CAMBRIDGE, Mass., Sept. 9, 2016 /PRNewswire/ -- Blueprint
Medicines Corporation (NASDAQ: BPMC), a leader in discovering and
developing highly selective kinase medicines for patients with
genomically defined diseases, today disclosed a new drug discovery
program targeting protein kinase cAMP-activated catalytic subunit
alpha (PRKACA) fusions for the treatment of fibrolamellar carcinoma
(FLC). The announcement was made during an oral presentation on
September 8, 2016 at the
10th ILCA Annual Conference in Vancouver, Canada on Blueprint Medicines'
efforts to develop targeted therapies for patients with
hepatocellular carcinoma.
![Blueprint Medicines Blueprint Medicines](http://photos.prnewswire.com/prnvar/20150605/221118LOGO)
"Patients with liver cancers have historically suffered from a
lack of genomically defined therapeutic options. Our new drug
discovery program represents our commitment to patients with orphan
cancers," said Christoph Lengauer,
Chief Scientific Officer of Blueprint Medicines. "The addition of
this program is a testament to the strength of our scientists and
genomics platform, as well as to the quality of our unique library
of kinase inhibitors and continued momentum of our discovery
efforts."
FLC is a rare and distinct subtype of liver cancer that
typically arises in young adults. Currently, there are no approved
therapies, and surgery is the only available treatment option for
some patients, but most patients inevitably progress. Research
published in 2014 by the laboratory of Dr. Sanford Simon, Professor and Head of the
Laboratory of Cellular Biophysics at The
Rockefeller University in New
York in Science (Honeyman J.N. et al.,
Detection of a recurrent DNAJB1-PRKACA chimeric transcript in
fibrolamellar hepatocellular carcinoma) and Blueprint Medicines in
Nature Communications (Stransky N. et al., The
landscape of kinase fusions in cancer) identified PRKACA kinase
fusions in FLC. Blueprint Medicines estimates that more than ninety
percent of patients with FLC harbor the PRKACA fusion, which is the
only known recurrent genomic event in FLC and is considered to be
the driver gene of the disease. Given the high medical need and the
opportunity for a potentially transformative therapeutic impact,
Blueprint Medicines is developing drug candidates for the selective
inhibition of PRKACA.
"FLC is a devastating disease, and patients are in great need of
effective therapeutic options," said Dr. Sanford Simon. "Targeting PRKACA represents a
promising opportunity to develop innovative therapeutics that could
make a difference in these patients' lives."
The unveiling of the PRKACA program expands Blueprint Medicines'
existing pipeline, which includes Phase 1 clinical programs for its
drug candidates BLU-554, a selective inhibitor of FGFR4 for
patients with hepatocellular carcinoma, and BLU-285, a selective
inhibitor of both exon 17 mutant KIT and D842V mutant PDGFRα for
patients with advanced systemic mastocytosis and unresectable,
treatment-resistant gastrointestinal stromal tumors.
Blueprint Medicines expects to report preliminary data from the
dose escalation portion of each of these Phase 1 clinical trials by
the end of 2016. Blueprint Medicines' existing pipeline also
includes preclinical programs for its drug candidate BLU-667, a
selective inhibitor of RET activating mutations, fusions and
predicted resistant mutants found in non-small cell lung cancer and
thyroid cancer, a rare genetic disease program in collaboration
with Alexion Pharma Holding and cancer immunotherapy programs in
collaboration with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche
Inc.
About Blueprint Medicines
Blueprint Medicines is developing a new generation of highly
selective and potent kinase medicines to improve the lives of
patients with genomically defined diseases. The Company's approach
is rooted in a deep understanding of the genetic blueprint of
cancer and other diseases driven by the abnormal activation of
kinases. Blueprint Medicines is advancing three programs in
clinical development for subsets of patients with gastrointestinal
stromal tumors, hepatocellular carcinoma and systemic mastocytosis,
as well as multiple programs in research and preclinical
development. For more information, please visit
www.blueprintmedicines.com.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995, as amended, including, without limitation, statements
regarding the estimated number of patients with FLC that harbor the
PRKACA fusion; and the timing of clinical data or proof of concept
for preclinical and clinical programs, including, without
limitation, the timing and type of preliminary clinical data for
Blueprint Medicines' Phase 1 clinical trials for BLU-285 and
BLU-554; and Blueprint Medicines' strategy, business plans and
focus. The words "may," "will," "could," "would," "should,"
"expect," "plan," "anticipate," "intend," "believe," "estimate,"
"predict," "project," "potential," "continue," "target" and similar
expressions are intended to identify forward-looking statements,
although not all forward-looking statements contain these
identifying words. Any forward-looking statements in this press
release are based on management's current expectations and beliefs
and are subject to a number of risks, uncertainties and important
factors that may cause actual events or results to differ
materially from those expressed or implied by any forward-looking
statements contained in this press release, including, without
limitation, risks and uncertainties related to the delay of any
current or planned clinical trials or the development of Blueprint
Medicines' drug product candidates, including BLU-285 and BLU-554;
Blueprint Medicines' advancement of multiple early-stage efforts;
Blueprint Medicines' ability to successfully demonstrate the
efficacy and safety of its drug product candidates; the preclinical
and clinical results for Blueprint Medicines' drug product
candidates, which may not support further development of such drug
product candidates; and actions of regulatory agencies, which may
affect the initiation, timing and progress of clinical trials;
Blueprint Medicines' ability to develop and commercialize companion
diagnostics for its current and future drug candidates, including a
companion diagnostic for BLU-554 with Ventana Medical Systems, Inc.
and a companion diagnostic for BLU-285 with QIAGEN Manchester
Limited; and the success of Blueprint Medicines' rare genetic
disease collaboration with Alexion Pharma Holding and its cancer
immunotherapy collaboration with F. Hoffmann-La Roche Ltd and
Hoffmann-La Roche Inc. These and other risks and uncertainties are
described in greater detail in the section entitled "Risk Factors"
in Blueprint Medicines' Quarterly Report on Form 10-Q for the
quarter ended June 30, 2016, as filed
with the Securities and Exchange Commission (SEC) on August 9, 2016, and other filings that Blueprint
Medicines may make with the SEC in the future. Any forward-looking
statements contained in this press release represent Blueprint
Medicines' views only as of the date hereof and should not be
relied upon as representing its views as of any subsequent date.
Blueprint Medicines explicitly disclaims any obligation to update
any forward-looking statements.
Logo - http://photos.prnewswire.com/prnh/20150605/221118LOGO
To view the original version on PR Newswire,
visit:http://www.prnewswire.com/news-releases/blueprint-medicines-announces-new-drug-discovery-program-targeting-prkaca-kinase-fusions-for-the-treatment-of-fibrolamellar-carcinoma-at-10th-international-liver-cancer-association-ilca-annual-conference-300325390.html
SOURCE Blueprint Medicines Corporation