Zusduri (mitomycin intravesicular) / UroGen - LARVOL DELTA

UroGen Pharma Expands Commercial Portfolio with Launch of ZUSDURI... (GlobeNewswire) - "ZUSDURI (mitomycin) for intravesical solution now available as the first and only FDA-approved medication for adults with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC)."

Launch US • Bladder Cancer

UroGen Announces 24-Month Duration of Response of 72.2% from the Pivotal Phase 3 ENVISION Trial of ZUSDURI, the First and Only FDA-Approved Medicine for Recurrent Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer (GlobeNewswire) - P3 | N=240 | ENVISION (NCT05243550) | Sponsor: UroGen Pharma Ltd. | "UroGen Pharma Ltd...announced the 24-month DOR of 72.2% (95% CI 64.1%, 78.8%) by Kaplan-Meier estimate in patients who achieved CR at three months from the Phase 3 ENVISION trial of ZUSDURI (mitomycin) for intravesical solution, a treatment for adults with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The median follow-up time after a three-month CR in this analysis was 23.7 months. The median DOR has not been reached....ARs were mainly mild to moderate. Serious ARs occurred in 12% of patients, including urinary retention (0.8%) and urethral stenosis (0.4%)."

P3 data • Bladder Cancer

Treatment of Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer With UGN-102: Outcomes From the 5-Year Long-Term Extension Study of the Single-Arm, Phase 2b Optima II Study. (PubMed, Clin Genitourin Cancer) - P2b | "For the 17 patients in the LTFU study the median DoR was 42.1 months (95% CI, 24.18-not estimable [NE]), with a median follow-up of 50.40 months (95% CI, 26.97-NE), These results demonstrate that treatment with UGN-102 results in clinically meaningful, and highly durable response in patients with LG-IR-NMIBC. UGN-102 may offer a promising non-surgical alternative to transurethral resection of bladder cancer (TURBT) for LG-IR-NMIBC patients."

Journal • P2b data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Novel intravesical delivery systems for nonmuscle invasive bladder cancer. (PubMed, Curr Opin Urol) - "Novel intravesical delivery systems show to enhance drug retention, improve tissue penetration, and potentially reduce adverse events. While traditional chemotherapy or BCG remain the gold-standard adjuvant treatments for NMIBC, these novel approaches offer promising alternatives for selected patients pending on ongoing clinical validation."

Journal • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • FGFR

UroGen Announces Five-Year Long-Term Extension Study of the OPTIMA II Trial Demonstrates Long-Term Durability of Response to ZUSDURI in Patients with Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer (GlobeNewswire) - P2b | N=63 | OPTIMA II (NCT03558503) | Sponsor: UroGen Pharma Ltd. | "UroGen Pharma Ltd...announced the publication of results of a five-year long-term extension study of the Phase 2b OPTIMA II trial evaluating ZUSDURI (mitomycin) for intravesical solution in patients with low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) demonstrating durable, long-term complete responses (CRs) in patients who initially achieved a CR following treatment with ZUSDURI....For the 41 patients achieving CR at three months, the median Kaplan-Meier estimate of duration of response (DOR) was 24.2 months (95% CI 9.7, 42.1) with a median follow-up of 35.8 months. For the 17 patients in the long-term follow-up study, the median DOR was 42.1 months by Kaplan-Meier estimate (95% CI: 24.2, NE), with a median follow-up of 50.4 months."

P2b data • Bladder Cancer • Urothelial Cancer

Impact of tumor burden or focality in recurrent low-grade intermediate-risk non-muscle invasive bladder cancer on response to treatment with UGN-102: A substudy of the phase 3 ENVISION trial. (ASCO 2025) - P3 | "The CRR and DoR were favorable in all subgroups and no significant differences were observed. Study limitations were the small sample size of comparator groups, single arm design, and post-hoc nature of the analysis. UGN-102 may represent a valuable treatment option for many patients with LG-IR-NMIBC."

P3 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Duration of response (DoR) following treatment with UGN-102 in patients with recurrent, low-grade, intermediate-risk, non-muscle invasive, bladder cancer: 18-month DoR data from the phase 3 ENVISION trial. (ASCO 2025) - P3 | "In the ENVISION study treatment with UGN-102 in patients with recurrent LG-IR-NMIBC resulted in a high and clinically meaningful CR rate. Patients who achieved an initial CR at 3 months had a high probability of remaining disease-free 18 months later. This data confirms that UGN-102 represents a valuable treatment option for patients with LG-IR-NMIBC."

Clinical • P3 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Treatment of low-grade intermediate-risk non-muscle invasive bladder cancer with UGN-102: Results of the phase 3 ATLAS and ENVISION studies. (ASCO 2025) - P3 | "In both studies, CR rate in patients initially treated with UGN-102 was robust, with the majority of patients remaining event free at 12 months follow-up. These results demonstrate that treatment with UGN-102 results in a high and clinically meaningful durable CR rate in patients with newly diagnosed or recurrent LG-IR-NMIBC. UGN-102 may represent a valuable nonsurgical treatment option for these patients."

P3 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

FDA approves mitomycin intravesical solution for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (FDA) - "On June 12, 2025, the Food and Drug Administration approved mitomycin intravesical solution (Zusduri, UroGen Pharma) for adult patients with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC)....Efficacy was evaluated in ENVISION (NCT05243550), a single-arm, multicenter trial in 240 adults with low-grade NMIBC that recurred after prior transurethral resection of bladder tumor (TURBT) and met 1-2 of the following criteria: multiple tumors, a solitary tumor >3 cm, and/or recurrence within 1 year....The major efficacy outcomes were complete response (CR) at 3 months and duration of response (DOR). CR was defined as no detectable disease in the bladder by cystoscopy and urine cytology....The recommended mitomycin dose is 75 mg (56 mL) instilled once weekly for six weeks into the bladder via a urinary catheter."

FDA approval • Bladder Cancer

UroGen Announces Data at ASCO 2025 Annual Meeting on UGN-102, an Investigational Treatment for Recurrent Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer (LG-IR-NMIBC) (Businesswire) - "UGN-102 data featured at ASCO: (i) Duration of Response (DOR) Following Treatment with UGN-102 in Patients with Recurrent, Low-Grade, Intermediate-Risk, Non-Muscle Invasive, Bladder Cancer: 18-Month DOR Data from the Phase 3 ENVISION Trial: Abstract #4598, Poster #398; (ii) Impact of Tumor Burden or Focality in Recurrent Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer on Response to Treatment with UGN-102: A Substudy of the Phase 3 ENVISION Trial: Abstract #4597, Poster #397; (iii) Treatment of Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer with UGN-102: Results of the Phase 3 ATLAS and ENVISION Studies. Published Abstract 501822."

P3 data • Bladder Cancer

UroGen Pharma (URGN) Shares Plummet After FDA Advisory Vote (Gurufocus) - "UroGen Pharma (URGN, Financial) experienced a significant stock drop of approximately 47% following a setback with the U.S. Food and Drug Administration (FDA). The FDA's Oncology Drug Advisory Committee (ODAC) voted against the company's bladder cancer treatment, UGN-102 (mitomycin), with a close vote of 4 in favor and 5 against. This decision comes as UGN-102 undergoes regulatory review in the U.S. The rejection poses a substantial challenge for UroGen Pharma, which is seeking approval for UGN-102 to treat recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC)."

ODAC • Stock price • Bladder Cancer

FDA ODAC Votes 5-to-4 Against UGN-102 Intravesical Solution for Bladder Cancer (Cancer Network) - "Based on a vote of 5-to-4, the FDA Oncologic Drug Advisory Committee has voted against a benefit/risk ratio of UGN-102 (mitomycin) for patients with low-grade intermediate-risk non-muscle invasive bladder cancer....The decision comes based on results from the phase 3 ENVISION trial....a single-arm study assessing UGN-102 in patients in the aforementioned population....TEAEs leading to treatment discontinuation occurred in 2.9% of patients, and 2.5% had TEAEs leading to study discontinuation, but none of which were related to the study procedure....Of note, 3 patients died during the study from pneumonia, cardiac failure, or an unknown reason. None of which was related to treatment."

ODAC • Bladder 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

UroGen Announces FDA Advisory Committee for UGN-102, an Investigational Treatment for Recurrent Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer (Businesswire) - "UroGen...today announced that the U.S. Food and Drug Administration (FDA) has scheduled an Oncologic Drugs Advisory Committee (ODAC) meeting on May 21, 2025 to review the new drug application (NDA) for UGN-102 (mitomycin) for intravesical solution, an investigational treatment for recurrent LG-IR-NMIBC....The FDA has stated in its correspondence to the Company that they intend to complete their review in time to meet the Prescription Drug User Fee Act (PDUFA) target action date of June 13, 2025....The NDA is supported by results from the pivotal Phase 3 ENVISION trial which demonstrated a 79.6% complete response (CR) rate at 3 months after first instillation of UGN-102..."

ODAC • PDUFA • Bladder Cancer

Neoadjuvant Mitomycin C Demonstrates Favorable Safety and Potential RFS Benefit in NMIBC (OncLive) - P3 | N=160 | 2021-003751-42 | "Treatment with neoadjuvant mitomycin C prior to transurethral resection of bladder tumor (TURBT) demonstrated a favorable safety profile in patients with non–muscle-invasive bladder cancer (NMIBC)....At a median follow-up of 19.4 months, findings showed that the Kaplan-Meier–estimated 12-months RFS rate was 88% (95% CI, 67%-96%) in both the standard arm (n = 27) and the neoadjuvant mitomycin C arm (n = 28; HR, 0.97; 95% CI, 0.2-4.8; P = .9). The 18-month RFS rates were 71% (95% CI, 46%-86%) in the standard arm vs 88% (95% CI, 67%-96%) in the neoadjuvant mitomycin C arm (HR, 0.4; 95% CI, 0.1-1.8; P = .27)."

P3 data • Bladder Cancer

New Patient-Reported Outcomes from UGN-102 Clinical Trials Show the Investigational Treatment Did Not Adversely Affect Functionality, Symptom Burden, and Quality of Life in Patients with LG-IR-NMIBC (Businesswire) - "The data (Moderated Poster - MP15) were presented at the AUA 2025 Annual Meeting in Las Vegas, Nevada...In the OPTIMA II, ATLAS, and ENVISION late-phase studies, most patients (≥91% in ATLAS, ≥94% in ENVISION) completed the questionnaires at baseline, three months, and 12 months or study end. Baseline scores indicated high levels of functioning and low symptom burden prior to treatment. UGN-102 did not cause sustained declines in functioning or symptom burden, and no measured domains or items exceeded the threshold for clinically significant worsening at three or 12 months, suggesting no negative impact on quality of life....Each study featured a six-week UGN-102 treatment period, a CR assessment at three months, and follow-up ranging from nine to 63 months after three-month CR. Patient-reported symptoms and health status were evaluated using the EORTC-QLQ-NMIBC24 at baseline, three months, and 12 months or study end."

Adverse events • Patient reported outcomes • Retrospective data • Bladder Cancer

New Long-Term Follow-Up Data from OPTIMA II Study of UGN-102 Demonstrates Median Duration of Response of Two Years in Patients with LG-IR-NMIBC (Businesswire) - P2b | N=63 | OPTIMA II (NCT03558503) | Sponsor: UroGen Pharma Ltd. | "Data Presented at the American Urological Association 2025...UroGen Pharma Ltd...announced new data from the OPTIMA II Phase 2b study of UGN‑102 (mitomycin) for intravesical solution demonstrate clinically meaningful two-year duration of response (24.2 months) by Kaplan-Meier analysis...Among the 41 patients achieving a complete response (CR) at three months, 25 remained in CR at 12 months, and 17 of these patients entered long-term follow-up. The median Kaplan–Meier estimate of duration of response for the 41 patients that achieved CR was 24.2 months (95% CI 9.72, 47.18), with a median follow-up time of 33.6 months (95% CI 10.78, 42.94). Twenty patients (48.8%) experienced recurrence of low-grade disease. One patient progressed to high-grade disease and one patient died due to a cardiac disorder. Five patients remained disease-free at the time of the four-year data analysis."

P2b data • Bladder Cancer

UroGen Announces Updated 18-Month Duration of Response (DOR) of 80.6% from the Phase 3 ENVISION Trial of UGN-102, an Investigational Treatment for Recurrent Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer (LG-IR-NMIBC) (Businesswire) - P3 | N=240 | ENVISION (NCT05243550) | Sponsor: UroGen Pharma Ltd. | "These data were featured today in an Oral Presentation Session (Abstract ID: PD12) at the AUA 2025 Annual Meeting....'This new update from the pivotal ENVISION trial of UGN-102 demonstrated a compelling probability of remaining in complete response of 80.6% at 18 months in patients who achieved a complete response (CR) at three months (79.6%)'....'The duration of response data from the ENVISION trial further underscores UGN-102’s potential to positively impact the treatment landscape for patients with recurrent LG-IR-NMIBC'....The ENVISION trial demonstrated a similar safety profile to that observed in other studies of UGN‑102. Median follow-up time at 18 months was 18.7 months after the three-month CR."

P3 data • Bladder 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

PATIENT-REPORTED SIDE-EFFECT BURDEN FOR PATIENTS WITH LOW-GRADE INTERMEDIATE-RISK NON-MUSCLE INVASIVE BLADDER CANCER RECEIVING TREATMENT WITH UGN-102 (AUA 2025) - P2b, P3 | "Treatment with UGN-102 did not appear to negatively impact symptom burden, patient function, or QoL. UGN-102 may be a potential nonsurgical treatment option that does not negatively impact QoL for patients with LG-IR-NMIBC."

Adverse events • Clinical • Anesthesia • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

TREATMENT OF LOW-GRADE INTERMEDIATE-RISK NON-MUSCLE INVASIVE BLADDER CANCER WITH UGN-102: LONG-TERM OUTCOMES OF THE OPTIMA II STUDY (AUA 2025) - P2b | "The estimated DoR in patients achieving CR after initial treatment with UGN-102 was about 2 years, and most cases of recurrence were of low-grade disease. These results demonstrate that treatment with UGN-102 results in a clinically meaningful DoR in patients with newly diagnosed or recurrent LG-IR-NMIBC. UGN-102 may represent a valid nonsurgical alternative to TURBT, the current standard of care, for treating these patients."

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

Psychometric validation of a quality of life scale in patients with low-grade intermediate-risk non-muscle invasive bladder cancer (AUA 2025) - " The Phase 3 ATLAS study included patients with newly diagnosed or recurrent LG-IR-NMIBC, randomized to 6 weekly intravesical instillations of UGN-102 (n=142) or transurethral resection of bladder tumor (n=140)... The results support the utilization of EORTC QLQ-NMIBC24 to assess HRQoL in individuals with LG-IR-NMIBC. Further, the values for MCID reported in these analyses, can aid in the clinical interpretation of the HRQoL in patients with LG-IR-NMIBC. Limitations of this study include the limited number of patients included in the analysis."

Clinical • HEOR • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Treatment of recurrent low-grade intermediate-risk non-muscle invasive bladder cancer with UGN-102: ongoing results from a single-arm, open-label, phase 3 trial (ENVISION) (AUA 2025) - P3 | "Results from ENVISION demonstrate that treatment with UGN-102 results in a robust CR rate in patients with recurrent LG-IR-NMIBC. Furthermore, trial participants who achieved an initial CR had a high probability of remaining disease-free 12 months later. UGN-102 may represent a well-tolerated, valuable alternative to TURBT for patients with LG-IR-NMIBC."

Clinical • P3 data • Bladder Cancer • Fatigue • Genito-urinary Cancer • Infectious Disease • Nephrology • Oncology • Solid Tumor

UroGen Pharma…Announces Updated 18-Month Duration of Response (DOR) of 80.6% from the Phase 3 ENVISION Trial of UGN-102 (Businesswire) - P3 | N=240 | ENVISION (NCT05243550) | Sponsor: UroGen Pharma Ltd. | "The Company reported updated 18-month DOR data from the Phase 3 ENVISION trial that evaluated UGN-102 in patients with LG-IR-NMIBC. The 18-month DOR data is consistent with prior Kaplan-Meier estimates, with a DOR rate of 80.6% (95% CI: 74.0, 85.7) at 18-months (N=101) compared to 82.5% (95% CI: 76.1, 87.3) at 12-months (N=146). Median follow-up time at 18 months was 18.7 months after the three-month CR."

P3 data • Bladder Cancer

Perceived impact on patient routines/responsibilities for surgery and a non-surgical primary treatment option in recurrent LG-IR-NMIBC: findings from the ENVISION phase 3 trial. (PubMed, J Urol) - P3 | "Patients perceived UGN-102 to be a favorable primary treatment alternative to traditional surgical resections for LG-IR-NMIBC. By focusing on the underexplored area of patient perceptions, this study provides key information urologists will need to conduct shared decision-making conversations."

Journal • P3 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Pain • Solid Tumor • Urology