GUGGENHEIM 3RD ANNUAL HEALTHCARE INNOVATION CONFERENCE - TRAVERE THERAPEUTICS INC

Explicit Guidance:

• PEG-tibadenase: Top-line data from the Phase 3 "Harmony" study expected in the second half of 2026.
• Sivorabrutinib: Investigational New Drug (IND) filing expected by year-end 2024.
• Filspari Revenue/Volume: Reported $200 million net revenue for Q3 2024; noted nearly 2,000 new patients added in Q3; indicated "continued strength" in prescribing as Q4 begins (no specific FY guidance provided in transcript).

Business-Segment Outlook:

• Filspari (IgA Nephropathy): Anticipate continued strong demand driven by physicians actively shifting from standard of care (ACE inhibitors, SGLT2s, steroids) to approved therapies; expect growth in patient diagnosis via increased biopsy rates for younger, symptomatic patients; predict shift toward combination therapies (kidney-targeted + immune-targeted) as physicians gain comfort with sequencing, particularly in sicker patients first.
• Filspari (FSGS): Expect continued strong uptake in the second quarter of the launch (post-Q3) with broad adoption across primary, genetic, and secondary FSGS etiologies and a wide range of nephrologists; no direct competitors expected in the near term.
• PEG-tibadenase (HCU): Target addressable market of ~3,500 diagnosed patients in the U.S. currently under physician care; potential to expand reach to 10,000–12,000 patients by identifying undiagnosed adults and re-engaging lost-to-follow-up patients via improved newborn screening and outreach.
• Sivorabrutinib (PMN): First indication will be Primary Membranous Nephropathy (PMN) targeting ~90,000 patients, with plans to evaluate immune-mediated FSGS and minimal change disease subsequently.
• Patient Adherence: Anticipate continued high patient engagement due to Filspari's mechanism replacing prior therapies (reducing pill burden) rather than adding to existing regimens; 90% of Filspari prescriptions in 2024 represent a patient's first exposure to any branded renal therapy.

Market and Competitive Outlook:

• FSGS Competitive Landscape: Management sees no direct competitors in the future; while other therapies in development may address specific genetic or immune-mediated subtypes, they lack the broad efficacy profile of Filspari and are viewed as complementary rather than substitutive.
• IgAN Competitive Landscape: Expect the market to grow dynamically due to updated KDIGO guidelines advocating for combination therapies (kidney-targeted + B-cell therapies); anticipate early combination use in sicker patients, evolving to broader combination use as physicians become more experienced; note that Novartis's ERA competitor is viewed as less preferred than Filspari because Filspari replaces existing meds (ACE/ARB) reducing pill burden, whereas alternatives often require adding to existing regimens.
• Diagnostic Trends: Expect accelerated diagnosis rates as physicians adopt a more aggressive stance, moving away from "watchful waiting" for patients with 1–2g proteinuria due to recognition of high long-term kidney failure risk.

Macro / Industry Outlook:

• Payer Environment: Do not anticipate payer pushback regarding combination therapy or access to Filspari; payers have been educated on the clinical imperative for complete remission and KDIGO guidelines; broad payer access is expected to remain stable.
• Regulatory Environment: Engaging with regulators on Sivorabrutinib dosing and endpoints to ensure global development readiness; emphasizing early data generation in U.S. populations to support bridging studies from the original East Asian trials.

Strategic Initiatives:

• Leadership Transition: Eric Dubé departing CEO role; Bradley Campbell to assume CEO duties on December 1, 2024.
• Strategic Focus: New leadership expected to continue "wise, disciplined investments" in business development; focus remains on commercializing existing portfolio and advancing pipeline assets (PEG-tibadenase, Sivorabrutinib).
• Pipeline Development:
  • PEG-tibadenase: Continue enrollment on "Harmony" Phase 3 study; aim to improve homocysteine control to allow for less strict dietary adherence compared to current standard of care.
  • Sivorabrutinib: Conduct bridging PK/PD studies in the U.S. population to validate dosing; move quickly from IND filing to later-stage development for PMN.
• Capacity/Reach Expansion: Initiatives to expand patient reach for HCU via better newborn screening and outreach to undiagnosed populations.

Risks and Headwinds to Outlook:

• Regulatory/Trial Timelines: Success of PEG-tibadenase and Sivorabrutinib remains contingent on successful completion of ongoing clinical studies (Phase 3 data in H2 2026; IND filing and subsequent dosing studies for BTK inhibitor).
• Competitive Uncertainty: While no direct competitors are seen now for FSGS, management acknowledges the potential for "another therapy that could be broad" in the longer term.
• Patient Identification: HCU commercial success depends on successfully identifying the 6,500–9,000 undiagnosed or lost-to-follow-up patients (10k–12k total vs. 3.5k current addressable).
• Execution Risk: Transition to new CEO (Dec 1) introduces a period of leadership change, though management expresses confidence in the board's selection and the continuity of strategy.

Management Tone on the Future:

• Confidence: High confidence in execution ("very strong quarter," "on track," "foundational positioning").
• Sentiment: Optimistic regarding market dynamism in nephrology, the alignment of payer/physician behavior with the treatment algorithm, and the unmet need in rare diseases (HCU, PMN).
• Hedges: Acknowledges seasonality in nephrology (summer slowdowns); notes that combination therapy adoption will likely be sequential (sicker patients first); acknowledges that FSGS competitors could emerge but remain distant.

Miscellaneous:

• Product Differentiation: Emphasizes Filspari's unique value proposition of reducing pill burden by replacing rather than adding to current therapies (e.g., stopping ACE inhibitors/ARBs), which resonates strongly with patient preference and control.
• Clinical Data: Reference to ASN meeting data showing that early treatment-naive patients can achieve both proteinuria remission and eGFR stabilization with Filspari.
• Company History: CEO departure marks the end of an 8-year tenure focused on establishing Travere as a leader in rare kidney diseases.