Upper Tract Urothelial Carcinoma (UTUC)
An Orphan disease
Upper tract urothelial carcinoma (UTUC) is an uncommon disease accounting for 5-10 percent of urothelial carcinomas with 6,000 cases per year in the U.S. alone. The current incident rate is approximately two out of every 100,000 inhabitants with incident rates in China and Japan recording up to double that rate. UTUC is highly recurrent, with 15-50 percent of tumors recurring after surgical management and 8.5-13 percent of UTUC cases being concurrent with bladder cancer. UTUC is more prevalent in the elderly and affects three times more men than women.
The European Association of Urology Standard of Care (EAU-SOC) and the U.S. National Comprehensive Cancer Network (NCCN) guidelines recommend first stratifying by risk, then kidney-sparing surgery for low risk patients and Radical Nephroureterectomy (RNU) for high risk patients. Patients are high risk where the disease has already invaded the muscle with number, size and location of tumors being prognostic factors. In 2020, the estimated prevalence of UTUC in the U.S. was approximately 60,000, of which 60 percent were diagnosed with high risk disease.
Significant unmet medical needs
The current standards of care entail kidney sparing surgery for low risk patients and radical Nephroureterectomy (RNU) for high risk patients with adjuvant post-operative chemotherapy believed to reduce disease-free survival (DFS) rates.
An effective treatment that could preserve more kidneys and reduce recurrence could greatly improve the quality of life in patients and lower the overall cost of treatment.
LiPax-UTUC
LIPAC is developing a new neoliposomal intracavitary paclitaxel formulation for intraluminal treatment of UTUC administered through a specialized advanced catheter. LiPax™ (UTUC) utilizes neoliposomal paclitaxel technology which enables paclitaxel loaded liposomes to adhere to the urothelial ureter wall for sustained persistence and enhanced penetration into the ureter to improve overall efficacy.
The company has filed for orphan designation with the U.S. Food and Drug Administration. Such a designation would make LiPax (UTUC) eligible for seven years of exclusivity from approval in the U.S., and ten years of exclusivity in the European Union.