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ELVN-001 is a potent, highly selective small molecule kinase inhibitor designed to specifically target the BCR-ABL fusion gene product, which is the oncogenic driver in chronic myeloid leukemia (CML). It binds to the ATP-binding site of the ABL1 kinase domain and stabilizes a unique "folded-in" active conformation of ABL1, resulting in high selectivity against other kinases. ELVN-001 was engineered to have activity against the T315I mutation—the most common BCR-ABL mutation conferring resistance to nearly all approved tyrosine kinase inhibitors (TKIs)—as well as mutations that confer resistance to allosteric BCR-ABL inhibitors. The drug is being developed as an oral therapy for patients with CML who are relapsed, refractory, or intolerant to TKIs. It is currently being evaluated in Phase 1 clinical trials and has shown promising safety and efficacy data[1][5][6][7].
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