CureDuchenne - Together we Can CureDuchenne
November 2012

Repeat Dosing of SMT C1100 For Treatment of Duchenne Muscular Dystrophy Meets Endpoints in Phase 1 Clinical Trial

Summit, a UK drug discovery company, announced on November 7 that the repeat dosing of the utrophin upregulator SMT C1100 for the treatment of the fatal muscle-wasting disease Duchenne Muscular Dystrophy (‘DMD’) has successfully met the endpoints as part of a Phase 1 clinical trial in healthy volunteers. The trial evaluated a new formulation of SMT C1100 and the results showed that upon repeat dosing, concentrations of the drug achieved in the blood plasma, stabilized at levels that from preclinical studies are expected to significantly increase utrophin protein production. The new formulation was also shown to be safe and well-tolerated in this Phase 1 trial.

SMT C1100 is a potential disease-modifying, oral small-molecule that works by upregulating (increasing) the amount of a naturally occurring protein called utrophin to maintain the healthy function of muscles. These data strongly support the progression of SMT C1100 into the next stages of development that includes biomarker and long-term safety studies, which will be required before a DMD patient efficacy trial could commence.

“Utrophin upregulation is a unique approach for treating DMD because it could benefit all DMD patients, regardless of their underlying genetic fault,” commented Glyn Edwards, Chief Executive Officer of Summit. “We are highly encouraged by these results, as the new formulation achieves blood concentrations that have the potential to significantly increase utrophin levels, with the outcome of maintaining the healthy function of muscles in patients with DMD. The results therefore strongly support continuing clinical evaluation of SMT C1100.”

The double blind, placebo-controlled Phase 1 trial examined a new nanoparticle aqueous suspension of SMT C1100 in a total of 48 healthy volunteers. The previously reported results from the single ascending dose cohort showed SMT C1100 to be safe and well-tolerated at all doses. These new data are being reported from the repeat dosing cohort where the volunteers received 100mg/kg twice daily for nine days. These results show that in all volunteers the blood plasma concentration of SMT C1100 stabilized after four days of dosing above the required level expected to increase utrophin protein production by 50% for at least 14 hours a day in a preclinical model. The plasma levels achieved were equivalent to those that gave significant therapeutic benefit in the gold standard disease model of DMD.

The Phase 1 trial has received funding from a group of U.S. DMD foundations: the Muscular Dystrophy Association, Charley’s Fund, CureDuchenne, the Foundation to Eradicate Duchenne, Nash Avery Foundation and Parent Project Muscular Dystrophy.

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