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BT01002 is a second-generation small molecule Menin inhibitor developed by Beyang Therapeutics, an AI-driven drug discovery company. It is designed to target the protein-protein interaction between Menin (encoded by the MEN1 gene) and KMT2A (formerly MLL), which is a critical driver in specific subsets of acute leukemia. BT01002 is intended for the treatment of acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL) harboring KMT2A rearrangements (KMT2Ar) or NPM1 mutations. A key differentiator of BT01002 is its molecular design, which is reported to overcome acquired resistance mechanisms, such as mutations in the Menin binding pocket, that limit the efficacy of first-generation Menin inhibitors. As of mid-2026, the drug is in early-stage development.
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