Blueprint Medicines' Continued Leadership in Redefining the Standard of Care in Systemic Mastocytosis Highlighted at 2024 ASH Annual Meeting
-- ASH data reinforce survival benefits of front-line AYVAKIT® (avapritinib) use in patients with advanced SM --
-- Bone density analyses reported in patients with advanced SM underscore disease-modifying effects of AYVAKIT --
CAMBRIDGE, Mass., Dec. 7, 2024 /PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC) today announced data presentations that continue to demonstrate the long-term clinical benefits of AYVAKIT® (avapritinib) in advanced systemic mastocytosis (advanced SM), and reflect the company's ongoing partnership with the SM community to redefine the future of patient care – from improving diagnostic rates to raising the bar on treatment outcomes. The datasets, which include one oral presentation and three poster presentations, will be reported at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, taking place December 7-10 in San Diego.
"Systemic mastocytosis is associated with mast cell infiltration across multiple organ systems, chronic inflammation caused by immune dysregulation, unpredictable symptoms that may worsen over time, and serious comorbidities such as osteoporosis, highlighting the urgency to treat patients early in the course of their disease," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "AYVAKIT has fundamentally changed the treatment paradigm by targeting the disease at its source, showing prolonged survival outcomes for patients with advanced SM, as well as durable symptom control and quality-of-life improvements for patients with ISM. Based on the substantial datasets we have amassed over more than a decade, and our continued collaboration with the SM community, we have designed the HARBOR trial of elenestinib – a next-generation KIT D816V inhibitor – to rigorously assess a broad range of endpoints reflecting disease-modifying impact."
Data reported at ASH highlight the survival benefits of front-line AYVAKIT use in patients with advanced SM, and support Blueprint Medicines' plans to evaluate novel clinical measures indicative of disease modification – such as bone density and biomarkers of chronic inflammation – in the registrational HARBOR trial. Key results include:
- In pooled analyses from the PATHFINDER and EXPLORER trials, treatment-naïve patients with advanced SM showed significant survival benefits with AYVAKIT, when indirectly compared to real-world outcomes for midostaurin.
- In the PATHFINDER trial, AYVAKIT led to sustained improvements in bone density for advanced SM patients who had low bone mass at baseline.
- In the PIONEER trial, patients with indolent SM (ISM) had significant baseline levels of immune dysregulation relative to healthy donors, reflecting the chronic inflammatory burden of the disease.
Non-Invasive, Blood-Based Assay Identified KIT Mutations Not Previously Detectable by Existing Tests
ASH data show that ultra-sensitive KIT testing in the peripheral blood – a novel tool in clinical development that is more sensitive than current commercially available methods – identified previously undetected KIT mutations in a number of PIONEER trial patients. Emerging clinical research with ultra-sensitive KIT assays suggest SM may be more prevalent than previously thought.
An additional data presentation highlights the application of machine learning techniques to analyze baseline, blood-based parameters of Blueprint Medicines' clinical trial participants. Following these analyses, a predictive model was developed to distinguish between advanced SM and ISM, and its accuracy was validated by an independent dataset from Dana-Farber Cancer Institute.
Collectively, these data build on Blueprint Medicines' collaborative efforts with clinical experts to improve and accelerate how SM is diagnosed and treated.
Data presentations will be made available in the "Science―Publications and Presentations" section of Blueprint Medicines' website.
- Oral Presentation: Analysis of Avapritinib Clinical Trial Data Generates a Highly Accurate Predictive Model for Advanced Systemic Mastocytosis Versus Indolent Systemic Mastocytosis Based on Peripheral Blood Testing (Abstract 107 – Saturday, December 7)
- Poster Presentation: Overall Survival and Duration of Treatment in Patients with Advanced Systemic Mastocytosis Receiving Avapritinib Versus Midostaurin or Best Available Therapy in a Real-World Setting (Abstract 1801 – Saturday, December 7)
- Poster Presentation: Ultra-Sensitive KIT Testing Uncovers Previously Undetected KIT Mutations in Patients with Indolent Systemic Mastocytosis: Results from the PIONEER Trial (Abstract 3164 – Sunday, December 8)
- Poster Presentation: Disease-Modifying Effects of Avapritinib in Patients with Advanced Systemic Mastocytosis: Improvements in Bone Density (Abstract 4544 – Monday, December 9)
- Publication-Only Abstract: Blood-Based Proteomics for Deeper Insights into Indolent Systemic Mastocytosis: the PIONEER Trial Experience (Abstract 6569)
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.
IMPORTANT SAFETY INFORMATION
Intracranial Hemorrhage — Serious intracranial hemorrhage (ICH) may occur with AYVAKIT treatment; fatal events occurred in
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