Utrophin is a protein that is found naturally in muscles in small quantities. Utrophin has the potential to operate as a substitute for dystrophin if muscle cells can be encouraged to produce more utrophin (up regulation).

Utrophin upregulation naturally occurs in dystrophin-deficient muscle fibers and can be enhanced through pharmacologic therapy. Several drugs have been developed and tested as utrophin up-regulators.

SMTC1100 is a novel small molecule that has been shown to increase utrophin production and reverse dystrophic pathology in the muscles of mdx (dystrophin deficient) mice. SMT C1100 is currently in early phase clinical trials, with the bio-tech, Summit Therapeutics.

Biglycan is a small, naturally occurring extracellular matrix protein naturally found in muscle. Biglycan activates a utrophin-based compensatory pathway that that can potentially overcome the damaging effects of dystrophin loss. Muscle health, structure and function are improved in mdx mice that are dosed with Biglycan. Tivorsan Pharmaceuticals is planning to initiate clinical trials with Biglycan in early 2017.

Clinical stage

Summit Therapeutics

SMT C1100 (Ezutromid): phase 2

  • Trial currently active. Has recruited 40 males between ages 5 and 10.
  • The 48 week trial is assessing the activity and safety of utrophin modulation with SMT C1100.
  • The study is being conducted across multiple centres in both the UK and USA.