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Venetoclax

Phase 3

Myelodysplastic Syndrome (MDS) | Small molecule | Hematology |AbbVie Inc.|Last Updated: May 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
RandomizedDouble-BlindACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment531
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04401748Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic SyndromePHASE3 ACTIVE NOT_RECRUITING 531Sep 10, 2020Jul 1, 2027May 6, 2026220 United States, Australia +21
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Study Endpoints
Primary Endpoints
Overall survival (OS)
Up To 5 Years

OS is defined as the number of days from the date of randomization to the date of death of any cause, or last known date to be alive.

Secondary Endpoints
Modified Overall Response (mOR)
Up To 5 Years
Percentage of Participants Achieving Overall Hematological Improvement (HI)
Up to 5 Years
Complete Remission (CR)
Up To 36 Months
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Arm 1: Venetoclax + Azacitidine (AZA)EXPERIMENTALParticipants will receive venetoclax once daily (QD) (Days 1-14) in combination with AZA QD (7 days of the first 9 days) of each 28 day cycle.
Arm 2: Placebo + AzacitidineACTIVE_COMPARATORParticipants will receive placebo once daily (QD) (Days 1-14) in combination with AZA QD (7 days of the first 9 days) of each 28 day cycle.
Interventions
NameTypeDescription
VenetoclaxDRUGTablet: Oral
AzacitidineDRUGSubcutaneous (SC) or Intravenous (IV) injection
PlaceboDRUGTablet; Oral
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites220

Inclusion Criteria: * Participants with a diagnosis of Myelodysplastic Syndrome (MDS) according to the 2016 World Health Organization (WHO) classification wtih presence of \< 20% bone marrow blasts per marrow biopsy/aspirate at screening. * Participants must meet the following disease activity crit...

Countries:United StatesAustraliaAustriaBelgiumBrazilCanadaChinaCzechiaFranceGermanyHungaryIsraelItalyJapanNetherlandsPolandPuerto RicoRussiaSouth KoreaSpainTaiwanTurkey (Türkiye)United Kingdom
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Competitive Landscape -Myelodysplastic Syndromes 76 trials
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Recent Changes (Last 90 Days)
MEDIUMMay 26, 2026NCT04401748primaryCompletionDate: changed
LOWMay 24, 2026NCT04401748studyFirstPostDate: changed