- Summit Corporation says SMT C1100 for the treatment of Duchenne Muscular Dystrophy (DMD) has successfully met its primary endpoint of safety and tolerability in a Phase 1b clinical trial in patients with the disease.

SMT C1100 is an oral small molecule utrophin modulator that has the potential to treat all patients with DMD, regardless of the underlying dystrophin fault causing the disease.

The Phase 1b dose-escalating trial was conducted in 12 patients with DMD aged between 5 and 11 years old.

"These preliminary results show that SMT C1100 was safe and well tolerated at all doses tested in the study, and that there were no issues with patient compliance," Summit said.

All the boys had variable blood plasma concentrations of SMT C1100 with only two of the boys achieving concentrations similar to those of the adult volunteers in the 2012 Phase 1 study.

"Initial evidence suggests that the variability in drug uptake may be due to differences in diet and to other disease-related factors."

The non-placebo controlled trial also measured creatine kinase (CK) levels, an enzyme that is associated with muscle fibre damage and elevated in boys with DMD.

In the majority of patients there was a reduction in CK levels during dosing with SMT C1100. These data are consistent with non-clinical in vivo efficacy studies in the mdx model of DMD that showed SMT C1100 reduced CK levels after only 15 days.

At 8:27am: [LON:SUMM] Summit Corporation PLC share price was -0.5p at 7.5p

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