DMD: Summit Granted Patents for Utrophin Booster

The drug SMT C1100 is still experimental, but an MDA-supported company has received two US patents that provide commercial protection for its development

Article Highlights:
  • Utrophin-boosting drug SMT C1100, still in development, has been granted two patents in the United States.
  • MDA-supported Summit PLC, developer of SMT C1100, says it expects to start testing of the drug in people with Duchenne muscular dystrophy later this year.
  • Raising and maintaining levels of utrophin, a protein that can partially substitute for dystrophin muscle cells, may be helpful to patients regardless of their specific dystrophin gene mutation.
by Quest Staff on September 3, 2013 - 5:00am

Summit PLC, a biotechnology company based in Abingdon, United Kingdom, has announced it now has two U.S. patents for its experimental drug SMT C1100 for Duchenne muscular dystrophy (DMD).

The patents will provide commercial protection to the company as it continues the further development of this drug. However, the drug will not become available to patients until it receives approval from regulatory agencies.

Summit has received $750,000 in support from MDA to develop SMT C1100, which is designed to boost utrophin protein levels in muscle fibers. The Association also has supported molecular geneticist Kay Davies at the University of Oxford in the United Kingdom to develop utrophin-based therapies. Davies, in collaboration with Summit, identified SMT C1100 in an MDA-supported screen.

Utrophin is a muscle protein similar to dystrophin, the protein that's missing in DMD and insufficient in Becker muscular dystrophy (BMD) because of any of a large number of mutations in the dystrophin gene. There is evidence that maintaining utrophin levels can compensate for dystrophin deficiency, at least in part. It may not matter exactly which mutation a patient has or whether the disease is classified as DMD or BMD for there to be benefit from a utrophin therapy, although this has not yet been demonstrated in humans.

In an Aug. 28, 2013, press release, Summit announced it had received a U.S. "composition of matter" patent for SMT C1100. Earlier in August, the company received a patent for the use of SMT C1100 in combination with corticosteroids, a standard treatment for DMD.

Summit says it expects to start a clinical trial of the drug in people with DMD during the second half of 2013.

An excerpt from Summit's Aug. 28 press release follows.

Oxford, UK, 28 August 2013 Summit (AIM: SUMM), a drug discovery and development company advancing therapies for Duchenne muscular dystrophy (DMD) and C. difficile infection, announces that the United States Patent and Trademark Office has issued a composition of matter patent that protects SMT C1100 and its use in the treatment of the fatal muscle-wasting disease DMD.

United States Patent number 8,518,980 is entitled "Treatment of Duchenne Muscular Dystrophy" and will provide a period of exclusivity for the small molecule utrophin modulator SMT C1100 through at least November 2028.  

"The grant of this cornerstone patent for SMT C1100 in a key commercial market represents a critical component in our strategy for advancing this promising utrophin modulator in the treatment of DMD," commented Glyn Edwards, chief executive officer of Summit. "Together with the recently issued U.S. patent covering the use of SMT C1100 in combination with steroids, the intellectual property estate protecting this potential life-changing drug has been significantly strengthened as we prepare to start patient clinical trials during the second half of this year."

The patent "Drug Combinations for the Treatment of Duchenne Muscular Dystrophy" was recently issued by the U.S. Patent and Trademark Officer and covers the use of SMT C1100 in combination with steroids such as prednisolone and deflazacort (U.S. Patent number 8,501,713).

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