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
UroGen Pharma (Nasdaq: URGN) announced promising Phase 3 ENVISION trial results for UGN-102, a mitomycin-based intravesical solution for treating recurrent low-grade intermediate-risk non-muscle-invasive bladder cancer (LG-IR-NMIBC). The trial demonstrated an 82.3% duration of response at 12 months in patients who achieved complete response at 3 months, with durability extending to 80.9% at 15 and 18 months.
The study achieved its primary endpoint with a 79.6% complete response rate at 3 months post-treatment. The safety profile remained consistent with previous trials, with common side effects including dysuria, hematuria, and urinary tract infection. The FDA has set a PDUFA goal date of June 13, 2025, for UGN-102, which could potentially become the first FDA-approved treatment for LG-IR-NMIBC.
UroGen Pharma (Nasdaq: URGN) ha annunciato risultati promettenti della fase 3 dello studio ENVISION per UGN-102, una soluzione intravesicale a base di mitomicina per il trattamento del cancro alla vescica non muscolo-invasivo a basso grado di rischio intermedio ricorrente (LG-IR-NMIBC). Lo studio ha dimostrato un 82,3% di durata della risposta a 12 mesi nei pazienti che hanno raggiunto una risposta completa a 3 mesi, con una durata estesa all'80,9% a 15 e 18 mesi.
Lo studio ha raggiunto il suo obiettivo primario con una tassa di risposta completa del 79,6% a 3 mesi dopo il trattamento. Il profilo di sicurezza è rimasto costante rispetto agli studi precedenti, con effetti collaterali comuni tra cui disuria, ematuria e infezione del tratto urinario. La FDA ha fissato una data obiettivo PDUFA del 13 giugno 2025 per UGN-102, che potrebbe diventare il primo trattamento approvato dalla FDA per LG-IR-NMIBC.
UroGen Pharma (Nasdaq: URGN) anunció resultados prometedores del ensayo de fase 3 ENVISION para UGN-102, una solución intravesical a base de mitomicina para tratar el cáncer de vejiga no invasivo de bajo grado y riesgo intermedio recurrente (LG-IR-NMIBC). El ensayo demostró una duración de respuesta del 82.3% a 12 meses en pacientes que lograron respuesta completa a los 3 meses, con una durabilidad que se extiende al 80.9% a los 15 y 18 meses.
El estudio alcanzó su objetivo primario con una tasa de respuesta completa del 79.6% a los 3 meses tras el tratamiento. El perfil de seguridad se mantuvo consistente con ensayos anteriores, con efectos secundarios comunes que incluyen disuria, hematuria e infección del tracto urinario. La FDA ha establecido una fecha objetivo PDUFA del 13 de junio de 2025 para UGN-102, que podría convertirse en el primer tratamiento aprobado por la FDA para LG-IR-NMIBC.
UroGen Pharma (Nasdaq: URGN)는 재발성 저급 중간 위험 비근육 침윤성 방광암(LG-IR-NMIBC) 치료를 위한 미토마이신 기반 방광 내 용액인 UGN-102에 대한 3상 ENVISION 임상시험의 유망한 결과를 발표했습니다. 이 연구는 3개월 만에 완전 반응을 기록한 환자에서 12개월 시점에서 82.3%의 반응 지속 시간을 보였으며, 15개월과 18개월 시점에서는 80.9%로 연장되었습니다.
연구는 치료 후 3개월에 79.6%의 완전 반응률로 주요 목표를 달성했습니다. 안전성 프로필은 이전 연구와 일관되었으며, 일반적인 부작용으로는 배뇨통, 혈뇨 및 요로 감염이 포함되었습니다. FDA는 UGN-102에 대해 2025년 6월 13일로 PDUFA 목표 날짜를 설정했으며, 이는 LG-IR-NMIBC에 대한 첫 번째 FDA 승인 치료제가 될 가능성이 있습니다.
UroGen Pharma (Nasdaq: URGN) a annoncé des résultats prometteurs de l'essai de phase 3 ENVISION pour UGN-102, une solution intravésicale à base de mitomycine pour traiter le cancer de la vessie non invasif, de bas grade et à risque intermédiaire récurrent (LG-IR-NMIBC). L'essai a démontré une durée de réponse de 82,3 % à 12 mois chez les patients ayant obtenu une réponse complète à 3 mois, avec une durabilité s'étendant à 80,9 % à 15 et 18 mois.
L'étude a atteint son objectif principal avec un taux de réponse complète de 79,6 % à 3 mois après le traitement. Le profil de sécurité est resté cohérent avec les essais précédents, avec des effets secondaires courants tels que la dysurie, l'hématurie et les infections des voies urinaires. La FDA a fixé une date cible PDUFA au 13 juin 2025 pour UGN-102, qui pourrait potentiellement devenir le premier traitement approuvé par la FDA pour LG-IR-NMIBC.
UroGen Pharma (Nasdaq: URGN) hat vielversprechende Ergebnisse der Phase-3-Studie ENVISION für UGN-102, eine mit Mitomycin basierende intravesikale Lösung zur Behandlung von rezidivierendem, niedriggradigem, intermediärem Risiko, nicht muskel-invasivem Blasenkrebs (LG-IR-NMIBC) angekündigt. Die Studie zeigte eine dauerhafte Antwort von 82,3% nach 12 Monaten bei Patienten, die nach 3 Monaten ein vollständiges Ansprechen erzielten, mit einer Haltbarkeit von 80,9% nach 15 und 18 Monaten.
Die Studie erreichte ihr primäres Ziel mit einer kompletten Ansprechrate von 79,6% 3 Monate nach der Behandlung. Das Sicherheitsprofil blieb konsistent mit früheren Studien, wobei häufige Nebenwirkungen Dysurie, Hämaturie und Harnwegsinfektionen umfassen. Die FDA hat ein PDUFA-Zieldatum vom 13. Juni 2025 für UGN-102 festgelegt, welches potenziell die erste von der FDA genehmigte Behandlung für LG-IR-NMIBC werden könnte.
- High complete response rate of 79.6% at 3 months post-treatment
- Strong durability of response at 82.3% after 12 months
- Maintained durability at 80.9% through 15 and 18 months
- FDA PDUFA date set for June 13, 2025
- Potential to be first FDA-approved treatment for LG-IR-NMIBC
- Treatment caused various adverse events including dysuria, hematuria, and urinary tract infection
Insights
The Phase 3 ENVISION trial results for UGN-102 represent a significant breakthrough in bladder cancer treatment. The
The durability of response extending to 18 months with
This development is especially significant as UGN-102 could become the first FDA-approved non-surgical treatment for LG-IR-NMIBC, addressing a major unmet medical need in an elderly patient population currently subjected to repeated surgeries.
From an investment perspective, UGN-102's strong clinical data significantly de-risks UroGen's regulatory pathway. With a market cap of
The potential first-to-market advantage in this indication could translate to significant market share capture. The durable response rates suggest potential for recurring revenue streams through maintenance therapy. Additionally, the non-surgical nature of UGN-102 could drive favorable reimbursement decisions, as it may reduce overall healthcare costs compared to current surgical standards.
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UGN-102 demonstrated
82.3% duration of response (DOR) at 12 months in patients who achieved complete response at 3 months -
79.6% complete response rate at 3 months in patients treated with UGN-102 - Safety profile consistent with prior clinical trials of UGN-102
"Finding options for patients with recurrent low-grade bladder cancer continues to be a major unmet need,” says Max Kates, R. Christian B. Evensen Professor and an Associate Professor of Urology and Oncology, and Director, Division of Urologic Oncology at the Brady Urological Institute at Johns Hopkins and ENVISION study investigator. “We are excited by the overall results of the ENVISION trial, especially the durability of response data, that support the potential of UGN-102 as a viable treatment option for these patients."
In the ENVISION study, UGN-102 treatment showed an impressive
The side effect profile of UGN-102 was consistent with previous clinical trials, further supporting its potential as a new treatment option for patients with LG-IR-NMIBC.
"We are excited by the progress made in advancing UGN-102 as a potential treatment for LG-IR-NMIBC and securing a PDUFA goal date of June 13, 2025, from the FDA,” said Mark Schoenberg, M.D., Chief Medical Officer, UroGen. “The strong durability of response observed in the ENVISION study highlights UGN-102’s promising potential for patients. Given that many LG-IR-NMIBC patients are elderly and endure multiple surgeries under general anesthesia for their condition that impact their health and quality of life, there is an urgent need for alternative treatment options that can prolong recurrence-free periods and enhance patient outcomes."
The most common treatment-emergent adverse events (TEAEs) in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. TEAEs were typically mild-to-moderate in severity and resolved or resolving. The ENVISION trial demonstrated a similar safety profile to that observed in other studies of UGN-102.
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 treat tumors by enabling longer exposure of bladder tissue to mitomycin. 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 for UGN-102 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 therapy for 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 potential for UGN-102 as the first FDA-approved treatment for LG-IR-NMIBC; the long-term results in the ENVISION trial supporting the potential of UGN-102 as a viable treatment option for LG-IR NMIBC; 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, Communications
cindy.romano@urogen.com
609-460-3583 ext. 1083
Source: UroGen Pharma Ltd.
FAQ
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