cedazuridine/azacitidine combination agent ASTX030

An orally available fixed-dose combination agent containing cedazuridine, a cytidine deaminase (CDA) inhibitor, and the cytidine antimetabolite azacitidine, with potential antineoplastic activity. Upon oral administration of the cedazuridine/azacitidine combination agent ASTX030, cedazuridine binds to and inhibits CDA, an enzyme primarily found in the gastrointestinal (GI) tract and liver that catalyzes the deamination of cytidine and cytidine analogs. This prevents the breakdown of azacitidine, increases its bioavailability and efficacy while decreasing GI toxicity due to the administration of lower doses of azacitidine. Azacitidine exerts its antineoplastic activity through the incorporation of its triphosphate form into DNA, which inhibits DNA methyltransferase (DNMT), thereby blocking DNA methylation and results in hypomethylation of DNA. This interferes with DNA replication and decreases tumor cell growth.