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]