Phase 3 Trial of New Drug To Treat Progressive MS Approved by FDA

The Food and Drug Administration (FDA) approved a phase 3 clinical trial (NCT05441488) for masitinib (AB Science; Paris, France) as a potential treatment for progressive forms of multiple sclerosis (MS), including primary progressive MS and nonactive secondary progressive MS. 

This randomized, double-blind study is actively recruiting partipants at 75 sites around the globe. Researchers expect to enlist up to 800 paricipants who have been diagnosed with progressive forms of MS and no active brain lesions to confirm results from earlier phase 2b/3 studies which demonstrated that masitinib was effective at delaying progression of disability. Participants in the study will be assigned randomly to take either twice daily doses of masitinib (4.5 mg/kg/d) or a placebo for 2 years. The main outcome will be disability progression as measured by the Expanded Disability Status Score; secondary outcomes include changes in brain volume and lesions and self-reported quality of life measurements. 

Professor Patrick Vermersch, MD, principal investigator of the study and professor of Neurology at the University of Lille, France said: “We are very pleased to receive FDA approval for this confirmatory study. This demonstrates interest from the health authorities in the masitinib program in progressive forms of multiple sclerosis and more broadly in neurodegenerative diseases.”

Results from the previous phase 3 study (NCT01433497) showed that individuals with various forms of progressive MS who took masitinib were less likely to experience worsening in their disability scores compared with those who took a placebo. Masitinib also is being studied for potential use as a treatment for other neurologic conditions, including amyotrophic lateral sclerosis and Alzheimer disease. Masitinib is a tyrosine kinase inhibitor that targets the c-Kit pathway.