Mallinckrodt halts Phase 2B trial investigating use of Acthar gel in ALS
Mallinckrodt announced that it is permanently discontinuing its Phase 2B study designed to assess the efficacy and safety of Acthar Gel as an investigational treatment for amyotrophic lateral sclerosis, or ALS. The drug is not FDA-approved for the ALS indication. Mallinckrodt made the decision to halt the trial after careful consideration of a recent recommendation by the study's independent Data and Safety Monitoring Board. The recommendation was based on the specific concern for pneumonia, which occurred at a higher rate in the ALS patients receiving Acthar Gel compared to those on placebo; the board also mentioned other adverse events specific to this patient population. The DSMB noted the proportion of patients who have completed Week 36 - the primary endpoint target - precludes a definitive determination of a treatment effect. The lack of a clear efficacy signal for this ALS patient population combined with the potential risk of pneumonia led to the board's recommendation.