SMT C1100

Clinical development of SMT C1100, a utrophin modulator for the treatment of Duchenne muscular dystrophy

Jon Tinsley
Summit PLC

21st June 2013

Summit has developed SMT C1100, a small molecule utrophin modulator drug to maintain the production of utrophin, the foetal form of dystrophin. Studies in mice show utrophin acts as a functional replacement for dystrophin. This strategy is not mutation specific and offers the potential of a broad therapy for DMD and BMD. SMT C1100 has completed Phase 1 trials in healthy human volunteers and has orphan drug status in Europe and the US. The TACT reviewed the preclinical package which importantly included evidence of increased utrophin in the heart, the Phase 1 results, and the plans for Phase 1b and the Phase 2 studies. The Phase 1 data and pre-clinical safety data suggest that SMT C1100 has an appropriate safety profile for acute use. Given the effects observed in the mdx mouse and human cell cultures, the drug is likely to increase levels of utrophin in humans if similar therapeutic blood levels of the drug can be achieved. For the Phase 2, the main challenge will be the development of assays capable of showing consistent and measurable increases of utrophin in patients. The TACT made a number of suggestions regarding additional preclinical studies, metabolism and safety studies, regulatory questions, selection of the patient population in the clinic, and trial design.

12 Apr 2017