BioMarin and Serono Form Strategic Alliance for the Development and Commercialization of Phenoptin(TM) and Phenylase(TM)
May 16 2005 - 1:30AM
PR Newswire (US)
BioMarin and Serono Form Strategic Alliance for the Development and
Commercialization of Phenoptin(TM) and Phenylase(TM) Phenoptin
Currently in Phase 3 Trials NOVATO, Calif. and GENEVA, 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. Contacts: For BioMarin: Investors Media Joshua A. Grass
Susan Ferris BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical
Inc. (415) 506-6777 (415) 506-6701 http://www.bmrn.com/ For Serono:
Corporate Media Relations: Corporate Investor Relations: Tel: +41
22 739 36 00 Tel: +41 22 739 36 01 Fax: +41 22 739 30 85 Fax: +41
22 739 30 22 http://www.serono.com/ Reuters: SEO.VX / SRA.N
Bloomberg: SEO VX / SRA US Media Relations, USA: Investor
Relations, USA: Tel: +1 781 681 2340 Tel: +1 781 681 2552 Fax: +1
781 681 2935 Fax: +1 781 681 2912 http://www.seronousa.com/
DATASOURCE: BioMarin Pharmaceutical Inc.; Serono CONTACT:
Investors: Joshua A. Grass, +1-415-506-6777, Media: Susan Ferris,
+1-415-506-6701, both of BioMarin Pharmaceutical Inc.; Corporate
Media Relations: +41-22-739-36-00, Fax: +41-22-739-30-85, Media
Relations, USA: +1-781-681-2340, Fax: +1-781-681-2935, Corporate
Investor Relations: +41-22-739-36-01, +41-22-739-30-22, Investor
Relations, USA: +1-781-681-2552, Fax: +1-781-681-2912, all of
Serono Web site: http://www.bmrn.com/ http://www.serono.com/
http://www.seronousa.com/
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