Ocugen completes dosing in subjects with GA secondary to dAMD in Phase 1/2 ArMaDa clinical trial of OCU410

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Ocugen Inc announced that dosing is complete in the third cohort of its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA)—a novel 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.

Majda Hadziahmetovic, MD, an associate professor of Ophthalmology, Vitreoretinal Diseases and Surgery at Duke University Eye Center, and the lead investigator for the OCU410 Phase 1/2 trial, pointed out in a news release there currently are 2 FDA approved anti-complement therapies for GA, which targets only one pathway for a disease that has a multifactorial and complex etiology.¹

“Though these treatments are a significant milestone, they provide limited benefits and involve a continuous regimen of multiple intravitreal injections over several years. This modifier gene therapy could potentially transform the landscape of GA treatment,” Hadziahmetovic

According to the company, OCU410 utilizes an adeno-associated virus (AAV) platform for the retinal delivery of the RORA (ROR Related Orphan Receptor A) gene. The RORA protein plays a key role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role as well as inhibiting the complement system in both in vitro and in vivo (animal model) studies.¹

The company noted in its news release, these results demonstrate the ability of OCU410 to target multiple pathways linked with dAMD pathophysiology. Ocugen is developing AAV-RORA as a one-time gene therapy for the treatment of geographic atrophy.

The ArMaDa clinical trial is designed to assess the safety of unilateral subretinal administration of OCU410 in patients with GA and will be conducted in 2 phases. Phase 1 is a multicenter, open-label, dose-ranging study consisting of 3 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.¹

The company noted in its news release that 3 subjects received a single subretinal administration of the highest dose (200 µL of 1.5x1011 vg/mL) being tested, which completed the dosing for the Phase 1 stage of the trial. The ArMaDa clinical trial for OCU410 is being performed at 14 leading retinal surgery centers across the United States.

“We are very encouraged about the potential of OCU410 as a one-time treatment option for GA,” said Huma Qamar, MD, MPH, CMI, chief medical officer of Ocugen. “We have initiated a Phase 2 clinical trial that will assess the safety and efficacy of OCU410 in a larger group of patients who will be randomized into either of two treatment groups (medium- or high-dose) or a control group.”

The company will continue to provide clinical updates on a periodic basis.

Reference:

1. Ocugen. Ocugen, Inc. Announces Completion of Dosing in Subjects with Geographic Atrophy Secondary to dAMD in High-Dose Cohort of Phase 1/2 ArMaDa Clinical Trial of OCU410—A Novel Modifier Gene Therapy. GlobeNewswire News Room. Published July 25, 2024. Accessed July 25, 2024. https://www.globenewswire.com/news-release/2024/07/25/2918663/0/en/Ocugen-Inc-Announces-Completion-of-Dosing-in-Subjects-with-Geographic-Atrophy-Secondary-to-dAMD-in-High-Dose-Cohort-of-Phase-1-2-ArMaDa-Clinical-Trial-of-OCU410-A-Novel-Modifier-Ge.html