UGN-102 Yields Positive Long-Term Results in Phase 3 Trial, Could Become First FDA-Approved LG-IR-NMIBC Treatment

UroGen Pharma Ltd., a biotech firm focused on pioneering treatments for urothelial and specialty cancers, has showcased promising results from its Phase 3 ENVISION trial at the Society of Urologic Oncology Annual Meeting in Dallas, Texas. These findings, published in the Journal of Urology, highlight the impressive effectiveness and safety of UGN-102, an investigational mitomycin-based intravesical solution, in treating patients with recurrent low-grade intermediate-risk non-muscle-invasive bladder cancer (LG-IR-NMIBC).

Max Kates, a key investigator in the ENVISION study, expressed optimism about the trial's outcomes, particularly the lasting response rates, which suggest UGN-102's potential as a viable treatment. The ENVISION trial demonstrated an 82.3% duration of response (DOR) at 12 months for patients who achieved a complete response (CR) at 3 months post-treatment. This high durability of response persisted at 15 and 18 months, with both periods showing an 80.9% DOR. The trial’s primary endpoint—a 79.6% CR rate three months after initial treatment with UGN-102—further underscores its efficacy.

The safety profile of UGN-102 in the ENVISION trial was consistent with previous studies, indicating its potential as a new treatment for LG-IR-NMIBC. Common treatment-emergent adverse events (TEAEs) included dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. These side effects were generally mild to moderate and resolved over time.

Mark Schoenberg, Chief Medical Officer at UroGen, highlighted the significance of these findings. He noted the promising potential of UGN-102 to provide a much-needed alternative to current treatments, especially for elderly patients who often undergo multiple surgeries under general anesthesia, affecting their health and quality of life. The ongoing development of UGN-102 aims to extend recurrence-free periods and improve patient outcomes.

UGN-102 represents a novel approach in the treatment of LG-IR-NMIBC, leveraging UroGen's proprietary RTGel technology. This hydrogel-based formulation allows for sustained release of mitomycin, enhancing the exposure of bladder tissue to the drug. Administered via a standard urinary catheter in an outpatient setting, UGN-102 aims to eliminate tumors without the need for invasive surgery. UroGen has completed the New Drug Application (NDA) for UGN-102, with the FDA assigning a Prescription Drug User Fee Act (PDUFA) goal date of June 13, 2025.

Bladder cancer remains a significant health issue in the United States, being the second most common urologic cancer in men. LG-IR-NMIBC accounts for around 22,000 new cases annually, with about 60,000 recurrences each year. The current standard of care, trans-urethral resection of bladder tumor (TURBT), often leads to multiple recurrences, necessitating repeated procedures. The high recurrence rate and the need for better treatment options emphasizes the importance of trials like ENVISION.

The Phase 3 ENVISION trial involved approximately 240 patients across 56 sites. Participants received six weekly intravesical instillations of UGN-102. The study focused on the CR rate at three months post-treatment and the durability of response over time for those who achieved CR.

UroGen Pharma Ltd. continues to pioneer in the field of urothelial and specialty cancer treatment, with innovative solutions like UGN-102. Their RTGel technology holds promise in enhancing the delivery and effectiveness of existing drugs, potentially providing better outcomes for patients with challenging conditions such as LG-IR-NMIBC. Based in Princeton, NJ, with operations in Israel, UroGen remains committed to offering improved therapeutic options for patients in need.