Jelmyto (mitomycin urothelial gel) / UroGen - LARVOL DELTA

Testing the Addition of the Immunotherapy Drug, Pembrolizumab, to Radiation Therapy Compared to the Usual Chemotherapy Treatment During Radiation Therapy for Bladder Cancer, PARRC Trial (clinicaltrials.gov) - P2 | N=160 | Recruiting | Sponsor: National Cancer Institute (NCI) | Initiation date: Dec 2025 ➔ Jun 2025

Trial initiation date • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

The uTRACT registry: A single-arm, multicenter, prospective, and retrospective registry study to evaluate the real-world use of UGN-101 in participants with upper tract urothelial carcinoma (UTUC) in the United States. (ASCO 2025) - P | "J Urol. 2024:101097ju0000000000004331."

Real-world • Real-world evidence • Retrospective data • Oncology • Solid Tumor • Urothelial Cancer

UroGen Presents uTRACT Registry at ASCO 2025 Annual Meeting Designed to Study Real-World Use of JELMYTO in Low-Grade Upper Tract Urothelial Cancer (Businesswire) - P=Obs | N=400 | uTRACT (NCT05874921) | Sponsor: UroGen Pharma Ltd. | "UroGen Pharma Ltd...today highlights the presentation of the uTRACT Registry study design at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, IL. The uTRACT Registry is a single-arm, multicenter, prospective and retrospective study evaluating the real-world use of JELMYTO (mitomycin) for pyelocalyceal solution for the treatment of adult patients with low-grade upper tract urothelial carcinoma (LG-UTUC) across the United States....As of May 2025, the uTRACT Registry (NCT05874921) has enrolled 274 participants across 22 clinical sites nationwide, with a target enrollment of approximately 400 patients. Of these, about 340 are expected to have LG-UTUC, the population for which JELMYTO is FDA-approved."

Observational data • Real-world • Trial status • Urothelial Cancer

Mitomycin/BCG Combo Could Alleviate BCG Shortage Concerns for NMIBC (OncLive) - P3 | N=501 | ANZUP 1301 (NCT02948543) | "The intravesical combination of BCG plus mitomycin showed similar efficacy and safety to that of BCG alone for patients with non–muscle-invasive bladder cancer (NMIBC) but with the added benefit of using 40% fewer BCG doses than the monotherapy arm...With a median follow-up of 48 months (IQR, 34-64), the disease-free survival (DFS) rate at 2 years was 75% (95% CI, 70%-81%) with BCG plus mitomycin (n = 249) compared with 71% (95% CI, 65%-77%) for BCG alone (n = 252; HR, 0.87; 95% CI, 0.65-1.16; P = .34). The complete response (CR) rate at 3 months was 90% with the combination and 86% with BCG alone (RR, 1.05; 95% CI, 0.98-1.12; P = .22). Adverse effects (AEs) were similar between the 2 arms, with fewer flu-like symptoms seen in the combination arm."

P3 data • Bladder Cancer • Urothelial Cancer

UroGen Pharma Reports First Quarter 2025 Financial Results and Provides a Business Update (Businesswire) - "New Drug Application for UGN-102 on track for FDA-PDUFA target action date of June 13, 2025; assuming approval, commercial launch to immediately follow with product availability in July...JELMYTO achieved net product sales of $20.3 million in Q1 2025, compared with $18.8 million in Q1 2024, driven by underlying demand growth of 12%...Results from a post-hoc sub-analysis of the ENVISION trial, showing that tumor burden and the number of tumors did not significantly affect the CR rate or durability of response for patients treated with UGN-102, were presented at the ASCO Genitourinary Cancers Symposium (ASCO-GU 2025) in February 2025."

Launch US • P3 data • Sales • Urothelial Cancer

In-home Intravesical Chemotherapy for the Treatment of Bladder Cancer, INVITE Trial (clinicaltrials.gov) - P1/2 | N=40 | Recruiting | Sponsor: Mayo Clinic | Not yet recruiting ➔ Recruiting

Enrollment open • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

OLYMPUS Long-Term Follow-Up Study Reports Nearly Four-Year Duration of Response in Subset of Patients with Low-Grade Upper Tract Urothelial Cancer Who Achieved a Complete Response to Initial JELMYTO Treatment (Businesswire) - P3 | N=71 | OLYMPUS (NCT02793128) | Sponsor: UroGen Pharma Ltd. | "Data Presented at the 2025 American Urological Association Annual Meeting....Among patients from the OLYMPUS trial who achieved a complete response after primary chemoablation with JELMYTO (n=41), the median duration of response was 47.8 months, irrespective of whether their cancer was new-onset or recurrent (median follow-up 28.1 months [95% CI 13.1, 57.5]). Of these patients, 21 had new-onset UTUC, and 20 had recurrent UTUC at baseline....Twenty patients entered long-term follow-up with a median follow-up of 53.3 months (95% CI 27.9, 65.3); median duration of response was not estimable (95% CI 43.5, not estimable) due to the low event rate....Investigators are currently enrolling participants in the JELMYTO uTRACT Registry (NCT05874921)....As of April 2025, 22 sites have been activated with 251 patients enrolled."

P3 data • Trial status • Urothelial Cancer

Emerging strategies: conservative management of upper tract urothelial carcinoma. (PubMed, Curr Opin Urol) - "The conservative management of UTUC is evolving, driven by advancements in imaging, molecular diagnostics, and minimally invasive treatments. While kidney-sparing approaches are increasingly utilized in low-risk patients, further prospective studies are needed to validate their efficacy."

Journal • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

Simulation Paired with Interdisciplinary Workflow to Prepare Oncology Nurses on Administration of Hazardous Drug via Nephrostomy Tube (ONS 2025) - "Jelmyto® is a chemotherapy treatment, which requires weekly instillation of mitomycin via nephrostomy tube for low-grade upper tract urothelial cancer...Simulation-based training with a multidisciplinary team approach is recommended to successfully implement education of hazardous drug administration via nephrostomy tube. Additional feedback will be obtained from nurses for follow-up to assess confidence long-term."

Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

UroGen Announces Data Presentations at the American Urological Association 2025 Annual Meeting Highlighting Emerging Evidence Supporting Our Portfolio for Urothelial Cancers (Businesswire) - "UroGen Pharma Ltd...announced that data on investigational drug UGN-102 (mitomycin) for intravesical solution, JELMYTO (mitomycin) for pyelocalyceal solution and UGN-301 (zalifrelimab) will be presented at the American Urological Association (AUA) 2025 Annual Meeting being held in Las Vegas, Nevada from April 26-29."

Clinical data • Bladder Cancer • Urothelial Cancer

Long-term outcomes of treatment of recurrent or new-onset low-grade upper tract urothelial carcinoma with UGN-101, a mitomycin reverse thermal gel (AUA 2025) - P, P3 | "Patients with LG-UTUC who achieved CR after receiving treatment with UGN-101 experienced clinically meaningful long-term response, with a median DoR of almost 4 years, irrespective of whether their cancer was new onset or recurrent. The ongoing uTRACT registry (NCT05874921) will provide an opportunity to collect and evaluate real-world data in a larger sample, to further inform the use of UGN-101 in UTUC patients."

Oncology • Respiratory Diseases • Septic Shock • Solid Tumor • Urothelial Cancer

Lower-Dose Chemoradiation in Treating Patients With Early-Stage Anal Cancer, the DECREASE Study (clinicaltrials.gov) - P2 | N=252 | Active, not recruiting | Sponsor: ECOG-ACRIN Cancer Research Group | Recruiting ➔ Active, not recruiting

Enrollment closed • Anal Carcinoma • Oncology • Solid Tumor • Squamous Cell Carcinoma • CD4

The Role of Kidney-Sparing Surgery for Patients with Upper Tract Urothelial Carcinoma (PubMed, Gan To Kagaku Ryoho) - "The phase Ⅲ trial assessing a mitomycin-containing reverse thermal gel (JelmytoTM)revealed the promising result despite their use not being approved in Japan. Therefore, KSS is assumed to have an increasing demand for patients with UTUC in our aged society and is expected to be widely utilized nationwide."

Journal • Cardiovascular • Nephrology • Oncology • Renal Disease • Solid Tumor • Urothelial Cancer

Testing the Addition of the Immunotherapy Drug, Pembrolizumab, to Radiation Therapy Compared to the Usual Chemotherapy Treatment During Radiation Therapy for Bladder Cancer, the PARRC Trial (clinicaltrials.gov) - P2 | N=160 | Recruiting | Sponsor: National Cancer Institute (NCI) | Not yet recruiting ➔ Recruiting | Initiation date: Mar 2025 ➔ Dec 2025

Enrollment open • Trial initiation date • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

SES AUA 2025: Long-Term Outcomes of Primary Chemoablation of Low-Grade UTUC with UGN-101, a Mitomycin Reversal Thermal Gel (UroToday) - P=NA | N=NA | "Of the 23 patients who maintained a complete response at the end of the 12-month post-complete response follow-up in OLYMPUS period, 20 entered the long term follow up study. Of the 41 patients with complete response in OLYMPUS (below: dark blue lines), 16 (39%) had documented events: 10 (24.4%) had urothelial carcinoma tumor recurrence and 6 (14.6%) patients died (not treatment-related). Of the 20 patients that entered long-term follow-up (below: light blue), 75% of patients had no evidence of recurrence at the last follow-up visit. Among the 41 patients followed up after initial complete response, the median follow-up was 28.1 months (95% CI 13.1, 57.5), and the median duration of response was 47.8 months (95% CI 13.0–NE). Of the 20 patients in the long term follow-up study, the median follow up was 53.3 months (95% CI 27.9, 65.3), and the median duration of response was not estimable (95% CI 43.5, NE) due to the low event rate."

Clinical data • Urothelial Cancer

UroGen Pharma Reports Fourth Quarter and Full Year 2024 Financial Results… (Businesswire) - "JELMYTO achieved net product revenue of $90.4 million in 2024, compared with $82.7 million in 2023, driven by underlying demand revenue growth of 12% for 2024 and 15% for Q4 2024....UroGen plans to initiate a Phase 3 trial to explore the safety and efficacy of UGN-104, its next generation product for LG-UTUC in the first half of 2025."

Sales • Trial status • Bladder Cancer • Urothelial Cancer

uTRACT Jelmyto Registry: A Registry of Patients With Upper Tract Urothelial Cancer (UTUC) Treated With Jelmyto (clinicaltrials.gov) - P=N/A | N=400 | Recruiting | Sponsor: UroGen Pharma Ltd. | Trial primary completion date: Apr 2025 ➔ Apr 2026

Real-world evidence • Trial primary completion date • Genito-urinary Cancer • Kidney Cancer • Oncology • Solid Tumor • Urothelial Cancer

SeqUential GeMcitabine and MITomycin Treatment for Favorable High-Risk Upper Urinary Tract Urothelial Carcinoma (clinicaltrials.gov) - P2 | N=28 | Not yet recruiting | Sponsor: Rutgers, The State University of New Jersey

New P2 trial • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

Data from a Long-Term Follow-up Study to the OLYMPUS Trial that Shows a Median Duration of Response of Nearly Four Years in Patients Achieving Complete Response with JELMYTO Published in The Journal of Urology (Businesswire) - P3 | N=71 | OLYMPUS (NCT02793128) | Sponsor: UroGen Pharma Ltd. | "UroGen Pharma...today highlights results from a long-term follow-up study with JELMYTO (mitomycin) for pyelocalyceal solution, which is FDA approved for the treatment of low-grade, upper tract urothelial cancer (LG-UTUC) in adult patients. Among patients from the OLYMPUS trial who achieved a complete response after primary chemoablation with JELMYTO (n=41, 20 of whom entered the long-term follow-up study), the median duration of response was 47.8 months (median follow-up 28.1 months [95% CI 13.1, 57.5]). The study results are published in the March issue of The Journal of Urology....Of the 71 patients enrolled in OLYMPUS, 41 achieved a complete response after treatment with JELMYTO and had a median duration of response of 47.8 months (95% CI 13.0, not estimable), with median follow-up of 28.1 months (95% CI 13.1, 57.5)."

P3 data • Bladder Cancer • Urothelial Cancer

New Real-World Durability of Response Data for JELMYTO Reports 68% Recurrence-Free Survival Rate (RFS) at Three Years Across a Broad Patient Population with Low-Grade Upper Tract Urothelial Cancer (LG-UTUC) (Businesswire) - "In this study on durability of response, 68% of patients with LG-UTUC who initially responded to JELMYTO had no evidence of disease recurrence at 3 years, as evaluated via endoscopy. The median follow-up was 23.5 months. RFS did not vary significantly based on use of JELMYTO for chemoablative versus adjuvant intent, tumor location (pyelocalyceal versus ureteral), tumor size before induction, single versus multiple tumors, or JELMYTO administration route (antegrade versus retrograde). The administration of maintenance treatment did appear to be associated with significantly better RFS, however, only 15 patients received maintenance therapy and, according to the authors, further study is required to determine the value of maintenance treatments."

Real-world • Retrospective data • Bladder Cancer • Urothelial Cancer

Durability of response of UGN-101: Longitudinal follow up of multicenter study. (PubMed, Urol Oncol) - "UGN-101 treatment has excellent durability in patients who initially respond to the treatment. Further study is needed to better understand the long term outcomes of this novel therapy and also the risks/benefits of maintenance therapy in this setting. Caution should be used in patients with high-grade disease who appear to be at higher risk of relapse and death despite initial response."

Clinical • Journal • Oncology • Solid Tumor • Urothelial Cancer

Testing the Addition of the Immunotherapy Drug, Pembrolizumab, to Radiation Therapy Compared to the Usual Chemotherapy Treatment During Radiation Therapy for Bladder Cancer, the PARRC Trial (clinicaltrials.gov) - P2 | N=160 | Not yet recruiting | Sponsor: National Cancer Institute (NCI)

New P2 trial • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

A Phase 3 Single-arm Study of UGN-104 for the Treatment of Low-grade Upper Tract Urothelial Cancer (clinicaltrials.gov) - P3 | N=50 | Not yet recruiting | Sponsor: UroGen Pharma Ltd.

New P3 trial • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

Letter: Long-Term Outcomes of Primary Chemoablation of Low-Grade Upper Tract Urothelial Carcinoma With UGN-101, a Mitomycin Reverse Thermal Gel. (PubMed, J Urol) - No abstract available

Journal • Oncology • Solid Tumor • Urothelial Cancer

Long-Term Follow-up Study to the OLYMPUS Trial Presented at SUO 2024 Reports Median Duration of Response of Four Years in Patients Who Achieved a Complete Response with JELMYTO (Businesswire) - P3 | N=71 | OLYMPUS (NCT02793128) | Sponsor: UroGen Pharma Ltd. | "UroGen Pharma...highlights results from a long-term follow-up study with JELMYTO (mitomycin) for pyelocalyceal solution, which is FDA approved for the treatment of adults with low-grade, upper tract urothelial cancer (LG-UTUC). Among patients from the OLYMPUS trial who achieved a complete response after primary chemoablation with JELMYTO (n=41, 20 of whom entered the long-term follow-up study), the median duration of response was 47.8 months (median follow-up 28.1 months (95% CI 13.1, 57.5). The study results were presented at the Society for Urologic Oncology (SUO) annual meeting in Dallas, Texas, and were recently published online in the Journal of Urology....The analysis has certain limitations, including its post-hoc nature and the inherent selection bias of the 20 patients enrolled in the long-term follow-up study."

P3 data • Urothelial Cancer