Blueprint Medicines Corporation presented compelling new data at the 66th American Society of Hematology (ASH) Annual Meeting, showcasing the long-term benefits of AYVAKIT® (avapritinib) in treating systemic mastocytosis (SM). These findings reflect the company’s commitment to transforming patient care through innovative therapies and deeper collaboration with the SM community.
Advancing the Standard of Care in Systemic Mastocytosis
Systemic mastocytosis is a complex disease characterized by mast cell infiltration, chronic inflammation, unpredictable symptoms, and severe comorbidities such as osteoporosis. Early intervention is crucial to managing its progression.
“AYVAKIT has revolutionized the treatment of systemic mastocytosis by addressing the disease at its root, offering prolonged survival for advanced SM patients and durable symptom control for those with indolent SM (ISM),” said Dr. Becker Hewes, Chief Medical Officer at Blueprint Medicines. “Building on this success, our HARBOR trial of elenestinib, a next-generation KIT D816V inhibitor, will assess disease-modifying impacts across a broad range of endpoints.”
Key Data Highlights
- Survival and Bone Density Improvements
- In pooled analyses from the PATHFINDER and EXPLORER trials, treatment-naïve advanced SM patients using AYVAKIT showed significant survival benefits compared to real-world outcomes with midostaurin.
- PATHFINDER trial results revealed sustained improvements in bone density for advanced SM patients with baseline low bone mass.
- Chronic Inflammation and Immune Dysregulation in ISM
- Data from the PIONEER trial highlighted significant immune dysregulation in ISM patients compared to healthy individuals, underscoring the chronic inflammatory burden of the disease.
- Breakthroughs in Detection with Ultra-Sensitive KIT Testing
- A novel, ultra-sensitive KIT assay detected previously undetectable KIT mutations in PIONEER trial patients. This innovative tool suggests SM prevalence may be higher than currently estimated.
- Predictive Models for SM Diagnosis
- Using machine learning, a predictive model was developed to distinguish advanced SM from ISM based on blood-based parameters. The model’s accuracy was validated with data from the Dana-Farber Cancer Institute.
Impactful Presentations at ASH 2024
Blueprint Medicines shared its findings through an oral presentation and three posters, including:
- Oral Presentation: Predictive model distinguishing advanced SM from ISM using blood-based data.
- Poster Presentations:
- Comparative survival analysis of AYVAKIT versus midostaurin.
- Ultra-sensitive KIT testing uncovering new mutations.
- Disease-modifying effects of AYVAKIT on bone density.
