Summit Corporation plc
("Summit plc" or "the Company")
BIOMARIN INITIATES PHASE 1 CLINICAL STUDY OF SMT C1100 FOR DUCHENNE MUSCULAR
DYSTROPHY
Oxford, UK, 12 January 2010, Summit (AIM: SUMM), a leading UK drug discovery
company with a portfolio of partnered drug programmes and an innovative
iminosugar technology platform, is pleased to note that one of the Company's
partners, BioMarin Pharmaceuticals Inc. (Nasdaq: BMRN), announced on 11 January
2010 that it has initiated a Phase 1 clinical study of SMT C1100 (also known as
BMN 195). SMT C1100 is a small molecule utrophin upregulator for the treatment
of the fatal genetic disorder Duchenne muscular dystrophy (DMD). BioMarin
indicated that initial top-line results are expected in Q3 2010.
The DMD programme is one of eight drug programmes that form Summit's partnered
product portfolio. This portfolio requires no further investment from Summit
but has contractual, success based development, regulatory and sales milestone
payments totalling over $160 million plus sales royalties of up to 13%.
The Phase 1 clinical trial is a single centre, double blind, placebo-controlled,
single dose-escalation study followed by a multiple-dose study of SMT C1100
administered orally in healthy volunteers. The primary objective is to assess
the safety, tolerability and pharmacokinetics of SMT C1100 in healthy
volunteers, and enable subsequent studies in patients with DMD.
Commenting on this news, Steven Lee, PhD, Chief Executive Officer of Summit
said, "This is a significant and exciting development for Summit and the
Duchenne muscular dystrophy programme, which was partnered with BioMarin in a
multi-million dollar deal in July 2008.
"This programme was one of Summit's original drug discovery programmes and it
was the work of our scientists that identified SMT C1100 as a potential first in
class treatment for DMD. The advances made since partnering the programme
endorse our belief that BioMarin, with their unparalleled track record in
developing orphan drugs to market, are the best partner to develop SMT C1100
into a medicine in the shortest possible timeframe for the benefit of all DMD
patients."
Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin added, "Duchenne
muscular dystrophy represents a serious unmet medical need affecting
approximately 40,000 patients in the developed world, and we are excited to
advance our program into the clinic in hopes of providing the first therapeutic
option to treat this disease, SMT C1100 has been shown to upregulate utrophin
levels in human muscle cells, as a means of augmenting muscle function. In mice
with mutations in the normal dystrophin gene, SMT C1100 has been shown to
improve strength. Therefore, SMT C1100 may have the potential to treat the
entire spectrum of DMD patients, regardless of the type of genetic abnormality."
- END -
For more information, please contact:
Summit
Steven Lee, PhD
Richard Pye, PhD
Tel: +44 (0)7825 313476
Singer Capital Markets
Shaun Dobson
Claes Spång
Tel: +44 (0)20 3205 7500
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a fatal neuromuscular disorder that affects 1 in
3,500 boys with an estimated patient population of over 40,000 in the developed
world.
DMD is caused by a genetic defect that results in DMD patients lacking an
important protein called dystrophin, which is crucial to maintaining muscle
integrity and function. The absence of dystrophin results in extensive muscle
wasting in all voluntary muscles as well as the heart and breathing muscles and
causes severe restriction in the mobility of DMD patients by their early teens
and is ultimately fatal, generally in their twenties. Currently there is no cure
for DMD. Corticosteroid treatment is the only frontline therapy and acts to
only delay the progression of the disease.
About SMT C1100
SMT C1100 (also known as BMN 195) is a proprietary, orally available small
molecule with a novel mechanism of action for DMD. SMT C1100 acts by increasing
expression of utrophin, an endogenous protein that is functionally similar to
dystrophin. The goal of therapy would be to preserve muscle function and
prevent the inexorable decline in strength seen in DMD patients. Summit and
BioMarin believes the primary advantage of SMT C1100 is that it offers the
potential to treat the entire DMD patient population, regardless of the mutation
the patient carries.
About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals for serious
diseases and medical conditions. The company's product portfolio comprises four
approved products and multiple clinical and pre-clinical product candidates.
Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI
(MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme®
(laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin
developed through a 50/50 joint venture with Genzyme Corporation; Kuvan®
(sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in
partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany;
and 3,4-diaminopyridine (amifampridine phosphate), which has been approved by
the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome
(LEMS). For additional information, please visit www.BMRN.com. Information on
BioMarin's website is not incorporated by reference into this press release.
About Summit plc
Summit plc is a leading UK based drug discovery company with a portfolio of
partnered drug programmes and a major focus on developing new therapeutics from
its iminosugar drug discovery platform.
Summit has a commercial track record of signing programme agreements and
currently has a product portfolio comprising of eight drug programmes with
partners including BioMarin, Orient Pharma, Evolva, the Wellcome Trust and the
Lilly TB Drug Discovery Initiative. This portfolio requires no further
investment from Summit but in the future may generate success based milestone
payments and sales royalties for the Company.
Summit believes iminosugars are the key to gaining access to several disease
mechanisms where classical drug candidates have had little success, and
therefore offer a major opportunity for the discovery and development of new
medicines.
Carbohydrates play critical roles in maintaining correct function of many normal
processes in healthy individuals and provide a wealth of new targets for drug
discovery. Iminosugars have the capability of accessing such targets and offer
the potential of generating new medicines in a variety of major therapy areas.
Summit is currently focussed on metabolic diseases, including diabetes, and
anti-virals.
The company listed on the alternative investment market (AIM) of the London
Stock Exchange in October 2004 - symbol: SUMM. Further information about the
company is available at www.summitplc.com <
http://www.summitplc.com/>.
Forward Looking Statements
This document contains "forward-looking statements" within the meaning of the
U.S. Private Securities Litigation Reform Act of 1995. Forward-looking
statements can be identified by words such as "anticipates", "intends", "plans",
"seeks", "believes", "estimates", "expects" and similar references to future
periods, or by the inclusion of forecasts or projections.
Forward-looking statements are based on the Company's current expectations and
assumptions regarding our business, the economy and other future conditions.
Because forward-looking statements relate to the future, by their nature, they
are subject to inherent uncertainties, risks and changes in circumstances that
are difficult to predict. The Company's actual results may differ materially
from those contemplated by the forward-looking statements. The Company cautions
you therefore that you should not rely on any of these forward-looking
statements as statements of historical fact or as guarantees or assurances of
future performance. Important factors that could cause actual results to differ
materially from those in the forward-looking statements and regional, national,
global political, economic, business, competitive, market and regulatory
conditions.
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