Data and Safety Monitoring Board approves simultaneous enrollment in Cohort 3 and Phase 2 initiation in OCU410 ArMaDa study for GA

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Ocugen, Inc. announced a positive outcome of the Data and Safety Monitoring Board (DSMB) Review for its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV5-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 2-3 million people in the U.S. and Europe combined.¹

The company noted that 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.

Moreover, the company noted that the 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.

According to the company's news release, 6 subjects diagnosed with GA were dosed in the Phase 1/2 clinical trial to date—3 subjects were dosed with the low dose and 3 subjects were dosed with the medium dose. An additional 3 patients will be dosed with the high dose of OCU410 in the dose-escalation phase.¹

Peter Chang, MD, FACS, DSMB chairman for the OCU410 clinical trial, noted the board to proceed with dosing subsequent GA subjects with the high dose of OCU410 in the dose-expansion phase of the study and concurrently initiate Phase 2 dosing.

“No serious adverse events (SAEs) related to OCU410 have been reported to date in both low- and medium-dose cohorts,” he said in the news release. “I believe that this marks a critical next step towards determining the maximum tolerated dose for OCU410 and is an important milestone for its clinical development.”

Huma Qamar, MD, MPH, CMI, chief medical officer of Ocugen, pointed out the second positive DSMB recommendation for the treatment of GA builds on the favorable safety and tolerability profile exhibited by OCU410.

“We are very enthusiastic about the potential of OCU410 as a potential one-time treatment for GA with a single sub-retinal injection,” Qamar said in the news release. “The currently approved treatments for GA target only the complement pathway and require approximately 6-12 intravitreal injections annually. OCU410 addresses multiple pathways causing dAMD, including complement, lipid metabolism, inflammation, and oxidative stress, providing long-term benefit to patients."

According to the company, the ArMaDa clinical trial will assess the safety and efficacy 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.¹

Reference

1. Ocugen. Data and Safety Monitoring Board Approves Simultaneous Enrollment in Cohort 3 and Phase 2 Initiation in OCU410 ArMaDa study for Geographic Atrophy. GlobeNewswire News Room. Published May 31, 2024. Accessed June 1, 2024. https://www.globenewswire.com/news-release/2024/05/31/2891519/0/en/Data-and-Safety-Monitoring-Board-Approves-Simultaneous-Enrollment-in-Cohort-3-and-Phase-2-Initiation-in-OCU410-ArMaDa-study-for-Geographic-Atrophy.html