Results of the First Post-Commercial Utilization Review of JELMYTO® Provide New Evidence that Reinforces its Efficacy and Safety in Patients with Low-Grade Upper Tract Urothelial Cancer

PRINCETON, N.J.--(BUSINESS WIRE)-- UroGen (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, today highlights results from the first and largest post-commercial utilization review of JELMYTO^® (mitomycin) for pyelocalyceal solution. This study evaluated 132 patients treated with JELMYTO from 15 high-volume academic and community centers and characterizes the manner in which urologists are now using JELMYTO in their practices. The study titled, “Early Experience with UGN-101 for the Treatment of Upper Tract Urothelial Cancer – A Multi-Center Evaluation of Practice Patterns and Outcomes,” is published in Urologic Oncology: Seminars and Original Investigations.

The authors describe several trends in JELMYTO use that differ from the pivotal Olympus study that led to drug approval. Doctors are using JELMYTO for the treatment of large tumors (>3cm in 15% of cases), high grade tumors (9% of cases), and ureteral tumors (35% of cases). In addition, the report documents the use of JELMYTO as a valuable multimodal adjunct following complete laser tumor ablation. JELMYTO is indicated for the treatment of adult patients with low-grade upper tract urothelial cancer (LG-UTUC). Primary chemoablation resulted in a 70% complete response (CR) rate for patients with tumors less than 1cm (CR rates following primary therapy were lower for larger tumors consistent with previous reporting) while 69% achieved CR when JELMYTO was combined with laser ablation. CR in the Olympus trial was 59%.

Almost half of the patients in the review were treated by antegrade administration of JELMYTO through a nephrostomy tube. The Olympus cohort was treated exclusively by retrograde catheter passed into the upper tract through the bladder. The authors of a recent single-center retrospective review observed that antegrade administration of JELMYTO was associated with a lower rate of ureteric stenosis than the retrograde approach as observed in OLYMPUS, and this larger multi-center review supports that earlier observation. The rate of ureteric stenosis in the Olympus trial was 44%. In this multi-center study it was 23%.

The limitations of this study include the retrospective design, lack of a control group, 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 longitudinal follow-up.

“This study provides valuable insights into how physicians are utilizing JELMYTO in the real-world and reinforces the genuine clinical value of this therapy for patients with LG-UTUC,” said Mark Schoenberg, M.D., Chief Medical Officer, UroGen. “We look forward to additional analyses from this retrospective study as well as prospective and retrospective data from the uTRACT Patient Registry.”

About UTUC
Approximately 5-7% of urothelial cancer occurs in the upper lining of the kidney, called the calyx and renal pelvis. It could also occur in one or both of the ureter(s), the tubes that lead from the kidneys to the bladder. Cancer in the renal pelvis or ureter(s) is called upper tract. LG-UTUC is usually not very aggressive and is slow to spread but has a high recurrence rate. High-grade UTUC can be more aggressive. It may spread to other parts of the urinary tract, or to other parts of the body.

JELMYTO is approved for the treatment of adults with LG-UTUC. LG-UTUC is a rare disease managed by endoscopic methods and radical nephroureterectomy. Endoscopic resection and laser ablation attempt to preserve the kidney, though there is a high risk of recurrence that may eventually necessitate removal of the kidney. Although kidney removal is the gold standard for treatment of high-grade UTUC, it may be over-treatment in LG-UTUC, as kidney removal offers similar five-year survival as kidney-sparing procedures but is associated with significant morbidity. JELMYTO is efficacious as a primary chemoablative therapy in patients with LG-UTUC.

About JELMYTO^®
JELMYTO^® (mitomycin) for pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel indicated for primary chemoablative treatment of LG-UTUC in adults. It is recommended for primary treatment of biopsy-proven LG-UTUC in patients deemed appropriate candidates for renal-sparing therapy. 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.

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 U.S. Food and Drug Administration. Visit www.fda.gov/medwatch or call 1800FDA1088. You may also report side effects to UroGen Pharma at 1-855-987-6436.

Please click here for JELMYTO Full Prescribing Information, including the Patient Information, for additional information and here for the Nephrostomy Administration Guide.

About UroGen Pharma Ltd.
UroGen is 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 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. UroGen’s first commercial product JELMYTO (mitomycin) for pyelocalyceal solution, 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 Twitter, @UroGenPharma.

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 additional analysis from the retrospective study of JELMYTO as well as prospective and retrospective data from the uTRACT Patient Registry; 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 initial reports from the retrospective study evaluating post-commercial utilization of JELMYTO in the publication 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; potential safety and other complications from JELMYTO use in diverse UTUC patient types; and our RTGel technology may not perform as expected and we may not successfully develop and receive regulatory approval of any other product that incorporates our 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 Form 10-Q filed with the United States Securities and Exchange Commission (SEC) on November 10, 2022 and other filings that UroGen makes with the SEC from time to time (which are 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.

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