GUGGENHEIM 3RD ANNUAL HEALTHCARE INNOVATION CONFERENCE - UROGEN PHARMA LTD

Financial Performance:

• Reported Q3 sales of $68 million, representing 35% year-over-year growth.
• Q3 net loss was $1.5 million, significantly improved from prior periods excluding one-time costs.
• Revenue growth was driven by a combination of new prescriber acquisition (rising from ~450 to >650 unique prescribers) and increased depth of use (repeat writers increasing from 45% to 50% of the base).
• Revenue impact from Gelmito was suppressed by a $1.5 million one-time CMS invoice adjustment regarding discarded drug provisions and competition from clinical trials.
• Company projects cash flow positivity and profitability to be achieved in Q4 2024 without requiring additional capital raises.
• Q2 included a one-time $5 million PDUFA fee expense; future quarters should see normalized operating expenses.

Guidance and Future Outlook:

• Management maintains a "billion-plus dollar" peak sales opportunity for Sesturi (Zasturi) within a 4-5 year timeline post-J-code implementation.
• Projected trajectory to peak sales is extended to 4-5 years due to the slower adoption curve of urologists in the "buy and bill" model.
• Expect profitability and positive cash flow to be sustained in Q4 2024, enabling a path to long-term sustainable growth.
• UGN-104 enrollment expected to complete by Q1 2027, removing trial-related revenue headwinds for Gelmito.
• Long-term strategy involves transitioning from a clinical-stage company to a commercial-and-clinical-stage entity with a focus on pipeline expansion into high-risk papillary disease and new oncology indications.

Business Segments and Product Lines:

• Sesturi (Ugn-103): First FDA-approved systemic product for intermediate-risk non-muscle invasive bladder cancer (NMIBC); launching permanent J-code uptake is accelerating adoption. 100% of doctors in active treatment units (ATU) who tried the drug expressed intent to use it again.
• Gelmito: Revenue pressured by rare disease market size (~6,000 US patients), competition from clinical trials, and operational challenges. A new 15-person "strike team" has been formed to drive growth.
• UGN-104: Next-generation formulation of Gelmito with enhanced solubility, extended post-reconstitution shelf life (weeks vs. days), and faster manufacturing (3 days lyophilization vs. 3 weeks). Currently enrolling in a Phase 3 study.
• UGN-501: First patient dosed in a Phase 1 study; a next-generation oncolytic virus with a dual mechanism (direct lysis + immune response) intended to bridge into other solid tumors beyond bladder cancer.
• Pipeline Expansion: Plans to study UGN-104 in high-risk papillary bladder cancer later this year, potentially expanding the total addressable market beyond the ~20,000 patients in the papillary-only segment.

Market and Competitive Landscape:

• Management rejects the "zero-sum game" narrative, arguing that multiple entrants in the NMIBC space grow the overall market by changing surgical paradigms.
• Sesturi holds a first-mover advantage as the only non-surgical option currently approved; competitors (e.g., CT Oncology) are not expected to launch for some time.
• Sesturi's value proposition emphasizes "treatment-free living" with an 80% complete response rate and durable responses at 36 months, contrasting with competitors who require surgery or longer treatment cycles.
• The competitive landscape in the rare disease Gelmito space is intensifying with new trials enrolling specifically in UTUC and US markets, though Sesturi remains the dominant growth driver.

Risks and Challenges:

• Adoption Speed: Urologists are traditionally slower to adopt new "buy and bill" drugs, requiring a steady ramp-up over 4-5 years rather than an immediate spike.
• Clinical Trial Cannibalization: Patients entering clinical trials for Gelmito and competing products (like UGN-104) reduce immediate revenue for the existing Gelmito product.
• Reimbursement/Operational Friction: While 95% of covered lives have access, the "buy and bill" workflow presents operational hurdles for community practices.
• Market Education: Significant effort required to shift urologist practice patterns from surgery-centric to drug-centric treatment for intermediate-risk patients.

Management Commentary and Tone:

• Tone is confident, optimistic, and focused on long-term sustainability.
• Management expresses high confidence in the $1B+ sales ceiling and the 4-5 year ramp period.
• CFO Chris Dagnon and CEO Liz Barnett emphasize the strategic decision to prioritize Sesturi, noting that the Q3 performance ($147M vs consensus $140M) validates this focus.
• Acknowledged recent challenges with Gelmito but framed them as temporary, with a clear operational fix (strike team) and timeline for resolution.

Analyst Questions and Answers:

• Repeat Usage: Analysts noted revenue is driven by depth (repeat prescriptions); Management confirmed early adopters are driving debt and 100% of ATU doctors plan repeat use.
• Market Share: Questioned if adjuvant therapies for new diagnoses would shrink the recurrent market; Management argued the prevalent pool (400k low-grade patients) ensures a stable, long-term patient base regardless of new diagnosis treatment.
• Product Durability: Real-world durability data is early but aligns with clinical study results; patients are seeing good responses in heavily pretreated populations.
• Profitability: Confirmed that Q4 2024 will mark the transition to profitability and positive cash flow.

Other Key Points:

• Intellectual Property: UGN-103 and Sesturi benefit from patents extending to mid-2035 (mitomycin) and 2041-2045 (method of use/RT gel).
• Operational Efficiency: UGN-104 offers COGS benefits via faster manufacturing and reduced waste through extended shelf life.
• Strategic Pivot: The company is actively transitioning from a pure clinical focus to a commercial execution focus, evidenced by the dedicated teams for Sesturi and the new Gelmito strike team.