Summit Corp plc - BioMarin and Summit sign worldwide licensing agreement for Duchenne Muscular Dystrophy program
22 Jul 2008
BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN)
and Summit Corporation plc (AIM: SUMM) announced today
that they have entered into an exclusive
worldwide
licensing agreement for Summit's novel
preclinical candidate SMT C1100 and all follow-on
molecules, which are being developed to treat the fatal genetic
disorder Duchenne muscular dystrophy (DMD).
Under the terms of the licensing agreement, Summit will receive an upfront payment of $7 million in the form of an equity investment in Summit shares, future development and regulatory milestones totaling $51 million, tiered royalties rising to low teens, depending on sales and product sales milestones giving a total deal value of up to $143 million.
Summit will be responsible for completing the preclinical development of SMT C1100. BioMarin will be responsible for the clinical development, regulatory filing and commercialization of the product candidate.
"We are pleased to work with Summit on the Duchenne muscular dystrophy program. SMT C1100, an oral small molecule utrophin upregulator, has shown promise in animal models of DMD and may have the potential for treating the entire spectrum of DMD patients, not just those with a particular type of mutation," said Jean-Jacques Bienaim, Chief Executive Officer of BioMarin. "The DMD indication aligns well with our growing product development pipeline as it is a genetic disorder with no approved treatments. IND-enabling studies with SMT C1100 are underway, and we plan on entering the clinic in 2009. By leveraging our expertise in rapidly developing and commercializing products for focused patient populations, we hope to soon provide a new treatment option for all DMD patients."
Steven Lee Ph.D., Chief Executive of Summit, said, "We are very pleased that BioMarin has become our partner for the DMD program. BioMarin has an unparalleled track record in developing orphan drugs to market and has developed and launched successfully three such drugs in record time. The expertise and commitment of the BioMarin team gives me great confidence that they are an excellent partner for this program. I believe they will help to deliver SMT C1100 into a medicine in the shortest timeframe possible for the benefit of all DMD patients.
"For Summit, this deal is important as it is the first of
many that we anticipate signing from our broad pipeline of
assets including two clinical and two preclinical programs with
future research driven by our world leadership in
two innovative technology platforms. Our
business strategy is focused
on out-licensing or partnering
candidates at a preclinical or early clinical stage, where there is
a strong demand from pharma and biotech companies
looking to enhance their own pipelines, and this deal
provides important validation of this strategy."
The equity investment of $7.0 million will be made at 69 pence per share, calculated from the 60-day trailing share price at the time issue, upon which BioMarin will hold approximately 9.16% of the enlarged share capital of Summit.
About Duchenne muscular dystrophy (DMD) and SMT C1100
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 meaning DMD patients lack 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.
Summit has identified SMT C1100, a
proprietary, orally available small molecule with
a novel mechanism of action for DMD. SMT C1100
acts to modify the progression of DMD by replacing dystrophin with
an endogenous, functionally similar protein
called utrophin. Summit believes the primary
advantage of SMTC1100 is that it offers
the
potential to treat the entire
DMD patient population. Summit
recently presented important
preclinical data for SMT C1100
demonstrating significantly improvements in the strength and
function of muscles in in vivo models.
Due to the low patient population and high unmet medical need,
DMD is designated as an orphan indication by the regulatory
agencies. Orphan products can expect to receive
additional regulatory support and accelerated approval
in addition to seven and ten years of
market exclusivity in the US and EU respectively upon designation
by the FDA
and the EMEA.
For further information:
BioMarin Pharmaceutical, Inc.
Eugenia
Shen, Investor Relations
Tel: +1 (415) 506-6570
Susan Berg, Media Relations
Tel: +1 (415) 506-6594
Summit plc
Steven Lee, PhD, Chief
Executive Officer
Darren Millington, ACMA, Chief Financial Officer
Richard Pye, PhD, Investor Relations
Tel: +44 (0)1235 443951
Citigate Dewe Rogerson
Mark Swallow, PhD / David Dible / Sylvie Berrebi / Emma
Palmer
Tel: +44 (0)207 638 9571
Panmure Gordon
Andrew Burnett / Rakesh Sharma (Corporate Finance)
Ashton Clanfield (Corporate Broking)
Tel: +44 (0)207 459 3600
About BioMarin
BioMarin develops and commercializes
innovative biopharmaceuticals for serious diseases and
medical conditions. The company's product portfolio
comprises three approved products and multiple clinical and
preclinical 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; and Kuvan
(sapropterin dihydrochloride) Tablets, a
product for the treatment of phenylketonuria
(PKU), developed in partnership with Merck
Serono, a division of Merck KGaA of Darmstadt, Germany.
Other product candidates include 6R-BH4 for
cardiovascular indications,
which is currently in Phase 2 clinical development for the
treatment of peripheral arterial disease and sickle cell
disease, and PEG-PAL (PEGylated recombinant
phenylalanine ammonia lyase),
which is currently in Phase 1 clinical development for
the treatment of PKU.
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 biotechnology company with a broad
preclinical and clinical pipeline, two world-leading technology
platforms and an innovative business model that is expected to
generate sustainable value for investors. Summit is
developing many
drug programmes that target unmet medical needs from which it
intends to generate value by out-licensing attractive late
preclinical or early clinical stage programmes in return for
upfront, milestone and royalty payments. Summit uses its scientific
expertise to target
orphan indications, neuro-disorders and infectious diseases.
Summit's drug pipeline is supported by its world leadership in two
innovative technology platforms: carbohydrate chemistry and
zebrafish biology. These platforms support existing programmes and
also will be the source of future programmes to refuel Summit's
drug pipeline. These platform technologies also form the basis of
the Company's revenue generating service business. 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.
Forward-Looking Statements
For BioMarin
This press release contains forward-looking
statements about the
business prospects of BioMarin
Pharmaceutical Inc., including,
without limitation, statements about: the expected development of
SMT
C1100, the continued clinical development and
commercialization of
BioMarin's products and other product
candidates and actions by
regulatory authorities. These
forward-looking statements are
predictions and involve risks and uncertainties
such that actual
results may differ materially from these statements. These
risks and
uncertainties include, among others: results of planned
and ongoing
preclinical and clinical trials, including preclinical trials
of SMT
C1100; the content and timing of decisions by the U.S. Food
and Drug
Administration, the European
Commission and other regulatory
authorities concerning each of the described
products and product
candidates; and those factors detailed in BioMarin's filings with
the
Securities and Exchange Commission, including,
without limitation,
the factors contained under the caption "Risk Factors" in
BioMarin's
2007 Annual Report on Form 10-K. Stockholders are urged not
to place
undue reliance on forward-looking statements, which speak only
as of
the date hereof. BioMarin is under no
obligation, and expressly
disclaims any obligation to update or
alter any forward-looking
statement, whether as a result of new information, future
events or
otherwise.
For Summit plc
This press release 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 Summit Corporation
plc'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. Summit's actual results may differ
materially
from those contemplated by the forward-looking statements.
Summit
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 include regional, national,
global
political, economic, business, competitive, market and
regulatory
conditions.
