Phenoptin Currently in Phase 3 Trials

    NOVATO, California, and GENEVA, Switzerland, May 16
/PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN)
and Serono (virt-x: SEO and NYSE: SRA) announced today that they have formed
a strategic alliance for the further development and commercialization of two
BioMarin product candidates, Phenoptin (sapropterin hydrochloride) and
Phenylase (phenylalanine ammonia lyase). Both products have shown potential
in the treatment of phenylketonuria (PKU), and there is preliminary clinical
evidence that suggests that the active ingredient in Phenoptin, a synthetic
form of the naturally occurring enzyme cofactor 6R-BH4, may also be useful in
the treatment of other serious diseases, including diabetes and
cardiovascular diseases.
    By the terms of their agreement, Serono acquires exclusive rights to
market the products in all territories outside the United States and Japan.
BioMarin retains exclusive rights to market the products in the United
States. Serono will make an upfront payment of $25 million to BioMarin, and
will make additional milestone payments of up to $232 million based on the
successful development and registration of both products in multiple
indications, of which $45 million are associated specifically with Phenoptin
in PKU. Serono will also pay BioMarin undisclosed royalties on its net sales
of the products. The companies will share equally all development costs
following successful completion of Phase 2 trials for each product candidate
in each indication.
    BioMarin is currently investigating Phenoptin, an orally administered
product, in a Phase 3 clinical trial for the treatment of PKU. PKU is an
inherited metabolic disease caused by a deficiency of the enzyme
phenylalanine hydroxylase, resulting in elevated levels of phenylalanine in
the blood, which can result in serious neurological damage. There is
currently no approved drug to treat PKU, which affects at least 50,000
diagnosed patients under the age of 40 worldwide. Phenylase, an enzyme
substitution therapy for the treatment of severe forms of PKU, is currently
in preclinical development.
    Emil Kakkis MD, PhD, Senior Vice President of Business Operations at
BioMarin commented, "We are very pleased to enter into this partnership with
Serono, a top-tier biotechnology company with strong global development
capabilities and commercial operations. Serono's expertise in developing and
marketing novel products for metabolic and genetic diseases will be an
invaluable asset for this partnership."
    "We are delighted to partner with BioMarin for the further development
and commercialization of Phenoptin and Phenylase, products that fit very
nicely into our metabolic endocrinology franchise," said Franck Latrille,
Senior Executive Vice President Global Product Development at Serono. "PKU is
a severely debilitating condition afflicting patients from birth and
throughout their lives. Serono's strategy is to invest in bringing to market
innovative products like Phenoptin and Phenylase that address significant
unmet medical needs."
    About BioMarin
    BioMarin develops and commercializes innovative biopharmaceuticals for
serious diseases and medical conditions. The company's product portfolio is
comprised of two approved products, Aldurazyme(R) (laronidase) for
mucopolysaccharidosis I (MPS I), and Orapred(R) (prednisolone sodium
phosphate oral solution) for severe asthma, and multiple product candidates
including rhASB (galsulfase), a BLA/MAA-stage product candidate for the
treatment of mucopolysaccharidosis VI (MPS VI), and Phenoptin(TM)
(sapropterin hydrochloride), a Phase 3 product candidate for the treatment of
phenylketonuria (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 Serono
    Serono is a global biotechnology leader. The Company has eight
biotechnology products, Rebif(R), Gonal-f(R), Luveris(R), Ovidrel(R
)/Ovitrelle(R), Serostim(R), Saizen(R), Zorbtive(TM) and Raptiva(R). In
addition to being the world leader in reproductive health, Serono has strong
market positions in neurology, metabolism and growth and has recently entered
the psoriasis area. The Company's research programs are focused on growing
these businesses and on establishing new therapeutic areas, including
oncology. Currently, there are approximately 30 ongoing development projects.
    In 2004, Serono achieved worldwide revenues of US$2,458.1 million, and a
net income of US$494.2 million, making it the third largest biotech company
in the world. Its products are sold in over 90 countries. Bearer shares of
Serono S.A., the holding company, are traded on the virt-x (SEO) and its
American Depositary Shares are traded on the New York Stock Exchange (SRA).
    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 development of its product candidates Phenoptin and
Phenylase; expectations related to the possible use of Phenoptin for the
treatment of indications other than PKU; and the possible milestone revenue
from development of these product candidates. 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 results of preclinical and clinical
trials related to Phenoptin and Phenylase, both for the treatment of PKU and
for indications other than PKU; results and timing of current and planned
clinical trials of Phenoptin for the treatment of PKU; the content and timing
of decisions by the U.S. Food and Drug Administration, the European Medicines
Agency and other regulatory authorities concerning Phenoptin and Phenylase;
the ability to develop suitable formulations of Phenylase; the ability to
develop commercial scale manufacturing for Phenoptin and Phenylase; and those
factors detailed in BioMarin's filings with the Securities and Exchange
Commission, including, without limitation, the factors contained under the
caption "Factors That May Affect Future Results" in BioMarin's 2004 Annual
Report on Form 10-K and the factors contained in BioMarin's reports on Forms
10-Q and 8-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
    For Serono
    Some of the statements in this press release are forward looking. Such
statements are inherently subject to known and unknown risks, uncertainties
and other factors that may cause actual results, performance or achievements
of Serono S.A. and affiliates to be materially different from those expected
or anticipated in the forward-looking statements. Forward-looking statements
are based on Serono's current expectations and assumptions, which may be
affected by a number of factors, including those discussed in this press
release and more fully described in Serono's Annual Report on Form 20-F filed
with the U.S. Securities and Exchange Commission on March 16, 2005. These
factors include any failure or delay in Serono's ability to develop new
products, any failure to receive anticipated regulatory approvals, any
problems in commercializing current products as a result of competition or
other factors, our ability to obtain reimbursement coverage for our products,
the outcome of government investigations and litigation and government
regulations limiting our ability to sell our products. Serono has no
responsibility to update the forward-looking statements contained in this
press release to reflect events or circumstances occurring after the date of
this press release.
    Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC.
    Orapred(R) is a registered trademark of Medicis Pediatrics, Inc., and is
used under license.

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