BY002

BY002 is an investigational, orally bioavailable small molecule menin inhibitor. It is specifically designed to target the interaction between menin and the histone-lysine N-methyltransferase 2A (KMT2A, formerly MLL) protein. This interaction is a critical driver in certain subsets of acute leukemia, particularly those with KMT2A rearrangements or NPM1 mutations, where it leads to the constitutive expression of pro-leukemogenic genes like HOXA9 and MEIS1. By disrupting this complex, BY002 aims to reverse the epigenetic program of the leukemic cells, inducing differentiation and apoptosis. The drug is currently undergoing Phase 1 clinical evaluation in an investigator-initiated trial for patients with relapsed or refractory acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and mixed phenotype acute leukemia (MPAL).