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Press Release: Drug for Duchenne Muscular Dystrophy Successfully Completes Phase 1 Trial; Trial in Boys with DMD May Be Next

MDA supported development of utrophin-boosting compounds for years

TUCSON, Ariz. — The Muscular Dystrophy Association announced today that SMT C1100, an experimental drug for Duchenne muscular dystrophy (DMD), has been found safe in healthy human volunteers and reached blood levels potentially adequate for a therapeutic benefit. Next steps are to move into trials in people with DMD.

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

The Association has been supporting the development of SMT C1100 through a $750,000 grant to Summit PLC, a drug discovery company based in Oxford, U.K. The grant was awarded in 2011 by the MDA Venture Philanthropy (MVP) drug development program for testing of a new formulation of SMT C1100 in humans. (A previous formulation failed to reach adequate blood levels.)

In addition to the 2011 MVP grant, the Association has given $1.8 million since 1997 to geneticist Kay Davies at Oxford University for laboratory studies related to boosting utrophin levels.

"We are very pleased by these encouraging results showing that SMT C1100 can reach plasma levels that might be expected to be effective," said Jane Larkindale, MDA's director of translational research. "MDA has been supporting the development of utrophin-increasing strategies for many years. We eagerly await later-stage trials of SMT C1100 to see if this has a clinical benefit in patients."

Summit's Chief Executive Officer Glyn Edwards said: "Utrophin upregulation is an important mechanism for treating DMD, as it is expected to have broad applicability for all patients regardless of the specific genetic fault causing their disease. We are highly encouraged that the new formulation of SMT C1100 shows improved bioavailability, as we believe it supports the progression of this potential breakthrough treatment into DMD patient clinical trials."

About SMT C1100

SMT C1100 is designed to upregulate and maintain the production of utrophin. Utrophin is a protein that is highly expressed in fetal and regenerating muscle but decreases as the muscle fiber mature and is eventually replaced by dystrophin, a similar protein that maintains the integrity and healthy function of muscles. Patients with DMD are unable to make dystrophin, resulting in muscle fiber degeneration. However, if utrophin is continually expressed in the mature fiber, it can functionally replace dystrophin and is expected to overcome the deficit in patients with DMD. This approach is expected to be a universal treatment for all DMD patients regardless of whether the disease was caused by an inherited or spontaneous mutation. Summit has demonstrated in nonclinical efficacy studies that SMT C1100 is capable of increasing utrophin to restore and maintain the healthy function of muscles, including the heart and diaphragm. SMT C1100 has been granted orphan drug status in Europe and the U.S.

To learn more, read the Quest News Online article DMD: Reformulated Utrophin Upregulator Looks Good in Healthy Volunteers.

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 a number of different 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 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.

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