Summit Therapeutics plc Summit Highlights Scientific Rigour Of Its Phaseout Dmd Clinical Trial At Mda Clinical Conference

 
TIDMSUMM 
 
 
   Summit Therapeutics plc 
 
   ("Summit" or the "Company") 
 
   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," said 
Dr David Roblin, Chief Medical Officer and President of R&D of Summit. 
"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." 
 
   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). 
 
   Contacts 
 
 
 
 
Summit 
Glyn Edwards / Richard Pye (UK office)       Tel:           [phone] 
Erik Ostrowski / Michelle Avery (US office)                    [phone] 
 
Cairn Financial Advisers LLP (Nominated 
 Adviser)                                    Tel:          [phone] 
Liam Murray / Tony Rawlinson 
 
N+1 Singer (Joint Broker)                    Tel:          [phone] 
Aubrey Powell / Jen Boorer 
 
Panmure Gordon (Joint Broker)                Tel:          [phone] 
Freddy Crossley, Corporate Finance 
Tom Salvesen, Corporate Broking 
 
MacDougall Biomedical Communications (US)    Tel:              [phone] 
Karen Sharma                                             [e-mail] 
 
Consilium Strategic Communications (UK)      Tel:          [phone] 
Mary-Jane Elliott / Jessica Hodgson /               [e-mail] 
Philippa Gardner 
 
 
 
   Summit Forward-looking Statements 
 
   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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contained therein. 
 
   Source: Summit Therapeutics plc via Globenewswire 
 
 
  http://www.summitplc.com/ 
 

(END) Dow Jones Newswires

March 14, 2018 07:00 ET (11:00 GMT)

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