VesiGel (mitomycin intravesicular) / UroGen - LARVOL DELTA

Meeting of the Oncologic Drugs Advisory Committee (FDA) - "On the morning of May 21, 2025, the Committee will discuss new drug application (NDA) 215793, for (mitomycin) intravesical solution, submitted by UroGen Pharma, Inc. The proposed indication (use) is for the treatment of adult patients with low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). On the afternoon of May 21, 2025, the Committee will discuss supplemental new drug application (sNDA) 211651/S-013, for TALZENNA (talazoparib) capsules, submitted by Pfizer Inc. The proposed indication (use) is in combination with enzalutamide for the treatment of adult patients with metastatic castration-resistant prostate cancer (mCRPC)."

FDA event • Bladder Cancer • Castration-Resistant Prostate 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

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 | "Clinical Trial Registration Number: NCT04688931/NCT05243550 The abstract will be released to the public on May 22, 2025 at 4:00 PM CDT"

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 | "Clinical Trial Registration Number: NCT05243550 The abstract will be released to the public on May 22, 2025 at 4:00 PM CDT"

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

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 | "Clinical Trial Registration Number: NCT05243550 The abstract will be released to the public on May 22, 2025 at 4:00 PM CDT"

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

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

Treatment of low-grade intermediate-risk non-muscle-invasive bladder cancer with UGN-102: Results of the phase 3 ATLAS and ENVISION studies. (ASCO-GU 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 non-surgical treatment option for these patients."

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

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-GU 2025) - P3 | "Although a non-significant higher event rate was observed at 3 months for patients with a higher tumor burden, the CRR was robust, and the majority of patients remained event free at 15 months. These results demonstrate that chemoablation using UGN-102 results in a high, clinically meaningful CRR in patients with recurrent LG-IR-NMIBC, regardless of tumor burden or multifocality. Study limitations were the small sample size of the comparator groups, single arm design, and post-hoc nature of the analysis."

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

UroGen Announces Results... (Businesswire) - P3 | N=240 | ENVISION (NCT05243550) | N=282 | ATLAS (NCT04688931) | Sponsor: UroGen Pharma Ltd. | "The analysis in another presentation....Studies highlights that in both studies, the CR rate in patients initially treated with UGN-102 was robust, with the majority of patients remaining event-free at 12 months after a three-month CR. A new analysis from the ATLAS trial highlights 54% decreased risk of recurrence, progression or death with UGN-102 compared to TURBT. The hazard ratio for DOR in ATLAS was 0.46 with a 95% confidence interval (0.24, 0.86) favoring the UGN-102 arm. Median DOR was not estimable in any arm due to low recurrence rates; the most common adverse event with UGN-102 in both studies was dysuria, occurring in 22.5% in ENVISION and 30.4% in ATLAS."

P3 data • Bladder Cancer

New Treatment Options for Non-Muscle-Invasive Bladder Cancer. (PubMed, Am Soc Clin Oncol Educ Book) - "Emerging approaches such as TAR-200 and UGN-102 offer novel intravesical delivery systems that enhance therapeutic efficacy while minimizing systemic adverse effects. Viral therapies, including nadofaragene firadenovec and CG0070, deliver immune-activating and oncolytic agents directly to urothelial tumor cells. Additionally, immune checkpoint inhibitors such as pembrolizumab and durvalumab have demonstrated potential for systemic treatments in BCG-unresponsive NMIBC and may show even more promise in combinations...For low-grade NMIBC, efforts are underway to de-escalate care through active surveillance and novel adjuvant therapies, reducing the need for repeated TURBT procedures. Together, these advancements highlight a promising shift toward personalized, bladder-preserving strategies that prioritize patient quality of life while addressing unmet needs in NMIBC management."

Journal • Review • Bladder Cancer • Gene Therapies • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

UroGen Announces Results from Subgroup Analyses of the Pivotal ENVISION Trial Evaluating Impact of Baseline Tumor Burden and Focality on Response to UGN-102 (Businesswire) - P3 | N=240 | ENVISION (NCT05243550) | Sponsor: UroGen Pharma Ltd. | "The poster presentation...was presented today at ASCO GU 2025...The ENVISION study included 240 patients with recurrent LG-IR-NMIBC who received at least one dose of UGN-102 and 95% (228 patients) received all six weekly intravesical instillations of UGN-102. In this pre-specified analysis CR rates were 82.8% for patients with a tumor burden ≤3 cm (n=149/180) compared to 73.2% for those with a tumor burden >3 cm (n=30/41). Of the patients with CR at three months, 15.4% vs 20.0% experienced recurrence of low-grade disease, progression (either in stage or grade), or death (unrelated to treatment) by 15 months. In the 191 patients with multiple vs single tumors who achieved a CR, the three-month CR was 79.3% vs 82.9%, with recurrence rates of 18.5% vs 11.8%....The ENVISION trial demonstrated a similar safety profile to that observed in other studies of UGN-102."

P3 data • Bladder Cancer

UTOPIA: A Phase 3 Study of UGN-103 for Treatment of Patients With Low-grade Intermediate-risk Non-muscle Invasive Bladder Cancer (clinicaltrials.gov) - P3 | N=87 | Recruiting | Sponsor: UroGen Pharma Ltd. | Trial completion date: Mar 2026 ➔ Jul 2026 | Trial primary completion date: Mar 2025 ➔ Jul 2025

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

ENVISION Trial Results Published in the February Issue of The Journal of Urology Highlight UGN-102 Achievement of 82.3% Duration of Response at 12 Months Paving the Way for the Potential First FDA-Approved Treatment for LG-IR-NMIBC in June 2025 (Businesswire) - P3 | N=240 | ENVISION (NCT05243550) | Sponsor: UroGen Pharma Ltd. | "In the ENVISION trial, UGN-102 treatment demonstrated an impressive 82.3% (95% CI, 75.9%, 87.1%) 12-month duration of response (DOR) by Kaplan-Meier estimate (n=108) in patients who achieved a CR at three months after the first instillation of UGN-102 (mitomycin) for intravesical solution. The Kaplan-Meier estimates for DOR at 15 months (n=43) and 18 months (n=9) following the 3-month CR were both 80.9% (95% CI, 73.9%, 86.2%). The ENVISION trial also met its primary endpoint, showing a 79.6% (95% CI, 73.9%, 84.5%) CR rate at three months in patients treated with UGN-102....The most common treatment-emergent adverse events (TEAEs) in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention."

P3 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Urothelial Cancer

UGN-102 Showed Promising Long-Term Results in the Phase 3 ENVISION Trial, Potentially Paving the Way for First FDA-Approved Treatment for LG-IR-NMIBC (Businesswire) - P3 | N=240 | ENVISION (NCT05243550) | Sponsor: UroGen Pharma Ltd. | "UroGen Pharma...announced the presentation of the Phase 3 ENVISION trial’s efficacy and safety results at the Society of Urologic Oncology (SUO) annual meeting....In the ENVISION study, UGN-102 treatment showed an impressive 82.3% (95% CI: 75.9%, 87.1%) duration of response (DOR) at 12 months, according to the Kaplan-Meier estimate, in patients who achieved a complete response (CR) at 3 months following the initial treatment with UGN-102. The DOR at 15 months (n=43) and 18 months (n=9) remained robust, both at 80.9% (95% CI: 73.9%, 86.2%) according to the Kaplan-Meier estimates. These results build upon the trial’s positive primary endpoint, a 79.6% (95% CI: 73.9%, 84.5%) CR rate 3 months after the first instillation of UGN-102."

P3 data • Bladder Cancer

UroGen Announces New Data Presentations at the Society of Urologic Oncology 2024 Annual Meeting Highlighting Urothelial Cancer Portfolio Aimed at Addressing Unmet Needs (Businesswire) - "UroGen Pharma...announced that new data on UGN-102 (mitomycin) for intravesical solution, JELMYTO (mitomycin) for pyelocalyceal solution, and UGN-301(zalifrelimab intravesical solution) will be presented at the Society of Urologic Oncology (SUO) 2024 annual meeting being held in Dallas, Texas from December 4 - 6."

Clinical data • Bladder Cancer • Urothelial Cancer