APTO-253

APTO-253 is a clinical-stage small molecule anticancer agent developed by Aptose. It functions primarily as an inhibitor of the MYC oncogene by targeting and stabilizing G-quadruplex DNA structures in the promoter region of MYC, leading to downregulation of MYC expression. This results in cell cycle arrest and apoptosis in both hematologic malignancies (such as acute myeloid leukemia) and solid tumors. The drug also induces the tumor suppressor Krüppel-like factor 4 (KLF4). Notably, its mechanism does not cause myelosuppression, distinguishing it from other chemotherapies. Preclinical studies have shown activity against a range of cancer cell lines and synergy with other therapies for AML or myelodysplastic syndromes. The active intracellular form is an iron complex [Fe(253)3]. Clinical trials have included patients with solid tumors and hematologic cancers, including those with BRCA1/2 mutations[1][2][4][5][6].

Other names

APTO-253 hydrochlorideAPTO253 hydrochlorideAPTO 253 hydrochloride