Ocugen has reported preliminary phase 2 data on its eye disease gene therapy, providing more evidence that the candidate may outperform existing treatments from Apellis Pharmaceuticals and Astellas Pharma.
The analysis covered 23 of the 51 patients enrolled in the phase 2 trial. Patients with geographic atrophy (GA) secondary to dry age-related macular degeneration received one of two doses of OCU410 or placebo. OCU410 is an AAV5 modifier gene therapy that delivers retinoid-related orphan receptor alpha. Ocugen believes the mechanism will affect inflammation, oxidative stress, lipid metabolism and complement activation.
In the preliminary phase 2 data, Ocugen linked the medium dose of OCU410 to a 54% reduction in lesion growth compared to placebo at 12 months. The high dose appeared less effective, with a 36% reduction, but the small size of the cohorts limits the conclusions that can be drawn about the one-time treatment.
On a call with analysts to discuss the data Thursday, Ocugen Chief Medical Officer Huma Qamar, M.D., said the absence of a traditional dose response could reflect baseline imbalances between the arms or a lack of statistical power. Qamar said the third possibility is that there are “biological factors related to excessive transgene expression ... where the additional vector doesn't provide additional benefit.”
Ocugen reported 27% slower lesion growth in a six-month analysis of 31 patients last year. At the time, Ocugen CEO Shankar Musunuri, Ph.D., compared the result favorably to approved intravitreal therapies, which he said achieved reductions of 12% and 13%.
Apellis became the first company to win FDA approval for a GA drug, the C3 inhibitor Syfovre, in 2023. Weeks later, Astellas won FDA approval for Izervay, which the company had acquired in its $5.9 billion takeover of Iveric Bio earlier that year.
Syfovre and Izervay hit the complement system. Because OCU410 is designed to act on the complement system and other pathways, Ocugen has predicted the candidate can improve on the outcomes achieved by existing products while freeing patients from safety risks and the need to receive frequent intravitreal injections.
Ocugen plans to report full data from its phase 2 trial later this quarter and enter phase 3 by the end of the year, although it has yet to choose a phase 3 dose. The biotech said the timeline puts it on track to file for approval of the gene therapy in 2028.