NTX-301

NTX-301 is an orally administered, small molecule hypomethylating agent that acts as a selective inhibitor of DNA methyltransferase 1 (DNMT1). By binding to and inhibiting DNMT1, NTX-301 prevents DNA methylation, induces DNA hypomethylation, and reactivates tumor suppressor genes silenced by hypermethylation. This leads to inhibition of tumor cell proliferation and induction of apoptosis. Preclinical studies have shown that NTX-301 exhibits potent anti-leukemia activity—more effective than existing agents like 5-azacytidine—and is also active in various solid cancer models. It has demonstrated efficacy both as monotherapy and in combination with venetoclax, particularly in acute myeloid leukemia (AML), including resistant subtypes. The drug is being developed primarily for hematologic malignancies such as AML, myelodysplastic syndromes (MDS), and chronic myelomonocytic leukemia (CMML), with additional investigation in certain solid tumors[1][2][3][4][5][6][8]. Developed and manufactured by Xennials Therapeutics Australia.

Other names

DNMT1 inhibitor NTX-301DNMT-1 inhibitor NTX-301DNMT 1 inhibitor NTX-301