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Lisaftoclax + azacitidine is an investigational combination therapy evaluated for the treatment of myeloid malignancies, notably higher-risk myelodysplastic syndrome (HR-MDS) and acute myeloid leukemia (AML)[1][2][3][4][5][7]. Lisaftoclax is a potent, selective, oral small molecule inhibitor of B-cell lymphoma 2 (Bcl-2), a key regulator of apoptosis, while azacitidine is a hypomethylating agent that inhibits DNA methyltransferase, inducing DNA hypomethylation and promoting cell differentiation or apoptosis[1][2][3]. Preclinical and clinical data indicate the combination acts synergistically to induce apoptosis in malignant hematopoietic cells and is effective even in patients who are refractory to other Bcl-2 inhibitors such as venetoclax[1][2][3]. The regimen is being developed primarily by Ascentage Pharma and has entered global phase 3 trials as a first-line therapy for HR-MDS; it is also studied in relapsed/refractory AML and MDS populations[1][2][3][4][5][6][7].
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