Summit PLC, a biotechnology company based in Abingdon, United Kingdom, has announced it has received approval from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) and Ethics Review Committee to begin testing its experimental drug SMT C1100 in children with Duchenne muscular dystrophy (DMD). Summit made the announcement in a Nov. 1, 2013, press release.
SMT C1100 is an orally administered drug designed to modulate levels of utrophin, a muscle protein that has the potential to compensate in part for the absence of the dystrophin protein, which is missing from the muscles of patients with DMD and diminished in those of patients with the related Becker muscular dystrophy (BMD). Both disorders result from any of a large number of mutations in the gene for dystrophin. (Utrophin-boosting compounds may also have implications for treating BMD.)
In May 2011, an MDA-supported study of SMT C1100 in dystrophin-deficient mice showed the drug reduced muscle abnormalities, increased overall strength, and improved the ability to resist fatigue after exercise. In December 2011, MDA awarded $750,000 to Summit PLC to advance SMT C1100 into clinical trials.
The drug has been found to be safe and well-tolerated in healthy volunteers, in whom it reached blood levels considered adequate for a therapeutic effect. Until now, there have been no tests of SMT C100 in people with DMD.
"We're extremely pleased to see this important compound moving forward into patient clinical trials," said Jane Larkindale, MDA's vice president of research. "SMT C1100 has the potential to benefit patients with DMD or possibly BMD arising from a number of different mutations in the dystrophin gene. Data from these studies will address critical questions in determining if the compound can be effective in DMD patients."
Summit CEO Glyn Edwards said: "Securing regulatory approval for the first in patient phase 1b clinical trial has achieved another important milestone in the development of utrophin modulator SMT C1100 for DMD. We believe that utrophin modulation is a novel disease-modifying approach for all boys with DMD, and this phase 1b trial forms an integral part of our wider clinical plans towards establishing SMT C1100 as a viable treatment for this devastating condition."
About the new trial
The new, phase 1b trial of SMT C1100 in DMD — which is not yet open to recruitment — will include 12 boys with the disorder who are between 5 and 11 years old and meet other criteria. It will be a dose-escalating, open-label study, meaning the dose of the experimental drug will gradually be increased over the course of the trial, and all participants will receive the experimental drug. (There will be no placebo group.) The trial will be conducted in the United Kingdom.
About Clinical Trials
A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.