U.K. Biotech Summit received a $750,000 grant from the Muscular Dystrophy Association (MDA) to help fund early clinical development of the firm’s SMT C110 candidate, a small molecule utrophin upregulator, which is in development for the treatment of Duchenne muscular dystrophy (DMD).
SMT C110 was originally discovered and developed by Summit, and has previously been evaluated in a healthy volunteer study by a U.S. pharmaceutical company. While the drug was found to be safe and well tolerated, administration led to variable blood concentrations, and therapeutic blood plasma levels were only achieved in some patients. Summit believes part of this problem was due to the formulation used in the trial, and aims to develop the SMT C1100 based on alternative formulations.
SMT C1100 has been granted orphan drug status in the U.S. and Europe. Previous preclinical studies indicated that treatment increases utrophin protein levels in dystrophin-deficient muscle cells from DMD patients, to levels expected to be of significant therapeutic benefit. In vivo studies also found the drug significantly increased utrophin levels, improved whole-muscle function, and reduced muscle degeneration, fibrosis, and chronic inflammation.