CAMBRIDGE, Mass., May 11, 2015 /PRNewswire/ -- Blueprint Medicines
(NASDAQ: BPMC) today announced that the Journal of Clinical
Investigation (JCI) published an overview of the Company's
kinase drug discovery and development strategy. The publication
underscores Blueprint Medicines' focus on identifying novel
genomically defined disease drivers and the use of novel chemistry
from its proprietary compound library to craft highly selective
kinase inhibitors for new and difficult-to-drug kinase targets.
"Targeting cancer with kinase inhibitors," authored by Blueprint
Medicines' scientists, was published in the May 2015 issue of JCI, a leading journal focused
on science and clinical research to advance medicine.
"Despite the success of kinase inhibitors in treating cancer,
approved drugs focus on less than 5 percent of all kinases and the
function of most kinases remains unknown," said Sir Philip Cohen, PhD, Professor of Enzymology,
Medical Research Council Protein Phosphorylation and Ubiquitylation
Unit, University of Dundee, Scotland.
"There is a tremendous need and opportunity to further our
understanding of kinase biology, identify novel disease drivers and
design highly targeted therapies. Blueprint Medicines' approach of
using novel chemistry to target newly discovered genomic drivers of
cancer as well as known drivers that have been historically
difficult to drug has the potential to greatly improve the lives of
patients who currently have no or limited treatment options."
The JCI review article describes how the abnormal activation of
kinases drives many hallmarks of tumor biology. Kinases are proven
cancer drug targets, but progress in recent years has been largely
incremental. In the article, Blueprint Medicines scientists call
attention to important opportunities for advancing the field that
serve as the foundation for the Company's drug discovery and
development strategy:
- Uncovering novel disease drivers and drug targets: New
genomics and sequencing approaches are needed to elucidate the
biology of the full kinome, which is comprised of 518 kinases, and
to uncover the function of kinases of unknown biology (or KUBs) and
their potential for drug discovery.
- Crafting highly selective kinase inhibitors: Novel
chemical matter and structure-informed design are needed to create
more selective and potent therapies that can inhibit new and
difficult-to-drug kinase targets. Complete target inhibition with
minimal off-target effects is also needed to provide more durable
responses with few toxicities, improving outcomes for
patients.
- Predicting resistance mutations: Novel approaches in
structural biology and computational chemistry are needed to
predict future resistance mutations which arise from targeted
therapies. Novel chemistry is needed to design a single therapy
that can target both the original primary disease driver and the
subsequent mutations that cause patients to become refractory to
treatment.
- Developing novel combination therapies: Combinations of
highly targeted kinase inhibitors acting within a single kinase
pathway or between parallel kinase pathways are needed to mount a
multi-pronged attack that improves efficacy and delays the onset of
treatment resistance.
"We're entering a new era of precision medicine, in which we can
craft highly selective kinase inhibitors against previously
unaddressed drivers of disease and identify patients who are most
likely to respond based on the molecular profile of their cancers,"
said Christoph Lengauer, PhD, MBA,
Blueprint Medicines Chief Scientific Officer. "The strategies we
describe in the JCI paper are at the heart of our drug discovery
and development efforts. We believe that our ability to identify
new drug targets, coupled with our proprietary library of
compounds, will enable us to uniquely fulfill our mission of
delivering targeted medicines for patients with genomically defined
diseases."
Blueprint Medicines expects to advance its lead programs into
Phase 1 clinical trials in mid-2015: BLU-554, a selective inhibitor
of fibroblast growth factor receptor 4 (FGFR4), in hepatocellular
carcinoma, and BLU-285, a selective inhibitor of PDGFRα D842V and
KIT Exon 17 mutants, in gastrointestinal stromal tumors and
systemic mastocytosis. The company also has a drug discovery
program targeting RET kinase fusions and predicted resistance
mutations.
About Blueprint Medicines
Blueprint Medicines
makes kinase drugs to treat patients with genomically defined
diseases. Led by a team of industry innovators, Blueprint Medicines
integrates a novel target discovery engine and a proprietary
compound library to understand the blueprint of cancer and craft
highly selective therapies. This empowers the Blueprint Medicines
team to develop patient-defined medicines aimed at eradicating
cancer.
Forward-Looking Statements
Various statements in this release concerning Blueprint
Medicines' future expectations, plans and prospects, including
without limitation, statements regarding the foundation for
Blueprint Medicines' drug discovery and development strategy,
expectations regarding Blueprint Medicines' ability to craft highly
selective kinase inhibitors and deliver targeted medicines for
patients with genomically defined cancers, Blueprint Medicines'
expectations regarding BLU-554 as a treatment for hepatocellular
carcinoma, Blueprint Medicines' expectations regarding BLU-285 as a
treatment for gastrointestinal stromal tumors and systemic
mastocytosis, statements concerning the timing of planned
Phase 1 clinical trials for BLU-554 and BLU-285, constitute
forward-looking statements for the purposes of the safe harbor
provisions under The Private Securities Litigation Reform Act of
1995. In particular, it should be noted that the strategies
described in the JCI paper are preliminary and interim in nature;
the Phase 1 clinical trials for BLU-554 and BLU-285 have not yet
commenced. Any forward-looking statements in this press release are
based on management's current expectations of future events and are
subject to a number of risks and uncertainties. Actual
results may differ materially from those indicated by these
forward-looking statements as a result of various important
factors, including, without limitation, the risk of delay of any
planned clinical trials and/or development of Blueprint Medicines'
drug product candidates, Blueprint Medicines' ability to
successfully demonstrate the efficacy and safety of its drug
product candidates, the pre-clinical and clinical results for its
drug product candidates, which may not support further development
of such drug product candidates, the risk that Blueprint Medicines'
collaboration with Alexion Pharma Holdings will not continue or
will not be successful, actions of regulatory agencies, which may
affect the initiation, timing and progress of clinical trials,
obtaining, maintaining and protecting intellectual property,
Blueprint Medicines' ability to enforce its patents against
infringers and defend its patent portfolio against challenges from
third parties, competition from others developing products for
similar uses, Blueprint Medicines' ability to manage operating
expenses, Blueprint Medicines' ability to obtain additional funding
to support its business activities and establish and maintain
strategic business alliances and new business initiatives,
Blueprint Medicines' dependence on third parties for various
functions, the outcome of litigation, and unexpected expenditures,
as well as those risks more fully discussed in the section entitled
"Risk Factors" in the final prospectus related to Blueprint
Medicines' initial public offering filed with the Securities and
Exchange Commission pursuant to Rule 424(b) of the Securities Act,
as well as discussions of potential risks, uncertainties, and other
important factors in Blueprint Medicines' subsequent filings with
the Securities and Exchange Commission. In addition, any
forward-looking statements represent Blueprint Medicines' views
only as of today 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.
CONTACT:
Investor Relations:
Beth DelGiacco
Stern Investor Relations, Inc.
212-362-1200
beth@sternir.com
Media Relations:
Beth Keshishian
inVentiv Health PR
212-229-8417
beth.keshishian@inventivhealth.com
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SOURCE Blueprint Medicines