Tibsovo (ivosidenib) / CStone Pharma, Servier, Schrodinger, Sagard Healthcare - LARVOL DELTA

Long-term results from the AGILE study of azacitidine plus ivosidenib vs placebo in newly diagnosed IDH1-mutated AML. (PubMed, Blood Adv) - P3 | "These long-term efficacy and safety results confirm the benefit of ivosidenib-azacitidine in this challenging-to-treat population and support its use as a standard of care with the longest reported survival benefit for intensive chemotherapy-ineligible patients with IDH1-mutated AML. ClinicalTrials.gov registration ID: NCT03173248."

Journal • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • IDH1

Comparative Efficacy and Safety of Biomarker-Guided Targeted Therapies Versus Conventional Treatment in Advanced Biliary Tract Cancer: A Systematic Review and Network Meta-Analysis (ACG 2026) - "14 studies involving 3,842 patients were included, comprising 2,116 males (55.1%) and 1,726 females (44.9%), with median age ranging from 58â69 years. Evaluated therapies included FGFR inhibitors (pemigatinib, futibatinib, infigratinib), the IDH1 inhibitor ivosidenib, HER2-directed regimens (trastuzumab-based therapies and zanidatamab), dabrafenib plus trametinib, zenocutuzumab, and conventional chemotherapy/immunotherapy controls. Most patients had metastatic disease (81.6%), ECOG 0â1 status (87.3%), and prior systemic therapy exposure."

Biomarker • IO biomarker • Metastases • Retrospective data • Review • Biliary Cancer • Biliary Tract Cancer • Oncology • Solid Tumor • FGFR2 • IDH1 • NRG1

Mechanisms of Ivosidenib Resistance Identified by ctDNA Profiling in Patients with IDH1-Mutated Cholangiocarcinoma: Findings from the Phase 3b ProvIDHe Study (ESMO 2026) - No abstract available

Circulating tumor DNA • Clinical • P3 data • Biliary Cancer • Cholangiocarcinoma • Oncology • Solid Tumor • IDH1

Phase 3 study of ivosidenib vs placebo in locally advanced or metastatic IDH1-mutant conventional chondrosarcoma untreated or previously treated with 1 systemic treatment regimen (CHONQUER). (ASCO 2025) - P1, P3 | "Other secondary endpoints include PFS by investigator, overall response, duration of response, time to response, disease control, duration of disease control, adverse events, and health-related quality of life. 92 sites from 12 countries are planned to participate, including North and South America, Europe and Asian countries."

Clinical • Metastases • P3 data • Oncology • Sarcoma • Solid Tumor • IDH1 • IDH2

IVOSIDENIB MONOTHERAPY IN IDH1 MUTATED MYELODYSPLASTIC SYNDROME, FINAL RESULTS OF THE IDIOME TRIAL, A GFM STUDY (EHA 2024) - P2 | "Background: Ivosidenib (IVO) is an oral, targeted, small-molecule inhibitor of mutant IDH1 (IDH1m) approved in associationwith azacytidine (AZA) for unfit adult patients with acute myeloid leukemia (AML) and as a single agent forrelapse/refractory AML. IVO monotherapy was associated with significant responses in all IDH1m MDS patients cohorts and, in this frailpopulation, was well tolerated. The high OR rate and prolonged OS observed in treatment naive HR-MDSpatients suggest that IVO monotherapy could be a first-line treatment in this population including incandidates for HSCT. Figure 1:"

Clinical • Monotherapy • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Myelomonocytic Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • IDH1

A phase Ib/II study of ivosidenib with venetoclax +/- azacitidine in IDH1-mutated hematologic malignancies. (ASCO 2022) - P1/2 | "IVO+VEN +/- AZA is an effective treatment for IDH1+myeloid malignancies with an expected toxicity profile and notable efficacy across disease groups. Single-cell sequencing and CyTOF correlatives will also be presented. Phase 2 enrollment is ongoing."

IO biomarker • P1/2 data • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Neutropenia • Oncology • Pneumonia • Respiratory Diseases • IDH1

Single-Center Experience with Vorasidenib in Patients with Grade 3 or 4 IDH-Mutant Gliomas (EANO 2026) - "Prior treatments included surgical resection (47, 84%), radiation (45, 80%), temozolomide (44, 78%), ivosidenib (13, 23%), bevacizumab (11, 20%), lomustine (10, 18%), lomustine + procarbazine (7, 13%), and pembrolizumab (4, 7%). In this heavily pre-treated cohort of patients with high-grade IDH mutant gliomas, vorasidenib was well-tolerated and associated with promising 6 and 12-month PFS rates. Optimal role of the agent in the treatment of high-grade gliomas requires prospective evaluation."

Clinical • Anaplastic Astrocytoma • Astrocytoma • Brain Cancer • Glioblastoma • Glioma • High Grade Glioma • Oligodendroglioma • Oncology • Solid Tumor

Ivosidenib in predominantly recurrent IDH1-mutant glioma: A single-institution experience. (PubMed, Neurooncol Pract) - "In this retrospective analysis of predominantly recurrent IDH-1-mutant glioma, ivosidenib was associated with longer PFS in lower-grade, non-enhancing tumors that received little or no prior anticancer treatment and less complex genomic profiles. These findings support a context-dependent benefit of IDH inhibition in recurrent disease."

Journal • Astrocytoma • Brain Cancer • Glioma • Oligodendroglioma • Oncology • Solid Tumor • CDKN2A • CDKN2B • IDH1

Final Overall Survival Efficacy Results of Ivosidenib for Patients With Advanced Cholangiocarcinoma With IDH1 Mutation: The Phase 3 Randomized Clinical ClarIDHy Trial. (PubMed, JAMA Oncol) - P3 | "These data, coupled with supportive quality of life data and a tolerable safety profile, demonstrate the clinical benefit of ivosidenib for patients with advanced cholangiocarcinoma with IDH1 mutation. ClinicalTrials.gov Identifier: NCT02989857."

Clinical • Journal • P3 data • Biliary Cancer • Cholangiocarcinoma • Gastrointestinal Cancer • Oncology • Solid Tumor • IDH1

Effectiveness of olutasidenib versus ivosidenib in patients with mutated isocitrate dehydrogenase 1 acute myeloid leukemia who are relapsed or refractory to venetoclax: the 2102-HEM-101 trial versus a US electronic health record-based external control arm. (PubMed, Leuk Lymphoma) - "Following weighting, treatment with OLU versus IVO was associated with significantly higher rates of complete response (RD: 0.25; 95%CI: 0.01, 0.49), transfusion independence (RD: 0.27; 95%CI: 0.01, 0.53), and OS (HR: 0.33; 95%CI: 0.11, 0.94). Results suggest favorable effectiveness of OLU versus IVO in this population."

Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • IDH1

A Phase I, Single-Center, Open-Label, Dose De-escalation and Expansion Study of Ivosidenib Plus mFOLFIRINOX in Patients With Resectable Pancreatic Ductal Adenocarcinoma (PDA) (ESMO 2026) - No abstract available

Clinical • Oncology • Pancreatic Ductal Adenocarcinoma

Allosteric kinase inhibition in hematological malignancies: from asciminib to emerging regulatory sites. (PubMed, Biochem Pharmacol) - "This review fills that gap with two contributions: mechanistic evidence that crizotinib engages BCR::ABL1 through a putative dual ATP-site/myristoyl-pocket mechanism, supported by indirect evidence and pending direct structural confirmation; and a hypothesis linking recurrent synonymous mutations in non-receptor tyrosine kinases to transiently structured regulatory regions, as a strategy for identifying latent allosteric sites Asciminib is the proof of concept...In the ASCEMBL trial, it achieved a major molecular response rate of 25.5% at 24 weeks versus 13.2% for bosutinib in heavily pretreated CML, with better tolerability-the first regulatory-site inhibitor approved for a hematological malignancy...Asciminib resistance is already real: A337V and P465S mutations reduce binding, and bypass signaling adds another layer. Each approved allosteric agent-asciminib, trametinib, and ivosidenib-required extensive structural and functional validation before reaching the clinic;..."

Journal • Review • Chronic Myeloid Leukemia • Hematological Disorders • Hematological Malignancies • Oncology • ABL1 • FLT3 • JAK2

Low-Intensity Metronomic Azacitidine Plus Venetoclax-Based Regimen Induces Complete Remission in Two Frail High-Risk MDS Patients With Severe Bleeding Risk (SOHO 2026) - "In Case 2, the patient presented with new-onset ICH, severe thrombocytopenia (platelet count, 17 × 109/L), and 12.37% aberrant blasts by flow cytometry and was treated with the same low-dose metronomic regimen, with ivosidenib (0.5 g once daily) added on day 22. A low-intensity weekly AZA plus VEN-based regimen represents a safe and viable salvage paradigm for frail high-risk MDS complicated by catastrophic bleeding. Extended-interval dosing provides a window for hematopoietic recovery while maintaining deep clonal suppression. NOTCH1: notch receptor 1, TP53: tumor protein p53."

Clinical • Colorectal Cancer • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • Rectal Cancer • Solid Tumor • IDH1 • NOTCH1 • TP53

Multi-center phase I trial of Ivosidenib as Maintenance Treatment following Allogeneic Hematopoietic Cell Transplantation for IDH1-Mutated Acute Myeloid Leukemia. (PubMed, Clin Cancer Res) - "Ivosidenib is safe and well-tolerated as maintenance therapy following HCT. Cumulative incidence of relapse and NRM, as well as estimations of PFS and OS, were promising in this phase 1 study."

Journal • P1 data • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Leukemia • Oncology • Transplantation • IDH1

Phase Ib/2 Study of Oral Decitabine/Cedazuridine (ASTX727) and Venetoclax in Combination with the Targeted Mutant IDH1 Inhibitor Ivosidenib or the Targeted Mutant IDH2 Inhibitor Enasidenib in IDH Mutated Acute Myeloid Leukemia (ASH 2022) - "(Table 1) In the R/R cohort, 78% (n=11) patients had received prior treatment with either an HMA (azacitidine or decitabine), BCL2i and/or IDHi, with a median of 2 prior treatments...There were two patients with possible/probable differentiation syndrome which resolved with medical management (dexamethasone and diuresis)...AEs are anticipated and tolerable. Enrollment to this study is ongoing."

Combination therapy • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Febrile Neutropenia • Hematological Malignancies • Hepatology • Mucositis • Myelodysplastic Syndrome • Neutropenia • Transplantation • IDH1

Phase I results of a multicenter, open-label, dose de-escalation and expansion study of gemcitabine and cisplatin with ivosidenib or pemigatinib for advanced cholangiocarcinoma. (PubMed, Invest New Drugs) - "Adding ivosidenib to gemcitabine and cisplatin in this study demonstrated a challenging safety profile in advanced CCA. Further research and dose optimization are warranted to confirm these findings and optimize the integration of targeted therapies into first-line regimens."

IO biomarker • Journal • P1 data • Biliary Cancer • Biliary Tract Cancer • Cholangiocarcinoma • Oncology • Solid Tumor • FGFR2 • IDH1

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 (ASH 2025) - P1, P3 | "Introduction Ivosidenib (IVO) is approved as monotherapy and in combination with azacitidine for frontline treatmentof patients (pts) with mIDH1 acute myeloid leukemia (AML) unfit for intensive chemotherapy (chemo)...Pts with ND mIDH1AML received induction therapy: cytarabine 200 mg/m2/d × 7 d and either daunorubicin 60 mg/m2/d oridarubicin 12 mg/m2/d × 3 d (up to 2 cycles of induction were permitted) and IVO 500 mg once dailystarting on d 1 of induction therapy...IVO maintenance has an acceptable safety profile, is associated with stablenormalization of blood counts, and results in durable responses and long-term survival acrosscomutational profiles. The benefit of this frontline regimen is being assessed in a phase 3 randomized,blinded trial (NCT03839771)."

Clinical • P1 data • Acute Kidney Injury • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Leukopenia • Neutropenia • Renal Disease • Thrombocytopenia • TP53

Preliminary efficacy and safety of ivosidenib in combination with PD-1/PD-L1 inhibitors and lenvatinib in advanced IDH1-mutant intrahepatic cholangiocarcinoma (ESMO 2026) - No abstract available

Clinical • Combination therapy • IO biomarker • Metastases • Biliary Cancer • Cholangiocarcinoma • Oncology • Solid Tumor • IDH1

Methods for assessing binding of a small-molecule radioligand to soluble enzyme isocitrate dehydrogenase 1 variants. (PubMed, EJNMMI Radiopharm Chem) - "The findings support the use of radioligand-based approaches for characterizing soluble intracellular protein targets, while highlighting the need for assay-specific optimization and careful interpretation of quantitative binding parameters."

Journal • Brain Cancer • Glioma • Oncology • Solid Tumor • IDH1

Updated efficacy and safety data from the AGILE study in patients with newly diagnosed acute myeloid leukemia treated with ivosidenib + azacitidine compared to placebo + azacitidine. (ASCO 2023) - P3 | "Updated data on OS (>5 months longer mOS and a greater risk reduction in deaths compared to the initial analysis), transfusion independence, blood count recovery and safety confirms the clinically and statistically robust benefit in favor of IVO+AZA at long-term follow up. Patient disposition. Clinical trial information: NCT03173248."

Clinical • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Oncology • IDH1

Mutations in IDH1 and IDH2 portend a poor prognosis in adult patients with acute lymphoblastic leukemia (ASH 2025) - "One pt with IDH1mut T-ALL and 2 ptsIDH2mut T-ALL received ivosidenib and enasidenib, respectively, in salvage: the IDH1mut pt had noresponse and the 2 IDH2mut pts achieved CR with a median duration of response of 17 months (3-31)before relapsing.The median event-free survival (EFS) for IDHmut pts was 16 months, with a 3-year EFS rate of 28%, vs. amedian EFS of 69 months and a 3-year EFS rate of 60% in IDHwt pts (p=0.0005). Co-occurring mutations include DNMT3A, NOTCH1, and NRAS, which were enriched inpts with T-ALL. Novel therapeutic strategies, including venetoclax and IDH inhibitors, are warranted inthis high-risk subgroup."

Clinical • Acute Lymphocytic Leukemia • Hematological Malignancies • Leukemia • T Acute Lymphoblastic Leukemia • BCOR • DNMT3A • IDH1 • IDH2 • IKZF1 • JAK1 • KRAS • NOTCH1 • NRAS • SRSF2 • TET2 • TP53

A MULTICENTER PHASE 1B/2 TRIAL OF AZACITIDINE, VENETOCLAX, AND IVOSIDENIB IN IDH1-MUTATED ACUTE MYELOID LEUKEMIA (AML) (EHA 2026) - P1/2 | "Reused with permission. This abstract was accepted and previously presented at the 2026 ASCO Annual Meeting."

Clinical • P1/2 data • Acute Myelogenous Leukemia • FLT3 • IDH1 • NPM1 • TP53

AGILE: A Global, Randomized, Double-Blind, Phase 3 Study of Ivosidenib + Azacitidine Versus Placebo + Azacitidine in Patients With Newly Diagnosed Acute Myeloid Leukemia With an IDH1 Mutation (SOHO 2022) - "In patients with ICineligible, newly diagnosed mIDH1 AML, IVO+AZA signifi cantly improved EFS, OS, and clinical response compared with PBO+AZA. Blood counts rapidly recovered in patients given IVO+AZA, patients were less dependent on RBC/platelet transfusion than those given PBO+AZA, with HRQoL improvements in the IVO+AZA group. The safety profi le of IVO+AZA was favorable, with fewer febrile neutropenia and infection events with IVO+AZA vs PBO+AZA."

Clinical • P3 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • IDH1

Targeting IDH1: Long-Term Efficacy and Tolerance of Ivosidenib in Advanced Chondrosarcoma. (PubMed, Acta Med Port) - "These cases highlight the usefulness of ivosidenib as a targeted therapeutic option for patients with metastatic or recurrent unresectable chondrosarcomas with IDH1 mutation. Long-term efficacy with very good tolerance was achieved."

Journal • Oncology • Sarcoma • Solid Tumor • IDH1

Baseline characteristics and molecular testing of patients with IDH1-mutated cholangiocarcinoma: Initial results from the phase IIIb ProvIDHe study (ESMO Asia 2024) - P3 | "Background Ivosidenib (IVO) has demonstrated efficacy as an oral inhibitor of the protein encoded by the mutant isocitrate dehydrogenase 1 ( mIDH1 ) gene in patients (pts) with cholangiocarcinoma (CCA) in the phase III ClarIDHy study...The median number of prior systemic regimens for advanced/metastatic disease was 2 (range, 1-6), most commonly gemcitabine + cisplatin alone (39.7%) or with immunotherapy (35.1%) Table: 138MO Baseline characteristics Baseline characteristic N=158 Median age, years (range) 62.0 (33, 85) Sex Male 67 (42.4) Female 91 (57.6) ECOG PS 0 72 (45.6) 1 76 (48.1) Missing 10 (6.3) CCA stage at diagnosis Metastatic 81 (51.3) Local/regional 44 (27.8) Advanced 30 (19.0) Missing 3 (1.9) CCA primary site Intrahepatic 142 (89.9) Perihilar 3 (1.9) Distal 2 (1.3) Unknown 8 (5.1) Missing 3 (1.9) Number of previous systemic therapy regimens for advanced/metastatic disease* Median (range) 2 (1-6) 1 37 (28.2) 2 30 (22.9) >2 39 (29.8) CCA,..."

Clinical • IO biomarker • P3 data • Biliary Cancer • Cholangiocarcinoma • Gastrointestinal Cancer • Oncology • Solid Tumor • BRAF • FGFR2 • IDH1 • PIK3CA