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
UroGen Pharma (NASDAQ: URGN) announced significant 24-month duration of response (DOR) data from its Phase 3 ENVISION trial for ZUSDURI™, the first FDA-approved treatment for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).
The trial demonstrated a 72.2% DOR at 24 months (95% CI 64.1%, 78.8%) in patients who achieved complete response (CR) at three months. The initial CR rate at three months was 79.6%, with a median follow-up time of 23.7 months. Notably, the median DOR has not been reached, indicating sustained efficacy.
ZUSDURI represents a significant advancement over the current standard of care, which relies on repeated surgical procedures (TURBT). An estimated 59,000 patients with LG-IR-NMIBC experience recurrence annually. The most common adverse reactions were primarily mild to moderate, with serious adverse reactions occurring in 12% of patients.
UroGen Pharma (NASDAQ: URGN) ha annunciato dati significativi sulla durata della risposta (DOR) a 24 mesi del suo studio di Fase 3 ENVISION per ZUSDURI™, il primo trattamento approvato dalla FDA per il carcinoma della vescica non muscolo-invasivo a basso grado e rischio intermedio ricorrente (LG-IR-NMIBC).
Lo studio ha evidenziato un 72,2% di DOR a 24 mesi (IC 95% 64,1%, 78,8%) nei pazienti che hanno raggiunto una risposta completa (CR) a tre mesi. Il tasso iniziale di CR a tre mesi è stato del 79,6%, con un tempo mediano di follow-up di 23,7 mesi. È importante sottolineare che la DOR mediana non è stata ancora raggiunta, indicando un'efficacia duratura.
ZUSDURI rappresenta un importante progresso rispetto allo standard di cura attuale, che si basa su procedure chirurgiche ripetute (TURBT). Si stima che 59.000 pazienti con LG-IR-NMIBC sperimentino una recidiva ogni anno. Le reazioni avverse più comuni sono state principalmente lievi o moderate, con reazioni avverse gravi riscontrate nel 12% dei pazienti.
UroGen Pharma (NASDAQ: URGN) anunció datos significativos de duración de la respuesta (DOR) a 24 meses de su ensayo de Fase 3 ENVISION para ZUSDURI™, el primer tratamiento aprobado por la FDA para el cáncer de vejiga no músculo invasivo de bajo grado y riesgo intermedio recurrente (LG-IR-NMIBC).
El ensayo mostró un 72,2% de DOR a 24 meses (IC 95% 64,1%, 78,8%) en pacientes que lograron una respuesta completa (CR) a los tres meses. La tasa inicial de CR a los tres meses fue del 79,6%, con un seguimiento medio de 23,7 meses. Cabe destacar que la DOR mediana no se ha alcanzado, lo que indica una eficacia sostenida.
ZUSDURI representa un avance significativo respecto al estándar actual de cuidado, que se basa en procedimientos quirúrgicos repetidos (TURBT). Se estima que 59,000 pacientes con LG-IR-NMIBC presentan recurrencia anualmente. Las reacciones adversas más comunes fueron principalmente leves a moderadas, con reacciones adversas graves en el 12% de los pacientes.
UroGen Pharma (NASDAQ: URGN)는 재발성 저등급 중간위험 비근육침윤성 방광암(LG-IR-NMIBC)에 대해 FDA 승인을 받은 최초의 치료제인 ZUSDURI™의 3상 ENVISION 임상시험에서 24개월 반응 지속 기간(DOR) 데이터를 발표했습니다.
임상시험 결과, 3개월 시점에 완전 관해(CR)를 달성한 환자에서 24개월 DOR이 72.2%(95% 신뢰구간 64.1%, 78.8%)로 나타났습니다. 3개월 시점 초기 CR 비율은 79.6%였으며, 중앙 추적 관찰 기간은 23.7개월입니다. 특히, 중앙 DOR에 도달하지 않아 지속적인 효능이 확인되었습니다.
ZUSDURI는 반복적인 수술 절차(TURBT)에 의존하는 현재 표준 치료법에 비해 중요한 진전을 의미합니다. 매년 약 59,000명의 LG-IR-NMIBC 환자가 재발을 경험합니다. 가장 흔한 부작용은 주로 경증에서 중등도의 반응이었으며, 심각한 부작용은 12% 환자에서 발생했습니다.
UroGen Pharma (NASDAQ : URGN) a annoncé des données significatives de durée de réponse (DOR) à 24 mois issues de son essai de phase 3 ENVISION pour ZUSDURI™, le premier traitement approuvé par la FDA pour le cancer de la vessie non invasif de bas grade à risque intermédiaire récurrent (LG-IR-NMIBC).
L’essai a montré un taux de DOR de 72,2 % à 24 mois (IC 95 % : 64,1 %, 78,8 %) chez les patients ayant obtenu une réponse complète (CR) à trois mois. Le taux initial de CR à trois mois était de 79,6 %, avec un suivi médian de 23,7 mois. Il est à noter que la DOR médiane n’a pas encore été atteinte, ce qui indique une efficacité durable.
ZUSDURI représente une avancée majeure par rapport au traitement standard actuel, qui repose sur des interventions chirurgicales répétées (TURBT). On estime que 59 000 patients atteints de LG-IR-NMIBC présentent une récidive chaque année. Les effets indésirables les plus fréquents étaient principalement légers à modérés, avec des effets indésirables graves survenus chez 12 % des patients.
UroGen Pharma (NASDAQ: URGN) gab bedeutende Daten zur 24-monatigen Ansprechdauer (DOR) aus der Phase-3-Studie ENVISION für ZUSDURI™ bekannt, der ersten von der FDA zugelassenen Behandlung für rezidivierendes niedriggradiges, intermediäres Risiko, nicht muskelinvasives Blasenkarzinom (LG-IR-NMIBC).
Die Studie zeigte eine 72,2%ige DOR nach 24 Monaten (95% KI 64,1%, 78,8%) bei Patienten, die nach drei Monaten eine komplette Remission (CR) erreichten. Die anfängliche CR-Rate nach drei Monaten lag bei 79,6%, bei einer medianen Nachbeobachtungszeit von 23,7 Monaten. Bemerkenswert ist, dass die mediane DOR noch nicht erreicht wurde, was auf eine anhaltende Wirksamkeit hinweist.
ZUSDURI stellt einen bedeutenden Fortschritt gegenüber dem aktuellen Standard dar, der auf wiederholten chirurgischen Eingriffen (TURBT) basiert. Schätzungsweise 59.000 Patienten mit LG-IR-NMIBC erleben jährlich ein Rezidiv. Die häufigsten Nebenwirkungen waren überwiegend mild bis moderat, ernsthafte Nebenwirkungen traten bei 12% der Patienten auf.
- First and only FDA-approved medicine for recurrent LG-IR-NMIBC
- High durability with 72.2% duration of response at 24 months
- Strong initial complete response rate of 79.6% at three months
- Median duration of response not yet reached, indicating sustained efficacy
- Outpatient treatment option avoiding repeated surgical procedures
- Most adverse reactions were mild to moderate
- Serious adverse reactions occurred in 12% of patients
- Multiple adverse reactions reported including urinary retention and urethral stenosis
Insights
UroGen's ZUSDURI shows strong 72.2% 24-month durability in bladder cancer patients who initially responded to treatment, representing significant advancement over surgical standard.
The Phase 3 ENVISION trial data for ZUSDURI (mitomycin) reveals impressive 72.2% duration of response at 24 months among patients who achieved complete response at three months (which was 79.6% of treated patients). This is particularly significant since the median duration of response has not yet been reached after 23.7 months of follow-up, indicating potentially longer-lasting effects.
The results are clinically meaningful for several reasons. Low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) is characterized by high recurrence rates, traditionally managed through repeated TURBT surgical procedures under general anesthesia. These surgeries significantly impact quality of life, especially considering the patient population is typically elderly with comorbidities. An estimated 59,000 patients with LG-IR-NMIBC recur annually.
ZUSDURI represents the first FDA-approved therapy specifically for recurrent LG-IR-NMIBC, offering an outpatient treatment alternative to surgery. The stable event rate over time among responders suggests the therapy provides durable protection against recurrence, potentially reducing the need for repeated surgical interventions.
The safety profile shows primarily mild to moderate adverse reactions, with serious adverse reactions occurring in 12% of patients. Common side effects include dysuria, electrolyte/enzyme changes, and hematuria, with urinary retention (0.8%) and urethral stenosis (0.4%) being the most significant serious adverse reactions reported.
This represents a potentially paradigm-shifting approach to managing this chronic cancer, offering patients a non-surgical option that could meaningfully extend the time between recurrences while avoiding the risks associated with repeated surgeries under anesthesia.
- 24-month Duration of Response (DOR) of
72.2% by Kaplan-Meier estimate was attained in patients who achieved a complete response (CR) at three months (79.6% )
PRINCETON, N.J., Aug. 05, 2025 (GLOBE NEWSWIRE) -- UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, today announced the 24-month DOR of
“This latest update from the pivotal ENVISION trial of ZUSDURI showed that, among patients who achieved a complete response at three months, the probability of remaining event-free 24 months after CR was
The existing standard of care for LG-IR-NMIBC is a surgical procedure typically conducted under general anesthesia called transurethral resection of bladder tumor (TURBT). Repeated TURBT procedures can impact patients’ physical health and quality of life and can even be associated with an increased risk in mortality. Due to high recurrence rates, LG-IR-NMIBC patients, who are typically elderly with comorbidities, will likely need multiple TURBTs under general anesthesia over the course of their lifetime. An estimated 59,000 patients with LG-IR-NMIBC recur annually.
According to Mark Schoenberg, M.D., Chief Medical Officer, UroGen, “The 24-month duration of response data from the ENVISION trial underscore the transformative potential of ZUSDURI (formerly known as UGN-102) for adult patients with recurrent LG-IR-NMIBC. This is a population that has long endured a cycle of recurrence and repeat surgeries, a burden that weighs heavily, particularly on older adults. With ZUSDURI now approved, we have a new treatment option that can help deliver durable complete responses and meaningfully extend event-free intervals. This represents a major advance in the way we care for these patients.”
The most common (≥
About ZUSDURI
ZUSDURI (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, approved for the treatment of adults with recurrent LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel® technology (a sustained release, hydrogel-based formulation), ZUSDURI is delivered directly into the bladder by a trained healthcare professional using a urinary catheter in an outpatient setting, thereby enabling the treatment of tumors by non-surgical means.
About Non-Muscle Invasive Bladder Cancer (NMIBC)
LG-IR-NMIBC affects around 82,000 people in the U.S. every year and of those, an estimated 59,000 are recurrent. Bladder cancer primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. Guideline recommendations for the management of NMIBC include TURBT as the standard of care. Up to 70 percent of NMIBC patients experience at least one recurrence, and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures. Learn more about non-muscle invasive bladder cancer at www.BladderCancerAnswers.com.
About ENVISION
The Phase 3 ENVISION trial is a single-arm, multinational, multicenter pivotal study evaluating the efficacy and safety of ZUSDURI (mitomycin) for intravesical solution as a chemoablative therapy in adult patients with recurrent LG-IR-NMIBC. The Phase 3 ENVISION trial completed target enrollment with 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of ZUSDURI. The primary endpoint evaluated the CR rate at three months after the first instillation, and the key secondary endpoint evaluates 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 our second product, ZUSDURI (mitomycin) for intravesical solution for adult patients with recurrent LG-IR-NMIBC, are designed to ablate tumors by non-surgical means. UroGen is headquartered in Princeton, NJ with operations in Israel. Visit www.UroGen.com to learn more or follow us on X (Twitter), @UroGenPharma.
IMPORTANT SAFETY INFORMATION
APPROVED USE FOR ZUSDURI
ZUSDURI (mitomycin) for intravesical solution is a prescription medicine used to treat adults with a type of cancer of the lining of the bladder called low-grade intermediate risk non-muscle invasive bladder cancer (LG-IR-NMIBC) after previously receiving bladder surgery to remove tumor that did not work or is no longer working.
IMPORTANT SAFETY INFORMATION
You should not receive ZUSDURI if you have a hole or tear (perforation) of your bladder or if you have had an allergic reaction to mitomycin or to any of the ingredients in ZUSDURI.
Before receiving ZUSDURI, tell your healthcare provider about all of your medical conditions, including if you:
- have kidney problems
- are pregnant or plan to become pregnant. ZUSDURI can harm your unborn baby. You should not become pregnant during treatment with ZUSDURI. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with ZUSDURI.
Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with ZUSDURI and for 6 months after the last dose.
Males being treated with ZUSDURI: You should use effective birth control (contraception) during treatment with ZUSDURI and for 3 months after the last dose. - are breastfeeding or plan to breastfeed. It is not known if ZUSDURI passes into your breast milk. Do not breastfeed during treatment with ZUSDURI and for 1 week after the last dose.
How will I receive ZUSDURI?
- You will receive your ZUSDURI dose from your healthcare provider 1 time a week for 6 weeks into your bladder through a tube called a urinary catheter. It is important that you receive all 6 doses of ZUSDURI according to your healthcare provider’s instructions.
- If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment.
- During treatment with ZUSDURI, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.
After receiving ZUSDURI:
- ZUSDURI may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 24 hours.
- To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
- Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.
The most common side effects of ZUSDURI include: increased blood creatinine levels, increased blood potassium levels, trouble with urination, decreased red blood cell counts, increase in certain blood liver tests, increased or decreased white blood cell counts, urinary tract infection, and blood in your urine.
You are encouraged to report negative side effects of prescription drugs to the FDA.
Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to UroGen Pharma at 1-855-987-6436.
Please see ZUSDURI Full Prescribing Information, including the Patient Information, for additional information.
ZUSDURI™ is a trademark and UroGen® is a registered trademark of UroGen Pharma, Ltd.
Copyright©2025 UroGen Pharma, Inc. All rights reserved.
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 potential benefits of ZUSDURI, including as an outpatient treatment option, its potential to provide durable CRs and clinically meaningful recurrence-free intervals and as a compelling alternative to TURBT given the high recurrence rates of LG-IR-NMIBC; the estimated annual U.S. patient population and demographics for LG-IR-NMIBC; the potential of UroGen’s proprietary RTGel technology to improve therapeutic profiles of existing drugs other than mitomycin; and UroGen’s sustained release technology making local delivery potentially more effective as compared to other treatment options. Words such as “can,” “estimate,” “likely,” “may,” “potential,” “will” or other words that convey uncertainty of future events or outcomes are used to identify these forward-looking statements. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: clinical results may not be indicative of results that may be observed in the future, including in larger populations; potential safety and other complications related to UroGen’s products; the ability to maintain regulatory approval; complications associated with commercialization activities; labeling limitations; competition in UroGen’s industry; the scope, progress and expansion of developing and commercializing UroGen’s products and product candidates; the size and growth of the market(s) therefor and the rate and degree of market acceptance thereof vis-à-vis alternative therapies or procedures, such as surgery; UroGen’s ability to attract or retain key management, members of the board of directors and other personnel; UroGen’s RTGel technology and ZUSDURI may not perform as expected; UroGen may not successfully develop and receive regulatory approval of any other product that incorporates RTGel technology; and the impacts of general macroeconomic and geopolitical conditions on UroGen’s business and financial position. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section of UroGen’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, filed with the SEC on May 12, 2025, the events and circumstances discussed in such forward-looking statements may not occur, and UroGen’s actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to UroGen as of the date of this release.
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
FAQ
What are the 24-month results for UroGen's ZUSDURI Phase 3 ENVISION trial?
What is the complete response rate for ZUSDURI in bladder cancer treatment?
How many patients with LG-IR-NMIBC could benefit from ZUSDURI annually?
What are the main side effects of ZUSDURI in bladder cancer treatment?
How does ZUSDURI treatment differ from current bladder cancer standard of care?
