zoftomenib

Zoftomenib (KO-539) is an orally bioavailable, potent, and selective small-molecule inhibitor of the Menin-KMT2A (formerly MLL) protein-protein interaction. Developed by Kura Oncology, it is primarily being investigated for the treatment of genetically defined subsets of acute myeloid leukemia (AML), specifically those harboring KMT2A rearrangements or NPM1 mutations. By blocking the interaction between Menin and KMT2A, zoftomenib disrupts the expression of leukemogenic genes such as HOXA9 and MEIS1, promoting cell cycle arrest and differentiation of leukemic blasts. Beyond hematologic malignancies, zoftomenib is also being explored in combination with imatinib for the treatment of gastrointestinal stromal tumors (GIST), where Menin inhibition may help overcome resistance to KIT inhibitors.

Other names

zoftomenib