Apellis update in ph 3 study of Pegcetacoplan in Treatment-Naïve Patients with PNH

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 United States and the European Union in the second half of 2020.

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. 

Apellis to submit NDA for Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria (PNH) in 2H-2020

The PEGASUS study met its primary endpoint, demonstrating the superiority of pegcetacoplan to eculizumab with a statistically significant improvement in hemoglobin levels at 16 weeks. Top-line results also showed that the safety profile of pegcetacoplan was comparable to eculizumab in the study. As previously announced, results from the PEGASUS study have been selected for an oral presentation at the 25th Congress of the European Hematology Association and will be available on June 12, 2020.