New Real-World Durability of Response Data for JELMYTO Reports 68% Recurrence-Free Survival Rate (RFS) at Three Years Across a Broad Patient Population with Low-Grade Upper Tract Urothelial Cancer (LG-UTUC)

January 22, 2025

-No RFS difference based on use of JELMYTO for primary chemoablation versus post-endoscopic ablation as adjuvant therapy

-No RFS difference based on tumor size, location, number of tumors or route of administration

-Maintenance treatment in a small group linked to higher RFS

PRINCETON, N.J.,--(BUSINESS WIRE)--Jan. 22, 2025-- UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, today highlights results from a study on the durability of response from the first and largest post-commercialization study of JELMYTO® (mitomycin) for pyelocalyceal solution. The long-term study evaluated 56 patients who achieved complete response after treatment with JELMYTO from 15 high-volume academic and community centers and helps characterize how urologists are now using JELMYTO in their practices. This long-term study titled, “Durability of Response of UGN-101: Longitudinal Follow-Up of Multicenter Study,” is published online in Urologic Oncology: Seminars and Investigations.

"The three-year durability data from this study further validate the potential of JELMYTO in providing long-term disease control for patients with low-grade upper tract urothelial cancer,” said Solomon L. Woldu, MD, Assistant Professor of Urology, UT Southwestern Medical Center, Dallas, Texas, and study investigator. “Notably, we found that recurrence-free survival was not influenced by factors like tumor size or location, highlighting the broad applicability of this treatment. The potential benefits of maintenance therapy are encouraging, and further research will be key in confirming its role in improving outcomes for these patients.”

In this study on durability of response, 68% of patients with LG-UTUC who initially responded to JELMYTO had no evidence of disease recurrence at 3 years, as evaluated via endoscopy. The median follow-up was 23.5 months. RFS did not vary significantly based on use of JELMYTO for chemoablative versus adjuvant intent, tumor location (pyelocalyceal versus ureteral), tumor size before induction, single versus multiple tumors, or JELMYTO administration route (antegrade versus retrograde). The administration of maintenance treatment did appear to be associated with significantly better RFS, however, only 15 patients received maintenance therapy and, according to the authors, further study is required to determine the value of maintenance treatments.

"We are excited by the three-year results showing the durability of JELMYTO in treating low-grade upper tract urothelial cancer, with 68% of patients remaining recurrence-free,” said Mark Schoenberg, M.D., Chief Medical Officer, UroGen. “These findings underscore the promising long-term potential of JELMYTO in managing this challenging disease. We are committed to improving the lives of patients with urothelial cancer and advancing our mission to deliver breakthrough therapies that transform the standard of care for patients with complex urological conditions."

The limitations of this study include the retrospective design, lack of a control group, and the lack of a centralized pathology review. Further study is needed to better understand the long-term outcomes of JELMYTO and the risks/benefits of maintenance therapy in this setting. In the phase 3 OLYMPUS study, the safety and efficacy of JELMYTO was not investigated in the adjuvant setting (although tumor debulking was permitted prior to study entry), patients with ureteral tumors and tumors larger than 15 mm were excluded, administration was limited to the retrograde technique, and complete response to treatment was assessed via urine cytology, ureteroscopy and biopsy (when warranted). Due to the risks associated with JELMYTO treatment following endoscopic ablation of UTUC or following placement of a nephrostomy tube for JELMYTO instillation (antegrade administration), an appropriate time interval consistent with institutional guidelines and standard medical practice should precede treatment with JELMYTO.

About JELMYTO

JELMYTO® (mitomycin) for pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel approved for the treatment of adult patients with low-grade-UTUC (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 a 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 have multiple comorbidities. There are limited treatment options for UTUC, with the most common being endoscopic surgery or nephroureterectomy (removal of the entire kidney and ureter). Treatment with endoscopic surgery can be associated with 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. Visit www.urogen.com to learn more or follow us on X (Twitter), @UroGenPharma.

APPROVED USE FOR JELMYTO

JELMYTO® is a prescription medicine used to treat adults with a type of cancer of the lining of the upper urinary tract including the kidney called low-grade Upper Tract Urothelial Cancer (LG-UTUC).

IMPORTANT SAFETY INFORMATION

You should not receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.

Before receiving JELMYTO, tell your healthcare provider about all your medical conditions, including if you:

  • are pregnant or plan to become pregnant. JELMYTO can harm your unborn baby. You should not become pregnant during treatment with JELMYTO. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with JELMYTO. Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with JELMYTO and for 6 months after the last dose. Males being treated with JELMYTO: If you have a female partner who is able to become pregnant, you should use effective birth control (contraception) during treatment with JELMYTO and for 3 months after the last dose.
  • are breastfeeding or plan to breastfeed. It is not known if JELMYTO passes into your breast milk. Do not breastfeed during treatment with JELMYTO and for 1 week after the last dose.
  • Tell your healthcare provider if you take water pills (diuretic).

How will I receive JELMYTO?

  • Your healthcare provider will tell you to take a medicine called sodium bicarbonate before each JELMYTO treatment.
  • You will receive your JELMYTO dose from your healthcare provider 1 time a week for 6 weeks. It is important that you receive all 6 doses of JELMYTO according to your healthcare provider’s instructions. If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment. Your healthcare provider may recommend up to an additional 11 monthly doses.
  • JELMYTO is given to your kidney through a tube called a catheter.
  • During treatment with JELMYTO, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.

After receiving JELMYTO:

  • JELMYTO may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 6 hours.
  • To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
  • Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.

JELMYTO may cause serious side effects, including:

  • Swelling and narrowing of the tube that carries urine from the kidney to the bladder (ureteric obstruction). If you develop swelling and narrowing, and to protect your kidney from damage, your healthcare provider may recommend the placement of a small plastic tube (stent) in the ureter to help the kidney drain. Tell your healthcare provider right away if you develop side pain or fever during treatment with JELMYTO.
  • Bone marrow problems. JELMYTO can affect your bone marrow and can cause a decrease in your white blood cell, red blood cell, and platelet counts. Your healthcare provider will do blood tests prior to each treatment to check your blood cell counts during treatment with JELMYTO. Your healthcare provider may need to temporarily or permanently stop JELMYTO if you develop bone marrow problems during treatment with JELMYTO.
  • The most common side effects of JELMYTO include: urinary tract infection, blood in your urine, side pain, nausea, trouble with urination, kidney problems, vomiting, tiredness, stomach (abdomen) pain.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1800FDA1088. You may also report side effects to UroGen Pharma at 1-855-987-6436.

Please see JELMYTO Full Prescribing Information, including the Patient Information, for additional information.

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 potential benefits of JELMYTO in providing long-term disease control for patients with LG-UTUC; the potential benefits of maintenance therapy; the estimated patient population and demographics for UTUC; 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: prior results may not be indicative of results that may be observed in the future; potential safety and other complications from JELMYTO use in diverse UTUC patient types; and UroGen’s RTGel technology may not perform as expected and we 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 Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, filed with the SEC on November 6, 2024 (which is available at http://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.

INVESTOR CONTACT:  
Vincent Perrone  
Senior Director, Investor Relations  
vincent.perrone@urogen.com  
609-460-3588 ext. 1093  

MEDIA CONTACT:  
Cindy Romano  
Director, Corporate Communications  
cindy.romano@urogen.com  
609-460-3583 ext. 1083  

Source: UroGen Pharma Ltd.