Travere Therapeutics (TVTX) 2025 Conference Transcript

Summary of Travere Therapeutics (TVTX) Conference Call Company Overview - Company: Travere Therapeutics - Focus: Rare diseases, specifically targeting IgA nephropathy and FSGS (Focal Segmental Glomerulosclerosis) [3][4] Key Programs and Strategic Focus - Filspari: Launched for IgA nephropathy, aiming to be the foundational therapy [3] - FSGS Program: Supplemental NDA accepted with a PDUFA date of January 13, 2026 [3][5] - Classical Homocystinuria (HCU): Phase three program set to initiate enrollment next year [4] Regulatory Insights - Advisory Committee (AdCom): Requested by the FDA for FSGS; reasons include questions around endpoints and safety [5][6] - Endpoints: Proteinuria identified as a valid surrogate endpoint for kidney failure risk, contrary to eGFR [10][11] - FDA Feedback: Consistent with previous submissions; focus on phase three results in context of Parasol [12][13] Market Opportunity - FSGS Patient Population: Over 30,000 diagnosed patients in the U.S. not on dialysis, facing significant risk of kidney failure [19][20] - Therapeutic Need: Current therapies are ineffective for many patients; Filspari shows a consistent benefit in reducing proteinuria by approximately 50% [19][20] - Rapid Uptake Expected: Anticipated due to high unmet need and lack of alternative therapies [20][21] Commercial Dynamics - IgA Nephropathy Launch: Sustained growth with over 700 new patient start forms per quarter since full approval [25][26] - Payer Access: Improved since full approval; broad access expected to continue [36] - Competitive Landscape: Atrasentan from Novartis expected to grow the endothelin class, but Filspari maintains a broader indication [27][28][30] Pipeline and Future Plans - HCU Program: Enrollment expected to restart next year [50] - Pipeline Expansion: Focus on rare renal diseases; disciplined approach to new opportunities [51] - Cash Position: $322 million at the end of Q1; runway expected into 2028 and beyond [54] Upcoming Catalysts - Key Dates: PDUFA for REMS modification on April 28; advisory committee for FSGS pending [55] - Market Access Milestones: Anticipated payments from partners for approvals and commercial milestones [54][55] Additional Insights - EMA Involvement: Less than FDA but still engaged; confidence in EU regulatory pathway [15][16] - Combination Therapy: Filspari expected to be used in combination with SGLT2s for enhanced efficacy [34][42] This summary encapsulates the critical points discussed during the conference call, highlighting the company's strategic focus, regulatory insights, market opportunities, commercial dynamics, pipeline developments, and upcoming catalysts.