Apellis complete enrollment of Phase 3 APL-2 head-to-head study
Apellis Pharmaceuticals announced the completion of enrollment for its PEGASUS Phase 3 trial assessing the safety and efficacy of APL-2 in patients with paroxysmal nocturnal hemoglobinuria compared to eculizumab. The study enrolled 80 patients, approximately half of whom were transfusion dependent. Apellis expects to release top-line data in December 2019. PEGASUS is a randomized, active-controlled study comparing APL-2 monotherapy to eculizumab monotherapy in patients currently on treatment with eculizumab who have a hemoglobin level less than10.5 g/dL, regardless of eculizumab dose or transfusion history. In the trial, patients are co-treated for 4 weeks with APL-2 and eculizumab, and then randomized to either APL-2 monotherapy or eculizumab monotherapy for a 16-week randomized controlled period. The primary endpoint is change from baseline to week 16 in hemoglobin level. Key secondary endpoints include transfusion avoidance during the 16-week randomized controlled period, as well as week 16 changes from baseline in reticulocyte count, lactate dehydrogenase levels and FACIT-Fatigue scale score. The 16-week randomized controlled period is followed by a 32-week open-label treatment period in which all subjects will receive treatment with APL-2 monotherapy. APL-2 is being developed for patients with PNH, autoimmune hemolytic anemia, complement-associated kidney diseases, and geographic atrophy. Cumulative systemic exposure is over 50 patient years of treatment on APL-2. There have been no serious adverse drug reactions in the systemic development programs for hematology indications to date.