Drug intelligence / Profile preview

azacitidine + birinapant

Development stage
Unknown
Lead developer
Medivir
Modality
Small Molecules
Administration
Intravenous, Subcutaneous
01

Overview

azacitidine + birinapant is an investigational drug combination comprising azacitidine, a hypomethylating agent (DNA methyltransferase inhibitor), and birinapant, a second mitochondrial-derived activator of caspases (SMAC) mimetic, which acts as an antagonist of inhibitor of apoptosis proteins (IAPs). Azacitidine is FDA-approved for myelodysplastic syndromes (MDS) and acts by reversing DNA methylation. Birinapant promotes apoptosis by degrading cIAP1 and cIAP2, thereby modulating apoptosis pathways. In studies, the combination has shown synergistic antileukemic activity, particularly in acute myeloid leukemia (AML) and MDS, including in patients naïve, refractory, or relapsed to HMAs. The combination augments apoptosis in AML/MDS stem and progenitor cells, showing promising preclinical and early clinical efficacy, but remains experimental[1][2][4][5].

Other names
5-azacitidine + birinapant5-azacytidine + TL32711
02

Targets

DNMT3A (DNA methyltransferase 3 alpha)BIRC2 (Cellular inhibitor of apoptosis protein 1)DNMT3B (DNA (cytosine-5)-methyltransferase 3B)XIAPDNMT1 (DNA (cytosine-5)-methyltransferase 1)BIRC3 (Baculoviral IAP repeat-containing protein 3)

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