Apellis Pharmaceuticals announced that a mid-stage trial investigating systemic pegcetacoplan for the treatment of amyotrophic lateral sclerosis (ALS) failed to meet endpoints, halting the drugmaker's chances of an approval trifecta, at least in the near future.
According to the Massachusetts-based biopharma and its partner, Swedish orphan drug specialist, Sobi, the phase 2 MERIDIAN study did not meet its primary endpoint of the Combined Assessment of Function and Survival (CAFS) rank score at Week 52. The study also did not meet key secondary efficacy endpoints.
Based on the lack of efficacy, Apellis and Sobi plan to discontinue development of systemic pegcetacoplan for ALS. The news doesn't come as a surprise after the partners discontinued treatment in the open-label portion of the study following a recommendation from an independent data monitoring committee, back in April.
Pegcetacoplan is already approved by the U.S. FDA in two indications.
In May 2021, the targeted C3 therapy, branded Empaveli, got the nod for paroxysmal nocturnal hemoglobinuria, a rare, life-threatening blood disease. This past February, under the brand name Syfovre, the drug won approval in a second indication, geographic atrophy — a leading cause of blindness.
Pegcetacoplan remains under investigation for several rare diseases across hematology, nephrology and neurology.
