Ocugen announced positive results from cohort 1 of the phase 1/2 ArMaDa clinical trial, designed to assess the safety of unilateral subretinal administration of OCU410 in subjects with geographic atrophy (GA). The company also announced the dosing of patients in cohort 2 of the trial. 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 1 of 2 OCU410 treatment groups or to an untreated control group. Up to 13 retinal surgery centers across the United States are participating in the ArMaDa trial. 

“OCU410 regulates multiple pathways involved with the disease, including lipid metabolism, inflammation, oxidative stress, and membrane attack complex complement with a single subretinal injection. We are very enthusiastic about the potential of OCU410 as a 1-time, gene-agnostic option for the treatment of geographic atrophy,” said Dr. Huma Qamar, chief medical officer of Ocugen, in a news release.

In related news, Ocugen announced that the FDA has cleared the company’s Investigational New Drug (IND) application to initiate a phase 3 clinical trial of OCU400, for treatment of retinitis pigmentosa (RP). The phase 3 study will have a sample size of 150 patients: 1 arm of 75 patients with the RHO gene mutation and the other arm with 75 patients who are gene agnostic. In each arm, patients will be randomized 2:1 to the treatment group (2.5 x 10¹⁰ vg/eye of OCU400) and untreated control group, respectively.