Person in lab with beaker

A pipeline for new possibilities

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.

Our Current Pipeline

URO-ONCOLOGY

Nonclinical

Phase 1

Phase 2

Phase 3

Registration

Unmet Need

Bladder cancers are described as muscle invasive or non-muscle invasive (NMIBC) based on whether they have invaded into the muscular layer of the wall of the bladder. Of the approximately 60,000 newly diagnosed cases of NMIBC per year, 54% 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 standard of care for treating LG-IR-NMIBC patients is a surgical procedure known as transurethral resection of bladder tumor (TURBT). Due to lack of treatment options to reduce recurrences in these patients, they are typically managed with repeat TURBT for each subsequent recurrence. 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, LG-IR-NMIBC patients will likely 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 LG intermediate risk NMIBC. Without a definitive alternative, TURBT remains the only first-line option for patients.

About

UroGen is developing UGN-102 (mitomycin) for intravesical solution as a 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 intermediate-risk NMIBC.

Instilled via standard catheters, UGN-102 is designed to dwell in the bladder for a period of several hours before being excreted via normal urine flow.

Status

UroGen announced positive topline results from the ATLAS and ENVISION Phase 3 clinical trials and has initiated submission of a rolling NDA to the FDA for the treatment of intermediate risk LG NMIBC. The secondary endpoint of duration of response from ENVISION is expected during the second quarter of 2024.

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IMMUNO-URO-ONCOLOGY

Unmet Need

Bladder cancers are described as muscle invasive or non-muscle invasive (NMIBC) based on whether they have invaded into the muscular layer of the wall of the bladder. Of the approximately 60,000 newly diagnosed cases of NMIBC per year, high grade (HG) NMIBC represents approximately 30% of all NMIBC. Standard treatment for HG-NMIBC is transurethral resection of bladder tumors followed by adjuvant intravesical immunotherapy with Bacillus Calmette-Guérin (BCG). Unfortunately, a third of patients will not respond to BCG, and half of patients who initially respond to BCG will experience recurrence or progression. Radical cystectomy is strongly advocated in patients who are BCG‑refractory or relapse following BCG treatment due to the risk of invasive and metastatic disease highlighting the need for nonsurgical treatment options in the setting of BCG failure.

Significant advances in the field of immunotherapy, have found that immune checkpoint inhibitors like anti-CTLA-4 can harness the body’s natural immune defenses to combat cancer. When administered systemically, anti-CTLA-4 therapy has 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 agent.

About

UGN-301 is our in-licensed, anti-CTLA-4 monoclonal antibody (zalifrelimab), prepared with reverse thermal hydrogel for local administration into the bladder. Local administration of UGN-301 is expected to increase drug concentrations in the bladder without significant systemic exposure, potentially diminishing the systemic toxicity associated with CTLA-4 blockade.

UroGen is evaluating UGN-301, our in-licensed anti-CTLA-4 antibody (zalifrelimab), as a monotherapy and as combination therapy for the intravesical treatment of high-grade NMIBC.

Status

UroGen is evaluating UGN-301, in a multi-arm Phase 1 study of UGN-301 as monotherapy and in combination with other agents. Safety and dosing data from the UGN-301 monotherapy arm are expected late-2024.

Unmet Need

Bladder cancers are described as muscle invasive or non-muscle invasive (NMIBC) based on whether they have invaded into the muscular layer of the wall of the bladder. Of the approximately 60,000 newly diagnosed cases of NMIBC per year, high grade (HG) NMIBC represents approximately 30% of all NMIBC. Standard treatment for HG-NMIBC is transurethral resection of bladder tumors followed by adjuvant intravesical immunotherapy with Bacillus Calmette-Guérin (BCG). Unfortunately, a third of patients will not respond to BCG, and half of patients who initially respond to BCG will experience recurrence or progression. Radical cystectomy is strongly advocated in patients who are BCG‑refractory or relapse following BCG treatment due to the risk of invasive and metastatic disease highlighting the need for nonsurgical treatment options in the setting of BCG failure.

About

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, we believe the combination may stimulate both innate and adaptive immune responses, both of which are important for fully harnessing the power of the immune system to fight cancer. We believe that this combination makes local immunotherapy a potentially more effective treatment option while minimizing systemic exposure and potential side effects.

Status

UroGen is evaluating UGN-301 in combination with UGN-201 in a multi-arm Phase 1 study. Safety and dosing data from this combination arm are expected in 2025.

Unmet Need

Bladder cancers are described as muscle invasive or non-muscle invasive (NMIBC) based on whether they have invaded into the muscular layer of the wall of the bladder. Of the approximately 60,000 newly diagnosed cases of NMIBC per year, high grade (HG) NMIBC represents approximately 30% of all NMIBC. Standard treatment for HG-NMIBC is transurethral resection of bladder tumors followed by adjuvant intravesical immunotherapy with Bacillus Calmette-Guérin (BCG). Unfortunately, a third of patients will not respond to BCG, and half of patients who initially respond to BCG will experience recurrence or progression. Radical cystectomy is strongly advocated in patients who are BCG‑refractory or relapse following BCG treatment due to the risk of invasive and metastatic disease highlighting the need for nonsurgical treatment options in the setting of BCG failure.

About

UGN-301 is UroGen’s in-licensed anti-CTLA-4 antibody (zalifrelimab). Gemcitabine is a chemotherapy that is used intravesically to treat high grade NMIBC where it is administered as a liquid formulation.

When combined, we believe the combination may stimulate both innate and adaptive immune responses, both of which are important for fully harnessing the power of the immune system to fight cancer. We believe that this combination makes local immunotherapy a potentially more effective treatment option while minimizing systemic exposure and potential side effects.

Status

UroGen is evaluating UGN-301 in combination with gemcitabine in a multi-arm Phase 1 study. Safety and dosing data from this combination arm are expected in 2025.

UGN-102, UGN-301 and UGN-201 are investigational drugs in development. The safety and efficacy of these agents has not been established by any regulatory body including the FDA.

Expanding Access to JELMYTO Outside the United States

Named Patient Program

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.