Blueprint Medicines highlights 'decade of leadership' in Mast Cell research with new AYVAKIT and BLU-808 data at AAAAI/WAO 2025.

-- Updated data show AYVAKIT® (avapritinib) was safe and effective for long-term treatment of ISM, with sustained symptom and quality-of-life benefit and no new safety signals with multiple years of therapy ---- AYVAKIT-treated patients achieved improvements in bone health based on an analysis of bone mineral density scans, underscoring the importance of treating patients early in the ISM disease course ---- BLU-808 healthy volunteer trial results demonstrate wide therapeutic window for modulating mast cell activity, affirming potential as best- and first-in-class oral wild-type KIT inhibitor --CAMBRIDGE, Mass., Feb. 28, 2025 /PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC) today announced data presentations reflecting its ongoing efforts to innovate the treatment of mast cell-driven diseases. The presentations include three-year median follow-up data from the registrational PIONEER trial demonstrating the strong long-term efficacy and safety of AYVAKIT® (avapritinib) in patients with indolent systemic mastocytosis (ISM), and real-world evidence further characterizing the substantial burden of the disease, highlighting the urgency to treat. In addition, the company will report positive data from the Phase 1 healthy volunteer trial evaluating BLU-808, an investigational, highly potent and selective oral inhibitor of wild-type KIT, consistent with top-line data previously announced in January 2025. A total of 14 data presentations, including two oral presentations, will be reported at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI) / World Allergy Organization (WAO) Joint Congress being held February 28-March 3 in San Diego."We are incredibly proud to showcase our mast cell therapy portfolio at AAAAI / WAO, with 14 presentations highlighting more than a decade of leadership and collaboration with the mast cell disease community, including renowned medical institutions pioneering innovative research and multi-disciplinary patient care," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "In ISM, new data have reinforced that AYVAKIT was very well-tolerated after multiple years of treatment, with a low discontinuation rate and safety results that have remained consistent with the safety profile from the placebo-controlled study as summarized in the FDA-approval label

Furthermore, Blueprint Medicines announced the appointment of Sherwin Sattarzadeh as Chief Business Officer, succeeding Helen Ho.