Blueprint Medicines Corporation announced new data at the 66th American Society of Hematology (ASH) Annual Meeting demonstrating the long-term clinical benefits of AYVAKIT® (avapritinib) in advanced systemic mastocytosis (SM). The data reinforce the survival benefits of front-line avapritinib use and highlight its disease-modifying effects, including improvements in bone density.
Survival Benefits in Advanced SM
Pooled analyses from the PATHFINDER and EXPLORER trials revealed that treatment-naïve patients with advanced SM experienced significant survival benefits with avapritinib compared to real-world outcomes with midostaurin. This suggests that early intervention with avapritinib can improve outcomes for patients with this rare and aggressive disease.
Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines, stated, "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."
Improvements in Bone Density
Data from the PATHFINDER trial also demonstrated that avapritinib led to sustained improvements in bone density for advanced SM patients who had low bone mass at baseline. This is particularly significant as systemic mastocytosis is often associated with comorbidities such as osteoporosis.
Enhanced KIT Mutation Detection
ASH data highlighted that ultra-sensitive KIT testing in peripheral blood, a novel tool in clinical development, identified previously undetected KIT mutations in PIONEER trial patients. This suggests that systemic mastocytosis may be more prevalent than previously thought, with current commercially available methods potentially underdiagnosing the condition.
Predictive Modeling for SM Subtypes
An additional data presentation showcased the application of machine learning techniques to analyze baseline, blood-based parameters of Blueprint Medicines' clinical trial participants. The resulting predictive model accurately distinguishes between advanced SM and ISM, with validation from an independent dataset from Dana-Farber Cancer Institute. This could lead to improved diagnostic accuracy and more tailored treatment strategies.
Ongoing Research and Future Directions
Blueprint Medicines is continuing its research in systemic mastocytosis with the HARBOR trial of elenestinib, a next-generation KIT D816V inhibitor. This trial aims to rigorously assess a broad range of endpoints reflecting disease-modifying impact. The company's collaborative efforts with clinical experts aim to improve and accelerate the diagnosis and treatment of systemic mastocytosis.
