Synonyms: (±)-4 | (±)-SMT022357 | compound 4 [Babbs et al., 2020]
Compound class:
Synthetic organic
Comment: SMT022357 is a second-generation, oral utrophin modulator. It was developed from the same chemical series as clinically evaluated ezutromid, but with a new chemotype [1]. SMT022357 has improved physicochemical and ADME properties compared to ezutromid, and has demonstrated efficacy in dystrophin-deficient mdx mice [1-2]. The molecular target of SMT022357 has not been reported, but may be the aryl hydrocarbon receptor, in common with ezutromid.
Clinical rationale: Up-regulation of utrophin (UTRN; P46939) as being investigated as a disease-modifying approach for Duchenne muscular dystrophy (DMD). In the mdx mouse DMD model, increased levels of utrophin effectively acts as a substitute for the dystrophin (DMD; P11532) that is missing in DMD [3]. |
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