APR-246 + venetoclax

APR-246 + venetoclax is an investigational combination therapy being studied primarily in patients with acute myeloid leukemia (AML) harboring TP53 mutations. APR-246 (also known as eprenetapopt) is a small molecule that reactivates mutant p53 protein by restoring its wild-type conformation and function, thereby promoting tumor cell apoptosis. Venetoclax is a selective BCL-2 inhibitor that induces apoptosis in cancer cells dependent on BCL-2 for survival. The combination aims to synergistically induce apoptosis in TP53-mutant myeloid malignancies, including AML, with clinical studies evaluating safety, recommended dosing, and preliminary efficacy. Development is most advanced in frontline and relapsed/refractory AML with TP53 mutation. Notably, some studies include azacitidine in triple regimens, but APR-246 + venetoclax alone is also investigated[1][3][7].

Brand names

Venclexta

Other names

APR-246 + venetoclaxeprenetapopt + venetoclax