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Ivosidenib

Phase 3

Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) | Small molecule | Hematology |Tango Therapeutics, Inc.|Last Updated: May 11, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment48
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06465953Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an IDH1 MutationPHASE3 RECRUITING 48Dec 3, 2024Dec 1, 2028May 11, 202662 United States, Australia +8
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Study Endpoints
Primary Endpoints
Number of participants achieving CR and PR by 4 months
Through 4 months after starting treatment

Complete remission (CR) or Partial remission (PR) as per International Working Group (IWG) 2006 criteria

Secondary Endpoints
Overall Response (OR) rate per IWG 2023 criteria
Through the end of the study (approximately 4 years)
Event-free survival (EFS)
Through the end of the study (approximately 4 years)
Overall Survival (OS)
Through the end of the study (approximately 4 years)
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Ivosidenib monotherapyEXPERIMENTAL -
Azacitidine monotherapyEXPERIMENTAL -
Interventions
NameTypeDescription
IvosidenibDRUGTwo 250 mg tablets, totaling 500 mg, administered orally once daily until disease relapse or progression, unacceptable toxicity, confirmed pregnancy, undergoing HSCT, death, withdrawal of consent, lost to follow-up, or Sponsor ending the study, whichever occurs first.
AzacitidineDRUGAzacitidine 75mg/m\^2/day administered by subcutaneous (SC) or intravenous (IV) injection for 1 week (7 days) of each 4-week (28 day) treatment cycle until disease relapse or progression, unacceptable toxicity, confirmed pregnancy, undergoing HSCT, death, withdrawal of consent, lost to follow-up, or Sponsor ending the study, whichever occurs first.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites62

Inclusion Criteria: * Diagnosis of HMA naive IDH1 R132 mutated MDS defined according to WHO criteria (5th edition): * Moderate high, high and very high-risk MDS per IPSS-M score will be eligible regardless of blood counts and with blast counts 0-19%. * Low and moderate low-risk MDS per IPSS-M score...

Countries:United StatesAustraliaBrazilFranceGermanyItalyJapanNetherlandsSpainUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT06465953studyFirstPostDate: changed