SMT022357

**SMT022357** is a second-generation small molecule utrophin modulator developed by Summit Therapeutics for the treatment of Duchenne muscular dystrophy (DMD). It upregulates endogenous utrophin expression in skeletal, respiratory, and cardiac muscles via oral administration, addressing the underlying dystrophin deficiency by enhancing sarcolemmal stability and restoring dystrophin-associated protein complex components like β-dystroglycan and dystrobrevin. Preclinical studies in mdx mice demonstrated increased utrophin localization along the entire muscle fiber length, independent of fiber type or regeneration, leading to reduced serum creatine kinase, centrally nucleated fibers, necrosis, fibrosis, and contraction-induced force drop, with benefits observed in diaphragm and heart tissues.

Other names

SMT 022357SMT022357SMT-022357