A pill could soon replace the needle for patients with two serious blood cancers. Taiho Oncology announced that the oral combination of azacitidine and cedazuridine (ASTX030) met the primary endpoint of the phase 3 AZTOUND trial in adults with myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML). The drug hit its pharmacokinetic target — matching total azacitidine drug exposure (AUC0–24) compared to the standard subcutaneous (injectable) form — and showed a safety profile consistent with parenteral azacitidine.

The regimen works by pairing azacitidine, a DNA methyltransferase inhibitor, with cedazuridine, which prevents azacitidine from being broken down in the gut, making an all-oral version viable. The phase 3 portion enrolled 88 adults in a randomized, open-label crossover design. Taiho now plans to submit a New Drug Application (NDA) to the FDA, with full results to be presented at an upcoming international conference.

Key Takeaways

• The trial enrolled 88 adults with MDS or CMML in a randomized crossover design comparing oral ASTX030 vs. subcutaneous azacitidine
• No specific adverse event rates or efficacy data were disclosed in the topline announcement
• An FDA NDA submission is planned based on these results

Why it matters: For patients with MDS or CMML, current azacitidine treatment requires frequent clinic visits for injections. An approved oral regimen could significantly reduce treatment burden and improve quality of life — a meaningful shift in how these blood cancers are managed day-to-day.