Date: 2015-11-12
Type of
information: Presentation of results at a congress
phase: preclinical
Announcement: presentation of results at the 57th American Society of Hematology (ASH) Annual Meeting in Orlando
Company: Blueprint Medicines (USA - MA)
Product: avapritinib (BLU-285)
Action
mechanism: kinase inhibitor.
Disease: acute myeloid leukemia (AML)
Therapeutic
area: Cancer - Oncology
Country:
Trial
details:
Latest
news:
- • On November 12, 2015, Blueprint Medicines announced that it will present new preclinical data demonstrating the potential utility of BLU-285 in a subset of patients with acute myeloid leukemia (AML) characterized by a mutation in KIT Exon 17 during an oral presentation at the 2015 American Society of Hematology (ASH) Annual Meeting and Exposition being held December 5-8 in Orlando, Fla. KIT Exon 17 mutations have been identified as drivers in multiple diseases, and the presence of these mutations greatly reduces the effective treatment options available to patients. BLU-285 is a potent and highly selective inhibitor of KIT Exon 17 and PDGFR? D842V mutants, which are drivers of disease in patients with gastrointestinal stromal tumors (GIST) and systemic mastocytosis (SM). In AML, a subset of patients with the t(8;21) translocation and an additional KIT Exon 17 mutation are at increased risk of recurrence compared to those without it, creating a strong need for new therapies to address their form of the disease.
- In preclinical results to be presented by Blueprint Medicines at ASH, BLU-285 showed potent inhibition of KIT activity in an AML cell line with the N822K mutation in Exon 17, including reduced phosphorylation, downstream signaling and cellular proliferation. BLU-285 administration in a xenograft model harboring AML cells driven by the KIT Exon 17 mutation resulted in a reduction of disease burden compared to cytarabine-treated animals. All BLU-285-dosed mice showed tumor regression, and several showed complete disease clearance.
Is
general: Yes
