On April 27, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported new data from the OPTIMA II Phase 2b study of UGN‑102 (mitomycin) for intravesical solution demonstrate clinically meaningful two-year duration of response (24.2 months) by Kaplan-Meier analysis (Press release, UroGen Pharma, APR 27, 2025, View Source [SID1234652196]). UGN-102 is UroGen’s sustained-release formulation of mitomycin being developed for the treatment of recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).

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"The median duration of response of two years highlights UGN-102’s durability, even in patients with recurrent disease and multiple prior TURBT procedures," said Neal Shore, MD, Medical Director, Carolina Urologic Research Center, Myrtle Beach, South Carolina. "These long-term data provide encouraging evidence of UGN-102’s sustained impact."

The majority of patients included in OPTIMA II had recurrent disease at baseline (77.8%), with multiple prior transurethral resection of bladder tumor (TURBT) procedures. Among the 41 patients achieving a complete response (CR) at three months, 25 remained in CR at 12 months, and 17 of these patients entered long-term follow-up. The median Kaplan–Meier estimate of duration of response for the 41 patients that achieved CR was 24.2 months (95% CI 9.72, 47.18), with a median follow-up time of 33.6 months (95% CI 10.78, 42.94). Twenty patients (48.8%) experienced recurrence of low-grade disease. One patient progressed to high-grade disease and one patient died due to a cardiac disorder. Five patients remained disease-free at the time of the four-year data analysis.

"As the burden of LG-IR-NMIBC persists, the need for long-lasting treatment options becomes increasingly urgent," said Mark Schoenberg, Chief Medical Officer, UroGen. "The growing body of evidence supporting UGN-102 underscores its potential to address this unmet need. These results emphasize UGN-102’s potential to deliver meaningful and sustained responses, offering hope to patients who have long struggled with recurrence and limited treatment options."

UroGen completed the submission of the UGN-102 rolling new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for UGN-102 in August 2024, ahead of schedule. The FDA accepted the NDA for UGN-102 with a Prescription Drug User Free Act (PDUFA) goal date of June 13, 2025.

The most common treatment-emergent adverse events (TEAEs) in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. The TEAEs were typically mild-to-moderate in severity and either resolved or were 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 recurrent LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional. UroGen completed the submission of the rolling NDA for UGN-102 in August 2024, 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)

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 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.

About OPTIMA II

OPTIMA II (OPTimized Instillation of Mitomycin for Bladder Cancer Treatment) was an open-label, single-arm, multi-center Phase 2b clinical trial of investigational drug UGN-102 to evaluate its safety and efficacy in patients with LG-IR-NMIBC.

Learn more about the Phase 2b OPTIMA II trial at www.clinicaltrials.gov (NCT03558503).