BTX-A51 + azacitidine

BTX-A51 + azacitidine is an investigational combination therapy under clinical evaluation for relapsed or refractory acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS)[1][2][3][5][6]. **BTX-A51** is a first-in-class oral small molecule inhibitor that directly targets casein kinase 1α (CK1α), cyclin-dependent kinase 7 (CDK7) and cyclin-dependent kinase 9 (CDK9), inducing apoptosis in leukemic cells through p53 activation and MCL1 suppression. **Azacitidine** is a hypomethylating agent that blocks the growth of cancer cells and is widely used in AML and MDS. Preclinical and phase 1/2 data indicate synergy between BTX-A51 and azacitidine, enabling improved suppression of leukemic blast expansion and thus providing scientific rationale for this combination[1][3][5]. Trials are ongoing to determine optimal dosing, safety and efficacy in AML and MDS[2][3][6]. Edgewood Oncology is currently developing BTX-A51 and leading combination studies[3].