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news.UK based drug discovery company Summit has dosed the first cohort of patients in a Phase 1 study of SMT C1100, an oral small molecule used for the treatment of Duchenne Muscular Dystrophy (DMD).
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The Phase 1 dose-escalating clinical trial in healthy volunteers is designed to assess whether the aqueous formulation of SMT C1100 can provide the consistent levels of drug in blood that non-clinical efficacy studies predicted would be required to confer therapeutic benefit in DMD patients.
Supported by a $1.5m grant from Muscular Dystrophy Association, Charley’s Fund, Cure Duchenne, the Foundation to Eradicate Duchenne, Nash Avery Foundation and Parent Project Muscular Dystrophy, the study will also evaluate the drug’s safety and tolerability.
Summit chief executive officer Glyn Edwards said, "SMT C1100 has the potential to modify the underlying disease, and the initiation of this Phase 1 trial represents a great step forward in bringing our breakthrough science to patients suffering from DMD. We expect to report top-line data from the full trial before the end of this year."
In non-clinical efficacy and safety studies, SMT C1100 has demonstrated its ability in increasing utrophin to restore and maintain the function of muscles.
The successful outcome from the Phase 1 trial is anticipated to advance SMT C1100 to a Phase 2 trial in DMD patients, the company said.