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Venetoclax

Phase 1

Acute Myeloid Leukemia | Small molecule | Oncology |Kura Oncology, Inc.|Last Updated: Dec 15, 2025

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 Enrollment22
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06448013A Phase I Study Investigating the Combination of the Menin Inhibitor Ziftomenib With Venetoclax and Gemtuzumab in Pediatric Patients With Acute Myeloid LeukemiaPHASE1 RECRUITING 22Mar 7, 2025Dec 31, 2030Dec 15, 20251 United States
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Study Endpoints
Primary Endpoints
Safety and adverse events (AEs)
Through study completion; an average of 1 year

Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0

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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Dose Escalation + Dose ExpansionEXPERIMENTALParticipants found to be eligible to take part in this study, you will be assigned to a dose level of ziftomenib based on when you join this study. Up to 4 dose levels of ziftomenib will be tested. Up to 6 participants will be enrolled at each dose level. The first group of participants will receive the lowest dose level. Each new group will receive a higher dose than the group before it, if no intolerable side effects were seen. This will continue until the recommended dose of ziftomenib is found.
Interventions
NameTypeDescription
VenetoclaxDRUGGiven by PO
GemtuzumabDRUGGiven by IV
ZiftomenibDRUGGiven by PO
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Eligibility Criteria
Age Range3 Years — 21 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: 1. Age ≥ 3 year to 21 years 2. Karnofsky for children \>16yo and Lansky \<16yo. 3. Relapsed/refractory AML, or MPAL with a myeloid phenotype. 1. Evidence of leukemia (AML, MPAL with a myeloid phenotype) in the bone marrow as detected by morphology or molecular diagnostics. ...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06448013primaryCompletionDate: changed
LOWMay 24, 2026NCT06448013studyFirstPostDate: changed