UroGen Reports 82.3% 12-Month Response in UGN-102 LG-IR-NMIBC Trial

UroGen Pharma Ltd., a biotechnology company focused on developing treatments for urothelial and specialty cancers, has announced promising results from its Phase 3 ENVISION trial. The trial evaluated the effectiveness of UGN-102 (mitomycin) for intravesical solution in patients with low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The 12-month duration of response (DOR) data, estimated by the Kaplan-Meier method, showed an 82.3% DOR (95% CI, 75.9%, 87.1%) among patients who achieved a complete response (CR) at three months.

The ENVISION trial had previously achieved its primary goal, demonstrating a 79.6% CR rate at three months post-instillation of UGN-102. This latest data further substantiates the potential of UGN-102 as a non-surgical treatment option for LG-IR-NMIBC. The study also revealed that the DOR Kaplan-Meier estimates at 15 and 18 months were both 80.9% (95% CI, 73.9%, 86.2%).

Liz Barrett, President and CEO of UroGen, highlighted the significance of these findings, emphasizing that UGN-102 may become the first FDA-approved non-surgical treatment for this recurrent disease, potentially benefiting around 82,000 U.S. patients annually.

The ENVISION trial's common treatment-emergent adverse events (TEAEs) included dysuria, hematuria, urinary tract infections, pollakiuria, fatigue, and urinary retention, most of which were mild to moderate. The safety profile was consistent with previous trials of UGN-102.

Dr. Sandip Prasad, Director of Genitourinary Surgical Oncology at Morristown Medical Center, expressed optimism about UGN-102’s potential to offer an alternative to repeated surgeries, which can adversely affect patients' quality of life and physical health. Dr. Mark Schoenberg, UroGen's Chief Medical Officer, also noted the potential for this treatment to reduce the frequency of surgeries for patients with LG-IR-NMIBC.

In early 2024, UroGen began a rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for UGN-102. The company plans to complete this submission by the third quarter of 2024, with an FDA decision anticipated as early as the first quarter of 2025.

UGN-102 is a novel drug formulation utilizing UroGen’s RTGel® technology, which allows for prolonged exposure of bladder tissue to mitomycin, potentially treating tumors non-surgically. The drug is administered via a standard urinary catheter in an outpatient setting by trained healthcare professionals.

In the U.S., bladder cancer is the second most common urologic cancer among men, with LG-IR-NMIBC affecting around 22,000 newly diagnosed patients annually and approximately 60,000 recurrences each year. The current standard of care involves Transurethral Resection of Bladder Tumor (TURBT) procedures. However, up to 70% of NMIBC patients experience recurrences, necessitating repeated surgeries.

The Phase 3 ENVISION trial involved about 240 patients across 56 sites, with participants receiving six weekly intravesical instillations of UGN-102. The trial's primary endpoint was the CR rate at three months, and the key secondary endpoint was the durability of response over time in those who achieved CR at three months.

UroGen Pharma Ltd., headquartered in Princeton, NJ with operations in Israel, is committed to developing innovative treatments for urothelial and specialty cancers. The company’s RTGel technology aims to enhance the therapeutic profiles of existing drugs by enabling longer exposure of urinary tract tissues to medications, potentially improving the efficacy of local therapies.