Apellis up on new clinical data for rare blood disease candidate

Shares of US biopharma company Apellis Pharmaceuticals (Nasdaq: APLS) were up more than 5% in pre-market trading Friday, after it released detailed results from the Phase III PEGASUS study, which showed superiority (p<0.0001) for pegcetacoplan over eculizumab in improving hemoglobin levels in adults with paroxysmal nocturnal hemoglobinuria (PNH).

Eculizumab is the active ingredient of Alexion Pharmaceuticals’ (Nasdaq: ALXN) blockbuster drug Solaris, which last year generated sales of $3.9 billion.

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 at the 25th Congress of the European Hematology Association (EHA).