Edgewise Therapeutics’ lead candidate has been shown to reduce levels of a biomarker related to skeletal muscle damage in a phase 2 trial.
The biotech had been testing sevasemten, an orally administered fast skeletal myosin inhibitor, in a trial of 40 adults and 29 adolescents with Becker muscular dystrophy. Edgewise described the CANYON study as the largest interventional trial to date for Becker, a genetic disorder that causes progressive muscle weakness and wasting.
The study hit the primary endpoint of showing a “significant change” in levels of creatine kinase (CK) in the adult cohort, with a 28% average decrease in CK levels compared to placebo between six and 12 months.
The company was less successful on the secondary endpoint of a change in motor function, with the North Star Ambulatory Assessment (NSAA) score remaining stable in the sevasemten group. Edgewise was only able to show “a trend towards improvement at 12 months” for this measurement, pointing out in the release that, “notably, the treatment population had more advanced disease than placebo.”
Investors seemed unfazed by the less clear-cut secondary endpoint data, sending Edgewise’s stock up 17% to $32.28 in premarket trading Monday from a Friday closing price of $27.62.
Edgewise will use NSAA as the primary endpoint of GRAND CANYON, another phase 2 trial in Becker that is due to complete recruitment by the first quarter of 2025. Should that study also hit its goal, the biotech is planning to begin discussions with the FDA about an approval application.
“We are very encouraged by the CANYON results in Becker and the potential of this novel muscle-targeted therapeutic,” Chief Medical Officer Joanne Donovan, M.D., Ph.D., said in the release. “This confirmed our previous observations in the ARCH study of significant decreases in biomarkers of muscle damage and similarly we are seeing evidence of preservation of function in Becker patients.”
There are currently no approved treatments for Becker, although Santhera Pharmaceuticals has been evaluating its approved Duchenne muscular dystrophy corticosteroid Agamree in an FDA-funded phase 2 study for Becker.