BioMarin Licenses Factor VIII Gene Therapy Program for Hemophilia A From University College London and St. Jude Children's

BioMarin Licenses Factor VIII Gene Therapy Program for Hemophilia A From
University College London and St. Jude Children's Research Hospital

SAN RAFAEL, Calif., Feb. 21, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) announced today that it has licensed a Factor VIII gene
therapy program for hemophilia A from University College London (UCL) and St.
Jude Children's Research Hospital. The company expects to select a development
candidate this year, initiate and complete IND-enabling toxicology studies
next year and initiate proof of concept human studies by the end of 2014. The
license and commitment to support the research program was made possible by
UCL Business, UCL's wholly-owned technology transfer company, working with
Professor Amit Nathwani of the UCL Cancer Institute.

"Gene therapy is emerging as a powerful and viable way to treat genetic
disorders and is complementary to our current suite of commercial products and
research programs," said Jean-Jacques Bienaimé, Chief Executive Officer of
BioMarin."Hemophilia is an attractive target for gene therapy as factor
levels in the blood serve as good biomarkers, relatively low factor levels are
required for a clinically important benefit in severe patients and the current
standard of care of intravenous infusions three times a week is quite
onerous.We remain committed to maintaining a rich pipeline with the goal of
filing an IND every twelve to eighteen months."

Mr. Cengiz Tarhan, Managing Director of UCL Business said, "This is an
excellent partnership for UCL Business, which combines the world class
translational research strengths of Professor Nathwani and his team with the
significant development and commercialization capabilities of BioMarin to
progress this ground breaking therapy for hemophilia A."

Professor Stephen Caddick, Vice-Provost (Enterprise) at University College
London added, "UCL and BioMarin each bring distinct strengths to the
partnership. UCL is a world leader in the biomedical sciences, with an
unremitting commitment to outstanding research and translation into healthcare
benefits for patients.We welcome this partnership which will continue to
build on the excellence of our research to fully explore the potential of gene
therapy as a life-saving treatment for people with hemophilia."

Andrew Davidoff, M.D., Chair, Surgery, St. Jude Children's Research Hospital,
added, "We are pleased that our research with UCL on gene therapy for
hemophilia has led to the development of a potential therapeutic tool for
treating this devastating disease.This licensing agreement underscores St.
Jude's commitment to rapidly translating our research into effective clinical
interventions."

About Hemophilia A

The current market for hemophilia A products is about $6.0 billion worldwide.
There are approximately 90,000 patients in territories where BioMarin has
commercial operations and an annual incidence of about 400 new patients in the
U.S.The standard of care for the 60 percent of hemophilia A patients who are
severe is a prophylactic regimen of IV infusions three times per week. Even
with the likely prospect of less frequently dosed products coming to the
market, feedback from thought leaders indicates that significant unmet need
will remain as factor replacement therapy will inevitably leave patients
vulnerable to bleeding events.Many patients on factor replacement therapy
still have bleeding events and experience debilitating damage to joints as a
result of chronically low factor levels.

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious
diseases and medical conditions. The company's product portfolio comprises
four 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; Kuvan® (sapropterin dihydrochloride) Tablets, for
phenylketonuria (PKU), developed in partnership with Merck Serono, a division
of Merck KGaA of Darmstadt, Germany; and Firdapse™ (amifampridine), which has
been approved by the European Commission for the treatment of Lambert Eaton
Myasthenic Syndrome (LEMS). Product candidates include BMN-110
(N-acetylgalactosamine 6-sulfatase), formally referred to as GALNS, which
successfully completed Phase III clinical development for the treatment of MPS
IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is
currently in Phase II clinical development for the treatment of PKU, BMN-701,
a novel fusion protein of insulin-like growth factor 2 and acid alpha
glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development
for the treatment of Pompe disease, BMN-673, a poly ADP-ribose polymerase
(PARP) inhibitor, which is currently in Phase I/II clinical development for
the treatment of genetically-defined cancers, and BMN-111, a modified
C-natriuretic peptide, which is currently in Phase I clinical development for
the treatment of achondroplasia. For additional information, please visit
www.BMRN.com. Information on BioMarin's website is not incorporated by
reference into this press release.

The BioMarin Pharmaceutical Inc. logo is available at
http://www.globenewswire.com/newsroom/prs/?pkgid=11419

About UCLB

UCLB is a leading technology transfer company that supports and commercialises
research and innovations arising from UCL, one of the UK's top research-led
universities.

UCLB has a successful track record and a strong reputation for identifying and
protecting promising new technologies and innovations from UCL academics. It
invests directly in development projects to maximise the potential of the
research and manages the commercialisation process of technologies from the
laboratory to market.

UCLB supports UCL's Grand Challenges of increasing UCL's positive impact on
and contribution to Global Health, Sustainable Cities, Intercultural
Interaction and Human Wellbeing.

For further information, please visit www.uclb.com

St. Jude Children's Research Hospital

St. Jude Children's Research Hospital is internationally recognized for its
pioneering research and treatment of children with cancer and other
life-threatening diseases. The hospital's research has helped push overall
survival rates for childhood cancer from less than 20 percent when the
institution opened to almost 80 percent today. It is the first and only
National Cancer Institute-designated Comprehensive Cancer Center devoted
solely to children, and no family ever pays St. Jude for anything. For more
information, visit www.stjude.org. Follow us on Twitter @StJudeResearch.

Forward-Looking Statement

This press release contains forward-looking statements about the business
prospects of BioMarin Pharmaceutical Inc., including, without limitation,
statements about: expectations related to the development of the Factor VIII
gene therapy program and the timing of a clinical trial of the product
candidate. 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 and
timing of current and planned preclinical studies and clinical trials; the
content and timing of decisions by the U.S. Food and Drug Administration, the
European Commission and other regulatory authorities concerning the program,;
the ability to manufacture the product candidate 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 2011 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^®, Kuvan^® and Firdapse™ are registered trademarks of
BioMarin Pharmaceutical Inc.

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

CONTACT: Investors:
         Eugenia Shen
         BioMarin Pharmaceutical Inc.
         (415) 506-6570
        
         Media:
         Debra Charlesworth
         BioMarin Pharmaceutical Inc.
         (415) 455-7451

BioMarin Pharmaceutical Inc. Logo
 
Press spacebar to pause and continue. Press esc to stop.