ezutromid

Ezutromid is an orally administered small molecule utrophin modulator developed for the treatment of Duchenne muscular dystrophy (DMD), a severe X-linked recessive disorder characterized by progressive muscle degeneration due to mutations in the dystrophin gene. Ezutromid acts by upregulating utrophin, a protein structurally and functionally similar to dystrophin, with the aim of compensating for the lack of functional dystrophin in DMD patients. Mechanistically, ezutromid functions as an antagonist of the aryl hydrocarbon receptor (AhR), which leads to increased expression of utrophin RNA and protein. Preclinical studies demonstrated improved muscle function in animal models, and early clinical trials showed safety and tolerability; however, later-stage clinical development was discontinued after failing to demonstrate efficacy in slowing disease progression. The drug was developed by Summit Therapeutics and received orphan drug designation from both FDA and EMA[1][3][4][5][6][7].

Other names

ezutromidSMTC-1100SMTC1100SMTC 1100