Summit Therapeutics gets nod for 'PhaseOut DMD' trial - Proactiveinvestors (UK): "Drug developer Summit Therapeutics (NASDAQ:SMMT, LON:SUMM) has received regulatory clearance to initiate a Phase 2 proof of concept clinical trial of its SMT C1100 candidate.
The “PhaseOut DMD” trial will be on patients with Duchenne Muscular Dystrophy (DMD), a muscle wasting disorder that affects boys and young men.
It aims to provide proof of concept for SMT C1100 and utrophin modulation through measurements of muscle fat infiltration, as well as measuring utrophin protein and muscle fibre regeneration in muscle biopsies.
SMT C1100 is an orally administered, small molecule utrophin modulator that the company believes has the potential to treat all boys and young men with DMD, regardless of their underlying dystrophin gene mutation. Utrophin is functionally and structurally similar to dystrophin, a protein that is is essential for the healthy function of muscles.
The 48-week open-label trial is expected to enrol up to 40 boys ranging in age from their fifth to their tenth birthdays at sites in Europe and the US, though trials in the latter will require the US Food and Drug Administration's (FDA) approval."
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