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TERN-701 is an investigational, oral, potent small molecule allosteric inhibitor of BCR-ABL tyrosine kinase, specifically targeting the ABL1 myristoyl pocket. It is being developed for the treatment of chronic myeloid leukemia (CML), particularly in patients who have relapsed or are refractory to prior therapies including second-generation and third-generation tyrosine kinase inhibitors (TKIs) as well as asciminib. Unlike ATP-competitive TKIs, TERN-701 binds to a non-active site on the BCR-ABL protein and induces conformational changes that inhibit its activity. Preclinical data show high selectivity and antiproliferative potency against both native and mutant CML cell lines, including those with the T315I mutation. Early clinical results from phase 1 trials demonstrate significant molecular responses and a strong safety profile in heavily pre-treated CML patients[1][2][3][5][6][7][8].
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