We are on the cutting edge of urothelial cancer treatment development with our investigational product candidates, which we believe have the potential to upend the status quo and open up new avenues to treat patients.
Bladder cancers are described as muscle invasive or non-muscle invasive (NMIBC) based on whether they have invaded into the wall of the bladder. Of the approximately 60,000 newly diagnosed cases of NMIBC per year, nearly 60% are considered low grade (LG). While there is a low risk of progression and mortality in this population, chronically relapsing disease remains a pervasive problem. In patients who recur, it is estimated that 68% will experience 2 or more recurrence episodes throughout the course of their disease.
The current primary standard of care for treating LG NMIBC patients is a surgical procedure known as transurethral resection of bladder tumor (TURBT). Repeated TURBT procedures can impact patients’ physical health and quality of life and are even associated with an increased risk in mortality. Due to the high disease recurrence rates, patients may need multiple TURBTs over the course of their lifetime, leaving them more prone to these surgical burdens.
There are currently no drugs approved by the US FDA for primary treatment of NMIBC—the 3 drugs currently approved are all indicated as adjuvant therapies. Without a definitive alternative, TURBT remains the only first-line option for patients.
UroGen is developing UGN-102 (mitomycin) for intravesical solution as a primary nonsurgical alternative to repetitive TURBT. UGN-102 is an investigational formulation that utilizes our innovative technology, RTGel™ reverse-thermal hydrogel, for the treatment of low-grade NMIBC.
Instilled via standard catheters, UGN-102 is designed to dwell for a period of several hours before being excreted via normal urine flow.
UroGen conducted a readout of a Phase 2b study to further investigate UGN-102 for the treatment of intermediate risk LG NMIBC. Currently UGN-102 is in Phase 3.Learn more >
Significant advances have been made in the field of immunotherapy, as scientists explore how to better harness the immune system to combat cancer. Checkpoint inhibitors such as anti-PD-1, anti-PD-L1, and anti-CTLA-4 are three such areas of study. These antibodies are designed to make tumors more susceptible to the body’s natural immune defenses.
In particular, CTLA-4 antibodies are seen as potentially potent and comprehensively acting immunomodulators due to the ability to stimulate cytotoxic T cells, while simultaneously inhibiting suppressive T-regulatory cells. When administered systemically, they have led to improved outcomes in patients suffering from advanced cancers.
However, experience in a variety of advanced cancers has shown there are significant risks to immunotherapies administered systemically, including side effects that range from mild to potentially life-threatening. Such dose-limiting toxicity is currently preventing patients from benefiting from the full therapeutic potential of this promising target.
UroGen is evaluating UGN-301, our in-licensed anti-CTLA-4 antibody (zalifrelimab), as a monotherapy and as combination therapy with UGN-201.
Nonclinical toxicity studies are underway to facilitate the initiation of a multi-arm Phase 1 study of UGN-301 as monotherapy and in combination with other agents. Phase 1 is expected to begin in early 2022.
Bladder cancers are described as muscle invasive or non-muscle invasive (NMIBC) based on whether they have invaded into the wall of the bladder. High-grade (HG) NMIBC is associated with an increased risk of recurrence and progression. Transurethral resection of bladder tumor (TURBT) followed by intravesical bacillus Calmette-Guérin (BCG) is currently the standard of care for treatment of high-grade NMIBC.
Treatment with BCG indicates that bladder cancer can be sensitive to immunomodulation—using the body’s immune system to attack cancer cells. However, some patients who experience BCG resistance or lack of response need to transition to other options like intravesical chemotherapy and systemic immunotherapies.
Challenges posed by these treatments can be severe. Recurrence rates are high with the standard of care, and systemic immunotherapies pose substantial risks—including side effects that range from mild to potentially life-threatening. Without alternatives that overcome these challenges, the highest-risk patients are left with radical bladder removal surgery as the best remaining option.
UGN-301 is UroGen’s in-licensed anti-CTLA-4 antibody (zalifrelimab). UGN-201 is our proprietary formulation of imiquimod, a toll-like receptor 7 (TLR 7) agonist. When combined, these medicines stimulate both the innate (UGN-201) and adaptive (UGN-301) immune responses, both of which are important for fully harnessing the power of the immune system to fight cancer.
UroGen is evaluating the combination of UGN-301 with hydrogel + UGN-201 as a local immunotherapy for the treatment of high-grade NMIBC.
Patients facing illnesses who have exhausted all available treatment options often want to know if and how they can receive early access to medicines that are not yet available in their country of residence.
UroGen has engaged Tanner Pharma Group to administer a Named Patient Program for JELMYTO® (mitomycin) for pyelocalyceal solution in select countries where JELMYTO is currently not available or approved for use by the local regulatory authority.
This program provides controlled, pre-approval access to JELMYTO in response to unsolicited requests by physicians, hospitals, pharmacies, distributors, ministries of health or other parties on behalf of specific, or named patients, in select countries where JELMYTO has not yet received a marketing authorization and in situations when patients have exhausted all available treatment options.
It is important to remember that potential risks and benefits are not yet established for medicines that have not received regulatory approval by the local government regulatory bodies. Healthcare providers and patients should consider all possible benefits and risks when seeking access to an unapproved medicine in their local jurisdiction.
Healthcare providers should direct Named Patient Program requests to Tanner Pharma Group via:
Patients seeking access to JELMYTO are advised to consult directly with their healthcare provider.