Alexion, AstraZeneca Rare Disease, is discontinuing CHAMPION-ALS, the global Phase III clinical trial of Ultomiris (ravulizumab) in adults with amyotrophic lateral sclerosis (ALS).

Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc.

“We are disappointed by this outcome and what it means for patients with this devastating disease. We would like to thank the entire ALS community as well as investigators and healthcare professionals who dedicated their time and expertise to this trial. We continue to be confident in the potential of targeting C5 for complement-driven diseases and remain fully committed to our efforts to serve the rare disease community,” says Gianluca Pirozzi, Senior Vice President, Head of Development and Safety, Alexion.

Based on the recommendation of the IDMC

This decision is based on the recommendation of the Independent Data Monitoring Committee (IDMC), following their review of data from a pre-specified interim analysis. The IDMC recommended that the trial be discontinued due to lack of efficacy. No new safety findings were observed and the data were consistent with the established safety profile of Ultomiris.

Patients who enrolled in the trial will discontinue study medication and complete any necessary follow-up evaluations. Data from the trial will be provided to inform ongoing research, states the company.

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