RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (Australia), Overseas New Drugs Urgently Needed in Clinical Settings (China)

Structure/Sequence

Molecular FormulaC20H21F6N7O4S

InChIKeyORZHZQZYWXEDDL-UHFFFAOYSA-N

CAS Registry1650550-25-6

Clinical Trials associated with Enasidenib Mesylate

NCT06577441

/ RecruitingPhase 2IIT

A Randomized Phase II Trial of Enasidenib-Based Therapies Versus Cedazuridine-Decitabine in Higher-Risk IDH2-Mutated Myelodysplastic Syndrome: A MyeloMATCH Sub-Study

This phase II MyeloMATCH treatment trial compares the usual treatment of cedazuridine-decitabine (ASTX727) to the combination treatment of ASTX727 and enasidenib in treating patients with higher-risk, IDH2-mutated myelodysplastic syndrome (MDS). ASTX727 is a combination of two drugs, decitabine and cedazuridine. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Enasidenib is an enzyme inhibitor that may stop the growth of cells by blocking some of the enzymes needed for cell growth. Giving ASTX727 in combination with enasidenib may be effective in treating patients with higher-risk IDH2-mutated MDS.

Start Date12 Jun 2025

Sponsor / Collaborator

National Cancer Institute

NCT06672146

/ RecruitingPhase 2IIT

A Randomized Phase II Trial of ASTX727 and Venetoclax Compared With ASTX727, Venetoclax, and Enasidenib for Newly Diagnosed Older Adults With IDH2 Mutant Acute Myeloid Leukemia: A MyeloMATCH Substudy

This phase II MyeloMATCH treatment trial studies how well ASTX727 and venetoclax plus enasidenib works compared to ASTX727 and venetoclax alone for the treatment of older patients with newly diagnosed acute myeloid leukemia (AML) or younger patients who are considered unfit for standard treatment, and who have an abnormal change (mutation) in the IDH2 gene. This gene mutation can cause AML to grow and spread. This trial is being done to see if adding enasidenib to the usual treatment can help more patients with the IDH2 gene get rid of AML.
ASTX727 is a fixed-dose formulation of two drugs, cedazuridine and decitabine. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Enasidenib works by stopping the growth and spread of tumor cells that have the IDH2 mutation. Giving ASTX727 and venetoclax plus enasidenib may work better in treating AML patients with the IDH2 mutation.

Start Date16 May 2025

Sponsor / Collaborator

National Cancer Institute

NCT06756308

/ RecruitingPhase 2IIT

A Phase 2 Study of Enasidenib in IDH2-Mutant Angioimmunoblastic T-Cell Lymphoma

The researchers are doing this study to find out whether enasidenib is a safe treatment for people with angioimmunoblastic T-cell lymphoma (AITL) that has an IDH2 mutation. The researchers will look at the safety of enasidenib when it is given alone or in combination with the drug rituximab.

Start Date24 Dec 2024

Sponsor / Collaborator

Memorial Sloan Kettering Cancer Center

[+1]

100 Clinical Results associated with Enasidenib Mesylate

100 Translational Medicine associated with Enasidenib Mesylate

100 Patents (Medical) associated with Enasidenib Mesylate

297

Literatures (Medical) associated with Enasidenib Mesylate

01 Jun 2026·EUROPEAN JOURNAL OF HAEMATOLOGY

Νοvel Therapies in High‐Risk Myelodysplastic Syndromes

Review

Author: Galanopoulos, Athanasios G. ; Papi, Christina

ABSTRACT:

Myelodysplastic syndromes/neoplasms (MDS) constitute a very heterogeneous group of clonal myeloid neoplasms characterized by a variable clinical course and recurrent genetic abnormalities. Their treatment relies on risk classification as lower or higher‐risk categories by the original International Prognostic Scoring System (IPSS) or the current revised (IPSS‐R) or molecular IPSS‐M. Higher‐risk MDS (HR‐MDS) are clonal hematopoietic disorders characterized by significant cytopenias, dysplastic changes, and a high propensity for progression to acute myeloid leukemia (AML). Up to 40% of them usually progress to AML within two years of diagnosis. Allogeneic stem cell transplantation (HSCT) remains the only potential cure and standard of care for eligible patients. Despite standard treatments such as Allo‐HSCT and hypomethylating agents (HMAs), outcomes remain suboptimal. Recent advances have led to the development of novel therapeutic strategies, such as BCL‐2 inhibitors (venetoclax), IDH1/2 inhibitors (ivosidenib, enasidenib), CD47 inhibitors (magrolimab), TIM‐3 inhibitors (sabatolimab), XPO1 inhibitors (eltanexor), NEDD8‐activating enzyme inhibitors (pevonedistat), TP53‐targeted agents (eprenetapopt), liposomal chemotherapy (CPX‐351), and oral HMA formulations. Combinations of hypomethylating agents with these new drugs, as first‐line treatment, have to date not proven more efficacious than HMA monotherapy. This review summarizes the current therapeutic landscape on novel therapies for HR‐MDS, highlighting their mechanism of action, efficacy, and demonstrates the unmet clinical need for more effective therapies.

01 Apr 2026·AMERICAN JOURNAL OF HEMATOLOGY

The Care and Cure of the Leukemias in 2026

Review

Author: Wierda, William ; Kantarjian, Hagop ; Kadia, Tapan ; Haddad, Fadi G. ; Freireich, Emil J. ; Ravandi, Farhad ; Chifotides, Helen T. ; Jabbour, Elias ; Jain, Nitin ; Welch, Mary Alma

ABSTRACT:

It is an exciting era in leukemia owing to the development of novel targeted therapies and advances in genomics, pathophysiology, prognostication, and monitoring (e.g., highly sensitive measurable residual disease assays). Currently, most leukemias are effectively treated with immunotherapies (highly effective monoclonal antibodies targeting CD19 [blinatumomab], or CD22 [inotuzumab ozogamicin]), BCR::ABL1 tyrosine kinase inhibitors (TKIs; e.g., dasatinib, ponatinib), Bruton TKIs (e.g., ibrutinib, acalabrutinib), BCL‐2 inhibitors (venetoclax), IDH1/2 inhibitors (ivosidenib, olutasidenib, and enasidenib), FLT3 inhibitors (e.g., midostaurin, quizartinib, and gilteritinib), menin inhibitors (revumenib, ziftomenib), and chimeric antigen receptor T‐cell therapies. These novel agents and their judicious use in combination strategies have transformed the treatment landscape across all leukemias, significantly increased survival and quality of life for patients, and attenuated the need for intensive chemotherapy and hematopoietic stem cell transplantation. Leukemia subtypes, such as Philadelphia‐positive acute lymphoblastic leukemia (incurable before 2000) and chronic lymphocytic leukemia (previously considered incurable) with historically dire prognoses were recently transformed to favorable leukemias with 5‐ and 10‐year survival rates of 80+% and 90+%, respectively. The BCR::ABL1 TKIs resulted in normal life expectancy in chronic myeloid leukemia. Notable advances have also been made in AML with targeted therapies, although some subsets (older/unfit patients for intensive chemotherapy, complex karyotype, TP53 ‐mutated, KMT2A ‐rearranged, and treated secondary AML) still have unfavorable outcomes. Herein, we provide a high‐level overview of prominent clinical developments across all leukemias. In contemporary times, harnessing the benefits of novel targeted therapies and the evolving treatment landscape bolster the optimistic view that most, if not all, leukemias are curable.

01 Feb 2026·Journal of oncology pharmacy practice : official publication of the International Society of Oncology Pharmacy Practitioners

Oral oncolytic monitoring pilot with patient-reported outcomes and adherence assessments

Article

Author: Monaco, Stephanie ; Bell, Christopher ; Signorelli, Jessie

Introduction:

Patients on oral oncolytics are responsible for self-monitoring adherence and adverse drug reactions (ADRs). Oncology pharmacists are in position to focus on quality and safety of care for patients on oncolytics while providing communication between the patient, physician, and specialty pharmacies. This pilot aimed to monitor patients treated by our leukemia team initiated on oral oncolytics.

Methods:

From July 2020 to February 2021, patients treated by our leukemia team newly started on oncolytics were included. Pharmacists performed medication reconciliation and drug interaction screening on initiation of oral oncolytic. Pharmacists followed up at predefined intervals. On follow up adherence was assessed using the Morisky Medication Adherence Scale-8 (MMAS-8) and patient reported outcomes (PROs) were assessed using the revised Edmonton Symptom Assessment Scale (ESAS-r). After each follow-up, a note was placed in the chart with assessment scores and recommendations.

Results:

A total of 32 patients were screened with 19 patients included. Oral oncolytics included: imatinib (4), dasatinib (5), ponatinib (1), gilteritinib (2), enasidenib (1), and venetoclax (6). Fourteen drug interactions were identified, 11 medications discontinued, nine medications added, and two medications doses were changed. Twenty-six adherence assessments were performed with 21, 4, and 1 assessment demonstrating adherence, medium adherence, and low adherence, respectively. 62 ESAS-r assessments were performed with 64% reported as no symptoms, 17% as mild, 13% as moderate, and 5% as severe symptoms. Twenty laboratory tests were ordered from pharmacist recommendation on initiation and follow-up.

Conclusion:

This pilot demonstrated the role pharmacists play in oral oncolytic monitoring and symptom management.

News (Medical) associated with Enasidenib Mesylate

13 Nov 2025

·www.prnewswire.com

Taiho Oncology Announces 14 Presentations, Including Six Oral Presentations, at the 67th American Society of Hematology Annual Meeting and Exposition

Six oral presentations will detail new data on the outcomes of all-oral regimens containing azacitidine and cedazuridine or decitabine and cedazuridine in patients with myelodysplastic syndromes, chronic myelomonocytic leukemia or acute myeloid leukemia PRINCETON, N.J., Nov. 13, 2025 /PRNewswire/ -- Taiho Oncology, Inc., a company developing and commercializing novel treatments for hematologic malignancies and solid tumors, announced the presentation of new data from 14 studies across company-sponsored and company-funded externally led studies at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, to be held Dec. 6-9, 2025, in Orlando, Florida. Taiho Oncology will share findings from clinical trials in two oral presentations and evidence from real-world studies in two online abstracts. The oral presentations will highlight results from the Phase 2 part of ASTX030-01, a randomized, open-label, crossover trial comparing oral combination of azacitidine and cedazuridine to subcutaneous azacitidine and the Phase 2 portion of the Phase 1/2 trial ASTX727-03 of low-dose versus standard-dose oral decitabine and cedazuridine in patients with lower-risk myelodysplastic syndromes (MDS). The two online posters will present real-world data on U.S. adults with acute myeloid leukemia (AML), describing treatment patterns and outcomes with hypomethylating agent (HMA) plus venetoclax, and evaluating the time burden of initiating parenteral HMA-based therapy in routine practice. Additionally, 10 presentations – including four oral presentations – led by independent investigators will feature data from Taiho-funded investigator-initiated or collaborative studies. These presentations will highlight: Results from a UK multicenter Phase 2 randomized trial comparing ASTX727 with hydroxycarbamide or best supportive care in advanced MDS/ myeloproliferative neoplasm (MPN). Data from a Phase 2 study evaluating the all-oral combination of ASTX727, venetoclax and revumenib in newly diagnosed AML patients with NPM1m, KMT2Ar or NUP98r AML mutations. Results of a Phase 1/2 clinical trial of the combination of oral decitabine/cedazuridine with the venetoclax in patients with untreated high-risk MDS and chronic myeloid leukemia (CMML). Safety and efficacy results from a Phase 1/2 trial evaluating an all-oral combination of ASTX727, venetoclax and gilteritinib in both relapsed refractory and newly diagnosed FLT3-mutated AML, high-risk MDS or MPN. "As the leader of oral HMA development, we're excited that these data will be featured in six oral presentations at the ASH Annual Meeting," said Harold Keer, MD, PhD, Taiho Oncology Chief Medical Officer. "These data highlight our increasing understanding of novel oral regimens in hematology. We are invested in developing treatments that have the potential to improve flexibility and lower the treatment burden for patients, and collaborating with leading institutions, evidenced by our continued support of independent clinical research to advance cancer care. We look forward to participating in the essential scientific exchange that contributes to progress in hematology at the ASH Annual Meeting." Data being presented by Taiho Oncology and its partners at the 2025 ASH Annual Meeting and Exposition include: Company-Sponsored Studies Oral Presentations Title: A phase 2 dose confirmation trial of oral ASTX030, a combination of oral azacitidine with cedazuridine among patients with myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia Abstract Number: 491 Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Moving the Needle Through Novel Approaches in MDS and CMML Session Date: Dec . 7, 2025 Session Time: 9:30 a.m. to 11a.m. Presentation Time: 10:15 to 10:30 a.m. Location: OCCC - Valencia Room W415D Presenter: Guillermo Garcia-Manero, MD Title: Low-dose oral decitabine and cedazuridine among patients with low-risk myelodysplastic syndromes Abstract Number: 790 Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Treatment Advances in Lower risk Myelodysplastic Syndromes and Myelodysplastic/Myeloproliferative Neoplasms Session Date: Dec . 8, 2025 Session Time: 10:30 a.m. to 12 p.m. Presentation Time: 11:15 to 11:30 a.m. Location: OCCC - Valencia Room W415A Presenter: Guillermo Garcia-Manero, MD Online Abstracts Title: Time burden of treatment with parenteral HMAs in combination with venetoclax among patients with newly diagnosed AML Abstract Number: 6990 Lead Author: Amer Zeidan, MD Title: Real-World Characteristics, Treatment Modifications, and Outcomes for AML Patients Receiving HMAs + Venetoclax (1L) Abstract Number: 8176 Lead Author: Amer Zeidan, MD Company-Funded Externally Led Studies Oral Presentations Title: Phase II Study of the All-Oral Combination of Revumenib (SNDX-5613) with Decitabine/Cedazuridine (ASTX727) and Venetoclax (SAVE) in Newly Diagnosed AML Abstract Number: 47 Session Name: 617. Acute Myeloid Leukemias: Commercially Available Therapies: Frontline treatment with intensive or lower-intensity regimens Session Date: Dec . 6, 2025 Session Time: 9:30 to 11 a.m. Presentation Time: 10:30 to 10:45 a.m. Location: OCCC - Valencia Room W415A Presenter: Wei-Ying Jen, BM BCh, FRCPath Title: Oral decitabine/cedazuridine in combination with venetoclax in treatment-naïve high-risk myelodysplastic syndrome or chronic myelomonocytic leukemia: Updates of a Phase 1/2 clinical trial Abstract Number: 237 Session Name: 637 . Myelodysplastic Syndromes : Clinical and Epidemiological: Treatment Advances in Higher Risk Myelodysplastic Syndromes Session Date: Dec . 6, 2025 Session Time: 2 to 3:30 p.m. Presentation Time: 2:30 to 2:45 p.m. Location: OCCC - Chapin Theater W415BC Presenter: Alex Bataller, MD, PhD Title: ASTX727 delivers superior response rates and associated survival benefit versus hydroxycarbamide/best supportive care in CMML and other MDS/MPN overlap syndromes: First results from the Phase 2 UK multicenter randomized ammo trial Abstract Number: 488 Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Moving the Needle Through Novel Approaches in MDS and CMML Session Date: Dec. 7, 2025 Session Time: 9:30 to 11 a.m. Presentation Time: 10:45 to 11 a.m. Location: OCCC - Valencia Room W415D Presenter: Daniel H. Wiseman, MB, ChB, PhD Title: Phase I/II Study of Decitabine/Cedazuridine (ASXT727), Venetoclax, and Gilteritinib for Patients with FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome Abstract Number: 997 Session Name: 617. Acute Myeloid Leukemias: Commercially Available Therapies: Frontline treatment with intensive or lower-intensity regimens Session Date: Dec . 8, 2025 Session Time: 4:30 to 6 p.m. Presentation Time: 4:30 to 4:45 p.m. Location: OCCC - Chapin Theater W320 Presenter: Roberta S. Azevedo, MD Poster Presentations Title: Translational Investigation of Tolinapant (ASTX660) in Acute Myeloid Leukemia Using Integrated Clinical, Bioinformatic, and Pharmacological Approaches Abstract Number: 2552 Session Name: 802. Chemical Biology and Experimental Therapeutics: Poster I Session Date: Dec. 6, 2025 Session Time: 5:30 to 7:30 p.m. Location: OCCC - West Halls B3-B4 Title: A Randomized Phase II Trial of ASTX727 and Venetoclax With or Without Enasidenib for Newly Diagnosed Older Adults With IDH2 Mutant Acute Myeloid Leukemia: A MyeloMATCH Substudy (MM1OA-S03) Abstract Number: 3466 Session Name: 617 . Acute Myeloid Leukemias: Commercially Available Therapies: Poster II Session Date: Dec. 7, 2025 Session Time: 6 to 8 p.m. Location: OCCC - West Halls B3-B4 Title: Trial in progress: A Phase I Study Evaluating the Safety of cirtuvivint (CIRT) as Monotherapy and in Combination with ASTX727 in Patients with MDS and AML Abstract Number: 3431 Session Name: 616 . Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Poster II Session Date: Dec. 7, 2025 Session Time: 6 to 8 p.m. Location: OCCC - West Halls B3-B4 Title: Trial in Progress - A Randomized Study of ASTX727 With or Without Ladademstat in Accelerated/Blast-Phase Myeloproliferative Neoplasms Abstract Number: 3827 Session Name: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster II Session Date: Dec. 7, 2025 Session Time: 6 to 8 p.m. Location: OCCC - West Halls B3-B4 Title: A Comparison of Overall Survival and Quality of Life in MDS Patients Treated with Azacitidine vs Decitabine: A Propensity-Matched Registry Study Abstract Number: 3862 Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Poster II Session Date: Dec. 7, 2025 Session Time: 6 to 8 p.m. Location: OCCC - West Halls B3-B4 Title: A Multiarm Phase 1b Study of Personalized Oral Maintenance Therapy with Decitabine/Cedazuridine (ASTX727) Plus Physician's Choice of Venetoclax, Gilteritinib, Enasidenib, or Ivosidenib in Acute Myeloid Leukemia Abstract Number: 5228 Session Name: 617. Acute Myeloid Leukemias: Commercially Available Therapies: Poster III Session Date: Dec. 8, 2025 Session Time: 6 to 8 p.m. Location: OCCC - West Halls B3-B4 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. Taiho Oncology Contact: Leigh Labrie (609) 664-9878 [email protected] SOURCE Taiho Oncology 21% more press release views with Request a Demo

Phase 2ASHClinical ResultPhase 1Cell Therapy

08 Apr 2025

·www.prnewswire.com

TIBSOVO's Continued Success Solidifies Its Position as a Leader in IDH1 Mutant Space | DelveInsight

With its ability to specifically inhibit the mutant IDH1 enzyme, TIBSOVO addresses an unmet need in personalized cancer treatment, offering new hope for patients with limited options. As clinical evidence continues to support its efficacy and safety, TIBSOVO is positioned for strong growth in the oncology market, especially with expanding indications and global market penetration. LAS VEGAS, April 8, 2025 /PRNewswire/ -- DelveInsight's " TIBSOVO Market Size, Forecast, and Market Insight Report" highlights the details around TIBSOVO, the first and only targeted therapy approved for patients with previously treated IDH1-mutated cholangiocarcinoma. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of TIBSOVO. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Servier's TIBSOVO (ivosidenib) Overview TIBSOVO (ivosidenib) is a small molecule developed by Servier, acts as an isocitrate dehydrogenase-1 (IDH1) inhibitor. It is indicated for patients with a susceptible IDH1 mutation, which is identified through an FDA-approved test. Currently, TIBSOVO is approved for the treatment of newly diagnosed acute myeloid leukemia (AML), relapsed or refractory AML, relapsed or refractory myelodysplastic syndromes (MDS), and locally advanced or metastatic cholangiocarcinoma. According to Servier's pipeline, TIBSOVO is also being actively investigated for a range of additional indications, with key areas of exploration including solid tumors and hematological malignancies. Learn more about TIBSOVO projected market size for AML, cholangiocarcinoma, and MDS @ TIBSOVO Market Potential The oncology market has experienced significant growth in recent years, driven by advances in cancer research, the development of targeted therapies, and the increasing global prevalence of cancer. As the second-leading cause of death worldwide, cancer continues to pose a major public health challenge, creating a substantial demand for innovative treatments. The market is marked by a shift toward personalized medicine, where therapies are tailored to an individual's genetic profile, allowing for more effective and less toxic treatments. Immunotherapies, such as checkpoint inhibitors, CAR-T cell therapies, and oncolytic virus therapies, are at the forefront of these advancements, offering new hope to patients with previously untreatable cancers. Moreover, the oncology market is expanding due to the growing adoption of precision oncology and molecular diagnostics, which enable earlier and more accurate detection of cancer. This has led to increased investments in research and development, especially in areas like liquid biopsies, companion diagnostics, and next-generation sequencing. The demand for more effective and accessible cancer treatments is fueling innovation in both the pharmaceutical and biotechnology sectors, with numerous new drugs and therapies entering clinical trials and moving toward commercialization. As a result, the oncology market is poised to continue growing, with significant opportunities for breakthrough therapies in immuno-oncology, targeted treatments, and gene therapies. DelveInsight has expertise in the oncology market with an experienced team handling the oncology domain proficiently. DelveInsight has released a series of epidemiology-based market reports on Acute Myeloid Leukemia, Cholangiocarcinoma, and Myelodysplastic Syndrome. These reports include a comprehensive understanding of current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Gain deeper insights into these diseases with a customized report tailored to your needs. Connect with us today—our experts are ready to assist you! Emerging Competitors of TIBSOVO TIBSOVO faces significant competition in the newly diagnosed AML market from several alternative treatments. Oral therapies like VENCLEXTA (venetoclax), which inhibits BCL-2, and DAURISMO (glasdegib), which targets the hedgehog pathway, present strong contenders. Additionally, emerging therapies such as Lisaftoclax (Ascentage Pharma), another BCL-2 inhibitor, and gilteritinib (Astellas), a kinase inhibitor, are expected to further challenge TIBSOVO in the newly diagnosed AML space. In the R/R AML market, TIBSOVO competes with several therapies, including XOSPATA (gilteritinib), IDHIFA (enasidenib), and others. Emerging therapies such as Ziftomenib (Kura Oncology) and RVU-120 (Ryvu Therapeutics) are also expected to compete with TIBSOVO upon their approval. Ziftomenib, a small molecule developed by Kura Oncology, is currently in Phase I/II trials. In the relapsed/refractory (R/R) MDS market, TIBSOVO faces competition from therapies such as RYTELO (Imetelstat), an IV injection that inhibits oligonucleotide telomerase, and REBLOZYL (Luspatercept), a subcutaneous injection, among other treatment options. In the metastatic cholangiocarcinoma market, TIBSOVO faces relatively less competition, as there are few approved therapies. One such competitor is PEMAZYRE (pemigatinib), a kinase inhibitor that was approved in the US in 2020 and in Europe in 2021. Whereas, Tyra Biosciences' TYRA-200, an FGFR1/2/3 inhibitor, is in phase I stage of development that might be a threat to TIBSOVO one approved. To know more about the number of competing drugs in development, visit @ TIBSOVO Market Positioning Compared to Other Drugs Key Developmental Activities of TIBSOVO In October 2023, Servier announced that the FDA has approved TIBSOVO (ivosidenib tablets) for the treatment of IDH1-mutated R/R MDS. In May 2023, Servier announced that the European Commission has approved TIBSOVO as a targeted therapy in two indications: in combination with azacitidine for the treatment of adult patients with newly diagnosed AML with an isocitrate dehydrogenase-1 R132 mutation who are not eligible to receive standard induction chemotherapy; as well as in monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy. In May 2022, Servier announced FDA approval of TIBSOVO (ivosidenib tablets) in combination with azacitidine for patients with newly diagnosed IDH1-mutated acute myeloid leukemia. In August 2021, Servier announced FDA approval for TIBSOVO (ivosidenib tablets) for the treatment of adult patients with previously treated, locally advanced, or metastatic cholangiocarcinoma with an IDH1 mutation as detected by an FDA-approved test. TIBSOVO is the first and only targeted therapy approved for patients with previously treated IDH1-mutated cholangiocarcinoma. In May 2019, Agios announced FDA approval of TIBSOVO (ivosidenib tablets) as monotherapy for newly diagnosed adult patients with IDH1 mutant acute myeloid leukemia not eligible for intensive chemotherapy. In July 2018, the FDA approved TIBSOVO (ivosidenib) for relapsed or refractory acute myeloid leukemia with an idh1 mutation. In February 2018, the FDA accepted a new drug application and granted priority review for ivosidenib in relapsed or refractory AML with an IDH1 mutation. In December 2017, Agios submitted an NDA to the FDA for ivosidenib for the treatment of patients with relapsed/refractory aml and an IDH1 mutation. TIBSOVO Patent Details TIBSOVO is a drug owned by Servier Pharmaceuticals. Currently, it is protected by 10 Orange book-listed patents filed from 2018 to 2023, out of which none have expired yet. It is also protected by ODE exclusivity for various indications. Discover how TIBSOVO is shaping the blood cancer treatment landscape @ TIBSOVO Side Effects TIBSOVO Market Dynamics In recent years, the therapeutic landscape has undergone significant changes, with promising approaches including immunotherapy, chemotherapy combined with targeted treatments, and the regulation of immune checkpoint-mediated signaling pathways. One such treatment, Ivosidenib, works by inducing the bone marrow to develop normal blood cells, helping to reduce the frequency of blood transfusions. Investment in the research and development of drugs has surged, contributing to a rich pipeline and forecasting promising opportunities in the treatment markets for AML and cholangiocarcinomas. Therapies like TIBSOVO have the potential to capture market attention due to their efficacy and innovation. As healthcare spending continues to rise globally, the pharmaceutical market is expected to expand, providing greater opportunities for market penetration by manufacturers and facilitating the development and distribution of new treatments. Despite progress in cancer treatment, significant gaps remain in understanding the full process that governs carcinogenesis and resistance to treatments, particularly in cancers like acute myeloid leukemia (AML). This highlights the need for more advanced research to address these challenges. Additionally, the limited biomarker testing for patients with the IDH1 mutation restricts the targeted patient pool, posing a challenge for therapies like TIBSOVO to achieve their expected market share. The increasing competition from emerging therapies, such as KEYTRUDA (pembrolizumab), IMFINZI (durvalumab), and the combination of Azacitidine + Venetoclax, further intensifies the pressure on TIBSOVO. Moreover, the difficulty in obtaining sufficient patient pools and diagnostic samples during disease recurrence presents significant hurdles to the effective development of translational research, potentially slowing progress in optimizing treatments. Dive deeper to get more insight into TIBSOVO's strengths & weaknesses relative to competitors @ TIBSOVO Market Drug Report Table of Contents Related Reports Acute Myeloid Leukemia Market Acute Myeloid Leukemia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key AML companies including Agios Pharmaceuticals, Abbvie, Jazz Pharmaceuticals, Astellas Pharma, Pfizer, Novartis Oncology, Daiichi Sankyo, AstraZeneca, Astex Pharmaceuticals, Inc., Chimerix, Takeda, Rafael Pharmaceuticals Inc., Delta-Fly Pharma, GlycoMimetics Incorporated, BerGenBio ASA, MacroGenics, Syndax Pharmaceuticals, Arog Pharmaceuticals, Forma Therapeutics, Sellas Life Sciences Group, Actinium Pharmaceuticals, Clear Creek Bio Inc., CellCentric Ltd., Biosight Ltd., Astex Pharmaceuticals, Inc., Curis, Inc., NexImmune Inc., Immunomedics, Inc., Sumitomo Dainippon Pharma Co., Ltd., Incyte Corporation, Aprea Therapeutics, Immunicum, DCPrime BV, GT Biopharma, Inc., Hanmi Pharmaceutical Company Limited, Cardiff Oncology, Bio-Path Holdings, Actinium Pharmaceuticals, Aptevo Therapeutics, Synimmune GmbH, GEMoaB Monoclonals, ImmunoGen, Inc., AGC Biologics S.p.A., Precigen, Inc., Novartis Pharmaceuticals, Xencor, Inc., Novartis, Celgene, Bristol Myers Squibb, among others. Relapsed/Refractory Acute Myeloid Leukemia Market Relapsed/Refractory Acute Myeloid Leukemia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key R/R AML companies including Agios Pharmaceuticals, Abbvie, Jazz Pharmaceuticals, Astellas Pharma, Pfizer, Novartis Oncology, Daiichi Sankyo, AstraZeneca, Astex Pharmaceuticals, Inc., Chimerix, Takeda, Rafael Pharmaceuticals Inc., Delta-Fly Pharma, GlycoMimetics Incorporated, BerGenBio ASA, MacroGenics, Syndax Pharmaceuticals, Arog Pharmaceuticals, Forma Therapeutics, Sellas Life Sciences Group, Actinium Pharmaceuticals, Clear Creek Bio Inc., CellCentric Ltd., Biosight Ltd., Astex Pharmaceuticals, Inc., Curis, Inc., NexImmune Inc., Immunomedics, Inc., Sumitomo Dainippon Pharma Co., Ltd., Incyte Corporation, Aprea Therapeutics, Immunicum, DCPrime BV, GT Biopharma, Inc., Hanmi Pharmaceutical Company Limited, Cardiff Oncology, Bio-Path Holdings, Actinium Pharmaceuticals, Aptevo Therapeutics, Synimmune GmbH, GEMoaB Monoclonals, ImmunoGen, Inc., AGC Biologics S.p.A., Precigen, Inc., Novartis Pharmaceuticals, Xencor, Inc., Novartis, Celgene, Bristol Myers Squibb, among others. 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Drug ApprovalImmunotherapyPhase 1Priority Review

14 Jan 2025

·www.taihooncology.com

Taiho Oncology Announces Decitabine and Cedazuridine to Be Included in NIH Precision Medicine Trials

Drug combination part of next-generation trials targeting specific genetic mutations in patients with myelodysplastic syndromes and acute myeloid leukemia PRINCETON, New Jersey, January 14, 2025 — Taiho Oncology, Inc., a company developing and commercializing novel treatments for hematologic malignancies and solid tumors, announced today that oral decitabine and cedazuridine will be included in next-generation clinical trials for precision medicine treatments initiated by the National Cancer Institute (NCI), part of the National Institutes of Health (NIH). Announced by the NIH on Oct. 23, 2024, the Myeloid Malignancies Molecular Analysis for Therapy Choice, or myeloMATCH, trials will be sponsored by NCI and conducted by the NCI-funded National Clinical Trials Network. The myeloMATCH program is comprised of trials evaluating new treatment combinations that target cancer-driving genetic mutations in myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML). Across the myeloMATCH program, patients will undergo rapid genetic testing of their tumors and then be matched to trials involving oral decitabine and cedazuridine, other studies within myeloMATCH or, if there are no other myeloMATCH targeted combination options for their cancer types, standard treatment. “The myeloMATCH program is designed to ensure patients with aggressive malignancies of the blood and bone marrow receive therapy directed at specific, identified abnormalities in a timely manner,” said Harold Keer, MD, PhD, Chief Medical Officer, Taiho Oncology. “We are excited to have oral decitabine and cedazuridine included in this innovative clinical trial program. The inclusion of all oral regimens in the setting of myeloid malignancies has the potential to produce practice-changing data and reduce the treatment burden that patients and their loved ones face.” Oral decitabine and cedazuridine will be included in two trials within the myeloMATCH program: A Phase 2 trial (ClinicalTrials.gov, NCT06577441) will compare oral decitabine and cedazuridine to the combination treatment of oral decitabine and cedazuridine and enasidenib in treating patients with higher-risk, IDH2-mutated MDS. The trial will compare the complete remission (CR) rate between the two study groups and assess other endpoints, including safety. A Phase 2 trial (ClinicalTrials.gov, NCT06672146) will compare oral decitabine and cedazuridine and venetoclax plus enasidenib compared to oral decitabine and cedazuridine and venetoclax alone for the treatment of older patients with newly diagnosed AML or younger patients who are unable to receive standard treatment, and who have a mutation in the IDH2 gene. The trial will evaluate safety and compare the rate of measurable residual disease (MRD) negative complete remission (CR), as well as other secondary endpoints. For more information about myeloMATCH and the sub-studies that are currently open, click here. 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 Contact: Leigh Labrie (609) 664-9878 llabrie@taihooncology.com

Phase 2

100 Deals associated with Enasidenib Mesylate

External Link

KEGG	Wiki	ATC	Drug Bank
-	Enasidenib Mesylate	L01XX59	DB13874

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Acute Myeloid Leukemia	United States	Bristol Myers Squibb Co.	01 Aug 2017

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	United States	Celgene Corp.	30 Dec 2015
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	China	Celgene Corp.	30 Dec 2015
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	Australia	Celgene Corp.	30 Dec 2015
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	Austria	Celgene Corp.	30 Dec 2015
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	Belgium	Celgene Corp.	30 Dec 2015
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	Brazil	Celgene Corp.	30 Dec 2015
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	Canada	Celgene Corp.	30 Dec 2015
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	Czechia	Celgene Corp.	30 Dec 2015
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	Denmark	Celgene Corp.	30 Dec 2015
IDH2 mutation Acute Myeloblastic Leukemia	Phase 3	France	Celgene Corp.	30 Dec 2015

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


EHA2026	Not Applicable	Immunoblastic Lymphadenopathy TET2 \| RHOA \| DNMT3A ... View more	9	MINE regimen + targeted agents	estwbnrban(mufdrskuvh) = bswpezreyu wwhiobwynw (vkqroztcrq ) View more	Positive	12 May 2026
NCT05282459 (CTgov)	Phase 1/2	Anemia \| Monocytic leukemia \| Secondary Myelodysplastic Syndrome ... View more	17	Enasidenib Mesylat (Enasidenib Mesylat 100mg)	feldohdcho = bmcujczfnx mtnnqyjfpx (txgdbisxhp, ngibihqqmb - ylqvpemtxi) View more	-	17 Apr 2026
				Enasidenib Mesylat (Enasidenib Mesylat 200mg)	feldohdcho = yztoyfkssg mtnnqyjfpx (txgdbisxhp, llupskxnfn - isuuoggpya) View more
ASCO_GI2026	Not Applicable	Bile Duct Neoplasms First line IDH2 mutation	40	Gemcitabine/platinum +/- devimistat or durvalumab	cmoehwpudr(byjijqzjva) = hcdqznztnh sjpzccbcch (cuhfjumvtg, 12.1 - 28.9) View more	Positive	08 Jan 2026
				Enasidenib	zkxbhplqlo(nytneqnhqg) = kxsimhtwmp pvuoullosu (xvhtdrtfed, 1.1 - 5.1)
NCT04522895 (ASH2025)	Phase 2	IDH2 mutation Acute Myeloblastic Leukemia Maintenance IDH2-mutated	47	Enasidenib maintenance therapy	jjazznsqyp(ulqzzmhxdg) = cmvmnthtin gaovrywlda (jrihlpzdxq ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Acute Myeloid Leukemia	387	Ivosidenib monotherapy (FL IDH1-mut AML)	zrdcwjmlqv(fgvkafsojc) = ajahhzkmqb ekqxsxynip (jpebxkcjuq, 46.0 - 84.6) View more	Positive	06 Dec 2025
				Ivosidenib+azacitidine (FL IDH1-mut AML)	zrdcwjmlqv(fgvkafsojc) = zpdgtardgz ekqxsxynip (jpebxkcjuq, 21.1 - 53.1) View more
NCT04092179 (ENAVEN-AML, Pubmed \| Lancet Haematol)	Phase 1/2	Relapsing acute myeloid leukemia \| Myelodysplastic Syndromes IDH2 mutation	27	Venetoclax 400 mgEnasidenib 100 mg + Venetoclax 400 mgEnasidenib 100 mg	kfruroaoeq(icafmcdomk) = xekibqqdfq kcrvnefjox (ywhwtouuxc, 41 - 80) View more	Positive	01 Nov 2025
NCT04774393 (PS1471, EHA2025)	Phase 1/2	Myeloid Tumor	105	Decitabine/Cedazuridine + Venetoclax + Ivosidenib	pmqzlfvgjf(zvqieecdof) = anvepbhzot tnjgosfftx (nvfbompter )	Positive	14 May 2025
				Decitabine/Cedazuridine + Venetoclax + Enasidenib	pmqzlfvgjf(zvqieecdof) = eqfqnbscvw tnjgosfftx (nvfbompter )
PB2774 (EHA2025)	Phase 1/2	Myelodysplastic Syndromes	770	Rigosertib	decghjkgph(pesxbrowzc) = jktwnffbsb zqavfvqyms (mkxbvywgog, 0.012 - 0.039) View more	Positive	14 May 2025
NCT04281498 (MPN-RC 119, CTgov)	Phase 2	Primary Myelofibrosis	6	Enasidenib+Ruxolitinib	tyfapzfbwu = ouhtjsxxea lgcxuylkkw (vyrimnllfh, jgvgwuhgdc - ugyxpyigst) View more	-	18 Dec 2024
NCT05010772 (4277, ASH2024)	Phase 1	Acute Myeloid Leukemia complex cytogenetics \| antecedent MDS/MPN \| therapy-related AML ... View more	31	ASTX727 alone	gaobvmvzrg(snemnhzwau) = zsamixzdue ogalaosgfx (swgyxnpkbz ) View more	Positive	09 Dec 2024
				ASTX727 plus venetoclax	gaobvmvzrg(snemnhzwau) = zyzljykejm ogalaosgfx (swgyxnpkbz ) View more

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