ENVISION Trial Results Published in the February Issue of The Journal of Urology Highlight UGN-102 Achievement of 82.3% Duration of Response at 12 Months Paving the Way for the Potential First FDA-Approved Treatment for LG-IR-NMIBC in June 2025

Rhea-AI Summary

UroGen Pharma (NASDAQ: URGN) announced the publication of Phase 3 ENVISION trial results for UGN-102 in The Journal of Urology. The study showed an 82.3% duration of response at 12 months and a 79.6% complete response rate at three months in patients with low-grade intermediate-risk non-muscle-invasive bladder cancer (LG-IR-NMIBC).

The company completed its New Drug Application (NDA) submission to the FDA in August, with a PDUFA date set for June 13, 2025. If approved, UGN-102 would be the first FDA-approved treatment for LG-IR-NMIBC, offering a non-surgical alternative for patients who currently face repeated surgeries under general anesthesia.

The trial reported mild-to-moderate treatment-emergent adverse events, including dysuria, hematuria, and urinary tract infection, which were generally resolving or resolved.

Positive

• Achieved 82.3% duration of response at 12 months in Phase 3 trial
• Met primary endpoint with 79.6% complete response rate at 3 months
• NDA submission completed ahead of schedule
• Potential to be first FDA-approved treatment for LG-IR-NMIBC

Negative

• PDUFA decision not expected until June 2025
• Presence of adverse events including urinary complications

Insights

Medical Research Analyst

The ENVISION Phase 3 trial results for UGN-102 represent a potential breakthrough in treating low-grade intermediate-risk non-muscle-invasive bladder cancer (LG-IR-NMIBC). The data reveals two important metrics: a 79.6% complete response rate at 3 months and an impressive 82.3% duration of response at 12 months.

These results are particularly noteworthy because current standard treatment typically involves repeated surgical procedures under general anesthesia. The demonstrated durability of response, maintaining 80.9% effectiveness even at 15 and 18 months, suggests UGN-102 could significantly reduce the need for frequent surgical interventions.

The safety profile appears manageable, with mostly mild-to-moderate adverse events that resolve over time. This is particularly important for the target population, who are often elderly and may have tolerance for aggressive treatments.

Financial Analyst

From a market perspective, UGN-102's potential FDA approval in June 2025 represents a significant catalyst for UroGen Pharma. With a current market cap of $422M, this therapeutic could substantially impact the company's valuation given it would be the first FDA-approved treatment for LG-IR-NMIBC.

The addressable market is considerable, as bladder cancer patients often require multiple treatments over their lifetime. The non-surgical nature of UGN-102 could drive strong adoption among both physicians and patients, potentially leading to substantial revenue growth. The completed NDA submission ahead of schedule and upcoming PDUFA date are important near-term catalysts that could drive stock movement.

Clinical Oncologist

The clinical implications of UGN-102's trial results are transformative for bladder cancer treatment. The 82.3% duration of response at 12 months is remarkable in this patient population, where recurrence is a major challenge. For context, current surgical approaches often see high recurrence rates, requiring repeated procedures.

The non-surgical delivery method through intravesical solution offers several advantages: reduced procedural risks, no need for general anesthesia and potentially better quality of life for patients. The consistent efficacy through 18 months suggests UGN-102 could significantly extend treatment-free intervals, a important benefit for elderly patients who may be poor candidates for repeated surgeries.

• ENVISION Reports 79.6% Complete Response Rate at 3 Months, 82.3% Duration of Response at 12 Months, and Consistent Safety Profile

PRINCETON, N.J.--(BUSINESS WIRE)-- UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, today announced that the 3-month complete response (CR) rate and 12-month durability of response from the Phase 3 ENVISION study of investigational drug UGN-102 in patients with low-grade intermediate-risk non-muscle-invasive bladder cancer (LG-IR-NMIBC) were published in the February issue of The Journal of Urology.

In the ENVISION trial, UGN-102 treatment demonstrated an impressive 82.3% (95% CI, 75.9%, 87.1%) 12-month duration of response (DOR) by Kaplan-Meier estimate (n=108) in patients who achieved a CR at three months after the first instillation of UGN-102 (mitomycin) for intravesical solution. The Kaplan-Meier estimates for DOR at 15 months (n=43) and 18 months (n=9) following the 3-month CR were both 80.9% (95% CI, 73.9%, 86.2%). The ENVISION trial also met its primary endpoint, showing a 79.6% (95% CI, 73.9%, 84.5%) CR rate at three months in patients treated with UGN-102.

“These data from the ENVISION trial provide compelling evidence that treatment with UGN-102 achieves a clinically meaningful complete response rate and also demonstrates remarkable durability in patients with LG-IR-NMIBC,” said Sandip Prasad, MD, M.Phil., Director of Genitourinary Surgical Oncology at Morristown Medical Center/Atlantic Health System, NJ, and Principal Investigator of the ENVISION trial. “The long-term results, with 82.3% duration of response at 12 months, further strengthen UGN-102’s potential as a non-surgical, effective treatment for patients facing the recurrent and challenging nature of LG-IR-NMIBC.”

According to Mark Schoenberg, M.D., Chief Medical Officer, UroGen, "The impressive duration of response data from the ENVISION trial further highlights UGN-102’s potential to transform the treatment landscape for patients with LG-IR-NMIBC. Many of these patients are elderly and face the burden of repeated surgeries under general anesthesia, so there is a critical need for innovative treatment options for this patient population. We believe that, if approved, UGN-102’s ability to achieve durable complete responses and potentially reduce recurrence rates while extending treatment-free intervals will represent a significant advance in managing LG-IR-NMIBC.”

UroGen initiated the submission of a rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for UGN-102 as a treatment for LG-IR-NMIBC in January 2024 and completed the NDA submission in August, ahead of schedule. The FDA accepted the NDA for UGN-102 with a PDUFA goal date of June 13, 2025.

The most common treatment-emergent adverse events (TEAEs) in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. The TEAEs were typically mild-to-moderate in severity and either resolved or were resolving. The ENVISION trial demonstrated a similar safety profile to that observed in other studies of UGN-102.

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently in Phase 3 development for the treatment of LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel® technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional. UroGen completed the NDA submission in August, ahead of schedule. The FDA accepted the NDA for UGN-102 and assigned a PDUFA goal date of June 13, 2025.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

In the U.S., bladder cancer is the second most common urologic cancer in men. LG-IR- NMIBC represents approximately 23,000 newly diagnosed bladder cancer patients each year and an estimated 59,000 recurrences annually among patients diagnosed from previous years. Bladder cancer primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. Guideline recommendations for the management of NMIBC include trans-urethral resection of bladder tumor (TURBT) as the standard of care. Up to 70 percent of NMIBC patients experience at least one recurrence and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures.

About ENVISION

The Phase 3 ENVISION trial is a single-arm, multinational, multicenter study evaluating the efficacy and safety of UGN-102 (mitomycin) for intravesical solution as a chemoablative therapy in patients with LG-IR-NMIBC. The Phase 3 ENVISION trial completed target enrollment with approximately 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of UGN-102. The primary endpoint evaluated the CR rate at the three-month assessment after the first instillation, and the key secondary endpoint evaluated durability over time in patients who achieved a CR at the three-month assessment. Learn more about the Phase 3 ENVISION trial at www.clinicaltrials.gov (NCT05243550).

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 low-grade upper tract urothelial cancer and investigational treatment UGN-102 (mitomycin) for intravesical solution for patients with LG-IR-NMIBC 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.

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 for UGN-102 as the first FDA-approved non-surgical treatment for LG-IR-NMIBC; the estimated annual U.S. patient population and demographics for LG-IR-NMIBC; the potential benefits to patients and opportunities for UGN-102, if approved, including to transform the treatment landscape for patients with LG-IR-NMIBC; statements related to UroGen’s NDA submission and expected PDUFA target action date for UGN-102 and the potential approval and timing thereof; 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: even though the NDA for UGN-102 has been accepted for filing by the FDA, there is no guarantee that such NDA will be sufficient to support approval of UGN-102 on the timeframe expected, or at all; the ability to maintain regulatory approval; complications associated with commercialization activities; the labeling for any approved product; competition in UroGen’s industry; the scope, progress and expansion of developing and commercializing UroGen’s product candidates; the size and growth of the market(s) therefor and the rate and degree of market acceptance thereof vis-à-vis alternative therapies; UroGen’s ability to attract or retain key management, members of the board of directors and other personnel; 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 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 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20250115929797/en/

INVESTOR CONTACT:

Vincent Perrone

Senior Director, Investor Relations

vincent.perrone@urogen.com

609-460-3588 ext. 1093

MEDIA CONTACT:

Cindy Romano

Director, Communications

cindy.romano@urogen.com

609-460-3583 ext. 1083

Source: UroGen Pharma Ltd.

FAQ

What were the key efficacy results from URGN's ENVISION Phase 3 trial for UGN-102?

The ENVISION trial showed an 82.3% duration of response at 12 months and a 79.6% complete response rate at three months in patients with LG-IR-NMIBC.

When is the PDUFA date for UroGen's UGN-102 bladder cancer treatment?

The FDA set a PDUFA goal date of June 13, 2025, for UGN-102 as a treatment for LG-IR-NMIBC.

What are the main side effects reported in the ENVISION trial for UGN-102?

The most common side effects were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention, typically mild-to-moderate in severity.

How long did the duration of response last in the ENVISION trial for UGN-102?

The duration of response was 82.3% at 12 months and remained at 80.9% at both 15 and 18 months following the 3-month complete response.