Key takeaways:
- Low-dose OCU410 slowed lesion growth, preserved retinal tissue, and stabilized visual function in three patients.
- No drug-related serious adverse events were reported.
OCU410, a single-dose modifier gene therapy, demonstrated positive preliminary outcomes for the treatment of geographic atrophy in a phase 1/2 clinical trial, according to a press release from Ocugen.
“Currently available therapies require six to 12 injections per year, which places an unreasonable burden on patients and caregivers,” Shankar Musunuri, PhD, MBA, chairman of the board, CEO and co-founder of Ocugen, told Healio. “Additionally, these therapies come with notable safety considerations while not demonstrating significant benefit in visual function.”
The ArMaDa trial is evaluating nine patients in low-, medium- and high-dose cohorts of OCU410.
At 6 months, three patients in the low-dose cohort experienced lesion growth that was 21.4% slower in treated vs. untreated fellow eyes from baseline, which is “favorable when compared to published data on pegcetacoplan injected every month or every other month over 6 months,” the release said.
These patients also experienced increased preservation of retinal tissue around the geographic atrophy lesions, with results comparable to pegcetacoplan, and stabilization of visual function in treated eyes.
Ocugen reported no drug-related serious adverse events.
“Our aim is to develop a ‘one-and-done’ approach that delivers meaningful structural and functional outcomes for patients with significant unmet needs. We will continue to monitor patients in the OCU410 phase 1 clinical trial at 9 and 12 months as we complete recruitment for phase 2,” Musunuri said. “Currently, Ocugen has three modifier gene therapies in the clinic, including our Phase 3 OCU400 liMeliGhT clinical trial for retinitis pigmentosa that is on track” for biologics license application and marketing authorization application filings in the first half of 2026.
