Apellis Says Mixed Phase III Results Position Geographic Atrophy Candidate For Filing

Apellis Pharmaceuticals, Inc. plans to submit a new drug application for targeted C3 inhibitor pegcetacoplan in geographic atrophy (GA) based on the results of two Phase III studies, despite one of them failing to achieve its primary endpoint. The biotech said on 9 September that it thinks the data from both trials plus a successful Phase II study form a strong rationale for approval in an unmet medical need, while a majority of analysts commenting on the top-line data readout concurred with Apellis’s view.

GA is an advanced form of age-related macular degeneration (AMD) and a leading cause of blindness, estimated to affect 1 million patients in the US and about 5 million globally. Analysts have projected blockbuster sales for pegcetacoplan in this indication, although those predictions were based on expected success in both the Phase III OAKS and DERBY studies. Roche Holding AG, Novartis AG and Alexion Pharmaceuticals Inc