GUGGENHEIM 3RD ANNUAL HEALTHCARE INNOVATION CONFERENCE - UROGEN PHARMA LTD

Explicit Guidance:

• Revenue Growth: Sisturi (Zesturi) sales guidance of $140 million for the year was exceeded with $147 million reported in the first three quarters; peak sales opportunity remains $1 billion+ with a 4–5 year ramp to peak from the January J-code approval.
• Profitability & Cash Flow: Management expects to reach profitability and become cash-flow positive in Q4 of the current year, driven by Sisturi growth and the absence of Q2 one-time costs (specifically the $5 million PDUFA fee).
• Capital Strategy: The company is on the "cusp" of reaching profitability without the need for additional capital raises.
• Jalmito Guidance: Jalmito revenue is expected to face headwinds in the current and upcoming quarters due to clinical trial enrollment (approx. $3.5–$4 million impact) and a one-time $1.5 million CMS adjustment charge, but is targeted to return to growth by 2027 once trial enrollment concludes.

Business-Segment Outlook:

• Sisturi (UGN-103):
  • Market Penetration: Currently only 10% (650/6,500) of core target urologists have tried the drug; growth drivers include shifting from "trial" to "repeat" adoption (repeat writer rate grew from 45% to 50% Q2 to Q3).
  • Adoption Curve: Projected 4–5 year time to peak sales as urologists, a slower-adopting segment, adjust workflows. 100% of doctors in the Expanded Access Program (ATU) who tried the drug indicated they would use it again.
  • High-Risk Expansion: UGN-103 (Sisturi) will expand into high-risk papillary bladder cancer with a study expected to start later this year.
• Jalmito:
  • Market Share: Facing pressure from a small patient pool (~6,000 patients) and competition from enrolling clinical trials in the US and UTUC space.
  • Revitalization: A dedicated 15-person "strike team" has been formed to drive growth, with a targeted return to growth trajectory by 2027.
• New Product (UGN-104): Next-gen formulation of Jalmito; enrollment expected to complete in Q4 of current year or early next year; offers improved stability (weeks vs. 7 days post-mix) and faster manufacturing (3 days vs. 3 weeks), leading to cost-of-goods improvements.
• New Asset (UGN-501): First patient dosed in Phase 1 study for an oncolytic virus; aims to bridge company capabilities beyond urothelial cancers to other solid tumors via intratumoral delivery.

Market and Competitive Outlook:

• Competitive Landscape: Management rejects the "zero-sum game" narrative; expects multiple entrants (e.g., CT Oncology, J&J, Lexo) to expand the overall market rather than cannibalize share.
• Differentiation: Sisturi remains the only FDA-approved non-surgical option for intermediate-risk NMIBC; competitors rely on surgery or different chemotherapies (e.g., gemcitabine).
• Treatment Paradigm: Anticipated shift toward bladder-sparing therapies; management expects patients to cycle through multiple therapies over time as no current treatment is a cure.
• Market Size: Potential for $1 billion peak sales in intermediate-risk space; high-risk papillary market (approx. 20,000 patients) adds a "similar opportunity" layer for UGN-103.

Macro / Industry Outlook:

• Reimbursement: Over 95% of covered lives have access to Sisturi, removing major reimbursement barriers despite physician hesitancy regarding operational workflows.
• Regulatory: No specific macro regulatory headwinds mentioned other than the standard adoption curve for urologists adapting to "buy and bill" models.
• Patient Demographics: 400,000 people living in the US with low-grade bladder cancer; 15% recurrence rate annually ensures a stable prevalent pool even if new diagnoses are treated with alternative therapies.

Strategic Initiatives:

• Commercial Focus: Deployment of a 15-person specialized "strike team" solely focused on revitalizing Jalmito growth.
• Pipeline Expansion: Advancement of UGN-103 into high-risk indications and UGN-501 into Phase 1 trials to diversify beyond urothelial cancers.
• Manufacturing & Operations: Transition to UGN-104 to reduce manufacturing time and improve supply chain efficiency for community practices.
• IP Moat: Secured strong intellectual property protection: UGN-103/104 method of use patent through July 2044; proprietary mitomycin through mid-2035; RT gel through mid-2045.

Risks and Headwinds to Outlook:

• Adoption Friction: Urologists are historically slow to adopt new commercial products and unfamiliar with "buy and bill" operational models, potentially delaying the peak sales timeline.
• Clinical Trial Enrollment: Ongoing trials (UGN-104 and others) divert patients from the commercial Jalmito patient pool, impacting short-to-mid-term revenue.
• Regulatory/Reimbursement Administration: CMS adjustments (e.g., discarded drug provision) can cause unexpected one-time financial charges.
• Market Saturation (Long-term): While minimal short-term risk, "cross-line reduction" (treating newly diagnosed patients upfront) could eventually shrink the recurrent patient pool, though management estimates a 10–20 year horizon before this significantly impacts the market.

Management Tone on the Future:

• Confidence: High confidence in the $1 billion+ peak sales target and the company's path to profitability in Q4.
• Strategic Shift: Explicit acknowledgment of a successful shift from clinical to commercial stage, with a focus on sustainable long-term growth for shareholders.
• Optimism on Competition: Positive view of increasing competitor activity, framing it as market education that benefits all players rather than a threat.
• Hedges: Growth for Jalmito is explicitly conditioned on the completion of trials in 2027 and the successful execution of the new strike team strategy.

Miscellaneous:

• Segment Transition: Company is transitioning from a "clinical stage" to a "commercial and clinical stage" entity.
• Real-World Data: Anecdotal evidence suggests real-world durability mirrors clinical study results, with patients experiencing complete or near-complete responses.
• Financial Context: Q3 net loss was $1.5 million, excluding one-time costs and revenue headwinds.