On May 12, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported financial results for the first quarter ended March 31, 2025, and provided an overview of recent developments (Press release, UroGen Pharma, MAY 12, 2025, View Source [SID1234652889]).

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"We are entering a pivotal and exciting period for UroGen as we approach the anticipated FDA approval of our lead pipeline product, UGN-102, in June for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer," said Liz Barrett, President and Chief Executive Officer of UroGen. "This milestone has the potential to mark the first major advancement in treatment for this patient population in decades, delivering a much-needed novel and innovative treatment option that may offer meaningful disease and treatment-free intervals. With momentum building across the organization, we are entering the final phase of launch readiness. If approved, UGN-102 represents a significant commercial opportunity, with a total addressable market of over $5 billion. Backed by a strong balance sheet and a growing pipeline, we are well-positioned to build a long-term, sustainable growth company. We remain steadfast in our mission to transform the treatment paradigm in uro-oncology and see a great opportunity to advance patient care and deliver value for shareholders."

Q1 2025 and Recent Business Highlights:

UGN-102 (mitomycin) for intravesical solution

UroGen’s New Drug Application (NDA) for investigational drug UGN-102 (mitomycin) for intravesical solution as a treatment for recurrent low-grade intermediate risk non-muscle invasive bladder cancer (LG-IR-NMIBC), is currently under review at the U.S. Food and Drug Administration (FDA). The FDA granted a Prescription Drug User Fee Act (PDUFA) target action date of June 13, 2025. The FDA has scheduled an Oncologic Drugs Advisory Committee (ODAC) meeting on May 21, 2025 to discuss the UGN-102 NDA.
Updated 18-month Duration of Response (DOR) data from the Phase 3 ENVISION trial evaluating UGN-102 in patients with recurrent LG-IR-NMIBC were featured in a podium presentation by Dr. Sandip Prasad at the American Urological Association (AUA) 2025 Annual Meeting on April 26 in Las Vegas. The data are consistent with prior Kaplan-Meier estimates, with the probability of remaining in complete response (CR) of 80.6% (95% CI: 74.0, 85.7) at 18-months after achieving CR at 3 months (N=101) compared to 82.5% (95% CI: 76.1, 87.3) at 12-months after 3-month CR (N=146). Median follow-up time at 18 months was 18.7 months after the three-month CR.
The results of a patient reported outcomes analysis from three UGN-102 studies (OPTIMA II, ATLAS and ENVISION) were presented in a poster at the AUA meeting. The results showed that UGN-102 did not have a negative impact on symptom burden, patient function, or quality of life.
Long-term outcomes of the OPTIMA II LT study of UGN-102 were also presented at the meeting and demonstrated median DOR of 24.2 months (95% CI 9.72, 47.18) with a median follow-up time of 33.6 months (95% CI 10.78, 42.94) in the 41 patients who achieved a CR in the parent study.
Results from a post-hoc sub-analysis of the ENVISION trial, showing that tumor burden and the number of tumors did not significantly affect the CR rate or durability of response for patients treated with UGN-102, were presented at the ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium (ASCO-GU 2025) in February 2025.
Results from the ENVISION trial were published in the February 2025 issue of The Journal of Urology.
JELMYTO (mitomycin) for pyelocalyceal solution in low-grade upper tract urothelial cancer (LG-UTUC)

Generated net product revenue of $20.3 million in the quarter ended March 31, 2025, an increase of 8% over the $18.8 million reported for the same quarter in 2024. Underlying demand grew by 12% year-over-year.
A poster featuring long-term outcomes of patients with recurrent and new-onset LG-UTUC who achieved CR in the Phase 3 trial of JELMYTO was presented at AUA 2025. As previously reported, the median DOR of all patients achieving CR in the JELMYTO Phase 3 study was 47.8 months, irrespective of whether their cancer was new onset or recurrent.
A long-term study on JELMYTO titled, "Durability of Response of UGN-101: Longitudinal Follow-Up of Multicenter Study," was published online in Urologic Oncology: Seminars and Investigations in January 2025. The results showed 68% recurrence-free survival rate at three years across a broad patient population with LG-UTUC.
UGN-301 (zalifrelimab) intravesical solution, an anti-CTLA4 antibody for use in high-grade non-muscle invasive bladder cancer (NMIBC)

Enrollment is complete in the multi-arm dose escalation Phase 1 clinical study of UGN-301 in patients with high-grade NMIBC, alone and in combination with fixed dose UGN-201 or gemcitabine. The investigational treatments demonstrated an acceptable safety profile and were generally well tolerated across dose levels. Responses were observed in both monotherapy and combination therapy arms, and patient follow-up is ongoing to further assess the durability of these responses.
Next-generation investigational oncolytic virus UGN-501 (ICVB-1042)

In February 2025, UroGen expanded its nonclinical oncology portfolio with the purchase of product candidate UGN-501 from IconOVir Bio, Inc (IconOVir). UGN-501 is a potent and fast-replicating investigational next generation oncolytic virus therapy, being developed as a locally administered treatment for bladder cancer and other specialty cancers. UroGen hosted a webinar to discuss UGN-501 (ICVB-1042) on February 20, 2025, and a replay can be accessed on the Company’s website here.
Next-generation novel mitomycin-based formulation for urothelial cancers

Enrollment is ongoing in the Phase 3 UTOPIA clinical trial of investigational drug UGN-103 (mitomycin) for intravesical solution in patients with LG-IR-NMIBC and enrollment is expected to be completed by mid-2025. UGN-103 is a next generation product that combines UroGen’s RTGel technology with a novel mitomycin formulation licensed from medac GmbH. UGN-103 is planned to follow the potential FDA approval and launch of UGN-102 for recurrent LG-IR-NMIBC. To learn more about the UTOPIA trial, refer to clinicaltrials.gov/NCT06331299.
UroGen plans to initiate a Phase 3 trial to explore the safety and efficacy of UGN-104 by mid-2025. UGN-104 is a next generation investigational product for LG-UTUC.
First quarter 2025 Financial Results

JELMYTO Revenue: JELMYTO net product revenues were $20.3 million for the three months ended March 31, 2025, compared with $18.8 million for the same quarter of 2024. Year-over-year revenue growth of 8% was driven by underlying demand growth of 12%, partially offset by higher 340B chargebacks.

R&D Expenses: R&D expenses for the first quarter of 2025 were $19.9 million, including non-cash share-based compensation expense of $0.6 million as compared to $15.5 million, including non-cash share-based compensation expense of $0.5 million, for the same period in 2024. The increase in R&D expenses of $4.4 million was primarily driven by the equity consideration issued to IconOVir for the acquisition of UGN-501 (ICVB-1042) which was expensed in the first quarter of 2025, higher manufacturing costs, and costs associated with the Phase 3 UTOPIA trial for UGN-103, partially offset by lower clinical trial costs and regulatory expenses in connection with UGN-102.

SG&A Expenses: SG&A expenses for the first quarter of 2025 were $35.0 million, including non-cash share-based compensation expense of $2.5 million. This compares to $27.3 million, including non-cash share-based compensation expense of $2.2 million, for the same period in 2024. The increase in SG&A expenses of $7.7 million was primarily driven by UGN-102 commercial preparation activities.

Financing on Prepaid Forward Obligation: UroGen reported non-cash financing expense related to the prepaid forward obligation to RTW Investments of $4.6 million in the first quarter of 2025, compared to $5.7 million in the same period in 2024. The decrease was primarily driven by changes in underlying assumptions for remeasuring the effective rate.

Interest Expense on Long-Term Debt: Interest expense related to the $125 million term loan facility with funds managed by Pharmakon Advisors was $4.1 million in the first quarter of 2025, compared to $2.4 million in the same period in 2024. The increase was primarily attributable to the interest expense on the third tranche of the loan that was funded in September 2024.

Net Loss: UroGen reported a net loss of $43.8 million or ($0.92) per basic and diluted share in the first quarter of 2025 compared with a net loss of $32.3 million or ($0.87) per basic and diluted share in the same period in 2024.

Cash and Equivalents: As of March 31, 2025, cash, cash equivalents and marketable securities totaled $200.4 million.

For further details on the Company’s financials, refer to Form 10-Q, filed with the SEC.

2025 JELMYTO Revenue and Company Operating Expense Guidance: Guidance for full-year 2025 net product revenues for JELMYTO remains unchanged and is expected to be in the range of $94 to $98 million. This implies a year-over-year growth rate of approximately 8% to 12% over the $87.4 million in demand driven JELMYTO sales in 2024, which excludes the $3.0 million in CREATES Act sales reported in 2024. Continue to expect full-year 2025 operating expenses to be in the range of $215 to $225 million, including non-cash share-based compensation expense of $11 million to $14 million.

Conference Call & Webcast Information: Members of UroGen’s management team will host a live conference call and webcast today at 10:00 AM Eastern Time to review UroGen’s financial results and provide a general business update.

The live webcast can be accessed by visiting the Investors section of the Company’s website at View Source Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast.