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UGN-102

Phase 3

Bladder Cancer | Small molecule | Oncology |UroGen Pharma Ltd.|Last Updated: May 11, 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 Trials3
Total Enrollment311
FDA Designations
No designations recorded
Clinical Trials (3)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05243550A Phase 3 Single-Arm Study of UGN-102 for Treatment of Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder CancerPHASE3 ACTIVE NOT_RECRUITING 240Mar 1, 2022Mar 1, 2028May 11, 202665 United States, Austria +8
NCT05136898Feasibility of Home Instillation of UGN-102 for Treatment of Low-Grade (LG) Non-Muscle Invasive Bladder Cancer (NMIBC)PHASE3 COMPLETED 8Nov 30, 2021Feb 2, 2023Sep 19, 20245 United States
NCT03558503A Phase 2b Study of UGN-102 for Low Grade Intermediate Risk Non-Muscle-Invasive Bladder CancerPHASE2 COMPLETED 63Oct 15, 2018Oct 21, 2020Sep 16, 202219 United States, Israel
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Study Endpoints
Primary Endpoints
Complete Response Rate (CRR)
3 months

CRR is defined as the percentage of patients who achieved a complete response (CR) at the 3-month Visit. A patient was considered a CR if there was no detectable disease in the bladder based on visual observation (white light cystoscopy), biopsy of remaining lesions (if applicable), and voiding urine cytology.

Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation, and TEAEs of Special Interest.
Up to 3 months

The number of patients with each type of event will be summarized. TEAEs were defined as adverse events (AEs) that occurred on or after the day of the first instillation of UGN-102 or pre-treatment AEs that worsened during the study.

Number of Participants With Post-baseline Potentially Clinically Significant (PCS) Laboratory Values
Up to 3 months

PCS laboratory criteria were defined for the following parameters: Hematology * Hemoglobin \< 0.8 × lower limit of normal and \> 20% decrease from baseline or \> 1.3 × upper limit of normal (ULN) and \> 30% increase from baseline. * Leukocytes ≤ 2.8 or ≥ 16 × 10\^3/µL. * Lymphocytes \< 0.5 or \> 20 × 10\^3/µL. * Neutrophils \< 1.0 × 10\^3/µL. * Platelets \< 75 or ≥ 700 × 10\^3/µL. Chemistry * Creatinine \> 2.2 mg/dL. * Sodium ≤ 130 or \> 150 mEq/L. * Potassium \< 3.0 or \> 5.5 mEq/L. * Total bilirubin \> 1.5 × ULN. * Alanine aminotransferase (ALT) \> 3 × ULN. * Aspartate aminotransferase (AST) \> 3 × ULN. * Gamma glutamyl transferase \> 2.5 × ULN.

Composite Score for the Post-instillation Patient Questionnaire
Up to 5 weeks

Patients were asked to rate their experience receiving UGN-102 at home after each instillation was completed. A total of 5 domains (8 questions) were assessed: * Comfort (3 questions, possible range from 3 "least favorable" to 12 "most favorable"). * Safety/concerns (1 question, possible range from 1 "least favorable" to 4 "most favorable"). * Communication (2 questions, possible range from 2 "least favorable" to 8 "most favorable"). * Preference compared to office instillation (1 question, possible range from 1 "least favorable" to 4 "most favorable"). * Overall experience/satisfaction (1 question, possible range from 1 "least favorable" to 4 "most favorable"). A composite score (sum of all 8 question scores) was also calculated (possible range from 8 "least favorable" to 32 "most favorable").

Responses to the Post-instillation Home Health Professional Questionnaire
Up to 5 weeks

Home health professionals (HHPs) were asked to share their experience administering UGN-102 at the patient's home after each instillation was completed. A total of 4 domains (4 questions) were assessed: * Comfort - Were you comfortable performing the instillation at this patient's home, yes (most favorable) or no (least favorable)? * Difficulty - Was it difficult to perform the instillation at this patient's home, yes (least favorable) or no (most favorable)? * Safety/concerns - Did you have any concerns performing the instillation in the home setting, yes (least favorable) or no (most favorable)? * Adequate support - Did you have sufficient support performing the instillation in the home setting, yes (most favorable) or no (least favorable)? The number of patients whose HHP had a "most favorable" response to all 4 questions is summarized for each home instillation.

Responses to End of Study Patient and Investigator Questionnaires
3 months

Patient recommendations regarding home instillation of UGN-102 were collected at the 3-month Visit or Early Termination Visit. Two questions were asked: * Will you recommend home instillations of UGN-102 for other patients with NMIBC, yes (most favorable) or no (least favorable)? * Will you recommend home instillations of UGN-102 instead of having transurethral resection of bladder tumor (TURBT) for other patients with NMIBC, yes (most favorable) or no (least favorable)? The number of patients who had a "most favorable" response is summarized by question. In addition, investigators were asked if the experience of having their patient receive instillations at home was less difficult (most favorable), more difficult (least favorable), or not different than having them receive instillations in the office. All responses are summarized.

Complete Response (CR) Rate for UGN-102 Treatment
3 months after the first instillation of UGN-102

To evaluate the tumor ablative effect of UGN-102 in patients with LG NMIBC. CR rate was defined as the percentage of patients with no detectable disease (NDD) as determined by cystoscopy, for cause biopsy, and urine cytology.

Secondary Endpoints
Duration of Response (DOR) in Patients Who Achieved CR at the 3-month Visit
Up to 60 months
Durable Complete Response (DCR) Rate in Patients Who Achieved CR at the 3-month Visit
Up to 60 months
Disease-free Survival (DFS) in Patients Who Achieved CR at the 3-month Visit
Up to 63 months
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
UGN-102EXPERIMENTALPatients will receive 6 once-weekly intravesical instillations of UGN-102 (75 mg mitomycin).
Interventions
NameTypeDescription
UGN-102DRUGUGN-102 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-102 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 bladder.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites65

Inclusion Criteria: 1. Capable of giving written informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and the protocol. 2. Patient who has LG-NMIBC (Ta) histologically confirmed by cold cup biopsy at Screening or within 8 weeks befor...

Countries:United StatesAustriaBulgariaEstoniaGeorgiaLatviaLithuaniaPolandSerbiaSpainIsrael
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Competitive Landscape -Bladder Cancer 147 trials
CompanyTickerTrialsLead PhaseDrugs
AstraZeneca PLCAZN15PHASE3Durvalumab, Bacillus Calmette-Guerin, Tremelimumab, Enfortumab Vedotin, Cisplatin
Bristol-Myers Squibb CompanyBMY5PHASE3Nivolumab, Gemcitabine, Cisplatin, Ipilimumab, Carboplatin
Johnson & JohnsonJNJ13PHASE3TAR-200, Mitomycin C, Gemcitabine, Cetrelimab, BCG Vesiculture
Merck & Co., Inc.MRK13PHASE3Pembrolizumab, BCG, Cisplatin, Fluorouracil, Mitomycin C
Pfizer Inc.PFE10PHASE3PF-06801591, Bacillus Calmette-Guerin, Avelumab, Lorlatanib, Talazoparib
Eli Lilly and CompanyLLY4PHASE3Vepugratinib, EV, Pembrolizumab, LY4101174, LY4052031
UroGen Pharma Ltd.URGN4PHASE3UGN-103, UGN-102, UGN-104, Jelmyto for pyelocalyceal
Natera, Inc.NTRA2PHASE3Atezolizumab
CG Oncology, Inc.CGON2PHASE3Cretostimogene Grenadenorepvec
Protara Therapeutics, Inc.TARA2PHASE3TARA-002
Relmada Therapeutics IncRLMD2PHASE3NDV-01, NDV01 intravesical controlled release formulation of gemcitabine and docetaxel
ImmunityBio IncIBRX6PHASE2N-803 and BCG, N-803 + Pembrolizumab, BCG + N-803, Intravesical Recombinant Mycobacterium, N803 plus Bacillus Calmette-Guerin
BeOne Medicines Ltd. Sponsored ADRONC3PHASE3Tislelizumab, Cisplatin, Gemcitabine, Carboplatin, BGB-A445
Exelixis, Inc.EXEL4PHASE2Cabozantinib, Atezolizumab, Zanzalintinib, cabozantinib, atezolizumab
Gilead Sciences, Inc.GILD2PHASE2Sacituzumab Govitecan-hziy, Pembrolizumab, Cisplatin, Avelumab, Zimberelimab
Tyra Bioscience, IncTYRA3PHASE2Dabogratinib, Dabogratinib TBD, TYRA-300, TYRA-300 Dose TBD
BioNTech SE Sponsored ADRBNTX1PHASE2Autogene Cevumeran, Nivolumab, Saline
ICON PlcICLR1PHASE3padeliporfin VTP
AbbVie, Inc.ABBV1PHASE2Livmoniplimab, Budigalimab, Docetaxel, Paclitaxel, Gemcitabine
Jazz Pharmaceuticals Public Limited CompanyJAZZ1PHASE2Zanidatamab
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05243550primaryCompletionDate: changed
LOWMay 24, 2026NCT05243550studyFirstPostDate: changed