CAMBRIDGE,
Mass., Nov. 9, 2015
/PRNewswire/ -- Blueprint Medicines (NASDAQ: BPMC), a leader
in discovering and developing highly selective kinase medicines for
patients with genomically defined diseases, today provided an
update on certain of its preclinical discovery efforts in oncology
and rare genetic diseases, in addition to reporting its financial
results for the third quarter ended September 30, 2015.
"We are excited about the ways in which we continue to
leverage our novel library of kinase inhibitors to advance our drug
discovery pipeline and target kinases as drivers of disease," said
Christoph Lengauer, Ph.D., Chief
Scientific Officer of Blueprint Medicines. "During the third
quarter of 2015, we presented preclinical data validating the
advancement of BLU-554 into clinical development and identifying
PRKACA as a novel target for therapeutic intervention. In addition,
we have predicted resistance mutations in NTRK, some of which have
recently been clinically observed by others, and we are advancing
compounds that inhibit NTRK and resistant mutants into preclinical
safety studies. We have also entered into a collaboration with the
Children's Hospital of Eastern
Ontario seeking to identify new therapeutic opportunities
for rare genetic diseases."
Oncology—Preclinical Discovery
Highlights
At the 9th Annual Conference of the
International Liver Cancer Association (ILCA) in September,
Blueprint Medicines presented new preclinical data on BLU-554, one
of Blueprint Medicines' lead drug candidates, and DNAJB1-PRKACA, a
recurrent fusion in certain hepatocellular carcinoma (HCC)
patients.
- The new preclinical data presented by Blueprint Medicines
at ILCA further validate the advancement of BLU-554 into clinical
development. BLU-554 is an exquisitely selective inhibitor of
fibroblast growth factor receptor 4 (FGFR4). The new data show that
BLU-554 demonstrated anti-tumor activity in preclinical models of
HCC with aberrant FGFR4 signaling. Blueprint Medicines is currently
enrolling patients in a dose-escalation, Phase 1 clinical trial of
BLU-554 for the treatment of advanced HCC and
cholangiocarcinoma.
- In addition, Blueprint Medicines continues to pursue
research on a novel drug target for fibrolamellar hepatocellular
carcinoma (FL-HCC). FL-HCC is a rare subtype of liver cancer that
arises in adolescents and young adults. Preclinical data presented
by Blueprint Medicines at ILCA indicate that more than 90 percent
of patients with FL-HCC have the DNAJB1-PRKACA fusion, which is
likely a driver of FL-HCC. The new data highlight the role of
PRKACA in FL-HCC and its potential as a novel target for
therapeutic intervention.
In addition, utilizing its proprietary platform, Blueprint
Medicines has predicted resistance mutations in the neurotrophic
tyrosine receptor kinase (NTRK). Recently, some of these
mutations were clinically observed by others in tumors that
developed resistance after initially responding to an NTRK
inhibitor.[1] Blueprint Medicines is advancing compounds
that inhibit NTRK and resistant mutants into preclinical safety
studies. Research published by Blueprint Medicines scientists in
the September 10, 2014 edition of
Nature Communications (Stransky N. et al., The
landscape of kinase fusions in cancer) suggests NTRK fusion
events may drive tumorigenesis in multiple cancers, with a total of
23 NTRK1, NTRK2 and NTRK3 fusions identified across nine tumor
types.
Rare Genetic Diseases—Preclinical Discovery
Highlights
Continuing its focus on rare genetic diseases, Blueprint
Medicines is currently collaborating with the Children's Hospital
of Eastern Ontario (CHEO).
Blueprint Medicines and CHEO are seeking to identify potential
therapeutic opportunities for rare genetic diseases utilizing
Blueprint Medicines' proprietary compound library, which will allow
Blueprint Medicines to initiate development of future programs in
rare genetic diseases.
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 Blueprint Medicines' discovery and development plans or
programs, including without limitation for NTRK, FL-HCC and rare
genetic diseases. 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' 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.
These and other risks and uncertainties are described in greater
detail in the section entitled "Risk Factors" in Blueprint
Medicines' Quarterly Reports on Form 10-Q for the quarters ended
March 31, 2015 and June 30, 2015, as filed with the Securities and
Exchange Commission (SEC) on June 11,
2015 and August 10, 2015, 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.
[1] Russo M, Misale S, Wei G et al.
Acquired resistance to the TRK inhibitor entrectinib in colorectal
cancer [published online November 6,
2015]. Cancer Discov. 2015. doi: 10.1158/2159-8290.
http://cancerdiscovery.aacrjournals.org/. Accessed November 8, 2015.
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SOURCE Blueprint Medicines