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Five-Day Azacitidine Best Among Shorter Hypomethylating Agent Regimens for Low-Risk MDS

Data from Taiho Oncology's studies show five-day azacitidine is most effective among shorter hypomethylating-agent regimens for low-risk myelodysplastic syndromes.

  • At the ASH meeting in Orlando, Taiho Oncology, Inc. presented ASTX030-01 and ASTX727-03 data showing 5-day azacitidine produced superior survival with no new safety signals, announced Dec. 8, 2025.
  • Researchers designed the trial to compare 3-day decitabine and 3- and 5-day azacitidine regimens in 249 patients, including transfusion-dependent and transfusion-independent groups.
  • In the transfusion-independent cohort, investigators found 5-day azacitidine significantly improved EFS and yielded a higher overall response rate .
  • Compared with best supportive care, 5-day azacitidine showed better EFS and OS , but randomization ended early and investigators urged a phase 3 trial.
  • The results position azacitidine-cedazuridine to inform optimal doses and durations, while a key unresolved question is how 5-day azacitidine compares with luspatercept-aamt or imetelstat for future combination strategies and sequencing questions.
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Taiho Oncology Presents Data on All-Oral Regimens Azacitidine and Cedazuridine, and Decitabine and Cedazuridine at the 2025 American Society of Hematology Annual Meeting and Exposition

Two oral presentations will highlight Phase 2 results from the ASTX030-01 trial of oral azacitidine and cedazuridine in patients with myelodysplastic syndromes, chronic myelomonocytic leukemia or acute myeloid leukemia and Phase 2 results from ASTX727-03 trial of low-dose oral decitabine…

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Healio broke the news in on Monday, December 8, 2025.
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