GUGGENHEIM 3RD ANNUAL HEALTHCARE INNOVATION CONFERENCE - TRAVERE THERAPEUTICS INC

Financial Performance:

• Reported $200 million in net revenue for the third quarter, driven by Filspari.
• Achieved strong patient conversion with nearly 2,000 new patients starting Filspari therapy during the quarter.
• Net revenue growth attributed to robust payer access and high conversion of new patient starts to paid prescriptions for both IgA Nephropathy (IgAN) and FSGS indications.
• Specific earnings per share (EPS) or net income margins were not explicitly detailed in the transcript, though the quarter was characterized as "very strong."

Guidance and Future Outlook:

• On track for data readout from the Phase 3 Harmony study of Pegtolibate (PEG-T) for classical Homocystinuria (HCU) in the second half of 2025.
• Pivotal data for Sivorabrutinib (Siv) expected to follow IND filing and bridging studies; IND filing targeted for end of year.
• CEO transition scheduled for December 1st with Brad Campbell taking over from Eric Dubé, who remains on board to support the transition.
• Strategic focus for 2027 and beyond includes expanding pipeline commercialization and disciplined business development investments.

Business Segments and Product Lines:

• Filspari (Sparsentan):
  • IgAN: Performance consistent with historical summer seasonality (slower patient visits), yet underlying demand remains strong due to shifting treatment algorithms away from ACE inhibitors/SGLT2s. 90% of new prescriptions are for patients with no prior branded therapy history.
  • FSGS: Entered first full quarter for launch; showing broad uptake across primary, genetic, and secondary FSGS types and across a wide base of nephrologists rather than concentrated in specific practices.
• PEGtolibate: Enzyme replacement therapy for classical HCU; currently in Phase 3 Harmony study.
• Sivorabrutinib: Newly licensed BTK inhibitor from Everest Medicines; first indication targeted is Primary Membranous Nephropathy (PMN), with potential for immune-mediated FSGS and minimal change disease.

Market and Competitive Landscape:

• Filspari Positioning: Viewed as having no direct competitors for FSGS currently; other developmental therapies are expected to be complementary or niche (e.g., specific genetic subtypes).
• IgAN Market Dynamics: Market growth driven by physician aggression in treating patients earlier to prevent kidney failure, supported by updated KDIGO guidelines advocating for combination therapy (Filspari + B-cell therapies).
• Competition vs. Verquvo: Physicians prefer Filspari for its "replacement" model (replacing ACE/ARB) which reduces pill burden and signals patient improvement, whereas competitors often require adding therapies.
• Payer Environment: No significant pushback on combination therapy usage; payers recognize the need for complete remission of proteinuria and have maintained broad access.

Risks and Challenges:

• FSGS Patient Identification: High unmet need remains, but challenges include identifying and reaching undiagnosed patients (missed at newborn screening) or those lost to follow-up.
• HCU Treatment Adherence: Current standard of care (strict low-protein diet) is a significant burden leading to psychological distress and non-adherence; current drugs (Vitamin B6, betaine) are ineffective for a majority of patients.
• Sivorabrutinib Development: Requires bridging studies to ensure dosing efficacy in US populations, as prior data is based on East Asian populations.
• Regulatory/Clinical Path: Ensuring IND and study designs align with regulatory needs to expedite development timelines.

Management Commentary and Tone:

• Tone: Confident and execution-focused; management expressed high satisfaction with the Q3 results and the rapid uptake of Filspari in FSGS.
• Strategic Vision: Emphasized the shift from "observation" to "aggressive early treatment" in IgAN and the critical need for combination therapies to achieve complete remission.
• Leadership Transition: Management praised outgoing CEO Eric Dubé and incoming CEO Brad Campbell, highlighting Campbell's experience in commercializing rare disease products and business development discipline.

Analyst Questions and Answers:

• Q: What drives FSGS uptake and growth? A: Broad utilization across FSGS etiologies and diverse nephrologist practices, rather than concentration in specific centers.
• Q: Is the competitive landscape intensifying in FSGS? A: No direct competition expected in the near term; potential future therapies are viewed as complementary.
• Q: How is the IgAN market evolving with new therapies? A: Market is expanding as physicians treat earlier and adopt combination regimens to achieve complete proteinuria remission and stabilize GFR.
• Q: Are payers pushing back on combination therapies? A: No; payers support the guidelines and Filspari is typically the first branded drug patients encounter.
• Q: Why target HCU first for PEG-T? A: Severe unmet need with devastating outcomes (ischemic events, eye issues) and ineffective current standard of care regarding diet adherence.
• Q: What is the strategy for Sivorabrutinib? A: File IND by year-end, conduct bridging studies, and target PMN first due to high symptom burden and need for rapid onset of action.

Other Key Points:

• Patient Experience: Filspari is noted to allow patients to stop other medications (ACE/ARB), improving patient sentiment and optimism compared to adding new drugs.
• KDIGO Guidelines: Updated guidelines explicitly recommend combination therapy involving kidney-targeted agents (like Filspari) and immune-targeted agents (like B-cell therapies).
• Addressable Market (HCU): Current diagnosed patient base in the US is ~3,500; total addressable market including undiagnosed could range from 10,000 to 12,000.
• Primary Membranous Nephropathy (PMN): Described as a symptomatic condition (edema, proteinuria) where patients currently rely on slow-acting off-label therapies like Rituximab.