Boston biotech giant Alexion Pharmaceuticals Inc. has new reason to fear for the future of its blockbuster drug Soliris after a small local biotech showed its drug is more effective in treating a rare blood disorder in a head-to-head trial.
Apellis Pharmaceuticals Inc. (Nasdaq: APLS) announced Tuesday that its lead drug candidate, APL-2, bested Alexion’s blockbuster drug in a Phase 3 trial of patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare disorder that impairs patients’ ability to produce blood cells.
After 16 weeks, participants treated with APL-2 showed an average increase of 2.4 grams of the oxygen-transporting protein hemoglobin, while patients taking Alexion’s drug Soliris had an average decrease of 1.5 grams.
“This study, in my opinion, more than anything established that there is an unmet medical need… We should all be grateful for Soliris being on the market for more than a decade, but there is room for improvement,” CEO Cedric Francois said during an analyst call.
