CLINICAL TRIALS PROFILE FOR JELMYTO

505(b)(2) Clinical Trials for Jelmyto

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Formulation	NCT06774131 ↗	A Phase 3 Single-arm Study of UGN-104 for the Treatment of Low-grade Upper Tract Urothelial Cancer	RECRUITING	UroGen Pharma Ltd.	PHASE3	2025-05-02	This study will evaluate the efficacy and safety of UGN-104, a new formulation of UGN-101 (approved in the United States and Israel as JELMYTO \[mitomycin\] for pyelocalyceal solution), instilled in the upper urinary tract (UUT) of patients with low-grade upper tract urothelial cancer (LG-UTUC).

All Clinical Trials for Jelmyto

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01004978 ↗	Chemoembolization With or Without Sorafenib Tosylate in Treating Patients With Liver Cancer That Cannot Be Removed by Surgery	Active, not recruiting	National Cancer Institute (NCI)	Phase 3	2009-10-28	This randomized phase III trial studies chemoembolization and sorafenib tosylate to see how well they work compared with chemoembolization alone in treating patients with liver cancer that cannot be removed by surgery. Drugs used in chemotherapy, such as doxorubicin hydrochloride, mitomycin, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Chemoembolization kills tumor cells by carrying drugs directly into blood vessels near the tumor and then blocking the blood flow to allow a higher concentration of the drug to reach the tumor for a longer period of time. Sorafenib tosylate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known whether giving chemoembolization together with sorafenib tosylate is more effective than chemoembolization alone in treating patients with liver cancer.
NCT03775265 ↗	Chemoradiotherapy With or Without Atezolizumab in Treating Patients With Localized Muscle Invasive Bladder Cancer	Recruiting	National Cancer Institute (NCI)	Phase 3	2019-04-19	This phase III trial studies how well chemotherapy and radiation therapy work with or without atezolizumab in treating patients with localized muscle invasive bladder cancer. Radiation therapy uses high energy rays to kill tumor cells and shrink tumors. Chemotherapy drugs, such as gemcitabine, cisplatin, fluorouracil and mitomycin-C, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving chemotherapy with radiation therapy may kill more tumor cells. Immunotherapy with monoclonal antibodies, such as atezolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving atezolizumab with radiation therapy and chemotherapy may work better in treating patients with localized muscle invasive bladder cancer compared to radiation therapy and chemotherapy without atezolizumab.
NCT04216290 ↗	A Study of Chemotherapy and Radiation Therapy Compared to Chemotherapy and Radiation Therapy Plus MEDI4736 (Durvalumab) Immunotherapy for Bladder Cancer Which Has Spread to the Lymph Nodes (The INSPIRE Study)	Recruiting	National Cancer Institute (NCI)	Phase 2	2020-08-25	This phase II trial studies the benefit of adding an immunotherapy drug called MEDI4736 (durvalumab) to standard chemotherapy and radiation therapy in treating bladder cancer which has spread to the lymph nodes. Drugs used in standard chemotherapy work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high-energy x-rays to kill tumor cells and shrink tumors. Immunotherapy with durvalumab may help the body's immune system attack the cancer and may interfere with the ability of tumor cells to grow and spread. Giving chemotherapy and radiation therapy with the addition of durvalumab may work better in helping tumors respond to treatment compared to chemotherapy and radiation therapy alone. Patients with limited regional lymph node involvement may benefit from attempt at bladder preservation, and use of immunotherapy and systemic chemotherapy.

Clinical Trial Conditions for Jelmyto

Condition Name

Condition Name for Jelmyto
Intervention	Trials
Bladder Urothelial Carcinoma	2
Stage III Bladder Cancer AJCC v8	2
Stage IIIA Bladder Cancer AJCC v8	2

Condition MeSH

Condition MeSH for Jelmyto
Intervention	Trials
Carcinoma	3
Urinary Bladder Neoplasms	3
Carcinoma, Transitional Cell	3

Clinical Trial Locations for Jelmyto

Trials by Country

Trials by Country for Jelmyto
Location	Trials
United States	117

Trials by US State

Trials by US State for Jelmyto
Location	Trials
Minnesota	4
Florida	4
California	4
Pennsylvania	4
Oregon	3

Clinical Trial Progress for Jelmyto

Clinical Trial Phase

Clinical Trial Phase for Jelmyto
Clinical Trial Phase	Trials
PHASE3	1
PHASE2	1
Phase 3	2

Clinical Trial Status

Clinical Trial Status for Jelmyto
Clinical Trial Phase	Trials
Not yet recruiting	3
RECRUITING	3
NOT_YET_RECRUITING	1

Clinical Trial Sponsors for Jelmyto

Sponsor Name

Sponsor Name for Jelmyto
Sponsor	Trials
National Cancer Institute (NCI)	6
Mayo Clinic	1
City of Hope Medical Center	1

Sponsor Type

Sponsor Type for Jelmyto
Sponsor	Trials
NIH	6
Other	3
INDUSTRY	1

Last updated: April 27, 2026

JELMYTO (mitomycin gel) — Clinical Trials Update, Market Analysis, and Projection (US)

What is JELMYTO and what indication is it tied to?

JELMYTO is mitomycin (mitomycin gel) for intraluminal delivery. In the US, the drug is built around treatment of upper tract urothelial cancer (UTUC) using a drug-eluting/retained gel platform delivered via ureteral instillation. The commercial and clinical position is primarily anchored to low-grade UTUC treated endoscopically, with the program’s regulatory path linked to durable response outcomes and local control.

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What is the clinical-trials status and how is it evolving?

A complete “current status” roll-up requires an authoritative trial registry snapshot (e.g., ClinicalTrials.gov last updated within a defined window) and is not available in the provided context. Under this constraint, this analysis does not publish a detailed per-study update.

Instead, the clinical-trials update below focuses on the program’s established evidence structure that supports ongoing commercialization and payer decision-making, using the highest-signal endpoints typically used for UTUC durability and recurrence-free outcomes.

Evidence pillars used for positioning

• Local control durability in low-grade UTUC after endoscopic therapy, measured by response durability and recurrence/ progression signals typical for UTUC trials.
• Renal preservation and tolerability given the need for ureteral instillation rather than systemic chemotherapy.

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What do the regulatory and label constraints imply for trial design and uptake?

Label and practice constraints drive which new trials can generate revenue-linked evidence:

• Population narrowing: UTUC is heterogeneous; evidence and uptake concentrate on low-grade disease where local-control endpoints map to regulatory decisions and guideline usage.
• Delivery logistics: ureteral instillation and imaging follow-up create operational constraints; trials that add breadth need to solve workflow (placement, dwell time, follow-up cystoscopy/ureteroscopy mapping).
• Comparator selection: adoption in routine urology depends on whether trials compare against standard endoscopic resection alone and provide clinically meaningful durability.

These factors influence trial sponsor choices toward:

• Single-arm durability confirmatory studies
• Comparative strategies vs. standard endoscopic management
• Sequencing trials that define how mitomycin gel fits after biopsy, ablation, or resection

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Market Analysis

How does JELMYTO compete in UTUC management?

In UTUC, the treatment landscape is dominated by:

• Endoscopic management (ureteroscopic resection/ablation)
• Radical surgery in higher-risk phenotypes
• Systemic therapy in advanced or metastatic disease

JELMYTO competes primarily where:

• Endoscopic management is preferred to avoid nephroureterectomy
• Durable local control is needed despite multifocal recurrence risk

Commercial thesis JELMYTO monetizes:

• Lower-margin procedures avoidance (reducing repeat resections) through durability
• Nephron sparing value to patients and health systems
• Guideline-concordant usage for the UTUC phenotype that matches label evidence

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Where does demand concentrate?

Demand concentrates in centers that can:

• Perform ureteroscopic access and instillation safely
• Support longitudinal surveillance (imaging and ureteroscopic evaluation)
• Manage ureteral anatomy and catheterization at scale

Buyer behavior

• Urology practices push adoption when clinical workflows integrate without disrupting OR throughput.
• Health systems adopt when expected episode reduction (fewer repeat interventions) offsets acquisition and procedure costs.

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What drives pricing power and reimbursement risk?

JELMYTO pricing power is tied to:

• Demonstrated durability versus repeated endoscopic procedures
• Health-economic value in avoiding or delaying nephroureterectomy
• Payer acceptance for the specific label-matched UTUC phenotype

Reimbursement risk clusters around:

• UTUC subtype coding variability and payer documentation requirements
• Site-of-care and administration coverage rules (how instillation is billed and reimbursed)
• Evidence acceptance thresholds for “durability” endpoints

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Forecast and Projection

What is the market projection framework for JELMYTO?

A credible projection requires an input set (prevalence, eligible proportion, uptake adoption curve, reimbursement coverage, and competitive displacement) that is not available in the provided context. Under the operating constraint, this analysis does not publish numerical forecasts.

Instead, the projection framework below describes how the market should be modeled for UTUC-focused local delivery products, using levers that directly affect revenue:

Revenue drivers

1. Eligible patient share within UTUC that matches the evidence-based phenotype (low-grade, endoscopically treatable)
2. Treatment setting access (high-capability urology centers and operative scheduling)
3. Adoption curve (patient selection refinement, physician experience, payer acceptance)
4. Treatment retreatment logic (how often patients require subsequent gel courses vs repeated endoscopic interventions)
5. Competition intensity (other local therapies and sequencing with endoscopic resection)

Downside drivers

• Lower than expected eligible proportion (real-world subtype mix)
• Slower payer adoption due to documentation requirements
• Adoption friction tied to instillation workflow variability
• Safety/tolerability concerns that reduce sustained usage

Upside drivers

• Faster conversion of real-world patients due to clear guideline uptake
• Evidence of durable control that reduces repeat procedures
• Site-of-care efficiencies lowering total cost of care

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Key Takeaways

• JELMYTO is positioned for local intraluminal management of UTUC where endoscopic therapy is preferred, with adoption tied to durable response evidence and operational feasibility.
• Clinical-trials evolution and uptake are shaped by population narrowing (low-grade UTUC), ureteral instillation workflow, and durability endpoints that map to reimbursement decisions.
• Numerical market projections cannot be produced from the provided inputs; revenue modeling should be built from eligible share, adoption curve, retreatment logic, and payer coverage dynamics.

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FAQs

1) Is JELMYTO a systemic chemotherapy?

No. JELMYTO is an intraluminal mitomycin gel designed for delivery to the upper urinary tract via ureteral instillation.

2) What patient type is most associated with JELMYTO uptake?

Uptake concentrates on UTUC patients whose disease phenotype matches the evidence base for local control after endoscopic management, with emphasis on low-grade disease.

3) Why does durability matter for JELMYTO’s market performance?

Because the therapy’s economic value comes from reducing repeat interventions and delaying more invasive management, durability is the core endpoint that influences both physician practice and payer coverage decisions.

4) What operational factors influence adoption?

Ureteral access capability, instillation workflow, and the ability to perform consistent post-treatment surveillance drive whether centers adopt and sustain use.

5) What is the biggest projection lever for sales?

The size of the eligible population that qualifies for label-matched use and can be treated in real-world workflow with acceptable payer coverage.

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References

[1] FDA. JELMYTO (mitomycin) prescribing information. US Food and Drug Administration.
[2] ClinicalTrials.gov. JELMYTO (mitomycin gel) trials database entries. National Library of Medicine.