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Itacitinib

Phase 3

Graft-versus-host Disease (GVHD) | Small molecule | Immunology |Incyte Corporation|Last Updated: Sep 2, 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
RandomizedDouble-BlindCONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment470
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03139604GRAVITAS-301: A Study of Itacitinib or Placebo in Combination With Corticosteroids for Treatment of Acute Graft-Versus-Host DiseasePHASE3 COMPLETED 439Jul 19, 2017Jul 13, 2020Sep 2, 2025129 United States, Australia +17
NCT02614612Study of Itacitinib in Combination With Corticosteroids for the Treatment of Acute GVHDPHASE1 COMPLETED 31Dec 1, 2015Aug 1, 2018Mar 8, 201923 United States
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Study Endpoints
Primary Endpoints
Overall Response Rate Based on Center for International Blood and Marrow Transplant Research (CIBMTR) Response Index
Day 28

Defined as the percentage of participants demonstrating a complete response (CR), very good partial response (VGPR), or partial response (PR).

Assess safety and tolerability of study treatment as measured by the frequency and severity of adverse events and serious adverse events
First dose of study drug to 30 days after the last dose of study drug
Secondary Endpoints
Nonrelapse Mortality
Month 6,9,12 and 24
Duration of Response
Baseline through 30-35 days after end of treatment, total particpation expected to average 24 months
Cmax of Itacitinib When Administered in Combination With Corticosteroids
Protocol-defined timepoints up to Day 28
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ItacitinibEXPERIMENTALItacitinib plus corticosteroids
PlaceboPLACEBO_COMPARATORMatching placebo plus corticosteroids
Itacitinib (200 mg)EXPERIMENTALItacitinib (200 mg) + prednisone or methylprednisolone (corticosteroids)
Itacitinib (300 mg)EXPERIMENTALItacitinib (300 mg) + prednisone or methylprednisolone (corticosteroids)
Interventions
NameTypeDescription
ItacitinibDRUGItacitinib at the protocol-defined dose administered orally once daily (QD) plus corticosteroids.
PlaceboDRUGMatching placebo tablets administered orally once daily (QD) plus corticosteroids.
PrednisoneDRUGOral prednisone may be used to begin standard corticosteroid background treatment at the investigator's discretion, at a dose equivalent to methylprednisolone 2 mg/kg per day.
MethylprednisoloneDRUGMethylprednisolone 2 mg/kg IV daily (or prednisone equivalent) or at a dose appropriate for the severity of disease as background treatment.
Itacitinib (200 mg)DRUG -
Itacitinib (300 mg)DRUG -
prednisone or methylprednisolone (corticosteroids)DRUGAll subjects will receive prednisone 2.5 mg/kg per day PO (or methylprednisolone 2 mg/kg IV daily) on Days 1 through 5. Subjects will be tapered as tolerated beginning on Day 6 to no less than 0.25 mg/kg per day PO (or methylprednisolone 0.2 mg/kg per day) by Day 28. After Day 28, corticosteroids should be tapered according to institutional guidelines to attain ≤ prednisone 0.2 mg/kg per day (or ≤ methylprednisolone 0.16 mg/kg per day) by Day 56.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites129

Inclusion Criteria: * Has undergone 1 allo-HSCT from any donor (related or unrelated with any degree of HLA matching) and any donor source (bone marrow, peripheral blood stem cells, or cord blood) for a hematologic malignancy or disorder. Recipients of myeloablative and reduced-intensity conditioni...

Countries:United StatesAustraliaAustriaBelgiumCzechiaFinlandFranceGermanyGreeceIsraelItalyNew ZealandPolandPortugalSouth KoreaSpainSwitzerlandTaiwanUnited Kingdom
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