 clinical trial of OCU410
OCU410 is a modifier gene therapy candidate being developed for geographic atrophy, an advanced stage of dry age related macular degeneration.
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Ocugen Inc announced that dosing is complete in the first cohort of its Phase 1/2 ArMaDa clinical trial for OCU410.
OCU410 (AAV-hRORA)—a modifier gene therapy candidate being developed for geographic atrophy (GA), an advanced stage of dry age related macular degeneration (dAMD). GA affects approximately 1 million people in the United States alone.
Shankar Musunuri, PhD, MBA, chairman, CEO and co-founder of Ocugen, said in a news release,1 the company is enthusiastic about the potential of OCU410 as a one-time treatment for life with a single sub-retinal injection.
“While there are currently two recently approved products for the treatment of GA, both require approximately 6-12 intravitreal injections annually and target only the complement system,” he explained. “OCU410 addresses multiple pathways causing dAMD, including complement, lipid metabolism, inflammation, and oxidative stress.”
According to Ocugen, 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 in-vitro and in-vivo (animal model) studies. The results show the ability of OCU410 to target multiple pathways linked with dAMD pathophysiology.
Up to 13 leading retinal surgery centers across the United States are participating in the ArMaDa clinical trial. The enrollment in the first cohort is now complete and 3 subjects received 200µL single subretinal administration of the low dose (2.5x1010 vg/mL) of OCU410.1
“As a retinal surgeon, I am encouraged by therapeutic options that can potentially provide long-term benefit to my patients,” said Lejla Vajzovic, MD, FASRS, Director of Duke Surgical Vitreoretinal Fellowship Program, Associate Professor of Ophthalmology with Tenure in Adult and Pediatric Vitreoretinal Surgery and Diseases, Duke University Eye Center. “OCU410 is a novel modifier gene therapy approach that could initiate a paradigm shift in the field of ophthalmology.”
Moreover, the company noted that its 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.
Matthew Levine, director of Grants, Advocacy and Partnerships at the American Macular Degeneration Foundation (AMDF), said the organization has supported the research of Neena Haider, PhD, inventor of modifier gene therapy, and OCU410 in particular, and is pleased that Ocugen is now spearheading the clinical trials necessary to bring this therapy closer to patients.
“The continued advancement of OCU410 offers hope to those whose vision is already deteriorating that their remaining vision could be preserved and could potentially prevent others with an early dAMD diagnosis from developing any significant vision loss,” he said in the news release.
The company will continue to provide clinical updates.
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.1
Moreover, 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% to 90% of the total AMD population.
