STIFEL 2026 HEALTHCARE CONFERENCE - TRAVERE THERAPEUTICS INC

Financial Performance:

• Achieved $200 million in revenue for the second quarter, driven by strong underlying demand and a robust base of continuing patients.
• Received 1,932 new patient start forms in the quarter, reaching nearly 2,000 patients.
• No specific guidance provided on full-year revenue or EPS; management indicated a path to profitability exists given current performance.
• Operating expenses (SG&A) expected to increase next year to support a full year of FSGS and IGAN launches and the advancement of pipeline assets.

Guidance and Future Outlook:

• Anticipated summer seasonality in IGAN patient starts, observing a similar low single-digit decrement in Q3 vs. Q2 consistent with historical patterns.
• FSGS launch trajectory aligned with expectations: rapid initial uptake followed by sustained growth without evidence of a "bolus" effect.
• Full year 2025 expected to see increased commercial activity and spending as the company moves past initial launch phases.
• Upcoming CEO Bradley Campbell is expected to define specific revenue and demand guidance for the next fiscal year.

Business Segments and Product Lines:

• Philspari (FSGS & IGAN): First approved medicine for FSGS; strong uptake with high adherence to dose titration (400mg to 800mg) in adults, though pediatric dosing remains at half-rate.
• Pipeline:
  • Sivo (Sivocabrutinib): BTK inhibitor for renal diseases; >150 patients dosed to date with encouraging safety; IND submission to FDA targeted for this year.
  • Pegditatinase: ERT for HCU (homocystinuria); Phase 3 "Harmony" trial on track with consistent 67% homocysteine reduction observed in Phase 2; focus on dietary protein intake as a sub-study endpoint.
• Combination Strategy: Positioning Philspari as foundational kidney-directed therapy (combined with SGLT2 inhibitors) alongside emerging immune-targeted therapies (B-cell or complement inhibitors).

Market and Competitive Landscape:

• IGAN: Competition from new B-cell and complement therapies (e.g., ciboprenolamab, atacicep) has not displaced Philspari; nephrologists face time constraints but continue to prescribe Philspari. Payers are adopting guidelines recommending aggressive early treatment.
• FSGS: Philspari holds a unique position as the first approved therapy; competitors include phenylenedione and genotype-specific therapies expected to be complementary rather than directly substitutive.
• Market Dynamics: KIDIGUI and KDIGO guidelines driving a shift toward complete remission and proteinuria reduction, increasing treatment urgency. Payers are generally receptive to combinations, with some requiring Philspari as a prerequisite for accessing immune therapies.

Risks and Challenges:

• Payer Dynamics: Potential for step edits and management of combination therapies; payers are evaluating net price trajectories, particularly with FSGS titration affecting net price stability.
• Pipeline Execution: IND submission timing for Sivo and Phase 3 readout for Pegditatinase depend on successful regulatory interactions and manufacturing scale-up.
• Market Share: Competition in emerging rare kidney disease spaces is increasing, requiring continuous differentiation through combination data and adherence to guidelines.

Management Commentary and Tone:

• Management expressed high confidence in the commercial trajectory of Philspari and the foundational role it plays in combination therapies.
• Tone was optimistic regarding the "renaissance" in rare kidney disease innovation and the regulatory clarity provided by the FDA.
• CEO Eric Dubé highlighted the alignment between physician adoption of new guidelines and commercial success, dismissing concerns about trade-offs to newer B-cell therapies.
• Transition to a new CEO (Bradley Campbell) is framed as a natural evolution as the company pivots to a broader growth phase.

Analyst Questions and Answers:

• IGAN Seasonality vs. Competition: Q: Is the Q3 dip seasonality or competition? A: Confirmed as seasonality with no evidence of physician trade-off to competing B-cell therapies.
• Payer Behavior: Q: How are payers handling combinations? A: Payers are paying for B-cell/complement therapies on top of Philspari; some payers are implementing step edits favoring Philspari background therapy.
• FSGS Pricing: Q: Impact of dose titration on net price? A: Strong adherence to 800mg target suggests upward pressure on net price as new patients enroll and titrate.
• Pegditatinase Endpoints: Q: Will functional endpoints be required for approval? A: FDA agreed to homocysteine reduction as primary endpoint; functional data on protein intake is being collected but not required for BLA.
• Sivo Development: Q: Timing of IND and independence from competitor data? A: IND filing expected this year; program development is independent of Paragon's MN data readouts.

Other Key Points:

• The company covers 6,000–7,000 nephrologists, with significant overlap between IGAN and FSGS prescribers, though the IGAN addressable population is >2x larger than FSGS.
• FSGS label excludes patients with active nephrotic syndrome, which physicians understand and manage by reserving Philspari for non-nephrotic cases while treating acute needs with other modalities.
• Manufacturing for Pegditatinase has transitioned from clinical to commercial scale, with no integrity issues reported despite previous delays.
• The company is exploring additional indications for Sivo, including immune-driven FSGS and Minimal Change Disease.