CAMBRIDGE, Mass., March 20, 2017 /PRNewswire/ -- Blueprint
Medicines Corporation (NASDAQ: BPMC), a leader in discovering
and developing targeted kinase medicines for patients with
genomically defined diseases, today announced that it has dosed the
first patient in a Phase 1 clinical trial of BLU-667, an
investigational RET inhibitor for patients with non-small cell lung
cancer (NSCLC), medullary thyroid cancer (MTC) and other advanced
solid tumors that harbor a RET alteration.
"By rapidly initiating a clinical trial for our potent and
selective RET inhibitor BLU-667, we have successfully achieved a
key 2017 milestone," said Jeff
Albers, Chief Executive Officer of Blueprint Medicines.
"This continued progress underscores our commitment to build
Blueprint Medicines into a sustainable company. BLU-667 has a
unique product profile differentiated from our other clinical-stage
programs by its potential to address predicted resistance mutations
that may arise. With BLU-667 enrollment underway, we are further
increasing our diversified pipeline of potent, highly selective
investigational medicines that target specific disease drivers in
genomically-defined patient populations."
"RET fusions and mutations are recognized as important drivers
in multiple cancers, but existing multi-kinase inhibitors with RET
activity do not provide sufficient, durable benefit for patients
with RET alterations," said Andy
Boral, M.D., Ph.D., Chief Medical Officer of Blueprint
Medicines. "BLU-667 is being developed to potently inhibit RET, and
simultaneously prevent the development of on-target resistance,
which we believe will provide more lasting clinical benefit and
prevent or delay disease recurrence. We look forward to working
with patients and physicians to explore BLU-667's potential."
BLU-667 is an orally available, potent and selective inhibitor
designed to target RET mutations, fusions and predicted resistance
mutants. RET activating fusions and mutations are a key disease
driver in multiple cancers, including NSCLC and MTC, with RET
fusions implicated in approximately 1-2% of patients with NSCLC,
and RET mutations implicated in approximately 60% of patients with
MTC. In addition, genomic analyses published by scientists at
Blueprint Medicines have identified RET fusions at low frequencies
in colon and breast cancer. Currently, there are no approved
therapies that selectively target RET-driven cancers, though there
are several approved multi-kinase inhibitors with RET activity
being evaluated in clinical trials. Thus far, clinical
activity attributable to RET inhibition has been uncertain for
these inhibitors, likely due to insufficient inhibition of RET and
off-target toxicities. In preclinical studies using biochemical and
cellular assays, and tumor models, BLU-667 was active against RET
fusions and mutations, including predicted resistance
mutations.
About the Phase 1 Clinical Trial for BLU-667 in RET
Blueprint Medicines' Phase 1 clinical trial of BLU-667 in RET is
designed to evaluate the safety and tolerability of BLU-667 in
multiple ascending doses in patients with NSCLC, MTC, and other
advanced solid tumors with the goal of establishing a maximum
tolerated dose (MTD) or a lower recommended dose if appropriate.
Once the MTD is reached, or a recommended dose is established,
Blueprint Medicines plans to open expansion cohorts for NSCLC
patients with a RET rearrangement, patients with MTC, and patients
with RET-altered solid tumors other than NSCLC and MTC. Secondary
objectives for this Phase 1 clinical trial include assessing the
pharmacokinetic profile of BLU-667, assessing RET gene status in
plasma and tumor tissue, characterizing the pharmacodynamic effects
of BLU-667 and assessing response rate by Response Evaluation
Criteria In Solid Tumors (RECIST) version 1.1, which are criteria
commonly used to measure clinical responses in solid tumors. The
Phase 1 clinical trial is designed to enroll approximately 115
patients, including approximately 35 patients during dose
escalation and approximately 80 additional patients in expansion
cohorts, at multiple sites in the United
States and the European Union. Please refer to
www.clinicaltrials.gov (NCT03037385) for additional details related
to this Phase 1 clinical trial. For more information, contact the
study director for this Phase 1 clinical trial at
studydirector@blueprintmedicines.com.
About Blueprint Medicines
Blueprint Medicines is developing a new generation of
targeted and potent kinase medicines to improve the lives of
patients with genomically defined diseases. Its 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 four programs
in clinical development for subsets of patients with
gastrointestinal stromal tumors, hepatocellular carcinoma, systemic
mastocytosis, non-small cell lung cancer, medullary thyroid cancer
and other advanced solid tumors, as well as multiple programs in
research and preclinical development.
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 plans and timelines for the clinical development of
BLU-667; Blueprint Medicines' ability to implement its clinical
development plans for BLU-667 for the treatment of NSCLC, MTC and
other advance solid tumors; Blueprint Medicines' ability to enroll
patients in its Phase 1 clinical trial for BLU-667; 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, uncertainties related
to market conditions. These and other risks and uncertainties are
described in greater detail in the section entitled "Risk Factors"
in Blueprint Medicines' Annual Report on Form 10-K for the year
ended December 31, 2016, as filed
with the Securities and Exchange Commission (SEC) on March 9, 2017, 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.
To view the original version on PR Newswire,
visit:http://www.prnewswire.com/news-releases/blueprint-medicines-announces-enrollment-of-first-patient-in-phase-1-clinical-trial-for-blu-667-300426336.html
SOURCE Blueprint Medicines Corporation