Taiho Oncology and Taiho Pharmaceutical said their experimental oral combination of azacitidine and cedazuridine met the primary endpoint in a Phase 3 trial involving adults with myelodysplastic syndromes or chronic myelomonocytic leukemia.
The AZTOUND study compared patients’ exposure to azacitidine after taking the oral combination, known as ASTX030, with exposure after azacitidine injections under the skin. Taiho said the trial met its measure of total drug exposure over a treatment cycle. It characterized the oral combination’s safety profile as consistent with injectable azacitidine.
The randomized, open-label crossover trial enrolled 88 adults. Its primary endpoint measured azacitidine exposure over 24 hours across a treatment cycle and compared the oral and injected regimens. The companies described the findings as topline results and plan to submit them for presentation at an international medical conference.
Taiho Oncology intends to file a new drug application with the FDA seeking approval of ASTX030 as an oral regimen for adults with either disease. The combination remains investigational and has not been approved by a health authority.
Azacitidine is a DNA methyltransferase inhibitor used in treating blood disorders. Cedazuridine inhibits an enzyme that can break down azacitidine, helping the drug remain active when taken by mouth.
Myelodysplastic syndromes are disorders involving abnormal blood cell production. Chronic myelomonocytic leukemia is another blood cancer affecting blood-forming cells. The proposed oral regimen is being developed as an alternative to treatment requiring injections.
The AZTOUND finding concerns drug exposure, the study’s primary measure. Taiho also evaluated safety and efficacy in the trial as it prepares its planned U.S. regulatory submission.
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