Phase III DERBY and OAKS studies of Empaveli show mixed results in geographic atrophy.- Apellis Pharma

In a prespecified analysis of the combined DERBY and OAKS studies, monthly and every-other-month treatment with pegcetacoplan reduced GA lesion growth by 17% (p<0.0001) and 14% (p="0.0012)," respectively, compared to pooled sham at 12 months. in a prespecified analysis of the primary endpoint, pegcetacoplan demonstrated a greater effect in patients with extrafoveal lesions at baseline. patients with ga typically present first with extrafoveal lesions, which then progress toward the fovea where central vision is impacted. in the combined studies, monthly and every-other-month treatment with pegcetacoplan decreased ga lesion growth by 26% (p><0.0001) and 23% (p="0.0002)," respectively, in patients with extrafoveal lesions compared to pooled sham at 12 months.></0.0001)></0.0001)>

Pegcetacoplan was well tolerated in both Phase III studies. The pooled rate of new-onset exudations was 6.0% of patients in the monthly pegcetacoplan groups, 4.1% in the every-other-month pegcetacoplan groups, and 2.4% in the sham groups.

Based on results from the studies, the company plans to submit a New Drug Application (NDA) for pegcetacoplan for GA to the FDA in the first half of 2022. GA is a leading cause of blindness that impacts more than five million people globally including one million people in the United States.