Summit Therapeutics Recognises 10th Annual Rare Disease Day
February 28 2017 - 6:00AM
Summit Therapeutics plc (NASDAQ:SMMT) (AIM:SUMM), the drug
discovery and development company advancing therapies for Duchenne
muscular dystrophy (‘DMD’) and Clostridium difficile infection,
recognises the tenth annual Rare Disease Day taking place today, 28
February 2017. The Rare Disease Day 2017 theme, ‘with research,
possibilities are limitless,’ emphasises the importance of
scientific research in helping to understand, diagnose and treat
rare diseases that affect millions of people and their families
worldwide.
Summit seeks to remain at the forefront of
utrophin modulation research through its strategic alliance with
the University of Oxford, under the guidance of Professor Kay
Davies. The collaboration is focussed on developing
future-generation utrophin modulators for the potential treatment
of all patients with the progressive muscle wasting disorder, DMD.
To date, the research team has identified two series of novel
utrophin modulators, one of which has a mechanism of action
potentially distinct from ezutromid, the Company’s lead utrophin
modulator that is in a Phase 2 clinical trial in DMD patients.
“In our quest to bring a potentially
disease-modifying treatment to all patients with DMD, we have
collaborated with the preeminent expert in utrophin modulation
biology, Professor Kay Davies, and her research team at the
University of Oxford,” said Glyn Edwards,
Chief Executive Officer of Summit. “We applaud EURORDIS,
the organisation representing rare disease patients in Europe, for
bringing an annual spotlight to the plight of millions of people
affected by rare diseases and in this year, recognising the immense
impact that research is having and will continue to have for those
living with rare diseases.”
In the European Union a rare disease is defined
as one that affects fewer than 5 in 10,000 of the general
population, while in the United States, it is defined as a disease
that affects fewer than 200,000 people. There are between
6,000 and 8,000 known rare diseases with around five new rare
diseases described in the literature each week. Rare diseases are
often chronic and life threatening and include rare conditions,
such as childhood cancers, and some other well-known conditions
including cystic fibrosis and DMD.
Rare Disease Day takes place on the last day of
February each year, and its objective is to raise awareness among
the general public and decision-makers about rare diseases and
their impact on patients' lives. Rare Disease Day was launched in
Europe in 2008 by EURORDIS. It is now observed in more than 80
nations, and is sponsored in the US by the National Organization
for Rare Disorders (NORD). For more information, please visit
www.rarediseaseday.org.
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 (‘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 programs 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).
For more information, please contact:
Summit Glyn Edwards / Richard Pye
(UK office)Erik Ostrowski / Michelle Avery (US office) |
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Tel: +44
(0)1235 443 951 +1 617 225 4455 |
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Cairn Financial Advisers LLP(Nominated
Adviser)Liam Murray / Tony Rawlinson |
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Tel: +44
(0)20 7213 0880 |
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N+1 Singer (Broker)Aubrey Powell / Lauren
Kettle |
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Tel: +44 (0)20 7496 3000 |
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MacDougall Biomedical Communications(US media
contact)Chris Erdman / Karen Sharma |
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Tel: +1
781 235 3060cerdman@macbiocom.comksharma@macbiocom.com |
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Consilium
Strategic Communications (Financial public relations,
UK)Mary-Jane Elliott / Sue Stuart / Jessica Hodgson / Lindsey
Neville |
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Tel: +44
(0)20 3709 5700 summit@consilium-comms.com |
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Forward-looking StatementsAny
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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