Venetoclax

Publication highlights positive results for the first all-oral decitabine-cedazuridine plus venetoclax regimen in newly diagnosed acute myeloid leukemia patients who are ineligible for intensive induction chemotherapy. PRINCETON, N.J.--(BUSINESS WIRE)--Taiho Oncology, Inc., a company developing and commercializing novel treatments for hematologic malignancies and solid tumors, today announced the publication in the New England Journal of Medicine of results from the ASCERTAIN-V Phase 1/2 clinical trial, which evaluated the first all-oral regimen of decitabine-cedazuridine plus venetoclax in patients with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive induction chemotherapy. The study demonstrated favorable response rates and survival with expected myelosuppressive effects. 1 Previously presented at the 2025 ASCO Annual Meeting and the 2025 European Hematology Association Congress, the results suggest this first all-oral regimen may provide outcomes consistent with existing hypomethylating agent–venetoclax–based approaches for patients with newly diagnosed AML who cannot receive intensive induction therapy. Nearly 23,000 people in the U.S. are expected to be diagnosed with AML, a cancer of the blood and bone marrow, in 2026. 2 More than half of those patients will likely be deemed ineligible for intensive induction chemotherapy because of factors such as older age (75 or older) or poor health. 3 For these patients, the standard treatment often combines oral venetoclax with parenteral hypomethylating agents, requiring frequent injections or infusions and an average of 12.9 days per month engaging in healthcare visits. 4 Oral decitabine-cedazuridine is a hypomethylating agent that has demonstrated oral bioavailability and equivalent pharmacokinetic exposure to intravenous decitabine. 5 “We are committed to advancing therapies that improve the lives of patients, their families and their caregivers,” said Harold Keer, MD, PhD, Chief Medical Officer, Taiho Oncology. “An all-oral regimen for AML could help reduce the significant time and cost burden associated with treatment in hospitals or infusion centers.” The ASCERTAIN-V trial evaluated the first all-oral regimen of oral decitabine-cedazuridine plus venetoclax in 189 patients (Phase 1, n=30; Phase 2a, n=58; Phase 2b, n=101). After an initial ramp-up period, patients were treated in 28-day cycles, receiving decitabine-cedazuridine on days one through five and venetoclax daily. Key results include complete response rates, durable remissions and survival, and a safety pro with hypomethylating agent-venetoclax regimens. In the Phase 2b of ASCERTAIN-V: The complete response rate was achieved in 47% of patients (95% CI: 36-57%). The composite of complete response or complete response with incomplete hematologic recovery was 63% (95% CI: 53-73%). The median time to marrow blasts of less than 5% was 28 days (range 22 to 57), and the median time to a complete response or complete response with incomplete hematologic recovery was 35 days (range 27 to 58). Among patients achieving complete response, 80% (95% CI: 64%, 90%) had an ongoing response at six months and 75% (95% CI: 57%, 87%) at 12 months. Median overall survival was 15.5 months (95% CI 7.6-not estimable). The 30- and 60-day mortality rates were 3% and 10%, respectively. The median follow-up period was 11.2 months. Serious adverse events were reported in 84% of patients, consistent with known hypomethylating agent–venetoclax regimens. Common related Grade ≥3 adverse events were anemia (30%), neutropenia (26%) and febrile neutropenia (25%). No drug–drug interactions were observed between decitabine-cedazuridine and venetoclax. In the Phase 2b of ASCERTAIN-V, decitabine-cedazuridine and venetoclax dosing was modified once leukemic blast clearance was achieved as measured by bone marrow morphology assessments. Serious adverse events and febrile neutropenia decreased as venetoclax dosing days decreased with each treatment cycle. This information potentially supports the need to explore a treatment strategy of an initial induction followed by dose-adjusting ongoing cycles to enhance tolerability and minimize cytopenia-related complications in patients who have reached remission. “The positive results of the trial — response rates, safety, pharmacokinetics and tolerability data — suggest that an all-oral regimen could potentially become a meaningful alternative to existing treatment protocols that involve parenteral hypomethylating agents for patients with AML who can’t undergo intensive induction chemotherapy,” said Gail J. Roboz, M.D., the paper’s lead author, a professor of medicine, director of the Clinical and Translational Leukemia Program and a member of the Sandra and Edward Meyer Cancer Center at Weill Cornell Medicine and a hematologist oncologist at NewYork-Presbyterian/Weill Cornell Medical Center in New York City. “An all-oral treatment regimen is a highly anticipated addition to current treatment options for patients with AML.” About Decitabine and Cedazuridine Fixed-Dose Combination This product is an orally administered, fixed dose combination of the approved anti-cancer DNA hypomethylating agent, decitabine, together with cedazuridine, 6 an inhibitor of cytidine deaminase. 7 By inhibiting cytidine deaminase in the gut and the liver, the fixed dose combination is designed to allow for oral delivery of decitabine over five days in a given cycle to achieve comparable systemic exposure to IV decitabine administered over five days. About Taiho Oncology, Inc. The mission of Taiho Oncology, Inc. is to improve the lives of patients with cancer, their families and their caregivers. The company specializes in the development and commercialization of orally administered anti-cancer agents for various tumor types. Taiho Oncology has a robust pipeline of small-molecule clinical candidates targeting solid-tumor and hematological malignancies, with additional candidates in pre-clinical development. Taiho Oncology is a subsidiary of Taiho Pharmaceutical Co., Ltd. which is part of Otsuka Holdings Co., Ltd. Taiho Oncology is headquartered in Princeton, New Jersey and oversees its parent company’s European and Canadian operations, which are located in Baar, Switzerland and Oakville, Ontario, Canada. For more information, visit , and follow us on LinkedIn and X. Taiho Oncology and the Taiho Oncology logo are registered trademarks of Taiho Pharmaceutical Co., Ltd. References Roboz G, Zeidan A, Mannis G, Montesinos P, Arnan M, Savona M, et al. All-Oral Treatment of Newly Diagnosed Acute Myeloid Leukemia. Published June 3, 2026. N Engl J Med 2026;394:2107-2116. Vol 394 No. 21. American Cancer Society. Key Statistics for Acute Myeloid Leukemia (AML). Revised January 13, 2026. Accessed February 4, 2026. Heuser M, Fernandez C, Hauch O, Klibanov OM, Chaudhary T, Rives V. Therapies for acute myeloid leukemia in patients ineligible for standard induction chemotherapy: a systematic review. Future Oncol . 2022;19(11):789-810. . Zeidan A, Zhao R, Voegel A, Christensen D, Zhdanava M, Tardif-Samson A, Vanden Eynde C, Keer H, Pilon D. Time burden of treatment with parenteral hypomethylating agents in combination with venetoclax among patients with newly diagnosed Acute Myeloid Leukemia. [ASH Abstract]. Blood (2025) 146 (Supplement 1): 6990. Garcia-Manero G, McCloskey J, Griffiths EA, et al. Oral decitabine-cedazuridine versus intravenous decitabine for myelodysplastic syndromes and chronic myelomonocytic leukaemia (ASCERTAIN): a registrational, randomised, crossover, pharmacokinetics, phase 3 study. Lancet Haematol 2024;11:e15-e26. (23)00338-1/abstract Oganesian A, Redkar S, Taverna P, Choy G, Joshi-Hangal R, Azab M. Preclinical data in cynomolgus (cyn) monkeys of ASTX727, a novel oral hypomethylating agent (HMA) composed of low-dose oral decitabine combined with a novel cytidine deaminase inhibitor (CDAi) E7727 [ASH Abstract]. Blood 2013;122(21): Abstract 2526. Ferraris D, Duvall B, Delahanty G, Mistry B, Alt, J, Rojas C, et al. Design, synthesis, and pharmacological evaluation of fluorinated tetrahydrouridine derivatives as inhibitors of cytidine deaminase. J Med Chem 2014; 57:2582-2588. Contacts Taiho Oncology Contact: Leigh Labrie +1 609.664.9878 llabrie@taihooncology.com