Upper tract urothelial carcinoma (UTUC) affects about 2 in 100,000 out of the total population in Western countries – about 10% of the prevalence of bladder cancer. The traditional treatment for this cancer is a complete surgical removal of the involved kidney and bladder cuff. For patients with a bilateral disease, an anatomic or functionally solitary kidney, or medical comorbidities – a kidney-conserving alternative is considered, if possible. But because of the specific anatomy and physiology, the performance of organ sparing endoscopic resection and instillation of neoadjuvant or adjuvant chemotherapy are extremely challenging. Difficulties in administering and maintaining MMC or any other drug to the upper tract due to low residual duration and short exposure time of the active agent in the treated area resulting in low treatment efficacy and limited chemoablative use in patients with UTUC. The recurrence rate is about 65% within 10 months – five times faster compared to bladder cancer. Furthermore and due to its rarity, clinical progress of UTUC treatments is limited and currently no clinically approved organ-sparing pharmaceutical treatment exists for UTUC.
UroGen is developing MitoGel, our novel sustained-release formulation of MMC, for the treatment of low-grade, non-muscle invasive UTUC. We have observed preliminary evidence of the efficacy of MitoGel in an ongoing investigator-initiated Compassionate Use program for the treatment of severe, non-resectable UTUC. Based on multiple discussions with the FDA, we do not believe that we will need to perform additional clinical trials prior to initiating a single pivotal Phase 3 clinical trial with MitoGel. Additionally, the FDA granted MitoGel Orphan Drug Designation for the treatment of UTUC. We plan to develop MitoGel through the FDA’s 505(b)(2) regulatory pathway. We intend to commence a single pivotal Phase 3 clinical in the first half of 2017.