[Overview and Business Highlights](index=1&type=section&id=Overview%20and%20Business%20Highlights) UroGen Pharma achieved a transformative milestone with ZUSDURI™ FDA approval and launch, complementing JELMYTO®'s continued growth and advancing its pipeline [Executive Summary](index=1&type=section&id=Executive%20Summary) UroGen Pharma's Q2 2025 results highlight the transformative FDA approval and commercial launch of ZUSDURI™, establishing the company as a multi-product uro-oncology entity alongside JELMYTO®'s continued growth - The FDA approval of ZUSDURI™ for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) is a **transformative milestone**, establishing UroGen as a multi-product company[3](index=3&type=chunk)[8](index=8&type=chunk) - The company is executing the commercial launch of ZUSDURI™ to address an estimated market opportunity of **over $5 billion**[3](index=3&type=chunk) Q2 2025 Key Metrics Snapshot | Metric | Value | Growth (YoY) | | :--- | :--- | :--- | | JELMYTO® Net Sales | $24.2 million | 11% | | Cash & Marketable Securities | $161.6 million | N/A | [Product Portfolio and Pipeline Update](index=1&type=section&id=Product%20Portfolio%20and%20Pipeline%20Update) UroGen's commercial portfolio expanded with ZUSDURI™'s launch, complementing JELMYTO®'s growth, while the pipeline advances with next-generation formulations and an immuno-oncology candidate [ZUSDURI™ (mitomycin) for intravesical solution](index=1&type=section&id=ZUSDURI%E2%84%A2%20(mitomycin)%20for%20intravesical%20solution) ZUSDURI™ received FDA approval as the first and only medication for recurrent LG-IR-NMIBC, supported by strong Phase 3 and Phase 2b clinical data - On June 12, 2025, ZUSDURI™ (formerly UGN-102) was approved by the FDA, becoming the **first and only approved medication** for adults with recurrent LG-IR-NMIBC[8](index=8&type=chunk) - Updated data from the Phase 3 ENVISION trial demonstrated a **72.2% probability** of patients remaining in complete response at 24 months[8](index=8&type=chunk) - Five-year results from the Phase 2b OPTIMA II trial showed a median duration of response of **approximately 3.5 years**[8](index=8&type=chunk) [JELMYTO® (mitomycin) for pyelocalyceal solution](index=1&type=section&id=JELMYTO%C2%AE%20(mitomycin)%20for%20pyelocalyceal%20solution) JELMYTO® demonstrated 11% year-over-year revenue growth in Q2 2025, driven by increased demand and price favorability, with ongoing real-world data collection JELMYTO® Q2 2025 Performance | Metric | Q2 2025 | Q2 2024 | YoY Growth | | :--- | :--- | :--- | :--- | | Net Product Revenue | $24.2 million | $21.8 million | 11% | - The **11% year-over-year revenue growth** was driven by a **7% increase in underlying demand** and price favorability[8](index=8&type=chunk)[11](index=11&type=chunk) - The uTRACT Registry, a real-world study of JELMYTO®, is ongoing to provide insights into treatment patterns, long-term outcomes, and patient safety[8](index=8&type=chunk) [Pipeline Development](index=1&type=section&id=Pipeline%20Development) UroGen's pipeline advanced with completed enrollment for UGN-103's Phase 3 trial, initiation of UGN-104's Phase 3 study, and completed dose escalation for UGN-301 - **UGN-103 (Next-gen ZUSDURI):** Enrollment is **complete** in the Phase 3 UTOPIA clinical trial for patients with recurrent LG-IR-NMIBC[6](index=6&type=chunk) - **UGN-104 (Next-gen JELMYTO):** A Phase 3 study to evaluate the safety and efficacy of this next-generation product for LG-UTUC has been **initiated**[9](index=9&type=chunk) - **UGN-301 (zalifrelimab):** Enrollment is **complete** in the dose escalation phase for this anti-CTLA4 antibody, showing an acceptable safety profile and observed responses[10](index=10&type=chunk) [Financial Performance](index=2&type=section&id=Financial%20Performance) UroGen's Q2 2025 financial performance reflects increased net loss due to ZUSDURI™ launch preparations and R&D investments, alongside JELMYTO® revenue growth and reaffirmed full-year guidance [Second Quarter 2025 Financial Results](index=2&type=section&id=Second%20Quarter%202025%20Financial%20Results) UroGen reported a wider Q2 2025 net loss of $49.9 million, primarily driven by increased SG&A for ZUSDURI™ launch and higher R&D expenses, despite JELMYTO® revenue growth Q2 2025 Financial Summary (vs. Q2 2024) | Metric | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | JELMYTO Revenue | $24.2 million | $21.8 million | | R&D Expenses | $18.9 million | $15.4 million | | SG&A Expenses | $43.2 million | $30.1 million | | Net Loss | $49.9 million | $33.4 million | | Net Loss per Share | ($1.05) | ($0.82) | | Cash & Equivalents (End of Period) | $161.6 million | N/A | - The increase in SG&A expenses was primarily driven by **commercial preparation activities for the ZUSDURI™ launch**[13](index=13&type=chunk) - Higher R&D expenses were mainly due to **increased manufacturing costs** and costs associated with the Phase 3 UTOPIA trial for UGN-103[12](index=12&type=chunk) [Financial Statements](index=2&type=section&id=Financial%20Statements) The condensed consolidated financial statements present detailed financial position and operational results for the three and six months ended June 30, 2025, compared to 2024 [Condensed Consolidated Statements of Operations](index=3&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) This statement details the company's revenues, expenses, and net loss for the three and six months ended June 30, 2025, and 2024 Condensed Consolidated Statements of Operations (in thousands, except per share data) | | Three months ended June 30, | Six months ended June 30, | | :--- | :--- | :--- | | | **2025** | **2024** | **2025** | **2024** | | **Revenue** | **$ 24,215** | **$ 21,848** | **$ 44,469** | **$ 40,629** | | Gross profit | 20,665 | 19,619 | 38,589 | 36,672 | | Total operating expenses | 62,113 | 45,458 | 116,951 | 88,251 | | **Operating loss** | **(41,448)** | **(25,839)** | **(78,362)** | **(51,579)** | | **Net loss** | **$ (49,940)** | **$ (33,403)** | **$ (93,783)** | **$ (65,689)** | | **Net loss per share** | **$ (1.05)** | **$ (0.82)** | **$ (1.97)** | **$ (1.69)** | [Selected Consolidated Balance Sheets](index=2&type=section&id=Selected%20Consolidated%20Balance%20Sheets) This statement provides a snapshot of the company's assets, liabilities, and equity as of June 30, 2025, and December 31, 2024 Selected Consolidated Balance Sheet Data (in thousands) | | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash, cash equivalents and marketable securities | $ 161,586 | $ 241,707 | | Total assets | $ 208,717 | $ 285,711 | | Total liabilities | $ 302,093 | $ 294,514 | [Full-Year 2025 Guidance](index=2&type=section&id=Full-Year%202025%20Guidance) UroGen reaffirmed its full-year 2025 guidance, projecting JELMYTO® net product revenues between $94 million and $98 million and total operating expenses from $215 million to $225 million Full-Year 2025 Financial Guidance | Metric | Guidance Range | | :--- | :--- | | JELMYTO Net Product Revenues | $94 - $98 million | | Total Operating Expenses | $215 - $225 million | | Non-cash Share-based Compensation | $11 - $14 million | - The revenue guidance implies an **8% to 12% year-over-year growth rate** for JELMYTO® over 2024 demand-driven sales[19](index=19&type=chunk) [Product and Company Information](index=3&type=section&id=Product%20and%20Company%20Information) This section provides detailed information on UroGen's commercial products, ZUSDURI™ and JELMYTO®, including their uses, mechanisms, and safety profiles, along with company overview [About ZUSDURI™](index=3&type=section&id=About%20ZUSDURI%E2%84%A2) ZUSDURI™ is an FDA-approved, mitomycin-based intravesical solution utilizing RTGel® technology for recurrent LG-IR-NMIBC, with detailed information on its use and safety - ZUSDURI™ is a prescription medicine for adults with recurrent LG-IR-NMIBC whose previous bladder surgery was not effective[25](index=25&type=chunk) - It utilizes UroGen's proprietary **RTGel® technology**, a sustained-release hydrogel, to enable non-surgical treatment of tumors in an outpatient setting[24](index=24&type=chunk) - Important safety information includes contraindication for patients with a perforated bladder and the need for effective contraception for both females and males during and after treatment[26](index=26&type=chunk)[27](index=27&type=chunk)[29](index=29&type=chunk) [About JELMYTO®](index=4&type=section&id=About%20JELMYTO%C2%AE) JELMYTO® is a mitomycin-containing reverse thermal gel approved for LG-UTUC, with details on its mechanism, approved use, and important safety information - JELMYTO® is a prescription medicine for the treatment of adult patients with low-grade Upper Tract Urothelial Cancer (LG-UTUC)[34](index=34&type=chunk) - Its **reverse thermal gel technology** becomes a semi-solid at body temperature, allowing the drug to remain in the collecting system for **four to six hours** for sustained chemoablative therapy[33](index=33&type=chunk) - Serious side effects can include swelling and narrowing of the ureter (ureteric obstruction) and bone marrow problems leading to decreased blood cell counts[44](index=44&type=chunk) [About UroGen Pharma Ltd.](index=5&type=section&id=About%20UroGen%20Pharma%20Ltd.) UroGen is a biotech company focused on developing and commercializing innovative solutions for urothelial and specialty cancers using its proprietary RTGel® platform, with two approved products - UroGen is a biotech company dedicated to developing and commercializing innovative solutions for urothelial and specialty cancers[42](index=42&type=chunk) - The company's proprietary **RTGel® platform** is a sustained-release, hydrogel-based technology designed to improve the therapeutic profiles of existing drugs for local therapy[42](index=42&type=chunk) [Other Information](index=2&type=section&id=Other%20Information) This section provides details on the conference call for Q2 results and includes cautionary forward-looking statements regarding potential risks and uncertainties [Conference Call & Webcast Information](index=2&type=section&id=Conference%20Call%20%26%20Webcast%20Information) UroGen management hosted a conference call and webcast on August 7, 2025, to discuss financial results and provide a business update - A live conference call and webcast were held on the day of the earnings release to review financial results and provide a general business update[20](index=20&type=chunk) [Forward-Looking Statements](index=5&type=section&id=Forward-Looking%20Statements) This section contains cautionary forward-looking statements, highlighting various risks and uncertainties that could cause actual results to differ materially from projections - The press release includes forward-looking statements regarding the market opportunity for ZUSDURI, pipeline progress, financial guidance, and the potential of its RTGel technology[43](index=43&type=chunk) - These statements are subject to numerous risks and uncertainties, including clinical results, regulatory approval, commercialization challenges, competition, and market acceptance[43](index=43&type=chunk)

Core Insights - UroGen Pharma has achieved a significant milestone with the FDA approval of ZUSDURI for treating adults with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC), marking its transition into a multi-product uro-oncology company [2][7] - The company is targeting a market opportunity exceeding $5 billion and is actively executing the launch plan for ZUSDURI while continuing to grow its existing product, JELMYTO [2][6] - UroGen's financial results for Q2 2025 show a net product revenue of $24.2 million for JELMYTO, reflecting an 11% year-over-year growth driven by underlying demand and price favorability [6][10] Financial Performance - JELMYTO net product revenues for Q2 2025 were $24.2 million, up from $21.8 million in Q2 2024, indicating an 11% increase [10] - Research and Development (R&D) expenses for Q2 2025 were $18.9 million, an increase from $15.4 million in Q2 2024, primarily due to higher manufacturing costs and expenses related to the Phase 3 UTOPIA trial [11] - Selling, General and Administrative (SG&A) expenses rose to $43.2 million in Q2 2025 from $30.1 million in Q2 2024, driven by commercial preparation activities for ZUSDURI [12] - The company reported a net loss of $49.9 million in Q2 2025, compared to a net loss of $33.4 million in the same period in 2024 [15][20] Product Development and Pipeline - Enrollment is complete for the Phase 3 UTOPIA trial of UGN-103, a next-generation mitomycin formulation aimed at improving treatment for recurrent LG-IR-NMIBC [5][8] - UroGen is also advancing its immuno-oncology candidates, with ongoing studies for UGN-301, an anti-CTLA4 antibody for high-grade non-muscle invasive bladder cancer [9] - The company is collecting real-world data through the uTRACT Registry to evaluate JELMYTO's use and outcomes in adult patients with upper tract urothelial carcinoma [7] Market Outlook - UroGen maintains its guidance for full-year 2025 net product revenues for JELMYTO in the range of $94 to $98 million, reflecting an expected growth rate of approximately 8% to 12% over 2024 [16] - The company is well-positioned to support the launch of ZUSDURI and advance its strategic initiatives for sustained growth and innovation [2][6]

Core Insights - UroGen Pharma announced a 24-month Duration of Response (DOR) of 72.2% for ZUSDURI in patients who achieved a complete response at three months from the Phase 3 ENVISION trial, indicating sustained efficacy for treating recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) [1][2][3] Company Overview - UroGen Pharma is focused on developing innovative solutions for urothelial and specialty cancers, with ZUSDURI being a significant product for treating recurrent LG-IR-NMIBC [7] - The company utilizes proprietary RTGel technology, which allows for sustained release of medication directly into the bladder, providing a non-surgical treatment option [4][7] Product Details - ZUSDURI (mitomycin) is an FDA-approved intravesical solution for adults with recurrent LG-IR-NMIBC, marking a transformative step in managing this chronic cancer [2][3][8] - The treatment is administered in an outpatient setting, which can improve patient quality of life compared to traditional surgical options [2][4] Clinical Trial Insights - The Phase 3 ENVISION trial involved 240 patients and evaluated the efficacy and safety of ZUSDURI, with a primary endpoint of complete response rate at three months [6] - The trial demonstrated that the event rate remained stable over time for patients who achieved a complete response, reinforcing the therapy's potential as a durable treatment option [2][3] Market Context - LG-IR-NMIBC affects approximately 82,000 individuals annually in the U.S., with around 59,000 experiencing recurrence, highlighting the need for effective treatment options [5] - The existing standard of care involves repeated surgical procedures, which can negatively impact patients' health and quality of life [2][5]

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 t ...

Core Viewpoint - A class action lawsuit has been filed against UroGen Pharma Ltd. for alleged misleading statements regarding its lead product UGN-102, which is intended for treating low-grade intermediate risk non-muscle invasive bladder cancer [1][3]. Company Overview - UroGen Pharma is engaged in the development and commercialization of solutions for specialty cancers, with its lead pipeline product being UGN-102, an intravesical solution for bladder cancer treatment [2]. Allegations in the Lawsuit - The lawsuit claims that UroGen made false statements and failed to disclose critical information about the ENVISION clinical study for UGN-102, including the lack of a concurrent control arm, which undermined the study's ability to demonstrate effectiveness [3]. - It is alleged that UroGen ignored FDA warnings regarding the study design for the new drug application (NDA) for UGN-102, leading to a substantial risk of NDA rejection [3][4]. Impact of FDA Findings - On May 16, 2025, the FDA released a briefing document indicating concerns about the ENVISION study's design, which contributed to a nearly 26% drop in UroGen's stock price [4]. - Following the Oncologic Drugs Advisory Committee's vote against the approval of UGN-102 on May 21, 2025, UroGen's stock price fell nearly 45% [5].

Core Insights - UroGen Pharma Ltd. announced positive results from a Phase 3b study on the home administration of ZUSDURI™ (mitomycin) for treating recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) [1][2] - The study demonstrated that trained home health professionals can safely administer ZUSDURI outside traditional clinical settings, potentially easing patient burden and reducing reliance on hospital resources [2][3] Study Findings - The study involved eight patients, with 75% completing all six scheduled treatments; 75% of patients reported a complete response rate at three months [2][4] - Patients expressed high satisfaction with the home-based treatment, with five out of six recommending it to others [2] - No new safety concerns were identified, and the safety profile was consistent with previous studies, primarily involving mild-to-moderate urinary symptoms [3][4] Treatment Administration - ZUSDURI was administered via urinary catheter once weekly for six weeks, with the first dose given in a clinic and subsequent doses at home [3] - Investigators found no significant differences in safety or efficacy between home and office administration [3] Company Overview - UroGen Pharma is focused on developing innovative treatments for urothelial and specialty cancers, utilizing proprietary RTGel technology for sustained drug release [5][8] - ZUSDURI is specifically approved for treating adults with recurrent LG-IR-NMIBC after unsuccessful bladder surgery [9] Market Context - Approximately 82,000 people in the U.S. are diagnosed with NMIBC annually, with around 59,000 experiencing recurrence [6] - The median age of diagnosis for bladder cancer is 73 years, highlighting the need for convenient treatment options for older patients [6]

Core Viewpoint - A class action lawsuit has been filed against UroGen Pharma Ltd. for alleged misleading statements regarding its lead product UGN-102, which is intended for treating low-grade intermediate risk non-muscle invasive bladder cancer [1][2]. Group 1: Lawsuit Details - The lawsuit covers investors who acquired UroGen securities between July 27, 2023, and May 15, 2025, with a deadline of July 28, 2025, to apply as lead plaintiff [1]. - Allegations include that UroGen's ENVISION clinical study for UGN-102 was inadequately designed, lacking a concurrent control arm, which undermined the evidence of effectiveness [3]. - The FDA had previously warned UroGen about the study design, suggesting a randomized trial, which UroGen did not conduct [4]. Group 2: Stock Price Impact - Following the FDA's briefing document on May 16, 2025, which raised concerns about the study's interpretability, UroGen's stock price fell nearly 26% [4]. - On May 21, 2025, after the Oncologic Drugs Advisory Committee voted against the approval of UGN-102, the stock price dropped nearly 45% [5].

Core Points - A shareholder class action lawsuit has been filed against UroGen Pharma Ltd. alleging that the company made materially false and misleading statements regarding its business and operations [1] - The lawsuit claims that the ENVISION clinical study was inadequately designed, lacking a concurrent control arm, which raises concerns about the effectiveness of UGN-102 [1] - It is alleged that UroGen ignored FDA warnings about the study design, leading to a substantial risk that the New Drug Application (NDA) for UGN-102 would not be approved [1] - The lawsuit asserts that the positive statements made by the company regarding its business and prospects were misleading and lacked a reasonable basis [1] Legal Context - Shareholders who purchased UroGen shares between July 27, 2023, and May 15, 2025, and suffered significant losses are encouraged to discuss their legal rights [2] - The deadline for shareholders to request to be appointed as lead plaintiff in the case is July 28, 2025 [3] - Holzer & Holzer, LLC is representing the shareholders and has a history of recovering substantial amounts for investors affected by corporate misconduct [3]

Core Insights - Investing in biotechnology stocks involves long periods of inactivity followed by sharp movements, particularly in small-cap biotech stocks which often lack profitability and revenue [1][2] Group 1: Urogen Pharma - UroGen Pharma is focused on treating urologic cancers with its proprietary RTGel technology, enhancing drug efficacy by prolonging retention in the urinary tract [4] - UroGen stock has increased approximately 43% in 2025 due to an application to expand the Jelymyto label for low-grade bladder cancer, potentially increasing its market [5] - Analyst forecasts suggest a price target of $32.86 for URGN stock, indicating a potential gain of over 115% from its closing price on July 18, although short interest is high at over 42% [6] Group 2: Nektar Therapeutics - Nektar Therapeutics has seen a 78% gain in 2025 but remains down over 93% in the last five years due to several clinical trial failures [8] - Positive Phase 2b clinical trial results for a candidate treating systemic lupus erythematosus and ulcerative colitis have generated bullish sentiment, along with a Fast Track designation from the FDA [9] - The consensus price target for NKTR stock is $88.33, representing a 254% increase from its closing price on July 18, but a pullback is anticipated due to recent price surges [10] Group 3: Verve Therapeutics - Verve Therapeutics is pioneering gene editing to treat cardiovascular disease, currently in the clinical stage with revenue primarily from partnerships [12] - The company received approval for a Phase 1b U.S. clinical trial for its lead candidate, VERVE-102, targeting the PCSK9 gene associated with high cholesterol [13] - Analysts have set a price target of $14.57 for VERV stock, indicating a 33% upside, although this is lower compared to the other stocks mentioned [13]

Core Insights - UroGen Pharma Ltd. announced the publication of a five-year long-term extension study of the Phase 2b OPTIMA II trial, demonstrating durable long-term complete responses in patients with low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) treated with ZUSDURI (mitomycin) [1][2] Company Overview - UroGen Pharma is a biotech company focused on developing innovative solutions for urothelial and specialty cancers, utilizing proprietary RTGel technology for sustained drug release [10] - ZUSDURI is specifically indicated for the treatment of adults with recurrent LG-IR-NMIBC, providing a non-surgical treatment option [5][11] Clinical Study Findings - The OPTIMA II trial included both newly diagnosed and recurrent adult patients with LG-IR-NMIBC, with 41 patients achieving complete response (CR) at three months post-treatment; 25 remained in CR at 12 months [2][4] - Among the 17 patients who entered the long-term follow-up study, the median duration of response (DOR) was 42.1 months, indicating ZUSDURI's potential for meaningful, lasting event-free periods [2][7] - The median Kaplan-Meier estimate of DOR for all 41 patients was 24.2 months, with a median follow-up of 35.8 months [2] Patient Demographics - The study cohort consisted of 17 patients, of which 16 (94%) were recurrent cases, highlighting the drug's relevance for patients facing repeated surgeries [4] Treatment Efficacy - ZUSDURI's long-term data emphasizes its ability to deliver sustained responses in an outpatient setting, which is particularly valuable for recurrent patients [2][3] - The study results reflect a commitment to providing innovative treatments that empower patients and physicians with more options [3] Safety Profile - Safety data were not collected during the long-term follow-up trial; however, the most commonly reported treatment-emergent adverse events in the parent OPTIMA II study included dysuria (41%), pollakiuria (21%), and hematuria (16%) [3][4]