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Mycophenolate Mofetil

Phase 2

Primary Myelofibrosis | Small molecule | Other |Incyte Corporation|Last Updated: Dec 4, 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 Enrollment61
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02251821JAK Inhibitor Before Donor Stem Cell Transplant in Treating Patients With Primary or Secondary MyelofibrosisPHASE2 ACTIVE NOT_RECRUITING 61Oct 20, 2014Dec 28, 2025Dec 4, 20251 United States
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Study Endpoints
Primary Endpoints
2-year Overall Survival (OS) in Patients With Myelofibrosis (MF) Who Receive Treatment With a JAK Inhibitor Followed by an Allogeneic Transplant
2 years

OS was defined as the time from date of transplantation to date of death due to any cause. In the absence of confirmation of death, survival time was censored to last date the participant was known to be alive. The 2-year survival probability was estimated using the Kaplan-Meier method and a 2-sided 95% confidence interval (CI).

Secondary Endpoints
Percentage of Participants With Acute Graft Versus Host Disease (GVHD) Grade II-IV
Up to 100 days post-transplant
Percentage of Participants With Chronic Graft Versus Host Disease (GVHD)
2 years
Percentage of Patients Who Had Relapsed Disease at 1 Year
1 year
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Treatment (ruxolitinib, transplant)EXPERIMENTALPatients receive a ruxolitinib and undergo myeloablative or reduced-intensity conditioning followed by transplant and GVHD prophylaxis; see detailed description.
Interventions
NameTypeDescription
Allogeneic Hematopoietic Stem Cell TransplantationPROCEDUREUndergo allogeneic hematopoietic stem cell transplant
BusulfanDRUGGiven IV
CyclophosphamideDRUGGiven IV
Fludarabine PhosphateDRUGGiven IV
Laboratory Biomarker AnalysisOTHERCorrelative studies
MelphalanDRUGGiven IV
MethotrexateDRUGGiven IV
Mycophenolate MofetilDRUGGiven IV or PO
RuxolitinibDRUGGiven PO
TacrolimusDRUGGiven IV or PO
Total-Body IrradiationRADIATIONUndergo TBI
Umbilical Cord Blood TransplantationPROCEDUREUndergo umbilical cord blood transplant
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: PART 1: * PART 1: Disease criteria * Diagnosis of primary MF (PMF) as defined by the 2008 World Health Organization classification system or diagnosis of secondary MF as defined by the International Working Group (IWG) for Myeloproliferative Neoplasms Research and Treatment ...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT02251821studyFirstPostDate: changed