bleximenib

Bleximenib is a novel, orally bioavailable small molecule that acts as a potent and selective inhibitor of the menin-KMT2A (also known as MLL) protein-protein interaction. This interaction is critical for the maintenance of aberrant gene expression in leukemias with KMT2A rearrangements or NPM1 mutations. By disrupting this complex, bleximenib downregulates key stemness genes such as HOX/Meis1 and activates differentiation genes in leukemic cells, leading to antiproliferative effects and induction of differentiation. Preclinical studies have shown robust efficacy against acute myeloid leukemia (AML) and B-cell acute lymphoblastic leukemia (B-ALL) models harboring these genetic alterations. Bleximenib has also demonstrated activity in patient samples with other mutations such as CEBPA or NUP98 rearrangements, suggesting broader potential utility within hematologic malignancies. The drug is currently being investigated in clinical trials for relapsed/refractory AML and newly diagnosed AML patients eligible or ineligible for intensive chemotherapy[3][4][5][6][7][8].

Other names

bleximenibJNJ-75276617JNJ75276617JNJ 75276617JNJ-6617JNJ6617JNJ 6617