ZYSM002

ZYSM002 is a small molecule Menin inhibitor currently under development by Rongchang Pharmaceuticals for the treatment of Acute Myeloid Leukemia (AML) and Acute Lymphocytic Leukemia (ALL). The drug targets the Menin protein, which acts as a critical cofactor for the Mixed Lineage Leukemia (MLL/KMT2A) protein. In leukemias involving MLL rearrangements or NPM1 mutations, the Menin-MLL complex drives the expression of oncogenic genes like HOXA and MEIS1, which prevent hematopoietic differentiation and promote leukemogenesis. By inhibiting the Menin-MLL interaction, ZYSM002 aims to suppress these oncogenic pathways and induce cell cycle arrest and differentiation in leukemic blasts. As of early 2024, ZYSM002 is in the IND-enabling phase of development.