BioMarin and Summit plc Sign Worldwide Licensing Agreement for Duchenne Muscular...

BioMarin and Summit plc Sign Worldwide Licensing Agreement for Duchenne
Muscular Dystrophy Program

NOVATO, Calif. and OXFORD, England, July 22 /PRNewswire-FirstCall/ --
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 the low teens, depending on sales and product sales
milestones, giving a total deal value of up to $143 million. The total
expense to BioMarin in 2008 is expected to be approximately $1.8 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 Bienaime, 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 (pounds Sterling 3.54 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 SMT C1100 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.
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(R) (galsulfase) for
mucopolysaccharidosis VI (MPS VI), a product wholly developed and
commercialized by BioMarin; Aldurazyme(R) (laronidase) for
mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a
50/50 joint venture with Genzyme Corporation; and Kuvan(R) (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 http://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 http://www.summitplc.com.
Forward-Looking Statements
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.
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.
BioMarin Summit plc
Investors Steven Lee, CEO
Eugenia Shen Darren Millington, CFO
+1 (415) 506-6570 Richard Pye, Investor Relations
+44 (0)1235 443951
Media
Susan Berg Citigate Dewe Rogerson
+1 (415) 506-6594 Mark Swallow /Sylvie Berrebi /David Dible/Emma
Palmer
+44 (0)20 7282 2948

SOURCE BioMarin Pharmaceutical Inc.

Investors, Eugenia Shen, +1-415-506-6570, or Media, Susan Berg,
+1-415-506-6594, both of BioMarin Pharmaceutical Inc.; or Steven Lee, CEO, or
Darren Millington, CFO, or Investor Relations, Richard Pye, all of Summit
Corporation plc, +44 (0)1235 443951; or, Mark Swallow, or Sylvie Berrebi, or
David Dible, or Emma Palmer, +44 (0)20 7282 2948, all of Citigate Dewe
Rogerson, for Summit Corporation plc