Clinical Pipeline

Phase 1Phase 2Phase 3

Azacitidine and Cedazuridine (ASTX030)

Oral DNMT inhibitor (Hematological Malignancies)


Ownership: Astex

Mechanism of Action: DNA methyltransferase (DNMT) inhibition

Indications: Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), or Acute Myeloid Leukemia (AML)

How this therapy can help

ASTX030 is an investigational oral formulation of azacitidine + cedazuridine designed to enhance systemic bioavailability of azacitidine delivered orally to the level achieved with parenteral administration of azacitidine alone. Cedazuridine is a cytidine deaminase inhibitor and inhibits azacitidine inactivation in the gut and liver.

Clinical status

ASTX030 is being evaluated in a first-in-human Phase 1-3 clinical study in patients with MDS, CMML, other myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN), or AML.

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