Apellis Completes Enrollment in Phase 3 Study of Pegcetacoplan

Apellis Pharmaceuticals, Inc. recently announced the completion of enrollment in the global Phase 3 PRINCE study, which is evaluating pegcetacoplan (APL-2) in patients with paroxysmal nocturnal hemoglobinuria (PNH) who are treatment-naïve, meaning they had not received a complement inhibitor within three months before entering the trial.

“Patient safety during this global pandemic has been our top priority as we worked to finalize enrollment in this trial, and we’d like to thank the patients and their families for participating in the PRINCE study as well as investigators for helping to ensure the well-being of all study participants,” said Federico Grossi, MD, PhD, Chief Medical Officer. “PNH patients can suffer debilitating fatigue and transfusion dependence even when treated with currently available therapies. Based on positive data announced earlier this year, we believe pegcetacoplan, our targeted C3 therapy, has the potential to redefine treatment for all patients with PNH by controlling both intravascular and extravascular hemolysis.”

The PRINCE study is a Phase 3, randomized, multicenter, open-label, controlled trial. The study was designed to evaluate the efficacy of pegcetacoplan in 48 adult patients who are treatment-naïve, showed evidence of hemolysis (elevated LDH ≥ 1.5x ULN) and had hemoglobin levels that were less than the lower limit of normal at the time of their screening. Primary outcome measures, to be evaluated after 26 weeks of treatment, include hemoglobin stabilization in the absence of transfusion and reduction in lactate dehydrogenase (LDH) level. A total of 53 patients were enrolled in the study.

Based on positive results from the pivotal PEGASUS study of pegcetacoplan in PNH, the company plans to submit marketing applications for pegcetacoplan for the treatment of PNH both in the US and the European Union in the second half of 2020. For more information about the Phase 3 PRINCE study, visit www.clinicaltrials.gov (NCT04085601).

Pegcetacoplan is an investigational, targeted C3 inhibitor designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies including paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (GA), cold agglutinin disease, and C3 glomerulopathy. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of PNH and the treatment of GA. For additional information regarding our clinical trials, visit www.apellis.com/clinical-trials.html.

PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue and difficulty breathing (dyspnea). Retrospective studies show that, even on eculizumab, approximately 70% of people with PNH have low hemoglobin levels,^1,2 and 36% require one or more transfusions a year.²

Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. By pioneering targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology. For more information, please visit http://apellis.com.