Biotech Firm Uncovers Long-Term Cancer Breakthrough in Nevada

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UroGen Pharma Ltd. (URGN:NASDAQ) announced new long-term data demonstrating a median duration of response of nearly four years for JELMYTO (mitomycin) for pyelocalyceal solution in treating low-grade upper tract urothelial cancer (LG-UTUC). The data were presented at the 2025 American Urological Association Annual Meeting in Las Vegas, Nevada.

Among 41 patients from the Phase 3 OLYMPUS trial who achieved a complete response after primary chemoablation with JELMYTO, the median duration of response was 47.8 months. This outcome was observed regardless of whether the cancer was new-onset or recurrent, with a median follow-up period of 28.1 months (95% confidence interval 13.1, 57.5). In long-term follow-up, 20 patients were evaluated, with a median follow-up of 53.3 months (95% confidence interval 27.9, 65.3). Median duration of response for this subset was not estimable due to the low event rate.

Brian Hu, MD, Associate Professor of Urology at Loma Linda University Health, commented, "This sub-analysis highlights new evidence for the impressive long-term benefits of JELMYTO, in both new-onset and recurrent low-grade upper tract urothelial cancer patients." He added that the findings "further establish JELMYTO as an effective treatment, providing patients with durable responses and a significant reduction in recurrence rates."

LG-UTUC is often managed with endoscopic techniques, but recurrence rates remain high. JELMYTO, a reverse thermal gel containing mitomycin, was developed to address the need for more durable, non-surgical treatment options. In the OLYMPUS trial, JELMYTO demonstrated the ability to eradicate disease and provide sustained responses.

H.C. Wainwright & Co. on April 28, 2025, analysts reiterated a Buy rating on UroGen Pharma Ltd. with a 12-month price target of US$55. 

Mark Schoenberg, MD, Chief Medical Officer of UroGen, stated in the news release, "The sub-analysis provides further compelling evidence that JELMYTO offers durable disease control and clinically meaningful responses, which could significantly reduce the need for repeated interventions."

To build on these results, UroGen is currently enrolling patients in the JELMYTO uTRACT Registry (NCT05874921), a real-world observational study. As of April 2025, 22 sites have been activated and 251 patients have been enrolled.

JELMYTO is the first and only FDA-approved non-surgical chemoablative treatment for adult patients with LG-UTUC. It is a cooled liquid formulation that becomes a gel at body temperature, enabling prolonged exposure of mitomycin to the urinary tract tissues before dissolving naturally over four to six hours.

Sector Snapshot: Biotechnology and Oncology

BioSpace reported on February 28 that the global oncology market was valued at US$356.20 billion in 2025 and was forecasted to surpass US$903.81 billion by 2034, representing a compound annual growth rate of 10.9%. The report stated that "the development of the global healthcare infrastructure and cancer continuing to be one of the leading causes of death worldwide" contributed significantly to market growth. Innovations such as immunotherapy, precision medicine, liquid biopsy, and next-generation sequencing were cited as driving factors that enhanced treatment outcomes and expanded early detection capabilities. The U.S. oncology market alone was valued at US$145.52 billion in 2024 and was projected to reach US$416.93 billion by 2034, supported by strong research initiatives and technological advancements.

According to a report published by Fortune Business Insights on April 7, the global urology devices market was valued at US$36.34 billion in 2024 and was projected to grow to US$64.33 billion by 2032, with a compound annual growth rate of 7.4%. The report indicated that technological progress, such as the introduction of single-use cystoscopes and improved imaging systems, was expected to propel demand for urology devices used in diagnosing and treating bladder, kidney, and urethral diseases. It also stated that "technological advancements in urology products are expected to propel the demand for these devices to treat these diseases," supported by the rising prevalence of kidney disorders and increased patient demand for minimally invasive procedures.

D. Boral Capital also issued a positive assessment of UroGen Pharma Ltd., maintaining a Buy rating with a price target of US$25.

Fortune Business Insights additionally pointed to the launch of new products like the VeraVue Single-Use Flexible Cystoscope by Boston Scientific in January 2024, highlighting how disposable devices offered "reduced infection transmission and consistent quality with no need for reprocessing or repairs." Growing demand for these products among hospitals and ambulatory surgical centers was noted as an important factor shaping future industry growth.

Stockhead wrote on April 10 that the broader biotechnology sector experienced a rebound following market fears over proposed pharmaceutical tariffs in the United States. The article explained that while biotech stocks had initially declined after tariff threats were announced, investor sentiment shifted after a 90-day pause was introduced. "Investors today pushed a slew of stocks up by more than double digits," Stockhead noted, as optimism returned to the sector. Although concerns over tariffs persisted, the report reflected continued investor interest in biotech innovation, particularly among companies working on critical therapies.

UroGen Pharma Earns Positive Analyst Reviews After AUA Updates

According to a report published by H.C. Wainwright & Co. on April 28, 2025, analysts reiterated a Buy rating on UroGen Pharma Ltd. with a 12-month price target of US$55. The report highlighted the presentation of new data at the 2025 American Urological Association (AUA) Annual Meeting, which showcased UGN-102’s “clinically meaningful two-year duration of response (24.2 months) by Kaplan–Meier analysis” from the Phase 2b OPTIMA II study. Analysts emphasized that UGN-102 demonstrated strong durability even among patients with multiple prior transurethral resection of bladder tumor (TURBT) procedures. The report stated that the long-term data “bode well for approvability and future commercial traction.” In addition, patient-reported outcomes across multiple studies showed that UGN-102 did not negatively impact quality-of-life measures, with H.C. Wainwright writing that “no measured domains or items exceeded the threshold for clinically significant worsening at three or 12 months.” The firm also noted that the Phase 1 data for UGN-301 demonstrated encouraging safety results, with no dose-limiting toxicities and limited systemic exposure, supporting future advancement into Phase 2 testing.

On the same date, D. Boral Capital also issued a positive assessment of UroGen Pharma Ltd., maintaining a Buy rating with a price target of US$25. In its report, D. Boral Capital pointed to the 2025 AUA Annual Meeting presentations as reinforcing momentum across UroGen’s bladder cancer franchise. Analysts described UGN-301 as having demonstrated “an acceptable safety profile and early signs of efficacy” in non-muscle invasive bladder cancer, and cited the Phase 3 ENVISION trial data showing an “18-month duration of response (DOR) of 80.6% among patients who achieved a complete response at three months.” Complementary findings from the Phase 2b OPTIMA II study further validated UGN-102’s treatment durability, with D. Boral Capital stating that the updates “continued to build a compelling profile for regulatory success.” The report noted that UroGen’s regulatory progress with the FDA, including the accepted New Drug Application for UGN-102, further supported the positive outlook.

Strong Pipeline Momentum Positions UroGen for Future Growth

According to its investor presentation, UroGen Pharma’s broader pipeline development aims to strengthen its presence in the urothelial cancer treatment space. The company’s RTGel technology platform, which underpins JELMYTO, is designed to enhance dwell time and local drug delivery within the urinary tract, overcoming physiological barriers to treatment.

Beyond JELMYTO, UroGen is advancing UGN-102, a minimally invasive, non-surgical therapy candidate for patients with low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The company expects a U.S. Food and Drug Administration (FDA) decision on UGN-102 by the target Prescription Drug User Fee Act (PDUFA) action date of June 13, 2025. If approved, UGN-102 would target a significantly larger addressable U.S. population of approximately 82,000 patients annually, compared to about 6,000 to 7,000 patients for LG-UTUC.

UroGen is also exploring additional candidates, including UGN-301, an anti-CTLA-4 monoclonal antibody being developed for high-grade non-muscle invasive bladder cancer (HG-NMIBC). Early-phase trials are evaluating UGN-301 as both a monotherapy and in combination with other agents. In addition, the acquisition of ICVB-1042, an investigational oncolytic virus therapy, expands UroGen’s future opportunities in immuno-oncology.

Financially, UroGen reported US$241.7 million in cash, cash equivalents, and marketable securities as of December 31, 2024. JELMYTO generated US$90.4 million in worldwide revenues in 2024, reflecting continued growth since its initial launch.

The company continues to invest in expanding the JELMYTO uTRACT registry and preparing for the potential commercialization of UGN-102. Management has indicated plans for strategic capital deployment and targeted expansion into other specialty oncology areas leveraging its RTGel technology.

Ownership and Share Structure

According to Refinitiv, 87.18% of Urogen is held by institutions. Among them, the top holders are RTW Investments with 8.32%, Adage Capital Management with 6.35%, Menora Mivtachim Insurance at 5%, BlackRock Institutional Trust with 4.76%, and TD Securities with 3.28%.

Management and Insiders hold 2.05% and strategic entities own 6.38%. Of those, Monograph Capital Partners has 3.41% and Akin Communication has 2.97%. The rest is retail.

UgoGen has a market cap of US$454.95 million, 41.7 million free float shares, and a 52 week range of US$8.94 to $20.70.