Summit Receives Fast Track Designation From US FDA for Ezutromid in the Treatment of Duchenne Muscular Dystrophy

OXFORD, United Kingdom, Sept. 26, 2016  -- Summit Therapeutics plc (SMMT) (SUMM.L), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy (‘DMD’) and Clostridium difficileinfection, today announces it has received Fast Track designation from the US Food and Drug Administration (‘FDA’) for ezutromid in the treatment of DMD. Ezutromid is a utrophin modulator and represents a potential disease modifying treatment for all patients with the fatal muscle wasting disease DMD.

 

“Fast Track designation underscores the importance that the FDA places on developing new treatments for life-threatening disorders, such as DMD, and aligns well with our recently outlined strategy to accelerate the development of ezutromid to market,” said Glyn Edwards, Chief Executive Officer of Summit. “As a utrophin modulator, ezutromid has the potential to significantly advance the state of care for all patients with DMD, regardless of their underlying genetic fault. We look forward to ezutromid’s continued progress in our ongoing Phase 2 clinical trial, PhaseOut DMD.”

Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions that address an unmet medical need. Advantages of Fast Track designation include opportunities for more frequent interactions with the FDA during all aspects of development, submission of a New Drug Application (‘NDA’) on a rolling basis, and eligibility for accelerated approval and priority review. This designation is in addition to ezutromid being granted Orphan Drug designation by the FDA and the European Medicines Agency.

 

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 has 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. DMD is an orphan disease, and the US Food and Drug Administration and the European Medicines Agency have granted Orphan Drug designation 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 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 programs focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficileinfection. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc).

 

Forward-looking Statements

Any statements in this press release about Summit’s future expectations, plans and prospects, including but not limited to, statements about the clinical and preclinical development of Summit’s product candidates, the therapeutic potential of Summit’s product candidates, and the timing of initiation, completion and availability of data from clinical trials, and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," and similar expressions, constitute forward looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of future clinical trials, availability and timing of data from on-going and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials or preclinical studies will be indicative of the results of later clinical trials, expectations for regulatory approvals, availability of funding sufficient for Summit’s foreseeable and unforeseeable operating expenses and capital expenditure requirements and other factors discussed in the "Risk Factors" section of filings that Summit makes with the Securities and Exchange Commission including Summit’s Annual Report on Form 20-F for the fiscal year ended January 31, 2016. Accordingly readers should not place undue reliance on forward looking statements or information. In addition, any forward looking statements included in this press release represent Summit’s views only as of the date of this release and should not be relied upon as representing Summit’s views as of any subsequent date. Summit specifically disclaims any obligation to update any forward-looking statements included in this press release.

 

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