enzomenib

Enzomenib is an investigational, orally bioavailable small molecule inhibitor of menin, developed for the treatment of acute leukemia. It specifically targets and binds to the nuclear protein menin, thereby preventing its interaction with mixed-lineage leukemia protein (KMT2A). This inhibition disrupts the formation of the menin–MLL complex, which is essential for leukemogenesis in certain subtypes of acute myeloid leukemia (AML), particularly those with KMT2A rearrangements or NPM1 mutations. By blocking this interaction, enzomenib downregulates oncogenic gene expression (such as HOXA9 and MEIS1), inhibits proliferation of leukemic cells, and promotes differentiation. Enzomenib has received Orphan Drug Designation from both the FDA and Japan’s PMDA for AML indications and Fast Track Designation from the FDA for relapsed or refractory AML with MLLr or NPM1m[1][3][4][5][7][8].

Other names

enzomenib [INN]2412555-70-3