A pipeline of
new possibilities

UNMET NEED

Bladder cancers are described as muscle invasive or non-muscle invasive 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 non-muscle invasive bladder cancer (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 patients with low-grade intermediate risk non-muscle invasive bladder cancer (LG-IR-NMIBC) is a surgical procedure known as transurethral resection of bladder tumor (TURBT). Due to a lack of treatment options to reduce recurrences in these patients, they are typically managed with repeated 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 U.S. FDA for the treatment of recurrent LG-IR-NMIBC. If approved, UGN-102 will become the first medicine for this patient population.

ABOUT

UroGen^® is developing UGN-102 (mitomycin) for intravesical solution for tumor ablation and as an alternative to repetitive TURBT. UGN-102 is an investigational formulation that utilizes UroGen's innovative technology, RTGel^® reverse-thermal hydrogel.

UNMET NEED

Bladder cancers are described as muscle invasive or non-muscle invasive based on whether they have invaded the muscular layer of the wall of the bladder. Of bladder cancer cases each year, approximately 75% are non-muscle invasive bladder cancer (NMIBC), and 55% of those cases 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% of recurrent patients will experience two or more recurrence episodes and 23% of recurrent patients will experience five or more recurrences throughout the course of their disease. Upon recurrence, the most common treatment today is repeated surgery with general anesthesia.

ABOUT

In January 2024, UroGen^® entered into a license and supply agreement with medac to develop next-generation products by combining our proprietary RTGel^® technology with medac’s proprietary formulation of mitomycin. This includes UGN-103, which combines UroGen’s RTGel technology with medac’s licensed mitomycin formulation. If approved, anticipated advantages include a new 75 mg mitomycin dosage strength that may considerably shorten the manufacturing process and simplify the reconstitution procedure. UroGen has two patents with claims relating to the combination of the Company’s RTGel technology combined with medac’s licensed proprietary lyophilized mitomycin formulation. These patents cover UGN-103 as well as the use of UGN-103 for the treatment of LG-IR-NMIBC and expire in December 2041.

UNMET NEED

Patients with low-grade upper tract urothelial cancer (LG-UTUC) are typically treated with kidney-sparing management, which includes endoscopic resection with or without intracavitary drug therapy. Kidney-sparing endoscopic surgery for small, solitary, low-grade tumors reduces the morbidity associated with radical surgery, without compromising oncological outcomes. However, endoscopic therapy is associated with high rates of local disease recurrence of about 50%-90% and requires lifelong surveillance, which can be associated with treatment-related complications such as ureteral stricture, perforation of the upper tract, bleeding, and infection. There remains a need for innovative and effective kidney-sparing approaches to LG-UTUC that avoid surgery, in turn reducing the likelihood of disease recurrence and improving patient outcomes. Over the past decade, research investigating the use of intravesical immunotherapy and chemotherapy agents for UTUC treatment has been challenged by the difficulty of achieving adequate dwell time, due to continuous urine production and drug dilution. This highlights a clinical need for an agent that conforms to the complex anatomy of the upper tract and demonstrates sufficient efficacy as well as durability of response for the treatment of LG-UTUC, either as adjuvant or primary therapy.

ABOUT

In January 2024, UroGen entered into a license and supply agreement with medac to develop next-generation products by combining our proprietary RTGel technology with medac’s proprietary formulation of mitomycin. This includes UGN-104, which combines UroGen’s RTGel technology with medac’s licensed mitomycin formulation. If approved, anticipated advantages include a new 75 mg mitomycin dosage strength that may considerably shorten the manufacturing process and simplify the reconstitution procedure. UroGen has two patents with claims relating to the combination of the Company’s RTGel technology combined with medac’s licensed proprietary lyophilized mitomycin formulation. These patents cover UGN-104 as well as the use of UGN-104 for the treatment of LG-UTUC and expire in December 2041.

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

In February 2025, UroGen acquired ICVB-1042 (now known as UGN-501) a next-generation investigational oncolytic virus. This addition meaningfully enhances UroGen’s pipeline by introducing a highly innovative approach to selectively targeting and destroying cancer cells while simultaneously activating a robust anti-tumor immune response. ICVB-1042 was thoughtfully engineered to achieve efficient cell entry, strong selectivity for malignant cells, and rapid replication within the tumor microenvironment—features that drive both direct tumor cell lysis and the induction of a durable, tumor-specific immune response.