Blueprint Medicines Corporation presented new data at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, held from December 7-10, 2024. The presentations reinforced the long-term clinical benefits of AYVAKIT (avapritinib) in advanced systemic mastocytosis (SM). Data highlighted survival benefits of front-line AYVAKIT use in patients with advanced SM.
Analyses of bone density in patients with advanced SM underscored the disease-modifying effects of AYVAKIT, indicating its impact beyond symptom control. Additionally, ultra-sensitive KIT testing in peripheral blood identified previously undetected KIT mutations in some PIONEER trial patients, suggesting a potentially higher prevalence of SM.
The company also reported on a predictive model developed using machine learning techniques to distinguish between advanced SM and indolent SM (ISM). These findings support Blueprint Medicines' ongoing efforts to improve SM diagnosis and treatment. The HARBOR trial of elenestinib, a next-generation KIT D816V inhibitor, is designed to rigorously assess disease-modifying impacts, including bone health and recurrent anaphylaxis.
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