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Eltanexor

Phase 1

Relapsed Myelodysplastic Syndrome | Small molecule | Oncology |Karyopharm Therapeutics Inc.|Last Updated: Mar 27, 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 Enrollment60
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06399640Eltanexor and Venetoclax in Relapsed or Refractory Myelodysplastic Syndrome and Acute Myeloid LeukemiaPHASE1 RECRUITING 60Aug 14, 2024Oct 1, 2027Mar 27, 20251 United States
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Study Endpoints
Primary Endpoints
Incidence of adverse events
Up to 2 years

Adverse medical events will be tabulated and graded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 5.

Biologically effective dose (BED) of eltanexor in combination with venetoclax
Up to 2 years

Measured by complete remission

Secondary Endpoints
Complete remission
Up to 2 years
Overall response rate
Up to 2 years
Progression free survival
Up to 2 years
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Eltanexor + VenetoclaxEXPERIMENTALParticipants receive eltanexor PO QD for 5 days per week for 14, 21, or 28 days every cycle, and venetoclax PO QD on days 1-14 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Participants undergo bone marrow aspiration and biopsy and blood sample collection throughout the study.
Interventions
NameTypeDescription
EltanexorDRUGEltanexor will be taken by mouth
VenetoclaxDRUGVenetoclax will be taken by mouth
Bone Marrow Aspiration and BiopsyPROCEDUREUndergo bone marrow aspiration and biopsy
Biospecimen CollectionPROCEDUREUndergo blood sample collection
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: \- Age \>/= 18 years at the time of signing the Informed Consent Form (ICF); must voluntarily sign an ICF; and must be able to meet all study requirements. For Myelodysplastic Syndrome (MDS): Morphologically confirmed diagnosis of MDS with increased blasts (\>/= 5%), with a pr...

Countries:United States
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Competitive Landscape -Myelodysplastic Syndromes 76 trials
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06399640primaryCompletionDate: changed
LOWMay 24, 2026NCT06399640studyFirstPostDate: changed