UroGen Pharma FY Conference Summary Company Overview - UroGen Pharma focuses on delivering local medicine to the urothelium, addressing unmet needs in treating urothelial cancers with its innovative RT Gel technology, which transforms from liquid to gel at body temperature [6][7] Key Product: ZUSDURI - ZUSDURI is the first FDA-approved non-surgical option for intermediate-risk non-muscle-invasive bladder cancer (NMIBC) [8] - The product addresses a significant unmet need in treating low-grade, intermediate-risk NMIBC, which has a high recurrence rate [9][10] - Historical treatment involved TURBT procedures, which are invasive and not comprehensive [10][11] Commercial Launch Insights - The commercialization of ZUSDURI is complex, akin to a drug-device combination, requiring operational logistics and physician education [12][13] - The transition from patient enrollment to actual dosing can take up to 60 days, presenting a challenge in revenue recognition [15][17] - Initial sales figures indicated a slower start, but there is optimism about future growth as logistical hurdles are addressed [23][25] Reimbursement Dynamics - Revenue is recognized upon shipment, with a just-in-time inventory model [17][18] - The introduction of a permanent J-code in January is expected to enhance reimbursement confidence among physicians [19][21] - A service warranty is offered to assist physicians with reimbursement processes, although actual usage is anticipated to be low due to positive reimbursement experiences [20] Market Expectations - Approximately 70% of physicians are reportedly waiting for the J-code before using ZUSDURI [21] - Anticipated revenue growth post-J-code is expected to mirror previous experiences with similar products, with a projected 220% increase in revenue within six months [26][27] Competitive Landscape - The entry of competitors into the intermediate-risk space is viewed positively, as it can drive innovation and provide more options for patients [33][34] - UroGen's treatment offers a significant advantage with an 80% complete response rate without the need for surgery, compared to competitors that require surgical intervention [35] Future Developments - UGN-103 and UGN-104 are next-generation formulations with patent protection until 2041, aimed at replacing UGN-102 post-J-code [40][42] - The company is confident in its ability to transition from UGN-102 to UGN-103 without regulatory issues [48][50] Long-term Outlook - The company anticipates continued growth for Jelmyto, its first approved product, as awareness increases with the launch of UGN-102 [51] - 2026 is expected to be a pivotal year for UroGen, with the first two quarters crucial for demonstrating market potential [53]

Group 1 - Brendan, a Pennsylvanian, completed a Ph.D. in organic synthesis at Stanford University in 2009 [1] - He worked for Merck from 2009 to 2013 and has experience in biotech startups such as Theravance and Aspira before joining Caltech [1] - Brendan is a co-founder and the first employee of 1200 Pharma, which spun out of Caltech and received significant investment in the 8 figures [1] - He remains an avid investor focused on market trends, particularly in biotechnology stocks [1]

Group 1 - The article discusses the expertise of a research firm focused on the U.S. restaurant industry, covering various segments from quick-service to fine dining [1] - The firm employs advanced financial modeling and sector-specific KPIs to identify hidden value in public equities, particularly in micro and small-cap companies [1] - The analyst has a strong academic background, holding an MBA in Controllership and Accounting Forensics, and has specialized training in valuation and financial modeling [1] Group 2 - The research has been featured on multiple platforms, indicating a broad reach and recognition in the investment community [1] - The firm also covers related sectors such as consumer discretionary, food & beverage, and casinos & gaming, showcasing a diverse analytical approach [1]

Core Insights - UroGen Pharma Ltd. will present at the Piper Sandler 37th Annual Healthcare Conference on December 2-4, 2025 [1][2] - The presentation will take place on December 2, 2025, at 1:00 PM ET in a fireside chat format [2] - UroGen is focused on developing innovative solutions for urothelial and specialty cancers, utilizing proprietary technologies [3] Company Overview - UroGen Pharma Ltd. is dedicated to treating urothelial and specialty cancers, aiming to provide better treatment options for patients [3] - The company has developed RTGel, a reverse-thermal hydrogel technology that allows for sustained release of medications, potentially enhancing therapeutic effectiveness [3] - UroGen's first product targets low-grade upper tract urothelial carcinoma (LG-UTUC), while its second product is an intravesical solution for adult patients with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) [3] - The company is headquartered in Princeton, NJ, with operations in Israel [3]

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]

UroGen Pharma FY Conference Summary Company Overview - **Company**: UroGen Pharma (NasdaqGM:URGN) - **Technology**: RTGel technology, a unique polymer combination that forms a soft gel at body temperature, facilitating drug delivery to the bladder and upper urinary tract [2][3] Core Product Insights - **Product**: Jelmyto, approved for bladder cancer treatment - **Market Opportunity**: Approximately 60,000 patients with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (NMIBC) [4] - **Unmet Medical Need**: High recurrence rates among patients, with 23% experiencing five or more recurrences and 68% having two or more [4] Market Dynamics - **Patient Population**: Focus on low-grade disease, which is highly recurrent but not life-threatening [6][7] - **Surgical Failures**: Current treatments often lead to repetitive surgeries, highlighting the need for effective non-surgical options [4][6] Launch and Sales Strategy - **Sales Force Expansion**: Increased from 40 to 82 representatives to support the launch of UGN-102, with a focus on clinical nurse educators [10][11] - **Reimbursement Challenges**: Identified as the biggest barrier to adoption; efforts are in place to address this with field reimbursement teams [10][11][19] Early Launch Metrics - **Revenue Performance**: $1.8 million in Q3 and $4.5 million in October, indicating strong early adoption [14][15] - **Patient Enrollment Forms**: Early indicators of demand are on par with Jelmyto's performance after five years, suggesting a strong market interest [15][16] Future Growth Potential - **J Code Impact**: Anticipated positive effects on revenue post-J code implementation in January 2026, with expectations of increased physician confidence and reimbursement facilitation [18][19] - **Market Penetration**: Projected peak market penetration of over $1 billion, with a conservative estimate of 20% market share [22] Competitive Landscape - **First-Mover Advantage**: UroGen Pharma is positioned as a primary therapy for low-grade intermediate-risk bladder cancer, differentiating from competitors that require surgery [24][25] - **Future Products**: Competitors like J&J and CG are developing adjuvant therapies, which may follow surgical interventions, contrasting with UroGen's non-surgical approach [25][26] Pipeline Developments - **UGN-103**: A new formulation of mitomycin with improved production efficiency, expected to file for NDA in the second half of 2026 [30][31] - **UGN-104**: Successor product for Jelmyto, anticipated to follow UGN-103 by about a year [34] - **Oncolytic Virus (501)**: Currently in IND enabling studies, with potential applications beyond bladder cancer [35][36] Commercial Synergies - **Cross-Promotion**: Increased commercial efforts around UGN-102 are expected to drive growth for Jelmyto, as physicians become more familiar with both products [38] Conclusion UroGen Pharma is strategically positioned in the bladder cancer treatment market with innovative technology and a strong pipeline. The company is addressing significant unmet needs in patient care while navigating challenges related to reimbursement and market adoption. The anticipated impact of the J code and the first-mover advantage in the low-grade intermediate-risk segment are expected to drive future growth.

Financial Data and Key Metrics Changes - Total revenues for Q3 2025 were $27.5 million, consisting of $25.7 million from Gemyto and $1.8 million from Zesturi, with Gemyto sales showing a 13% year-over-year growth when excluding CreateX sales from the previous year [22][24] - Net loss for Q3 2025 was $33.3 million, or $0.69 per share, compared to a net loss of $23.7 million, or $0.51 per share in Q3 2024 [24] - Cash, cash equivalents, and marketable securities totaled $127.4 million as of September 30, 2025 [24] Business Line Data and Key Metrics Changes - Gemyto generated net product revenue of $25.7 million, reflecting a 13% increase in underlying demand revenue over the same period in 2024 [6][22] - Zesturi sales were $1.8 million in Q3 2025, with a preliminary demand revenue estimate of $4.5 million for October, indicating strong early momentum [15][22] Market Data and Key Metrics Changes - Zesturi addresses an estimated $5 billion annual market, with expectations to become a standard of care and deliver over $1 billion in peak revenue [5][6] - The company has secured broad coverage across major payers, with Zesturi now accessible to over 95% of covered lives, approximately 296 million eligible patients [16] Company Strategy and Development Direction - The company is focused on the ongoing commercialization of Zesturi and Gemyto, with Zesturi being the primary growth driver [3][4] - A permanent product-specific J code for Zesturi is expected to take effect on January 1, 2026, which is anticipated to accelerate adoption [19][20] - The company plans to submit a New Drug Application (NDA) for UGN-103 in the second half of 2026, with potential approval anticipated in 2027 [8][10] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term potential for Zesturi, citing positive physician feedback and increasing patient enrollment forms [4][5] - The company acknowledged logistical and operational challenges affecting the conversion of patient enrollment forms to actual patient dosing but expects improvements as practices gain experience [18][32] - Management emphasized the importance of the upcoming permanent J code in simplifying reimbursement and reducing barriers to adoption [19][20] Other Important Information - R&D expenses for Q3 2025 were $14 million, primarily driven by costs associated with the phase 3 Utopia trial for UGN-103 [22] - Selling, general, and administrative expenses increased to $37.6 million, driven by Zesturi commercial preparation activities and expansion of the sales force [23] Q&A Session Summary Question: Can you explain the timing for revenue recording and remittance? - Management indicated that the average lag time between patient enrollment form submission and patient dosing is currently 45-60 days, with expectations for gradual improvement as practices gain experience [31][33] Question: What visibility is there into physicians waiting for the permanent J code? - Management noted that many physicians are interested in prescribing Zesturi but are waiting for the permanent J code to take effect [36][40] Question: How are patient enrollment forms tracking month over month? - Management reported steady growth in patient enrollment forms, with some weeks showing equal or greater numbers compared to Gemyto [45][48] Question: What is the expected impact of the J code on lag time for patient dosing? - Management anticipates that the lag time will gradually decrease, potentially reaching below 30 days as practices become more familiar with the process [58][61] Question: Will additional capital be needed in 2026? - Management expressed confidence in their current cash position and operational plans, believing they can reach profitability without needing additional capital [70]

Market Opportunity and Product Approval - Zusduri has an estimated total addressable market opportunity of over $5.0 billion for recurrent low-grade intermediate risk NMIBC, with an annual treatable population of approximately 82,000 in the U.S.[151] - The FDA approved Zusduri on June 12, 2025, as the first and only FDA-approved non-surgical treatment for recurrent low-grade intermediate risk NMIBC[151][164] - Jelmyto, approved on April 15, 2020, is designed for the treatment of low-grade UTUC and has a median duration of response (DOR) of 47.8 months in patients who achieved a complete response[159] Clinical Trial Results - The ATLAS trial showed Zusduri reduced the risk of recurrence, progression, or death by 55% compared to TURBT alone, with a complete response (CR) rate of 64.8% at three months[166] - The ENVISION trial demonstrated a 79.6% CR rate at three months following the initial instillation of Zusduri, meeting its primary endpoint[166] - In the Phase 3 ENVISION trial, Zusduri achieved a 79.6% complete response (CR) rate at three months and 60.8% maintained CR at 12 months among 240 patients[167] - The 18-month duration of response (DOR) for patients who achieved CR at three months was 80.6%, consistent with the 12-month DOR of 82.3%[168] - At 24 months, the DOR for patients who achieved CR at three months was 72.2%[169] - The FDA approved Zusduri on June 12, 2025, based on a CR rate of 78% at three months and 79% maintaining CR at 12 months[173] - The company initiated the Phase 3 UTOPIA trial for UGN-103, enrolling 99 patients globally, with a reported CR rate of 77.8% at three months[178] Financial Performance - Revenue for the three months ended September 30, 2025, was $27.5 million, and for the nine months, it was $72.0 million[190] - Revenue for the three months ended September 30, 2025, was $27.5 million, an increase of $2.3 million from $25.2 million in 2024, primarily due to higher sales volume of Jelmyto and the launch of Zusduri[212] - For the nine months ended September 30, 2025, revenue was $72.0 million, an increase of $6.2 million from $65.8 million in 2024, attributed to increased sales of Jelmyto and the launch of Zusduri[222] Expenses and Losses - Research and development expenses for the nine months ended September 30, 2025, totaled $52.8 million, up from $42.3 million in 2024[193] - Selling and marketing expenses increased to $23.7 million for the three months ended September 30, 2025, from $17.8 million in 2024, reflecting costs related to Zusduri commercial activities and expanded sales force[215] - General and administrative expenses rose to $13.9 million for the three months ended September 30, 2025, compared to $11.2 million in 2024, driven by higher compensation and commercialization support for Zusduri[216] - The net loss for the three months ended September 30, 2025, was $33.3 million, compared to a net loss of $23.7 million in 2024, reflecting an increase in operating loss[210] - Total operating expenses for the three months ended September 30, 2025, were $51.6 million, an increase of $11.3 million from $40.3 million in 2024[210] Cash Flow and Financing - As of September 30, 2025, the company had $127.4 million in cash and cash equivalents and marketable securities, indicating a strong liquidity position[231] - The company reported an accumulated deficit of $933.4 million as of September 30, 2025, and expects to continue incurring losses as it executes its strategy for the commercialization of Zusduri and Jelmyto[243] - The company raised approximately $120.0 million through a Securities Purchase Agreement in July 2023, selling 12,579,156 ordinary shares at a price of $9.54 per share[240] - The company has incurred negative cash flows from operations and expects to finance its cash needs through a combination of equity or debt financings and collaboration arrangements[245] - Future funding requirements will depend on various factors, including clinical trial progress and commercialization activities for Jelmyto and Zusduri[246] Debt and Obligations - The company entered into a loan agreement with Pharmakon for a senior secured term loan of up to $100.0 million, with the first tranche of $75.0 million funded in March 2022 and the second tranche of $25.0 million funded in December 2022[256] - The obligations under the loan agreement are guaranteed by UroGen Pharma Ltd. and secured by substantially all tangible and intangible assets[259] - The company received a one-year extension on the repayment period for all outstanding loans following FDA approval of the NDA for Zusduri, with repayments commencing in Q2 2027[258] Market Coverage and Exclusivity - Medicare patients with supplemental coverage are covered for Jelmyto, with over 150 million lives covered by commercial plans[161] - Jelmyto's new product exclusivity expired on April 15, 2023, but orphan drug exclusivity extends until April 15, 2027[157] - Zusduri is now accessible to over 296 million eligible patients in the U.S., with more than 95% of covered lives having open access[175] Other Financial Metrics - Interest expense on long-term debt increased to $3.4 million for the three months ended September 30, 2025, from $2.7 million in 2024, primarily due to the Pharmakon loan[218] - Interest expense on long-term debt increased to $11.6 million for the nine months ended September 30, 2025, up from $8.6 million in 2024, attributed to the $25.0 million third tranche under the Pharmakon loan funded in September 2024[229] - Net cash provided by investing activities was $33.8 million during the nine months ended September 30, 2025, compared to net cash used of $81.2 million in the same period of 2024, reflecting a net change of $115 million[262] - Net cash provided by financing activities decreased to $8.6 million in the nine months ended September 30, 2025, from $194.5 million in the same period of 2024, a decrease of $185.9 million[263] Currency and Inflation - The company does not currently engage in currency hedging activities to reduce currency exposure but may consider it in the future[267] - The company experienced a 1.2% appreciation of the U.S. dollar against the New Israeli Shekel during 2024, which could affect dollar-denominated results of operations[266] - Inflation has not had a material effect on the company's business or financial condition during the nine months ended September 30, 2025 or 2024[265]

Financial Performance - UroGen reported total revenues of $27.5 million for Q3 2025, with JELMYTO generating $25.7 million, reflecting a year-over-year growth of approximately 13%[12][8] - ZUSDURI achieved net product revenue of $1.8 million in its first quarter on the market, with preliminary demand revenue for October 2025 estimated at $4.5 million, indicating accelerating growth[12][6] - UroGen reported a net loss of $33.3 million or ($0.69) per share in Q3 2025, compared to a net loss of $23.7 million or ($0.51) per share in Q3 2024[18][26] - The company expects full-year 2025 net product revenues for JELMYTO to be in the range of $94 to $98 million, implying a growth rate of approximately 8% to 12% over 2024[21] Research and Development - R&D expenses for Q3 2025 were $14.0 million, an increase from $11.4 million in Q3 2024, primarily due to costs associated with the UTOPIA trial[13] - The three-month complete response rate for UGN-103 was reported at 77.8% in the Phase 3 UTOPIA trial, consistent with previous results, and the FDA agreed to the NDA submission plan[16][6] - UroGen has made the strategic decision to discontinue the development of UGN-301 after its Phase 1 study, as it did not meet internal benchmarks for advancement[16] - UroGen plans to submit a New Drug Application (NDA) for UGN-103 and is conducting ongoing clinical trials for UGN-104 and UGN-501[46] Commercial Activities - SG&A expenses rose to $37.6 million in Q3 2025, compared to $28.9 million in the same period in 2024, driven by ZUSDURI commercial launch activities[14] - As of October 31, 2025, UroGen reported 592 activated sites of care and 54 unique prescribers for ZUSDURI since its launch on July 1, 2025[7] - Preliminary demand revenue estimate for ZUSDURI in October indicates accelerating commercial uptake and growing physician adoption[46] Product Information - ZUSDURI (mitomycin) is approved for treating adults with recurrent low-grade intermediate risk non-muscle invasive bladder cancer (LG-IR-NMIBC) after ineffective prior surgery[28] - JELMYTO (mitomycin) is indicated for adult patients with low-grade upper tract urothelial cancer (LG-UTUC) and is administered via a ureteral catheter or nephrostomy tube[37] - UroGen's proprietary RTGel technology allows for sustained release of medication, improving therapeutic profiles and potentially enhancing treatment effectiveness[45] - UroGen's sustained release technology aims to improve local delivery of complex immunotherapies, enhancing treatment options[46] Safety and Side Effects - The most common side effects of ZUSDURI include increased blood creatinine levels and urinary tract infections[35] - JELMYTO may cause serious side effects, including ureteric obstruction and bone marrow problems, necessitating blood tests prior to treatment[47] Company Overview - UroGen is headquartered in Princeton, NJ, with operations in Israel, focusing on innovative solutions for urothelial and specialty cancers[45]