Duchenne Muscular Dystrophy

UK-based drug discovery company Summit has received clearance from the Medicines and Healthcare products Regulatory Agency (MHRA) for its Phase I Clinical Trial Application (CTA) for SMT C1100 drug.

The move allows the company to commence a Phase I study of SMT C1100 in healthy humans for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal genetic neuromuscular disorder.

The double-blind placebo-controlled Phase I study will enrol 48 healthy male volunteers to assess the safety and tolerability of a new aqueous oral formulation of SMT C1100.

In addition, the trial will evaluate whether the new formulation can provide consistent levels of SMT C1100 in the blood that non-clinical efficacy studies predict will be required to have a therapeutic benefit in DMD patients.

The findings from the study are anticipated to be reported by the end of this year and a successful outcome from the Phase I trial will lead to evaluation of SMT C1100 in DMD patients.

In non-clinical efficacy studies, SMT C1100 has demonstrated its potential as a disease-modifying drug.

Summit chief executive officer Glyn Edwards said the company’s plans to commence a new Phase I trial for SMT C1100 continue to progress well with the necessary regulatory approval now in place.

"This study represents an important development milestone for this programme with a positive outcome having the potential to add considerable value to this asset, and bring an urgently needed treatment for this fatal disease a step closer," Edwards added.
The Phase I trial is being funded by the Muscular Dystrophy Association (MDA), Parent Project Muscular Dystrophy, Charley’s Fund, Cure Duchenne, Foundation to Eradicate Duchenne and the Nash Avery Foundation.

MDA translational research director Jane Larkindale said the SMT C1100 will now move from testing in vivo to testing in healthy humans.

"We have good evidence from laboratory studies that this drug’s mechanism of action is valid for slowing the progression of this degenerative muscle disease, and we hope that this new trial will show that it can be effectively and safely delivered to humans," Larkindale added.


Image: Histopathology of gastrocnemius muscle from patient who died of pseudohypertrophic muscular dystrophy, Duchenne type. Photo: Dr. Edwin P. Ewing, Jr.