Cedazuridine (E7727) (Compound 7a) hydrochloride, an orally active cytidine deaminase (CDA) inhibitor with an IC50 of 0.4 μM, can be used for cancer research [1].

Cedazuridine (Compound 7a) demonstrates superior acid stability [1], and at concentrations of 0-10 μM over 72 hours, it does not enhance the growth inhibition effects of AZA (5-Azacytidine) in AML cell lines [2].

Cedazuridine, when administered orally at a dose of 3 mg/kg daily for 7 days, in conjunction with 2.5 mg/kg AZA, induced tumor regression in both MOLM-13 cell line-derived (CDX) and patient-derived xenograft (PDX) mouse models of leukemia. Female NSGS mice, aged 6–8 weeks, were used for these experiments, demonstrating not only a reduction in leukemic expansion in synergy with AZA in a CDX setup but also showcasing preliminary safety and efficacy in a primary AML PDX scenario. Further investigations in NSGS male mice, with Cedazuridine doses ranging from 1 to 30 mg/kg combined with 2.5 mg/kg AZA, revealed a dose-dependent increase in the bioavailability (AUC) of orally administered AZA, compared to its standard intraperitoneal administration, suggesting enhanced pharmacokinetic performance.