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SMT C1100 For Duchenne Muscular Dystrophy Moves To Human Testing

TUCSON, Ariz., April 24, 2012 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association announced today that SMT C1100, an experimental drug for Duchenne muscular dystrophy (DMD), has received approval from regulatory agencies in the United Kingdom to move from laboratory to human testing, starting with healthy human volunteers.


The drug is designed to increase production of a muscle protein utrophin that can potentially compensate for the one that's missing in DMD.

The Association has been supporting the development of SMT C1100 through its MDA Venture Philanthropy drug development program, via a grant to Summit Corporation PLC of Oxford, United Kingdom.

"We're extremely pleased that SMT C1100 will now move from testing in vivo to testing in healthy humans," said Jane Larkindale, MDA's director of translational research. "We have good evidence from laboratory studies that this drug's mechanism of action is valid for slowing the progression of this degenerative muscle disease, and we hope that this first trial will show that it can be effectively and safely delivered to humans."

Summit's Chief Scientific Officer Richard Storer said: "Utrophin upregulation will be beneficial to all DMD boys, and SMT C1100 has demonstrated disease-modifying potential in nonclinical efficacy studies. This trial will evaluate if our improved formulation of SMT C1100 can produce consistent levels of the drug in the blood which, based on the preclinical studies, might be expected to have a therapeutic benefit in patients."

About SMT C1100

SMT C1100 is designed to boost production of the muscle protein utrophin. Utrophin closely resembles and can mimic some of the functions of dystrophin, the protein missing in the muscle fibers of patients with DMD.

In May 2011, an MDA-supported study showed that daily treatment with SMT C1100 dramatically reduced symptoms in dystrophin-deficient mice with a DMD-like disease. The mice showed reduced muscle abnormalities, increased overall strength and improved ability to resist exercise-related fatigue.

An earlier version of the drug, tested by California-based BioMarin Pharmaceutical, was found safe and well-tolerated in healthy human volunteers, but it failed to reach blood levels considered adequate for therapy.

The reformulated Summit PLC version of the drug is designed to reach adequate blood levels in humans following oral administration.

To learn more, read the Quest News Online article DMD Drug SMT C1100 Moves to Human Testing.

About DMD

DMD is a degenerative muscle disease — affecting boys almost exclusively — that involves progressive degeneration of voluntary and cardiac muscles, with resulting weakness and heart abnormalities. The disease manifests in early childhood, causing delayed motor milestones and, in some cases, cognitive, behavioral or language deficits. Loss of the ability to walk occurs in most children with DMD between the ages of 10 and 12 years; weakened cardiac and respiratory muscles severely limit life span.

The disease is caused by any number of mutations in the X-chromosome gene that carries instructions for the muscle protein dystrophin. Without dystrophin, muscle fibers are abnormally fragile and break down under the stress of contractions.

Corticosteroid medications, such as prednisone and its chemical cousin prednisolone, slow disease progression.

About Summit

Summit is an Oxford, U.K.-based drug discovery company with an innovative Seglin™ technology platform for the discovery of new medicines and a portfolio of drug program assets.  Summit's program portfolio consists of a number of drug programs targeting high-value areas of unmet medical need, including Duchenne muscular dystrophy and C. difficile infection. Summit is listed on the AIM market of the London Stock Exchange and trades under the ticker symbol SUMM. Further information is available at

About MDA Venture Philanthropy (MVP)

MVP is the Muscular Dystrophy Association's drug development program, which operates within MDA's translational research program. MVP is exclusively focused on funding the discovery and clinical application of treatments and cures for neuromuscular diseases.

For more information, visit and follow MDA on Facebook ( and Twitter (@MDAnews).

SOURCE Muscular Dystrophy Association
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