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Pembrolizumab

Phase 3

UC (Urothelial Cancer) | Small molecule | Oncology |Incyte Corporation|Last Updated: Aug 22, 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
RandomizedACTIVE_CONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment177
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03374488Pembrolizumab + Epacadostat vs Pembrolizumab + Placebo in Recurrent or Progressive Metastatic Urothelial CarcinomaPHASE3 COMPLETED 84Dec 22, 2017Jul 23, 2020Aug 22, 2025135 United States, Australia +16
NCT03361865Pembrolizumab in Combination With Epacadostat or Placebo in Cisplatin-ineligible Urothelial Carcinoma (KEYNOTE-672/ECHO-307)PHASE3 COMPLETED 93Dec 4, 2017Aug 4, 2020Aug 22, 2025143 United States, Australia +16
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Study Endpoints
Primary Endpoints
Objective Response Rate (ORR) With Pembrolizumab + Epacadostat Versus Pembrolizumab + Placebo
up to 9 weeks +14 days

ORR was defined as the percentage of participants who had a complete response (CR), disappearance of all target lesions or partial response (PR), \>=30% decrease in the sum of the longest diameter of target lesions per RECIST v1.1 by investigator determination.

Secondary Endpoints
Safety and Tolerability of Pembrolizumab + Epacadostat Versus Pembrolizumab + Placebo as Measured by Number of Participants Experiencing Adverse Events (AEs)
Up to 8 months
Safety and Tolerability of Pembrolizumab + Epacadostat Versus Pembrolizumab + Placebo as Measured by Number of Participants Discontinuing Study Treatment Due to AE
Up to 8 months
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Pembrolizumab 200 mg + Epacadostat 100 mg BIDEXPERIMENTALPembrolizumab + epacadostat
Pembrolizumab 200 mg + placebo BIDACTIVE_COMPARATORPembrolizumab + placebo
Interventions
NameTypeDescription
PembrolizumabDRUGPembrolizumab administered intravenously Day 1 of each cycle every 3 weeks.
EpacadostatDRUGEpacadostat administered orally twice daily.
PlaceboDRUGMatching placebo administered orally twice daily.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites135

Inclusion Criteria: * Histologically-confirmed diagnosis of urothelial carcinoma of the renal pelvis, ureter, bladder, or urethra, that is transitional cell, or mixed transitional/non-transitional (predominantly transitional) cell type. * Progression or recurrence of urothelial carcinoma following ...

Countries:United StatesAustraliaCanadaDenmarkFranceGermanyHungaryIrelandIsraelItalyJapanNetherlandsRussiaSouth KoreaSpainTaiwanTurkey (Türkiye)United KingdomBelgiumPolandUkraine
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