-- AYVAKIT® (avapritinib) launch in
indolent systemic mastocytosis to drive strong revenue growth in
2024, with ongoing U.S. launch and recent EU approval --
-- Expanding mast cell disease leadership with
oral wild-type KIT inhibitor, BLU-808, advancing into clinical
development; IND submission planned in Q2 2024 --
-- Maintaining durable cash position through
focused investment and global commercial execution --
-- Kate
Haviland, Chief Executive Officer, to present at J.P. Morgan
conference today at 10:30 a.m. PT
(1:30 p.m. ET) --
SAN
FRANCISCO, Jan. 8, 2024 /PRNewswire/ -- Blueprint
Medicines Corporation (Nasdaq: BPMC) today outlined its 2024
corporate strategy to deliver accelerated revenue growth,
sustainable research and development, and a clear path to
profitability.
Kate Haviland, Chief Executive
Officer of Blueprint Medicines, said:
"As we enter 2024, AYVAKIT's early launch success in indolent
systemic mastocytosis has grown our conviction that AYVAKIT has the
potential to be a multi-billion-dollar therapy that will drive
long-term growth into the next decade. We know that the first few
quarters of a launch are critical in defining the trajectory of a
new medicine, and we have built a strong foundation for
success with AYVAKIT as we continue to drive growth in the
U.S. and expand our launch in Europe this year.
Throughout 2023, we also made significant progress across our
research and development pipeline enabling us to focus investments
on our most promising programs. A core component of our growth
strategy is to build on our leadership position in SM by expanding
to other allergic-inflammatory diseases where mast cells play a
core role in the biology, and we have integrated infrastructure
that we can efficiently scale. Across our portfolio, we are
investing in our most compelling opportunities to deliver
innovative, life-changing medicines to patients, while maintaining
a strong and sustainable financial profile."
Focused investment strategy in 2024 to drive long-term growth
and maintain durable cash position
- Prioritized programs for investment
Blueprint Medicines is building portfolio scale in
therapeutic areas where there are significant medical needs in
large patient populations and the company has a deep understanding
of biological pathways, a potential to drive best-in-class efficacy
and an ability to leverage expertise and infrastructure.
Mast cell diseases
- Extend the company's leadership position in systemic
mastocytosis with the ongoing launch of AYVAKIT in the U.S. and EU
and continued development of the next-generation KIT D816V
inhibitor elenestinib.
- Expand into larger patient populations with
allergic-inflammatory diseases with BLU-808, an oral wild-type KIT
inhibitor, including chronic urticaria and other diseases where
mast cells are core to the biology.
Breast cancer and other solid tumors
- Advance combination development of BLU-222, a highly selective
CDK2 inhibitor with best-in-class potential, in HR+/HER2- breast
cancer based on positive previously reported monotherapy clinical
data.
- Progress ongoing strategic partnership discussions to maximize
the potential of BLU-222 as a backbone combination therapy in
HR+/HER2- breast cancer and other CDK2-vulnerable cancers.
- Advance additional programs including BLU-956, a
next-generation CDK2 inhibitor development candidate nominated in
2023, and targeted protein degrader research programs for CDK2 and
an undisclosed target to support long-term lifecycle
management.
- De-prioritized programs
Blueprint Medicines is discontinuing investment in
specific programs, based on the evolving external landscape,
emerging clinical data and partnering considerations.
Lung cancer
-
- Discontinue further investment in the early clinical-stage
therapies BLU-945 and BLU-451 for EGFR-mutant NSCLC and
explore strategic options, including potential out-licensing, based
on the evolving external landscape and emerging clinical data.
- In February 2023, Blueprint
Medicines announced Roche's decision to terminate the global
collaboration agreement for GAVRETO® (pralsetinib).
Given Blueprint Medicines' lack of global infrastructure in lung
and thyroid cancer, the company has decided to discontinue global
development and marketing of GAVRETO in territories excluding the
U.S. and Greater China. The
companies will continue working on transition and wind-down
activities anticipated to begin in the first quarter of 2024;
further information on product discontinuation timing to be
provided in the near future.
- Blueprint Medicines has identified a potential alternate
partner for GAVRETO in the U.S. and is continuing to work with
the involved parties to define a scenario that enables continued
availability of GAVRETO in the U.S.
- Blueprint Medicines expects the wind-down of the Roche
collaboration for GAVRETO will result in significantly lower
year-over-year operating expenses related to GAVRETO in 2024 and
will not affect the $175 million
upfront payment received under a 2022 financing agreement with
Royalty Pharma.
As a result of continued strategic portfolio prioritization,
Blueprint Medicines expects a year-over-year decline in operating
expenses in 2024. The company plans to provide financial guidance
for 2024, including anticipated AYVAKIT revenue, when it
reports fourth quarter and full-year 2023 financial results in
February 2024.
2024 Corporate Milestones
The company's anticipated 2024 corporate milestones include:
Mast cell diseases
- Present long-term safety and efficacy data from the PIONEER
trial of AYVAKIT in indolent SM (ISM) in the first half of
2024.
- Submit an investigational new drug (IND) application for
BLU-808 in the second quarter of 2024.
- Initiate the registration-enabling Part 2 of the HARBOR trial
of elenestinib in ISM in the second half of 2024.
Breast cancer and other solid tumors
- Continue ongoing strategic business development
discussions.
- Present data for BLU-222 in combination with ribociclib and
fulvestrant in patients with HR+/HER2- breast cancer in the first
half of 2024.
- Provide update on BLU-222 registration plan in HR+/HER2- breast
cancer in the second half of 2024.
J.P. Morgan Healthcare Conference Presentation
Information
Kate Haviland, Chief Executive
Officer of Blueprint Medicines, will present a company overview and
2024 outlook at the 42nd Annual J.P. Morgan Healthcare Conference
on Monday, January 8 at 10:30 a.m. PT / 1:30 p.m.
ET. A live webcast of the presentation and Q&A breakout
session will be available by visiting the "Events and
Presentations" section of Blueprint Medicines' website at
http://ir.blueprintmedicines.com. A replay of the webcast will be
archived on Blueprint Medicines' website for 30 days following the
presentation.
About Blueprint Medicines
Blueprint Medicines is a global precision therapy company that
invents life-changing medicines. Applying an approach that is both
precise and agile, we create therapies that selectively target the
root cause of disease, 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 have brought our approved medicines to patients in
the United States and Europe, and we are globally advancing multiple
programs for mast cell disorders, including systemic mastocytosis
and chronic urticaria, breast cancer and other solid tumors. For
more information, visit www.BlueprintMedicines.com and follow us on
X (formerly 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' views with respect to AYVAKIT's
potential to be a multi-billion-dollar therapy; the continued
growth of AYVAKIT in the U.S. and the expansion of AYVAKYT's launch
in Europe; the expansion of
Blueprint Medicines' mast cell disease franchise with the
development of BLU-808; the advancement of its clinical development
of BLU-222, preclinical development of BLU-956 and progression of
targeted protein degrader research programs for CDK2; plans,
strategies, timelines and expectations for Blueprint Medicines'
current or future approved drugs and drug candidates; the potential
benefits of any of Blueprint Medicines' current or future approved
drugs or drug candidates in treating patients; and Blueprint
Medicines' financial performance, strategy, goals and anticipated
milestones, business plans, outlook 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: preliminary activity and safety data may not be
representative of more mature data; the risk of delay of any
current or planned clinical trials or the development of Blueprint
Medicines' current or future drug candidates; risks related to
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; preclinical and clinical
results for Blueprint Medicines' drug candidates may not support
further development of such drug candidates either as monotherapies
or in combination with other agents or may impact the anticipated
timing of data or regulatory submissions; the timing of the
initiation of clinical trials and trial cohorts at clinical trial
sites and patient enrollment rates may be delayed or slower than
anticipated; actions of regulatory agencies may affect the
initiation, timing and progress of clinical trials; the success of
Blueprint Medicines' current and future collaborations, financing
arrangements, partnerships or licensing arrangements may impact
Blueprint Medicines' ability to capitalize on the market potential
of its approved drugs and drug candidates; and risks related to
Blueprint Medicines' ability to obtain, maintain and enforce patent
and other intellectual property protection for its products and
current or future drug candidates it is developing. 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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