- Results from positive Phase 3 PRINCE study in treatment-naïve patients with PNH accepted for oral presentation
- In new analysis, EMPAVELI demonstrated clinically meaningful improvements in key markers of disease in PNH patients with baseline hemoglobin levels greater than or equal to 10.0 g/dL
WALTHAM, Mass., Nov. 04, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in complement, today announced that five abstracts were accepted for presentation at the American Society of Hematology (ASH) Annual Meeting to be held December 11-14, 2021. The new data further support the positive efficacy and safety profile of EMPAVELI, the first and only targeted C3 therapy, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
Highlights include the first oral presentation of the positive Phase 3 PRINCE study of EMPAVELI in adults with PNH who are treatment-naïve, meaning they had not received a complement inhibitor within three months before entering the study. As previously reported, EMPAVELI demonstrated superiority in both hemoglobin stabilization and reduction in lactate dehydrogenase (LDH) compared to standard of care, which did not include complement inhibitors, at Week 26.
Additionally, in a new post hoc analysis of the PRINCE, PEGASUS, and PADDOCK studies, EMPAVELI demonstrated clinically meaningful improvements in key markers of disease in PNH patients with pre-study hemoglobin levels greater than or equal to 10.0 g/dL. This highlights the potential of EMPAVELI to elevate the standard of care for all patients with PNH regardless of previous hemoglobin levels. The analysis included treatment-naïve patients and patients who were taking eculizumab, a C5 inhibitor. “The data that we are presenting at ASH underscore our leadership in PNH as well as the potential of EMPAVELI to redefine treatment for all adults with this debilitating disease,” said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. “EMPAVELI demonstrated superiority to eculizumab in improving hemoglobin levels in PNH in our PEGASUS study and further evidence shows that EMPAVELI provides meaningful improvements in the treatment-naïve patient population.”
Accepted abstracts at ASH 2021 include:
- Oral presentation: Efficacy and Safety of Pegcetacoplan Treatment in Complement-Inhibitor Naïve Patients with Paroxysmal Nocturnal Hemoglobinuria: Results from the Phase 3 Prince Study – #606 – December 13, 11:45 AM ET, Georgia World Congress Center, Georgia Ballroom 1-3, in collaboration with Sobi
- Poster presentation: Post Hoc Analysis of the Effect of Pegcetacoplan Treatment of Patients with Paroxysmal Nocturnal Hemoglobinuria and Baseline Hemoglobin Levels Greater Than 10 Grams per Deciliter – #2194 – December 12, 6:00 PM - 8:00 PM ET, in collaboration with Sobi
- Poster presentation: Evaluation of the Long-Term Safety and Efficacy of Pegcetacoplan Treatment for Paroxysmal Nocturnal Hemoglobinuria Patients: An Extension Study – #2175 – December 12, 6:00 PM - 8:00 PM ET, in collaboration with Sobi
- Poster presentation: Categorized Hematologic Response to Pegcetacoplan and Correlations with Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria: Post Hoc Analysis of Data from Phase 1b, Phase 2a, and Phase 3 Trials – #1104 – December 11, 5:30 PM - 7:30 PM ET
- Poster presentation: Changes in Hemoglobin Measures Observed in PNH Patients Treated with Both C5 Inhibitors Ravulizumab and Eculizumab: Real-World Evidence from a US-Based EMR Network – #1112 – December 11, 5:30 PM - 7:30 PM ET
About the PRINCE StudyThe PRINCE study (NCT04085601) is a randomized, multi-center, open-label, controlled Phase 3 study in 53 treatment-naïve adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of EMPAVELI™ (pegcetacoplan) in patients who have not received treatment with any complement inhibitor within three months prior to screening. During the 26-week randomized, controlled period, patients received either 1080 mg of EMPAVELI twice weekly or standard of care therapy, which did not include complement inhibitors. Patients in the standard of care group had the option to receive EMPAVELI as escape therapy if their hemoglobin decreased by 2 g/dL or more from their baseline value.
About the PEGASUS StudyThe PEGASUS study (NCT03500549) is a multi-center, randomized, head-to-head Phase 3 study in 80 adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of EMPAVELI compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a hemoglobin level of