Blueprint Medicines, a Sanofi company, announced AYVAKIT®? (avapritinib) data showing clinically meaningful outcomes and a well-tolerated long-term safety profile in patients with indolent systemic mastocytosis (ISM). Key presentations include four-year PIONEER clinical study results highlighting sustained symptom control and quality of life improvement in AYVAKIT-treated patients, and real-world data showing meaningful symptom benefit in patients receiving the therapy in a community practice setting.

In addition, a study conducted with the Advanced Practitioner Society for Hematology and Oncology (APSHO) and The Mast Cell Disease Society (TMS) reinforces the severe symptoms and impaired quality of life experienced by patients with ISM. Among patients who initiated the 25 mg daily dose of AYVAKIT (n=16), 87.5% have remained on therapy, reflecting a well-tolerated safety profile. An additional presentation features a systematic literature review of AYVAKIT data, including results from the real-world AVATAR study in patients with uncontrolled ISM (N=17).

In this study, the median MC-QoL score decreased from 61 (baseline) to 17 (24 weeks), representing a 76% improvement. Avapritinib continues to Demonstrate Durable Symptom Control with a Well-Tolerated Safety Profile: Long-Term Outcomes from PIONEER (Abstract 509 - Saturday, February 28), Real-World Experience of Patients with Indolent Systemic Mastocytosis Treated with Avapritinib in a Community Oncology setting (Abstract 516 - Saturday, February 28); Avapritinib Durably Improves Gastrointestinal Symptoms of Indolent Systemic Mastocosis: Long-Term Bone Health Outcomes in Patients Treated with Avaprit Inib for Indolent Systemicastocytosis: Findings from the PIONEER Study (Abstract 530 - Saturday, February 28); A Systematic literature Review of Avapritinib Outcomes in Indolent Systemic Mastomatosis (ISM) Patients (Abstract 529 - Saturday, February 28); Understanding Disease Control: What Shapes Patient Experience in Indolent Systemicmastocytosis (Abstract 514 - Saturday, February 28); Systemic Mastocytosis: Five-Year Mortality Comparison with Matched Reference Cohort Analysis (Abstract 533 - Saturday, February 28); Ultra-Sensitive SuperRCA Assay Enhances the ability to Detect KIT D816V Mutations in Peripheral Blood with a sensitivity Comparable to That of Bone Marrow Testing (Abstract 524 - Saturday, February 28); Are Tryptase Thresholds for Systemic Mastocytosis Adequate? Electronic Health Record (EHR) Data Weigh In (Abstract 527 - Saturday, February 28); Project BEACON: Developing a Predictive Model to Accelerate Diagnosis of Indolent Systemicastocosis (Abstract 517 - Saturday, February 28).