Low Grade Non Muscle Invasive Bladder Cancer

Patients treated with the current standard of care have up to an approximately 60% rate of recurrence of NMIBC within one year, and the rate of progression of NMIBC to MIBC is between 20% and 30%. As a consequence, NMIBC patients often undergo multiple repeat TURBT procedures and adjuvant chemotherapy and immunotherapy treatments.  The standard of care for treating NMIBC patients is TURBT followed by adjuvant chemotherapy or immunotherapy treatment. TURBT is a surgical operation for tumor removal conducted under general anesthesia in a hospital setting and often requires at least an overnight stay. Moreover, TURBT’s success is tied to the physician’s ability to overcome challenges in properly identifying, reaching and resecting all tumors. No drugs have been approved by the FDA as first-line treatment for NMIBC and only three drugs have been approved by the FDA for NMIBC, all used as adjuvant treatment, following TURBT. Efficacy of drug treatments has historically been limited due to challenges presented by bladder physiology, specifically the fact that urine is produced and voided frequently, thus diluting the concentration of the drug almost immediately and causing the excretion of the drug from the bladder at first urine voiding.

UGN-102 (VESIGEL™) (mitomycin) urothelial gel, 0.18% , an RTGel-based formulation of high dose mitomycin C (“MMC”), is our product candidate that is being studied for the potential treatment of low-grade NMIBC. UGN-102 is administered locally using standard catheters and is designed to conform to the bladder’s anatomy and persist in the bladder despite urine flow and bladder movement. Once instilled, UGN-102 converts into gel form within approximately 15 minutes at body temperature. Subsequently, upon contact with urine, UGN-102 gradually dissolves and releases the active drug, MMC, over a period of several hours.  Conversely, MMC in its current water-based formulation may be released upon first voiding.  We are evaluating the safety and efficacy of this drug candidate, and our preliminary research suggests that the gel formulation does not interfere with the natural flow of urine out of the bladder.   Use our proprietary gel appears to significantly increase dwell time of MMC in the bladder thereby prolonging exposure of MMC to the tissue, which suggests that the drug candidate has the potential to chemoablate visible and unseen tumors. As a result of these properties, the Company has undertaken clinical trials to evaluate UGN-102 as a first-line chemoablation non-surgical alternative to TURBT for the treatment of low-grade NMIBC.  Clinical trials to assess safety and efficacy remain on-going.