zefamenib

Zefamenib is an investigational oral **small-molecule menin inhibitor** being developed for genetically defined acute leukemias, particularly **acute myeloid leukemia** and other acute leukemias harboring **NPM1 mutation**, **KMT2A rearrangement**, and potentially **NUP98 rearrangement**. Public clinical-trial and meeting materials identify zefamenib with the development code **BN104** and describe it as a potent inhibitor of the menin pathway that disrupts oncogenic menin-driven transcriptional programs, including the HOXA/MEIS1 axis, to promote leukemic blast differentiation and anti-leukemic activity. The program has been studied as **monotherapy** in relapsed/refractory acute leukemia and is also being evaluated in **combination** with intensive chemotherapy or venetoclax plus azacitidine in AML. Development is associated with **Servier** and **BioNova Pharmaceuticals**, with clinical studies reported primarily in China.

Other names

zefamenib