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Apellis Completes Enrollment in Phase 3 Study of Pegcetacoplan in Treatment-Naïve Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)
PRINCE study enrolled 53 patients. Trial designed to further establish the potential of pegcetacoplan, a targeted C3 therapy, in all patients living with
About this update from Apellis Pharmaceuticals, Inc.
[{"type":"text","content":"PRINCE study enrolled 53 patients. Trial designed to further establish the potential of pegcetacoplan, a targeted C3 therapy, in all patients living with PNHTop-line results expected in early 2021 WALTHAM, Mass., July 02, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today 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.\n “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, M.D., Ph.D., 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 United States and the European Union in the second half of 2020. For more information about the Phase 3 PRINCE study, visit www.clinicaltrials.gov (NCT04085601). About Pegcetacoplan (APL-2) Pegcet...