CAMBRIDGE, Mass. - Blueprint Medicines (NASDAQ:BPMC) Corporation (NASDAQ:BPMC) has reported encouraging long-term results from the PIONEER trial of AYVAKIT (avapritinib) in patients with indolent systemic mastocytosis (ISM), as well as promising preclinical data for BLU-808, a potent oral KIT inhibitor. These findings were presented at the 2024 American Academy of Allergy, Asthma & Immunology Annual Meeting.
The PIONEER trial data revealed that AYVAKIT produced significant and sustained improvements in symptoms across various domains such as skin, gastrointestinal, and neurocognitive over 48 weeks. The safety profile was consistent with earlier results, showing no new safety signals and most adverse events being mild or moderate. The treatment also led to a reduction or discontinuation of other medications in 35% of patients after 48 weeks.
BLU-808, targeting wild-type KIT, demonstrated a high degree of selectivity and potency in preclinical studies, with potential implications for chronic urticaria and other mast cell diseases. Blueprint Medicines plans to submit an investigational new drug application for BLU-808 in the second quarter of 2024 and initiate a Phase 1 study.
The company's commitment to advancing treatments for mast cell disorders is underscored by nine data presentations at the AAAAI meeting. Blueprint Medicines is working towards establishing AYVAKIT as a new standard of care for ISM and expanding its reach with potential treatments like BLU-808.
Systemic mastocytosis is a rare condition characterized by the proliferation and activation of mast cells, leading to a range of severe symptoms. AYVAKIT is an FDA-approved treatment for ISM, advanced SM, and certain types of gastrointestinal stromal tumor (GIST).
Blueprint Medicines is a biopharmaceutical company focused on developing medicines for allergy/inflammation and oncology/hematology. The company's approach involves targeting known drivers of disease with precision therapies.
This news is based on a press release statement from Blueprint Medicines Corporation.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.