CAMBRIDGE, Mass., Jan. 11, 2021 /PRNewswire/ -- Blueprint Medicines
Corporation (NASDAQ: BPMC) today provided an update on key
portfolio milestones and outlined a strategic roadmap to become the
world's leading precision therapy company.
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"For the first time, we enter a new year as a fully integrated,
global biopharmaceutical company, with four regulatory approvals in
the United States and Europe in 2020, a pipeline of eight wholly
owned or partnered precision therapies, and the strongest financial
position since our inception," said Jeff
Albers, Chief Executive Officer of Blueprint Medicines.
"With this solid foundation, we are now scaling our ambition and
aim to make real the promise of precision medicine to improve and
extend life for as many people with cancer and hematologic
disorders as possible. We will do this by bringing our medicines to
more patients globally, rapidly advancing a wave of new therapeutic
candidates to clinical proof-of-concept, and further expanding our
platform-enabled research pipeline."
In addition, Blueprint Medicines today announced the achievement
of several portfolio milestones:
- AYVAKIT received breakthrough therapy designation from the U.S.
Food and Drug Administration (FDA) for the treatment of moderate to
severe indolent systemic mastocytosis (SM), which encompasses the
majority of patients with SM, highlighting the medical need in this
population as well as the clinical potential of AYVAKIT to
demonstrate substantial improvement over the current standard of
care.
- Positive top-line results from a Phase 1 trial in healthy
volunteers showed BLU-263 was well-tolerated across a range of
single- and multiple-ascending doses predicted to potently inhibit
D816V mutant KIT, the underlying SM disease driver. These data
support development of BLU-263 as a potential treatment for
patients with SM and other mast cell disorders.
- The company nominated a selective, brain-penetrant development
candidate for treatment-resistant double-mutant EGFR-driven
non-small cell lung cancer (NSCLC), with the potential to be
first-in-class, showing potent activity against the activating
L858R or exon 19 deletion mutations and the acquired C797S
mutation, the most common on-target resistance mutation to
osimertinib.
- The company nominated a development candidate targeting MAP4K1,
a kinase believed to play a role in T-cell regulation, with the
potential to be best-in-class. The program was developed under the
company's cancer immunotherapy collaboration with Roche. In
addition, Blueprint Medicines and Roche have amended their
agreement to focus on MAP4K1 and one additional undisclosed target,
collectively identified as the most promising targets of the
collaboration to date.
Entering 2021, the company's key strategies and goals
include:
1. Accelerate global adoption of AYVAKIT and GAVRETO™
(pralsetinib)
AYVAKIT, a selective KIT and PDGFRA inhibitor, is approved in
the U.S. and Europe for the
treatment of patients with unresectable or metastatic
gastrointestinal stromal tumor driven by certain PDGFRA
mutations.
- Obtain FDA approval and launch AYVAKIT in advanced SM in the
U.S. in the second half of 2021.
- Submit a Type II variation marketing authorization application
(MAA) to the European Medicines Agency (EMA) for AYVAKYT®
(avapritinib) for advanced SM in the first quarter of 2021.
- Present registrational data from the PATHFINDER trial of
AYVAKIT in advanced SM in the first half of 2021.
- Complete enrollment of the registration-enabling Part 2 of the
PIONEER trial of AYVAKIT in non-advanced SM in mid-2021.
GAVRETO, a selective RET inhibitor, is approved in the U.S.
for the treatment of patients with certain advanced or metastatic
RET fusion-positive NSCLC, RET-mutant medullary thyroid cancer
(MTC) and RET fusion-positive thyroid cancer. Under a global
collaboration, Blueprint Medicines and Roche are developing and
commercializing GAVRETO for the treatment of RET-altered
cancers.
- Obtain regulatory approval from the European Commission and
launch GAVRETO in RET fusion-positive NSCLC in Europe in the first half of 2021.
- Submit a Type II variation MAA to the EMA for GAVRETO for
RET-altered thyroid cancers in the second half of 2021.
- Initiate a GAVRETO cohort in Roche's TAPISTRY tumor-agnostic
platform trial in the second half of 2021.
- Submit marketing applications for GAVRETO for RET-altered NSCLC
and thyroid cancers across multiple additional global geographies
in 2021.
2. Advance a new wave of innovative therapeutic candidates
into clinical development, with plans to achieve rapid
proof-of-concept and regulatory approval.
BLU-263, a
next-generation selective KIT inhibitor
- Initiate the Phase 2 HARBOR trial of BLU-263 in patients with
non-advanced SM in mid-2021.
Development candidates for treatment-resistant EGFR-driven
NSCLC
- Initiate a Phase 1 trial of BLU-945, a triple-mutant EGFR
inhibitor, in patients with treatment-resistant EGFR-driven NSCLC
in the first half of 2021.
- Initiate a Phase 1 trial of the company's double-mutant EGFR
inhibitor in patients with treatment-resistant EGFR-driven NSCLC by
the end of 2021.
- Present foundational preclinical data for the company's
double-mutant EGFR inhibitor in the first half of 2021.
- Present preclinical data supporting combination of the
company's wholly owned double- and triple-mutant EGFR inhibitors in
treatment-naïve EGFR-driven NSCLC in the second half of 2021.
Development candidate targeting MAP4K1, under the cancer
immunotherapy collaboration with Roche
- Present foundational preclinical data in the first half of
2021.
3. Further expand the company's precision medicine pipeline
with a focus on delivering transformational benefit to patients
with cancer and hematologic disorders.
- Expand pipeline with one or more development candidates in
2021.
- Pursue external opportunities to complement the company's
precision medicine pipeline.
Financial Guidance
Based on its current operating plans, Blueprint Medicines
continues to anticipate its existing cash, cash equivalents and
investments, together with anticipated future product revenues,
will provide sufficient capital to enable the company to achieve a
self-sustainable financial profile.
About Blueprint Medicines
Blueprint Medicines is a global precision therapy company that
invents life-changing therapies for people with cancer and
hematologic disorders. Applying an approach that is both precise
and agile, we create medicines that selectively target genetic
drivers, with the goal of staying one step ahead across stages of
disease. Since 2011, we have leveraged our research platform,
including expertise in molecular targeting and world-class drug
design capabilities, to rapidly and reproducibly translate science
into a broad pipeline of precision therapies. Today, we are
delivering approved medicines directly to patients in the United States and Europe, and we are globally advancing multiple
programs for genomically defined cancers, systemic mastocytosis,
and cancer immunotherapy. For more information, visit
www.BlueprintMedicines.com and follow us on Twitter
(@BlueprintMeds) and LinkedIn.
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' 2021 goals and anticipated
milestones; plans, strategies, timelines and expectations for
Blueprint Medicines' current or future approved drugs and drug
candidates, including timelines for marketing applications and
approvals, the initiation of clinical trials or the results of
ongoing and planned clinical trials; the potential benefits of any
of Blueprint Medicines' current or future approved drugs or drug
candidates in treating patients; and Blueprint
Medicines' strategy, goals and anticipated milestones,
business plans and focus. The words "aim," "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 impact of the COVID-19 pandemic
to Blueprint Medicines' business, operations, strategy,
goals and anticipated milestones, including Blueprint
Medicines' ongoing and planned research and discovery
activities, ability to conduct ongoing and planned clinical trials,
clinical supply of current or future drug candidates, commercial
supply of current or future approved products, and launching,
marketing and selling current or future approved
products; Blueprint Medicines' ability and plans in
establishing a commercial infrastructure, and successfully
launching, marketing and selling current or future approved
products, including AYVAKIT and GAVRETO; Blueprint
Medicines' ability to successfully expand the approved
indications for AYVAKIT and GAVRETO or obtain marketing approval
for AYVAKIT and GAVRETO in additional geographies in the future;
the delay of any current or planned clinical trials or the
development of Blueprint Medicines' current or future
drug candidates; Blueprint Medicines' advancement of
multiple early-stage efforts; Blueprint
Medicines' ability to successfully demonstrate the safety and
efficacy of its drug candidates and gain approval of its drug
candidates on a timely basis, if at all; the preclinical and
clinical results for Blueprint Medicines' drug
candidates, which may not support further development of such drug
candidates; actions of regulatory agencies, which may affect the
initiation, timing and progress of clinical trials; Blueprint
Medicines' ability to develop and commercialize companion
diagnostic tests for its current and future drug candidates; and
the success of Blueprint Medicines' current and future
collaborations, partnerships or licensing arrangements,
including Blueprint Medicines' global collaboration with
Roche for the development and commercialization of GAVRETO. These
and other risks and uncertainties are described in greater detail
in the section entitled "Risk Factors" in Blueprint
Medicines' filings with the Securities and Exchange
Commission (SEC), including Blueprint
Medicines' most recent Annual Report on Form 10-K, as
supplemented by its most recent Quarterly Report on Form 10-Q and
any other filings that Blueprint Medicines has made or
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.
Except as required by law, Blueprint Medicines explicitly
disclaims any obligation to update any forward-looking
statements.
Trademarks
Blueprint Medicines, AYVAKIT, AYVAKYT, GAVRETO and associated
logos are trademarks of Blueprint Medicines Corporation.
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SOURCE Blueprint Medicines Corporation