Recent Updates
Recently added Catalysts

Enfortumab vedotin

Phase 3

Urothelial Cancer | Small molecule | Oncology |Merck & Company, Inc.|Last Updated: Jul 29, 2026

Target and mechanism

Target class-Mab (Antibody)
ModalitySmall molecule

Also known as Enfortumab vedotin (EV)

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment886

FDA Designations

No designations recorded

Clinical trial landscape

Enfortumab vedotin · 6 trials · 16 indications

Phase 3 1Phase 2 5
NCT04223856Enfortumab Vedotin and Pembrolizumab vs. Chemotherapy Alone in Untreated Locally Advanced or Metastatic Urothelial CancerUrothelial Cancer
ACTIVE NOT_RECRUITING886 Analytics
PHASE3ACTIVE NOT_RECRUITING
Enfortumab Vedotin and Pembrolizumab vs. Chemotherapy Alone in Untreated Locally Advanced or Metastatic Urothelial Cancer
Urothelial CancerUnlock trial analytics

Study Endpoints

Primary Endpoints

Progression-Free Survival (PFS) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version (v) 1.1 by Blinded Independent Central Review (BICR)
From the date of randomization to first documentation of PD or death due to any cause, whichever occurred first (maximum exposure to treatment was up to 39.2 months)

PFS was defined as the time from date of randomization to first documentation of disease progression (PD), or to death due to any cause, whichever occurred first. PD: at least a 20 percent (%) increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 millimeter (mm). PD could also be unequivocal progression of non-target lesions or the presence of unequivocal new lesions. Kaplan-Meier method was used for analysis.

Overall Survival (OS)
From randomization to date of death due to any cause or censoring date, whichever occurred first (maximum exposure to treatment was up to 39.2 months)

OS was defined as the time from the date of randomization to the date of death from any cause. In the absence of death, OS was censored at the date the participant was last known to be alive. Kaplan-Meier method was used for analysis.

Complete response rate with enfortumab vedotin plus pembrolizumab for MIBC
9 weeks

Clinical complete response rate will be defined as cT0 or cTa (low grade) disease at the time of restaging after 3 cycles of induction enfortumab vedotin plus pembrolizumab

Estimated 2-year bladder-intact event-free survival (BI-EFS) for the intention-to-treat population, measured from day 1 of treatment until the moment of analysis
From the first dose of study treatment up to 2 years of follow-up

Bladder-intact event-free survival (BI-EFS). The primary analysis will be performed after the last participant has completed at least 15 months of follow-up from the first dose of study treatment or when 21 BI-EFS events have been observed, whichever occurs first.

Pathological objective response (ORR) rate
from radical nephroureterectomy through study completion, an average of 1 year

Will be assessed by the combination of partial response (PR) and complete response (CR). PR is defined as pathologic stage \<T2 disease. CR is defined as pT0 disease and assessed by Response Evaluation Criteria in Solid Tumors (RECIST) version (v)1.1.

Radiographic recurrence-free survival
From radical nephroureterectomy to 1 year
Overall response rate
Up to 2 years

Will be measured by Response Evaluation Criteria in Solid Tumors version 1.1, and estimated by the Clopper-Pearson method with 95% confidence intervals.

Cohorts 1-8: Confirmed Overall Response Rate (ORR) (Complete Response [CR] and Partial Response [PR]) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version (V)1.1 Per Investigator Assessment
Up to 20.04 months

Confirmed ORR was defined as the percentage of participants whose objective response was confirmed as CR or PR according to RECIST V1.1 per investigator assessment, using exact method based on binomial distribution (Clopper-Pearson).

Cohort 9: Confirmed ORR (CR and PR) Per RECIST V1.1 Per Investigator Assessment
Up to 12.85 months

Confirmed ORR was defined as the percentage of participants whose objective response was confirmed as CR or PR according to RECIST V1.1 per investigator assessment, using exact method based on binomial distribution (Clopper-Pearson).

Secondary Endpoints

Percentage of Participants With Objective Response Rate (ORR) Per RECIST v1.1 by BICR
From first dose till CR/PR or PD or death, whichever occurred first (maximum up to approximately 7.4 years)
Time to Pain Progression (TTPP)
From the date of randomization to date of pain progression (maximum up to approximately 7.4 years)
Change From Baseline in Worst Pain Using BPI-SF at Week 26
Baseline, Week 26
Unlock Study Endpoints

Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Arm AEXPERIMENTALEnfortumab vedotin + pembrolizumab
Arm BACTIVE_COMPARATORGemcitabine + cisplatin or carboplatin
Enfortumab vedotin plus pembrolizumabEXPERIMENTALPatients with MIBC will receive 3 cycles (C1-C3) of induction enfortumab vedotin (1.25 mg/kg for a maximum dose of 125 mg) plus pembrolizumab (200 mg) followed by restaging including MRI of the bladder, urine cytology, and cystoscopy with TURBT of any visible tumor and/or resection site plus random biopsies using a recommended template. Patients achieving a stringently defined cCR (defined below) will receive 14 cycles of "maintenance" treatment. Enfortumab vedotin (1.25 mg/kg for a maximum dose of 125 mg) will be administered during the first 6 cycles (C4-C9) of "maintenance" treatment and pembrolizumab (200 mg) will be given all 14 cycles (C4-C14). Patients with any residual disease at clinical restaging (i.e., \>cTa disease) will undergo cystectomy.
After induction therapy and cCR: maintenance pembrolizumab onlyEXPERIMENTALPatients receive four 3-weekly cycles of induction Enfortumab Vedotin on days 1, 8 and Pembrolizumab day 1. If, after tumor assessment, the response is deemed a cCR, patients can be randomized to this group. This arm will receive Pembrolizumab maintenance: 400 mg q6weeks.
After induction therapy and cCR: consolidative radiotherapy, followed by maintenance pembrolizumab.EXPERIMENTALPatients receive four 3-weekly cycles of induction Enfortumab Vedotin on days 1, 8 and Pembrolizumab day 1. If, after tumor assessment, the response is deemed a cCR, patients can be randomized to this group. This arm will receive consolidative radiotherapy followed by Pembrolizumab maintenance: 400 mg q6weeks.
By residual disease may still receive bladder-sparing treatmentOTHERPatients receive four 3-weekly cycles of induction Enfortumab Vedotin on days 1, 8 and Pembrolizumab day 1. If, after tumor assessment, the response is deemed a non-cCR, patients can be placed in this group. In this arm, patients may still receive bladder-sparing treatment using chemoradiotherapy (by local protocol; Mitomycin C/fluoropyrimidines in the Netherlands), followed by Pembrolizumab maintenance: 400 mg q6weeks.
By residual disease cannot receive bladder-sparing treatmentOTHERPatients receive four 3-weekly cycles of induction Enfortumab Vedotin on days 1, 8 and Pembrolizumab day 1. If, after tumor assessment, the response is deemed a non-cCR, patients can be placed in this group. In this arm, patients will undergo a radical cystectomy, followed by Pembrolizumab maintenance: 400 mg q6weeks.
Treatment (pembrolizumab, enfortumab vedotin)EXPERIMENTALPatients receive pembrolizumab IV and enfortumab vedotin IV on study. Patients undergo radical nephroureterectomy and receive pembrolizumab IV on study Patients also undergo MRU imaging and undergo blood, urine and tissue sample collection throughout the study.
Treatment (enfortumab vedotin, pembrolizumab)EXPERIMENTALPatients receive enfortumab vedotin IV and pembrolizumab IV on study. Patients also undergo CT scan or MRI, and collection of blood throughout the trial.
Cohort 1: HR+/HER2- breast cancerEXPERIMENTALParticipants will receive enfortumab vedotin as an intravenous (IV) infusion on days 1, 8 and 15 of each 28-day cycle. HR+/HER2- = Hormone receptor-positive/ human epidermal growth factor receptor 2-negative
Cohort 2: Triple negative breast cancer (TNBC)EXPERIMENTALParticipants will receive enfortumab vedotin as an intravenous (IV) infusion on days 1, 8 and 15 of each 28-day cycle.
Cohort 3: Squamous non-small cell lung cancerEXPERIMENTALParticipants will receive enfortumab vedotin as an intravenous (IV) infusion on days 1, 8 and 15 of each 28-day cycle.
Cohort 4: Non-squamous non-small cell lung cancerEXPERIMENTALParticipants will receive enfortumab vedotin as an intravenous (IV) infusion on days 1, 8 and 15 of each 28-day cycle.
Cohort 5: Head and neck cancerEXPERIMENTALParticipants will receive enfortumab vedotin as an intravenous (IV) infusion on days 1, 8 and 15 of each 28-day cycle.
Cohort 6: Gastric or GEJ or esophageal cancerEXPERIMENTALParticipants enrolled into Cohort 6 will be reallocated based on disease type and histology into Cohorts 7 or 8. GEJ= gastroesophageal junction
Cohort 7: Gastric and esophageal adenocarcinoma (EAC) including GEJ adenocarcinomaEXPERIMENTALParticipants will receive enfortumab vedotin as an intravenous (IV) infusion on days 1, 8 and 15 of each 28-day cycle.
Cohort 8: Esophageal squamous cell carcinoma (ESCC)EXPERIMENTALParticipants will receive enfortumab vedotin as an intravenous (IV) infusion on days 1, 8 and 15 of each 28-day cycle.
Cohort 9: Head and neck squamous cell carcinoma (HNSCC)EXPERIMENTALParticipants will receive enfortumab vedotin as an IV infusion on days 1 and 8 of each 21-day cycle. Pembrolizumab will be administered as an IV infusion on day 1 of each 21-day cycle.

Interventions

NameTypeDescription
Enfortumab vedotinDRUGEnfortumab vedotin administered as an IV infusion on Days 1 and 8 of every 3-week cycle
PembrolizumabDRUGIV infusion on Day 1 of every 3-week cycle
CisplatinDRUGadministered as IV infusion on Day 1 of each 3-week cycle
CarboplatinDRUGDosed according to local guidelines and will be administered as IV infusion on Day 1 of each 3-week cycle
GemcitabineDRUGIV infusion on Days 1 and 8 of every 3 week cycle
RadiationRADIATIONThe preference will be a four-week schedule, in which 55 Gy radiotherapy will be administered using intensity modulated radiation therapy (IMRT)
ChemoradiationOTHERby local protocol; by local protocol; Mitomycin C/fluoropyrimidines in the Netherlands) * No disease outside the bladder (e.g. involvement of ureter, prostatic urethra or (suspected) lymph node metastases) * No bilateral hydronephrosis * No multifocal CIS * Adequate bladder function: Post-micturition residual volume of \< 200 cc, International Prostate Symptom Score (IPSS) \< 15 points; revised urinary incontinence scale \< 8 points; no daily or continuous catheter use.
CystectomyPROCEDURESurgical removal of the bladder
BiopsyPROCEDUREUndergo tissue biopsy
Biospecimen CollectionPROCEDUREUndergo blood and urine collection
MR UrographyPROCEDUREUndergo MRU
NephroureterectomyPROCEDUREUndergo nephroureterectomy
Computed TomographyPROCEDUREUndergo CT
Magnetic Resonance ImagingPROCEDUREUndergo MRI
Questionnaire AdministrationOTHERAncillary studies
Unlock Study Design Details

Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites260

Inclusion Criteria: * Histologically documented, unresectable locally advanced or metastatic urothelial carcinoma * Measurable disease by investigator assessment according to RECIST v1.1 * Participants with prior definitive radiation therapy must have measurable disease per RECIST v1.1 that is o...

Countries:United StatesArgentinaAustraliaBelgiumCanadaChinaCzechiaDenmarkFranceGermanyHungaryIsraelItalyJapanNetherlandsPolandRussiaSingaporeSouth KoreaSpainSwitzerlandTaiwanThailandTurkey (Türkiye)United Kingdom
Unlock Eligibility Criteria

Frequently asked questions about Enfortumab vedotin

What is Enfortumab Vedotin used for?

Enfortumab Vedotin is an investigational antibody-drug conjugate being studied for multiple urothelial cancers, including muscle invasive bladder urothelial carcinoma, urothelial bladder cancer, renal pelvis and ureter urothelial carcinoma, bladder squamous cell carcinoma, and locally advanced or metastatic malignant solid tumors. It is also being evaluated in combination with pembrolizumab for various bladder cancer settings.

What does Enfortumab Vedotin target?

Enfortumab Vedotin is a monoclonal antibody (mab) that targets Nectin-4, a cell adhesion protein highly expressed on urothelial cancer cells. It delivers a microtubule-disrupting agent directly to tumor cells, leading to cell death. This targeted mechanism is being investigated across multiple clinical trials for bladder and urothelial cancers.

Who is developing Enfortumab Vedotin?

Enfortumab Vedotin is being developed by Merck & Company, Inc., traded on the New York Stock Exchange under the ticker MRK. The company is conducting clinical trials to evaluate the drug's safety and efficacy in various urothelial cancer indications, including combination studies with pembrolizumab.

What phase is Enfortumab Vedotin in?

Enfortumab Vedotin is currently in Phase 2 clinical development for several indications, including muscle invasive bladder urothelial carcinoma, urothelial bladder cancer, and renal pelvis and ureter urothelial carcinoma. It is also being studied in a Phase 3 trial for untreated locally advanced or metastatic urothelial cancer, though the overall development stage is Phase 2.

What clinical trials is Enfortumab Vedotin in?

Enfortumab Vedotin is being evaluated in multiple trials, including NCT04223856 (Phase 3, active not recruiting, 886 participants) for urothelial cancer, NCT05756569 (Phase 2, recruiting) for bladder cancer of variant histology, NCT05775471 (Phase 2, recruiting) for high-risk upper tract urothelial cancer, and NCT07663747 (Phase 2, not yet recruiting) for muscle invasive bladder cancer.

Is Enfortumab Vedotin the same as Enfortumab vedotin (EV)?

Yes, Enfortumab Vedotin is also known as Enfortumab vedotin (EV). This alternative name is commonly used in clinical trial titles and medical literature. Researchers and clinicians may refer to the drug as EV when discussing combination regimens, such as EV plus pembrolizumab, in the context of urothelial cancer treatment.