Summit Therapeutics’ SMT C1100 is a small molecule drug that targets increasing the production of utrophin. Utrophin is a protein that is present in early muscle development but “switched off” as muscle fibers mature and dystrophin is produced. SMT C1100 hopes to maintain the production of utrophin, in order to compensate for the absence of dystrophin in Duchenne. More information about SMT C1100 can be found here.


SMT C1100 is continuing to progress through clinical trials. Summit has recently been given the go-ahead for their next trial in the UK and expects to begin enrolling UK patients into their phase 2B study (“PhaseOut DMD”) very soon; enrollment in the US is planned for third quarter 2016. PhaseOut DMD plans for a total of 40 boys, ages 5 years/0 months – 10 years/0 months with the ability to walk at least 300 meters in the 6 minute timed walk test and who have been on a stable dose/schedule of steroids for at least 6 months. Participants who have participated in other clinical trials will need to “wash out” for 3 months prior to participating in PhaseOut DMD. Eight sites across the US, UK and Europe will participate.


Information is being updated in, but has been slightly delayed. Until is updated, information may be found in Summit’s FAQ.


Should you have questions, please contact PPMD or Michelle Avery with Summit at

Please speak with your clinical site to inquire if they will be participating in the study. Site contracts are still being finalized and are not yet listed on

Related Links

Summit’s FAQ

Duchenne Therapeutic Pipeline

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