Ocugen completes dosing of subjects with geographic atrophy in Cohort 2 of Phase 1/2 ArMaDa clinical trial of OCU410

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Ocugen Inc announced dosing is complete in the second cohort of its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA)—a modifier gene therapy candidate being developed for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD).

According to the company’s news release, GA affects approximately 1 million people in the United States alone.¹ It noted dAMD affects approximately 10 million Americans and more than 266 million people worldwide.

Huma Qamar, MD, MPH, chief medical officer of Ocugen, said in a news release the company is enthusiastic about the potential of OCU410 as a one-time, gene-agnostic option for the treatment of GA.

“OCU410 regulates multiple pathways involved with the disease, including lipid metabolism, inflammation, oxidative stress, and membrane attack complex (complement) with a single sub-retinal injection,” Qamar said in the release.

The company noted that dosing in the second cohort is complete and 3 subjects received 200 mL single subretinal administration of the medium dose (5x1010 vg/mL) of OCU410. Up to 13 leading retinal surgery centers across the United States are participating in the ArMaDa clinical trial.

Syed M Shah, MD, vice chairman of Research and Digital Medicine and Director of Retina Service at Gundersen Health System, La Crosse, Wisconsin, and the lead investigator for the OCU410 Phase 1/2 trial, explained that there currently are 2 FDA approved, anti-complement therapies for GA targeting a single pathway of the disease, which has multifactorial and complex etiology.

“The limited benefit comes with the burden of continued multiple intravitreal injections spanning over several years,” Shah said in the news release. “This novel modifier gene therapy has the potential to transform the therapeutic landscape in GA treatment.”

A Data and Safety Monitoring Board meeting will convene next month to review the 4-week safety data of the medium dose cohort before proceeding with high dose, which is the final dose in the Phase 1 dose-escalation study.

According to the company, 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. The company noted in its news release tThese 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.

The ArMaDa Phase 1/2 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.

The company will continue to provide clinical updates on an ongoing basis.

Reference:

1. Ocugen. Ocugen Announces Dosing Completion of Subjects with Geographic Atrophy in Cohort 2 of Phase 1/2 ArMaDa Clinical Trial of OCU410—A Modifier Gene Therapy. GlobeNewswire News Room. Published April 19, 2024. Accessed April 22, 2024. https://www.globenewswire.com/news-release/2024/04/19/2866086/0/en/Ocugen-Announces-Dosing-Completion-of-Subjects-with-Geographic-Atrophy-in-Cohort-2-of-Phase-1-2-ArMaDa-Clinical-Trial-of-OCU410-A-Modifier-Gene-Therapy.html