Why Did AstraZeneca's Alexion Stop Amyotrophic Lateral Sclerosis Trial?

  • AstraZeneca Inc's AZN newly acquired Alexion division will stop developing a treatment for the neurological disorder amyotrophic lateral sclerosis (ALS) due to lack of efficacy in a late-stage trial.
  • Following an interim analysis, Alexion says it will scrap a Phase 3 study testing the long-acting C5 inhibitor Ultomiris as a treatment for amyotrophic lateral sclerosis. 
  • Since starting in January 2020, the trial has enrolled 382 patients, per trial records. 
  • All of them will now stop taking study medication, either Ultomiris or placebo, and complete any necessary follow-up evaluations.
  • The Independent Data Monitoring Committee recommended trial discontinuation "due to lack of efficacy," Alexion said, as measured by the ALS functional rating scale-revised (ALSFRS-R) score. 
  • No safety concerns were reported.
  • Related content: Benzinga's Full FDA Calendar.
  • Price Action: AZN shares are up 0.52% at 59.49 during the market session on the last check Friday.
Posted In: Briefsmultiple sclerosisPhase 3 TrialBiotechNewsHealth CareFDAGeneral