CAMBRIDGE, Mass., Nov. 23, 2020 /PRNewswire/ -- Blueprint Medicines
Corporation (NASDAQ: BPMC), a precision therapy company focused on
genomically defined cancers, rare diseases and cancer
immunotherapy, today announced plans to report data presentations
highlighting the medical needs in systemic mastocytosis (SM) and
the broad potential of AYVAKIT™ (avapritinib) across multiple SM
patient populations at the 62nd American Society of
Hematology (ASH) Annual Meeting and Exposition. AYVAKIT is designed
to potently and selectively target D816V mutant KIT, the primary
driver of SM, and is being developed for the treatment of advanced
and non-advanced forms of the disease.
"The breadth of our upcoming ASH presentations highlights our
commitment to fundamentally change the way systemic mastocytosis is
diagnosed and treated across a broad population of patients
impacted by this disease," said Andy Boral, M.D., Ph.D., Chief
Medical Officer at Blueprint Medicines. "These presentations
reflect our collaborative efforts with the systemic mastocytosis
community to better understand the burden of disease, advance
important strategies for facilitating diagnosis and establish new
methods for measuring treatment response."
The accepted abstracts are listed below and available on the ASH
conference website:
https://www.hematology.org/meetings/annual-meeting.
Oral Presentation
Presentation Title: Pure Pathologic Response Is
Associated with Improved Overall Survival in Patients with Advanced
Systemic Mastocytosis Receiving Avapritinib in the Phase I EXPLORER
Study
Session Title: Myeloproliferative Syndromes: Clinical:
Translational Science in MPN—Hitting the Mark
Session Date & Time: Sunday, December 6, 2020 at
10:15 a.m. PT (1:15 p.m. ET)
Abstract Number: 345
Poster Presentations
Presentation Title: Patient Reported Outcomes Among
Systemic Mastocytosis (SM) Patients in Routine Clinical Practice:
Results from the TouchStone Survey
Session Title: Outcomes Research—Non-Malignant Conditions:
Poster I
Session Date & Time: Saturday,
December 5, 2020 from 7:00
a.m. – 3:30 p.m. PT (10:00
a.m. – 6:30 p.m. ET)
Abstract Number: 1638
Presentation Title: Results from PIONEER: A
Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of
Avapritinib in Patients with Indolent Systemic Mastocytosis
Session Title: Myeloproliferative Syndromes: Clinical:
Poster I
Session Date & Time: Saturday, December 5, 2020
from 7:00 a.m. – 3:30 p.m. PT (10:00
a.m. – 6:30 p.m. ET)
Abstract Number: 1248
Presentation Title: Increased Detection of KIT D816V
Mutation in Peripheral Blood Samples from Patients with Indolent
Systemic Mastocytosis (ISM) in the Phase 2 PIONEER Study Using a
High Sensitivity Droplet Digital (dd) PCR Assay Compared with Next
Generation Sequencing (NGS)
Session Title: Hodgkin Lymphoma and T/NK Cell
Lymphoma—Clinical Studies: Poster III
Session Date & Time: Monday, December 7, 2020 from
7:00 a.m. – 3:30 p.m. PT
(10:00 a.m. – 6:30 p.m.
ET)
Abstract Number: 3004
Presentation Title: Perceptions of Patient Disease Burden
and Management Approaches of Systemic Mastocytosis By Healthcare
Providers: Results from the TouchStone SM Survey
Session Title: Health Services Research—Non-Malignant
Conditions: Poster III
Session Date & Time: Monday,
December 7, 2020 from 7:00
a.m. – 3:30 p.m. PT (10:00 a.m. – 6:30 p.m. ET)
Abstract Number: 3403
Presentation Title: PIONEER Part 2: A Randomized,
Double-Blind, Placebo-Controlled, Phase 2 Study to Evaluate Safety
and Efficacy of Avapritinib in Indolent Systemic Mastocytosis
Session Title: Hodgkin Lymphoma and T/NK Cell
Lymphoma—Clinical Studies: Poster III
Session Date & Time: Monday,
December 7, 2020 from 7:00
a.m. – 3:30 p.m. PT (10:00 a.m. – 6:30 p.m. ET)
Abstract Number: 2988
About AYVAKIT (avapritinib)
AYVAKIT (avapritinib) is a kinase inhibitor approved by the U.S.
Food and Drug Administration (FDA) for the treatment of adults with
unresectable or metastatic gastrointestinal stromal tumor (GIST)
harboring a PDGFRA exon 18 mutation, including PDGFRA D842V
mutations. For more information, visit www.AYVAKIT.com. This
medicine is approved in Europe
under the brand name AYVAKYT® for the treatment of
adults with unresectable or metastatic GIST harboring the PDGFRA
D842V mutation.
AYVAKIT is not approved for the treatment of any other
indication, including SM, in the U.S. by the FDA or in Europe by the European Commission, or for any
indication in any other jurisdiction by any other health
authority.
Blueprint Medicines is developing AYVAKIT globally for the
treatment of advanced and indolent SM. The FDA granted breakthrough
therapy designation to AYVAKIT for the treatment of advanced SM,
including the subtypes of aggressive SM, SM with an associated
hematologic neoplasm and mast cell leukemia.
Blueprint Medicines has an exclusive collaboration and license
agreement with CStone Pharmaceuticals for the development and
commercialization of AYVAKIT in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains
development and commercial rights for AYVAKIT in the rest of the
world.
About Blueprint Medicines
Blueprint
Medicines is a precision therapy company striving to improve
human health. With a focus on genomically defined cancers, rare
diseases and cancer immunotherapy, we are developing
transformational medicines rooted in our leading expertise in
protein kinases, which are proven drivers of disease. Our uniquely
targeted, scalable approach empowers the rapid design and
development of new treatments and increases the likelihood of
clinical success. We have two approved precision therapies and are
currently advancing multiple investigational medicines in clinical
and pre-clinical development, along with a number of earlier-stage
research programs. 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,
timelines and expectations for interactions with the FDA and other
regulatory authorities; expectations regarding the potential
benefits of AYVAKIT in treating patients with SM; 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™ (pralsetinib);
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. 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