UGN-101

UGN-101

Unmet need

Low-grade UTUC is a urothelial malignancy that presents in the lining of the upper urinary tract, in the ureters and renal collecting system of the kidneys. It is a rare but critical cancer that affects approximately 6,000-8,000 new patients in the United States every year.1

The standard of care for patients with high-grade UTUC is radical nephroureterectomy (RNU), which is the complete removal of the affected kidney, ureter, and bladder cuff. 1,2

However, due to significant treatment challenges associated with the complex anatomy of the upper urinary tract, up to 80% of low-grade UTUC is also treated with radical surgery, typically RNU.3

Endoscopic tumor resection, a kidney-sparing surgical procedure, is sometimes performed in patients with low-grade disease. However, this approach is typically limited to those who present with a limited number of smaller tumors that can be easily visualized and removed using standard instrumentation.1,3 The upper urinary tract’s anatomical constraints limit the effectiveness of these procedures, and they are associated with high rates of recurrence.4

There are currently no drugs approved by the US Food & Drug Administration (FDA) for the treatment of low-grade UTUC, representing a significant unmet medical need.

Pipeline candidate

Urology Immuno- uro-oncology Uro-oncology Overactive bladder (OAB) BOTOX ® /RTGel TM reverse- thermal hydrogel High-grade non-muscle invasive bladder cancer (NMIBC) UGN-201 Low-grade non-muscle invasive bladder cancer (NMIBC) UGN-102 Low-grade upper tract urothelial carcinoma (UTUC) UGN-101 (orphan) REGISTRATION PHASE 3 PHASE 2 PHASE 1 NONCLINICAL PROGRAM CATEGORY

*Licensed to Allergan. The safety and efficacy of the above product candidates for the specified conditions have not been established.
BOTOX is a registered trademark of Allergan plc.

PHASE 2 BOTOX ® /RTGel TM reverse-thermal hydrogel Overactive bladder (OAB) Urology NON- CLINICAL UGN-201High-grade non-muscleinvasive bladder cancer (NMIBC) Immuno-uro-oncology PHASE 2 UGN-102Low-grade non-muscleinvasive bladder cancer (NMIBC) REGISTRATION UGN-101 (orphan)Low-grade upper tract urothelial carcinoma (UTUC) Uro-oncology

*Licensed to Allergan. The safety and efficacy of the above product candidates for the specified conditions have not been established.
BOTOX is a registered trademark of Allergan plc.

UGN-101 (mitomycin gel) for instillation is an investigational drug formulation that utilizes our innovative technology, RTGel reverse-thermal hydrogel, for the treatment of low-grade UTUC.

Administered locally via ureteral catheter, UGN-101 is designed to fill and conform to the anatomy of the renal collecting system and dwell for a period of several hours before being excreted via normal urine flow.5

Status

  • The FDA has granted Orphan Drug, Fast Track, and Breakthrough Therapy designations to UGN-101 for the treatment of low-grade UTUC
  • Updated efficacy data from the phase 3 OLYMPUS trial was reported at the American Urological Association Annual Meeting in Chicago on May 5, 2019
    Read about the data update >
  • UroGen has completed enrollment of the pivotal phase 3 OLYMPUS trial to assess the safety and efficacy of UGN-101 for the treatment of patients with low-grade UTUC
    Learn more about OLYMPUS >
  • In December 2018, UroGen initiated a rolling submission of a New Drug Application for UGN-101 to the FDA and intends to complete the submission in 2019. If approved, UGN-101 would be the first medical therapy approved for the treatment of low-grade UTUC
References: 1. Rouprêt M, Babjuk M, Compérat E, et al. European Association of Urology guidelines on upper urinary tract urothelial carcinoma: 2017 update. Eur Urol. 2018;73(1):111-122. 2. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Bladder Cancer V.2.2017. © National Comprehensive Cancer Network, Inc. 2017. All rights reserved. Accessed June 26, 2019. To view the most recent and complete version of the guideline, go online to NCCN.org. 3. Upfill-Brown A, Lenis AT, Faiena I, et al. Treatment utilization and overall survival in patients receiving radical nephroureterectomy versus endoscopic management for upper tract urothelial carcinoma: evaluation of updated treatment guidelines. World J Urol. 2019;37(6):1157-1164. doi: 10.1007/s00345-018-2506-1. 4. Cutress ML, Stewart GD, Wells-Cole S, Phipps S, Thomas BG, Tolley DA. Long-term endoscopic management of upper tract urothelial carcinoma: 20-year single-centre experience. BJU Int. 2012;110(11):1608-1617. 5. Data on file. UroGen Pharma, Inc. 2019.