Core Viewpoint - UroGen Pharma Ltd. has been downgraded from Buy to Neutral by HC Wainwright & Co. analyst Raghuram Selvaraju due to the FDA's Oncologic Drugs Advisory Committee voting 5-4 against the benefit-risk profile of its investigational therapy UGN-102 for bladder cancer [1][2]. Financial Position - UroGen concluded Q1 2025 with approximately $200 million in cash, cash equivalents, and marketable securities, which is expected to sustain operations through late 2026 even if UGN-102 fails to secure regulatory approval [4]. - The analyst forecasts a net loss of $2.14 per share for 2026, a significant change from the previous projection of earnings of 27 cents [4]. Development Pipeline - UroGen is advancing the development of UGN-103 and UGN-104, next-generation mitomycin-based formulations, which utilize the RTGel delivery platform combined with a novel mitomycin formulation licensed from medac GmbH [5]. - The Phase 3 UTOPIA trial for UGN-103 is currently enrolling patients, with enrollment expected to conclude by mid-2025 and top-line data anticipated shortly thereafter [5][6]. - If the results from UGN-103 are positive, a regulatory submission could occur in 2026, potentially leading to FDA approval by 2027 [6].

SAN FRANCISCO, May 22, 2025 (GLOBE NEWSWIRE) -- On May 21, 2025, investors in UroGen Pharma Ltd. (NASDAQ: URGN) saw the price of their shares crash 44% after the company announced that the FDA’s Oncologic Drugs Advisory Committee (“ODAC”) voted against the risk/benefit profile of UroGen’s UGN-102, a therapy intended to treat patients with low-grade, intermediate-risk non-muscle invasive bladder cancer (“LG-IR-NMIBC”) for which no drugs are currently FDA-approved. Hagens Berman has opened an investigation in ...

Group 1 - The author has a PhD in biochemistry and extensive experience analyzing clinical trials and biotech companies, emphasizing the importance of understanding the science behind investments in this sector [1] - The mission is to educate investors on conducting due diligence to avoid pitfalls in biotech investments [1] Group 2 - There is a potential for initiating a long position in URGN through stock or options purchases within the next 72 hours [2] - The article reflects the author's personal opinions and is not influenced by compensation from any company mentioned [2]

Core Viewpoint - UroGen Pharma Ltd. is under investigation for potential securities fraud and unlawful business practices, following concerns raised by the FDA regarding the clinical trial design of its bladder cancer drug UGN-102 [1][2]. Group 1: Investigation and Legal Actions - Pomerantz LLP is investigating claims on behalf of UroGen investors, focusing on possible securities fraud or other unlawful practices by the company and its officers [1]. - The investigation is prompted by the FDA's concerns regarding the Envision trial for UGN-102, which lacked a concurrent control arm, making the trial's primary endpoints difficult to interpret [2]. Group 2: FDA Concerns and Market Reaction - The FDA's briefing document indicated that it had recommended a randomized trial design to UroGen multiple times to address concerns about the trial's validity [2]. - Following the news of the FDA's concerns, UroGen's stock price dropped by $2.54 per share, representing a decline of 25.79%, closing at $7.31 per share on May 16, 2025 [3].

Core Insights - Holzer & Holzer, LLC is investigating UroGen Pharma Ltd. for potential compliance issues with federal securities laws following FDA comments on the company's clinical trial results [1] - The FDA indicated that the lack of a concurrent control arm in the ENVISION trial makes it difficult to interpret the primary endpoints of complete response (CR) and duration of response (DOR) [1] - UroGen's stock price experienced a decline after the FDA's statement regarding the investigational product UGN-102 [1] Company Overview - UroGen Pharma Ltd. is a publicly traded company on NASDAQ under the ticker URGN [1] - The company is involved in developing treatments for urological diseases, with a focus on innovative therapies [1] Legal Context - Holzer & Holzer, LLC is a law firm specializing in securities litigation, representing shareholders and investors in class action and derivative litigation [3] - The firm has a history of recovering significant amounts for shareholders affected by corporate misconduct [3]

Financial Data and Key Metrics Changes - UroGen reported net product revenues of $20.3 million for Q1 2025, an 8% year-over-year increase from $18.8 million in Q1 2024, driven by a 12% underlying demand growth [30][32] - The net loss for Q1 2025 was $43.8 million, or $0.92 per share, compared to a net loss of $32.3 million, or $0.87 per share, in the same period in 2024 [32] - Cash, cash equivalents, and marketable securities totaled $200.4 million as of March 31, 2025 [13][32] Business Line Data and Key Metrics Changes - Jelmyto sales for Q1 2025 were $20.3 million, reflecting an 8% year-over-year growth, with underlying demand growth of 12% [12][30] - UGN-102 is expected to target an addressable population of approximately 60,000 patients annually, translating to a market opportunity of over $5 billion, significantly larger than the Jelmyto market [11][12] Market Data and Key Metrics Changes - The company anticipates that the Medicare population will comprise about 70% of the business for UGN-102, consistent with the overall patient demographic in low-grade intermediate risk NMIBC [35] - The market for UGN-102 is expected to be more accessible, with a significant portion of patients managed by community urologists [11][12] Company Strategy and Development Direction - UroGen aims to transition from a rare disease-focused company to a multi-product team, with plans to expand the sales force from approximately 50 to over 80 representatives at the launch of UGN-102 [10][12] - The company is preparing for the potential launch of UGN-102, targeting commercial readiness by June 2025, with product availability expected in July [10][11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming ODAC meeting and the PDUFA target date, highlighting the strength of clinical data supporting UGN-102 [16][55] - The management team emphasized the importance of addressing the unmet medical need in the recurrent low-grade intermediate risk NMIBC patient population [46][55] Other Important Information - UroGen has a strong balance sheet with over $200 million in cash and equivalents, allowing for continued investment in innovation [13][32] - The company is actively engaged in prelaunch activities for UGN-102, including educational efforts with urologists and payer engagement strategies [24][26] Q&A Session Summary Question: Breakdown of Medicare and Medicaid exposure for UGN-102 and Jelmyto - Management anticipates that the Medicare population will comprise about 70% of the business for UGN-102, with a focus on driving reimbursement confidence with providers [35] Question: Preparation for the ODAC meeting and expected pushback - Management has conducted mock ODAC panels and feels confident about presenting the clinical data, particularly addressing the single-arm study design of ENVISION [41][46] Question: Current status of the UGN-103 clinical development program - Enrollment for the UGN-103 trial is progressing ahead of plan, with top-line data anticipated in 2026 [21][71] Question: Expectations regarding the gross-to-net for Jelmyto and UGN-102 - The gross-to-net profile for Jelmyto is in the mid-70s percent, with expectations for UGN-102 to be more favorable due to a higher proportion of community-based business [101]

Financial Data and Key Metrics Changes - UroGen reported net product revenues of $20.3 million for Q1 2025, an 8% year-over-year increase from $18.8 million in Q1 2024, driven by a 12% underlying demand growth [28][30] - The net loss for Q1 2025 was $43.8 million, or $0.92 per share, compared to a net loss of $32.3 million, or $0.87 per share, in the same period in 2024 [30] - Cash, cash equivalents, and marketable securities totaled $200.4 million as of March 31, 2025 [12][30] Business Line Data and Key Metrics Changes - Jelmyto sales for Q1 2025 were $20.3 million, reflecting an 8% year-over-year growth, supported by a 12% increase in underlying demand [11][28] - UGN-102 is expected to target an addressable population of approximately 60,000 patients annually, translating to a market opportunity exceeding $5 billion, significantly larger than the Jelmyto market [10][11] Market Data and Key Metrics Changes - The company anticipates that approximately 70% of UGN-102's business will come from the Medicare population, consistent with the demographics of low-grade intermediate-risk NMIBC patients [34] - The market for UGN-102 is expected to be more accessible than the fragmented UTUC setting, as NMIBC patients are primarily managed by community urologists [10][11] Company Strategy and Development Direction - UroGen aims to transition from a rare disease-focused company to a multi-product organization, with UGN-102 as a primary growth driver alongside Jelmyto [5][9] - The company is expanding its sales force from approximately 50 to over 80 representatives in preparation for the potential launch of UGN-102 [9][24] - UroGen is committed to advancing its pipeline, including next-generation programs and immuno-oncology initiatives, while maintaining a strong balance sheet [12][29] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming ODAC meeting and the PDUFA target date, highlighting the strength of clinical data supporting UGN-102 [6][14] - The company is focused on ensuring timely access for patients and driving reimbursement confidence among providers [22][24] - Management emphasized the importance of UGN-102 as a transformative therapy for patients with recurrent low-grade intermediate-risk NMIBC [22][24] Other Important Information - The FDA has scheduled an ODAC meeting for UGN-102 on May 21, 2025, which is seen as a critical opportunity to present clinical data [6][10] - UroGen has engaged in extensive market research with physicians, indicating strong interest and demand for UGN-102 [55] Q&A Session Summary Question: Breakdown of Medicare and Medicaid exposure for UGN-102 and Jelmyto - Management anticipates that the Medicare population will comprise about 70% of UGN-102's business, aligning with the patient demographic for NMIBC [34] Question: Preparation for the ODAC meeting and expected pushback - Management has conducted mock ODAC panels and feels confident about presenting the clinical data, particularly addressing the single-arm study design of ENVISION [38][40] Question: Current status of the UGN-103 clinical development program - Enrollment for the UGN-103 trial is ongoing, with completion expected by mid-2025 and top-line data anticipated in 2026 [20][69] Question: Expectations regarding the gross-to-net for UGN-102 - Management expects the gross-to-net profile for UGN-102 to be more favorable than Jelmyto, primarily due to a higher proportion of community-based business [100]

Company Performance - Urogen Pharma reported a quarterly loss of $0.92 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.83, and an improvement from a loss of $0.97 per share a year ago, indicating a surprise of -10.84% [1] - The company posted revenues of $20.25 million for the quarter ended March 2025, missing the Zacks Consensus Estimate by 3.67%, but showing an increase from $18.78 million in the same quarter last year [2] - Over the last four quarters, Urogen Pharma has surpassed consensus EPS estimates only once and has topped consensus revenue estimates just once [2] Stock Outlook - Urogen Pharma shares have declined approximately 2.5% since the beginning of the year, while the S&P 500 has seen a decline of 3.8%, indicating relative outperformance [3] - The current consensus EPS estimate for the upcoming quarter is -$0.85 on revenues of $24.45 million, and for the current fiscal year, it is -$3 on revenues of $120.83 million [7] Industry Context - The Medical - Biomedical and Genetics industry, to which Urogen Pharma belongs, is currently ranked in the top 34% of over 250 Zacks industries, suggesting a favorable outlook compared to lower-ranked industries [8] - Empirical research indicates a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can be tracked by investors [5]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-38079 UROGEN PHARMA LTD. (Exact Name of Registrant as Specified in its Charter) (State or other jurisdiction of incorporation or ...

[Business and Financial Highlights](index=1&type=section&id=Business%20and%20Financial%20Highlights) UroGen is approaching a pivotal period with the anticipated FDA approval of its lead product, UGN-102, in June 2025, targeting a potential $5 billion market for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer - The company is entering a pivotal period with the anticipated FDA approval of its lead pipeline product, UGN-102, in June 2025, representing a significant commercial opportunity with a total addressable market of over **$5 billion**[3](index=3&type=chunk) Q1 2025 Key Highlights | Metric | Value | Note | | :--- | :--- | :--- | | **UGN-102 PDUFA Date** | June 13, 2025 | FDA target action date | | **JELMYTO® Net Sales** | $20.3 million | Q1 2025 | | **JELMYTO® Underlying Demand Growth** | 12% | Year-over-year | | **Cash and Equivalents** | $200.4 million | As of March 31, 2025 | [Pipeline and Product Updates](index=1&type=section&id=Pipeline%20and%20Product%20Updates) UroGen provided significant updates across its portfolio, with UGN-102 on track for its PDUFA date, JELMYTO showing steady demand growth, and the early-stage pipeline expanding with UGN-501 and UGN-301 advancements [UGN-102 (mitomycin) for intravesical solution](index=1&type=section&id=UGN-102%20%28mitomycin%29%20for%20intravesical%20solution) The New Drug Application (NDA) for UGN-102 is under FDA review with a PDUFA target date of June 13, 2025, supported by positive 18-month data from the Phase 3 ENVISION trial - The FDA has set a PDUFA target action date of **June 13, 2025**, for UGN-102 and scheduled an ODAC meeting for **May 21, 2025**[7](index=7&type=chunk) - Updated 18-month data from the ENVISION trial showed the probability of remaining in complete response was **80.6%**, consistent with prior 12-month estimates[7](index=7&type=chunk) - Patient-reported outcomes from three studies (OPTIMA II, ATLAS, ENVISION) showed that UGN-102 did not have a negative impact on symptom burden, patient function, or quality of life[7](index=7&type=chunk) [JELMYTO (mitomycin) for pyelocalyceal solution](index=1&type=section&id=JELMYTO%20%28mitomycin%29%20for%20pyelocalyceal%20solution) JELMYTO generated $20.3 million in net product revenue in Q1 2025, an 8% increase over Q1 2024, driven by 12% underlying demand growth, with long-term data showing a median duration of response of 47.8 months JELMYTO Q1 Performance | Metric | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Net Product Revenue | $20.3M | $18.8M | - Year-over-year revenue growth of **8%** was driven by underlying demand growth of **12%**, partially offset by higher 340B chargebacks[11](index=11&type=chunk) - Long-term follow-up from the JELMYTO Phase 3 study showed a median Duration of Response (DOR) of **47.8 months** for all patients achieving complete response[18](index=18&type=chunk) [Other Pipeline Developments](index=2&type=section&id=Other%20Pipeline%20Developments) UroGen is actively advancing its broader pipeline, with enrollment complete for the Phase 1 study of UGN-301, the acquisition of UGN-501, and expected completion of UGN-103 Phase 3 enrollment by mid-2025 - Enrollment is complete in the Phase 1 study of UGN-301 (zalifrelimab) in high-grade NMIBC, with the investigational treatment demonstrating an acceptable safety profile[8](index=8&type=chunk) - In February 2025, UroGen acquired UGN-501 (ICVB-1042), a next-generation oncolytic virus, from IconOVir Bio to expand its nonclinical oncology portfolio[9](index=9&type=chunk) - Enrollment in the Phase 3 UTOPIA trial of UGN-103 is expected to be completed by **mid-2025**, and a Phase 3 trial for UGN-104 is planned to initiate by **mid-2025**[19](index=19&type=chunk) [Q1 2025 Financial Performance](index=2&type=section&id=First%20quarter%202025%20Financial%20Results) UroGen reported a net loss of $43.84 million for Q1 2025, compared to a $32.29 million loss in Q1 2024, primarily due to higher operating expenses for UGN-102 commercial preparations and R&D increases, ending the quarter with $200.41 million in cash and marketable securities [Key Financial Metrics](index=2&type=section&id=Key%20Financial%20Metrics) In Q1 2025, UroGen's revenue grew to $20.25 million from $18.78 million year-over-year, but increased operating expenses led to a higher net loss of $43.84 million, or ($0.92) per share Condensed Consolidated Statements of Operations (in millions USD, except per share data) | Metric | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Revenue | $20.25M | $18.78M | | Gross Profit | $17.92M | $17.05M | | R&D Expenses | $19.87M | $15.49M | | SG&A Expenses | $34.97M | $27.30M | | Operating Loss | $(36.91)M | $(25.74)M | | Net Loss | $(43.84)M | $(32.29)M | | Net Loss per Share | $(0.92) | $(0.87) | - The **$4.4 million** increase in R&D expenses was primarily driven by the acquisition of UGN-501, higher manufacturing costs, and costs for the Phase 3 UTOPIA trial[12](index=12&type=chunk) - The **$7.7 million** increase in SG&A expenses was primarily driven by UGN-102 commercial preparation activities[13](index=13&type=chunk) [Financial Position](index=2&type=section&id=Financial%20Position) As of March 31, 2025, UroGen held $200.41 million in cash, cash equivalents, and marketable securities, a decrease from $241.71 million at the end of 2024, resulting in a total shareholders' deficit of $46.46 million Selected Consolidated Balance Sheet Data (in millions USD) | Metric | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash, cash equivalents and marketable securities | $200.41M | $241.71M | | Total assets | $247.62M | $285.71M | | Total liabilities | $294.08M | $294.51M | | Total shareholders' deficit | $(46.46)M | $(8.80)M | [2025 Financial Guidance](index=3&type=section&id=2025%20Financial%20Guidance) UroGen reaffirmed its full-year 2025 financial guidance, expecting JELMYTO net product revenues between $94 million and $98 million, and total operating expenses between $215 million and $225 million - Guidance for full-year 2025 net product revenues for JELMYTO remains unchanged at **$94 million to $98 million**[21](index=21&type=chunk) - Full-year 2025 operating expenses are expected to be in the range of **$215 million to $225 million**, including **$11 million to $14 million** in non-cash share-based compensation[21](index=21&type=chunk) [Product and Disease Information](index=3&type=section&id=Product%20and%20Disease%20Information) This section provides background on UroGen's approved product JELMYTO, its lead investigational candidate UGN-102, and the disease they target, Upper Tract Urothelial Cancer (UTUC), all based on the proprietary RTGel® technology [About JELMYTO](index=3&type=section&id=About%20JELMYTO) JELMYTO (mitomycin) is a prescription medicine for adult patients with low-grade Upper Tract Urothelial Cancer (LG-UTUC), utilizing a reverse thermal gel for prolonged chemoablative therapy, with key safety warnings including ureteric obstruction and bone marrow problems - JELMYTO is a mitomycin-containing reverse thermal gel indicated for the treatment of adult patients with LG-UTUC[28](index=28&type=chunk) - Important safety information includes a contraindication for patients with a hole or tear (perforation) of the bladder or upper urinary tract[30](index=30&type=chunk) - Serious side effects may include swelling and narrowing of the ureter (ureteric obstruction) and bone marrow problems causing a decrease in blood cell counts[36](index=36&type=chunk) [About UGN-102](index=4&type=section&id=About%20UGN-102) UGN-102 is an investigational formulation of mitomycin using UroGen's proprietary RTGel® technology, in Phase 3 development for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC), with an FDA PDUFA goal date of June 13, 2025 - UGN-102 is an innovative drug formulation of mitomycin in Phase 3 development for recurrent LG-IR-NMIBC, utilizing UroGen's proprietary RTGel® technology[35](index=35&type=chunk) - The FDA accepted the NDA for UGN-102 and assigned a PDUFA goal date of **June 13, 2025**[37](index=37&type=chunk) [About Upper Tract Urothelial Cancer (UTUC)](index=4&type=section&id=About%20Upper%20Tract%20Urothelial%20Cancer%20%28UTUC%29) Upper Tract Urothelial Cancer (UTUC) accounts for 5-10% of all urothelial cancers, with an estimated 6,000-7,000 new or recurrent low-grade cases annually in the U.S., predominantly affecting patients over 70 years old - UTUC accounts for **five to ten percent** of primary urothelial cancers, with approximately **6,000 - 7,000** new or recurrent LG-UTUC patients annually in the U.S[34](index=34&type=chunk) [Forward-Looking Statements](index=5&type=section&id=Forward-Looking%20Statements) This section provides a standard legal disclaimer regarding forward-looking statements, cautioning that actual results could differ materially from expectations due to numerous risks and uncertainties, including regulatory approvals, clinical trial success, and commercialization challenges - The press release contains forward-looking statements regarding the UGN-102 regulatory timeline, market opportunities, clinical trial progress, and financial guidance[39](index=39&type=chunk) - These statements are subject to significant risks, including uncertain outcomes of the ODAC meeting and FDA review, potential clinical trial delays, commercialization complications, and competition[39](index=39&type=chunk)