Taiho Oncology and Taiho Pharmaceutical Announce U.S. FDA Acceptance of Supplemental New Drug Application for INQOVI® in Combination with Venetoclax to Treat Patients with Acute Myeloid Leukemia

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Jul 09, 2025, 16:00 ET

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

PRINCETON, N.J., July 9, 2025 /PRNewswire/ -- Taiho Oncology, Inc., and Taiho Pharmaceutical Co., Ltd., 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 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."

Summary of ASCERTAIN-V^*Data

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

INDICATIONS
Decitabine and cedazuridine, marketed under the brand name INQOVI®, is indicated for treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and chronic myelomonocytic leukemia [CMML]) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups.

INQOVI is the first and only oral hypomethylating agent approved by the FDA for the treatment of adults with intermediate and high-risk MDS including CMML.

Commercialization of INQOVI in the U.S. is conducted by Taiho Oncology, Inc.

IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS

Myelosuppression
Fatal and serious myelosuppression can occur with INQOVI. Based on laboratory values, new or worsening thrombocytopenia occurred in 82% of patients, with Grade 3 or 4 occurring in 76%. Neutropenia occurred in 73% of patients, with Grade 3 or 4 occurring in 71%. Anemia occurred in 71% of patients, with Grade 3 or 4 occurring in 55%. Febrile neutropenia occurred in 33% of patients, with Grade 3 or 4 occurring in 32%. Myelosuppression (thrombocytopenia, neutropenia, anemia, and febrile neutropenia) is the most frequent cause of INQOVI dose reduction or interruption, occurring in 36% of patients. Permanent discontinuation due to myelosuppression (febrile neutropenia) occurred in 1% of patients. Myelosuppression and worsening neutropenia may occur more frequently in the first or second treatment cycles and may not necessarily indicate progression of underlying MDS.

Fatal and serious infectious complications can occur with INQOVI. Pneumonia occurred in 21% of patients, with Grade 3 or 4 occurring in 15%. Sepsis occurred in 14% of patients, with Grade 3 or 4 occurring in 11%. Fatal pneumonia occurred in 1% of patients, fatal sepsis in 1%, and fatal septic shock in 1%.

Obtain complete blood cell counts prior to initiation of INQOVI, prior to each cycle, and as clinically indicated to monitor response and toxicity. Administer growth factors and anti–infective therapies for treatment or prophylaxis as appropriate. Delay the next cycle and resume at the same or reduced dose as recommended.

Embryo-Fetal Toxicity
INQOVI can cause fetal harm. Advise pregnant women of the potential risk to a fetus. Advise patients to use effective contraception during treatment and for 6 months (females) or 3 months (males) after last dose.

ADVERSE REACTIONS
Serious adverse reactions in > 5% of patients included febrile neutropenia (30%), pneumonia (14%), and sepsis (13%). Fatal adverse reactions included sepsis (1%), septic shock (1%), pneumonia (1%), respiratory failure (1%), and one case each of cerebral hemorrhage and sudden death.

The most common adverse reactions (≥ 20%) were fatigue (55%), constipation (44%), hemorrhage (43%), myalgia (42%), mucositis (41%), arthralgia (40%), nausea (40%), dyspnea (38%), diarrhea (37%), rash (33%), dizziness (33%), febrile neutropenia (33%), edema (30%), headache (30%), cough (28%), decreased appetite (24%), upper respiratory tract infection (23%), pneumonia (21%), and transaminase increased (21%). The most common Grade 3 or 4 laboratory abnormalities (≥ 50%) were leukocytes decreased (81%), platelet count decreased (76%), neutrophil count decreased (71%), and hemoglobin decreased (55%).

USE IN SPECIFIC POPULATIONS

Lactation
Because of the potential for serious adverse reactions in the breastfed child, advise women not to breastfeed during treatment with INQOVI and for 2 weeks after the last dose.

Renal Impairment
No dosage modification of INQOVI is recommended for patients with mild or moderate renal impairment (creatinine clearance [CLcr] of 30 to 89 mL/min based on Cockcroft-Gault). Due to the potential for increased adverse reactions, monitor patients with moderate renal impairment (CLcr 30 to 59 mL/min) frequently for adverse reactions. INQOVI has not been studied in patients with severe renal impairment (CLcr 15 to 29 mL/min) or end-stage renal disease (ESRD: CLcr <15 mL/min).

Please see the accompanying Full Prescribing Information.

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,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.

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

Taiho Oncology and the Taiho Oncology logo are registered trademarks of Otsuka Holdings Co., Ltd. or its subsidiaries.

About Taiho Pharmaceutical Co., Ltd.
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/

References

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. doi: https://doi.org/10.1200/JCO.2025.43.16_suppl.6504.
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.
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.
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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.