Travere Therapeutics, Inc. Q3 FY2024 earnings call

• FILSPARI Launch: The company achieved full approval of FILSPARI for IgA nephropathy and is in the ongoing commercial launch. There was over 500 new patient start forms in Q3, with growth in net sales. The new label and draft KDIGO guidelines are driving momentum.
• FSGS Program: Data from DUET and DUPLEX studies show sparsentan's superiority in reducing proteinuria in FSGS. A meeting with the FDA is scheduled to discuss an sNDA submission for FSGS, with potential full approval next year. Also, PARASOL group recommendations support using proteinuria as a clinical trial endpoint for FSGS.
• International Expansion: FILSPARI launched in Germany, Austria, and received temporary marketing approval in Switzerland with partners CSL Vifor and Renalys Pharma.
• Medical Perspectives: Clinical data from ASN Kidney Week supports FILSPARI's role in IgA nephropathy (proteinuria reduction, kidney function preservation, patient-reported outcomes) and FSGS (rapid and sustained proteinuria reduction in high-risk patients).
• Regulatory Updates: Submitted an sNDA to modify liver monitoring frequency for FILSPARI, requesting quarterly monitoring instead of monthly, and requested priority review for this submission.

In the third quarter of 2024, Travere Therapeutics saw strong performance in its FILSPARI product. Net product sales of FILSPARI were $61 million in Q3 2024, representing more than a 30% quarter-over-quarter growth. This compares to $33.9 million in net product sales for the same period in 2023. Additionally, the company recognized $1.9 million of license and collaboration revenue, resulting in total revenue of $62.9 million for Q3 2024 compared to $37.1 million in 2023.

• FILSPARI sales are expected to continue growing, with anticipation of milestone payments from CSL Vifor upon FILSPARI's full approval in Europe and market access achievements.
• Anticipate an update on FSGS discussions by the Q4 earnings call, with plans to potentially submit an sNDA for FSGS and aim for full approval next year.
• Confidence in FILSPARI's growth as foundational care in IgAN, leveraging the new label and draft KDIGO guidelines.

• Voluntary Pause of Enrollment in the Phase 3 HARMONY Study for Pegtibatinase due to necessary commercial scale up process improvements.
• Uncertainties in regulatory approvals for FSGS and potential differences between expected and actual outcomes.
• Risks associated with forward-looking statements, including known and unknown risks, uncertainties, and assumptions that may cause actual results to differ materially from forward-looking statements.

Q: Concern about minor dip in PSF metrics between Q2 and Q3, including summer seasonality and training week in September. How are things trending early in Q4?

A: Peter Heerma noted strong growth trajectory, with over 500 new patient start forms in Q3, and encouraging demand uptake in the first month post-full approval, indicating strong Q4 trends.

Q: Regarding PARASOL recommendations and FSGS, how will discussions with FDA focus on proteinuria thresholds, totality of data, and biologic plausibility?

A: Bill Rote stated that the FDA will look at the full data in submission, with proteinuria targets, totality, and biologic plausibility (podocyte-driven disease and Sparsentan's effect) being key elements.

Q: Scope of additional data on liver monitoring for FILSPARI beyond clinical experience?

A: Bill Rote mentioned data from ongoing clinical trials, open label extensions for DUET and DUPLEX, and commercial experience, showing consistent low levels of LFT elevations and no drug-induced liver injury.

Q: What will be proposed during Type C meeting with FDA and key questions to ask?

A: Bill Rote stated they will leverage PARASOL discussions, propose thresholds like 0.7 gram per gram per day, and ask the FDA what additional data is needed in the submission.

Q: Include data from open label extension in FSGS Type C meeting and post-hoc analysis needs?

A: Bill Rote said open label extension data from DUET and DUPLEX will be included, and pre-specified threshold proteinuria cut points were already analyzed, with no extensive post-hoc analysis planned ahead of the meeting.

Q: Patient and physician education efforts regarding KDIGO draft guidelines for earlier diagnosis and treatment?

A: Peter Heerma mentioned physicians are showing higher urgency and call to action due to KDIGO guidelines, and FILSPARI's data shows consistent benefit across patient populations.

Q: Broader opportunity for FSGS, market building, and challenges for new entrants?

A: Peter Heerma and Jula Inrig discussed FSGS having high unmet need, same prescribers as IgAN, strong brand awareness, and potential rapid uptake if approved. Jula noted FSGS patients have few effective treatments, making FILSPARI a significant opportunity.

Q: Thoughts on KDIGO updating FSGS treatment guidelines in response to PARASOL observational data?

A: Jula Inrig stated KDIGO typically updates guidelines with new treatments or threshold changes, and full approval of Sparsentan for FSGS would be an appropriate time for an update.

Q: Compliance/adherence metrics for FILSPARI with monthly REMS monitoring and change with reduced frequency?

A: Peter Heerma noted high compliance and persistency rates for FILSPARI, attributing it to patient experience of reaching targets and remission, and the company would ensure continued support and engagement with reduced monitoring frequency.

Q: Payer reimbursement for FILSPARI in IgAN below 1 gram proteinuria, given trial enrolled above that?

A: Peter Heerma mentioned payers are recognizing progressive nature of disease, ongoing education, and some authorization criteria are updating, with good uptake expected early in 2025.

Q: Expectations for LOE in IgAN and orphan drug exclusivity?

A: Eric Dube stated current planning assumption for LOE with sparsentan is into 2033, and additional orphan drug exclusivity term from full approval in IgAN takes them to September 2031.

Q: Update on message reach to community vs academic nephrologists for FILSPARI launch?

A: Peter Heerma mentioned targeting ~6,000 nephrologists out of ~10,000 in US, with strong feedback from academia and community physicians, and FILSPARI positioned as foundational care aligning with KDIGO guidelines.