-- Breadth of pipeline featured in multiple
early clinical data presentations, reflecting diversity of
fundamental growth drivers across development programs --
CAMBRIDGE, Mass., June 3, 2023
/PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC)
today announced clinical data for multiple programs across its
precision therapy portfolio at the 2023 American Society of
Clinical Oncology (ASCO) Annual Meeting. The presentations showcase
the company's progress in developing precision therapies to address
the needs of broad patient populations, including
hormone-receptor-positive/HER2-negative (HR+/HER2-) breast cancer
and EGFR-mutant non-small cell lung cancer (NSCLC).
"At ASCO, we are reporting early clinical data for three
therapeutic candidates highlighting the breadth of our pipeline,
which lays the foundation for our next wave of precision therapy
programs to tackle significant medical challenges," said Becker
Hewes, M.D., Chief Medical Officer at Blueprint Medicines.
"Together, these presentations reflect how we are developing
differentiated, highly selective therapies against promising
disease targets, and demonstrate strong execution in advancing
potential treatment options for large patient populations,
including HR+/HER2- breast cancer and EGFR-mutant lung cancer. The
datasets represent a diversity of fundamental value drivers in our
clinical portfolio, which create opportunities to substantially
expand our patient impact and highlight our ambition to deliver
many more transformational medicines to patients in the
future."
CDK2 Program
CDK2 is a cell cycle regulator and important cancer target
across a broad range of malignancies, including HR+/HER2- breast
cancer. Based on the underlying science on the role of CDK2
inhibition in cancer, and the potential for this mechanism to
impact large populations of patients, BLU-222 represents one of the
most important programs within Blueprint Medicines' early clinical
development pipeline. The data reported at ASCO begin to
demonstrate the clinical profile of BLU-222, a highly selective and
potent oral CDK2 inhibitor.
VELA trial: BLU-222 in HR+/HER2- metastatic breast cancer
and other advanced cancers
Abstract number:
3095
Presentation location and time: Hall A; Saturday, June 3 from 8:00
a.m. — 11:00 a.m. CT
Results from the ongoing dose escalation part of the VELA trial
of BLU-222 (n=27) showed encouraging safety and evidence of cell
cycle pathway modulation consistent with the treatment's
best-in-class preclinical profile. Based on these data, Blueprint
Medicines is continuing monotherapy dose escalation to identify a
maximum tolerated dose, and has initiated dose escalation of
BLU-222 in combination with the CDK4/6 inhibitor, ribociclib, and
the estrogen receptor antagonist, fulvestrant, in patients with
HR+/HER2- metastatic breast cancer.
- BLU-222 was generally well-tolerated with no discontinuations
due to adverse events (AEs). Treatment-related hematologic AEs
commonly seen with CDK4/6 inhibitors were generally mild and
primarily reported in patients with a history of low blood cell
counts. No cardiac AEs or QTc prolongation were observed.
- BLU-222 showed evidence of cell cycle pathway modulation as
demonstrated by circulating and tumor tissue-based biomarker data.
Increased BLU-222 doses led to robust reductions in thymidine
kinase 1 (TK1) activity and phosphorylated retinoblastoma
(pRB).
- In addition, the presentation highlighted a confirmed partial
response in a BLU-222 monotherapy-treated patient with HR+/HER2-
metastatic breast cancer who previously received five lines of
therapy, including the CDK4/6 inhibitors palbociclib and
abemaciclib.
EGFR Programs
EGFR represents one of the most common oncogenic drivers in
NSCLC, and Blueprint Medicines is developing a portfolio of
investigational precision therapies to inhibit the broad spectrum
of EGFR activating and resistance mutations. With three novel EGFR
inhibitors, the company seeks to transform treatment options for
patients with EGFR-mutant NSCLC, including with highly active,
well-tolerated combinations in the front-line setting. Data
reported at ASCO feature two of the company's investigational EGFR
inhibitors:
- BLU-451, a wildtype-EGFR-sparing, CNS-penetrant oral inhibitor
of EGFR exon 20 insertions and atypical mutations with
best-in-class potential
- BLU-945, a potent oral EGFR inhibitor designed to be highly
selective over wildtype EGFR, supporting its potential as a
differentiated combination partner
CONCERTO trial: BLU-451 in advanced NSCLC driven by EGFR
exon 20 insertions or atypical mutations
Abstract
number: 9064
Presentation location and time: Hall A;
Sunday, June 4 from 8:00 a.m. — 11:00 a.m.
CT
Results from the ongoing dose escalation part of the CONCERTO
trial of BLU-451 in heavily pretreated patients with NSCLC driven
by EGFR exon 20 insertions (n=48) or atypical mutations (n=9)
showed evidence of safety and clinical benefits, including central
nervous system (CNS) activity. The data support continued dose
escalation to determine the recommended Phase 2 dose (RP2D), and
further development in patients with EGFR exon 20 insertions as
well as additional patients with atypical EGFR mutations.
Collectively, these mutations represent approximately 20 percent of
EGFR-mutant NSCLC cases.
- BLU-451 was generally well-tolerated in patients, with no grade
3 or higher AEs associated with wildtype EGFR inhibition, no
dose-limiting toxicities and no discontinuations due to
treatment-related AEs.
- In patients with EGFR exon 20 insertion-positive NSCLC, BLU-451
demonstrated evidence of clinical activity including circulating
tumor DNA (ctDNA) clearance, tumor reductions including confirmed
partial responses, and CNS activity. Multiple patients showed CNS
benefits including one who had a partial CNS response per RECIST
version 1.1 criteria and one who had a CNS complete response with
resolution of multiple non-target brain lesions, as highlighted by
patient vignettes in the presentation.
- In patients with NSCLC driven by atypical EGFR mutations,
BLU-451 showed emerging dose-dependent ctDNA clearance and tumor
shrinkage, reflecting an expanded development opportunity.
SYMPHONY trial: BLU-945 monotherapy and in combination
with osimertinib in late-line, EGFR-mutant
NSCLC
Abstract number: 9011
Presentation
location and time: S406; Monday, June
5 from 11:30 a.m. —
12:30 p.m. CT
Updated results from the dose escalation part of the SYMPHONY
trial showed the safety and clinical activity of BLU-945 as a
monotherapy (n=112) and in combination with osimertinib (n=55) in
patients with late-line, osimertinib-refractory, EGFR-mutant NSCLC.
Based on the unprecedented and favorable safety profile for the
combination of BLU-945 and osimertinib observed to-date, dose
escalation is continuing which impacts the timeframe to establish
the RP2D and begin the randomized, first-line treatment portion of
the study. Consequently, the company no longer anticipates
presenting initial dose expansion data for BLU-945 in combination
with osimertinib in first-line, EGFR L858R-positive NSCLC by the
end of 2023.
- BLU-945, as a monotherapy and in combination with osimertinib,
was generally well-tolerated, and AEs associated with wildtype EGFR
inhibition were infrequent, with the majority reported as Grade 1.
For the combination, the discontinuation rate due to
treatment-related AEs was 3.6 percent.
- BLU-945 monotherapy and the combination showed evidence of
clinical activity, with ctDNA clearance and tumor reductions
including confirmed partial responses observed in patients whose
tumors had progressed following treatment with osimertinib. For
combination regimens with a total BLU-945 daily dose of 300 mg or
higher, tumor shrinkage was observed in 51 percent of patients with
molecularly heterogenous, osimertinib-refractory, late-line
EGFR-mutant NSCLC, with multiple confirmed responses.
Copies of Blueprint Medicines data presentations from the ASCO
Annual Meeting are available in the "Science—Publications and
Presentations" section of the company's website at
www.blueprintmedicines.com.
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 our
approved medicines to patients in the United
States and Europe, and we are globally advancing multiple
programs for systemic mastocytosis, lung cancer, breast cancer and
other genomically defined cancers, 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' views with respect to its
ability to expand its patient impact and deliver more
transformational medicines to patients; the potential benefits of
Blueprint Medicines' current and 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
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although not all forward-looking statements contain these
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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
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limitation, risks and uncertainties related to our ability and
plans in continuing to expand Blueprint Medicines' 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
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 our 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 either as monotherapies or in combination with other
agents or may impact the anticipated timing of data 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 its products or any drug
candidates it is developing; Blueprint Medicines' ability to
develop and commercialize companion diagnostic tests for its
products or any of its current and future drug candidates;
Blueprint Medicines' ability to successfully expand its research
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 its Annual Report on Form 10-K for the
year ended December 31, 2022, as
filed with the SEC on February 16,
2023, 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 assumes no
obligation to update or revise these forward-looking statements for
any reason, even if new information becomes available in the
future.
Trademarks
Blueprint Medicines, AYVAKIT, AYVAKYT and associated logos are
trademarks of Blueprint Medicines Corporation.
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