Apellis Reports Positive Top-line Results from Phase 3 Head-to-Head Study of Pegcetacoplan (APL-2)

News Release

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)

  • Pegcetacoplan met the primary endpoint in the PEGASUS study, demonstrating superiority to eculizumab (p<0.0001) with an improvement in adjusted means of 3.8 g/dL of hemoglobin at week 16
  • Safety profile of pegcetacoplan was comparable to eculizumab in this study
  • Conference call scheduled today at 8:30 a.m. ET

WALTHAM, Mass., Jan. 07, 2020 (GLOBE NEWSWIRE) —  Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced positive results from the Phase 3 PEGASUS study evaluating pegcetacoplan (APL-2) in adults with paroxysmal nocturnal hemoglobinuria (PNH). Top-line data show that pegcetacoplan met the study’s primary efficacy endpoint, demonstrating superiority to eculizumab with a statistically significant improvement in adjusted means of 3.8 g/dL of hemoglobin at week 16 (p<0.0001). At week 16, pegcetacoplan-treated patients (n=41) had an adjusted mean hemoglobin increase of 2.4 g/dL from a baseline of 8.7 g/dL, compared to eculizumab-treated patients (n=39) who had a change of -1.5 g/dL from a baseline of 8.7 g/dL.

Additionally, pegcetacoplan showed promising results in key secondary endpoints. Pegcetacoplan met non-inferiority on transfusion avoidance and absolute reticulocyte count. Pegcetacoplan also showed positive trends on lactate dehydrogenase (LDH) and fatigue as measured by the Functional Assessment of Chronic Illness Therapy (FACIT)-fatigue score.

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