WALTHAM, Mass. and STOCKHOLM, Sweden, June 10, 2022 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Sobi® (STO:SOBI) today reported new analyses of Phase 3 studies that reinforce the robust efficacy and safety profile of EMPAVELI®/Aspaveli® (pegcetacoplan) for paroxysmal nocturnal hemoglobinuria (PNH). The data will be presented at the hybrid European Hematology Association (EHA) Congress in Vienna, Austria.
New analyses demonstrated that treatment with EMPAVELI resulted in meaningful improvements in quality of life for treatment-naïve patients and suggested the incidence of thrombosis was comparable to eculizumab, a C5 inhibitor. Additionally, a matching-adjusted indirect comparison (MAIC) showed significant improvements in clinical outcomes in treatment-naïve patients who received EMPAVELI compared to C5 inhibitors.
“The data presented at EHA add to a growing body of evidence, which shows that EMPAVELI leads to both clinically meaningful efficacy and improved quality of life regardless of prior treatment,” said Peter Hillmen, M.B. Ch.B., Ph.D., head of hematology engagement at Apellis. “Many patients experience a significant disease burden, even with C5 inhibitor treatment, so these data further emphasize that EMPAVELI has the potential to become a new standard of care for PNH.”
“We are very pleased that these new data further reinforce the safety and efficacy of EMPAVELI/Aspaveli in treating such a rare, chronic, and life-threatening condition,” said Anders Ullman, head of research and development and chief medical officer at Sobi. “Sobi and Apellis are firmly committed to improving the care and quality of life for those affected by this rare blood disease.”
EMPAVELI Demonstrated Meaningful Improvements in Quality of Life in Treatment-Naïve Patients
In an analysis of the Phase 3 PRINCE study, EMPAVELI patients who were previously treatment-naïve demonstrated meaningful quality-of-life improvements through 26 weeks, reaching normal or near-normal levels of the general population. These data, which were assessed using multiple measures including the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 scale, will be reported during an oral presentation at the congress.
Results Suggested Incidence of Thrombosis Comparable across EMPAVELI- and Eculizumab-Treated Patient Groups
A post hoc analysis of data from all PNH clinical trials of EMPAVELI revealed that there were 1.54 thrombotic events per 100 patient-years for EMPAVELI-treated patients compared to 1.77 thrombotic events per 100 patient-years for eculizumab-treated patients prior to entry in the Phase 3 PEGASUS study.
Additionally, D-dimer normalization was comparable across EMPAVELI- and eculizumab-treated patient groups in a post hoc analysis of the Phase 3 studies. D-dimer is a marker of thrombotic risk, one of the most common life-threatening complications of PNH.
EMPAVELI Demonstrated Significant Improvements in Clinical Outcomes Versus C5 Inhibitors in Treatment-Naïve Patients
Using a MAIC methodology, individual patient data from the Phase 3 PRINCE study were compared to aggregate, published data from the ALXN1210-PNH-301 study,1 which compared C5 inhibitors ravulizumab and eculizumab in PNH patients who were treatment naïve.
Patients treated with EMPAVELI showed significant improvements compared to C5 inhibitors across all key disease measures evaluated, including lactate dehydrogenase normalization, hemoglobin stabilization, and transfusion avoidance at Week 26.
In the absence of a clinical head-to-head study, MAIC is a valid and accepted method for comparative effectiveness research used by health technology assessment bodies across the world.2,3 As with other MAIC analyses, matching may not adjust for all confounding factors due to differences inherent in study design and entry criteria. Key limitations include differences in the route of administration, treatment administration schedule, and dosing regimen.
About EMPAVELI®/Aspaveli® (pegcetacoplan)EMPAVELI®/Aspaveli® (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. It is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in the United States, Australia, and Saudi Arabia as EMPAVELI and in the European Union and the United Kingdom as Aspaveli. The therapy is also under investigation for several other rare diseases across hematology, nephrology, and neurology.
About the PRINCE StudyThe PRINCE study (NCT04085601) was 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®/Aspaveli® (pegcetacoplan) in patients who had 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 escape to the EMPAVELI group if their hemoglobin decreased by 2 g/dL or more from their baseline value.
About the PEGASUS StudyThe PEGASUS study (NCT03500549) was a multi-center, randomized, open-label, 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® compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a hemoglobin level of