DDP-38003

DDP-38003 is a **novel, orally available small molecule inhibitor** of **histone lysine-specific demethylase 1A (KDM1A/LSD1)**, with an IC50 of approximately 84 nM. It has been shown to induce differentiation and apoptotic cell death in acute myeloid leukemia (AML) cell lines, demonstrating antiproliferative and antitumor efficacy in preclinical studies, including increased survival in mouse leukemia models. DDP-38003 can trigger upregulation of LSD1-repressed genes, impact mTORC1 signaling, and shows enhanced anti-leukemia effects in combination with rapamycin. Mechanistically, it acts via inhibition of KDM1A/LSD1, and secondarily affects GSE1 functions by downregulating chromatin protein GSE1 to induce myeloid differentiation. It was initially developed by Istituto Europeo di Oncologia Srl, with evidence supporting its preclinical activity in neoplasms, particularly acute myeloid leukemia, and exploratory work in settings like glioblastoma and immunomodulatory combinations.

Other names

DDP-38003 trihydrochlorideDDP38003 trihydrochlorideDDP 38003 trihydrochlorideDDP-38003 dihydrochlorideDDP38003 dihydrochlorideDDP 38003 dihydrochloride