14 March 2018

Summit Highlights Scientific Rigour of its PhaseOut DMD Clinical Trial at MDA Clinical Conference


Oxford, UK, and Cambridge, MA, US, 14 March 2018 – Summit Therapeutics plc (NASDAQ: SMMT, AIM: SUMM) highlighted the rigour being utilised in the collection and analysis of muscle biopsies and magnetic resonance spectroscopy (‘MRS’) related data in its PhaseOut DMD clinical trial, at the 2018 MDA Clinical Conference in Arlington, VA.

PhaseOut DMD is a Phase 2 open-label trial of the Company’s utrophin modulator, ezutromid, in patients with Duchenne muscular dystrophy (‘DMD’). Through this study, Summit is compiling a deep dataset for each patient in the trial, which includes the analysis of several thousand muscle fibres per biopsy sample. As part of this effort, steps are being taken to remove human bias through the automated reading of biopsy and MRS measurements at central sites.

“Since DMD is a muscle-wasting disorder, it is critical for us to have high quality data from objective muscle health measurements as we assess ezutromid’s activity. As the collection of muscle biopsy and MRS measurements are a major intervention for patients in clinical trials, it means it is crucial this is done in a robust and reliable way,” “Our data collection and analysis methods used in PhaseOut DMD helped yield a high-quality dataset from all evaluable boys in the trial after 24-weeks of treatment. We are grateful to all the boys in the trial and excited about the interim findings that showed compelling signs of ezutromid activity.”

Dr David Roblin

Chief Medical Officer and President of R&D of Summit

The recently announced positive interim data from PhaseOut DMD showed that after 24 weeks of treatment, there was a significant and meaningful reduction in muscle damage, as well as a significant reduction in muscle inflammation, in patients treated with ezutromid. These data provide early evidence that ezutromid is modulating the production of utrophin protein and having its intended effect of stabilising muscle membranes, which has in turn led to early improvements in muscle health. Utrophin is a protein that can substitute for dystrophin, the protein that is missing in those with DMD the absence of which results in disease progression and premature death.  Because Summit’s utrophin modulation approach is independent of the patient’s underlying dystrophin gene mutation, it could be applicable to all patients with DMD.


Details of the Methodologies from the Poster Presentation

One of the methods being used to measure muscle damage in PhaseOut DMD is biopsy analysis. Summit has made a significant effort to ensure that these biopsies are handled, processed and analysed with great care.  For the biopsy procedure, two pieces of muscle are taken during each biopsy. These pieces are frozen and shipped separately to a central reading facility. Multiple sections from each biopsy are assayed for muscle damage, yielding a total of six biopsy sections for each patient for both the baseline and post-treatment samples. After quality control checks, the muscle sections are then read via an automated system to remove human bias. For the 24-week assessment, several thousand muscle fibres were analysed per sample, producing a robust dataset for each patient with high concordance across individual samples.

Muscle inflammation is measured by T2- relaxation time using MRS. MRS of the soleus (calf) and vastus lateralis (thigh) muscles is performed at central imaging centres following a training and certification process. The MRS quantification is then performed by an automated blinded process.

A copy of the poster is available in the Publications section of Summit’s website, www.summitplc.com.

About PhaseOut DMD

PhaseOut DMD is an open-label, multi-centre trial that has enrolled 40 patients in the US and UK, aged from their fifth to their tenth birthdays. PhaseOut DMD is 48 weeks in length after which patients have the option of enrolling into an extension phase and continuing to be dosed with ezutromid. The primary endpoint is the change from baseline in magnetic resonance spectroscopy parameters related to the leg muscles. Biopsy measures evaluating utrophin and muscle damage are included as secondary endpoints. Exploratory endpoints include the six-minute walk distance, the North Star Ambulatory Assessment and patient reported outcomes. Top-line 48-week results are expected to be reported in the third quarter of 2018.

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 patient’s underlying dystrophin gene mutation. Summit also believes that utrophin modulation could 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 www.summitplc.com and Summit can be followed on Twitter (@summitplc).






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Any statements in this press release about the Company’s future expectations, plans and prospects, including but not limited to, statements about the clinical and preclinical development of the Company’s product candidates, the therapeutic potential of the Company’s product candidates, the potential commercialisation of the Company’s product candidates, the sufficiency of the Company’s cash resources, the timing of initiation, completion and availability of data from clinical trials, the potential submission of applications for marketing approvals 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 ongoing 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, laws and regulations affecting government contracts, availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements and other factors discussed in the "Risk Factors" section of filings that the Company makes with the Securities and Exchange Commission, including the Company’s Annual Report on Form 20-F for the fiscal year ended 31 January 2017. 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 the Company’s views only as of the date of this release and should not be relied upon as representing the Company’s views as of any subsequent date. The Company specifically disclaims any obligation to update any forward-looking statements included in this press release.


This announcement contains inside information for the purposes of Article 7 of EU Regulation 596/2014 (MAR).


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