Apellis Reports Positive Top-line Results from Phase 3 Head-to-Head Study of Pegcetacoplan (APL-2) Compared to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - Seite 3

About the PEGASUS Study
The PEGASUS study (APL2-302; NCT03500549) is a multi-center, randomized, open-label, active-comparator controlled Phase 3 study in 80 adults with 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 3 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 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. All participants completing the randomized controlled period entered the open-label pegcetacoplan treatment period where they received pegcetacoplan, regardless of the prior treatment received in the randomized, controlled period.

Key secondary endpoints were tested in a hierarchical manner after statistical significance was reached for the primary endpoint. Key secondary endpoints were tested first for non-inferiority and, if all were met, then superiority was tested sequentially for transfusion avoidance, absolute reticulocyte count, and FACIT-fatigue score. Once one hypothesis was tested as not significant, all subsequent tests were not assessed. Estimates were computed for all key secondary and secondary endpoints regardless of whether a hypothesis was tested.

The study was conducted in collaboration with SFJ Pharmaceuticals, who supported the development of pegcetacoplan in PNH. SFJ is a global drug development company, which provides a unique and highly customized co-development partnering model for the world’s top pharmaceutical and biotechnology companies.

About Pegcetacoplan (APL-2)
Pegcetacoplan is an investigational, targeted C3 inhibitor designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies including paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (GA), and C3 glomerulopathy. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of PNH and the treatment of GA. For additional information regarding our clinical trials, visit www.apellis.com/clinical-trials.html.