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TAK-226

Phase 2

Myelodysplastic Syndromes | Small molecule | Hematology |Takeda Pharmaceutical Company Limited|Last Updated: Jan 6, 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
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment27
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07319845A Study of TAK-226 for Transfusion-Dependent Anemia in Japanese Patients With Lower-Risk Myelodysplastic SyndromesPHASE2 NOT YET_RECRUITING 27Feb 6, 2026Jan 10, 2033Jan 6, 2026 -
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Study Endpoints
Primary Endpoints
Percentage of Participants Achieving Transfusion Independence (TI) for Greater Than or Equal to 8 Weeks from Baseline through Week 24
Baseline, Up to Week 24

Transfusion independence is defined as the absence of any red blood cells (RBC) transfusions in a period of at least 8 weeks after the first dose of the study treatment through week 24.

Secondary Endpoints
Percentage of Participants Achieving TI for Greater Than or Equal to 24 Weeks from Baseline through Week 48
Baseline, Up to Week 48
Percentage of Participants with High Transfusion Burden (HTB) Achieving TI for Greater Than or Equal to 8 Weeks from Baseline through Week 24
Baseline, Up to Week 24
Percentage of Participants Achieving Mean Haemoglobin (Hgb) Increase of Greater Than or Equal to 1.5 grams per deciliter (g/dL) for Greater Than or Equal to 8 Weeks from Baseline through Week 24
Baseline, Up to Week 24
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
TAK-226EXPERIMENTALParticipants will receive the study drug, TAK-226, administered subcutaneously every four weeks (Q4W), up to 48 weeks or until the end of for approximately one year during Treatment Period.
Interventions
NameTypeDescription
TAK-226DRUGTAK-226 subcutaneous injection
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo

Inclusion Criteria: 1. Participants or their legally authorized representative must be willing and able to sign the ICF and to adhere to the protocol requirements. 2. Japanese adult male or female participant ≥18 years of age at the time of signing informed consent. 3. Diagnosis of MDS with or with...

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Competitive Landscape -Myelodysplastic Syndromes 76 trials
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07319845primaryCompletionDate: changed
LOWMay 24, 2026NCT07319845studyFirstPostDate: changed