BioMarin Provides Preliminary Data on Ongoing Phase 1/2 Trial for BMN 673 for the Treatment of Solid Tumors at 2013 American

BioMarin Provides Preliminary Data on Ongoing Phase 1/2 Trial for BMN 673 for
the Treatment of Solid Tumors at 2013 American Society of Clinical Oncology
Annual Meeting

Phase 3 Trial in Metastatic gBRCA Breast Cancer Planned to Start in Q4 2013

SAN RAFAEL, Calif., June 3, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) today announced an update on the ongoing Phase 1/2 study
for its poly ADP-ribose polymerase (PARP) inhibitor BMN 673 for the treatment
of solid tumors. BMN 673 has exhibited substantial single-agent anti-tumor
activity in deleterious germline BRCA ovarian and breast cancers in the trial.
The data were presented during a poster presentation at the 2013 American
Society of Clinical Oncology Annual Meeting.

In the 28 gBRCA ovarian cancer patients, the RECIST response rate was 44% or
11 of 25 evaluable patients, the CA-125 response rate was 70% or 19 of 27
evaluable patients and the clinical benefit response rate was 82% or 23 of 28
In the 18 gBRCA breast cancer patients, the RECIST response rate was 39% or 7
of 18 patients and the clinical benefit rate was 67% or 12 of 18 patients. Of
the 18 gBRCA breast cancer patients, there were six partial responses (three
yet to be confirmed) and one complete response.Four ongoing patients have had
stable disease for less than 12 weeks.Treatment is ongoing in 12 of the 18
breast cancer patients in the study.

"Patients with germline BRCA-associated tumors have no targeted treatment
options.There is a need for therapies that target specific molecular defects
in tumors, and PARP inhibitors offer that potential in BRCA-related cancers.
We have seen excellent anti-tumor activity in some of our patients treated
with BMN 673. Continued study of this molecule will be meaningful for
advancing the care for patients suffering from these cancers," said Professor
Johann de Bono, Professor of Experimental Cancer Medicine at The Institute of
Cancer Research, London, and Honorary Consultant in Medical Oncology at The
Royal Marsden NHS Foundation Trust.

BMN 673 was generally well-tolerated. The dose-limiting toxicity was Grade 3
thrombocytopenia. Myelosuppression, most of which was moderate in severity,
occurred in 10-20% of patients with chronic dosing.Fatigue, nausea and
alopecia were observed in 20-30% of patients.Signs of activity were seen as
low as 100 µg/day, and the maximum tolerated dose was 1.0 mg/day, which is the
expected dose for further development.BMN 673 also has good bioavailability
and a long half-life which supports once daily dosing.

"We are encouraged by this early stage data on BMN 673, including the safety
profile, and substantial anti-tumor activity.We look forward to initiating a
Phase 3 trial in metastatic gBRCA breast cancer patients to pursue a safe and
effective therapy in a once-daily, oral dose that meets an unmet medical need
in this aggressive form of cancer," said Hank Fuchs, M.D., Chief Medical
Officer of BioMarin.

More complete data in breast and ovarian cancers, as well as Ewings sarcoma
and small cell lung cancer (SCLC) will be presented at a future medical

Based on this preliminary data, BioMarin expects to start a Phase 3 trial in
gBRCA breast cancer in the fourth quarter of 2013.

Study Objectives and Design

The Phase 1/2 trial is an open-label study of once daily, orally administered
BMN 673 in approximately 70 patients ages 18 and older with advanced or
recurrent solid tumors.The primary objective of the study is to establish the
maximum tolerated dose (MTD) of daily oral BMN 673.The secondary objectives
are to assess the safety, tolerability, preliminary efficacy and
pharmacodynamic activity of BMN 673, and to determine the pharmacokinetic
profile.The study design was a standard 3 + 3 dose escalation followed by
expansion at MTD in cohorts with selected tumor types to further characterize
safety and anti-tumor activity.

To access the poster presented at ASCO

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 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 of the development of BMN 673, including
the timing of the clinical trials of the candidate, and the possible safety
and efficacy of such 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 content and timing of decisions by the U.S. Food
and Drug Administration, the European Commission and other regulatory
authorities, results and timing of current and planned clinical and
preclinical studies related to such product; our ability to successfully
manufacture the product; 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:
         Eugenia Shen
         BioMarin Pharmaceutical Inc.
         (415) 506-6570
         Debra Charlesworth
         BioMarin Pharmaceutical Inc.
         (415) 455-7451

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