CAMBRIDGE, Mass., Sept. 10, 2015 /PRNewswire/ -- Blueprint
Medicines (NASDAQ: BPMC) today announced that the U.S. Food and
Drug Administration (FDA) accepted the Company's Investigational
New Drug (IND) application to begin a Phase 1 clinical trial of
BLU-285 in patients with advanced systemic mastocytosis (SM), a
disorder of the mast cells.
"BLU-285 has the potential to be a highly targeted therapy for
patients with severe forms of SM who lack effective treatment
options," said Jeffrey Albers, Chief
Executive Officer of Blueprint Medicines. "With the achievement of
this milestone, we are simultaneously advancing multiple drug
candidates into clinical trials for genomically defined subsets of
patients in dire need of new therapies. We remain singularly
focused on using our deep understanding of the genetic blueprint of
cancer and other diseases driven by the abnormal activation of
kinases to craft highly selective medicines aimed at improving
patients' lives."
BLU-285 is a potent and highly selective inhibitor of the KIT
D816V mutant, the primary driver of disease in more than 94 percent
of SM patients. SM is characterized by the abnormal accumulation of
mast cells. In patients with advanced forms of the disease, mast
cells accumulate in organs, such as the bone marrow, intestines,
spleen and liver, compromising organ function. There are no
approved targeted therapies for SM patients with KIT D816V-driven
disease.
In this Phase 1 trial, Blueprint Medicines plans to enroll
approximately 60 patients with advanced SM, which includes
aggressive SM with associated hematological non-mast cell disorders
(SM-AHNMD) and mast cell leukemia (MCL), and other relapsed or
refractory myeloid malignancies at multiple sites in Europe and the
United States. The trial will test the safety and
tolerability of escalating doses of BLU-285, with the goal of
establishing a maximum tolerated dose (MTD), or a recommended dose
if the MTD is not achieved. Additional study objectives include
assessing early signs of biological activity using disease-specific
biomarkers and clinical efficacy as measured by response rate and
patient-reported outcomes.
In July, Blueprint Medicines also announced the acceptance of
INDs to advance BLU-285 into a Phase 1 trial for unresectable,
treatment-resistant gastrointestinal stromal tumor (GIST) and
BLU-554 into a Phase 1 trial for advanced hepatocellular carcinoma
(HCC).
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
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' ability to craft highly selective
therapies and deliver targeted medicines for patients with
genomically defined diseases and its planned Phase 1 clinical
trials for BLU-554 and BLU-285. 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 planned clinical trials
and/or development of Blueprint Medicines' drug product candidates,
including BLU-554 and BLU-285; Blueprint Medicines' ability to
successfully demonstrate the efficacy and safety of its drug
product candidates; the pre-clinical 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 trial. 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.
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SOURCE Blueprint Medicines