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UGN-104

Phase 3

Upper Urinary Tract Urothelial Carcinoma | Small molecule | Oncology |UroGen Pharma Ltd.|Last Updated: Apr 29, 2026

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
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment70
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06774131A Phase 3 Single-arm Study of UGN-104 for the Treatment of Low-grade Upper Tract Urothelial CancerPHASE3 RECRUITING 70Aug 6, 2025Mar 1, 2028Apr 29, 202652 United States, Bulgaria +6
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Study Endpoints
Primary Endpoints
Complete response rate (CRR)
3 months

CRR is defined as the proportion of patients who achieved CR at the PDE Visit.

Secondary Endpoints
Duration of response (DOR)
12 months
Durable complete response (DCR) rate
12 months
Incidence of treatment-emergent adverse events (TEAEs), serious TEAEs, TEAEs of special interest, and abnormal clinical laboratory test results (hematology and serum chemistry)
15 months
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
UGN-104EXPERIMENTALPatients will receive UGN-104 once weekly for 6 weeks (a total of 6 doses). The dose of UGN-104 to be instilled is 4 mg mitomycin per 1 mL sterile hydrogel via ureteral catheter (retrograde administration) or nephrostomy tube (antegrade administration). Patients who have a CR at the PDE Visit will enter the Follow-up Period and may receive UGN-104 as maintenance treatment once monthly for 11 months (a total of 11 doses) at the discretion of the investigator.
Interventions
NameTypeDescription
UGN-104DRUGUGN-104 consists of mitomycin and sterile hydrogel (a proprietary thermally responsive gel) that is used to reconstitute mitomycin before instillation. The reverse thermal properties of UGN-104 allow for local administration of mitomycin as a liquid under chilled conditions, with subsequent conversion to a semi-solid gel depot following instillation into the UUT.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites52

Inclusion Criteria: 1. Provide written informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and protocol. 2. Naive or recurrent patients with LG, non-invasive UTUC in the pyelocalyceal system, with the following disease characteristi...

Countries:United StatesBulgariaGeorgiaHungaryIsraelPolandRomaniaSpain
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Recent Changes (Last 90 Days)
MEDIUMMay 26, 2026NCT06774131primaryCompletionDate: changed
LOWMay 24, 2026NCT06774131studyFirstPostDate: changed