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ASTX727

Phase 1

MDS/MPN | Small molecule | Other |Incyte Corporation|Last Updated: Apr 21, 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
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment94
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04061421Active Myeloid Target Compound Combinations in MDS/MPN Overlap Syndromes Overlap Syndromes (ABNL-MARRO)PHASE1 RECRUITING 94Nov 24, 2021Dec 1, 2027Apr 21, 20264 United States
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Study Endpoints
Primary Endpoints
Phase 1: Determination of dose
Up to 28 days

Per CTCAE 5.0

Phase 2: Overall response rate (ORR). Overall response includes patients who achieve best response of CR, PR, MR, or CB as defined by the MDS/MPN IWG proposed response criteria
Up to 2 years
Secondary Endpoints
Adverse Event/DLT severity and frequency
Up to 2 years
CR/MR rate defined as the proportion of patients who achieve either complete remission or marrow response as a best response to treatment.
Up to 2 years
Overall survival (OS)
Up to 2 years
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ASTX727 + itacitinibEXPERIMENTALASTX727 and itacitinib will be taken by mouth
ASTX727 + ruxolitinibEXPERIMENTALASTX727 and ruxolitinib will be taken by mouth
Interventions
NameTypeDescription
ASTX727DRUGTaken by mouth daily during days 1-5 of every 28-day cycle.
ItacitinibDRUGTaken by mouth daily during each 28-day cycle
RuxolitinibDRUGDosage will be 5,15, or 20mg Taken by mouth BID for 28-day cycle
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: 1. Must be ≥ 18 years of age at the time of signing the Informed Consent Form (ICF); must voluntarily sign an ICF; and must be willing and able to meet all study requirements. 2. Must have morphologically confirmed diagnosis of MDS/MPN, excluding JMML, in accordance with WHO (20...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT04061421primaryCompletionDate: changed
LOWMay 24, 2026NCT04061421studyFirstPostDate: changed