CAMBRIDGE, Mass., Jan. 10, 2022 /PRNewswire/ -- Blueprint Medicines
Corporation (NASDAQ: BPMC) today outlined upcoming portfolio
milestones that are anticipated to extend its position as a leading
precision therapy company.
"With two precision therapies expanding their global reach
across multiple approved indications, four INDs filed from our
portfolio in 2021, and a range of clinical data inflection points
anticipated over the next 12 to 18 months, Blueprint Medicines is
entering a new, transformative phase of growth," said Jeff Albers, Chief Executive Officer of
Blueprint Medicines. "As we look ahead to 2022, we have the
foundation in place to dramatically expand our opportunity to
improve the lives of increasingly broad populations of patients
with cancer and blood disorders. We will do this by advancing our
systemic mastocytosis franchise to improve treatment across the
spectrum of disease, progressing multiple innovative precision
therapies for lung cancer and other genomically defined cancers
toward registration, and diversifying our scientific platform to
maintain unparalleled research productivity. Supported by strong
commercial performance for AYVAKIT in advanced systemic
mastocytosis and a clear pathway to financial independence, we are
well positioned to usher in a new era of leadership and innovation
in the field of precision medicine."
The company's key strategies and upcoming goals are to:
1. Expand the company's leadership position
in systemic mastocytosis (SM), improving treatment options for
patients across the spectrum of the disease.
AYVAKIT®/AYVAKYT® (avapritinib) and
BLU-263: SM
- Obtain regulatory approval from the European Medicines Agency
and launch AYVAKYT in advanced SM in Europe in the first half of 2022.
- Report topline data from the registration-enabling Part 2 of
the PIONEER trial of AYVAKIT in non-advanced SM in mid-2022.
- Submit a supplemental new drug application to the U.S. Food and
Drug Administration for AYVAKIT in non-advanced SM in the second
half of 2022.
- Present data from the HARBOR trial of BLU-263 in non-advanced
SM in the second half of 2022.
2. Advance a robust portfolio of
innovative clinical programs towards registration.
BLU-945 and BLU-701: EGFR-driven NSCLC
- Present preclinical data supporting the combination of BLU-945
and BLU-701 in the first quarter of 2022.
- Present initial clinical data from the Phase 1/2 SYMPHONY trial
of BLU-945 in the first half of 2022.
- Present initial clinical data from Phase 1/2 HARMONY trial of
BLU-701 in the second half of 2022.
BLU-451 (formerly LNG-451): EGFR exon 20 insertion-positive
NSCLC
- Initiate a Phase 1/2 trial of BLU-451 in the first quarter of
2022.
- Present preclinical data for BLU-451 in the first half of
2022.
BLU-222: Cyclin-E aberrant cancers
- Initiate the Phase 1/2 VELA trial of BLU-222 in the first
quarter of 2022.
- Present preclinical data for BLU-222 in the first half of
2022.
GAVRETO® (pralsetinib): RET-altered cancers
- Submit additional marketing applications for GAVRETO for
RET-altered NSCLC and thyroid cancers across multiple additional
global geographies in 2022, via the company's ongoing global
collaboration with Roche.
3. Grow the R&D pipeline with
diverse, high-value programs from company's prolific scientific
platform.
Research
- Expand pipeline with two new development candidates in
2022.
- Share the company's research vision, including scientific
platform expansion plans, at an R&D Day in the second half of
2022.
Financial Guidance
Blueprint Medicines also announced preliminary1
global product revenues for AYVAKIT for full year 2021 and the
fourth quarter of 2021 were approximately $52.9 million, and $20.0
million, respectively, representing an increase of
approximately 150 percent and 230 percent over the same periods in
2020 based on strong initial U.S. demand in advanced SM. Full year
2021 total revenues, including collaboration revenues, are expected
to be at the higher-end of previous guidance of $170-$180 million.
Cash, cash equivalents and investments as of December 31, 2021 were approximately $1.0 billion.1 Based on its current
operating plans, Blueprint Medicines continues to expect that 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.
1The preliminary selected financial results
are unaudited, subject to adjustment, and provided as an
approximation in advance of the Company's announcement of complete
financial results in February
2022.
About Blueprint Medicines
Blueprint Medicines is a global precision therapy company
that invents life-changing therapies for people with cancer and
blood 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 systemic mastocytosis, lung cancer and other
genomically defined cancers. 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 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; plans to expand Blueprint
Medicines' scientific platform; Blueprint Medicines' plans,
strategies and timelines to nominate development candidates; plans
and timelines for additional marketing applications for avapritinib
and pralsetinib and, if approved, commercializing avapritinib and
pralsetinib in additional geographies or for additional
indications; the potential benefits of any of Blueprint Medicines'
current or future approved drugs or drug candidates in treating
patients; the potential benefits of Blueprint Medicines'
collaborations; preliminary selected financial
results; and Blueprint Medicines' strategy, goals and
anticipated financial performance, 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 continuing to establish and expand a
commercial infrastructure, and successfully launching, marketing
and selling current or future approved products; Blueprint
Medicines' ability to successfully expand the approved indications
for AYVAKIT/AYVAKYT and GAVRETO or obtain marketing approval for
AYVAKIT/AYVAKYT 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 timing and results of
preclinical and clinical studies for Blueprint Medicines' drug
candidates, which may not support further development of such drug
candidates or may impact the timing of data publications or
regulatory submissions; actions of regulatory agencies, which may
affect the initiation, timing and progress of clinical trials;
Blueprint Medicines' ability to obtain, maintain and enforce patent
and other intellectual property protection for AYVAKIT/AYVAKYT,
GAVRETO or any drug candidates it is developing; Blueprint
Medicines' ability to develop and commercialize companion
diagnostic tests for AYVAKIT/AYVAKYT, GAVRETO or any of its current
and future drug candidates; Blueprint Medicines' ability to
successfully expand its operations and scientific platform and the
costs thereof; and the success of Blueprint Medicines' current and
future collaborations, partnerships or licensing arrangements.
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