UroGen Pharma Ltd. (Nasdaq: URGN), a biotechnology company focused on developing innovative treatments for urothelial and specialty cancers, has announced the presentation of the Phase 3 ENVISION trial’s efficacy and safety results at the Society of Urologic Oncology (SUO) annual meeting in Dallas, TX. These results, published online in the Journal of Urology in October, show that the investigational therapy UGN-102, a mitomycin-based intravesical solution, led to a high and clinically significant complete response rate, with durable results in patients with recurrent low-grade intermediate-risk non-muscle-invasive bladder cancer (LG-IR-NMIBC).
The treatment of recurrent low-grade bladder cancer remains a significant unmet need, according to Dr. Max Kates, a leading urologic oncology expert at Johns Hopkins. Speaking as an investigator for the ENVISION study, Dr. Kates expressed excitement over the trial’s promising results, particularly the durability of response data, which highlights UGN-102’s potential as a viable treatment for patients with low-grade, intermediate-risk non-muscle-invasive bladder cancer (LG-IR-NMIBC).
The ENVISION study showed that UGN-102, a mitomycin-based intravesical therapy, resulted in an impressive 82.3% duration of response (DOR) at 12 months for patients who achieved a complete response (CR) three months after their first treatment. The DOR at 15 and 18 months remained robust, at 80.9%, according to Kaplan-Meier estimates. These findings build on the trial’s positive primary endpoint, a 79.6% CR rate three months after initial treatment with UGN-102.
The safety profile of UGN-102 was consistent with previous trials, supporting its potential as a new treatment option for LG-IR-NMIBC. “We are thrilled to advance UGN-102 as a potential treatment for LG-IR-NMIBC, with a PDUFA goal date of June 13, 2025,” said Dr. Mark Schoenberg, UroGen’s Chief Medical Officer. He emphasized the need for alternative treatment options for this patient population, many of whom are elderly and face repeated surgeries that affect their quality of life.
The most common treatment-emergent adverse events (TEAEs) in the ENVISION trial included dysuria, hematuria, urinary tract infections, pollakiuria, fatigue, and urinary retention. These TEAEs were typically mild-to-moderate and resolved or were resolving by the end of the trial. UGN-102 demonstrated a similar safety profile to that observed in other studies.
UGN-102 is a mitomycin-based intravesical solution designed for patients with LG-IR-NMIBC. Utilizing UroGen’s RTGel® technology, a sustained-release, hydrogel-based formulation, UGN-102 is delivered via urinary catheter to provide prolonged exposure of bladder tissue to mitomycin. UroGen submitted the New Drug Application (NDA) for UGN-102 ahead of schedule, and the FDA has set a PDUFA goal date of June 13, 2025.
Low-grade intermediate-risk non-muscle-invasive bladder cancer (LG-IR-NMIBC) is common in older populations, with the median age of diagnosis at 73. Each year, about 22,000 people are diagnosed with bladder cancer, with many patients experiencing recurrences that require repeated surgeries, such as transurethral resection of bladder tumors (TURBT). LG-IR-NMIBC patients are particularly prone to recurrences and multiple treatments.
The Phase 3 ENVISION trial is a multinational study evaluating UGN-102’s efficacy and safety in patients with LG-IR-NMIBC. Approximately 240 patients were enrolled across 56 sites, receiving six once-weekly intravesical treatments of UGN-102. The study’s primary endpoint focused on the CR rate at three months, with key secondary endpoints assessing durability in patients who achieved a CR.
