On September 30, 2026 Taiho Oncology, Inc., and Taiho Pharmaceutical Co., Ltd., reported positive topline results from the Phase 3 AZTOUND trial evaluating pharmacokinetic (PK) exposure, safety and efficacy of an orally available azacitidine and cedazuridine (ASTX030) in patients with myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML). The study met its primary endpoint of total cycle area under the curve (AUC) from 0 to 24 hours (AUC0-24) exposures, measured as the ratio of azacitidine total cycle AUC0-24 exposures following oral azacitidine and cedazuridine compared with subcutaneous azacitidine. The safety profile was consistent with parenteral azacitidine.
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These results from AZTOUND will be submitted for presentation at an international medical conference. Based on these results, Taiho Oncology intends to submit a New Drug Application to the U.S. Food and Drug Administration (FDA) seeking approval of azacitidine and cedazuridine as an oral treatment regimen for adults with MDS or CMML.
"The positive topline results of the Phase 3 AZTOUND trial support the potential of an all-oral regimen of azacitidine and cedazuridine to meet the treatment needs of patients with MDS and CMML without the time and treatment burden associated with parenteral therapies," said Harold Keer, MD, PhD, Chief Medical Officer, Taiho Oncology. "We look forward to pursuing regulatory approval of oral azacitidine and cedazuridine to potentially bring this treatment option to patients."
"We believe the positive topline results of the AZTOUND Phase 3 trial mark a key milestone in our collective efforts to develop a portfolio of novel treatments that improve clinical outcomes and quality of life for people with hematological malignancies," said Fabio Benedetti, MD, Global Chief Medical Officer, Taiho Pharmaceutical. "We are grateful to the patients, caregivers and investigators participating in the AZTOUND trial and remain committed to advancing azacitidine and cedazuridine for patients with MDS and CMML."
About the AZTOUND Trial
Phase 3 of the AZTOUND trial is a randomized open-label crossover study that enrolled 88 adults with myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML). The primary objective was to assess total cycle area under the curve (AUC) from 0 to 24 hours (AUC0-24) exposures, measured as the ratio of azacitidine total cycle AUC0-24 exposures after oral azacitidine and cedazuridine over subcutaneous azacitidine.
About Oral Azacitidine and Cedazuridine
Azacitidine and cedazuridine (ASTX030) is an oral combination of azacitidine, a DNA methyltransferase inhibitor, and cedazuridine, a cytidine deaminase inhibitor added to help azacitidine stay active in the body without being degraded. Azacitidine and cedazuridine (ASTX030) is investigational and has not been approved by any health authority. Azacitidine and cedazuridine is being developed by Taiho Oncology, Inc. and its parent company, Taiho Pharmaceutical Co., Ltd.
About Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML)
Myelodysplastic syndromes (MDS) are a heterogeneous group of hematopoietic stem cell disorders. Each year, there are approximately 10,000 to 15,000 new cases in the U.S., though the disease is believed to be underdiagnosed.1,2,3 Approximately one-third of these cases may progress to acute myeloid leukemia.4,5 Chronic myelomonocytic leukemia (CMML) is a clonal hematopoietic malignancy characterized by accumulation of abnormal monocytes in the bone marrow and in blood. Approximately 2,000 new cases of CMML are diagnosed annually in the U.S. 6,7 Between 15% and 30% of CMML cases may progress to acute myeloid leukemia.
(Press release, Taiho, SEP 30, 2026, View Source [SID1234671197])