UroGen Announces Results from Subgroup Analyses of the Pivotal ENVISION Trial Evaluating Impact of Baseline Tumor Burden and Focality on Response to UGN-102
UroGen Pharma (Nasdaq: URGN) announced results from subgroup analyses of the pivotal ENVISION trial evaluating UGN-102 for treating recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The study demonstrated strong complete response (CR) rates across different patient subgroups:
- 82.8% CR for patients with tumor burden ≤3 cm vs 73.2% for >3 cm
- 79.3% CR for patients with multiple tumors vs 82.9% for single tumors
The ENVISION trial included 240 patients, with 95% receiving all six weekly intravesical instillations. The overall CR rate was 79.6% at three months, with 82.3% probability of maintaining response after 12 months. UroGen completed its NDA submission to the FDA with a PDUFA date of June 13, 2025. A separate ATLAS trial analysis showed UGN-102 reduced risk of recurrence, progression or death by 54% compared to TURBT.
UroGen Pharma (Nasdaq: URGN) ha annunciato i risultati delle analisi di sottogruppo dello studio clinico fondamentale ENVISION che valuta UGN-102 per il trattamento del cancro della vescica non muscolo invasivo a basso grado e rischio intermedio ricorrente (LG-IR-NMIBC). Lo studio ha dimostrato tassi di risposta completa (CR) elevati tra i diversi sottogruppi di pazienti:
- 82,8% di CR per i pazienti con carico tumorale ≤3 cm rispetto al 73,2% per >3 cm
- 79,3% di CR per i pazienti con tumori multipli rispetto all'82,9% per tumori singoli
Il trial ENVISION ha incluso 240 pazienti, con il 95% che ha ricevuto tutte e sei le instillazioni intravesicali settimanali. Il tasso complessivo di CR è stato del 79,6% a tre mesi, con una probabilità dell'82,3% di mantenere la risposta dopo 12 mesi. UroGen ha completato la sua sottomissione NDA alla FDA con una data PDUFA del 13 giugno 2025. Un'analisi separata del trial ATLAS ha mostrato che UGN-102 ha ridotto il rischio di recidiva, progressione o morte del 54% rispetto al TURBT.
UroGen Pharma (Nasdaq: URGN) anunció los resultados de los análisis de subgrupos del ensayo pivotal ENVISION que evalúa UGN-102 para el tratamiento del cáncer de vejiga no musculoinvasivo de bajo grado y riesgo intermedio recurrente (LG-IR-NMIBC). El estudio demostró tasas de respuesta completa (CR) fuertes en diferentes subgrupos de pacientes:
- 82.8% de CR para pacientes con carga tumoral ≤3 cm frente al 73.2% para >3 cm
- 79.3% de CR para pacientes con tumores múltiples frente al 82.9% para tumores únicos
El ensayo ENVISION incluyó a 240 pacientes, con el 95% recibiendo las seis instilaciones intravesicales semanales. La tasa general de CR fue del 79.6% a los tres meses, con una probabilidad del 82.3% de mantener la respuesta después de 12 meses. UroGen completó su presentación NDA a la FDA con una fecha PDUFA del 13 de junio de 2025. Un análisis separado del ensayo ATLAS mostró que UGN-102 redujo el riesgo de recurrencia, progresión o muerte en un 54% en comparación con TURBT.
UroGen Pharma (Nasdaq: URGN)은 재발성 저등급 중간 위험 비근육 침습성 방광암(LG-IR-NMIBC) 치료를 위한 UGN-102를 평가하는 주요 ENVISION 시험의 하위 그룹 분석 결과를 발표했습니다. 이 연구는 다양한 환자 하위 그룹에서 높은 완전 반응(CR) 비율을 보여주었습니다:
- 종양 부담이 ≤3 cm인 환자의 CR 비율은 82.8%이고, >3 cm인 경우는 73.2%
- 다발성 종양 환자의 CR 비율은 79.3%이고, 단일 종양 환자는 82.9%
ENVISION 시험은 240명의 환자를 포함하였으며, 95%가 6회의 주간 방광 내 주입을 모두 받았습니다. 전체 CR 비율은 3개월 후 79.6%였으며, 12개월 후에도 반응을 유지할 확률은 82.3%였습니다. UroGen은 FDA에 NDA 제출을 완료하였으며 PDUFA 날짜는 2025년 6월 13일입니다. 별도의 ATLAS 시험 분석에서는 UGN-102가 TURBT에 비해 재발, 진행 또는 사망의 위험을 54% 줄였다고 보고했습니다.
UroGen Pharma (Nasdaq: URGN) a annoncé les résultats des analyses de sous-groupes de l'essai pivot ENVISION évaluant UGN-102 pour le traitement du cancer de la vessie non invasif à bas grade et à risque intermédiaire récurrent (LG-IR-NMIBC). L'étude a démontré des taux de réponse complète (CR) élevés parmi différents sous-groupes de patients :
- 82,8 % de CR pour les patients ayant une charge tumorale ≤3 cm contre 73,2 % pour >3 cm
- 79,3 % de CR pour les patients avec des tumeurs multiples contre 82,9 % pour des tumeurs uniques
L'essai ENVISION a inclus 240 patients, dont 95 % ont reçu les six instillations intravésicales hebdomadaires. Le taux global de CR était de 79,6 % à trois mois, avec une probabilité de 82,3 % de maintenir la réponse après 12 mois. UroGen a complété sa soumission NDA à la FDA avec une date PDUFA du 13 juin 2025. Une analyse séparée de l'essai ATLAS a montré qu'UGN-102 réduisait le risque de récidive, de progression ou de décès de 54 % par rapport à TURBT.
UroGen Pharma (Nasdaq: URGN) hat die Ergebnisse der Subgruppenanalysen der entscheidenden ENVISION-Studie bekannt gegeben, die UGN-102 zur Behandlung von wiederkehrendem, niedriggradigem, intermediärem Risiko bei nicht-muskelinvasivem Blasenkrebs (LG-IR-NMIBC) evaluiert. Die Studie zeigte starke vollständige Ansprechquoten (CR) in verschiedenen Patientensubgruppen:
- 82,8% CR für Patienten mit Tumorlast ≤3 cm im Vergleich zu 73,2% für >3 cm
- 79,3% CR für Patienten mit multiplen Tumoren im Vergleich zu 82,9% für Einzeltumoren
Die ENVISION-Studie umfasste 240 Patienten, von denen 95% alle sechs wöchentlichen intravesikalen Instillationen erhielten. Die Gesamtrate der CR betrug 79,6% nach drei Monaten, mit einer Wahrscheinlichkeit von 82,3%, die Antwort nach 12 Monaten aufrechtzuerhalten. UroGen hat seine NDA-Einreichung bei der FDA abgeschlossen, mit einem PDUFA-Datum vom 13. Juni 2025. Eine separate Analyse der ATLAS-Studie zeigte, dass UGN-102 das Risiko von Rückfällen, Progression oder Tod um 54% im Vergleich zu TURBT reduzierte.
- High complete response rates across all patient subgroups (73.2-82.9%)
- 82.3% probability of maintaining response after 12 months
- 54% decreased risk of recurrence compared to standard treatment (TURBT)
- NDA submission completed ahead of schedule
- 15.4-20% of patients experienced disease recurrence, progression, or death by 15 months
- Study limitations include small sample size of comparator groups
- Multiple adverse events reported including dysuria, hematuria, and urinary tract infection
Insights
The ENVISION trial subgroup analyses reveal compelling efficacy data that significantly strengthen UGN-102's regulatory and commercial prospects. The high complete response rates across different tumor burdens (82.8% for ≤3 cm and 73.2% for >3 cm) demonstrate remarkable consistency, suggesting broad applicability across the patient population. This is particularly significant as tumor burden often impacts treatment efficacy in oncology.
The durability of response is especially noteworthy, with only 15.4% to 20.0% of patients experiencing disease recurrence, progression, or death by 15 months post-treatment. This compares favorably to historical recurrence rates with standard TURBT procedures, which can exceed 50% within one year. The ATLAS trial's demonstration of a 54% reduced risk of recurrence versus TURBT provides compelling evidence for UGN-102's superior efficacy.
The safety profile appears manageable, with primarily mild-to-moderate adverse events that resolve over time. The consistent safety data across multiple trials strengthens the regulatory package. With the NDA submission completed and a PDUFA date set for June 13, 2025, UroGen is well-positioned for potential commercialization in the second half of 2025.
The positive data across both ENVISION and ATLAS trials, combined with the unmet need in LG-IR-NMIBC, suggests significant market potential. The ability to reduce repeated surgical interventions could drive strong adoption among both physicians and patients, potentially establishing UGN-102 as a new standard of care in this indication.
- Results presented at the American Society of Clinical Oncology’s Genitourinary Cancers Symposium (ASCO GU 2025)
- Treatment with UGN-102 resulted in durable complete response (CR) rates across subgroups with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC)
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CR rates were:
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82.8% for patients with a tumor burden ≤3 cm compared to73.2% for those with a tumor burden >3 cm -
79.3% for patients with multiple tumors compared to82.9% for those with single tumors
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- CR rates and duration of response (DOR) patterns were similar across tumor burden and focality subgroups
"In this subgroup analysis on tumor burden and focality from the ENVISION study, the overall CR rate was consistent, and a majority of patients maintained their response 12 months after achieving CR at three months," said Sandip M. Prasad, M.D., Director of Urology at Morristown Medical Center and lead author of the study. "These results provide evidence of UGN-102’s potential to become an effective and durable treatment option for patients with recurrent LG-IR-NMIBC, regardless of tumor size or focality."
The ENVISION study included 240 patients with recurrent LG-IR-NMIBC who received at least one dose of UGN-102 and
“We are encouraged by these results, which highlight the potential of UGN-102 to provide durable responses in patients with recurrent LG-IR-NMIBC,” said Mark Schoenberg, M.D., Chief Medical Officer of UroGen. “These pre-specified subgroup analyses further strengthen the growing body of evidence supporting UGN-102 as a treatment option for patients dealing with the challenges of disease recurrence and the need for repeated surgeries.”
The ENVISION trial’s primary efficacy and safety results, previously presented, showed that UGN-102 delivered a
These findings further support UGN-102’s potential to alter the treatment landscape for patients with LG-IR-NMIBC, a condition where recurrence and progression remain significant clinical challenges.
UroGen initiated the submission of a rolling new drug application (NDA) to the
The most common treatment-emergent adverse events (TEAEs) in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. The TEAEs were typically mild-to-moderate in severity and either resolved or were resolving. The ENVISION trial demonstrated a similar safety profile to that observed in other studies of UGN-102.
Study limitations include the small sample size of the comparator groups and the single arm design.
The analysis in another presentation titled, Treatment of Low-Grade Intermediate-Risk Non-Muscle-Invasive Bladder Cancer with UGN-102: Results of the Phase 3 ATLAS and ENVISION 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
About UGN-102
UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently in Phase 3 development for the treatment of LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel® technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional. UroGen completed the NDA submission in August, ahead of schedule. The FDA accepted the NDA for UGN-102 and assigned a PDUFA goal date of June 13, 2025.
About Non-Muscle Invasive Bladder Cancer (NMIBC)
In the
About ENVISION
The Phase 3 ENVISION trial is a single-arm, multinational, multicenter study evaluating the efficacy and safety of UGN-102 (mitomycin) for intravesical solution as a chemoablative therapy in patients with LG-IR-NMIBC. The Phase 3 ENVISION trial completed target enrollment with approximately 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of UGN-102. The primary endpoint evaluated the CR rate at the three-month assessment after the first instillation, and the key secondary endpoint evaluated durability over time in patients who achieved a CR at the three-month assessment. Learn more about the Phase 3 ENVISION trial at www.clinicaltrials.gov (NCT05243550).
About UroGen Pharma Ltd.
UroGen is a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers because patients deserve better options. UroGen has developed RTGel reverse-thermal hydrogel, a proprietary sustained-release, hydrogel-based platform technology that has the potential to improve the therapeutic profiles of existing drugs. UroGen’s sustained release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. Our first product to treat low-grade upper tract urothelial cancer and investigational treatment UGN-102 (mitomycin) for intravesical solution for patients with LG-IR-NMIBC are designed to ablate tumors by non-surgical means. UroGen is headquartered in
Forward-Looking Statements
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding: the ENVISION subgroup analyses providing evidence of UGN-102's potential to become an effective and durable treatment option for patients with recurrent LG-IR-NMIBC, regardless of tumor size or focality and further strengthening the growing body of evidence supporting UGN-102 as a treatment option for patients dealing with the challenges of disease recurrence and repeated surgeries; the potential benefits to patients and opportunities for UGN-102, if approved, including to alter the treatment landscape for patients with LG-IR-NMIBC; statements related to UroGen’s NDA submission and expected PDUFA target action date for UGN-102; the estimated annual
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INVESTOR CONTACT:
Vincent Perrone
Senior Director, Investor Relations
vincent.perrone@urogen.com
609-460-3588 ext. 1093
MEDIA CONTACT:
Cindy Romano
Director, Corporate Communications
cindy.romano@urogen.com
609-460-3583 ext. 1083
Source: UroGen Pharma Ltd.
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