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Taiho Oncology's Innovative Oral Cancer Treatments at ASH 2025

Taiho Oncology showcased pivotal findings from its recent studies on novel oral cancer therapies during the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, which took place from December 6-9, 2025, in Orlando, Florida. The company is focused on advancing treatments for hematological malignancies and solid tumors, emphasizing patient compliance and quality of life.

The presentations primarily focused on the Phase 2 results from two significant trials: ASTX030-01 and ASTX727-03. Both studies explored the effectiveness of oral azacitidine and cedazuridine, along with low-dose oral decitabine and cedazuridine, respectively, in patients diagnosed with myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), and acute myeloid leukemia (AML).

ASTX030-01 Trial Findings

The ASTX030-01 trial was a multicenter, randomized, open-label crossover study designed to compare the oral combination of azacitidine and cedazuridine against subcutaneous azacitidine.

As of the data cutoff in May 2025, a total of 30 patients participated, with 22 suffering from MDS, five from CMML, two from AML, and one from myelodysplastic/myeloproliferative neoplasms (MPN). Random assignments distributed patients into two groups to receive either ASTX030 or subcutaneous azacitidine during specific cycles.

The primary endpoint measured the geometric mean ratio (GMR) of drug exposure between the two formulations, which yielded a GMR of 0.913, indicating a comparable drug absorption. Among the MDS patients, the complete response (CR) rate stood at an encouraging 22.7%, with an overall response rate of 50%. Notably, patients who were reliant on red blood cell (RBC) transfusions showed significant improvement, with 30.8% achieving transfusion independence for over 56 days.

Safety and Tolerability

Despite the promising efficacy, adverse events (AEs) were reported in all patients, with a substantial percentage classified as grade 3 or higher. The most frequently occurring treatment-emergent AEs included nausea (70%), constipation (66.7%), and fatigue (60%).

ASTX727-03 Trial Insights

The other trial, ASTX727-03, involved patients with lower-risk MDS. It compared a low-dose all-oral regimen of decitabine and cedazuridine against a standard dose. As per the findings released as of October 2024, 81 patients participated, and there was a notable focus on those experiencing one or more cytopenias along with RBC transfusion dependence.

Patients in the low-dose (LD) arm received 10 mg of oral decitabine alongside 100 mg of cedazuridine, while the standard-dose (SD) group received 35 mg decitabine with 100 mg cedazuridine. The median overall survival (OS) for the LD arm was reported at 23.9 months compared to 26.0 months for the SD arm, showcasing comparable outcomes in terms of survival rates.

Improved Safety Profile

While both regimens resulted in reduced blood counts, the LD arm demonstrated more favorable outcomes with stable or improved blood counts across cycles as opposed to the SD group. Adverse events of grade 3 or higher were reported in 85% of patients within the LD cohort, contrasting with 90.2% in the SD group. Treatment discontinuation due to AEs was significantly lower in the LD arm (2.5%) compared to the SD arm (17.1%).

Commitment to Patient Care

Taiho Oncology’s Chief Medical Officer, Dr. Harold Keer, expressed satisfaction over the results, stating, "We are pleased to present positive data suggesting that our oral therapies may be comparable in safety and effectiveness to traditional parenteral treatments.” This approach recognizes the potential benefits of home-based therapy, which can significantly reduce treatment burdens for patients suffering from these severe conditions.

The company’s efforts in oral drug development underscore a holistic approach to patient care, aiming for treatments that not only maximize efficacy but also improve the overall treatment experience. Taiho Oncology continues to advocate for the advancement of innovative cancer therapies, with the goal of bettering the lives of cancer patients worldwide.

For further details and updates, please visit Taiho Oncology's website.