PRINCETON - UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing novel solutions that treat urothelial and specialty cancers, today highlights the results of a sub-analysis from the first and largest real-world patient cohort review of JELMYTO (mitomycin) for pyelocalyceal solution presented at the American Urological Association Meeting 2024 in San Antonio, TX.

Among patients with low-grade Ta upper tract urothelial carcinoma (UTUC) who were complete responders to induction therapy (n=53), JELMYTO was associated with an 86% recurrence-free survival rate at 24 months across diverse patient types regardless of initial disease characteristics or usage for chemoablation versus post-endoscopic ablation. Among the 30% of the complete responders who received maintenance therapy, RFS at 24 months was 100%, vs. 61% for those who did not receive maintenance therapy.

'The 86% recurrence-free survival rate at the 24-month mark among low-grade UTUC patients (n=53) exhibited a favorable response to the initial induction of this new treatment,' said Adam Feldman, M.D., M.P.H., Urologic Oncologist in the Department of Urology at Massachusetts General Hospital, and Director of the Combined Harvard Urologic Oncology Fellowship. 'Notably, there seems to be no discernible difference in disease recurrence based on tumor characteristics or timing of administration, including primary chemoablation or post-endoscopic ablation. These results offer additional evidence for the clinical utility of JELMYTO and may also offer hope for improved long-term outcomes for our patients.'

Furthermore, a different analysis of the same patient cohort, presented by Yair Lotan, M.D., Professor of Urology and Chief of Urologic Oncology at UT Southwestern and Parkland Health and Hospital System and study investigator, reports that 'maintenance therapy with JELMYTO following successful induction treatment (n = 16) yielded a RFS rate of 100%, supplying more evidence of JELMYTO's pivotal role in treating this challenging condition,' according to Dr. Lotan.

Data was collected from 15 centers on patients treated with JELMYTO for upper tract urothelial cancers (UTUC). Recurrence-free survival was calculated in 53 patients with LGTa disease at baseline who had no evidence of disease following JELMYTO induction. Chemoablative use was defined as the administration of JELMYTO treatment in the setting of known residual UTUC, while post-chemoablation use was defined as receipt of JELMYTO following visually complete endoscopic ablation. Exploratory analyses were performed to assess impact of size of tumor, presence of ureteral involvement, and multifocality of UTUC prior to JELMYTO induction on RFS at 24 months.

'These findings provide additional evidence reinforcing JELMYTO's position as a valuable therapeutic option for patients with low-grade upper tract urothelial cancer and add to the growing body of real-world evidence in extending the long-term positive outcomes for patients,' said Mark Schoenberg, MD, Chief Medical Officer at UroGen. 'This real-world evidence reaffirms JELMYTO's role as a valuable tool in our fight against this disease.'

There were 136 cases of UTUC treated with JELMYTO with a cumulative median (IQR) follow-up of 22 (12-27) months including 107 cases of LGTa UTUC. After initial treatment, 74% of post-endoscopic ablation and 39% of chemoablative patients were disease-free totaling 53 cases with LGTa UTUC without evidence of disease following JELMYTO induction. The limitations of these sub-analyses include the sample size, retrospective design, lack of a control group, and the lack of a centralized pathology review and standardized clinicopathologic assessment.

To further explore the full potential of JELMYTO for the treatment of patients with UTUC, investigators are in the process of enrolling the prospective and retrospective uTRACT Registry to capture data in a large-scale, standardized manner to report further on patient outcomes following JELMYTO treatment including long-term longitudinal follow-up.

About JELMYTO

JELMYTO (mitomycin) for pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel indicated for the treatment of adult patients with low-grade-UTUC (LG-UTUC). It is approved for adult patients with LG-UTUC. JELMYTO is a viscous liquid when cooled and becomes a semi-solid gel at body temperature. The drug slowly dissolves over four to six hours after instillation and is removed from the urinary tract by normal urine flow and voiding. It is approved for administration in a retrograde manner via ureteral catheter or antegrade through nephrostomy tube. The delivery system allows the initial liquid to coat and conform to the upper urinary tract anatomy. The eventual semisolid gel allows for chemoablative therapy to remain in the collecting system for four to six hours without immediately being diluted or washed away by urine flow.

About Upper Tract Urothelial Cancer (UTUC)

Urothelial cancer is the ninth most common cancer globally and the eighth most lethal neoplasm in men in the U.S. Between five percent and ten percent of primary urothelial cancers originate in the ureter or renal pelvis and are collectively referred to as UTUC. In the U.S., there are approximately 6,000 - 7,000 new or recurrent LG-UTUC patients annually. Most cases are diagnosed in patients over 70 years old, and these older patients often face comorbidities. There are limited treatment options for UTUC, with the most common being endoscopic surgery or nephroureterectomy. Treatment with endoscopic surgery can lead to a high rate of recurrence and relapse.

About UroGen Pharma Ltd.

UroGen is a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers because patients deserve better options. UroGen has developed RTGel reverse-thermal hydrogel, a proprietary sustained-release, hydrogel-based platform technology that has the potential to improve the therapeutic profiles of existing drugs. UroGen's sustained release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. Our first product to treat LG-UTUC and investigational treatment UGN-102 (mitomycin) for intravesical solution for patients with low-grade non-muscle invasive bladder cancer are designed to ablate tumors by non-surgical means. UroGen is headquartered in Princeton, NJ with operations in Israel.

Forward-Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding the results from the real-world study of JELMYTO offering hope for improved long-term outcomes for patients; the enrollment of the prospective and retrospective data from the uTRACT Patient Registry and plans to analyze and report on such data; the potential of UroGen's proprietary RTGel technology to improve therapeutic profiles of existing drugs and UroGen's sustained release technology making local delivery potentially more effective as compared to other treatment options. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: results from of the real-world study of JELMYTO may not be indicative of results that may be observed in future clinical practice and may differ from additional analysis of the data from the study or uTRACT Patient Registry; potential safety and other complications from JELMYTO use in diverse UTUC patient types; the ability to maintain regulatory approval; the ability to obtain and maintain adequate intellectual property rights and adequately protect and enforce such rights; complications associated with commercialization activities; UroGen's RTGel technology may not perform as expected and UroGen may not successfully develop and receive regulatory approval of any other product that incorporates UroGen's RTGel technology.

In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section of UroGen's Annual Report on Form 10-K for the year ended December 31, 2023, filed with the SEC on March 14, 2024 (which is available at www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and UroGen's actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to UroGen as of the date of this release.

Contact:

Vincent Perrone

Email: vincent.perrone@urogen.com

Tel: 609-460-3588

Cindy Romano

Email: cindy.romano@urogen.com

Tel: 609-460-3583

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