Posted Date: 12/20/2016

UroGen Pharma Announces FDA Acceptance of Investigational New Drug Application

UroGen Pharma Ltd. recently announced the acceptance of its Investigational New Drug (IND) application by the US FDA for MitoGel for the treatment of low-grade upper tract urothelial carcinoma (UTUC). There are currently no FDA-approved drugs for the treatment of UTUC. UroGen plans to initiate a single pivotal, open-label, single-arm Phase III clinical trial of MitoGel for the treatment of low-grade UTUC in the first quarter of 2017. The single pivotal, open-label, single-arm Phase III clinical trial design is based on preliminary evidence of the safety and efficacy of MitoGel from an ongoing investigator-initiated Compassionate Use program for the treatment of severe, non-resectable, low-grade UTUC.

Utilizing RTGel, a proprietary sustained-release, hydrogel-based formulation, MitoGel is designed to enable longer exposure of Mitomycin C to the urinary tract tissue, thereby potentially enabling the treatment of tumors by non-surgical means.  MitoGel has been granted Orphan Drug Designation by the FDA for the treatment of UTUC.

"IND acceptance by the FDA is an important milestone for UroGen and our MitoGel program, and it highlights our commitment to developing new therapies for urological diseases that we believe have high unmet clinical needs. We look forward to commencing the single pivotal, open-label, single-arm Phase III clinical trial for MitoGel for the treatment of low-grade UTUC in the first quarter of 2017,” said Ron Bentsur, Chief Executive Officer of UroGen Pharma.

"As we continue to advance our product candidates, we look forward to potentially establishing a leadership position in the treatment of various forms of urothelial cancer for the benefit of patients. Concurrently, we are exploring the broad applicability of the RTGel platform beyond uro-oncology, and our recently announced Allergan collaboration for overactive bladder is one such example,” added Arie Belldegrun, MD, UroGen Pharma's Chairman.

Upper tract urothelial carcinoma (UTUC) has an estimated annual incidence in the US of up to 7,500 cases – about 5% to 10% of all new cases of urothelial cancer. UTUC refers to cancer of the upper tract, which connects the bladder to the kidney, and the renal pelvis. The current standard of care for this cancer is complete surgical removal of the involved kidney and upper tract. For patients with a bilateral disease, an anatomic or functionally solitary kidney, medical comorbidities, or low-grade disease that present with a limited number of tumors, a kidney-conserving alternative is considered, if possible. However, due to the specific anatomy and physiology of the upper tract and renal pelvis, the performance of organ-sparing endoscopic resection and instillation of neoadjuvant or adjuvant chemotherapy are extremely challenging, leading to high rates of recurrence and risk for progression. Difficulties in administering and maintaining Mitomycin C or any other drug in the upper tract due to low residual duration and short exposure time of the active agent in the treated area results in low treatment efficacy and limited use of chemoablative agents in patients with UTUC.  Furthermore, due to the rarity of UTUC, clinical development of UTUC treatments is limited and there are currently no FDA-approved drugs for the treatment of UTUC.

UroGen Pharma is a clinical-stage biopharmaceutical company developing advanced non-surgical, local treatments to address unmet needs in the field of urology, with a focus on uro-oncology. The company has developed RTGel, a proprietary sustained-release, hydrogel-based formulation for potentially improving the efficacy and safety profiles of existing drugs. UroGen Pharma's sustained-release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. UroGen Pharma's lead product candidates, MitoGel and VesiGel, are designed to potentially remove tumors by non-surgical means and to treat several forms of non-muscle invasive urothelial cancer, including low-grade UTUC and bladder cancer. Moreover, UroGen Pharma has recently completed a worldwide licensing agreement with Allergan Pharmaceuticals International Limited, a wholly owned subsidiary of Allergan plc, for the use of RTGel with neurotoxins for the treatment of overactive bladder and related conditions. UroGen Pharma is headquartered in Israel and also maintains a corporate office in New York City. For more information, visit

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