Phase 3 Study Initiated For Ravulizumab in Amyotrophic Lateral Sclerosis

A pivotal phase 3 study of ravulizumab (Ultomiris, Alexion Pharmaceuticals, New Haven, CT ) in participants with amyotrophic lateral sclerosis (ALS) has been announced. In the fourth quarter of 2019, the investigational new drug application (IND) was submitted to the Food and Drug Administration (FDA) for ravulizumab for treatment of ALS. The phase 3 trial will be initiated in the first quarter of 2020.

“Given the significant need for new and improved treatments for ALS, we are committed to advancing this clinical program with urgency,” said John Orloff, MD, executive vice president and head of Research & Development at Alexion. “Based on preclinical data and the significant role complement activation is known to play in other neuromuscular diseases, we believe Ultomiris has the potential to inhibit complement-mediated damage in people with ALS, which may slow disease progression. We thank the ALS community for their involvement in designing this phase 3 program and look forward to continued close collaboration as we move it forward.”

The phase 3 Champion-ALS trial is a randomized double-blind, placebo-controlled multicenter global study designed to evaluate the efficacy and safety of ravulizumab across a broad ALS population. The study will enroll approximately 350 adults with sporadic or familial ALS who have had disease onset (in the form of first motor symptoms) within the prior 36 months, demonstrate a slow vital capacity (SVC) of at least 65% predicted, and are not dependent on respiratory support.

“We’ve made great progress advancing ALS research in recent years, but there is still more work to be done to ensure patients benefit from these advances in the form of new treatments,” said Calaneet Balas, CEO and president of The ALS Association. “It’s great that Alexion, which has an established record of bringing new treatments to patients with devastating rare diseases, is joining the fight against ALS.”

The participants will be randomized on a 2:1 basis to receive ravulizumab or placebo every 8 weeks following an initial loading dose. They may be able to continue to receive their existing standard of care treatment for ALS. After 50 weeks, all participants will receive ravulizumab in a 2-year open-label extension phase of the study. The study will be conducted at approximately 90 clinical trial sites across North America, Europe and Asia-Pacific.