BioMarin Initiates Phase 3 Trial for PEG-PAL for the Treatment of PKU

BioMarin Initiates Phase 3 Trial for PEG-PAL for the Treatment of PKU

SAN RAFAEL, Calif., June 5, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) announced today that it has initiated the Phase 3 program
for PEG-PAL (PEGylated recombinant Phenylalanine Ammonia Lyase) for the
treatment of phenylketonuria (PKU).

"In the Phase 2 trial, PEG-PAL was shown to dramatically reduce blood Phe
levels, and we are hopeful that we will achieve the same outcome with the
Phase 3 program," stated Hank Fuchs, M.D., Chief Medical Officer of BioMarin.
"Adult patients with PKU andpatients on the severe end of the disease
spectrum still represent a very high unmet medical need. With PEG-PAL, it may
be possible to provide a treatment benefit to this population."

The Phase 3 study (165-301) is an open-label, multi-center study to assess the
safety and tolerability of an induction, titration and maintenance dose
regimen of PEG-PAL self-administered by approximately 90 naïve adult PKU
subjects. The primary objective of the 165-301 study is to characterize the
safety and tolerability of PEG-PAL during induction, titration, and
maintenance dosing. The secondary objective of the study is to evaluate blood
Phe levels during induction, titration, and maintenance dosing.

After completion of the open label 165-301 study, subjects are expected to
enroll in 165-302, a Phase 3 double-blind, placebo-controlled, randomized
discontinuation study to evaluate the efficacy and safety of PEG-PAL
self-administered by adults with PKU. The study will also enroll approximately
60 subjects from the Phase 2 program who are currently being treated with
PEG-PAL. The primary objective of the 165-302 study is to evaluate blood Phe
levels. The secondary objective of this study is to evaluate changes in
neuropsychiatric assessments as measured by the Inattentive portion of the
Attention Deficit and Hyperactivity Disorder Rating Scale (ADHD-RS) and the
Profile of Mood States (POMS). These will be administered at baseline, four
and eight weeks.

About PKU

PKU, a genetic disorder affecting approximately 50,000 diagnosed patients in
the developed world, is caused by a deficiency of the enzyme phenylalanine
hydroxylase. PAH is required for the metabolism of phenylalanine, an essential
amino acid found in most protein-containing foods. If the active enzyme is not
present in sufficient quantities, Phe accumulates to abnormally high levels in
the blood and becomes toxic to the brain, resulting in a variety of
complications including severe mental retardation and brain damage, mental
illness, seizures, tremors, and limited cognitive ability. As a result of
newborn screening efforts implemented in the 1960s and early 1970s, virtually
all PKU patients under the age of 40 in developed countries have been
diagnosed at birth. Currently, PKU can only be managed by a Phe-restricted
diet, which is supplemented by nutritional replacement products, like formulas
and specially-manufactured foods; however, the strict diet is difficult for
most patients to adhere to the extent needed for achieving adequate control of
blood Phe levels.To learn more about PKU, please visit www.PKU.com.
Information on this website is not incorporated by reference into this press
release.

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 Vimizim
(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 III 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.

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 related to the recently initiated Phase 3
trial for PEG-PAL, the expected future Phase 3 trial of PEG-PAL and the
PEG-PAL development program generally. 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 of PEG-PAL; our ability to successfully
manufacture PEG-PAL; the content and timing of decisions by the U.S. Food and
Drug Administration and other regulatory authorities concerning PEG-PAL; and
those risks that are discussed in BioMarin's filings with the Securities and
Exchange Commission, including, without limitation, BioMarin's 2012 Annual
Report on Form 10-K, and our periodic reports on Form 10-Q and Form 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-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:
         Eugenia Shen
         BioMarin Pharmaceutical Inc.
         (415) 506-6570
        
         Media:
         Debra Charlesworth
         BioMarin Pharmaceutical Inc.
         (415) 455-7451

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