BioMarin Announces Selection of Factor VIII Gene Therapy Drug Development Candidate BMN 270 for the Treatment of Hemophilia A

BioMarin Announces Selection of Factor VIII Gene Therapy Drug Development
Candidate BMN 270 for the Treatment of Hemophilia A

SAN RAFAEL, Calif., Jan. 13, 2014 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) announced today that it has selected an AAV-factor VIII
vector, BMN 270, to develop for the treatment of hemophilia A and has
initiated IND-enabling studies. BioMarin expects to initiate clinical studies
with BMN 270 in early 2015. The Company's gene therapy program for hemophilia
A was originally licensed from University College London (UCL) and St. Jude
Children's research Hospital in February 2013 and has since been developed at
BioMarin's facilities. 

"In our hemophiliac factor VIII deficient mouse models, BMN 270 restored
factor VIII plasma concentrations to levels projected to be adequate for
normal clotting in humans," said Barrie Carter, Ph.D., Vice President, Vector
Biology, of BioMarin."In the past eleven months since we acquired the
AAV-factor VIII technology from UCL and St. Jude, we have evaluated a large
number of candidate vectors for treating hemophilia A, and we are pleased to
have selected BMN 270 as BioMarin's next IND candidate."

"Gene therapy is emerging as a powerful 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 and the current standard of care of multiple
intravenous infusions per week is quite onerous for patients. We remain
committed to maintaining a rich pipeline with the goal of filing an IND every
twelve to eighteen months."

Professor Amit Nathwani, Ph.D., at University College London added, "UCL is a
world leader in the biomedical sciences, with an unremitting commitment to
outstanding research and translation into healthcare benefits for patients. We
are pleased to collaborate with BioMarin in this exciting field of gene
therapy for hemophilia and keen to see a revolution in treatment for this
debilitating condition."

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 with 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 severely affected is a prophylactic regimen of IV infusions three times
per week. Even with prophylactic regimens, many patients still experience
spontaneous bleeding events that result in progressive and debilitating joint
damage due to 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 MPS VI, a
product wholly developed and commercialized by BioMarin; Aldurazyme®
(laronidase) for 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 VIMIZIM™ (N-acetylgalactosamine 6-sulfatase), formally referred to as
GALNS, which successfully completed Phase 3 clinical development for the
treatment of MPS IVA, PEG PAL (PEGylated recombinant phenylalanine ammonia
lyase), which is currently in Phase 3 clinical development for the treatment
of PKU, BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, which is
currently in Phase 3 clinical development for the treatment of germline BRCA
breast cancer, BMN 701, a novel fusion of acid alpha glucosidase (GAA) with a
peptide derived from insulin like growth factor 2, which is currently in Phase
1/2 clinical development for the treatment of Pompe disease, BMN 111, a
modified C-natriuretic peptide, which is currently in Phase 1 clinical
development for the treatment of achondroplasia and BMN 190, a recombinant
human tripeptidyl peptidase-1 (rhTPP1) for the treatment of late-infantile
neuronal ceroid lipofuscinosis (CLN2), a form of Batten Disease. For
additional information, please visit www.BMRN.com. Information on BioMarin's
website is not incorporated by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business
prospects of BioMarin Pharmaceutical Inc., including, without limitation,
statements about BMN 270, including the expected timing of the pre-clinical
trials and initiation of clinical trials of the 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: the results of current and
ongoing preclinical trials, particularly the IND-enabling toxicology; the
content and timing of decisions by the U.S. Food and Drug Administration, the
European Commission and other regulatory authorities; our ability to
successfully manufacture the product candidtate for the preclinical and
clinical trials; 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 2012 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.

Vimizim™ is our trademark, and BioMarin^®, Naglazyme^®, Kuvan^®, Firdapse^®
are registered trademarks of BioMarin Pharmaceutical Inc.

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

CONTACT: Investors:
         Traci McCarty
         BioMarin Pharmaceutical Inc.
         (415) 455-7558
        
         Media:
         Debra Charlesworth
         BioMarin Pharmaceutical Inc.
         (415) 455-7451

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