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ASTX727

Phase 1

MDS/MPN | Small molecule | Hematology |Incyte Corporation|Last Updated: Apr 21, 2026

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment94

FDA Designations

No designations recorded

Clinical trial landscape

ASTX727 · 1 trial · 1 indication

Phase 1 1
NCT04061421Active Myeloid Target Compound Combinations in MDS/MPN Overlap Syndromes Overlap Syndromes (ABNL-MARRO)MDS/MPN
RECRUITING94 Analytics
PHASE1RECRUITING
Active Myeloid Target Compound Combinations in MDS/MPN Overlap Syndromes Overlap Syndromes (ABNL-MARRO)
MDS/MPNUnlock trial analytics

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 to 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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Frequently asked questions about ASTX727

What is ASTX727 used for?

ASTX727 is an investigational small molecule being studied for the treatment of myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN), a group of blood cancers. It is currently in Phase 1 clinical development and has not been approved by the FDA.

Who makes ASTX727?

ASTX727 is being developed by Incyte Corporation (NASDAQ: INCY). The company is conducting a Phase 1 clinical trial to evaluate the drug in patients with MDS/MPN overlap syndromes.

What phase is ASTX727 in?

ASTX727 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA. The ongoing Phase 1 trial is actively recruiting participants in the United States.

What clinical trials is ASTX727 in?

ASTX727 is being studied in a Phase 1 clinical trial with the identifier NCT04061421, titled "Active Myeloid Target Compound Combinations in MDS/MPN Overlap Syndromes (ABNL-MARRO)." The trial is recruiting 94 participants aged 18 years and older in the United States.

Is ASTX727 the same as decitabine and cedazuridine?

ASTX727 is a combination of decitabine and cedazuridine, which are the active ingredients in the drug. This combination is being studied for the treatment of MDS/MPN. The drug is also known by the code name ASTX727.