FDA Clears IND For UGN-501 in Non-Muscle Invasive Bladder Cancer

The FDA has cleared an investigational new drug application (IND) for the next-generation oncolytic virus UGN-501 in the treatment of patients with non-muscle invasive bladder cancer (NMIBC), according to a news release from the developer, UroGen Pharma.¹

Moreover, a phase 1 study is expected to begin in the fourth quarter of 2026, which will evaluate the safety, tolerability, and feasibility of intravesical UGN-501 among patients with NMIBC.

"Patients with [NMIBC] continue to [experience] a significant risk of disease recurrence despite available treatment options," said Mark Schoenberg, MD, chief medical officer of UroGen, explained in the news release.¹ "UGN-501 is an investigational next-generation oncolytic virus designed to selectively destroy tumor cells while generating an anti-tumor immune response. FDA clearance of the IND allows us to begin evaluating whether the encouraging nonclinical profile of UGN-501 can translate into a safe and meaningful therapeutic approach for patients with NMIBC.”

The oncolytic virus is designed to selectively replicate within tumor cells, facilitating direct tumor cell destruction and eliciting an anti-tumor immune response. Although UGN-501 is initially being developed for the treatment of NMIBC, the developers expressed that the underlying properties of the agent could have broader applicability across additional solid tumor indications. They intend to evaluate development opportunities informed by emergent clinical and translational data.

According to the developers, nonclinical data to date demonstrate cytotoxic activity across a panel of bladder cancer cell lines, which represent a range of tumor stages and grades.² The agent will be delivered via a proprietary RTGel technology, potentially enabling prolonged dwell time and enhanced local activity.

“We look forward to initiating the phase 1 study and advancing our efforts to develop innovative treatment options for patients with bladder cancer,” Schoenberg concluded.¹

Regarding other agents in the UGN pipeline, the developers plan to submit a new drug application for mitomycin (UGN-103) as a treatment for recurrent low-grade intermediate risk NMIBC (LG-IR-NMIBC) in the second half of 2026 for a potential approval in 2027. Supporting the NDA submission are data from the phase 3 UTOPIA trial (NCT06331299),in which mitomycin exhibited a 3-month complete response (CR) rate of 77.8% (95% CI, 68.3%-85.5%), which was consistent with liquid mitomycin (Zusduri), which attained a 3-month CR rate of 79.6% (95% CI, 73.9%-84.5%) in the phase 3 ENVISION trial (NCT05243550).³

The single-arm phase 3 UTOPIA trial treated patients with 75 mg of UGN-103, which consists of mitomycin and a sterile hydrogel, to reconstitute mitomycin prior to instillation.⁴ Patients who did not have detectable disease at a 3-month visit entered the study follow-up period, returning every 3 months or until disease progression or recurrence, death, or 12 months of follow-up.

Moreover, a phase 3 clinical trial (NCT06774131) exploring the safety and efficacy of UGN-104 among patients with treatment of low-grade upper tract urothelial cancer (UTUC) is ongoing, with an expected completion of enrollment by the end of 2026.⁵ Those in the trial will undergo treatment with the investigational agent for 6 weekly doses consisting of 4 mg of mitomycin per 1 mL of sterile hydrogel via ureteral catheter or nephrostomy tube.

Those with a CR after the initial treatment period will enter the follow-up period and could be eligible to receive UGN-104 as maintenance for 11 monthly doses at investigator discretion. Regardless of receipt of maintenance treatment, patients will return for 3-month follow to evaluate response and will remain on study for 12 months in the absence of disease recurrence, progression, or death.

References

1. UroGen announces FDA Clearance of the IND for UGN-501, an investigational next-generation oncolytic virus for non-muscle invasive bladder cancer. News release. UroGen Pharma. July 8, 2026. Accessed July 8, 2026. https://tinyurl.com/4jk4m35y
2. UroGen reports ZUSDURI™ revenue more than doubled quarter-over-quarter and provides first quarter 2026 financial results and highlights. News release. UroGen Pharma. May 6, 2026. Accessed July 8, 2026. https://tinyurl.com/3kyyfx6w
3. UroGen reports 77.8% three-month complete response rate from phase 3 UTOPIA trial of UGN-103 and receives FDA agreement on NDA submission strategy in recurrent LG-IR-NMIBC based on UTOPIA trial. News release. UroGen Pharma. November 6, 2025. Accessed July 8, 2026. https://tinyurl.com/4jzzpwxm
4. A phase 3 study of UGN-103 for treatment of patients with low-grade intermediate-risk non-muscle invasive bladder cancer (UTOPIA). ClinicalTrials.gov. Updated January 15, 2026. Accessed July 8, 2026. https://tinyurl.com/y3azb2rd
5. A phase 3 single-arm study of UGN-104 for the treatment of low-grade upper tract urothelial cancer. ClincalTrials.gov. Updated June 16, 2026. Accessed July 8, 2026. https://tinyurl.com/mswaausb