Summit Presents New Data from Phase 1b Clinical Trial of SMT C1100 for Treatment of DMD

Today, Summit released further results with new data from its recently completed Phase IB clinical trial of SMT C1100 at the 13th International Congress of Neuromuscular Disease in Nice, France. (

The poster (Poster session: PS1-72 / #298) was entitled, “Utrophin modulators to treat Duchenne Muscular Dystrophy (DMD): Phase 1b clinical trial results of SMT C1100” by Francesco Muntoni et al.

The results showed a statistically significant decrease in certain key enzymes (creatine kinase, aspartate aminotransferase and alanine aminotransferase) associated with muscle damage and the Company believes that this supports the proposed mechanism of action of SMT C1100.

“These new data from the Phase 1b clinical trial of SMT C1100 provide further encouragement regarding the potential of our utrophin modulator as a disease modifying treatment for all patients with DMD,” commented Glyn Edwards, Chief Executive Officer of Summit.  “The reduction during drug dosing in enzyme markers normally associated with muscle damage provides an intriguing indication of SMT C1100 activity in these patients. We look forward to progressing SMT C1100 into future trials.”

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 trial was a dose-escalating, open-label study conducted in pediatric patients with DMD to evaluate safety, tolerability and drug exposure.  The trial enrolled 12 patients aged between 5 and 11 years, divided equally into three dose cohorts and received daily oral doses of SMT C1100 for a total of ten days.

Previously reported preliminary results show that SMT C1100 was safe and well tolerated at all doses tested in the study.  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 I study. Additionally, 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

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