In a series of tests using the ‘gold standard’ preclinical in-vivo model of Duchenne muscular dystrophy (DMD), SMT C1100 was shown to increase significantly the strength of muscles when compared to no treatment. A synergistic benefit in reducing muscle fatigue during exercise (ability to walk longer distances) was seen with SMT C1100 and steroid treatment (prednisolone). Steroids are currently the only frontline therapy for DMD.
Importantly, this study, which demonstrates a reduction in muscle fatigue during exercise, is a surrogate for the agreed primary clinical endpoint in human clinical trials. It is designed to demonstrate that patients’ muscle function is improved by measuring the increase in distance they can walk. Summit’s plan is to conclude preclinical development work by the end of 2008 and begin Phase I clinical trials shortly afterwards.
Richard Storer, chief scientific officer of Summit, said: “In preclinical studies to date, SMT C1100 has shown positive results consistently that give us great confidence that we will soon be able to progress this novel candidate into patients. If it is successful in the clinic, this will be a first in class drug offering considerable hope to all DMD patients.”