News Releases

• The submission is based on clinical trial data demonstrating that INQOVI in combination with venetoclax met complete response endpoints; no new safety concerns were reported
• If approved, INQOVI in combination with venetoclax would be the first all-oral combination treatment for patients with newly diagnosed AML who are ineligible for intensive induction chemotherapy
• INQOVI is currently approved in the U.S. as a treatment for myelodysplastic syndromes and chronic myelomonocytic leukemia

Taiho Pharmaceutical Co., Ltd. and its U.S. subsidiary, Taiho Oncology, Inc., announced today that the U.S. Food and Drug Administration (FDA) has accepted their supplemental new drug application (sNDA) for INQOVI (decitabine and cedazuridine) plus venetoclax as a treatment for adults with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive induction chemotherapy. The FDA assigned a standard review with a Prescription Drug User Fee Act (PDUFA) target action date of February 25, 2026.

The sNDA is supported by results from ASCERTAIN-V, a Phase 2b study of INQOVI plus venetoclax in 101 adult patients with newly diagnosed AML who were ineligible for intensive induction chemotherapy.¹

INQOVI is an orally administrated hypomethylating regimen, currently indicated in the U.S. for the treatment of adults with myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML).²

Approximately 22,000 people in the U.S. will receive a diagnosis of AML, a cancer of the blood and bone marrow, in 2025.³ More than about half those patients will likely be deemed ineligible for intensive induction chemotherapy.⁴

“We have an unwavering dedication to developing innovative new cancer treatments, and the FDA’s acceptance of our sNDA for INQOVI in combination with venetoclax highlights the need for novel approaches in AML,” said Harold Keer, MD, PhD, Chief Medical Officer, Taiho Oncology. “If approved for patients with AML who are not eligible to undergo intensive induction chemotherapy, INQOVI in combination with venetoclax would offer the first all-oral alternative to current therapies.”

In each 28-day treatment cycle, the patients received INQOVI on days one through five and venetoclax daily. Median follow-up period was 11.2 months. The trial met its primary endpoint with a complete response (CR) rate of 46.5% (n=47). In considering secondary endpoints, the study found that CR plus CR with incomplete hematologic recovery totaled 63.4% (n=64). Median overall survival was estimated to be 15.5 months. At 12 months, median duration of response had not been reached, and over 75% of patients achieving CR status remained in CR. No new safety concerns were reported. Adverse events (AEs) of grade 3 and higher were reported in 98% of patients (n=99); most commonly, febrile neutropenia (49.5%), anemia (38.6%) and neutropenia (35.6%) were reported. No drug-drug interactions were observed between INQOVI and venetoclax. At 30 and 60 days after the first dose of INQOVI, deaths attributed to either AEs or disease progression totaled 3% (n=3) and 9.9% (n=10) of study participants, respectively.
Results were presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting and the 2025 European Hematology Association (EHA) Congress.

※ASCERTAIN-V Study: AStx727-07: decitabine + CEdazuRidine TreAtment IN AML, adding Venetoclax

This product is an orally administered, fixed dose combination of the anti-cancer DNA hypomethylating agent decitabine, approved in the U.S., together with cedazuridine,⁵ an inhibitor of cytidine deaminase.⁶ 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.

Taiho Pharmaceutical, a subsidiary of Otsuka Holdings Co., Ltd. ( https://www.otsuka.com/en/ ), is an R&D-driven specialty pharma focusing on the fields of oncology and immune-related diseases. Its corporate philosophy takes the form of a pledge: “We strive to improve human health and contribute to a society enriched by smiles.” In the field of oncology, in particular, Taiho Pharmaceutical is known as a leading company in Japan for developing innovative medicines for the treatment of cancer, a reputation that is rapidly expanding through their extensive global R&D efforts. In areas other than oncology, as well, the company creates and markets quality products that effectively treat medical conditions and can help improve people’s quality of life. Always putting customers first, Taiho Pharmaceutical also aims to offer consumer healthcare products that support people’s efforts to lead fulfilling and rewarding lives. For more information about Taiho Pharmaceutical, please visit https://www.taiho.co.jp/en/

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 https://www.taihooncology.com/, and follow us on LinkedIn and X.

1. 1. Zeidan A, Griffiths E, Dinardo C, et al. An all-oral regimen of decitabine-cedazuridine (DEC-C) plus venetoclax (VEN) in patients (pts) with newly diagnosed acute myeloid leukemia (AML) ineligible for intensive induction chemotherapy: Results from a phase 2 cohort of 101 pts. Presented at: the 2025 Annual Meeting of the American Society of Clinical Oncology; May 30-June 3, 2025; Chicago. Abstract 6504. https://ascopubs.org/doi/10.1200/JCO.2025.43.16_suppl.6504
2. Oncology Practice Management. FDA Approves Inqovi, a New Oral Combination Therapy, for Patients with MDS. Published July 7, 2020. Accessed June 12, 2025. https://oncpracticemanagement.com/issues/2020/august-2020-vol-10-no-8/1719-fda-approves-inqovi-a-new-oral-combination-therapy-for-patients-with-mds.
3. American Cancer Society. Key Statistics for Acute Myeloid Leukemia (AML). Revised March 4, 2025. Accessed June 12. 2025. https://www.cancer.org/cancer/types/acute-myeloid-leukemia/about/key-statistics.html.
4. 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. https://doi.org/10.2217/fon-2022-1286.
5. 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.
6. 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.