UroGen Pharma's investigational therapy, UGN-102, a mitomycin-based intravesical solution, has demonstrated promising long-term results in the Phase 3 ENVISION trial for patients with recurrent low-grade intermediate-risk non-muscle-invasive bladder cancer (LG-IR-NMIBC). The data, presented at the Society of Urologic Oncology (SUO) annual meeting, reveal a high and durable complete response rate, potentially offering a new treatment avenue for this patient population. The FDA has accepted the NDA for UGN-102 and assigned a PDUFA goal date of June 13, 2025.
High Duration of Response
The ENVISION study showcased an 82.3% duration of response (DOR) at 12 months (95% CI: 75.9%, 87.1%) in patients who achieved a complete response (CR) at 3 months following the initial UGN-102 treatment, according to Kaplan-Meier estimates. Furthermore, the DOR at 15 months and 18 months remained robust at 80.9% (95% CI: 73.9%, 86.2%). These results build upon the trial's primary endpoint, which demonstrated a 79.6% CR rate (95% CI: 73.9%, 84.5%) at 3 months post-treatment.
Consistent Safety Profile
The side effect profile of UGN-102 in the ENVISION trial was consistent with previous clinical trials. The most common treatment-emergent adverse events (TEAEs) included dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. These TEAEs were generally mild to moderate in severity and resolved without significant intervention.
Addressing Unmet Needs in Bladder Cancer
LG-IR-NMIBC presents a significant challenge, with approximately 22,000 new diagnoses and 60,000 recurrences annually in the U.S. Standard treatment involves trans-urethral resection of bladder tumor (TURBT), but recurrence rates are high, with up to 70% of patients experiencing at least one recurrence. Max Kates, R. Christian B. Evensen Professor at Johns Hopkins, highlighted the unmet need for better options for patients with recurrent low-grade bladder cancer, expressing excitement about the durability of response data from the ENVISION trial.
UGN-102: A Potential Breakthrough
UGN-102 (mitomycin) for intravesical solution utilizes UroGen's proprietary RTGel technology, a sustained-release, hydrogel-based formulation designed to prolong the exposure of bladder tissue to mitomycin. This innovative approach aims to improve treatment efficacy and reduce the need for repeated surgeries. Mark Schoenberg, M.D., Chief Medical Officer at UroGen, emphasized the urgent need for alternative treatment options that can prolong recurrence-free periods and enhance patient outcomes, especially given that many LG-IR-NMIBC patients are elderly and undergo multiple surgeries.
