Alexion Scraps Ultomiris ALS Trial

AstraZeneca’s Alexion drug unit is discontinuing its CHAMPION-ALS Phase 3 clinical trial of Ultomiris (ravulizumab) in adults with amyotrophic lateral sclerosis due to lack of efficacy. The decision is based on the recommendation of the trial’s independent data monitoring committee after it reviewed data from a pre-specified interim analysis. No new safety findings were observed and the data were consistent with the established safety profile of Ultomiris, the company said.

Ultomiris is currently approved in the U.S. for treating adults and children (one month of age and older) with paroxysmal nocturnal hemoglobinuria, and for treating atypical hemolytic uremic syndrome to inhibit complement-mediated thrombotic microangiopathy. The long-acting C5 complement inhibitor works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system, according to the company.