UroGen Submits Completed UGN-102 NDA Seeking Approval as the First FDA-Approved Treatment for Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer

Rhea-AI Summary

UroGen Pharma (Nasdaq: URGN) has submitted a New Drug Application (NDA) for UGN-102, a potential first FDA-approved treatment for low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The company anticipates possible FDA approval in early 2025, pending acceptance and priority review. The NDA is supported by the Phase 3 ENVISION study, which demonstrated a 79.6% complete response rate at three months and an 82.3% 12-month duration of response. UGN-102 aims to provide an alternative to repeated surgeries, offering potential quality of life benefits and clinically meaningful recurrence-free intervals for patients with this challenging disease.

Positive

• Successful completion of NDA submission for UGN-102
• Potential to be the first FDA-approved treatment for LG-IR-NMIBC
• Phase 3 ENVISION study met its primary endpoint with 79.6% complete response rate
• 82.3% 12-month duration of response in patients who achieved complete response
• Potential for FDA approval in early 2025 if granted priority review

Negative

• Most common treatment-emergent adverse events included dysuria, hematuria, and urinary tract infection
• FDA approval not guaranteed and subject to review process

Medical Research Analyst

The submission of UGN-102's NDA is a significant milestone in the treatment landscape for LG-IR-NMIBC. The 79.6% complete response rate and 82.3% 12-month duration of response are impressive figures that could potentially revolutionize patient care. These results suggest UGN-102 could offer a non-surgical alternative with durable efficacy, addressing a critical unmet need in bladder cancer treatment.

The safety profile, with mostly mild-to-moderate TEAEs, appears manageable. However, it's important to note that long-term safety data beyond 18 months is still The potential FDA approval in early 2025 could be a game-changer, but investors should be aware that regulatory processes can be unpredictable, especially for novel therapies.

Financial Analyst

UroGen's NDA submission for UGN-102 represents a pivotal moment for the company's growth trajectory. If approved, UGN-102 could be the first FDA-approved treatment for LG-IR-NMIBC, potentially securing a significant market share in a largely untapped segment. This could translate into substantial revenue growth for UroGen, especially given the high recurrence rates of this cancer type.

Investors should consider the potential impact on UroGen's stock value if UGN-102 receives FDA approval. However, it's important to note that the timeline for potential approval extends to early 2025, which means any significant financial impact won't be immediate. The company's ability to navigate the regulatory process and prepare for a successful commercial launch will be critical factors to watch in the coming months.

PRINCETON, N.J.--(BUSINESS WIRE)-- UroGen Pharma Ltd. (Nasdaq: URGN), a leading biotech company specializing in novel therapies for urothelial and specialty cancers, today announced the successful completion of its New Drug Application (NDA) submission for investigational drug UGN-102, (mitomycin) for intravesical solution, a significant step forward in potentially addressing the urgent need for innovative treatments for low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). UroGen anticipates potential FDA approval in early 2025, if the NDA is accepted for filing by the FDA and priority review is granted.

“The completion of the NDA submission for UGN-102 marks a crucial milestone for UroGen and underscores our dedication to advancing this groundbreaking treatment for patients with LG-IR-NMIBC,” said Liz Barrett, President and CEO of UroGen. “By providing a viable alternative to repeated surgeries, if approved UGN-102 may offer patients quality of life benefits and clinically meaningful recurrence-free intervals. The high recurrence rates associated with LG-IR-NMIBC make the need for innovative therapies like UGN-102 urgent. UGN-102 could become a valuable new option for managing this challenging disease.”

The NDA is supported by the clinical program for UGN-102, including the long-term durability results from the Phase 3 ENVISION study. The ENVISION trial met its primary endpoint by demonstrating that patients treated with UGN-102 had a 79.6% complete response (CR) rate at three months following the first instillation. UGN-102 demonstrated an 82.3% 12-month duration of response (DOR) by Kaplan-Meier estimate (n=108) in patients who achieved a CR at three months. The DOR estimates at 15 (n=43) and 18 (n=9) months after 3-month CR were both 80.9%.

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

For more information and perspective on the ENVISION study results and the unmet need for patients with LG-IR-NMIBC click here to view the webcast: UGN-102 12-Month Durability of Response Data Event.

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.

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 22,000 newly diagnosed bladder cancer patients each year and an estimated 60,000 recurrences annually among patients diagnosed from previous years. Bladder cancer primarily affects older populations with the median age of diagnosis 73 years and an increased risk of comorbidities. Guideline recommendations for the management of NMIBC include 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 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 treatment for LG-IR-NMIBC; the estimated annual U.S. patient population and demographics for LG-IR-NMIBC; the potential patient benefits and opportunities for UGN-102, if approved, the potential timing for a decision from the FDA on the UGN-102 NDA submission and the potential priority review; 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: the NDA may not be accepted for filing by the FDA, and even if it is, there is no guarantee that such NDA will be given priority review or 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 March 31, 2024, filed with the SEC on August 13, 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/20240814480495/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

908-963-7827

Source: UroGen Pharma Ltd.

FAQ

What is UGN-102 and what is it designed to treat?

UGN-102 is an investigational drug designed to treat low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). It's potentially the first FDA-approved treatment for this specific condition.

What were the key results of the Phase 3 ENVISION study for UGN-102 (URGN)?

The ENVISION study showed a 79.6% complete response rate at three months and an 82.3% 12-month duration of response for patients treated with UGN-102.

When does UroGen Pharma (URGN) expect potential FDA approval for UGN-102?

UroGen anticipates potential FDA approval for UGN-102 in early 2025, if the NDA is accepted for filing and granted priority review.

What are the potential benefits of UGN-102 for patients with LG-IR-NMIBC?

If approved, UGN-102 may offer patients an alternative to repeated surgeries, potentially providing quality of life benefits and clinically meaningful recurrence-free intervals.