On March 29, 2022 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that the U.S. Food and Drug Administration (FDA) has cleared UroGen’s Investigational New Drug (IND) application to begin a novel Phase 1 clinical study of the anti-CTLA-4 immunotherapy UGN-301 (zalifrelimab) in patients with recurrent NMIBC (Press release, UroGen Pharma, MAR 29, 2022, View Source [SID1234611132]). The multi-arm Phase 1 study is expected to start in April and support the development of UGN-301 in high-grade (HG) NMIBC.
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UroGen’s pursuit to harness the power of the immune system to fight cancer begins with UGN-301, which UroGen views as a potential cornerstone of a variety of combination therapies targeting recurrent NMIBC and high-grade cancers. UroGen initially plans to combine UGN-301 with UGN-201, the Company’s proprietary formulation of imiquimod a toll-like receptor 7 (TLR7) agonist, which has demonstrated single-agent activity in high-risk bladder cancer patients.
The novel study design will utilize a Master Protocol that UroGen believes is a more efficient and streamlined approach to development. It will provide more flexibility to add study arms as the trial progresses and increase efficiency and reduces costs. UroGen expects the Master Protocol will allow the Company to more quickly evaluate safety, tolerability and dosing of UGN-301 in combination with additional immunomodulators and chemotherapies, with the goal of developing optimized medicines for patients.
"We are pleased that our IND application was cleared to proceed, and we can begin to explore our innovative approach to meeting the high unmet needs in bladder cancer, especially for patients with high-grade disease," says Mark Schoenberg, Chief Medical Officer, UroGen Pharma. "Intravesical delivery of combination therapies is unique with the goal of improving efficacy while avoiding the toxicities associated with systemic treatment of immunotherapies. Our proprietary technology enables local delivery of treatments, which provide opportunities to pursue several promising drug combinations."
Unmet Needs in Bladder Cancer
Bladder cancers are described as muscle invasive or non-muscle invasive based on whether they have invaded the wall of the bladder. HG NMIBC is associated with an increased risk of recurrence and progression. Approximately 25,000 people are diagnosed with HG NMIBC annually. Transurethral resection of bladder tumor (TURBT) followed by intravesical bacillus Calmette-Guérin (BCG) is currently the standard of care for treatment of HG NMIBC.
HG NMIBC patient response to BCG therapy has long been interpreted as evidence that bladder cancer is sensitive to immunotherapy. Unfortunately, many patients with HG disease do not respond to BCG or relapse following therapy. In these cases, patients have limited therapeutic options and often proceed to bladder removal as a means of forestalling disease progression which has a significant association with cancer specific mortality. UroGen’s proprietary RTGel technology provides a novel method for delivering alternatives to BCG including immunomodulatory molecules such as UGN-301 as well as chemotherapeutic agents and other drugs with diverse molecular characteristics and sizes.