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Ivosidenib+ azacitidine

Phase 2

Acute Myeloid Leukemia | Small molecule | Oncology |Tango Therapeutics, Inc.|Last Updated: Mar 11, 2026

Target and mechanism

Molecular targetIDH1
Target classInhibitor
ModalitySmall molecule

Also known as Ivosidenib, Ivosidenib Oral Tablet, Ivosidenib 500mg, Ivosidenib 500mg Oral Tablet

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment60

FDA Designations

No designations recorded

Clinical trial landscape

Ivosidenib+ azacitidine · 1 trial · 1 indication

Phase 2 1
NCT07463768Study Evaluating 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.Acute Myeloid Leukemia
NOT YET_RECRUITING60 Analytics
PHASE2NOT YET_RECRUITING
Study Evaluating 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.
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Study Endpoints

Primary Endpoints

RFS 24 months
24 months

relapse-free-survival (RFS) at 24 months in patients receiving oral azacitidine with ivosidenib

Secondary Endpoints

RFS 12 months
12 months
Overall survival
24 months
EFS MRD neg
24 months
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ivosidenib + Oral azacitidineEXPERIMENTALcf intervention

Interventions

NameTypeDescription
Ivosidenib + oral azacitidineDRUGSubjects will be administered treatment over 28-day cycles until progression or another reason (unacceptable toxicity, patient choice to discontinue study treatment, sponsor decision, HSCT at any time, patients who require the use of any of the excluded therapies). The treatment is the association of ivosidenib with oral azacitidine. * The investigational medicinal product is ivosidenib. Two tablets (500 mg) will be administered as a single dose, once daily, during 28 days-cycles until discontinuation. * The auxiliary treatment is oral azacitidine. Oral AZA 300 mg will be given once-daily for 14 days of repeated 28-day treatment cycles. For all patients, oral AZA will be interrupted systematically after day 14 of each 28-days cycle.
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Eligibility Criteria

Age Range55 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites20

Inclusion Criteria: 1. Male or female ≥ 55 years of age at the time of signing informed consent 2. Patients with confirmation of newly diagnosed AML by 2022 WHO criteria 3. Presence of IDH1 R132 mutation at AML diagnosis 4. Achievement CR or CRi following induction therapy by intensive chemotherapy...

Countries:France
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Frequently asked questions about Ivosidenib+ azacitidine

What is Ivosidenib used for?

Ivosidenib is an investigational small molecule being studied for acute myeloid leukemia, cholangiocarcinoma, clonal cytopenia of undetermined significance, IDH1-mutated malignancies, and locally advanced or metastatic conventional chondrosarcoma with an IDH1 mutation. It is also being evaluated in combination with oral azacitidine for AML patients in complete remission after intensive chemotherapy.

What does Ivosidenib target?

Ivosidenib targets IDH1 mutations. It is a kinase inhibitor, belonging to the -nib class of drugs. The drug is being studied in biomarker-selected patient populations with IDH1-mutated cancers, including acute myeloid leukemia, cholangiocarcinoma, and chondrosarcoma.

Who makes Ivosidenib?

Ivosidenib is being developed by Tango Therapeutics, Inc. (NASDAQ: TNGX). The company is conducting clinical trials of the drug across multiple oncology indications, including a Phase 3 study in chondrosarcoma and Phase 2 studies in other IDH1-mutated malignancies.

What phase is Ivosidenib in?

Ivosidenib is in Phase 3 clinical development for locally advanced or metastatic conventional chondrosarcoma with an IDH1 mutation. It is also being studied in Phase 2 trials for clonal cytopenia of undetermined significance, cholangiocarcinoma, and acute myeloid leukemia. The drug remains investigational and is not yet approved.

What clinical trials is Ivosidenib in?

Ivosidenib is being studied in four clinical trials. NCT06127407 is a Phase 3 trial in chondrosarcoma. NCT05030441 is a Phase 2 trial in clonal cytopenia of undetermined significance. NCT06081829 is a Phase 2 trial in cholangiocarcinoma. NCT07463768 is a Phase 2 trial combining ivosidenib with oral azacitidine in AML.

Is Ivosidenib the same as Ivosidenib plus oral azacitidine?

Ivosidenib is the single-agent drug, while Ivosidenib plus oral azacitidine refers to a combination regimen being studied in acute myeloid leukemia. The combination is being evaluated in a Phase 2 trial for patients over 55 with an IDH1 mutation who are in complete remission after intensive chemotherapy.