Summit plc, an Oxford-based biotech company, has announced preliminary results of their phase 1b trial of SMT C1100 – a potential drug developed to increase levels of utrophin in the muscles of boys with Duchenne muscular dystrophy (DMD). The potential drug was developed in Professor Dame Kay Davies’ laboratory and can increase levels of utrophin in vitro and in mdx mice. This is the first trial to test SMT C1100 in people. The trial tested different doses of the potential drug in 12 boys (aged between five and eleven) with the condition. Four boys received a lower dose twice each day, four received a higher dose twice each day, and four received a higher dose three times each day. The preliminary results show that all doses of the potential drug were safe and that it was well tolerated in all individuals. However, the levels of SMT C1100 that reached the bloodstream (or plasma) varied between trial participants – with good levels in two boys but lower levels in ten. Researchers believe this may be due to difference in diets between boys, with a possibility that low-fat diets may reduce uptake of the potential drug. The company is working to understand this result using food diaries kept by some of the trial participants and plans to make small changes to SMT C1100 to make uptake of the drug less dependent on the diet being eaten. Encouragingly, the researchers noted that levels of creatine kinase (CK) in the serum were reduced in boys during the trial, suggesting that muscle damage was reduced in DMD boys receiving SMT C1100. Further trials will be required to confirm this and to test the effectiveness of the potential drug.
