Blueprint Medicines announces PIONEER trial results

Blueprint Medicines announced PIONEER trial results highlighting the long-term efficacy and safety of AYVAKIT in patients with indolent systemic mastocytosis, as well as foundational preclinical data for BLU-808, an investigational highly selective and potent oral inhibitor of wild-type KIT. Blueprint Medicines will report a total of nine data presentations, including two oral presentations, reflecting the company’s long-standing commitment to transform care for patients living with mast cell disorders. The datasets are being reported at the 2024 American Academy of Allergy, Asthma & Immunology Annual Meeting being held February 23-26 in Washington, D.C. expectations for continued growth of Caplyta, upcoming data readouts in MDD, and progress of its pipeline in the medium/long term. “PIONEER data show that long-term treatment with AYVAKIT led to robust and durable clinical efficacy across a wide range of symptoms, and a well-tolerated safety profile that has remained remarkably consistent over time,” said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. “These compelling long-term data, combined with the real-world experience of more than a thousand patients currently on therapy in the U.S., indicate our continued momentum in establishing AYVAKIT as a new standard of care for patients living with ISM. With an SM franchise anchored by AYVAKIT, and the opportunity to expand and extend our reach with elenestinib, Blueprint Medicines is transforming treatment across the spectrum of the disease and redefining what well-controlled means for patients.”