Investigational Therapy UGN-102 May Be Delivered at Home for the Treatment of Bladder Cancer

UroGen Pharma, a biotech company dedicated to developing and commercializing innovative treatment of urothelial and specialty cancers, today announced preliminary results of a study to assess the feasibility of home instillation of UGN-102 (mitomycin) for intravesical solution, an investigational therapy in development for primary chemoablation of low-grade, intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). In this study, UGN-102 was suitable to administer at home by a visiting nurse under the supervision of a treating physician and resulted in 75% of patients achieving a complete response, defined as no detectable disease 3 months after starting treatment.

“Many bladder cancer patients are elderly and suffer from comorbidities that make frequent office visits for treatment a real burden,” said study investigator David Morris, M.D., F.A.C.S., Urologist at Urology Associates PC, Nashville, TN. “Moving healthcare out of the clinical setting and into a patient’s home would represent a new treatment paradigm for bladder cancer, which may reduce clinic and hospital costs while increasing patient comfort and convenience.”

Eight patients were enrolled and treated with UGN-102 by 4 investigators. Median age was 75 years (range 55-84). Six patients completed treatment (6 doses), and 2 patients discontinued the study (5 doses and 4 doses, respectively) due to adverse events (AEs) unrelated to treatment. All 8 patients were evaluable for treatment response, and 6 of 8 (75%) achieved a complete response 3 months after starting treatment. The 2 patients who discontinued were assessed as non-responders. Treatment-related AEs were mild to moderate in severity (most common: dysuria and fatigue in 2 patients), and 3 patients with significant comorbidities had a serious AE, all of which were unrelated to treatment with UGN-102.

Patients, nurses and investigators completed home instillation feasibility questionnaires. These standardized feasibility questionnaires highlighted that all 8 patients preferred at-home to in-office treatment, and 5 of 6 patients recommended UGN-102 home instillation instead of transurethral resection of bladder tumors (TURBT). Home instillation was reported as feasible for visiting nurses, and 3 of 4 investigators considered at-home treatment “not different” than in-office treatment.

“As a urologic oncologist, I feel confident that the home instillation study results, appearing on the heels of our Phase 2 program and following the complete enrollment of our pivotal Phase 3 ENVISION study for UGN-102, provide us with even more reasons to believe that our novel approach to treating LG-IR-NMIBC has the potential to address genuine unmet needs of patients with bladder cancer,” said Arie Belldegrun, M.D., Board Chair of UroGen.

The study enrolled patients with recurrent LG-IR-NMIBC who agreed to receive UGN‑102 at home. Six weekly doses of UGN-102 were administered. The first dose was administered on-site by a physician and the five remaining doses were administered at home by a visiting nurse. Patients were followed for AEs and treatment response was evaluated by visual observation, for cause biopsy, and urine cytology 3 months after treatment initiation. Complete response was defined as the absence of disease by white light cystoscopy, cytology, and histopathology.

“This study is another example of how UroGen continues to advance its mission to pioneer the way urothelial cancers are treated,” says Liz Barrett, President and Chief Executive Officer, UroGen. “2023 will be a pivotal year for UroGen as we report on the ATLAS study of UGN-102 and start combinatorial treatments in the Phase 1 study of UGN-301. UroGen is in a strong position to achieve leadership in uro-oncology.”