- Rapid improvements in key markers of disease were sustained for up to three years across all adults with PNH
- Majority of patients remained transfusion free over the long term
- Zero cases of meningococcal infection observed over three years in this analysis
- Data shared during an oral presentation
WALTHAM, Mass., Dec. 11, 2023 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Sobi today presented post hoc data that reinforce the long-term efficacy and safety of EMPAVELI® (pegcetacoplan) in adults with paroxysmal nocturnal hemoglobinuria (PNH) for up to three years. The data were reported during an oral presentation at the American Society of Hematology (ASH) Annual Meeting.
“These results show that treatment with EMPAVELI can help PNH patients achieve rapid and sustained control of their disease over the long term,” said Carlos de Castro, M.D., presenting author and professor of medicine, Duke University. “Furthermore, it is impressive that the majority of patients remained transfusion free up to three years, alleviating a significant and common disease burden for many people living with PNH.”
The analysis integrated data across the Phase 3 PEGASUS and PRINCE studies and the long-term extension study. After starting treatment with EMPAVELI, key markers of disease rapidly improved and were sustained in both treatment-naïve patients and patients previously treated with eculizumab. Improvements in hemoglobin reached normal or near-normal levels, and mean lactate dehydrogenase (LDH) was maintained below the upper limit of normal.
Additionally, 67% of treatment-naïve patients from PRINCE were transfusion free for up to 2.5 years, and 52% of patients from PEGASUS remained transfusion free for up to 3 years. Less than 25% of patients were transfusion free in the year prior to entering the PRINCE and PEGASUS studies.
The safety profile was consistent with previous clinical study results, and no new or unexpected safety findings were identified. Approximately 4.5% of patients experienced a serious adverse event deemed related to treatment with pegcetacoplan. No meningococcal infections were reported.
The prescribing information for EMPAVELI contains a boxed warning, which states that EMPAVELI may increase the risk of meningococcal and other serious infections caused by encapsulated bacteria that may become rapidly life threatening or fatal if not recognized and treated early.
“The strength of these long-term EMPAVELI results highlight why we are seeing compliance rates of 97% with real-world use,” said Peter Hillmen, M.B. Ch.B., Ph.D., head, rare disease advisor, Apellis. “By achieving and maintaining normal clinical measures over time, EMPAVELI has the potential to elevate the standard of care for all adults with PNH."
About the Long-Term Efficacy and Safety Extension (APL2-307) StudyThe APL2-307 study was a nonrandomized, multicenter, open-label Phase 3 extension study of 137 adults with paroxysmal nocturnal hemoglobinuria (PNH) who completed previous EMPAVELI®/Aspaveli® (pegcetacoplan) Phase 1 (PHAROAH, PADDOCK), Phase 2 (PALOMINO), and Phase 3 (PEGASUS, PRINCE) clinical trials. Patients in these studies were either anemic despite eculizumab treatment or were naïve to complement inhibitors. During the trial, patients continued to receive 1080 mg of EMPAVELI twice weekly or once every three days (PEGASUS, PRINCE) or switched to 1080 mg of EMPAVELI twice weekly (PHAROAH, PADDOCK, PALOMINO). The primary objective was to establish the long-term efficacy and safety of EMPAVELI.
About the PEGASUS StudyThe PEGASUS study (APL2-302; NCT03500549) was a randomized, multi-center, 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®/Aspaveli® (pegcetacoplan) compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a hemoglobin level of