New Pivotal Data Demonstrate Superiority of Apellis’ Pegcetacoplan to Eculizumab in Improving Hemoglobin Levels, Independent of Prior Transfusions, in PNH

• 71% of pegcetacoplan-treated patients achieved LDH normalization vs. 15% of eculizumab-treated patients
  
  
• 73% of pegcetacoplan-treated patients achieved a clinically meaningful improvement in FACIT-fatigue score vs. 0% of eculizumab-treated patients
   
• Detailed results from positive head-to-head Phase 3 PEGASUS study in patients with paroxysmal nocturnal hemoglobinuria (PNH) presented in oral session at 25^th Congress of the European Hematology Association

• NDA and MAA submissions planned in H2 2020. Conference call scheduled today at 8:30 a.m. ET

WALTHAM, Mass., June 12, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today presented detailed results from the Phase 3 PEGASUS study, which in January showed superiority (p<0.0001) for pegcetacoplan over eculizumab in improving hemoglobin levels in adults with paroxysmal nocturnal hemoglobinuria (PNH). New data from the pivotal study showed that pegcetacoplan’s effect was seen consistently across the study population, both in patients who had low or no transfusion requirements (fewer than four transfusions in the 12 months before study entry) and high transfusion requirements (four or more transfusions). Pegcetacoplan also demonstrated a robust response across several key hematologic and clinical measures for PNH. The results were presented in a scientific oral presentation at the 25^th Congress of the European Hematology Association (EHA).

Pegcetacoplan met the study’s primary endpoint for efficacy, 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), 53% higher than the eculizumab arm.

• In patients with low or no transfusion requirements, pegcetacoplan-treated patients (n=20) had an adjusted mean hemoglobin increase of 2.97 g/dL vs. eculizumab-treated patients (n=16) who had a mean change of -0.01 g/dL from the 8.9 g/dL baseline.
   
• In patients with high transfusion requirements, pegcetacoplan-treated patients (n=21) had an adjusted mean hemoglobin increase of 2.11 g/dL vs. eculizumab-treated patients (n=23) who had a mean change of -4.02 g/dL from the 8.5 g/dL baseline.¹

“The PEGASUS results showed that pegcetacoplan, our targeted C3 therapy, demonstrated a substantial improvement over C5 inhibition in PNH,” said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. “These data reinforce the potential for pegcetacoplan to elevate the standard of care in PNH, and we are in discussions with regulatory authorities to bring pegcetacoplan to the PNH community as quickly as possible.”