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Onureg + Venetoclax

Phase 1

Untreated Myelodysplastic Syndrome | Small molecule | Hematology |AbbVie Inc.|Last Updated: Mar 13, 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
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment36
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05782127Oral Azacitidine Combined With Venetoclax in Previously Untreated Higher-risk Myelodysplastic SyndromesPHASE1 ACTIVE NOT_RECRUITING 36Dec 6, 2023Nov 1, 2028Mar 13, 202625 France
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Study Endpoints
Primary Endpoints
Dose-limiting toxicity
at day 28 of cycle 1

Dose-limiting toxicities according to CTCAE (common terminology criteria for adverses events) 5.0 occurring within the first cycle of treatment

Overall response
at day 28 of cycle 1

Overall response measured after the first cycle of treatment according to modified IWG-MDS (International Working Group-Myelodysplastic Syndromes) 2006

Secondary Endpoints
Best response
after 6 cycles of treatment (each cycle is 28 days)
Hematological improvement
at end of treatment (an average of 4 years)
Time to response
at end of treatment (an average of 4 years)
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Onureg + VenetoclaxEXPERIMENTALOnureg (CC-486, oral azacitidine) will be administered orally at 200 or 300 mg once daily for 7 or 14 consecutive days, beginning on Day 1 of repeated 28-day cycles. Venetoclax will be administered orally at 400 mg once daily for 14 consecutive days on days 1 to 14, beginning on Day 1 of repeated 28-day cycles. Patients will be treated up to 4 cycles and for a maximum of 24 cycles.
Interventions
NameTypeDescription
Onureg + VenetoclaxDRUGCombination of Onureg and Venetoclax
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites25

Inclusion Criteria: 1. Subjects must understand and voluntarily sign and date an ICF indicating the investigational nature of the study, approved by an independent EC/IRB, prior to the initiation of any screening or study-specific procedures. 2. Age ≥ 18 years at the date of signing the ICF. 3. Dia...

Countries:France
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT05782127studyFirstPostDate: changed