Summit Reports Positive Data on it Utrophin Modulation Programme for the Treatment of DMD at 2014 WMS Congress

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October 08, 2014 02:00 ET

Summit Therapeutics plc

Summit Therapeutics plc

Summit Corporation plc

('Summit' or 'the Company')

SUMMIT REPORTS POSITIVE DATA ON ITS UTROPHIN MODULATION PROGRAMME FOR THE TREATMENT OF DMD AT 2014 WMS CONGRESS

Phase 1b clinical trial data on lead utrophin modulator SMT C1100

Positive preclinical data unveiled on second generation candidates

Oxford, UK, 8 October 2014 - Summit (AIM: SUMM), the drug discovery and development company advancing therapies for Duchenne Muscular Dystrophy ('DMD') and C. difficile infection, reports clinical data on SMT C1100, the Company's lead utrophin modulator, highlighting its safety profile and further detailing the observed reduction in enzymes associated with muscle damage in a Phase 1b study in boys with DMD.  In addition, Summit is unveiling new positive preclinical data on its second generation utrophin modulator programme for the treatment of DMD.

All data are being presented at the 19th International World Muscle Society Congress ('WMS') being held in Berlin Germany from 7-11 October 2014.

Glyn Edwards, Chief Executive Officer of Summit commented, "SMT C1100 is paving the way in the clinic for utrophin modulation and the initial signs of activity point to this mechanism as a potential treatment for all boys with DMD.  Our second generation utrophin modulators build on this mechanism with the new data illustrating how they increase utrophin protein levels and improve muscle health, while having enhanced drug properties compared to the first generation drug SMT C1100."

"This exciting progress shows SMT C1100 has the potential to become the first utrophin modulator drug to treat all DMD patients, to be followed by second generation candidates with improved properties, as we seek to change the treatment paradigm for this devastating condition."

The data on the second generation programme comprise results from in vivo and in vitro studies that highlight the promising profile of two lead candidates as potential disease modifying treatments:

Significant improvement in systemic exposure in vivo compared to first generation utrophin modulator SMT C1100 with blood plasma levels 10-40 times higher following oral dosing;

Modulation of utrophin expression in vivo with an increase in protein levels observed in skeletal muscle, diaphragm and heart compared to the control;

Significant improvement in disease pathology with reduction in muscle fibre fibrosis ('scarring') and membrane damage;

Improvement in muscle health indicated by a significant reduction in the number of regenerating muscle fibres;

Protection of muscle function with the increased utrophin protein levels resulting in significant improvement in muscle membrane stability and resistance to damage.

These studies were conducted as part of the UtroDMD Alliance, a collaboration to develop utrophin modulator drugs for the treatment of DMD.

Overview of WMS Presentations

Summit and its collaborators will be making three poster presentations on the utrophin modulator programme at WMS and the details of these are below.  Copies of the presentations are also available on the Company's website, www.summitplc.com.

Utrophin modulators to treat Duchenne Muscular Dystrophy (DMD): Phase 1b clinical trial results of SMT C1100 (Poster # GP102)

Francesco Muntoni, Stefan Spinty, Helen Roper, Imelda Hughes, Valeria Ricotti, Alison Bracchi, Bina Tejura, David Roblin, Kay Davies, Jon Tinsley

A review of the Phase 1b clinical trial results that showed SMT C1100 was safe and well tolerated at all doses tested and a reduction observed in plasma levels of three enzymes associated with muscle damage; these reductions were statistically significant.  Inter-patient variability in the blood plasma levels of SMT C1100 were also observed and will be explored in future patient clinical studies.

New orally available compounds which modulate utrophin expression for the therapy of Duchenne Muscular Dystrophy (DMD) (Poster # GP89)

Simon Guiraud, Sarah E. Squire, Ben Edward, Nandini Shah, David T. Burns, Huijia Chen, Graham M. Wynne, Angela J. Russell, David J. Elsey, Francis X. Wilson, Jon Tinsley, Kay E. Davies

Positive preclinical data on second generation utrophin modulators reporting their enhanced bioavailability compared to SMT C1100 and their ability to increase expression of utrophin, improve the disease pathology, reduce muscle regeneration and protect muscle function.

Development of a multifaceted biomarker strategy to support clinical development of utrophin modulators for Duchenne Muscular Dystrophy (DMD) therapy (Poster # GP103)

Jonathon Tinsley, Francis X. Wilson, Narinder Janghra, Jennifer Morgan, Caroline Sewry, Francesco Muntoni, Kay E. Davies

Feasibility data on the quantification of utrophin protein in muscle biopsies and data on a biomarker measuring the level of muscle fibre regeneration are reported.  These two projects have been supported by Joining Jack and form part of Summit's wider programme developing exploratory biomarkers for use in future patient clinical trials.

About DMD and Utrophin Modulation

DMD is a progressive muscle wasting disease that affects around 50,000 boys in the developed world.  It is estimated that the value of the DMD market is in excess of $10 billion per annum based on the current pricing commanded by orphan drugs.  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 the functional equivalent of dystrophin and studies have shown that maintaining utrophin production has the potential to slow down or even stop the progression of DMD, regardless of the underlying dystrophin mutation.  It is also expected to be complementary to other therapeutic approaches in clinical trials.

About the UtroDMD Alliance

The UtroDMD Alliance is a multi-year strategic collaboration that combines the extensive biology, chemistry and drug discovery expertise of the University of Oxford and Summit, supported by the Medical Research Council and Muscular Dystrophy Campaign, with the sole aim to accelerate the development of utrophin modulator therapies for all DMD patients.

Summit is a drug discovery and development company targeting two high-value areas of unmet medical need:  the muscle wasting disease Duchenne Muscular Dystrophy and C. difficile infection. Summit is listed on the AIM market of the London Stock Exchange and trades under the ticker symbol SUMM. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc).

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Forward Looking Statements

This announcement, and the presentations referred to in it, contains "forward-looking statements", including, but not limited to, statements about the discovery, development and commercialisation of programme assets. These forward-looking statements are statements based on the Company's current intentions, beliefs and expectations, which include, among other things, the Company's results of operations, financial condition, prospects, growth, strategies and the industry in which the Company operates. No forward-looking statement is a guarantee of future performance and actual results could differ materially from those expressed or implied in the forward-looking statements. Accordingly, readers should not place undue reliance on forward-looking statements or information. Forward-looking statements and information by their nature involve known and unknown risks, uncertainties and other factors which may cause actual results, performance or achievements, or industry results, to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements or information. These include but are not limited to: adverse results in clinical or preclinical development studies; delays in obtaining regulatory approval; failure to obtain patent protection for inventions; commercial limitations imposed by patents owned or controlled by third parties; being unable to secure partnership agreements to develop and commercialise programme assets; being unable to secure the necessary funding to conduct any proposed research and development studies; and the ability to retain and recruit key personnel. The Company expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statement contained in this announcement to reflect any changes in expectations with regard thereto or any changes in events, conditions or circumstances on which any such statement is based, except as required by applicable law.