Benzinga | Sep 30, 2021 11:52AM EDT

Apellis Pharmaceuticals Announces Detailed Results From Phase 3 DERBY And OAKS Studies Presented At Retina Society Annual Meeting

Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in complement, today announced that detailed data from the Phase 3 DERBY and OAKS studies were presented for the first time as part of two oral presentations at the Retina Society Annual Scientific Meeting in Chicago. The studies evaluated the efficacy and safety of pegcetacoplan, an investigational, targeted C3 therapy, in geographic atrophy (GA) secondary to age-related macular degeneration (AMD). GA is a leading cause of blindness that impacts more than five million people globally including one million people in the United States.1,2

In the OAKS study, monthly (p=0.0003) and every-other-month treatment (p=0.0052) with pegcetacoplan met the primary endpoint, significantly reducing GA lesion growth compared to pooled sham at 12 months. The DERBY study narrowly missed the primary endpoint, showing a reduction in GA lesion growth with monthly (p=0.0528) and every-other-month treatment (p=0.0750) compared to pooled sham at 12 months. In a prespecified analysis of the combined studies, pegcetacoplan showed a reduction in lesion growth in patients with foveal and extrafoveal lesions, with a greater effect in patients with extrafoveal lesions at baseline. Apellis plans to submit a New Drug Application (NDA) for pegcetacoplan for GA to the U.S. Food and Drug Administration (FDA) in the first half of 2022.

"The data presented at Retina Society reinforce that pegcetacoplan is a breakthrough for patients with GA, a relentless disease that leads to blindness and has no treatment," said Charles Wykoff, M.D., Ph.D., investigator of the OAKS study and director of research, Retina Consultants of Texas. "Pegcetacoplan demonstrated a clinically meaningful treatment effect and favorable safety profile with both monthly and every-other-month dosing, highlighting the potential of pegcetacoplan to become the first treatment for GA."