Clinical Pipeline

Phase 1Phase 2Phase 3

Oral Decitabine and Cedazuridine (ASTX727)

DNA methyltransferase (DNMT) inhibitor (Hematological Malignancies)


Ownership: Astex

Mechanism of Action: DNA methyltransferase (DNMT) inhibition

Indication: Hematological Malignancies

How this therapy can help

Oral decitabine and cedazuridine (ASTX727) is a unique fixed-dose combination of the hypomethylating agent decitabine, the active ingredient in Dacogen®, and the novel cytidine deaminase inhibitor, E7727 (cedazuridine).  Oral decitabine and cedazuridine was designed to deliver decitabine by oral administration. By inhibiting cytidine deaminase, cedazuridine inhibits the major mechanism by which decitabine is degraded in the gut and liver, and the combination therefore permits the efficient delivery of decitabine orally.

Clinical studies

Astex has completed a Phase 3 clinical study of oral decitabine and cedazuridine in treatment for adults with intermediate and high-risk myelodysplastic syndromes (MDS) including chronic myelomonocytic leukemia (CMML), the ASCERTAIN study. This study was a multicenter, randomized, open-label, crossover PK study of oral decitabine and cedazuridine versus IV decitabine. The ASCERTAIN study has been expanded to include enrollment of acute myeloid leukemia (AML) patients in the European Union (EU).

Astex is developing a low-dose formulation of oral decitabine and cedazuridine for the treatment of chronic conditions where oral delivery of a hypomethylating agent would be preferred. These include lower-risk MDS, and in AML maintenance following intensive chemotherapy.

Oral decitabine and cedazuridine  is also being studied in combination with tolinapant (ASTX660) in the treatment of AML.

View all clinical studies with oral decitabine and cedazuridine (ASTX727)

View Presentations and Publications