- A strong showing in a Phase 3 head-to-head trial in patients with rare blood disease has sent shares of the small biotech Apellis Pharmaceuticals soaring.
Shares of Apellis Pharmaceuticals (APLS) rocketed Tuesday after the biotech company's experimental drug outperformed Alexion Pharmaceuticals' (ALXN) blockbuster, Soliris, in a head-to-head test.
The biotech company tested its drug, dubbed pegcetacoplan, in patients with a blood disorder called paroxysmal nocturnal hemoglobinuria, or PNH. A rare disorder, PNH destroys red blood cells and blood clots, and impairs bone marrow function.
At week 16 of the study, patients who received pegcetacoplan showed a stronger increase in hemoglobin vs. those who took Soliris, Apellis said in a news release. Hemoglobin is a protein responsible for transporting oxygen in the blood.
"Pegcetacoplan is the first and only investigational therapy to demonstrate superiority compared to (Soliris) on hemoglobin levels," Apellis Chief Medical Officer Federico Grossi said in a written statement. "We are also excited to see 85% of patients treated with pegcetacoplan were transfusion free."
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