Taiho Oncology, Inc. and Taiho Pharmaceutical Co., Ltd. have released positive topline results from a phase 3 trial testing ASTX030 in patients with either myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML).
ASTX030 is orally-available azacitidine and cedazuridine. Azacitidine (currently approved for subcutaneous administration) is a DNA methyltransferase inhibitor. Cedazuridine is a cytidine deaminase inhibitor that prevents the degradation of azacitidine and enables it to remain active in the body.
The phase 3 AZTDOUND trial evaluated pharmacokinetic exposure, safety, and efficacy of ASTX030. It met its primary endpoint of total cycle area under the curve from 0 to 24 hours (AUC0-24) exposures. The study compared the ratio of azacitidine total cycle AUC0-24 exposures in patients taking the drug to those receiving subcutaneous azacitidine. The study also found a safety profile consistent with parenteral azacitidine.