Five Data Presentations on BioMarin's BMN 673 PARP Inhibitor at the 2013
AACR-NCI-EORTC International Conference on Molecular Targets and Cancer
BMN 673 Potentially 100-Fold More Potent Than Other PARP Inhibitors
SAN RAFAEL, Calif., Oct. 20, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) today announced that five poster presentations on BMN 673
will be featured at the 2013 AACR-NCI-EORTC International Conference on
Molecular Targets and Cancer Therapeutics in Boston from October 19-23, 2013.
The five pre-clinical and clinical data abstracts include:
*Stereospecific Trapping of PARP-DNA Complexes by BMN 673 and Comparison
with Olaparib and Rucarparib (Abstract A257) to be presented on Sunday,
October 20, 2013 from 12:30 – 3:00 PM.
*Inhibition of PBMC PARP activity with the novel PARP 1/2 inhibitor BMN 673
in patients with advanced solid tumors (Abstract A220) to be presented on
Sunday, October 20, 2013 from 12:30 – 3:00 PM.
*Preclinical evaluation of BMN 673 in combination with temozolomide (TMZ)
in various tumor types including small cell lung cancer (SCLC) cells
(Abstract B93) to be presented on Monday, October 21, 2013 from 12:30 PM -
*Pediatric Preclinical Testing Program (PPTP) evaluation of BMN 673, an
inhibitor of Poly-ADP Ribose Polymerase (PARP), alone and with
Temozolomide (TMZ) (Abstract C206) to be presented Tuesday, October 22,
2013 from 12:30 PM - 3:00 PM
*Update on first-in-human trial of novel oral PARP inhibitor BMN 673 in
patients with solid tumors (Abstract C295) to be presented on Tuesday,
October 22 from 12:30-3:00.
Junko Murai and Yves Pommier from the National Cancer Institute, will present
Stereospecific trapping of PARP-DNA complexes by BMN 673 and comparison with
olaparib and rucaparib.The research as reported on the poster concluded that
BMN 673 was approximately 100-fold more potent than olaparib and rucaparib at
trapping PARP, making it the most potent clinical PARP inhibitor to date with
the highest efficiency at trapping PARP-DNA complexes.
PARP-DNA trapping helps explain why PARP inhibitors show different potency in
killing tumor cells, even when they are similar in inhibiting enzymatic
activity in PARP.While reducing the level of PARP enzymatic activity seems to
be important, trapping PARP onto DNA is lethal to the cancer cell if not
repaired.Tumor cells defective in DNA repair function are more sensitive to
PARP inhibitors than normal cells with full DNA repair capability.The
research concluded that PARP inhibitors should be evaluated on both catalytic
PARP inhibition and PARP-DNA trapping to fully understand the impact.
"We believe PARP trapping seen with BMN 673 is differentiating because BMN 673
appears to be significantly more lethal to cancer cells than olaparib and
rucaparib, and PARP trapping may explain why BMN 673 shows far greater
potency," said Hank Fuchs, M.D., Chief Medical Officer at BioMarin. "We're
looking forward to further studies in gBRCA metastatic breast cancer patients
to confirm and extend the early clinical results of the compound."
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, BMN-111, a modified
C-natriuretic peptide, which is currently in Phase I 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.
This press release contains forward-looking statements about the business
prospects of BioMarin Pharmaceutical Inc., including, without limitation,
statements about: the data presented at the conference, the expectations of
the development of BMN 673, including the possible safety and efficacy of such
candidate, and the observed and expected potency and anti-tumor activity 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: 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.
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