UroGen Highlights 'Unprecedented' Data for Gel-Based Bladder Cancer Therapy

UroGen Pharma has unveiled promising 12-month data from its Phase III ENVISION trial, highlighting the efficacy of its innovative gel-based treatment, UGN-102, for non-muscle invasive bladder cancer (NMIBC). This significant development led to a surge in the company's shares, jumping over 41% on Thursday.

UGN-102 is a sustained-release, hydrogel-based formulation designed using UroGen's proprietary RTGel technology. This formulation allows bladder tissue to be in prolonged contact with the chemotherapy drug mitomycin. In the previous year, UroGen had announced that UGN-102 met its primary endpoint for patients with low-grade, intermediate-risk NMIBC, achieving a complete response (CR) rate of 79.6% at three months post the initial intravesical instillation. The ENVISION trial enrolled approximately 240 participants.

The latest results revealed that 82.3% of patients who achieved a CR at three months maintained their response for at least a year, according to Kaplan-Meier estimates. Furthermore, the duration of response estimates stood at 80.9% at both the 15 and 18-month marks.

Safety data from the ENVISION trial indicated that UGN-102 was generally well-tolerated, with most side effects being mild to moderate. The most common treatment-emergent adverse events included dysuria, hematuria, urinary tract infection, frequent urination, fatigue, and urinary retention.

Mark Schoenberg, the Chief Medical Officer, pointed out the "highly recurrent nature" of low-grade, intermediate-risk NMIBC, which often necessitates multiple surgeries for patients throughout their lives. He suggested that UGN-102 could offer a less invasive alternative. Liz Barrett, the CEO, estimated that around 82,000 patients in the US are affected by this recurrent form of cancer annually.

Earlier this year, UroGen began a rolling submission to the FDA, which they aim to complete by the third quarter. A potential FDA decision could come as early as the first quarter of 2025. If approved, UGN-102 might become the first non-surgical treatment option for low-grade, intermediate-risk NMIBC.

UroGen’s only product currently available on the market is Jelmyto, another hydrogel-based formulation of mitomycin. Approved by US regulators in 2020, Jelmyto is used to treat low-grade upper tract urothelial cancer. Sales of Jelmyto saw a 28% increase last year, reaching $82.7 million.

This new development with UGN-102 not only underscores UroGen’s commitment to pioneering advanced treatments for bladder cancer but also holds the potential to significantly improve the quality of life for countless patients by reducing the need for invasive surgical procedures.