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Summit Therapeutics gets nod for 'PhaseOut DMD' trial

The UK Medicines and Healthcare products Regulatory Agency and the Research Ethics Committee have given the trial the go-ahead

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About 1 in 3,500 boys in the UK are born with DMD

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.

The primary endpoint of the trial is the change from baseline in magnetic resonance imaging parameters related to fat infiltration and inflammation of the leg muscles.

Functional endpoints, including the six-minute walk test, North Star Ambulatory Assessment and patient reported outcomes, are also being explored.

Summit expects to report data from the first group of patients enrolled in the trial periodically from the second half of 2016 onwards with the first set of 24-week muscle biopsy data expected to be available before the end of 2016.

"Our lead utrophin modulator, SMT C1100, has demonstrated disease-modifying potential in preclinical studies to date, and with the initiation of PhaseOut DMD, we are at a stage in SMT C1100's clinical development where we are evaluating the possibility of this benefit in patients," said Ralf Rosskamp, MD, chief medical officer of Summit. 

Summit's shares were outperforming the London market in lunchtime trading, up 1.4% at 126.75p.

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