Summit Highlights Utrophin Modulation as a Potential Universal Treatment Option in DMD at 15th Action Duchenne International Conference


  • New Preclinical Data Show Utrophin’s Positive Effect on Mitochondria Improving Muscle Health in DMD Model

OXFORD, United Kingdom, Nov. 09, 2017 (GLOBE NEWSWIRE) -- Summit Therapeutics plc (NASDAQ:SMMT) (AIM:SUMM) the drug discovery and development company advancing therapies for Duchenne muscular dystrophy (‘DMD’) and Clostridium difficile infection, announces that the Company and collaborators from the University of Oxford will highlight the potential of utrophin modulation as a universal treatment for DMD in several presentations at the 15th Action Duchenne International Conference 2017, taking place November 10-12, 2017, in Birmingham, UK. The conference brings together families living with Duchenne and Becker muscular dystrophy, along with experts and industry members aiming to treat these diseases.

The Company’s scientific adviser and co-founder, Professor Kay Davies from the University of Oxford, will present recently published preclinical data highlighting the benefits of utrophin modulation on muscle health in animal models of DMD. The data show that continuously expressing utrophin in a dystrophin-deficient background can reduce mitochondrial aberration and oxidative stress. Aberrant mitochondria drive, in part, oxidative stress, which is a contributing factor to muscle damage in DMD. In addition, Professor Davies is also expected to discuss previously published preclinical data of utrophin modulators, including ezutromid, that have shown these compounds’ potential to prevent molecular disease, leading to functional improvements in mdx mice.

Professor Kay Davies FRS of the University of Oxford commented, “My team at the University of Oxford continues to gather scientific evidence showing how utrophin can substitute for dystrophin in animal muscle and prevent many of the molecular hallmarks of DMD from occurring in these animal models. Importantly, these underlying changes in muscle health have the potential to lead to functional benefits, providing hope for a universal, disease-modifying treatment for families living with Duchenne.”

Separately, Summit’s presentations will focus on the ongoing Phase 2 proof of concept clinical trial, PhaseOut DMD, which is evaluating the Company’s lead utrophin modulator, ezutromid. PhaseOut DMD is a 48-week open-label clinical trial that is fully enrolled with a total of 40 patients with DMD. A key milestone of this clinical trial is the 24-week assessment that could provide initial clinical proof of mechanism for ezutromid and these data are expected to be reported in the first quarter of 2018.

“These new preclinical data from Professor Davies’ team at the University of Oxford provide another piece of evidence highlighting the potential of utrophin modulation in being able to treat this devastating muscle wasting disease,” said Dr David Roblin, Chief Operating Officer and Medical Officer at Summit. “We remain on-track to report the first results of ezutromid treatment in boys with DMD in PhaseOut DMD in the first quarter of 2018, and if results provide evidence of the mechanism of utrophin modulation in patients, we believe it would represent a major advancement for ezutromid. It would bring closer to all patients a therapy that has the potential to be disease modifying in DMD.”

About Utrophin Modulation in DMD
DMD is a progressive muscle wasting disease that affects around 50,000 boys and young men in the developed world. The disease is caused by different genetic faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of all muscles. There is currently no cure for DMD and life expectancy is into the late twenties. Utrophin protein is functionally and structurally similar to dystrophin. In preclinical studies, the continued expression of utrophin had a meaningful, positive effect on muscle performance. Summit believes that utrophin modulation has the potential to slow down or even stop the progression of DMD, regardless of the underlying dystrophin gene mutation. Summit also believes that utrophin modulation could potentially be complementary to other therapeutic approaches for DMD. The Company’s lead utrophin modulator, ezutromid, is an orally administered, small molecule drug. DMD is an orphan disease, and the US Food and Drug Administration (‘FDA’) and the European Medicines Agency have granted orphan drug status to ezutromid. Orphan drugs receive a number of benefits including additional regulatory support and a period of market exclusivity following approval. In addition, ezutromid has been granted Fast Track designation and Rare Pediatric Disease designation by the FDA.

About Summit Therapeutics
Summit is a biopharmaceutical company focused on the discovery, development and commercialisation of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programmes focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficile infection. Further information is available at and Summit can be followed on Twitter (@summitplc).


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