-- Breadth of data across 7 presentations,
including 4 oral, reflects continued leadership in advancing the
understanding and treatment of systemic mastocytosis --
CAMBRIDGE, Mass., May 30, 2024
/PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC)
today announced multiple upcoming datasets across two key
conferences that reinforce the significant real-world burden of
systemic mastocytosis (SM), and highlight the durable clinical
outcomes of AYVAKIT®/AYVAKYT® (avapritinib)
across the spectrum of the disease. The presentations, which build
on over a decade of pioneering research with clinical experts and
patient advocates, will be reported at the following meetings:
- European Academy of Allergy and Clinical Immunology (EAACI)
Congress 2024, May 31 to June 3
- European Hematology Association 2024 (EHA24) Hybrid Congress,
June 13 to 16
"Our data reinforce the efficacy and safety of prolonged
durations of AYVAKIT therapy, and highlight the urgency to diagnose
and treat patients living with systemic mastocytosis," said Becker
Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "Based
on unprecedented clinical datasets and the strength of our ongoing
commercial launches in the U.S. and Europe, we have made significant progress
toward establishing AYVAKIT as the global standard of care. We look
forward to continued collaboration with the SM community to
redefine what 'well-controlled' means for patients across the
spectrum of the disease."
PIONEER: With a median follow-up of more than two years,
AYVAKIT showed durable efficacy and a favorable safety profile in
patients with ISM. Safety data were consistent for the small number
of patients (~10 percent) who dose escalated to 50 mg once
daily.
- AYVAKIT led to sustained improvements in total symptom score
and all symptom domains (assessed by the Indolent Systemic
Mastocytosis Symptom Assessment Form), as well as quality of life
(assessed by the Mastocytosis Quality of Life Score).
- AYVAKIT had a favorable safety and tolerability profile, with
patients receiving up to four-plus years of treatment. The most
common treatment-related adverse event (AE) was edema, with the
majority reported as Grade 1, and the rate of treatment-related AEs
leading to discontinuations remained low (3 percent).
PATHFINDER: With a median follow-up of more than three years,
the median overall survival (OS) for AYVAKIT has not been reached
in patients with advanced SM.
- The median OS has not yet been reached regardless of disease
subtype or prior therapy, reflecting the durable clinical benefits
of AYVAKIT. In advanced SM, the historical median OS has ranged
from less than six months to about 3.5 years.1
- The overall response rate2 was 73 percent, and the
rate of complete remission with full or partial hematologic
recovery was 29 percent.
- AYVAKIT had a favorable benefit/risk profile consistent with
previously reported data. Common treatment-related AEs were
periorbital edema, thrombocytopenia, peripheral edema and
anemia.
PRISM: The largest European study to evaluate patient
perspectives in SM reinforces the significant burden of disease,
highlighting the urgency to treat.
- The Perceptions, Realities & Insights on SM (PRISM) Survey
– a collaborative research project led by Blueprint Medicines and
involving disease experts and patient organizations across seven
countries – further illustrates the substantial impact of SM on
patients' quality of life.
- Patients reported debilitating symptoms, impaired physical and
mental health functioning, meaningful impacts on the ability to
work, and significant polypharmacy use.
- Healthcare providers broadly recognized the burden of SM; 65
percent reported the disease affected patients' lives "quite a bit"
or "a great deal," and 58 percent reported patients lost employment
opportunities.
At the start of their respective oral and poster sessions, data
presentations will be made available in the "Science―Publications
and Presentations" section of Blueprint Medicines' website.
EAACI Congress 2024
- Oral Presentation: Safety of Avapritinib in Indolent Systemic
Mastocytosis (ISM): Longer Term Follow-up from the PIONEER Study
(Abstract 000223)
- Oral Presentation: The Burden of Indolent Systemic Mastocytosis
in Europe: Results from the PRISM
Patient Survey (Abstract 000405)
- Oral Presentation: Identifying KIT D816V Mutation in Patients
with Evidence of Systemic Mast Cell Activation (MCA) and Enriched
for Hereditary Alpha-Typtasemia (HaT): Results from the PROSPECTOR
Clinical Trial (Abstract 000403)
- Poster Presentation: Healthcare Provider Perspectives on
Management of European Patients with Systemic Mastocytosis
(Abstract 000413)
EHA2024 Congress
- Oral Presentation: Avapritinib in Patients with Advanced
Systemic Mastocytosis (AdvSM): Efficacy and Safety Analysis from
the Phase 2 PATHFINDER Study with 3-Year Follow-up (Abstract
S224)
- Poster Presentation: The Burden of Systemic Mastocytosis in
Europe: Results from the PRISM
Patient Survey (Abstract P1676)
- ePoster Presentation: Patient Diagnostic Journey of Systemic
Mastocytosis in Europe: Results
from the PRISM Survey (Abstract P2292)
About Systemic Mastocytosis
Systemic mastocytosis (SM) is a rare disease driven by the KIT
D816V mutation in about 95 percent of cases. Uncontrolled
proliferation and activation of mast cells result in chronic,
severe and often unpredictable symptoms across multiple organ
systems. The vast majority of those affected have indolent systemic
mastocytosis (ISM). A broad range of symptoms, including
anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog,
fatigue and bone pain, frequently persist in patients with ISM
despite treatment with multiple symptom-directed therapies. This
burden of disease can lead to a profound, negative impact on
quality of life. Patients often live in fear of severe, unexpected
symptoms, have limited ability to work or perform daily activities,
and isolate themselves to protect against unpredictable triggers.
Until 2023, there were no approved therapies for the treatment of
ISM.
A minority of patients have advanced SM, which encompasses a
group of high-risk SM subtypes including aggressive SM (ASM), SM
with an associated hematological neoplasm (SM-AHN) and mast cell
leukemia (MCL). In addition to mast cell activation symptoms,
advanced SM is associated with organ damage due to mast cell
infiltration and poor survival.
About AYVAKIT (avapritinib)
AYVAKIT (avapritinib) is approved by the U.S. Food and Drug
Administration (FDA) for the treatment of three indications:
adults with ISM, adults with advanced SM, including ASM, SM-AHN and
MCL, and adults with unresectable or metastatic gastrointestinal
stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including
PDGFRA D842V mutations. Under the brand name AYVAKYT
(avapritinib), this medicine is approved by the European
Commission for the treatment of adults with ISM with moderate
to severe symptoms inadequately controlled on symptomatic
treatment, adults with ASM, SM-AHN and MCL, after at least one
systemic therapy, and adults with unresectable or metastatic GIST
harboring the PDGFRA D842V mutation. The therapy is not recommended
for the treatment of patients with low platelet counts (less than
50,000/µL).
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.
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 the 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, fully integrated
biopharmaceutical company that invents life-changing medicines. We
seek to alleviate human suffering by solving important medical
problems in two core focus areas: allergy/inflammation and
oncology/hematology. Our approach begins by targeting the root
causes of disease, using deep scientific knowledge in our core
focus areas and drug discovery expertise across multiple
therapeutic modalities. We have a track record of success with two
approved medicines, including
AYVAKIT®/AYVAKYT® (avapritinib) which we are
bringing to patients with systemic mastocytosis (SM) in the U.S.
and Europe. Leveraging our
established research, development, and commercial capability and
infrastructure, we now aim to significantly scale our impact by
advancing a broad pipeline of programs ranging from early science
to advanced clinical trials in mast cell diseases including SM 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 plans, strategies, timelines and expectations for
Blueprint Medicines' operations, including its expectations for our
current or future approved drugs and drug candidates; the potential
benefits of AYVAKIT/AYVAKYT or any of our current or future drug
candidates in treating patients; the strength of the
AYVAKIT/AYVAKYT launches in the U.S. and Europe; and Blueprint Medicines' ability to
establish AYVAKIT/AYVAKYT as the global standard of care for the
treatment of SM. The words "aim," "may," "will," "could," "would,"
"should," "expect," "plan," "anticipate," "intend," "believe,"
"estimate," "predict," "project," "potential," "opportunity,"
"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 delay of any current or planned
clinical trials or the development of the company's current or
future drug candidates; the company's 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
possibility that preclinical and clinical results for the company's
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 the possibility patient
enrollment rates may be delayed or slower than anticipated; the
actions of regulatory agencies and how this may affect the
initiation, timing and progress of clinical trials; the company's
ability to obtain, maintain and enforce patent and other
intellectual property protection for its products and current or
future drug candidates it is developing; and the success of the
company's 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 the company's 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, the company explicitly disclaims
any obligation to update any forward-looking statements contained
in this press release as a result of new information, future events
or otherwise. Accordingly, readers are cautioned not to place undue
reliance on these forward-looking statements.
Footnotes
1 Sperr WR, Kundi M, Alvarez-Twose I, et al.
International prognostic scoring system for mastocytosis (IPSM): a
retrospective cohort study. Lancet Haematol.
2019;6(12):e638-e649.
2 Treatment response was defined as complete
remission with full or partial hematologic recovery, partial
remission or clinical improvement.
Trademarks
Blueprint Medicines, AYVAKIT, AYVAKYT and associated logos are
trademarks of Blueprint Medicines Corporation.
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