CAMBRIDGE, Mass., Nov. 12, 2015 /PRNewswire/ -- Blueprint
Medicines (NASDAQ: BPMC) today announced that it will present new
preclinical data demonstrating the potential utility of BLU-285 in
a subset of patients with acute myeloid leukemia (AML)
characterized by a mutation in KIT Exon 17 during an oral
presentation at the 2015 American Society of Hematology (ASH)
Annual Meeting and Exposition being held December 5-8 in Orlando, Fla.
KIT Exon 17 mutations have been identified as drivers in
multiple diseases, and the presence of these mutations greatly
reduces the effective treatment options available to patients.
BLU-285 is a potent and highly selective inhibitor of KIT Exon 17
and PDGFRα D842V mutants, which are drivers of disease in patients
with gastrointestinal stromal tumors (GIST) and systemic
mastocytosis (SM).
Blueprint Medicines is enrolling patients in a dose-escalation,
Phase 1 clinical trial of BLU-285 for the treatment of
unresectable, treatment-resistant GIST. Additionally, the U.S. Food
and Drug Administration in September accepted Blueprint Medicines'
Investigational New Drug application to begin a Phase 1 clinical
trial of BLU-285 for the treatment of advanced SM, and Blueprint
Medicines is in the process of initiating clinical sites for this
trial.
In AML, a subset of patients with the t(8;21) translocation and
an additional KIT Exon 17 mutation are at increased risk of
recurrence compared to those without it, creating a strong need for
new therapies to address their form of the disease.
In preclinical results to be presented by Blueprint Medicines at
ASH, BLU-285 showed potent inhibition of KIT activity in an AML
cell line with the N822K mutation in Exon 17, including reduced
phosphorylation, downstream signaling and cellular proliferation.
BLU-285 administration in a xenograft model harboring AML cells
driven by the KIT Exon 17 mutation resulted in a reduction of
disease burden compared to cytarabine-treated animals. All
BLU-285-dosed mice showed tumor regression, and several showed
complete disease clearance.
"We are encouraged by these preclinical results, which further
strengthen our belief that BLU-285 may play an important role in
multiple hematologic and solid tumor malignancies," said
Andy Boral, M.D., Ph.D., Senior Vice
President of Clinical Development. "We continue to advance our
clinical programs for BLU-285 while also exploring new therapeutic
areas where targeting the KIT Exon 17 mutation may bring
substantial clinical benefit."
ASH presentation details:
Date &
Time:
|
Monday Dec. 7, 2015;
11:15 AM EST
|
Presentation
Title:
|
BLU-285, a Potent and
Selective Inhibitor for Hematologic Malignancies with KIT Exon 17
Mutations
|
Presenter:
|
Erica K. Evans, PhD,
Director, Biology, Blueprint Medicines
|
Abstract
Number:
|
568
|
Location:
|
Orange County
Convention Center, W 109
|
|
9800 International
Drive, Orlando, FL 32819
|
About the Phase 1 Clinical Trials for BLU-285
The Phase 1 clinical trial for BLU-285 for the treatment of
unresectable, treatment-resistant GIST will evaluate the safety and
tolerability of BLU-285 in multiple ascending doses with the goal
of establishing a maximum tolerated dose (MTD) or a recommended
dose if the MTD is not achieved. Blueprint Medicines expects to
enroll approximately 60 patients in this clinical trial at multiple
sites in the United States,
European Union and Asia. All
patients will be tested retrospectively for both KIT Exon 17 and
PDGFRα D842V mutational status. Once the MTD is reached, or a
recommended dose is established, Blueprint Medicines will open
expansion cohorts for genomically selected patients. Secondary
objectives include assessing response rate by Response Evaluation
Criteria In Solid Tumors (RECIST) criteria commonly used to measure
clinical responses in solid tumors, the pharmacokinetics of BLU-285
and allelic burden using circulating tumor DNA.
The planned Phase 1 clinical trial for BLU-285 for the treatment
of advanced SM will evaluate the safety and tolerability of BLU-285
in multiple ascending doses with the goal of establishing an MTD or
a recommended dose if the MTD is not achieved. Blueprint Medicines
expects to enroll approximately 60 patients in this clinical trial
at multiple sites in the United
States and European Union. All patients will be tested
retrospectively for KIT D816V mutational status. Once the MTD is
reached, or a recommended dose is established, Blueprint Medicines
will open expansion cohorts for specific subtypes of SM. Secondary
objectives include assessment of the pharmacokinetic profile of
BLU-285, assessment of response rate by the International Working
Group Myeloproliferative Neoplasms Research and Treatment criteria,
changes in KIT D816V mutant allele fractions in bone marrow and
circulating tumor DNA, and changes in patient reported
outcomes.
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 potential for BLU-285 to provide clinical benefit to
patients and Blueprint Medicines' preclinical and clinical plans or
programs. 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,
including its RET program; 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 Report on
Form 10-Q for the quarter ended September
30, 2015, as filed with the Securities and Exchange
Commission (SEC) on November 9, 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.
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