-- HARBOR Part 1 trial data in indolent
systemic mastocytosis showed elenestinib was well-tolerated with
broad symptom improvement, supporting further development to expand
and extend company's SM franchise leadership --
CAMBRIDGE, Mass., Dec. 9, 2023
/PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC)
today announced data showcasing its commitment to advance the
scientific understanding and treatment of systemic mastocytosis
(SM) at the 65th American Society of Hematology (ASH) Annual
Meeting and Exposition being held December
9-12 in San Diego. Data
that will be presented include results from the HARBOR Part 1 trial
of elenestinib in indolent systemic mastocytosis (ISM) and analyses
of real-world data highlighting the burden of and urgency to treat
ISM.
"Blueprint Medicines has transformed the standard of care for
advanced and indolent systemic mastocytosis with
AYVAKIT® (avapritinib), and its proven and compelling
clinical profile is redefining what well-controlled disease means
for patients living with SM," said Becker Hewes, M.D., Chief
Medical Officer at Blueprint Medicines. "Building on the success of
AYVAKIT and the clinical expertise amassed during its development,
we are strategically advancing our investigational next-generation
KIT D816V inhibitor, elenestinib, to expand and extend Blueprint
Medicines' SM franchise leadership over the long term."
HARBOR Part 1 trial data in patients with ISM showed elenestinib
was well-tolerated and clinically active at all dose levels tested,
supporting further development. In patients treated with
elenestinib, most adverse events (AEs) were Grade 1 or 2, and there
were no discontinuations due to AEs. Elenestinib showed clinically
meaningful symptom improvements as assessed by the validated
Indolent Systemic Mastocytosis Symptom Assessment Form Total
Symptom Score (ISM-SAF TSS), and rapid and profound reductions
across multiple measures of mast cell burden.
At ASH, new data on the burden of disease highlight the urgency
to treat patients with ISM. A real-world analysis of U.S. health
claims data showed patients with ISM had lower survival compared to
a matched population cohort (p<0.0001), and a model-based
analysis assessed that the lifetime risk of progression from ISM to
advanced SM was approximately 20 percent. In addition, a data
presentation reports on a diagnostic tool to aid in the
identification of patients with SM, which was developed based on a
real-world analysis at The Quality Cancer Care Alliance (QCCA).
In total, Blueprint Medicines' presence at ASH builds on over a
decade of innovative research in the field of SM, and reflects the
company's ongoing leadership in transforming care for patients
living with the disease.
ASH abstracts are listed below:
Oral Presentations
Presentation Title: Decreased Survival Among Patients
with Indolent Systemic Mastocytosis: A Population-Level
Retrospective Cohort Analysis Using Healthcare Claims Dataset
Session Title: 634. Myeloproliferative Syndromes: Clinical
and Epidemiological: Rare Myeloproliferative Neoplasms: Unveiling
Promising Pathways and Novel Therapies
Session Date & Time: Today, December 9 from 9:30 – 11:00 a.m. PT (12:30 – 2:00 p.m. ET)
Presentation Date & Time: Today, December 9 at 10:00 a.m.
PT (1:00 p.m. ET)
Abstract Number: 75
Location: San Diego
Convention Center, Ballroom 20AB
Presentation Title: Elenestinib, an Investigational, Next
Generation KIT D816V Inhibitor, Reduces Mast Cell Burden, Improves
Symptoms, and Has a Favorable Safety Profile in Patients with
Indolent Systemic Mastocytosis: Analysis of the HARBOR Trial
Session Title: 634. Myeloproliferative Syndromes: Clinical
and Epidemiological: Rare Myeloproliferative Neoplasms: Unveiling
Promising Pathways and Novel Therapies
Session Date & Time: Today, December 9 from 9:30 – 11:00 a.m. PT (12:30 – 2:00 p.m. ET)
Presentation Date & Time: Today, December 9 at 10:15 a.m.
PT (1:15 p.m. ET)
Abstract Number: 76
Location: San Diego
Convention Center, Ballroom 20AB
Poster Presentation
Presentation Title: Development and Validation of a
Diagnostic Tool for the Timely Diagnosis of Patients with Systemic
Mastocytosis
Session Title: 906. Outcomes Research—Myeloid Malignancies:
Poster II
Session Date & Time: Sunday,
December 10 from 6:00 – 8:00 p.m.
PT (9:00 – 11:00 p.m. ET)
Abstract Number: 3800
Location: San Diego
Convention Center, Halls G-H
Publication-Only Abstract
Title: A Model of Cumulative Risk of Disease Progression
Among Patients with Indolent Systemic Mastocytosis
Abstract Number: 6406
Copies of Blueprint Medicines data presentations from the ASH
annual meeting will be available in the "Science—Publications and
Presentations" section of the company's website at
www.BlueprintMedicines.com.
About AYVAKIT (avapritinib)
AYVAKIT (avapritinib) is a precision therapy approved by the
U.S. Food and Drug Administration (FDA) for the treatment of three
indications: adults with ISM, adults with advanced SM, including
aggressive SM (ASM), SM with an associated hematological neoplasm
(SM-AHN) and mast cell leukemia (MCL), and adults with unresectable
or metastatic gastrointestinal stromal tumor (GIST) harboring a
PDGFRA exon 18 mutation, including PDGFRA D842V mutations. For more
information, visit AYVAKIT.com. This medicine is approved by
the European Commission (AYVAKYT®) for the treatment of
adults with ASM, SM-AHN or MCL, after at least one systemic
therapy, and adults with unresectable or metastatic GIST harboring
the PDGFRA D842V mutation. Please click here to see the
full U.S. Prescribing Information for AYVAKIT, and
click here to see the European Summary of Product
Characteristics for AYVAKYT.
In November 2023, the Committee
for Medicinal Products for Human Use (CHMP) of the European
Medicines Agency (EMA) issued a positive opinion recommending the
approval of AYVAKYT for the treatment of adult patients with ISM
with moderate to severe symptoms inadequately controlled on
symptomatic treatment.
To learn about ongoing or planned clinical trials,
contact Blueprint Medicines at
medinfo@blueprintmedicines.com or 1-888-BLU-PRNT
(1-888-258-7768). Additional information is available at
blueprintclinicaltrials.com or clinicaltrials.gov.
Important Safety Information
Intracranial Hemorrhage—Serious intracranial hemorrhage (ICH)
may occur with AYVAKIT treatment; fatal events occurred in <1%
of patients. Overall, ICH (eg, subdural hematoma, ICH, and cerebral
hemorrhage) occurred in 2.9% of 749 patients who received AYVAKIT
in clinical trials. In Advanced SM patients who received AYVAKIT at
200 mg daily, ICH occurred in 2 of 75 patients (2.7%) who had
platelet counts ≥50 x 109/L prior to initiation of
therapy and in 3 of 80 patients (3.8%) regardless of platelet
counts. In ISM patients, no events of ICH occurred in the 246
patients who received any dose of AYVAKIT in the PIONEER study.
Monitor patients closely for risk factors of ICH, which may
include history of vascular aneurysm, ICH or cerebrovascular
accident within the prior year, concomitant use of anticoagulant
drugs, or thrombocytopenia.
Symptoms of ICH may include headache, nausea, vomiting, vision
changes, or altered mental status. Advise patients to seek
immediate medical attention for signs or symptoms of ICH.
Permanently discontinue AYVAKIT if ICH of any grade occurs. In
Advanced SM patients, a platelet count must be performed prior to
initiating therapy. AYVAKIT is not recommended in Advanced SM
patients with platelet counts <50 x 109/L. Following
treatment initiation, platelet counts must be performed every 2
weeks for the first 8 weeks. After 8 weeks of treatment, monitor
platelet counts every 2 weeks or as clinically indicated based on
platelet counts. Manage platelet counts of <50 x
109/L by treatment interruption or dose reduction.
Cognitive Effects—Cognitive adverse reactions can occur in
patients receiving AYVAKIT and occurred in 33% of 995 patients
overall in patients who received AYVAKIT in clinical trials
including: 28% of 148 Advanced SM patients (3% were Grade ≥3), and
7.8% of patients with ISM who received AYVAKIT + best supportive
care (BSC) versus 7.0% of patients who received placebo + BSC
(<1% were Grade 3). Depending on the severity and indication,
withhold AYVAKIT and then resume at same dose or at a reduced dose
upon improvement, or permanently discontinue.
Photosensitivity—AYVAKIT may cause photosensitivity reactions.
In all patients treated with AYVAKIT in clinical trials (n=1049),
photosensitivity reactions occurred in 2.5% of patients. Advise
patients to limit direct ultraviolet exposure during treatment with
AYVAKIT and for one week after discontinuation of treatment.
Embryo-Fetal Toxicity—AYVAKIT can cause fetal harm when
administered to a pregnant woman. Advise pregnant women of the
potential risk to a fetus. Advise females and males of reproductive
potential to use an effective method of contraception during
treatment with AYVAKIT and for 6 weeks after the final dose of
AYVAKIT. Advise women not to breastfeed during treatment with
AYVAKIT and for 2 weeks after the final dose.
Adverse Reactions—The most common adverse reactions (≥20%) in
patients with Advanced SM were edema, diarrhea, nausea, and
fatigue/asthenia.
The most common adverse reactions (≥10%) in patients with ISM
were eye edema, dizziness, peripheral edema, and flushing.
Drug Interactions—Avoid coadministration of AYVAKIT with strong
or moderate CYP3A inhibitors. If coadministration with a moderate
CYP3A inhibitor cannot be avoided in patients with Advanced SM,
reduce dose of AYVAKIT. Avoid coadministration of AYVAKIT with
strong or moderate CYP3A inducers.
To report suspected adverse reactions, contact Blueprint
Medicines Corporation at 1-888-258-7768 or FDA at 1-800-FDA-1088 or
http://www.fda.gov/medwatch.
Please click here to see the full Prescribing
Information for AYVAKIT.
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 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 cancers vulnerable
to CDK2 inhibition, as well as EGFR-mutant lung cancer. 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' expectations regarding the potential
benefits of AYVAKIT for the treatment of patients with ISM,
including with respect to the burden of and urgency to treat ISM;
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 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. 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 and associated logos are
trademarks of Blueprint Medicines Corporation.
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SOURCE Blueprint Medicines Corporation