UroGen Pharma (URGN) Study Update summary

Study background and objectives

• ENVISION is a pivotal phase 3 single-arm study evaluating UGN-102 for low-grade, intermediate-risk non-muscle invasive bladder cancer (NMIBC).

• The study focused on patients with recurrent disease within one year, a population with high recurrence rates.

• UGN-102 is a novel, non-surgical, intravesical chemoablation therapy using RTGel technology for sustained drug release.

• The primary endpoint was complete response rate at 3 months; key secondary endpoint was duration of response.

• Over 80,000 patients in the U.S. are affected annually, with limited non-surgical treatment options.

Key efficacy and safety results

• ENVISION showed a 79.6% complete response rate at 3 months among 240 patients.

• Estimated 12-month duration of response for complete responders was 82.3%, the highest seen in this population.

• Median duration of response predicted at 40 months for complete responders using Weibull model.

• Less than 3% of patients progressed to high-grade disease; severe treatment-related adverse events occurred in under 1%.

• Adverse events were mostly mild to moderate, primarily lower urinary tract symptoms, with two severe events (urethral stenosis, urinary retention) resolved.

Patient and physician perspectives

• Patient interviews revealed UGN-102 had less impact on daily activities, work, and sexual function compared to TURBT.

• Patients reported shorter recovery, less bleeding, and fewer catheter issues with UGN-102.

• Most patients would recommend UGN-102, citing it as less invasive, painful, and time-consuming.

• Physicians highlighted the paradigm shift, noting UGN-102 offers a durable, non-surgical alternative with a favorable safety profile.

• Panelists agreed UGN-102 could be offered as first-line therapy, especially for patients with comorbidities or limited surgical access.