DMD: Utrophin Modulator SMT C1100 To Undergo Further Testing

MDA-supported Summit PLC announced it will continue testing it experimental drug in Duchenne MD patients, along with dietary modifications that may improve drug absorption

A new trial will probe whether changing the diet will increase the therapeutic effect of utrophin modulator SMT C1100 in Duchenne muscular dystrophy.
Article Highlights:

  • SMT C1100, in development by British biotechnology company Summit PLC with support from MDA, is designed to increase levels of and change localization of the protein known as utrophin in muscle fibers.
  • Animal experiments have suggested that increasing utrophin can improve muscle health when there is a lack of the dystrophin protein, which is the case in boys with Duchenne muscular dystrophy ; a trial in DMD patients has recently shown that the drug has therapeutic potential in this disorder.
  • The new phase 1b trial, to be conducted in the United Kingdom, will investigate whether changing the diet of patients taking SMT C1100, an oral drug, can increase its effectiveness.


by Margaret Wahl on December 22, 2014 - 11:22am

Update (Feb. 20, 2015): In a Feb. 20, 2015, press release, Summit announced that the first patients in this phase 1b modified-diet clinical trial of SMT C1100 have received the drug. "We believe that utrophin modulation has the potential to benefit all patients with DMD, irrespective of the underlying fault in the dystrophin gene causing the disease," said Glyn Edwards, company CEO, "and this new trial forms part of our broader clinical development path that seeks to provide this drug with the best chance of reaching the market. We look forwadr to reporting data from this study in Q3 2015 [third quarter of 2015]." The company name is now Summit Therapeutics.


original story:

SMT C1100, an experimental compound designed to boost levels of the potentially therapeutic protein utrophin in patients with Duchenne muscular dystrophy (DMD), will undergo further testing in boys with this disorder along with dietary specifications that may improve its absorption by the body, its developer has announced.

Summit PLC, an Oxford, United Kingdom-based biotechnology company that has received MDA support for the development of SMT C1100, announced the modified diet trial of this compound in a Dec. 11, 2014, press release.

In October 2014, the company released positive data from a phase 1b trial in boys with DMD without dietary modification. The orally administered drug showed a favorable safety profile and was associated with reduction in enzymes that are markers of muscle damage.

Summit received a $750,000 grant from MDA in 2011.

SMT C1100 is a utrophin modulator

Utrophin is a protein that is similar to dystrophin, which is missing in patients with DMD and in animal models of the disease. Experiments in dystrophin-deficient animals have suggested that utrophin "modulators" – compounds that increase the amount of utrophin and expand its location in muscle fibers – could be therapeutic, regardless of the specific genetic mutation causing dystrophin deficiency. Utrophin modulation is a strategy that MDA has long supported.

About the phase 1b modified diet trial

In its Dec. 14 announcement, Summit said it has received approval from the UK Medicines and Healthcare Products Regulatory Agency and the Ethics Review Committee to begin a phase 1b modified diet trial of SMT C1100.

"We believe it is possible to enhance absorption of SMT C1100 through dietary means, and this new patient trial is designed to test this so that we can confidently evaluate the efficacy of utrophin modulation in subsequent clinical trials," said Glyn Edwards, CEO of Summit. "We believe that utrophin modulation has the opportunity to benefit all boys with DMD, and we are working to ensure this molecule has the best chance to reach the market through a well-designed clinical path." Edwards said preliminary data are expected in mid-2015.

The trial will enroll 12 patients with DMD who are 5 to 13 years old and meet study criteria at four hospitals in the United Kingdom. Participants will be divided into three groups, and each participant will receive two different doses of SMT C1100 and a placebo during three 14-day treatment periods. The three treatment periods will be divided by 14 days of no treatment ("wash-out" periods). Participants will be asked to follow specific dietary guidance with respect to balanced proportions of fat, protein and carbohydrates during the trial.

If this trial is successful, it will be followed by a phase 2 trial that will seek to generate longer-term data on effectiveness and safety. It is expected that participants in the phase 1b modified diet trial will be eligible to join the phase 2 trial.


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