CAMBRIDGE, Mass., April 10, 2021 /PRNewswire/ -- Blueprint
Medicines Corporation (NASDAQ: BPMC) today announced data from
multiple poster presentations highlighting the breadth of the
company's precision therapy pipeline at the virtual American
Association for Cancer Research (AACR) Annual Meeting 2021.
Collectively, the presentations, including foundational preclinical
data for multiple programs, demonstrate the productivity of the
company's scientific platform. Additional presentations of clinical
data for AYVAKIT™ (avapritinib) and BLU-263 will be reported on
Sunday, April 11, 2021.
"Our data presentations at the AACR annual meeting showcase
Blueprint Medicines' next wave of precision therapies, which have
the potential to impact large global patient populations," said
Fouad Namouni, M.D., President, Research & Development at
Blueprint Medicines. "Building on the foundation of our approved
medicines AYVAKIT and GAVRETO®, these presentations
highlight our efforts to address significant patient needs in areas
such as EGFR-driven lung cancer, cyclin E-aberrant cancers and
cancer immunotherapy. As we celebrate our 10th
anniversary as a company, and our rapidly expanding pipeline now
includes our ninth development candidate, we look forward to
continuing to leverage our productive research platform to advance
potent and selective inhibitors that have the potential to enable
transformative benefit for patients."
BLU-701 and BLU-945: Double- and triple-mutant inhibitors in
EGFR-driven NSCLC
Lung cancer is the leading cause of cancer death globally, and
approximately 17 percent of patients with lung adenocarcinoma, the
most common form of non-small cell lung cancer (NSCLC), have
EGFR-driven disease. While first- and third-generation EGFR
inhibitors have improved treatment outcomes for patients with
EGFR-driven NSCLC, resistance inevitably emerges, with the T790M
and C797S mutations being the most common on-target resistance
mechanisms. Together, BLU-701 and BLU-945 are designed to provide
comprehensive coverage of the most common activating and on-target
resistance mutations, spare wild-type EGFR to limit toxicities
driven by wild-type EGFR inhibition and treat or prevent central
nervous system (CNS) metastases. Ultimately, with these
characteristics, BLU-701 and BLU-945 have the potential to be used
either as monotherapy or in combination, together or with other
agents, to potentially overcome or prevent on-target resistance
across multiple lines of treatment.
BLU-701 is a potential best-in-class, selective, potent,
fourth-generation double-mutant EGFR inhibitor with activity
against EGFR activating mutations and the C797S
osimertinib-resistant mutation. Preclinical data presented at the
conference showed strong and durable inhibition of tumor growth at
doses that are EGFR wild-type sparing. BLU-701 also indicated
significant CNS penetration in preclinical models, with comparable
exposure in the plasma and brain, which illustrates its potential
to treat or prevent CNS metastases in patients with EGFR-driven
tumors. With activity also shown against the activating EGFR
mutants, BLU-701 has potential to be used in both first- and
second-line settings.
BLU-945 is a potential first- and best-in-class, selective,
potent, fourth-generation triple-mutant EGFR inhibitor with
activity against the T790M and C797S resistance mutations.
BLU-945 is highly selective over wild-type EGFR and off-target
kinases, highlighting its potential to enable tolerable
combinations with BLU-701 or other therapies. Data presented at the
conference demonstrated potent anti-tumor activity in triple-mutant
osimertinib-resistant tumor models, as well as activity in a
triple-mutant intracranial patient-derived xenograft model. In
addition, the combination of BLU-945 with either gefitinib
or osimertinib showed enhanced anti-tumor activity when
compared with either gefitinib or osimertinib alone.
The preclinical data presented for BLU-701 and BLU-945 support
the continued development of both candidates in patients with
EGFR-driven NSCLC. An investigational new drug (IND) application
for BLU-945 has been cleared by the U.S. Food and Drug
Administration (FDA) and an international Phase 1 dose escalation
trial is expected to begin this quarter. Future clinical
development of BLU-945 in combination with other agents across
multiple treatment settings is planned. BLU-701 is expected to
enter clinical development later this year.
BLU-222: CDK2 inhibitor in cyclin E-aberrant cancers
Cyclin dependent kinases (CDKs) and their cyclin partners
regulate the cell cycle. In subsets of patients across multiple
cancer types, aberrant cyclin E (CCNE) hyperactivates CDK2,
resulting in cell cycle dysregulation and tumor proliferation.
Aberrant CCNE has been observed as a primary driver of disease as
well as a mechanism of resistance to CDK4/6 inhibitors and other
therapies. In addition, data have shown that ovarian and
hormone-receptor-positive breast cancer patients with aberrant CCNE
have poor outcomes. Prior drug discovery efforts targeting CDK2
have been hindered by challenges in achieving selectivity over
other CDK family members associated with toxicity.
At AACR, preclinical data highlighted a set of potent and
selective CDK2 inhibitors designed by Blueprint Medicines. The data
showed that selective CDK2 inhibition arrested the cell cycle and
blocked tumor proliferation in CCNE-amplified cell lines and
demonstrated robust and sustained anti-tumor activity in vivo in
models of CCNE-amplified ovarian, breast and gastric cancer. A
selective CDK2 inhibitor also showed improved tolerability compared
to a pan-CDK inhibitor and chemotherapy, as measured by animal body
weight.
Based on this work and further optimization, Blueprint Medicines
today announced the nomination of a potentially best-in-class
selective and potent CDK2 inhibitor development candidate, BLU-222,
which is expected to enter clinical development in the first half
of 2022.
BLU-852: MAP4K1 inhibitor
MAP4K1 is a well-characterized immunokinase target involved in
the regulation of immune cells; however, prior drug discovery
efforts have been hindered by challenges in achieving selectivity
over other MAP4K family members associated with toxicity. In
January 2021, Blueprint Medicines
announced the nomination of a highly selective and potent MAP4K1
inhibitor development candidate, BLU-852, with best-in-class
potential.
Data presented at AACR highlighted a set of potent and highly
selective MAP4K1 inhibitors designed by Blueprint Medicines,
including BLU-852. The inhibitors were shown to enhance
intratumoral immune cell activation, overcome T cell suppression,
and reduce tumor burden both as a monotherapy and in combination
with checkpoint inhibition. The data support the continued
development of BLU-852 under the company's cancer immunotherapy
collaboration with Roche, with Phase 1 trial initiation anticipated
in 2022.
Copies of Blueprint Medicines data presentations from the AACR
annual meeting are available in the "Science—Publications and
Presentations" section of the company's website at
www.BlueprintMedicines.com.
Conference Call Information
Blueprint Medicines will host a live webcast on Monday, April 12, 2021 beginning at 8:00 a.m. ET to review data for multiple
research- and clinical-stage programs presented at the AACR annual
meeting. To access the live call, please dial (855) 728-4793
(domestic) or (503) 343-6666 (international) and refer to
conference ID 5548976. A webcast of the conference call will be
available under "Events and Presentations" in the
Investors & Media section of Blueprint Medicines' website
at http://ir.blueprintmedicines.com. The archived webcast will
be available on Blueprint Medicines' website approximately two
hours after the conference call and will be available for 30 days
following the call.
About Blueprint Medicines
Blueprint Medicines is a global precision therapy company that
invents life-changing medicines for people with cancer and
hematologic disorders. Applying an approach that is both precise
and agile, we create therapies 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 our approved medicines 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 the plans, timing, designs, implementation, enrollment
and announcement of results regarding Blueprint Medicines' ongoing
and planned clinical trials for its drug candidates; the potential
benefits of Blueprint Medicines' current and future 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™/AYVAKYT® (avapritinib) and
GAVRETO® (pralsetinib); Blueprint
Medicines' ability to successfully expand the approved
indications for AYVAKIT/AYVAKYT and GAVRETO or obtain marketing
approval for AYVAKIT/AYVAKYT 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. 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.
View original content to download
multimedia:http://www.prnewswire.com/news-releases/blueprint-medicines-presents-preclinical-data-highlighting-broad-precision-therapy-research-pipeline-at-aacr-annual-meeting-2021-301266154.html
SOURCE Blueprint Medicines Corporation