Dear ALS Community,

Today, we are sharing the disappointing news that we have made the difficult decision to discontinue the CHAMPION-ALS Phase III trial, based on the recommendation of the Independent Data Monitoring Committee (IDMC), following their review of data from the pre-planned interim analysis. The IDMC recommended the trial be discontinued due to lack of efficacy. For more information, please see the pressrelease here.

We are disappointed by this outcome and what it means for patients with this devastating disease and your families.

We would like to sincerely thank the entire ALS community as well as investigators and healthcare professionals who dedicated their time and expertise to this trial. From early protocol development, we partnered with the community to design the CHAMPION-ALS trial with a direct- to-Phase III approach to allow for a faster development pathway leading to robust scientific answers as quickly as possible given the significant and urgent treatment need.

Patients who enrolled in the global trial will discontinue study medication and complete any necessary follow-up evaluations. Data from the trial will be provided to the research community to inform future scientific efforts. There were no new safety findings observed and the data were consistent with the established safety profile of Ultomiris (ravulizumab).

We remain confident in the potential of targeting C5 for complement-driven diseases and remain fully committed to our efforts to serve the rare disease community.

Thank you for your support and insight to this program. It is with our sincere admiration and appreciation that we thank all those who volunteered to participate in this study.

Source: patient advocacy mailbox Alexion