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
UroGen Pharma (NASDAQ: URGN) has published promising five-year extension study results of their Phase 2b OPTIMA II trial for ZUSDURI™ (mitomycin) in treating low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).
The study demonstrated significant durability of response: Among 41 patients who achieved complete response (CR) at three months, the median duration of response was 24.2 months. For the 17 patients who entered long-term follow-up, the median duration of response reached 42.1 months. Notably, 94% of long-term follow-up patients were recurrent cases.
The most common adverse events included dysuria (41%), pollakiuria (21%), and hematuria (16%). ZUSDURI is specifically indicated for adults with recurrent LG-IR-NMIBC, offering a non-surgical treatment alternative in an outpatient setting.
UroGen Pharma (NASDAQ: URGN) ha pubblicato risultati promettenti a cinque anni dello studio di estensione della Fase 2b OPTIMA II per ZUSDURI™ (mitomicina) nel trattamento del carcinoma della vescica non muscolo-invasivo a basso grado e rischio intermedio (LG-IR-NMIBC).
Lo studio ha mostrato una significativa durata della risposta: tra 41 pazienti che hanno raggiunto una risposta completa (CR) a tre mesi, la durata mediana della risposta è stata di 24,2 mesi. Per i 17 pazienti seguiti a lungo termine, la durata mediana della risposta ha raggiunto i 42,1 mesi. È importante sottolineare che il 94% dei pazienti in follow-up a lungo termine erano casi recidivi.
Gli eventi avversi più comuni includevano disuria (41%), pollachiuria (21%) ed ematuria (16%). ZUSDURI è indicato specificamente per adulti con LG-IR-NMIBC recidivante, offrendo un'alternativa non chirurgica in regime ambulatoriale.
UroGen Pharma (NASDAQ: URGN) ha publicado resultados prometedores a cinco años del estudio de extensión de su ensayo de Fase 2b OPTIMA II para ZUSDURI™ (mitomicina) en el tratamiento del cáncer de vejiga no músculo invasivo de bajo grado y riesgo intermedio (LG-IR-NMIBC).
El estudio demostró una durabilidad significativa de la respuesta: entre 41 pacientes que lograron respuesta completa (CR) a los tres meses, la mediana de duración de la respuesta fue de 24,2 meses. Para los 17 pacientes que entraron en seguimiento a largo plazo, la mediana de duración de la respuesta alcanzó 42,1 meses. Cabe destacar que el 94% de los pacientes en seguimiento a largo plazo eran casos recurrentes.
Los eventos adversos más comunes incluyeron disuria (41%), polaquiuria (21%) y hematuria (16%). ZUSDURI está indicado específicamente para adultos con LG-IR-NMIBC recurrente, ofreciendo una alternativa de tratamiento no quirúrgico en un entorno ambulatorio.
UroGen Pharma (NASDAQ: URGN)는 저등급 중간 위험 비근육 침습성 방광암(LG-IR-NMIBC) 치료를 위한 ZUSDURI™ (미토마이신)의 2b상 OPTIMA II 시험 5년 연장 연구 결과를 발표했습니다.
연구 결과는 반응의 지속성이 뛰어남을 보여주었습니다: 3개월 시점에 완전 반응(CR)을 보인 41명의 환자 중 중앙 반응 지속 기간은 24.2개월이었습니다. 장기 추적 관찰에 들어간 17명 환자의 경우 중앙 반응 지속 기간은 42.1개월에 달했습니다. 특히 장기 추적 환자의 94%가 재발 환자였습니다.
가장 흔한 부작용은 배뇨통(41%), 빈뇨(21%), 혈뇨(16%)였습니다. ZUSDURI는 재발성 LG-IR-NMIBC 성인 환자를 위해 특별히 승인되었으며, 외래 환경에서 비수술적 치료 대안을 제공합니다.
UroGen Pharma (NASDAQ : URGN) a publié des résultats prometteurs à cinq ans de l'étude d'extension de phase 2b OPTIMA II pour ZUSDURI™ (mitomycine) dans le traitement du cancer de la vessie non muscle-invasif à faible grade et risque intermédiaire (LG-IR-NMIBC).
L'étude a démontré une durabilité significative de la réponse : parmi 41 patients ayant obtenu une réponse complète (RC) à trois mois, la durée médiane de la réponse était de 24,2 mois. Pour les 17 patients suivis à long terme, la durée médiane de la réponse a atteint 42,1 mois. Notamment, 94% des patients en suivi à long terme étaient des cas récurrents.
Les effets indésirables les plus fréquents comprenaient la dysurie (41%), la pollakiurie (21%) et l'hématurie (16%). ZUSDURI est spécifiquement indiqué pour les adultes atteints de LG-IR-NMIBC récurrent, offrant une alternative de traitement non chirurgicale en ambulatoire.
UroGen Pharma (NASDAQ: URGN) hat vielversprechende fünfjährige Verlängerungsstudienergebnisse ihrer Phase-2b-OPTIMA-II-Studie für ZUSDURI™ (Mitomycin) zur Behandlung von niedriggradigem, intermediärem Risiko, nicht muskelinvasivem Blasenkrebs (LG-IR-NMIBC) veröffentlicht.
Die Studie zeigte eine signifikante Dauerhaftigkeit der Ansprechrate: Von 41 Patienten, die nach drei Monaten eine komplette Remission (CR) erreichten, betrug die mediane Ansprechdauer 24,2 Monate. Bei den 17 Patienten, die in die Langzeitnachbeobachtung gingen, erreichte die mediane Ansprechdauer 42,1 Monate. Bemerkenswert ist, dass 94% der Langzeitnachbeobachtungspatienten Rezidivfälle waren.
Die häufigsten Nebenwirkungen waren Dysurie (41 %), Pollakisurie (21 %) und Hämaturie (16 %). ZUSDURI ist speziell für Erwachsene mit rezidivierendem LG-IR-NMIBC indiziert und bietet eine nicht-chirurgische Behandlungsalternative in der ambulanten Versorgung.
- Long-term durability demonstrated with 42.1 months median duration of response in follow-up patients
- 94% of long-term follow-up patients were recurrent cases, showing effectiveness in challenging cases
- Treatment can be administered in outpatient setting, avoiding repeated surgical interventions
- Provides non-surgical alternative for recurring bladder cancer patients
- Significant adverse events reported including dysuria in 41% of patients
- Study sample size was relatively small with only 17 patients in long-term follow-up
- Safety data not collected during long-term follow-up trial
Insights
ZUSDURI shows impressive 3.5-year durability in recurrent bladder cancer patients, offering a valuable non-surgical alternative.
The five-year extension data from UroGen's OPTIMA II trial represents a significant clinical advancement for patients with low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The extension study shows remarkable durability of response with ZUSDURI (mitomycin intravesical solution), with patients remaining event-free for a median of 24.2 months after achieving complete response.
Most notably, among the 17 patients who entered the long-term extension study, the median duration of response reached 42.1 months (approximately 3.5 years). This is particularly impressive considering that 94% of these patients had recurrent disease, which typically requires repeated surgical interventions that can significantly impact quality of life.
The non-surgical nature of ZUSDURI addresses a critical unmet need in bladder cancer management. Traditionally, patients with recurrent LG-IR-NMIBC face multiple transurethral resections of bladder tumors (TURBTs), which carry cumulative surgical risks and healthcare costs. ZUSDURI's administration in an outpatient setting with durable responses potentially reduces the burden of repeated procedures.
While safety data wasn't collected during the extension study, the original OPTIMA II data showed manageable adverse events, primarily localized urinary symptoms like dysuria (41%), pollakiuria (21%), and hematuria (16%)—generally expected with intravesical therapies and less severe than surgical complications.
This long-term data substantiates ZUSDURI as a valuable treatment option that could significantly change the management paradigm for recurrent LG-IR-NMIBC, potentially reducing surgical interventions while maintaining disease control for extended periods.
The publication of this 5-year extension data significantly strengthens UroGen's commercial positioning for ZUSDURI. The demonstrated 3.5-year median duration of response in predominantly recurrent patients (94% of the extension cohort) provides compelling evidence for ZUSDURI's value proposition in the treatment paradigm.
This durability data addresses a critical commercial hurdle for non-surgical bladder cancer treatments—proving long-term efficacy. With bladder cancer being the sixth most common cancer in the U.S. and having one of the highest recurrence rates of all cancers, the market opportunity for effective non-surgical options is substantial.
The outpatient administration profile combined with multi-year response durability creates a compelling economic case for both payers and healthcare systems. Each avoided surgical procedure (TURBT) represents significant cost savings, potentially accelerating payer coverage and physician adoption.
For UroGen, this data will likely enhance market penetration for ZUSDURI's approved indication in recurrent LG-IR-NMIBC. The publication in a peer-reviewed journal (Clinical Genitourinary Cancer) adds scientific credibility that can be leveraged in marketing materials and discussions with healthcare providers.
While the study size is modest (17 patients in long-term follow-up), the consistency of results and the extended follow-up period (median 50.4 months) provide meaningful evidence for ZUSDURI's durability claim. This should help UroGen in its ongoing efforts to establish ZUSDURI as a standard of care option for recurrent LG-IR-NMIBC, potentially expanding its market share in this indication.
- Findings published in the journal of Clinical Genitourinary Cancer show patients remained event-free for a median of two years after achieving complete response with ZUSDURI (mitomycin) intravesical solution.
- Among the 17 patients who achieved complete response and entered a long-term extension study, the median duration of response was 3.5 years by Kaplan-Meier estimate.
PRINCETON, N.J., July 21, 2025 (GLOBE NEWSWIRE) -- UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions for urothelial and specialty cancers, today 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. The publication, titled “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”, is now available online at https://doi.org/10.1016/j.clgc.2025.102392.
“Low-grade intermediate-risk bladder cancer is a chronic, recurring disease that often requires repeated surgical intervention,” said Neal D. Shore, MD, FACS Medical Director for the START Carolinas/Carolina Urologic Research Center and lead author of the study. “The long-term data from the extension study of OPTIMA II highlight ZUSDURI’s ability to deliver sustained responses in an outpatient setting, which may be especially valuable for recurrent patients and thus for physicians who prefer a different, non-surgical treatment option.”
Among the 41 patients who achieved CR at three months post-treatment with ZUSDURI in the OPTIMA II trial, 25 remained in CR at 12 months and 17 entered the long-term follow-up study. For the 41 patients achieving CR at three months, the median Kaplan-Meier estimate of duration of response (DOR) was 24.2 months (
“These results reflect our continued commitment to bringing forward innovative treatments that give patients and physicians more options,” said Mark Schoenberg, MD, Chief Medical Officer, UroGen. “For recurrent patients facing repeated surgeries, it offers a non-surgical approach that can empower patients and providers to choose a path that best fits individual needs and preferences.”
Safety data were not collected during the long-term follow-up trial. The most commonly reported treatment emergent adverse events (>
The OPTIMA II trial included both newly diagnosed and recurrent adult patients with LG-IR-NMIBC. Of the 17 patients that entered long-term follow up, 16 (
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 in an out-patient procedure by a trained healthcare professional using a urinary catheter to enable the treatment of tumors by non-surgical means. Visit ZUSDURI.com for more information.
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 trans-urethral resection of bladder tumor (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 OPTIMA II
OPTIMA II (OPTimized Instillation of Mitomycin for Bladder Cancer Treatment) was an open-label, single-arm, multi-center Phase 2b clinical trial of ZUSDURI to evaluate its safety and efficacy in patients with LG-IR-NMIBC.
Learn more about the Phase 2b OPTIMA II trial at www.clintrials.gov (NCT03558503).
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 ZUSDURI (mitomycin) for intravesical solution, our first product to treat recurrent LG-IR-NMIBC are both 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, @UroGenPharma.
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.
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 to patients; 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,” “may,” “plan,” “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; 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 key findings from UroGen's OPTIMA II trial 5-year extension study for ZUSDURI?
What is the success rate of ZUSDURI (URGN) in treating bladder cancer?
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How does ZUSDURI treatment differ from traditional bladder cancer treatments?
What type of bladder cancer is ZUSDURI approved to treat?
