marked.bio

IDH1 inhibitor · small molecule

olutasidenib

10 active trials across 2 of our biomarkers, targeting IDH1. 1 active approval for a biomarker on marked.bio.

Also known as FT-2102, Rezlidhia.

Counts only trials that select patients by one of the biomarkers on marked.bio, not every trial of this drug.

Approvals and pivotal results

Biomarker-selected approvals in the United States and the European Union, with the pivotal trial's results and side effects where we have them. Results from different trials are not directly comparable.

TherapyBiomarkerTumourUnited StatesEuropean Union
olutasidenib
FDA, 2022
relapsed or refractory, susceptible IDH1 mutation FDA approval 01/12/2022; companion test Abbott RealTime IDH1 Assay. Boxed warning: differentiation syndrome. Source
Rezlidhia 		IDH1/2 mutation AML Approved 2022 Not approved

2102-HEM-101 single-arm, 147 patients
CR+CRh 35% (27-43)Lasting 25.9 months

Trial details and side effects
Patients
relapsed or refractory IDH1-mutant AML (NCT02719574)
Most common side effects
nausea, fatigue, arthralgia, constipation, leukocytosis, dyspnoea, fever, rash, mucositis, diarrhoea, raised liver enzymes
Notes
FDA approval summary (2022); side effects are those in 20% or more of patients.
Source
FDA approval summary

Biomarkers

BiomarkerActive trialsAll trialsFurthest active phase
IDH1/2 mutation 10 14 Phase 4
FLT3 mutation (ITD/TKD) 1 1 Phase 2

Sponsors

Industry sponsors leading active biomarker-selected trials of olutasidenib.

  1. Rigel Pharmaceuticals 1
  2. Kissei Pharmaceutical 1

Trials

10 of 14 trials shown

  1. Olutasidenib DDI Study in Patients With IDH1 Mutation Positive Malignancies

    Recruiting Phase 4 Rigel Pharmaceuticals Started 2026Primary completion 2027-04 NCT07486713

    olutasidenib FDACYP1A2, CYP2B6, CYP2C8, CYP2C9, CYP2C19, CYP3A4, and OATP1B1 Probe Substrates

    Olutasidenib DDI Study in Patients With IDH1 Mutation Positive Malignancies

    IDH1/2 mutationAML (Acute Myeloid Leukemia), Glioma, Cholangiocarcinoma, Solid Tumor Malignancies

  2. A Clinical Trial of Olutasidenib in Patients With Acute Myeloid Leukemia

    Recruiting Phase 2 Kissei Pharmaceutical Started 2026Primary completion 2030-09 NCT07604064

    olutasidenib FDA

    Patients with IDH1 gene mutation confirmed by central confirmation after relapse or refractoriness

    IDH1/2 mutationRelapsed or Refractory IDH1 Mutation-Positive Acute Myeloid Leukemia

  3. Olutasidenib, Venetoclax, and Azacitidine in IDH1 Mutated Newly Diagnosed Acute Myeloid Leukemia Patients Eligible for Intensive Induction Chemotherapy

    Recruiting Phase 2 Justin Watts, MD Started 2026Primary completion 2029-03 NCT06782542

    olutasidenib FDAvenetoclaxazacitidine

    Newly diagnosed AML Isocitrate dehydrogenase 1 (IDH1) R132 mutated disease as assessed locally.

    IDH1/2 mutationAcute Myeloid Leukemia, IDH1 Mutation

  4. Olutasidenib in Relapsed IDH1 Mutated AML Patients Who Have Previously Received Venetoclax

    Recruiting Phase 2 Timothy Pardee Started 2026Primary completion 2028-06 NCT07471841

    olutasidenib FDAazacitidine

    Acute Myeloid Leukemia with a documented IDH1 mutation.

    IDH1/2 mutationIDH1 Mutation, Relapsed / Refractory AML

  5. Testing the Use of an IDH1 Inhibitor, Olutasidenib, in Acute Myeloid Leukemia Added to ASTX727 and Venetoclax; in High-Risk MDS Added to ASTX727; and Alone in Low Risk MDS (A MyeloMATCH Treatment Substudy)

    Not yet recruiting Phase 2 National Cancer Institute (NCI) Started 2026Primary completion 2030-04 NCT07153497

    decitabineolutasidenib FDAvenetoclax

    …n the MYELOMATCH Master Screening and Reassessment Protocol (MSRP), determined to have an IDH1-R132 mutation and assigned to this trial via MATCHBox

    IDH1/2 mutationAcute Myeloid Leukemia, Myelodysplastic Syndrome

  6. Olutasidenib With Azacitidine Followed by Olutasidenib Maintenance for the Treatment of IDH1-mutated Acute Myeloid Leukemia in Patients With Prior Treatment With Venetoclax Plus a Hypomethylating Agent

    Recruiting Phase 2 University of California, Davis Started 2025Primary completion 2027-12 NCT07304011

    azacitidineolutasidenib FDA

    Documented IDH1-R132 mutations (≥ 0.01%) detected in the bone marrow or blood.

    IDH1/2 mutationAcute Myeloid Leukemia

  7. Olutasidenib Combined With Co-targeted Therapy in Relapsed or Refractory IDH1-mutated Myeloid Malignancies Harboring Activated Signaling Pathway Mutations

    Recruiting Phase 2 M.D. Anderson Cancer Center Started 2025Primary completion 2027-06 NCT07032727

    olutasidenib FDAcladribinevenetoclaxgilteritinibRuxolitinibcytarabine

    Participants must have a documented IDH1 mutation.

    IDH1/2 mutationFLT3 mutation (ITD/TKD)Targeted Therapy, IDH1-Mutated Malignancies, Mutations

  8. A Phase 2 Study Evaluating Olutasidenib in Patients With IDH1-mutated Clonal Cytopenia of Undetermined Significance and Lower-risk Myelodysplastic/Syndromes/Chronic Myelomonocytic Leukemia.

    Recruiting Phase 2 M.D. Anderson Cancer Center Started 2024Primary completion 2027-08 NCT06566742

    olutasidenib FDA

    A Phase 2 Study Evaluating Olutasidenib in Patients With IDH1-mutated Clonal Cytopenia of Undetermined Significance and Lower-risk Myelodysplastic/Syndromes/Ch…

    IDH1/2 mutationMyelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, Clonal Cytopenia of Undetermined Significance

  9. A Phase II Open-label Study of Olutasidenib Post-transplant Maintenance Therapy for Patients With IDH1-mutated Myeloid Malignancies

    Recruiting Phase 2 M.D. Anderson Cancer Center Started 2024Primary completion 2027-12 NCT06668584

    olutasidenib FDA

    Evdence of an IDH1 mutation presence by next generation sequencing panel at original diagnosis.

    IDH1/2 mutationMyeloid Malignancies

  10. Olutasidenib Single Plus Combo Therapy in IDH1mut AML After Induction and Consolidation

    Recruiting Phase 1 Virginia Commonwealth University Started 2026Primary completion 2028-10 NCT07130695

    olutasidenib FDA

    …ologically or cytologically confirmed non-acute promyelocytic isocitrate dehydrogenase (1 IDH1) mutant acute myeloid leukemia (AML).

    IDH1/2 mutationAcute Myeloid Leukemia