Decitabine/Cedazuridine

Scientists are exploring if early intervention can prevent AML in high-risk patients, shifting the focus from monitoring to proactive prevention. CAMBRIDGE, Mass., July 10, 2025 /PRNewswire/ -- Break Through Cancer, a Boston-based cancer research foundation, has launched a clinical trial to evaluate the feasibility of early intervention for Clonal Hematopoiesis (CH), a precursor to acute myeloid leukemia (AML). This multi-institutional study focuses on high-risk patients, exploring a new approach to managing this pre-malignant condition and advancing the groundwork for preventing this aggressive blood cancer. Understanding CH and AML Risk Continue Reading Break Through Cancer launches study to test early action against AML Post this AML is a malignant blood disorder characterized by the rapid production of abnormal myeloid cells in the bone marrow and bloodstream. This aggressive cancer disrupts normal blood cell production and is associated with severe clinical outcomes, including a five-year survival rate of approximately 30%. CH can occur naturally with aging or as a consequence of chemotherapy or radiation treatment. Before receiving an AML diagnosis, many patients already have CH with AML-associated mutations, making it a pre-malignant condition. While often symptomless, some individuals with CH may have low blood counts—a condition known as Clonal Cytopenia of Uncertain Significance (CCUS). Those with high-risk CCUS have a greater than 50% chance of progressing to AML within five years. "A lot of patients have already survived one cancer, and it's devastating when they receive a diagnosis of a therapy-related AML. The ability to potentially target the disease before it progresses to that point is what really excites me," said Dr. Kelly S. Chien, Assistant Professor at The University of Texas MD Anderson Cancer Center and a clinician on the study. A New Approach: Early Intervention As part of the new interventional trial, patients diagnosed with high-risk CCUS have the opportunity to choose proactive treatment for their precursor condition. The trial (NCT06802146) will evaluate Inqovi (Astex Pharmaceuticals) in patients with high-risk CCUS, assessing the safety and feasibility of intervention at this precursor stage. Inqovi—an oral drug currently FDA-approved to treat a related blood cancer—works by altering the DNA of abnormal blood cells, thus slowing their growth and reducing the risk of progression to more advanced disease stages. The two-arm study will enroll patients with high-risk CCUS who opt for one year of treatment with Inqovi or observation only. "Many patients feel anxious when given a CCUS diagnosis and information about AML risk," says Dr. Lachelle Weeks, Assistant Professor, Dana-Farber Cancer Institute and a clinician on the study. "There are a number of patients who want to do something about it, and this trial could be the first step." All patients will be observed for three years to assess the effects of Inqovi on AML progression and other health outcomes. "Patients with high-risk CCUS already have low blood counts, which can significantly affect their quality of life," said Dr. Chien. "We're also looking at whether treatment improves quality of life, stabilizes blood counts, or reduces the burden of comorbidities, like heart disease, that are sometimes associated with certain mutations." The trial will also assess mutational burden—whether the percentage of cells with mutations increases, decreases, or stays the same with treatment. A comparison of treated and untreated patients will provide insights into potential benefits of early intervention in CCUS. This trial aims to pave the way for future larger studies testing the efficacy of intervention as part of developing comprehensive AML prevention strategies. "The ultimate goal is to prevent diagnoses like AML altogether," Dr. Weeks notes. "This trial represents a critical step in understanding CCUS progression and exploring how early intervention could redefine care standards. If successful, it could spare patients and their families the profound challenges of living with and treating AML." Prioritizing Diversity in Cancer Research In addition to its scientific goals, this trial centers inclusivity in cancer research. By leveraging Break Through Cancer's model of radical collaboration and tapping into the broad reach of four disparate communities, this trial seeks to recruit a diverse and representative patient population and proactively circumvent long-standing inequities in cancer prevention research. "By recruiting from community sites and reflecting the demographics of our catchment areas, we're ensuring our findings benefit all populations at risk," says Dr. Weeks. To achieve this, the trial partners with community-based nurse navigators linked to Break Through Cancer's four partner sites. Together, researchers at Dana-Farber Cancer Institute, Memorial Sloan Kettering Cancer Center, Johns Hopkins Sidney Kimmel Comprehensive Cancer Center and MD Anderson are working to close gaps in care and improve outcomes for all patients. About the trial Title: Early Intervention in High Risk CCUS (NCT06802146) About Break Through Cancer Founded in 2021, Break Through Cancer empowers outstanding researchers and physicians to both intercept and find cures for several of the deadliest cancers by stimulating radical collaboration among outstanding cancer research institutions, including its founding partners: Dana-Farber Cancer Institute, Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Memorial Sloan Kettering Cancer Center, MIT's Koch Institute for Integrative Cancer Research, and The University of Texas MD Anderson Cancer Center. The Foundation is supported by a Board of Directors from the five partner institutions and a Scientific Advisory Board of U.S. cancer experts. The Foundation was launched with an extraordinary challenge pledge of $250 million from Mr. and Mrs. William H. Goodwin, Jr. and their family, and the estate of William Hunter Goodwin III. For further information, please visit the Foundation's website at . References Surveillance, Epidemiology, and End Results (SEER) Program. Cancer Stat Facts: Acute Myeloid Leukemia (AML). Weeks LD, Ebert BL. Causes and consequences of clonal hematopoiesis. Blood. 2023;142(26):2235-2245. Savona M, Bewersdorf JP, Allen SL, et al. FDA approval summary: decitabine and cedazuridine tablets for myelodysplastic syndromes. Clin Cancer Res. 2022;28(16):3411-3414. Media Contact: Soracha Ward [email protected] SOURCE Break Through Cancer WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

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.