Apellis Pharmaceuticals, Inc. and Sobi(R) reported new analyses of Phase 3 studies that reinforce the robust efficacy and safety profile of EMPAVELI®/Aspaveli® (pegcetacoplan) for paroxysmal nocturnal hemoglobinuria. The data will be presented at the hybrid European Hematology Association 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 showed significant improvements in clinical outcomes in treatment-naïve patients who received EMPAVELI compared to C5 inhibitors. 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.