UroGen Reports 77.8% Three-Month Complete Response Rate from Phase 3 UTOPIA Trial of UGN-103 and Receives FDA Agreement on NDA Submission Strategy in Recurrent LG-IR-NMIBC Based on UTOPIA Trial
UroGen (Nasdaq: URGN) reported a 77.8% three-month complete response (CR) rate (95% CI, 68.3%–85.5%) from the ongoing Phase 3 UTOPIA trial of UGN-103 in recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).
The result is consistent with a 79.6% three-month CR observed with ZUSDURI in ENVISION. The FDA agreed that CR and durability results from UTOPIA can support a New Drug Application (NDA) for UGN-103, and the company plans to prepare an NDA submission in 2026. UroGen also holds two U.S. patents covering UGN-103 that expire in December 2041.
UroGen (Nasdaq: URGN) ha riportato un tasso di risposta completa (CR) a tre mesi del 77,8% (IC del 95%, 68,3%–85,5%) dal trial di fase 3 in corso UTOPIA di UGN-103 nel cancro della vescica recidivante a basso grado, rischio intermedio non muscolo-invasivo (LG-IR-NMIBC).
Il risultato è in linea con un CR a tre mesi del 79,6% osservato con ZUSDURI in ENVISION. La FDA ha concordato che i risultati di CR e la loro durabilità provenienti da UTOPIA possono supportare una Nuova Domanda di Farmaco (NDA) per UGN-103, e l’azienda prevede di preparare una sottomissione NDA nel 2026. UroGen detiene anche due brevetti statunitensi che coprono UGN-103 e che scadranno nel dicembre 2041.
UroGen (Nasdaq: URGN) informó una tasa de respuesta completa (CR) a tres meses de 77,8% (IC del 95%, 68,3%–85,5%) proveniente del ensayo de fase 3 UTOPIA de UGN-103 en cáncer de vejiga no músculo-invasivo de bajo grado, de riesgo intermedio y recidivante (LG-IR-NMIBC).
El resultado es consistente con un CR a tres meses del 79,6% observado con ZUSDURI en ENVISION. La FDA acordó que los resultados de CR y durabilidad de UTOPIA pueden apoyar una Solicitud de Nuevo Fármaco (NDA) para UGN-103, y la compañía planea preparar una presentación de NDA en 2026. UroGen también posee dos patentes en EE. UU. que cubren UGN-103 y que vencen en diciembre 2041.
UroGen (나스닥: URGN)은 재발성 저등급 중간 위험 비근육침습성 방광암(LG-IR-NMIBC)에서 진행 중인 3상 UTOPIA 시험의 UGN-103으로부터 3개월 완전 반응(CR)률이 77.8%(95% CI, 68.3%–85.5%)를 보고했습니다.
이 결과는 ENVISION에서 ZUSDURI로 관찰된 3개월 CR 79.6%와 일치합니다. FDA는 UTOPIA의 CR 및 내구성 결과가 UGN-103에 대한 신약 승인(NDA)를 뒷받침할 수 있다고 동의했으며, 회사는 2026년에 NDA 제출을 준비할 계획입니다. UroGen은 또한 UGN-103을 커버하는 미국 특허 2건을 보유하고 있으며, 이 특허는 2041년 12월에 만료됩니다.
UroGen (Nasdaq : URGN) a annoncé un taux de réponse complète (CR) à trois mois de 77,8% (CI à 95% : 68,3%–85,5%) issus de l’essai de phase 3 UTOPIA sur UGN-103 dans le cancer de la vessie récurrent non-muscle-invasive à faible risque intermédiaire (LG-IR-NMIBC).
Le résultat est cohérent avec un CR à trois mois de 79,6% observé avec ZUSDURI dans ENVISION. La FDA a accepté que les résultats CR et leur durabilité issus d’UTOPIA puissent soutenir une demande de médicament nouvelle (NDA) pour UGN-103, et l’entreprise prévoit de préparer une soumission NDA en 2026. UroGen détient également deux brevets américains couvrant UGN-103 et qui expireront en décembre 2041.
UroGen (Nasdaq: URGN) meldete eine dreimonatige vollständige Ansprechrate (CR) von 77,8% (95%-KI 68,3%–85,5%) aus der laufenden Phase-3-UTOPIA-Studie von UGN-103 bei rezidivierendem LG-IR-NMIBC (niedriges bis mittleres Risiko, nicht muskelinvasiv).
Das Ergebnis stimmt mit einer dreimonatigen CR von 79,6% überein, die mit ZUSDURI in ENVISION beobachtet wurde. Die FDA hat zugestimmt, dass CR- und Haltbarkeitsdaten aus UTOPIA eine NDA für UGN-103 unterstützen können, und das Unternehmen plant, im Jahr 2026 eine NDA-Einreichung vorzubereiten. UroGen hält außerdem zwei US-Patente, die UGN-103 abdecken und am Dezember 2041 ablaufen.
UroGen (ناسداك: URGN) أبلغت عن معدل استجابة كاملة (CR) عند ثلاثة أشهر بنسبة 77.8% (فاصل ثقة 95%: 68.3%–85.5%) من تجربة المرحلة 3 المستمرة UTOPIA لـ UGN-103 في سرطان المثانة المتكرر من الدرجة المنخفضة-المخاطر المتوسطة غير الغازية للعضلة (LG-IR-NMIBC).
النتيجة تتماشى مع CR عند ثلاثة أشهر بنسبة 79.6% التي لوحظت مع ZUSDURI في ENVISION. وافقت إدارة الغذاء والدواء الأمريكية (FDA) على أن نتائج CR والمتانة من UTOPIA يمكن أن تدعم طلب دواء جديد (NDA) لـ UGN-103، وتخطط الشركة لإعداد تقديم NDA في 2026. كما تمتلك UroGen اثنين من براءات الاختراع الأمريكية التي تغطي UGN-103 وتنتهي صلاحيتها في ديسمبر 2041.
- Three-month CR 77.8% (95% CI 68.3%–85.5%)
- FDA agreed UTOPIA results can support an NDA
- Company plans NDA submission in 2026
- Two U.S. patents covering UGN-103 expire Dec 2041
- Three-month CR < ZUSDURI’s 79.6% observed in ENVISION
- UTOPIA data described as preliminary and trial ongoing
Insights
Phase 3 shows a high early complete response and FDA agrees UTOPIA can support an NDA.
The UTOPIA trial reports a
The clinical mechanism is straightforward: measured CR at three months serves as an efficacy endpoint for recurrent LG-IR-NMIBC and the trial produced a point estimate close to a prior pivotal result. Key dependencies include the durability data and full trial dataset, which the company cites as necessary to complete before filing. Watch for the durability outcomes and the complete UTOPIA dataset ahead of the planned NDA submission in
FDA acceptance that UTOPIA can support an NDA materially de-risks regulatory pathway pending full data.
The FDA’s agreement that CR and durability results from UTOPIA can support an NDA provides procedural alignment on submission content for UGN-103. The release also highlights two U.S. patents covering UGN-103 that expire in
Risks and near-term monitors are explicit: completion of the UTOPIA trial, the durability readout, and the formal NDA filing process expected in
PRINCETON, N.J., Nov. 06, 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 robust preliminary results from its ongoing Phase 3 UTOPIA trial, demonstrating a
“The robust
UGN-103 is designed to offer key improvements over ZUSDURI (mitomycin) for intravesical solution, the first and only FDA-approved treatment for adults with recurrent LG-IR-NMIBC. These include a shorter manufacturing process and a simplified reconstitution procedure, while maintaining UroGen’s established approach that enables prolonged drug exposure within the bladder. UroGen has two U.S. patents with claims relating to the combination of the Company’s RTGel® technology combined with medac’s licensed proprietary lyophilized mitomycin formulation. These patents cover UGN-103 as well as the use of UGN-103 for the treatment of LG-IR-NMIBC and expire in December 2041.
About UTOPIA
The UTOPIA trial is a single-arm, multicenter study evaluating the efficacy and safety of UGN-103 in 99 patients across global sites. Enrolled patients received 75 mg of UGN-103 via intravesical instillation once weekly for six weeks. The primary endpoint is complete response rate at three months, with responders entering a follow-up phase of up to 12 months to assess durability of response. For more information on the UTOPIA study, please visit https://clinicaltrials.gov/study/NCT06331299.
About UGN-103
In January 2024, UroGen entered into a licensing and supply agreement with medac to develop UGN-103 for LG-IR-NMIBC. UGN-103 is an innovative mitomycin formulation that aims to streamline manufacturing and reconstitution processes while providing intellectual property coverage until December 2041. It utilizes UroGen’s RTGel® technology for prolonged mitomycin exposure.
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 transurethral 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 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 is approved to treat low-grade upper tract urothelial cancer, and our second product, ZUSDURI (mitomycin) for intravesical solution, is approved for adult patients with recurrent LG-IR-NMIBC. Both products 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, @UroGenPharma.
About medac CDMO
The belief that health is humanity’s most valuable resource drives medac group. Since 1970, the mission of medac has been to improve patients’ quality of life worldwide by making the best medical treatments available. Since 2000, medac has been dedicated to improving patient outcomes globally by supporting pharmaceutical companies in bringing the best medical treatments to market. As a trusted Contract Development and Manufacturing Organization (CDMO), headquartered in Germany, medac CDMO is specialised in providing customised, high-quality services to customers and worldwide markets.
With a team of over 2,000 highly skilled professionals, medac CDMO offers comprehensive solutions tailored to the needs of clients worldwide. The cutting-edge facilities of medac group in Germany and the Czech Republic are equipped with the latest technologies to ensure precision, efficiency and compliance with the most stringent industry standards. From early-stage development to large-scale commercial production, medac CDMO is committed to foresight, progress, reliability and creative thinking which makes them a solution ahead.
The deep expertise, commitment to quality and flexible manufacturing capabilities enable medac CDMO to serve as a trusted partner for pharmaceutical and biotech companies looking to scale their operations and bring life-changing treatments to patients around the globe.
For more information, please visit www.medac-cdmo.com.
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.
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 to patients and opportunities for UroGen’s product candidates, including the potential of UGN-103 to deliver meaningful benefits to patients, as a key growth driver for UroGen, to streamline manufacturing and reconstitution processes and to offer intellectual protection until December 2041; the expected duration of intellectual property protection for UGN-103; UroGen’s ongoing UTOPIA clinical trial of UGN-103; the planned NDA submission for UGN-103 and the timing thereof; the belief that UGN-103, if approved, will offer several improvements over ZUSDURI; the FDA’s agreement that the UTOPIA trial can support the submission of an NDA for UGN-103 representing a significant regulatory milestone, a strong validation of UroGen’s clinical strategy and giving UroGen a clear path toward potential approval of UGN-103; 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,” “expect,” “may,” “plan,” “potential,” 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: preliminary clinical results may not be indicative of results that may be observed in the future; potential safety and other complications related to UroGen’s products and product candidates; risks related to our and our licensors’ ability to protect our respective patents and other intellectual property, including the fact that UroGen’s or our licensors’ pending patent applications may not be successful, and in such event, the duration of intellectual property protection would be more limited; 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 UroGen’s products and product candidates may not perform as expected; the data from the UTOPIA trial may not be sufficient to support approval of UGN-103; 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 June 30, 2025, filed with the SEC on August 7, 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 was UroGen's reported three-month complete response rate for UGN-103 in the UTOPIA trial (URGN)?
Did the FDA accept UTOPIA trial results to support an NDA for UGN-103 (URGN)?
When does UroGen plan to submit an NDA for UGN-103 (URGN)?
How does UGN-103's three-month CR compare to ZUSDURI in ENVISION for URGN investors?
What patent protection does UroGen have for UGN-103 (URGN)?
Are UTOPIA results final and available for regulatory filing for URGN shareholders?
