BioMarin Acquires Huxley Pharmaceuticals, Inc.

Mon Oct 26, 2009 4:40pm EDT
 
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Sole Product of Huxley is a Proprietary Form of 3,4-Diaminopyridine,
Amifampridine Phosphate










NOVATO, Calif., Oct. 26 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc.
(Nasdaq: BMRN) announced today that it has acquired Huxley Pharmaceuticals,
Inc. (Huxley), which has rights to a proprietary form of 3,4-diaminopyridine
(3,4-DAP), amifampridine phosphate, for the rare autoimmune disease Lambert
Eaton Myasthenic Syndrome (LEMS).  Last week, the Committee for Medicinal
Products for Human Use of the European Medicines Evaluations Agency adopted a
positive opinion recommending approval of amifampridine phosphate for LEMS. 
If approved by the European Commission, amifampridine phosphate will be the
first approved treatment for LEMS, thereby conferring orphan drug protection
and providing ten years of market exclusivity in Europe. Huxley licensed the
rights to 3,4-DAP from EUSA Pharma, which was developing the product after
acquiring the rights from the original developer, Assistance Publique Hopitaux
de Paris (AP-HP).  

"This acquisition represents a natural extension of BioMarin's core business
operations and strategy.  LEMS is a rare, serious and debilitating autoimmune
disease treated by neuromuscular specialists," said Jean-Jacques Bienaime,
Chief Executive Officer of BioMarin.  "This deal leverages our existing
European infrastructure and commercial capabilities and provides the
opportunity for near-term revenue growth and operating income growth.  We
expect to launch the product in Europe in the first quarter of 2010, and are
evaluating the best development strategy for amifampridine phosphate in LEMS
in the U.S. and in other indications in the U.S. and Europe. We will also
evaluate development of amifampridine phosphate in other indications including
multiple sclerosis. We expect this deal to be dilutive in 2010 and accretive
in 2011 and beyond."   

Stephen Aselage, Senior Vice President and Chief Business Officer of BioMarin
added, "3,4-DAP is currently the treatment of choice for LEMS.  Although its
use has been limited as an unapproved product due to regulatory restrictions
and limited availability of drug product, 3,4-DAP has been studied in six
randomized controlled trials and has been shown to improve muscle strength in
LEMS patients as measured by a variety of means.  3,4-DAP has been widely
recommended for use in LEMS, and the introduction of amifampridine phosphate
will enable regular access to a high quality, stable proprietary product."  

Bryan Morton, President and Chief Executive Officer of EUSA Pharma said, "We
are pleased now to be working with BioMarin to develop and commercialize
3,4-DAP. BioMarin is well placed to launch this important treatment for LEMS,
and to continue its future broader development." 

Under the terms of the agreement, BioMarin paid Huxley stockholders $15.0
million upfront and will pay an additional $7.5 million upon final European
Commission approval of amifampridine in LEMS, which is expected in late 2009
or early 2010.  Additionally, Huxley stockholders are eligible to receive up
to approximately $36.0 million in milestone payments if certain annual,
cumulative sales and U.S. development milestones are met. In addition,
successful development of multiple sclerosis will result in milestone payments
to EUSA.

Conference Call Details

BioMarin will host a conference call and webcast to discuss the acquisition of
Huxley Pharmaceuticals today, Monday, October 26, at 5:30 p.m. ET. This event
can be accessed on the investor section of the BioMarin website at
www.BMRN.com.


    Date: October 26, 2009
    Time: 5:30 p.m. ET
    U.S. / Canada Dial-in Number:  866.788.0544
    International Dial-in Number:  857.350.1682
    Participant Code: 53851486
    Replay Dial-in Number: 888.286.8010
    Replay International Dial-in Number: 617.801.6888
    Replay Code: 48079886


About LEMS 

Lambert Eaton Myasthenic Syndrome (LEMS) is a rare autoimmune disease with the
primary symptoms of muscle weakness. Muscle weakness in LEMS is caused by
autoantibodies to voltage gated calcium channels leading to a reduction in the
amount of acetylcholine released from nerve terminals. The prevalence of LEMS
is estimated at four to ten per million, or approximately 2,000 to 5,000
patients in the EU and 1,200 to 3,100 patients in the U.S. Approximately 50
percent of LEMS patients diagnosed have small cell lung cancer.

Patients with LEMS typically present with fatigue, muscle pain and stiffness.
The weakness is generally more marked in the proximal muscles particularly of
the legs and trunk. Other problems include reduced reflexes, drooping of the
eyelids, facial weakness and problems with swallowing. Patients often report a
dry mouth, impotence, constipation and feelings of light headedness on
standing. On occasion these problems can be life threatening when the weakness
involves respiratory muscles.  A diagnosis of LEMS is generally made on the
basis of clinical symptoms, electromyographic testing and the presence of
autoantibodies against voltage gated calcium channels. 

Current treatment of LEMS can consist of strategies directed at the underlying
malignancy if one is present.  Unfortunately, therapy of small cell lung
cancer is limited and outcomes are generally poor.  Immunosuppressive agents
have been tried but success is limited by toxicity, and difficulty
administering the regimens.  A mainstay of therapy has been 3,4-DAP but its
use in practice has been limited by the drug's availability.  This problem
will be addressed by the introduction of BioMarin's product.

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 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; and Kuvan® (sapropterin dihydrochloride) Tablets,
for phenylketonuria (PKU), developed in partnership with Merck Serono, a
division of Merck KGaA of Darmstadt, Germany. Other product candidates include
PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is
currently in Phase II clinical development for the treatment of PKU and GALNS
(N-acetylgalactosamine 6-sulfatase), which is currently in Phase I/II clinical
development for the treatment of MPS IVA. For additional information, please
visit www.BMRN.com. Information on BioMarin's website is not incorporated by
reference into this press release.

About Huxley Pharmaceuticals 

Huxley Pharmaceuticals, Inc. is private life sciences company founded and
managed by Aceras BioMedical, LLC.  Aceras is dedicated to funding and
developing novel innovations that address unmet medical needs. 

About EUSA Pharma

EUSA Pharma is a transatlantic specialty pharmaceutical company focused on
in-licensing, developing and marketing late-stage oncology, pain control and
critical care products. The company currently has eight marketed products,
including Caphosol® for the treatment of oral mucositis, a common and
debilitating side-effect of radiation therapy and high dose chemotherapy,
Erwinase® and Kidrolase® for the treatment of acute lymphoblastic leukemia,
Collatamp® G, a surgical implant impregnated with the antibiotic gentamicin,
ProstaScint® for imaging the extent and spread of prostate cancer and
Quadramet® for the treatment of pain in patients whose cancer has spread to
the bones. EUSA also has several products in development.

About AP-HP

Assistance Publique - Hopitaux de Paris (AP-HP) is a public health federation
that gathers together 37 hospitals for the Paris metropolitan area. Its
activities are medical care (750 medical units / 52 medical and biological
specialties), education (37 formation centers), and research performed either
independently (AP HP labs and pharmaceutical departments), in partnership with
hosted research units (112 INSERM and 30 CNRS units), or in partnership with
external entities (public and private).


Forward-Looking Statement

This press release contains forward-looking statements about the business
prospects of BioMarin Pharmaceutical Inc., including, without limitation,
statements about: the expectations of the development and potential approval
of Huxley's 3,4-Diaminopyridine product for the treatment of LEMS. 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: the content and timing of
decisions by the U.S. Food and Drug Administration, the European Commission
and other regulatory authorities, particularly the pending decision by the
European Commission on the Marketing Authorization Application for such
product, our success in the commercialization of such product, if approved;
results and timing of current and planned preclinical studies and clinical
trials related to such product; our ability to successfully manufacture the
product ; 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 2008 Annual Report on Form
10-K, and the factors contained in BioMarin's reports on Form 10-Q.
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.

BioMarin®, Naglazyme® and Kuvan® are registered trademarks of BioMarin
Pharmaceutical Inc.

Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.

    Contact:

    Investors                          Media
    Eugenia Shen                       Susan Berg
    BioMarin Pharmaceutical Inc.       BioMarin Pharmaceutical Inc.
    (415) 506-6570                     (415) 506-6594






SOURCE  BioMarin Pharmaceutical Inc.

Investors, Eugenia Shen, +1-415-506-6570, or Media, Susan Berg,
+1-415-506-6594, both of BioMarin Pharmaceutical Inc.

 

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