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UGN-301

Phase 1

Non-muscle Invasive Bladder Cancer | Small molecule | Oncology |UroGen Pharma Ltd.|Last Updated: Jan 14, 2026

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment51

FDA Designations

No designations recorded

Clinical trial landscape

UGN-301 · 1 trial · 6 indications

Phase 1 1
NCT05375903A Phase 1 Dose-escalation Study of UGN-301 in Patients With Recurrent Non-muscle Invasive Bladder Cancer (NMIBC)Non-muscle Invasive Bladder Cancer
COMPLETED51 Analytics
PHASE1COMPLETED
A Phase 1 Dose-escalation Study of UGN-301 in Patients With Recurrent Non-muscle Invasive Bladder Cancer (NMIBC)
Non-muscle Invasive Bladder CancerUnlock trial analytics

Study Endpoints

Primary Endpoints

Incidence of dose-limiting toxicities (DLTs) and treatment-emergent adverse events (TEAEs)
Up to 15 months

The number of patients with each type of event will be summarized.

Concentration of UGN-301 in blood and urine
6 weeks

Data will be summarized using descriptive statistics.

Complete response rate (CRR)
3 months

CRR is defined as the proportion of CIS patients who achieved CR at the Week 12 (3-month) Visit.

Recurrence-free survival (RFS) rate
3 months

RFS rate is defined as the proportion of patients with Ta/T1 disease who are recurrence-free at the Week 12 (3-month) Visit.

Secondary Endpoints

Presence of anti-drug antibodies (ADA) in serum
3 months
UGN-301 maximum serum concentration (Cmax) following single and repeat dose administration
6 weeks
UGN-301 area under the concentration-time curve (AUC) following single and repeat dose administration
6 weeks
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
UGN-301 monotherapy dose escalation (Arm A)EXPERIMENTALDose escalation of UGN-301 monotherapy in patients with recurrent NMIBC with high grade (HG) Ta and/or T1 disease and/or CIS or recurrent intermediate risk (IR) low grade (LG) Ta and/or T1 disease.
UGN-301 dose escalation + UGN-201 combination (Arm B)EXPERIMENTALDose escalation of UGN-301 in combination with a fixed dose of UGN-201 in patients with recurrent NMIBC with HG Ta and/or T1 disease and/or CIS.
UGN-301 dose escalation + gemcitabine combination (Arm C)EXPERIMENTALDose escalation of UGN-301 in combination with a fixed dose of gemcitabine in patients with recurrent NMIBC with HG Ta and/or T1 disease and/or CIS.

Interventions

NameTypeDescription
UGN-301DRUGInduction Period: Intravesical administration once weekly for 6 weeks. Optional Maintenance Period: Intravesical administration once every 3 months (at 6, 9, and 12 months after the start of treatment).
UGN-201DRUGInduction Period: Intravesical administration once weekly for 6 weeks. Optional Maintenance Period: Intravesical administration once every 3 months (at 6, 9, and 12 months after the start of treatment).
GemcitabineDRUGInduction Period: Intravesical administration once weekly for 6 weeks. Optional Maintenance Period: Intravesical administration once every 3 months (at 6, 9, and 12 months after the start of treatment).
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites13

Inclusion Criteria: 1. Able to give informed consent. 2. Arm A: Have confirmed recurrent NMIBC with HG Ta and/or T1 disease and/or CIS or recurrent IR LG Ta and/or T1 disease. Arm B: Have confirmed recurrent NMIBC with HG Ta and/or T1 disease and/or CIS. Arm C: Have confirmed recurrent NMIBC wi...

Countries:United StatesItalySpain
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Frequently asked questions about UGN-301

What is UGN-301 used for?

UGN-301 is an investigational small molecule being developed for the treatment of non-muscle invasive bladder cancer (NMIBC). It is intended for patients with recurrent NMIBC, including carcinoma in situ of the bladder and urothelial carcinoma. As of the latest data, UGN-301 is in Phase 1 clinical development and is not yet approved by regulatory authorities.

Who makes UGN-301?

UGN-301 is being developed by UroGen Pharma Ltd., a biopharmaceutical company traded on NASDAQ under the ticker symbol URGN. The company is conducting clinical trials to evaluate the safety and efficacy of UGN-301 in patients with recurrent non-muscle invasive bladder cancer.

What phase is UGN-301 in?

UGN-301 is currently in Phase 1 clinical development. A Phase 1 dose-escalation study has been completed, involving 51 patients with recurrent non-muscle invasive bladder cancer. The drug is investigational and has not yet received approval from the U.S. Food and Drug Administration or other regulatory bodies.

What clinical trials is UGN-301 in?

UGN-301 has been studied in a Phase 1 dose-escalation trial identified as NCT05375903. This completed trial enrolled 51 patients with recurrent non-muscle invasive bladder cancer, including those with carcinoma in situ of the bladder and urothelial carcinoma. The study was conducted in the United States, Italy, and Spain.

How does UGN-301 work?

UGN-301 is a small molecule therapeutic, but its specific molecular target has not been disclosed in available information. The drug is being evaluated for its ability to treat recurrent non-muscle invasive bladder cancer, though the precise mechanism of action has not been publicly detailed.