UroGen Pharma FY Conference Summary Company Overview - **Company**: UroGen Pharma (NasdaqGM:URGN) - **Industry**: Biotechnology, specifically focused on bladder cancer treatments Key Highlights - **Transformational Year**: UroGen is experiencing a pivotal year with the approval of JELMYTO for bladder cancer, marking a significant milestone since the company's inception [10][11] - **Financial Position**: The company has successfully completed debt refinancing, enhancing its financial stability and flexibility for future growth [60][61] - **Product Launch**: JELMYTO's launch is unique as it involves a procedure rather than a traditional pill or infusion, impacting the treatment timeline for patients [16][17] Financial Performance - **Q4 Results**: UroGen reported Q4 revenues of approximately $14 million, with $4.5 million generated in October alone, indicating strong initial uptake [19] - **Growth Expectations**: The company anticipates accelerated growth following the introduction of a permanent J-code, which is crucial for reimbursement confidence among physicians [17][20] Market Dynamics - **Patient Enrollment**: About 80% to 90% of patients are processed through a hub that manages patient enrollment forms, which are critical for tracking demand [23][25] - **Conversion Rates**: The conversion rate from patient enrollment forms to actual treatments is currently around 70%-80%, with efforts to reduce the time from enrollment to treatment [31][25] Competitive Landscape - **Unique Positioning**: UroGen is the only company offering a treatment for low-grade non-muscle invasive bladder cancer, differentiating itself from competitors [18] - **Market Potential**: The bladder cancer market is estimated to be a $5 billion opportunity, with UroGen aiming to capture a significant share through its innovative treatments [35][36] Pipeline Developments - **UGN-103**: This next-generation formulation is expected to have patent protection until 2042, with clinical data anticipated mid-year [66] - **UGN-501**: An oncolytic virus with potential applications beyond bladder cancer, currently in early development stages [70][71] Strategic Vision - **Long-term Growth**: UroGen aims to build a sustainable growth company by leveraging its unique product offerings and expanding its market presence [10][12] - **Focus on Education**: The company is committed to educating physicians on the benefits of its treatments to drive adoption and increase patient access [35][46] Undervalued Aspects - **Market Valuation**: The CEO highlighted that the market valuation does not fully reflect the potential of ZUSDURI, which could represent a $1.2 billion opportunity if the company captures 20% of the patient population [76] Conclusion - UroGen Pharma is positioned for significant growth in the bladder cancer treatment market, with a strong financial foundation, innovative products, and a strategic focus on education and market penetration. The upcoming quarters will be critical in determining the trajectory of its growth and market acceptance.

Financial Data and Key Metrics Changes - Total revenues for the year ended December 31, 2025, were $109.8 million, a 21% increase from $90.4 million in 2024, driven by the commercial launch of ZUSDURI and increased sales of JELMYTO [19][20] - The net loss for the year was $153.5 million, or $3.19 per share, compared to a net loss of $126.9 million, or $2.96 per share in 2024 [21] - Cash equivalents and marketable securities totaled $120.5 million as of December 31, 2025 [21] Business Line Data and Key Metrics Changes - ZUSDURI generated $15.8 million in revenue for 2025, reflecting early launch dynamics [5][14] - JELMYTO generated net product revenue of $94 million for the full year 2025, indicating continued underlying demand growth [7][17] Market Data and Key Metrics Changes - As of December 31, 2025, there were 838 activated sites of care with 102 unique prescribers and 32 repeat prescribers for ZUSDURI [15] - Over 95% of covered lives had open access to ZUSDURI by year-end 2025 [15] Company Strategy and Development Direction - The company’s top priority is the commercial launch of ZUSDURI, which addresses a large and underserved market, with potential peak revenue exceeding $1 billion [5][7] - The company is advancing its pipeline with UGN-103 and UGN-104, with plans to submit an NDA for UGN-103 in the second half of 2026 [8][12] - A refinancing of the debt facility with Pharmakon Advisors has strengthened the balance sheet and enhanced financial flexibility [8][22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the early trajectory of ZUSDURI's launch and noted that the permanent J-code has facilitated more predictable patient access [5][6] - The company anticipates that the conversion timeline from patient enrollment forms to dosing will narrow as sites gain familiarity with the product [16] - Management is optimistic about the potential for ZUSDURI and its pipeline products to create long-term value for patients and shareholders [9][23] Other Important Information - The company expects JELMYTO net product revenues for 2026 to be in the range of $97 million to $101 million, reflecting a year-over-year growth rate of approximately 3% to 7% [23] - Full-year 2026 operating expenses are expected to be in the range of $240 million to $250 million, driven by increased sales force expansion and lifecycle management plans [24] Q&A Session Summary Question: Insights on patient enrollment forms and potential guidance for ZUSDURI - Management indicated that they would consider providing formal guidance for ZUSDURI after gaining visibility into steady-state demand, approximately two quarters post the permanent J-code [29] - The number of patient enrollment forms has increased since the permanent J-code became effective, with new and repeat prescribers contributing to this growth [31][32] Question: Trends in repeat prescribers and their likelihood of becoming repeat prescribers - There is steady growth in both new and repeat prescribers, with positive experiences leading to increased confidence in reimbursement and repeat prescribing [39] Question: Timing for UGN-103 market introduction and its commercial positioning - UGN-103 is expected to be introduced after obtaining a permanent J-code, with a potential launch in early 2028 [41] Question: Current use of ZUSDURI among patients and expected shifts in use patterns - ZUSDURI is being used primarily for patients who recur early, have frequent recurrences, or are unfit for surgery, with expectations for increased utilization as community practices gain experience [48][49] Question: Opportunities for ZUSDURI in the adjuvant setting - There is enthusiasm for expanding UGN-103 into the adjuvant setting for new diagnoses, with ongoing discussions about potential combinations with other agents [60][61]

Core Insights - UroGen Pharma Ltd. reported a transformative year in 2025, marked by the FDA approval and launch of ZUSDURI, the first approved treatment for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer, with expectations of over $1 billion in peak sales opportunity [2][5][10] Financial Performance - Total revenue for 2025 was $109.8 million, a 21% increase from $90.4 million in 2024, primarily driven by ZUSDURI's launch and growth in JELMYTO sales [10] - JELMYTO generated net product sales of $94.0 million in 2025, reflecting a 7% year-over-year growth [4][5] - UroGen reported a net loss of $153.5 million for 2025, compared to a net loss of $126.9 million in 2024, with a loss per share of $3.19 [16][22] Product Developments - ZUSDURI achieved net sales of $15.8 million in its initial launch period in 2025, with a permanent J Code effective January 1, 2026, enhancing reimbursement processes [5][6] - The UTOPIA trial for UGN-103 showed a 77.8% complete response rate, with plans to submit a New Drug Application (NDA) in the second half of 2026 [12] - UroGen is advancing UGN-501, an investigational oncolytic virus therapy, with plans for an IND submission and Phase 1 trial initiation by the end of 2026 [8] Strategic Initiatives - The company refinanced its term loan with Pharmakon Advisors, securing $200 million to strengthen its balance sheet and support long-term growth strategies [9] - UroGen plans to explore label expansion opportunities for UGN-103 and continue evaluating lifecycle management strategies [12] Market Outlook - Guidance for 2026 JELMYTO sales is projected between $97 million and $101 million, indicating a growth rate of approximately 3% to 7% over 2025 [17] - The company is not providing full-year sales guidance for ZUSDURI at this time due to its early commercial stage [17]

ZUSDURI (formerly UGN-102) Approved by FDA! ZUSDURI is the first and only FDA-approved medication for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer Transforming Urothelial Cancer Care Through Innovation March 2026 Disclaimers This investor presentation contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation: the estimated addressable patient population and market and revenue opportunity for ...

Core Insights - UroGen Pharma Ltd. (NASDAQ:URGN) is recognized as a promising small-cap biotech stock by analysts [1] Financial Performance - For Q3 2025, UroGen Pharma reported a net loss of $33.3 million, an increase from $23.7 million in Q3 2024, primarily due to rising R&D expenses and delayed revenue recognition [4] - The company ended the quarter with $127.4 million in cash and marketable securities [2] Revenue Growth - Preliminary figures for October indicate that demand revenue more than doubled compared to the previous three months, reflecting increased physician adoption despite operational delays [2] - UroGen provided revenue guidance for its product JELMYTO, expecting net revenues between $94 million and $98 million for the full year [4] Market Position and Strategy - The company is expanding its sales force to 82 representatives to enhance physician outreach and patient access, achieving coverage for over 95% of insured lives [3] - With the implementation of a permanent billing code in early 2026, UroGen anticipates a smoother reimbursement process, although administrative lags of up to 60 days may still occur [3] Product Focus - UroGen Pharma specializes in developing and marketing specialty cancer treatments, offering targeted and minimally invasive therapies [5]

Core Viewpoint - Small-cap biotech stocks are experiencing a resurgence, driven by market rotation and improving technical indicators, with healthcare emerging as a constructive sector [2][4] Industry Overview - The Health Care Select Sector SPDR Fund (XLV) has increased by 11.09% in 2025, while the iShares Biotechnology ETF has gained approximately 25% year-to-date [2][3] - Analysts suggest that capital is flowing out of high-flying AI stocks into more attractive healthcare and biotech opportunities [3][4] M&A Activity - Increased mergers and acquisitions (M&A) activity in the biotech sector has been noted, with significant deals including Pfizer's $10 billion acquisition of Metsera and Roche's $3.5 billion acquisition of 89bio [4][5] - This M&A activity is expected to bolster potential upside for both large-cap and small-to-mid-cap biotech companies [4] Methodology for Stock Selection - The list of the best small-cap biotech stocks was curated based on companies with market capitalizations between $300 million and $2 billion, focusing on those with the highest upside potential as of November 10, 2025 [8] Company Highlights - **Viridian Therapeutics, Inc. (NASDAQ:VRDN)**: - Upside potential of 36.71% with a cash position of $888 million and key patient enrollments in late-stage clinical studies [10][11][12] - **UroGen Pharma Ltd. (NASDAQ:URGN)**: - Upside potential of 49.17%, reporting a significant increase in demand revenue and a cash position of $127.4 million [14][15][16] - **Liquidia Corporation (NASDAQ:LQDA)**: - Upside potential of 55.49%, with YUTREPIA achieving profitability earlier than expected and a cash position of $157.5 million [19][20][22]

Core Insights - UroGen Pharma Ltd. reported financial results for Q3 2025, highlighting the launch momentum of ZUSDURI, the first FDA-approved treatment for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer [1][2] - The company expressed optimism about the commercial potential of ZUSDURI, supported by strong physician engagement and broad reimbursement coverage [2][5] Financial Performance - Total revenues for Q3 2025 were $27.5 million, with JELMYTO generating $25.7 million, reflecting a year-over-year growth of approximately 13% [11][7] - ZUSDURI achieved net product revenue of $1.8 million in its first quarter, with preliminary estimates for October 2025 indicating $4.5 million, suggesting accelerating growth [11][6] - As of September 30, 2025, UroGen had $127.4 million in cash, cash equivalents, and marketable securities [17] Product Developments - ZUSDURI received a unique J-Code (J9282) effective January 1, 2026, enhancing patient access through various insurance programs [6] - The Phase 3 UTOPIA trial for UGN-103 reported a complete response rate of 77.8%, with plans to submit an NDA in the second half of 2026 [12][5] - UroGen discontinued the development of UGN-301 due to not meeting internal benchmarks, while continuing with UGN-103 and UGN-104 [12] Research and Development - R&D expenses for Q3 2025 were $14.0 million, driven by costs associated with the UTOPIA trial [13] - The company is focused on next-generation formulations to improve manufacturing efficiencies and extend product lifecycles [2][12] Operational Highlights - UroGen activated 592 sites of care and had 54 unique prescribers for ZUSDURI since its launch [6] - The company anticipates full-year 2025 net product revenues for JELMYTO to be between $94 million and $98 million, indicating an 8% to 12% growth from 2024 [18]

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].

Financial Data and Key Metrics Changes - UroGen Pharma reported net product revenues of $90.4 million for the full year 2024, an increase from $82.7 million in 2023, representing a year-over-year growth of approximately 9.3% [17][44] - The net loss for the fourth quarter of 2024 was $37.5 million, or $0.80 per share, compared to a net loss of $26 million, or $0.72 per share, in the same period in 2023 [47] - For the full year 2024, the net loss was $126.9 million, or $2.96 per share, compared to a net loss of $102.2 million, or $3.55 per share, in 2023 [47] Business Line Data and Key Metrics Changes - JELMYTO generated net product revenues of $24.6 million in Q4 2024, up from $23.5 million in Q4 2023, with underlying demand revenue increasing by 15% [44] - The underlying demand revenue for JELMYTO for the full year 2024 was $87.4 million, reflecting a 12% increase compared to the previous year [39][44] - The company reported a compelling complete response rate of 79.6% at three months and an 82.3% duration of response at 12 months for UGN-102, with updated data showing an 80.6% duration of response at 18 months [9][10] Market Data and Key Metrics Changes - The total addressable market for UGN-102 is estimated to be over $5 billion, significantly larger than the market for JELMYTO [15] - The anticipated launch of UGN-102 is expected to transform UroGen's market position, moving from a rare disease-focused company to a multiproduct specialty-driven team [34] Company Strategy and Development Direction - UroGen aims to enhance its commercial infrastructure by expanding its sales force from 52 to approximately 83 representatives to support the launch of UGN-102 [37][100] - The company is focused on raising awareness of unmet needs in low-grade intermediate risk non-muscle invasive bladder cancer through educational programs and scientific exchanges [35][36] - UroGen has acquired ICVB-1042, a next-generation investigational oncolytic virus, to enhance its pipeline and address critical gaps in cancer care [19][20] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strength of the data package submitted for UGN-102 and its potential to address significant unmet needs in the bladder cancer treatment landscape [14] - The company anticipates a transformative year in 2025 with the expected launch of UGN-102, which is projected to generate over $1 billion in revenue [54][109] - Management highlighted the importance of preparing for reimbursement processes and ensuring smooth integration of UGN-102 into treatment protocols [38][66] Other Important Information - UroGen ended 2024 with a strong balance sheet, holding $241.7 million in cash, cash equivalents, and marketable securities [21][43] - The company expects full-year 2025 JELMYTO revenues to be in the range of $94 million to $98 million, indicating a growth rate of approximately 8% to 12% [48] Q&A Session Summary Question: Can UGN-102 be used off-label for patients ineligible for surgery? - Management indicated that using UGN-102 in this context would be considered off-label, and it would be up to physicians to work with insurance companies for reimbursement [53][54] Question: How should investors think about the initial launch trajectory of UGN-102? - The launch trajectory is expected to be similar to JELMYTO, with a larger patient population leading to higher absolute numbers [61][72] Question: What are the expected differences in reimbursement levels with the J-code transition? - Initially, a miscellaneous J-code will be used, which may result in longer reimbursement times, but this will improve with a unique permanent J-code expected in January 2026 [66][68] Question: How will UGN-102 be positioned relative to UGN-103? - UGN-102 is expected to be phased out after UGN-103 is approved, with a focus on ensuring no automatic substitution occurs [94][95] Question: What is the rationale behind increasing the sales force size? - The increase is based on patient epidemiology, ensuring coverage of approximately 85% of the market for low-grade intermediate risk NMIBC [102][104]