Drug intelligence / Profile preview

sevasemten

Development stage
Phase 2
Lead developer
Edgewise Therapeutics
Modality
Small Molecules
Administration
Oral
01

Overview

Sevasemten is an orally administered, first-in-class small molecule inhibitor of fast skeletal myosin. It is designed to protect muscle against contraction-induced damage in muscular dystrophies such as Becker and Duchenne muscular dystrophy. By selectively inhibiting skeletal muscle myosin, sevasemten reduces muscle damage biomarkers and fibrosis while increasing muscle strength and activity. The drug aims to minimize progressive muscle damage that leads to functional impairment in patients lacking fully functional dystrophin protein. Sevasemten has demonstrated significant reductions in biomarkers of skeletal muscle injury (such as creatine kinase and fast skeletal troponin I) and stabilization or improvement of motor function assessments in clinical trials for Becker muscular dystrophy. Developed by Edgewise Therapeutics, it is currently being evaluated in late-stage clinical trials for both Becker and Duchenne muscular dystrophies[1][3][5][8].

Other names
2-[(5-fluoropyridin-3-yl)methyl]-6-[2-(2,2,2-trifluoroethoxy)pyrimidin-5-yl]pyridazin-3-one2417395-15-2
02

Targets

Skeletal muscle myosin (SkM-Myosin)

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