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 - As of June 30, 2025, the company had cash, cash equivalents, and short-term investments of approximately $20.6 million, down from $44.9 million as of December 31, 2024 [22] - Cash used in operations in Q2 2025 was $6.4 million, compared to $13.3 million for the same period in 2024 [22] - The net loss for Q2 2025 was $9.9 million or $0.30 per share, compared to a net loss of $17.8 million or $0.59 per share for Q2 2024 [24] Business Line Data and Key Metrics Changes - Research and development expenses for Q2 2025 totaled $2.8 million, down from $10.7 million in Q2 2024, primarily due to lower study costs from the wind down of clinical trials for REL-ten seventeen [23] - General and administrative expenses for Q2 2025 were $7.4 million, compared to $8.1 million in Q2 2024, driven by a decrease in stock-based compensation [24] Market Data and Key Metrics Changes - The company is focusing on two product candidates: NDV-one for non-muscle invasive bladder cancer and sopranolone for compulsivity disorders, targeting significant and underserved markets [6][20] - NDV-one has shown a complete response rate of 91% at any time point following treatment in high-risk patients [14] Company Strategy and Development Direction - The company aims to initiate a Phase III study for NDV-one in 2026 and a proof of concept study for sopranolone in Prader Willi syndrome also in 2026 [18][27] - The management team has expanded with the addition of experts in bladder cancer and urologic oncology, which is expected to enhance the company's strategic objectives [26] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of NDV-one as a promising treatment for non-muscle invasive bladder cancer, with plans to engage with the FDA regarding trial design [18][36] - The company believes that the recent approval of a competitor's product sets a precedent for their own approach in the market [33] Other Important Information - The company reported a clean balance sheet and a strong cash position, which positions it well for future value-creating steps [27] - Management emphasized the importance of upcoming milestones and the disciplined development strategy to enhance the long-term value proposition [22] Q&A Session Summary Question: How do recent data and competitor approvals affect NDV-one's strategy? - Management acknowledged the opportunity in both high-grade and low-grade non-muscle invasive bladder cancer, noting the significant efficacy data for high-grade disease while also considering the larger market for low-grade patients [31][33] Question: What are the goals for the upcoming FDA meeting? - The main goal is to discuss the viability of a single-arm open-label study for chemoablation, similar to the path taken by a competitor [36] Question: How should R&D expenses be viewed going forward? - R&D expenses have decreased due to a lack of patient enrollment, but costs will rise again as the company prepares to start new trials [41]