Apellis to Present New Data Supporting the Efficacy and Safety of Pegcetacoplan in PNH at the American Society of Hematology Annual Meeting - Seite 2

• Real-World Eculizumab Dosing Patterns among Patients with Paroxysmal Nocturnal Hemoglobinuria in a US Population – #3412 – December 7, 7:00 a.m. – 3:00 p.m. PT
• Real-World Treatment Patterns and Healthcare Resource Utilization (HRU) of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Receiving Eculizumab in a US Population – #3415 – December 7, 7:00 a.m. – 3:00 p.m. PT

About the PEGASUS Study
The PEGASUS study (APL2-302; NCT03500549) is a multi-center, randomized, active-comparator controlled 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 pegcetacoplan compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a hemoglobin level of <10.5 g/dL at the screening visit. During the four-week run-in, patients were dosed with 1080 mg of pegcetacoplan twice weekly (n=41) in addition to their current dose of eculizumab. During the 16-week randomized, controlled period, patients were randomized to receive either 1080 mg of pegcetacoplan twice weekly or their current dose of eculizumab (n=39). All participants completing the randomized controlled period entered the open-label pegcetacoplan treatment period.

About the Matching-Adjusted Indirect Comparison Analysis
Using a matching-adjusted indirect comparison (MAIC) methodology, individual patient data from the PEGASUS study were compared to aggregate, published results from the ALXN1210-PNH-302 study, which compared ravulizumab and eculizumab among patients with PNH who previously were treated with eculizumab. To adjust for cross-study differences in baseline characteristics, propensity score weighting was used to balance demographic and clinical characteristics. Outcomes assessed from the PEGASUS study at week 16 and the ALXN1210-PNH-302 study at week 26 included transfusion avoidance, number of units transfused, hemoglobin stabilization and change in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score. As with other MAIC analyses, matching may not adjust for all confounding factors due to differences inherent in study design and entry criteria.