Apellis Pharmaceuticals, Inc. announced that five oral presentations and two on-demand papers are being highlighted at the American Society of Retina Specialists Annual Scientific Meeting taking place July 13 -16, 2022 in New York City. New data include an analysis from the Phase 3 DERBY and OAKS studies, which showed the mean rate (slope) of GA lesion growth at 18 months with both monthly and every-other-month intravitreal pegcetacoplan, an investigational, targeted C3 therapy for geographic atrophy (GA) secondary to age-related macular degeneration. The results were consistent with previously reported 18-month data, with all p-values (nominal) below 0.05.

Apellis announced the submission of a New Drug Application (NDA) for pegcetacoplan for GA to the U.S. Food and Drug Administration (FDA) in June 2022. The FDA decision on NDA filing acceptance is expected in August 2022. Apellis also plans to submit a marketing authorization application to the European Medicines Agency in the second half of 2022.

DERBY (621 patients enrolled) and OAKS (637 patients enrolled) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies comparing the efficacy and safety of intravitreal pegcetacoplan with sham injections in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The primary objective of the studies is to evaluate the efficacy of pegcetacoplan in patients with GA assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence (p-value less than 0.05) at 12 months. Patients in DERBY and OAKS will continue to receive masked treatment for 24 months.

Secondary functional endpoints will be evaluated after all patients have received treatment for 24 months. Geographic atrophy (GA) is an advanced form of age-related macular degeneration (AMD) and a leading cause of blindness that impacts more than 5 million people worldwide, including one million people in the United States. This progressive disease can severely impair visual function, independence, and quality of life as it takes on average 2.5 years for GA lesions to encroach the fovea, which is responsible for central vision.

GA is caused by destruction of retinal cells through irreversible lesion growth that is driven by excessive complement activation. C3 is the only target that can precisely control the complement cascade due to its central location. There are currently no approved treatments for GA. Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases.

Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of geographic atrophy.