Servier has announced topline results of its phase 2 study of sevasemten in which no statistically significant benefits were observed in treating Becker muscular dystrophy (BMD).
BMD is a rare, genetic, X-linked recessive disorder that causes slowly progressive muscle degeneration and weakness. In the GRAND CANYON study, sevasemen failed to demonstrate a statistically-significant benefit on the North Star Ambulatory Assessment (NSAA) scale, designed to measure gross motor function and walking ability, compared to a placebo. It also did not meet any of the secondary endpoints. However, it did have a favorable safety and tolerability profile.
Sevasemen is an orally-administered, first-in-class, fast skeletal myosin inhibitor designed to preserve and protect unstable muscle against contraction-induced damage in individuals living with rare muscular dystrophy. Servier acquired the drug from Edgewise Therapeutics in June, paying $1.55 billion upfront. In addition to being tested for BMD, sevasemen is in phase 2 trials for Duchenne muscular dystrophy.