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Mycophenolate Mofetil

Phase 1

Acute Myeloid Leukemia | Small molecule | Oncology |Incyte Corporation|Last Updated: Jul 6, 2026

Target and mechanism

Molecular targetIMPDH1, IMPDH2
Target classInhibitor
ModalitySmall molecule

Also known as Mycophenolate mofetil (MMF)

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment40

FDA Designations

No designations recorded

Clinical trial landscape

Mycophenolate Mofetil · 1 trial · 1 indication

Phase 1 1
NCT07548983Ruxolitinib With Azacitidine Maintenance for the Treatment of Patients With Acute Myeloid Leukemia Undergoing Reduced Intensity Allogeneic Stem Cell TransplantationAcute Myeloid Leukemia
RECRUITING40 Analytics
PHASE1RECRUITING
Ruxolitinib With Azacitidine Maintenance for the Treatment of Patients With Acute Myeloid Leukemia Undergoing Reduced Intensity Allogeneic Stem Cell Transplantation
Acute Myeloid LeukemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Incidence of dose limiting toxicities (DLTs)
From cycle 1 day 1 (day +5 post-transplant) to end of cycle 2 (Cycle length = 28 days)

Will determine the safety and tolerability of ruxolitinib monotherapy (part A) and ruxolitinib plus azacitidine therapy (part B). DLTs will be used to identify a maximum tolerated dose. Dose levels will either be de-escalated, escalated, or retained-according to pre calculated Bayesian Optimal Interval decision rules.

Incidence of grade 3+ treatment-related adverse events (TRAEs) (Part B)
Up to 30 days after last dose of study drug

TRAEs of grade 3 and higher experienced during treatment with the doublet of ruxolitinib + azacitidine will be reported (with frequencies and percentages) as a co-primary endpoint. TRAEs are defined as adverse events that are definitely or possibly attributable to study treatment. Will be assessed using Common Terminology Criteria for Adverse Events version 5.0, where adverse events are assessed on a scale of 1-5, with higher scores indicating worse outcomes.

Secondary Endpoints

Graft versus host disease (GVHD)-free, relapse-free survival
From date of transplant to disease relapse, grade III-IV acute (a) GVHD, chronic (c) GVHD requiring systemic immunosuppressive therapy, or death from any cause or last known alive (censored), whichever occurs earlier, assessed up to 2 years
Overall survival
From date of transplant to last known alive (censored) or death date, assessed up to 2 years
Cumulative incidence of neutrophil engraftment
From date of transplant to 30 days post-HSCT
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Treatment (ruxolitinib, azacitidine)EXPERIMENTALSee Detailed Description.

Interventions

NameTypeDescription
Allogeneic Hematopoietic Stem Cell TransplantationPROCEDUREUndergo alloHSCT
AzacitidineDRUGGiven IV
Biospecimen CollectionPROCEDUREUndergo blood sample collection
Bone Marrow AspirationPROCEDUREUndergo bone marrow aspiration
Bone Marrow BiopsyPROCEDUREUndergo bone marrow biopsy
CyclophosphamideDRUGGiven IV
Echocardiography TestPROCEDUREUndergo ECHO
Electronic Health Record ReviewOTHERAncillary studies
Multigated Acquisition ScanPROCEDUREUndergo MUGA
Mycophenolate MofetilDRUGGiven PO or IV
Reduced-Intensity Transplant Conditioning ProcedureOTHERUndergo SOC RIC or NMA conditioning
RuxolitinibDRUGGiven PO
Survey AdministrationOTHERAncillary studies
TacrolimusDRUGGiven PO or IV
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * PART A: Willingness to provide written informed consent before any study-specific procedures or interventions are performed. For participants unable to independently provide consent, a legally authorized representative (LAR) must provide consent * PART A: Age ≥ 18 years, at th...

Countries:United States
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Recent Changes (Last 90 Days)

LOWJul 6, 2026NCT07548983Status: NOT_YET_RECRUITING → RECRUITING
LOWJul 6, 2026NCT07548983Status: NOT_YET_RECRUITING → RECRUITING

Frequently asked questions about Mycophenolate Mofetil

What is Mycophenolate Mofetil used for in Acute Myeloid Leukemia?

Mycophenolate Mofetil is being studied for use in Acute Myeloid Leukemia (AML) as part of a Phase 1 clinical trial. The trial combines ruxolitinib with azacitidine maintenance therapy for patients with AML undergoing reduced intensity allogeneic stem cell transplantation. It is an investigational small molecule in early-stage clinical development.

What does Mycophenolate Mofetil target?

Mycophenolate Mofetil is an immunosuppressant that inhibits inosine monophosphate dehydrogenase, an enzyme needed for guanine nucleotide synthesis in lymphocytes. This selectively blocks T and B cell proliferation. In the context of the AML trial, it is used to prevent graft-versus-host disease after stem cell transplantation.

Who makes Mycophenolate Mofetil?

Mycophenolate Mofetil is being developed by Incyte Corporation, traded on the NASDAQ under the ticker INCY. The company is conducting a Phase 1 clinical trial of the drug in Acute Myeloid Leukemia. Incyte is a biopharmaceutical company focused on oncology and inflammation.

What phase is Mycophenolate Mofetil in?

Mycophenolate Mofetil is in Phase 1 clinical development for Acute Myeloid Leukemia. It is an investigational drug and has not been approved by the FDA for this indication. The ongoing trial is recruiting patients and is not yet completed.

What clinical trials is Mycophenolate Mofetil in?

Mycophenolate Mofetil is being evaluated in one active Phase 1 trial, NCT07548983, titled 'Ruxolitinib With Azacitidine Maintenance for the Treatment of Patients With Acute Myeloid Leukemia Undergoing Reduced Intensity Allogeneic Stem Cell Transplantation.' The trial is recruiting 40 patients in the United States.

Is Mycophenolate Mofetil the same as Mycophenolate mofetil (MMF)?

Yes, Mycophenolate Mofetil is the same as Mycophenolate mofetil (MMF). MMF is a common abbreviation for the drug. In the context of this Phase 1 trial, it is being studied in combination with ruxolitinib and azacitidine for Acute Myeloid Leukemia patients.