RegulationBreakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Japan), Orphan Drug (South Korea), Orphan Drug (Australia), Overseas New Drugs Urgently Needed in Clinical Settings (China), Priority Review (Australia), Priority Review (United States), Conditional marketing approval (China), Priority Review (China)

Structure/Sequence

Molecular FormulaC28H22ClF3N6O3

InChIKeyWIJZXSAJMHAVGX-DHLKQENFSA-N

CAS Registry1448347-49-6

Clinical Trials associated with Ivosidenib

CTIS2025-521322-14-00

/ RecruitingPhase 2IIT

FILO-AML-01-MIVONU A single arm phase II study investigating the efficacy and safety of the addition of ivosidenib to oral azacitidine (Onureg®) in patients over 55 with Acute Myeloid Leukemia (AML) and IDH1 mutation, in complete remission after intensive chemotherapy. A study of the French AML Intergroup.

Start Date11 Dec 2025

Sponsor / Collaborator-

NCT06707493

/ RecruitingPhase 2IIT

A Randomized, Placebo-Controlled Phase 2 Study of IDH1 Inhibition Using Ivosidenib as Maintenance Therapy for IDH1-mutant Acute Myeloid Leukemia Following Allogeneic Stem Cell Transplantation

This is a Phase 2 study of the study drug, ivosidenib (a mutant IDH1 inhibitor), compared to placebo, given to patients with IDH1-mutant acute myeloid leukemia (AML) after hematopoietic stem cell transplantation (HCT).

Start Date08 Dec 2025

Sponsor / Collaborator

The Massachusetts General Hospital

[+1]

NCT07260175

/ RecruitingPhase 2IIT

adIVO - A Phase II Trial of Ivosidenib Maintenance After SOC Adjuvant Chemotherapy in Curative mIDH1 Cholangiocarcinoma

This study trial is a prospective, multicentre, exploratory, single-arm, open-label phase II study to evaluat ivosidenib maintenance after SOC adjuvant chemotherapy in curative mIDH1 cholangiocarcinoma

Start Date18 Nov 2025

Sponsor / Collaborator

Servier Deutschland GmbH

[+1]

100 Clinical Results associated with Ivosidenib

100 Translational Medicine associated with Ivosidenib

100 Patents (Medical) associated with Ivosidenib

371

Literatures (Medical) associated with Ivosidenib

01 Nov 2025·JOURNAL OF NEURO-ONCOLOGY

Single center experience of IDH inhibitors in recurrent high-grade gliomas

Article

Author: Highsmith, Kaitlin ; Leung, Cheuk Hong ; Andrade, Ivan Pradilla ; Garcia, Catherine R ; Kamiya-Matsuoka, Carlos ; Puduvalli, Vinay ; Knight, Stephanie

PURPOSE:

The role of IDH inhibitors in recurrent high-grade gliomas (HGG) is not well described. We present the outcomes of patients with HGG that were treated with these agents at our institution.

METHODS:

We reviewed patients with recurrent IDH-mutant HGG treated with FDA approved IDH inhibitors (ivosidenib, enasidenib, and vorasidenib) from December 2019 to December 2024 at the University of Texas MD Anderson Cancer Center.

RESULTS:

23 patients were identified of which 65.2% were male. Tumors included astrocytoma (n = 14, 60.9%) and WHO grade 3 oligodendroglioma (n = 9, 39.1%), with four patients having a WHO grade 4 astrocytoma and 10 a WHO grade 3 astrocytoma. Twenty patients had enhancing disease at the time an IDH inhibitor was recommended. One patient was treated with enasidenib, 17 with ivosidenib, and 5 with vorasidenib. Of those initially treated with ivosidenib, 9 were switched to vorasidenib. Two patients discontinued the drugs due to side effects, that included elevated liver enzymes (n = 1), and diarrhea (n = 1). All patients had received prior surgery, radiation, and chemotherapy before starting an IDH inhibitor. Median overall survival (OS) was not reached. OS after IDH inhibitor was 20.7 months and was longer in patients that had not received initial chemotherapy (p = 0.032). Median progression-free survival (PFS) was 6.8 months and was not different between tumor type, sex, or number of recurrences, but was longer in patients that did not receive initial chemotherapy (p = 0.041).

CONCLUSION:

IDH inhibitors were well tolerated in patients with IDH-mutant HGG previously treated with DNA-damaging agents. A median PFS longer than 6 months is encouraging in this patient population and may be due to antitumor activity of IDH inhibitors in recurrent HGG.

01 Nov 2025·Blood neoplasia

Ivosidenib or venetoclax combined with hypomethylating agents in IDH1-mutated acute myeloid leukemia: a real-world study

Article

Author: Ambinder, Alexander Joseph ; LeBlanc, Thomas W ; Papademetriou, Eros ; Angiolillo, Anne ; Potluri, Ravi ; Binder, Gary ; Lachowiez, Curtis A ; Smith, B Douglas

Approximately 10% of patients with newly diagnosed acute myeloid leukemia (ND-AML) harbor the isocitrate dehydrogenase 1 gene mutation (mIDH1). In this real-world study evaluating ivosidenib (IVO) + hypomethylating agents (HMAs; n = 181) vs venetoclax (VEN) + HMAs (n = 99) in patients with mIDH1 ND-AML, those treated with IVO+HMA had higher rates of complete remission (CR; 42.5% vs 26.3%; P = .007), higher rates of composite CR + CR with incomplete platelet count recovery (63.0% vs 48.5%; P = .019), shorter median time to best response (3.3 vs 4.1 months; P = .006), and improved 6-month event-free survival (55.8% vs 38.4%; P = .006). Most patients treated with VEN received well under 28 days of VEN per cycle, likely due to anticipation of toxicity; outcomes with this short-schedule VEN were proportionately worse with fewer days of exposure per cycle. The between-group rate of grade ≥3 adverse events was similar within 30 days of treatment initiation, except for higher rates of febrile neutropenia for VEN+HMA vs IVO+HMA (8.1% vs 1.7%; P = .008). These findings support results from the phase 3 AGILE trial demonstrating IVO+HMA's efficacy and favorable toxicity profile in patients with mIDH1 ND-AML. IVO + azacitidine should be considered as the preferred standard of care treatment regimen in this patient subgroup.

01 Nov 2025·ANTI-CANCER DRUGS

Safety and efficacy of ivosidenib in the treatment of isocitrate dehydrogenase 1 mutant cholangiocarcinoma and acute myeloid leukemia: a systematic review and meta-analysis

Review

Author: Qasim, Rameez ; Haseeb, Bakhtawar ; Raza, Mubashir ; Shakeel, Izza ; Anmol, Laraib ; Iqbal, Uzair ; Ahmad, Shaheer ; Bhatti, Hurmat Fatima ; Hassan, Muhammad

Isocitrate dehydrogenase 1 (IDH1) mutations have gained interest because of their association with malignancies, including cholangiocarcinoma and acute myeloid leukemia. Ivosidenib, an inhibitor of IDH1 mutations, inhibits the formation of the oncometabolite D-2-HG, restoring normal cellular turnover and inhibiting tumorigenesis. In July 2024, a literature search was done using these databases: PubMed, Cochrane Library, and Embase. Studies were to show the safety and efficacy of ivosidenib using 95% confidence intervals (CIs). Preferred Reporting Items for Systematic reviews and Meta-Analyses flow guidelines were followed. Four articles involving 533 patients were included. The objective response rate (ORR) and progression-free survival (PFS) were significantly improved in the control group where risk ratio was 0.79, 95% CI: 0.71–0.89, Z = 4.05, a P value less than 0.001 for PFS, and odds ratio was 0.45, 95% CI: 0.30–0.68, Z value of 3.86, and P = 0.001 for ORR. The safety profile was favorable. Overall survival (OS) did not change significantly within the groups, as indicated by a P value of 0.78, risk ratio of 0.98, 95% CI: 0.83–1.15, and Z = 0.27. Ivosidenib demonstrated a PFS advantage and improved ORR with a favorable safety profile, but no effect on the OS. Evidence is suggestive of its plausibility for clinical usage as an adjunct therapy.

125

News (Medical) associated with Ivosidenib

10 Dec 2025

·pharma.economictimes.indiatimes.com

Servier gets CDSCO nod for brain cancer drug Voranigo

Mumbai: French drugmaker Servier , has secured CDSCO nod for marketing its brain cancer therapy Voranigo ( Vorasidenib ) in India. Voranigo is approved for treating Grade 2 IDH-mutant astrocytoma and oligodendroglioma–types of brain cancer. Notably, the oral small-molecule which delays tumour progression is the first and only FDA-approved targeted therapy over the two indications in the world. According to the company unlike conventional chemotherapy or radiotherapy, which affects both healthy and cancerous cells, Vorasidenib is a targeted therapy that inhibits the mutant IDH enzyme responsible for tumor growth and spread. Grade 2 gliomas are a rare indication and are classified as an unmet medical need across multiple countries. Servier is placing a strong focus on India and earlier this year, the French drugmaker launched Tibsovo, another targeted therapy for IDH1-mutated acute myeloid leukemia (blood cancer) and cholangiocarcinoma. “Vorasidenib, marks a significant breakthrough directly addressing a critical unmet need in rare neuro-oncology and is a key part of Servier’s global journey in advancing cancer care,” said Aurelien Breton, Managing Director, Servier India . By Online Bureau ,

Drug Approval

21 Nov 2025

·www.prnewswire.com

Hackensack Meridian John Theurer Cancer Center Unveils Research at the 67th American Society of Hematology Annual Meeting

Advances span leukemia, lymphoma, myeloma, myeloproliferative neoplasms, sickle cell disease, and AI-enabled diagnostics HACKENSACK, N.J., Nov. 21, 2025 /PRNewswire/ -- Investigators from Hackensack Meridian John Theurer Cancer Center (JTCC)—a leading research partner of the NCI-designated Lombardi Comprehensive Cancer Center at Georgetown University, and number one Cancer Center in New Jersey—will present 65 studies at the 67th American Society of Hematology (ASH) Annual Meeting, taking place December 6–9, 2025, in Orlando. This represents one of JTCC's largest and most diverse scientific contributions to ASH to date, highlighting innovations in cell therapy, targeted agents, AI-driven diagnostics, stem cell transplantation, and real-world evidence across virtually every hematologic disease area. "John Theurer Cancer Center continues to help shape the future of blood cancer care," said André Goy, MD, chair, physician-in-chief and vice president of oncology at Hackensack Meridian Health. "Our teams are redefining transplantation, advancing CAR-T science, and co-leading trials testing next-generation targeted therapies and immunotherapies. The depth and breadth of our ASH presentations underscore our mission: to bring transformational science rapidly to the clinic for patients with blood cancers and other serious blood disorders." Highlights of JTCC Research to be presented at ASH 2025 can be found here. Leukemia High event-free (EFS) and overall survival (OS) after non-total body irradiation (TBI) conditioning and allogeneic hematopoietic cell transplantation (HCT) in next-generation-sequencing minimal residual disease (NGS-MRD) negative B-acute lymphoblastic leukemia (B-ALL): Results from the EndRAD trial (PTCTC ONC1701) (ABSTRACT 25-12959) 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 25-7509) Ziftomenib in combination with venetoclax and azacitidine in relapsed/refractory NPM1-m or KMT2A-r acute myeloid leukemia: Updated phase 1a/b safety and clinical activity results from KOMET-007 (ABSTRACT 25-3910) MRD-guided therapy of sonrotoclax (BGB-11417) + obinutuzumab (O) in patients with treatment-naive CLL: Initial results from an ongoing phase 1/1b study, BGB-11417-101 (ABSTRACT 25-7489) Real world outcomes of bispecific T-cell engagers in plasma cell leukemia (ABSTRACT 25-4651) Etoposide can be safely removed from induction chemotherapy without impacting survival for pediatric acute myeloid leukemia – a report from the Children's oncology group study AAML1831 (ABSTRACT 25-12270) Updated response and safety analyses from a Phase 1 study of ivosidenib combined with intensive chemotherapy in patients with newly diagnosed (ND) Acute Myeloid Leukemia with isocitrate dehydrogenase (IDH)1 mutation (ABSTRACT 25-421) AI-derived prediction of response and relapse to venetoclax plus hypomethylating agent based therapy in Acute Myeloid Leukemia (ABSTRACT 25-14588) TSC-101 eliminates recipient hematopoietic cells and demonstrates potential for improved relapse-free survival in patients with AML, ALL, or MDS undergoing allogeneic HCT: Updated results from the Phase 1 (ALLOHA) trial (ABSTRACT 25-12098) Trials in progress: Design of a registrational Phase 2 trial (ALLOHA) using an external control arm for TSC-101 for prevention of relapse post allogeneic HCT in patients with ALL, AML, or MDS (ABSTRACT 25-13827) Developing artificial intelligence-based transcriptomic signature for selecting patients with HOXA-MEIS1 pathway abnormalities for the treatment with menin inhibitors (ABSTRACT 25-4126) Evaluation of ventoclax initiation prophylaxis and monitoring outcomes at each dose level and time point in patients with chronic lymphocytic leukemia: A real-world experience (ABSTRACT 25-2129) Developing transcriptomic signature for IDH1 and IDH2 acute leukemia and the demonstration of high prevalence of these signatures in mutation-negative leukemia (ABSTRACT 25-4127) Impact of DUSP22 and TP63 rearrangements in patients with ALK-negative ALCL treated with frontline BV-CH(E)P (ABSTRACT 25-1798) Harnessing repressive LEF1/ β-catenin complexes to overcome drug resistance in chronic lymphocytic leukemia (ABSTRACT 25-14485) Reducing Acute Myeloid Leukemia resistance to CAR T cell therapy by epigenetic activation of the tumor inflammasome-pyroptosis signaling (ABSTRACT 25-13458) Lymphoma Epcoritamab + R-mini-CHOP results in 2-year remissions and high MRD negativity rates in elderly patients with newly diagnosed DLBCL: Results from the EPCORE NHL-2 trial (ABSTRACT 25-3828) Epcoritamab with rituximab + lenalidomide (R2) and epcoritamab maintenance deliver deep and durable remissions in previously untreated (1L) follicular lymphoma (FL): 3-year outcomes from epcore NHL-2 arms 6 and 7 (ABSTRACT 25-2787) Liquid-biopsy mutation landscape and its concordance with skin biopsies in cutaneous T-cell lymphoma (ABSTRACT 25-2858) ZUMA-25 preliminary analysis: A Phase 2 study of brexucabtagene autoleucel (brexu-cel) in patients (Pts) with relapsed/refractory (R/R) Burkitt lymphoma (BL), substudy C (ABSTRACT 25-2841) Final results of a phase 1 trial with soquelitinib (SQL), a selective interleukin-2-inducible T cell kinase (ITK) inhibitor for treatment of relapsed/refractory (R/R) T cell lymphomas (TCL) (ABSTRACT 25-2574) Acalabrutinib plus venetoclax and rituximab in patients with treatment-naive (TN) mantle cell lymphoma (MCL): Results from the Phase 2 TrAVeRse study (ABSTRACT 25-7289) Two-year update of ZUMA-2 Cohort 3: Brexucabtagene autoleucel (Brexu-cel) in patients (pts) with relapsed/refractory mantle cell lymphoma (R/R MCL) who had not received prior Bruton tyrosine kinase inhibitor (BTKi) therapy (ABSTRACT 25-2240) Phase 2 bellwave-003 cohort f: Updated clinical outcomes of nemtabrutinib in participants with relapsed or refractory marginal zone lymphoma (ABSTRACT 25-2322) A real-world analysis of safety and outcomes with first line nivolumab in combination with doxorubicin, vinblastine, and dacarbazine (NAVD) in patients with classic Hodgkin lymphoma (cHL) – a multicenter cohort study (ABSTRACT 25-2368) Safety and efficacy of brexucabtagene autoleucel in elderly patients with relapsed or refractory Mantle Cell Lymphoma: A retrospective, multicenter, international study (ABSTRACT 25-2034) Developing artificial intelligence-based transcriptomic signature for the diagnosis of dark zone lymphoma in patients without MYC gene rearrangement (ABSTRACT 25-7855) A multicenter real-world analysis of combined chemotherapy followed by consolidative radiation versus chemotherapy alone in the management of early-stage Hodgkin lymphoma – the HODGKIN25 study (ABSTRACT 25-1471) Nivolumab with doxorubicin, vinblastine, and dacarbazine (NAVD) in older adults with classic Hodgkin lymphoma: Do S1826 results hold up in the real world? (ABSTRACT 25-7840) First-line salvage therapies in relapsed/refractory large B-cell lymphoma after second- or third-line CD19-directed CAR T-cell therapy (ABSTRACT 25-3402) Multicenter, randomized Phase II study of epcoritamab for patients with large B-cell lymphomas achieving a partial response after CD19-directed CAR T-cell therapy: Trial in progress (ABSTRACT 25-15528) Zanubrutinib + obinutuzumab + sonrotoclax in patients with treatment-naive chronic lymphocytic leukemia/small lymphocytic lymphoma (TN CLL/SLL): Initial results from an ongoing phase 1/1b study, BGB-11417-101 (ABSTRACT 25-4113) Sustained remissions beyond 4 years with epcoritamab monotherapy: Long term follow-up results from the pivotal EPCORE NHL-1 trial in patients with relapsed or refractory large B-cell lymphoma (ABSTRACT 25-7543) Multiple Myeloma Real-world outcomes with elranatamab in multiple myeloma: A multi-center analysis from the United States multiple myeloma immunotherapy consortium (ABSTRACT 25-2557) Phase 1 study of ktx-1001, a first-in-class oral MMSET/NSD2 inhibitor, demonstrates clinical activity in relapsed/refractory multiple myeloma (ABSTRACT 25-2077) Phase 2 registrational study of anitocabtagene autoleucel for the treatment of patients with relapsed and/or refractory multiple myeloma: Updated results from iMMagine--1 (ABSTRACT 25-4541) Alterations in the gut microbiome and the association of butyrate producers with progression-free survival in multiple myeloma patients undergoing autologous stem cell transplantation (ABSTRACT 25-14794) Talquetamab outcomes in relapsed/refractory myeloma with extramedullary and paraskeletal soft tissue plasmacytomas (ABSTRACT 25-7804) Safety and efficacy of talquetamab in patients with relapsed and refractory multiple myeloma (RRMM) with and without renal impairment (ABSTRACT 25-7724) Enhancing the safety of ciltacabtagene autoleucel in relapsed multiple myeloma (MM): Identification of potentially modifiable risk-factors associated with delayed neurotoxicity and non-relapse mortality (ABSTRACT 25-2357) An open-label, multi-center Phase 2 study to assess the safety and efficacy of burixafor (GPC-100) and propranolol with G-CSF for the mobilization of hematopoietic progenitor cells in patients with multiple myeloma (ABSTRACT 25-14982) Prospective real-world evaluation of SKY92 for risk stratification in multiple myeloma: Comparison with updated ims/IMWG criteria in the prommis study (ABSTRACT 25-8818) Pomalidomide salvage in T-cell engager monotherapy failures: Real-world experience with talquetamab or elranatamab with pomalidomide combinations in heavily pretreated multiple myeloma (ABSTRACT 25-10539) Prolonged elranatamab treatment interruption in patients with relapsed or refractory multiple myeloma (RRMM) is feasible: A retrospective analysis from MagnetisMM-3 (ABSTRACT 25-8338) Talquetamab, a GPRC5D×CD3 bispecific antibody, in combination with pomalidomide in patients with Relapsed/Refractory multiple myeloma: Updated safety and efficacy results from the Phase 1b monumental-2 study (ABSTRACT 25-11949) Prospective study of fluoroquinolone resistance colonization in patients undergoing autologous hematopoietic stem cell transplantation in the treatment of multiple myeloma (ABSTRACT 25-13625) Identifying high-risk profiles and adverse prognoses in relapsed/refractory multiple myeloma treated with bispecific antibodies: A real-world analysis of 943 treatment initiations (ABSTRACT 25-8860) Intratumoral cellular immunotherapy with autologous hyperactivated M1 SIRP α -low macrophages in non-Hodgkin lymphoma: Clinical results from a first-in-human Phase 1 study (ABSTRACT 25-8309) Real-world disease burden and treatment patterns among triple-class–exposed patients with relapsed/refractory multiple myeloma and extramedullary disease in the US: A retrospective analysis using Flatiron Health electronic medical records (ABSTRACT 25-9053) Shared immune features correspond to high-risk multiple myeloma across multiple human subtypes and murine models (ABSTRACT 25-13910) Real-world efficacy and safety of teclistamab in relapsed or refractory multiple myeloma: Results from 87 patients treated by the polish myeloma group (ABSTRACT 25-11305) Myeloproliferative Neoplasms Preliminary data from the Phase I/II study of nuvisertib, an oral investigational selective PIM1 inhibitor, in combination with momelotinib showed clinical responses in patients with relapsed/refractory myelofibrosis (ABSTRACT 25-3882) Safety and efficacy results from A phase 1b study of R289, a dual irak 1/4 inhibitor, in patients with Relapsed/Refractory (R/R) lower risk myelodysplastic syndrome (LR-MDS) (ABSTRACT 25-13480) Nuvisertib, an oral investigational selective PIM1 kinase inhibitor, showed clinical responses strongly correlating with cytokine modulation in patients with relapsed/refractory myelofibrosis in the ongoing global phase I/II study (ABSTRACT 25-2614) Bone marrow microenvironment overlap between vexas and myelodysplastic syndrome demonstrated by targeted transcriptomic and artificial intelligence (ABSTRACT 25-7376) Noncancerous Blood Disorders Reduced intensity haploidentical bone marrow transplantation in children with severe sickle cell disease (SCD): BMT CTN 1507 (ABSTRACT 25-11982) End-of-study results from the ICON3 pines trial, a phase 3, randomized trial of eltrombopag vs. standard first-line treatment for newly diagnosed immune thrombocytopenia in children (ABSTRACT 25-4324) The real-world safety and efficacy of BCMA-directed chimeric antigen receptor T-cell therapy in systemic AL amyloidosis (ABSTRACT 25-2732) Robust HbF induction and improvement of anemia and hemolysis with base editing in sickle cell disease: Safety and efficacy findings from the ongoing BEACON study (ABSTRACT 25-2531) Children and adolescents with sickle cell disease demonstrate improved health-related quality of life and low decisional regret after hematopoietic cell transplantation: A sickle cell transplant advocacy and research alliance (STAR) study (ABSTRACT 25-12128) Increased age-adjusted mortality rates from hemophagocytic lymphohistiocytosis (HLH), 2010-2023 (ABSTRACT 25-7365) Rapid decrease in age-adjusted mortality rates associated with ITP following eltrombopag and romiplostim approvals, but not in TMA following eculizumab approval, 1999-2023 (ABSTRACT 25-9103) Technology B- and T-cell clonality using peripheral blood cell-free RNA (cfRNA) in liquid biopsy (ABSTRACT 25-7865) Not so exclusive: Co-mutations in JAK2, MPL and CALR define distinct hematologic and clonal signatures (ABSTRACT 25-10661) Temporal control of CAR expression enables thymic generation of autoreactive T cells targeting tumor-associated antigens (ABSTRACT 25-8390) About Hackensack Meridian John Theurer Cancer Center Hackensack Meridian John Theurer Cancer Center at Hackensack University Medical Center is recognized as New Jersey's #1 cancer center in New Jersey by U.S. News & World Report. They are also the largest and most comprehensive center dedicated to diagnosis, treatment, management, research, screening, and preventive care as well as survivorship of patients with all types of cancers. The 16 specialized divisions covering the complete spectrum of cancer care have developed a close-knit team of medical, research, nursing, and support staff with specialized expertise that translates into more advanced, focused care for all patients. Each year, more people in the New Jersey/New York metropolitan area turn to John Theurer Cancer Center for cancer care than to any other facility in New Jersey. John Theurer Cancer Center is part of the NCI-designated Lombardi Comprehensive Cancer Center at Georgetown University. Housed within a 781-bed not-for-profit teaching, tertiary care, and research hospital, John Theurer Cancer Center provides state-of-the-art technological advances, compassionate care, research innovations, medical expertise, and a full range of aftercare services that distinguish John Theurer Cancer Center from other facilities. The center expanded to Jersey Shore University Medical Center, serving patients in southern New Jersey and parts of Pennsylvania. Toms River Regional Cancer Center and Saint Joseph's Health are affiliated with the cancer center. For additional information, please visit . SOURCE Hackensack Meridian John Theurer Cancer Center 21% more press release views with Request a Demo

Clinical ResultPhase 2Phase 1ImmunotherapyASH

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

100 Deals associated with Ivosidenib

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KEGG	Wiki	ATC	Drug Bank
D11090	Ivosidenib	L01XX62	DB14568

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Approved

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Indication	Country/Location	Organization	Date
Myelodysplastic Syndromes	United States	Servier Pharmaceuticals LLC	24 Oct 2023
IDH1 Mutation Cholangiocarcinoma	Australia	Servier Laboratories (Australia) Pty Ltd.	06 Apr 2023
IDH1 Mutation Acute Myeloid Leukemia	China	Cornerstone Pharmaceutical Co., Ltd	30 Jan 2022
IDH1 Mutation Acute Myeloid Leukemia	China	Servier Pharmaceuticals LLC	30 Jan 2022
Bile Duct Neoplasms	United States	Servier Pharmaceuticals LLC	25 Aug 2021
Acute Myeloid Leukemia	United States	Servier Pharmaceuticals LLC	20 Jul 2018

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Chondrosarcoma	Phase 3	United States	Institut de Recherches Intern Servier	09 Jul 2024
Chondrosarcoma	Phase 3	United States	Servier Bio-Innovation LLC	09 Jul 2024
Chondrosarcoma	Phase 3	China	Institut de Recherches Intern Servier	09 Jul 2024
Chondrosarcoma	Phase 3	China	Servier Bio-Innovation LLC	09 Jul 2024
Chondrosarcoma	Phase 3	Japan	Institut de Recherches Intern Servier	09 Jul 2024
Chondrosarcoma	Phase 3	Japan	Servier Bio-Innovation LLC	09 Jul 2024
Chondrosarcoma	Phase 3	Australia	Servier Bio-Innovation LLC	09 Jul 2024
Chondrosarcoma	Phase 3	Australia	Institut de Recherches Intern Servier	09 Jul 2024
Chondrosarcoma	Phase 3	Belgium	Servier Bio-Innovation LLC	09 Jul 2024
Chondrosarcoma	Phase 3	Belgium	Institut de Recherches Intern Servier	09 Jul 2024

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06081829 (ClarIDHy, CTgov)	Phase 2	Metastatic Cholangiocarcinoma	12	Ivosidenib	pckybayhtm = wmetybkgai oglizyyshj (znekolyiad, uebazjtntl - ifsbqjnoij) View more	-	09 Dec 2025
ASH2025	Phase 2	CCUS Clonal Cytopenia of Undetermined Significance IDH1 mutation	20	Ivosidenib	jjducqwouy(soptcsccqa) = zawnyoezpd hdbnmuivjh (iiljvqjjhq ) View more	Positive	06 Dec 2025
NCT03173248 (AGILE, ASH2025)	Phase 3	IDH1 Mutation Acute Myeloid Leukemia mutant isocitrate dehydrogenase 1 (mIDH1)	146	Ivosidenib+AZA	lflvqpygap(mgacldjclj) = zutiribnco caashrybqh (qolygkkqlr ) View more	Positive	06 Dec 2025
NCT03173248 (AGILE, ASH2025)	Phase 3		146	Placebo+AZA	lflvqpygap(mgacldjclj) = hppbsykfnj caashrybqh (qolygkkqlr ) View more	Positive	06 Dec 2025
NCT05030441 (ASH2025)	Phase 2	IDH1 Mutation Acute Myeloid Leukemia IDH1/2-mutant	20	Ivosidenib (CCUS)	xtwxqnlutp(cftvqhojpi) = cjjncjknsn ndaylghbfs (omvxtnkjwk ) View more	Positive	06 Dec 2025
NCT05876754 (ProvIDHe, ESMO_ASIA2025)	Phase 3	IDH1 Mutation Cholangiocarcinoma IDH1 Mutation	44	Ivosidenib 500 mg	ibdskfxzxw(vzcdaaclok) = not reached katydpembl (cyixelursc ) View more	Positive	05 Dec 2025
NCT06127407 (CHONQUER, ESMO2025)	Phase 3	IDH1 mutation	75	Ivosidenib 500 mg once daily	dqgzixbilm(boorykzdzo) = ikuuyzjkmk trkslkmfoa (dvubnhlxig ) View more	Negative	17 Oct 2025
NCT06127407 (CHONQUER, ESMO2025)	Phase 3	IDH1 mutation	75	Placebo	dqgzixbilm(boorykzdzo) = nxbffybehr trkslkmfoa (dvubnhlxig ) View more	Negative	17 Oct 2025
NCT05876754 (ProvIDHe, ESMO2025)	Phase 3	Bile Duct Neoplasms Second line mIDH1	249	Ivosidenib 500mg	ngvksoitbf(mafpoisitr) = igapsfcyoe ohdqqnwged (culwisargw ) View more	Positive	17 Oct 2025
NCT05876754 (ProvIDHe, ESMO_GI2025)	Phase 3	IDH1 Mutation Cholangiocarcinoma IDH1 mutated	262	Ivosidenib 500mg	cygtcezugj(ozosknjclg) = rlkdddwnec nwzhhulwcj (pjbxgjffvs ) View more	Positive	05 Jul 2025
NCT06081829 (ESMO_GI2025) Manual	Phase 2	Metastatic Cholangiocarcinoma Second line IDH1 Mutation	12	Ivosidenib 500 mg	kficoysebv(xfxdbccrlk) = mrgubmdpop axhkdfdslz (gxthzppcsy, 8.1 - 46.5) Met View more	Positive	03 Jul 2025
ESMO_GI2025	Not Applicable	IDH1 Mutation Cholangiocarcinoma Second line IDH1m	46	Ivosidenib 500 mg once daily in 28-day cycles	rxkrfchmga(fdnfzzhzty) = ozitjtfsov qbckhfxieb (hcwlkqcrwz, 2.2 - 36.5) View more	Positive	03 Jul 2025
ESMO_GI2025	Not Applicable	IDH1 Mutation Cholangiocarcinoma Second line IDH1m	46	FOLFOX/CAPOX	rxkrfchmga(fdnfzzhzty) = otejwzndki qbckhfxieb (hcwlkqcrwz ) View more	Positive	03 Jul 2025

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