Financial Data and Key Metrics Changes - The company reported net product sales of $75.9 million for Q1 2025, representing a 90% increase year-over-year and continued sequential growth [27] - Net sales for VILSPARI grew 182% year-over-year and 13% compared to the previous quarter, reflecting strong demand and uptake [6][20] - The net loss for Q1 2025 was $41.2 million, or $0.47 per basic share, compared to a net loss of $136.1 million, or $1.76 per basic share, for the same period in 2024 [32] Business Line Data and Key Metrics Changes - VILSPARI maintained strong momentum with $55.9 million in net product sales for Q1 2025, despite higher gross-to-net discounts due to insurance coverage changes [27][28] - Thiola and Thiola EC contributed $20 million in net product sales for the first quarter [28] - The company expects to receive a $17.5 million milestone payment from CSL V4 due to the conversion of VILSPARI's conditional approval to full approval in Europe [32] Market Data and Key Metrics Changes - The European Commission and MHRA in the UK converted FILSPARI's conditional approvals to full approvals for the treatment of adults with IgA nephropathy, enabling broader access across Europe and the UK [7] - Approximately 75% of nephrologists are now targeting proteinuria below 0.5 grams per gram, with nearly a third targeting even more ambitious goals of 0.3 grams [25] Company Strategy and Development Direction - The company aims to solidify VILSPARI's foundational positioning in IgA nephropathy and unlock additional growth through a potential new indication in FSGS [5] - The company is preparing for a potential launch of VILSPARI for FSGS, which is expected to be a significant opportunity, potentially larger than in IgA nephropathy [10][26] - The company is committed to advancing its investigational therapy PEG T for classical homocystinuria (HCU) and plans to restart patient enrollment in the Phase III HARMONY study next year [10][19] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strategy with strong fundamentals and a clear focus on executing key priorities [11] - The company anticipates continued strong demand for FILSPARI and IgA nephropathy, projecting meaningful growth in net product sales throughout the year [33] - Management is monitoring legislative developments and geopolitical uncertainties but believes that potential tariffs on pharmaceutical products would not have a material impact on VILSPARI [33] Other Important Information - The company reported a decrease in R&D expenses to $46.9 million for Q1 2025, down from $49.4 million in the same period in 2024 [29] - Selling, general, and administrative expenses increased to $72.8 million for Q1 2025, compared to $64.2 million for the same period in 2024, largely due to increased investment in the FILSPARI launch [30] Q&A Session Summary Question: Can you elaborate on any interactions with the agency regarding the sNDA for FSGS? - Management indicated that interactions with the FDA have been consistent and progressing as expected, similar to previous experiences with the IGAN indication [36][38] Question: What do you think the label for FSGS will look like? - The expectation is that the indication will be for the treatment of FSGS in patients ages eight and up, based on the broad inclusion criteria of the duplex study [42][45] Question: How is the recent approval of Novartis' therapy impacting your sales reps in the field? - Management noted that it is early to assess the impact, but continued demand for FILSPARI has been observed, indicating confidence in its efficacy profile [50][52] Question: What is the impact of the removal of the REMS program on new patient starts in IGAN? - Management stated that REMS has not been an obstacle to performance and anticipates that modifications will enhance convenience for patients and physicians [76][78] Question: What is the split between new versus repeat prescribers for FILSPARI? - The split is slightly skewed towards experienced prescribers, with a healthy continuation of new prescribers as well [86]

Core Points - The European Commission has converted the conditional marketing approval of FILSPARI (sparsentan) into a standard marketing authorization for the treatment of IgA nephropathy (IgAN) [1][2] - This decision follows a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) in February 2025 [1][2] - The approval is based on comprehensive data from the phase-III PROTECT study, which demonstrated that FILSPARI significantly slowed kidney function decline compared to irbesartan [1][2][9] Company Overview - CSL Vifor is a global partner specializing in pharmaceuticals and innovative therapies for iron deficiency and nephrology, headquartered in St. Gallen, Switzerland [3] - Travere Therapeutics is a biopharmaceutical company focused on developing therapies for rare diseases, emphasizing the urgent need for treatment options [5] Product Information - FILSPARI is the only Dual Endothelin Angiotensin Receptor Antagonist (DEARA) approved in Europe for IgAN, currently available in Germany, Austria, and Switzerland [3][11] - It is a non-immunosuppressive therapy with high selectivity for the endothelin A receptor and the angiotensin II subtype 1 receptor [10][11] Clinical Study Insights - The PROTECT study is one of the largest interventional studies in IgAN, involving 404 patients and comparing the efficacy of FILSPARI to irbesartan [8][9] - The study met its primary endpoint, showing a mean reduction in proteinuria of 49.8% for FILSPARI compared to 15.1% for irbesartan after 36 weeks [9]

Core Viewpoint - Travere Therapeutics has submitted a supplemental New Drug Application (sNDA) to the FDA for FILSPARI, aiming for it to be the first FDA-approved treatment for focal segmental glomerulosclerosis (FSGS), a rare kidney condition [1][2] Company Overview - Travere Therapeutics is focused on developing therapies for rare diseases, emphasizing the urgent need for effective treatment options for conditions like FSGS [2][8] - FILSPARI is currently approved for slowing kidney function decline in adults with IgA nephropathy and is a non-immunosuppressive oral medication [2][9] Clinical Studies - The sNDA submission is supported by results from the Phase 3 DUPLEX Study and the Phase 2 DUET Study, which are among the largest interventional studies in FSGS [1][6] - The DUPLEX Study achieved its interim endpoint with statistical significance at 36 weeks, showing significant proteinuria reduction and a lower rate of end-stage kidney disease compared to the active control [6][7] - The DUET Study demonstrated a greater than two-fold reduction in proteinuria compared to irbesartan, with a consistent safety profile across trials [7] Regulatory Process - The FDA has 60 days to determine whether to accept the sNDA for review, with an expected notice in the second quarter of 2025 [3] - The FDA has indicated that REMS monitoring for embryo-fetal toxicity is no longer necessary, and the company plans to submit a modification to the REMS [4] Disease Context - FSGS is a rare kidney disorder affecting over 40,000 patients in the U.S., characterized by progressive scarring of the kidney and leading to kidney failure [5] - There are currently no FDA-approved pharmacologic therapies for FSGS, highlighting the significance of FILSPARI's potential approval [5]

Financial Data and Key Metrics Changes - In Q4 2024, net product sales of FILSPARI reached approximately $50 million, representing nearly 40% growth compared to the previous quarter [30] - For the full year 2024, net product sales totaled $226.7 million, an increase of nearly 80% compared to 2023 [39] - The net loss for Q4 2024 was $60.3 million, or $0.73 per basic share, compared to a net loss of $90.2 million, or $1.18 per basic share in Q4 2023 [43] - Total revenue for Q4 2024 was $74.8 million, with $1.2 million recognized from license and collaboration revenue [41] Business Line Data and Key Metrics Changes - FILSPARI was the key growth driver, generating $132.2 million in net product sales for the full year 2024, outperforming benchmarks for the first full year of launch [39] - Thiola and Thiola EC contributed $23.9 million in net product sales for Q4 2024, with a slight decrease attributed to generic competition [40] Market Data and Key Metrics Changes - The company received 693 new patient start forms in Q4 2024, a 37% increase from the prior quarter, indicating growing adoption in the IgAN community [28] - The median starting proteinuria level for patients initiating FILSPARI is now below 1.5 grams per gram, signaling progress in targeting a broader patient population [32] Company Strategy and Development Direction - The company aims to solidify FILSPARI's foundational positioning in IgAN as the only non-immunosuppressive kidney-targeted therapy with demonstrated superiority over historical standards of care [12] - Plans include submitting a supplemental New Drug Application (sNDA) for a potential FSGS indication by the end of Q1 2025 [10] - The company is also focused on optimizing manufacturing for the pegtibatinase program and preparing for a potential launch in FSGS [13] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in FILSPARI's growth trajectory following full approval and highlighted the importance of updated KDIGO guidelines in driving physician behavior [33] - The company anticipates continued strength in demand for FILSPARI and expects to maintain disciplined investments to support growth [46] Other Important Information - The company had cash, cash equivalents, and marketable securities totaling $370.7 million as of December 31, 2024, including net proceeds from a successful financing [45] - The company expects to see a moderate increase in gross to net discounts compared to 2024, but anticipates significant growth in net product sales of FILSPARI in 2025 [46] Q&A Session Summary Question: What are you seeing in terms of patient uptake with the expanded FILSPARI label? - The company reported a 37% increase in new patient start forms, with more use in patients with lower proteinuria levels following full approval [55][56] Question: How might new competitors impact FILSPARI's uptake? - Management remains confident in FILSPARI's profile and growth outlook, noting that competition has not significantly affected sales thus far [63][66] Question: When might guidance for the IgAN setting be provided? - The company is monitoring uptake and will provide further updates but is not yet in a position to offer guidance [75] Question: What is the strategy for business development in the rare renal space? - The company is exploring opportunities to diversify its pipeline while prioritizing the launch of FILSPARI for FSGS [82] Question: What steps remain towards the ultimate goal for full removal of the REMS? - The first step is to modify the REMS to quarterly monitoring, with the next step being the potential removal of the REMS after sufficient patient exposure [90][127] Question: How will pricing be determined for FILSPARI in FSGS? - Pricing strategy will focus on ensuring broad access, with potential for linear pricing based on dosing [129][130] Question: How long will the accelerated approval path in IgAN remain open? - The FDA may eventually determine that the unmet need in IgAN has been sufficiently met, but specifics are difficult to predict [138] Question: Can you provide context on the conversion of patient start forms to patients on therapy? - The company has seen strong fulfillment and compliance rates, with improvements in the transition from patient start forms to therapy [97][100]

Drug Approvals and Efficacy - FILSPARI® (sparsentan) received full FDA approval on September 5, 2024, for slowing kidney function decline in adults with primary IgAN, following accelerated approval in February 2023 [493]. - In the PROTECT Study, FILSPARI demonstrated a significant reduction in kidney function decline with a mean eGFR slope of -3.0 mL/min/1.73 m²/year compared to -4.2 mL/min/1.73 m²/year for irbesartan, resulting in a treatment effect of 1.2 mL/min/1.73 m²/year (p=0.0168) [495]. - The FDA-approved label for FILSPARI includes two-year efficacy data showing a 3.8 mL/min/1.73 m² difference in mean change from baseline in eGFR between FILSPARI and irbesartan [495]. - FILSPARI has been granted seven years of Orphan Drug Exclusivity in the U.S. for both the reduction of proteinuria and slowing kidney function decline in adults with primary IgAN [496]. - The estimated prevalence of IgAN in the U.S. is up to 150,000 patients, with more than 70,000 potentially addressable under FILSPARI's full approval indication [498]. - In April 2024, the European Commission granted conditional marketing authorization for FILSPARI for adults with primary IgAN, with a regulatory milestone payment of $17.5 million expected upon full approval [501]. - The Phase 3 DUPLEX Study for sparsentan in FSGS showed that 42.0% of patients achieved FSGS partial remission of proteinuria compared to 26.0% for irbesartan (p=0.0094) [507]. - The FDA indicated that the two-year results from the DUPLEX Study alone were insufficient for an sNDA submission, prompting a collaborative effort to define alternative proteinuria-based endpoints for FSGS [508]. - The company plans to submit an sNDA for traditional approval of FILSPARI for FSGS by the end of Q1 2025, based on existing data from the Phase 3 DUPLEX and Phase 2 DUET studies [508]. Financial Performance - Total revenue for the year ended December 31, 2024, was $233.2 million, an increase of $87.9 million compared to $145.2 million in 2023, primarily driven by a $103.0 million increase in FILSPARI sales [549]. - FILSPARI sales reached $132.2 million in 2024, compared to $29.2 million in 2023, reflecting a full year of sales following its launch in February 2023 [550]. - Total net product sales increased by $99.2 million to $226.7 million in 2024, while license and collaboration revenue decreased by $11.2 million to $6.5 million [549]. - Operating expenses totaled $557.0 million in 2024, an increase of $23.6 million from $533.4 million in 2023, driven by restructuring costs and in-process research and development [552]. - The company recognized a non-recurring charge of $65.2 million in in-process research and development expense in March 2024 related to pegtibatinase [562]. - The loss from discontinued operations for the year ended December 31, 2024, was $0.9 million, a significant decrease from a gain of $264.9 million in 2023 due to the sale of the bile acid business [567]. - Cash used in operating activities from continuing operations decreased to $230.0 million in 2024 from $325.4 million in 2023, attributed to a $99.2 million increase in total net product sales [602]. - Cash provided by investing activities was $99.3 million in 2024, down from $151.6 million in 2023, due to a decrease in net purchases of marketable debt securities [603]. - Cash provided by financing activities was $139.4 million in 2024, compared to $220.1 million in 2023, influenced by a public offering that generated $134.7 million in net proceeds [604]. Corporate Actions and Workforce Management - The company implemented a 20% workforce reduction in December 2023, expected to result in annualized savings of approximately $25.0 million starting in 2024 [525]. - The company implemented a 20% workforce reduction in December 2023 to align resources for the FILSPARI launch and ongoing clinical studies [569]. - As of December 31, 2024, total non-recurring charges incurred due to restructuring amounted to $13.8 million [525]. - The company currently has four Phase 3 clinical trials in process, with ongoing non-clinical support trials that may impact future expenses [558]. Strategic Partnerships and Licensing - An exclusive licensing agreement with Renalys Pharma was established to bring sparsentan to patients in Japan and other Asian countries, with an open label registration study initiated in 2024 [502]. - The company has a license agreement with CSL Vifor that could yield up to $845.0 million in milestone payments and royalties for the commercialization of sparsentan [576]. - The company received an upfront cash payment of $210.0 million from the sale of its bile acid product portfolio to Mirum Pharmaceuticals [522]. - The company is eligible to receive up to $235.0 million in milestone payments based on specified annual net sales of the bile acid products sold to Mirum [523]. - The company received an upfront cash payment of $210.0 million from the sale of its bile acid business in August 2023, with potential additional payments of up to $235.0 million based on sales milestones [575]. - The company has a contingent payment obligation of up to $427.0 million related to the acquisition of Orphan Technologies Limited, based on development and regulatory milestones [590]. Research and Development - Pegtibatinase demonstrated a 55.1% mean relative reduction in total homocysteine (tHcy) from baseline in the highest dose cohort of 1.5 mg/kg during the Phase 1/2 COMPOSE Study [512]. - In the sixth cohort of the Phase 1/2 COMPOSE Study, treatment with 2.5 mg/kg of pegtibatinase resulted in a 67.1% mean relative reduction in tHcy from baseline [514]. - The pivotal Phase 3 HARMONY Study was initiated in December 2023 to evaluate the efficacy and safety of pegtibatinase for classical HCU [515]. - A voluntary pause of enrollment in the Phase 3 HARMONY Study was announced in September 2024 to address manufacturing scale-up improvements, with plans to restart enrollment in 2026 [516]. Market and Economic Conditions - The company is exposed to interest rate risk, with a potential $1.7 million impact on investments from a 100 basis point change in interest rates [605]. - Inflation has primarily impacted the company through increased labor costs, but it has not affected the current outlook or business objectives [608].

Core Insights - Travere Therapeutics reported a revenue of $74.79 million for Q4 2024, marking a 66% year-over-year increase and exceeding the Zacks Consensus Estimate of $73.44 million by 1.84% [1] - The company posted an EPS of -$0.73, an improvement from -$1.16 a year ago, but fell short of the consensus estimate of -$0.58, resulting in an EPS surprise of -25.86% [1] Revenue Breakdown - Revenue from Tiopronin products (Thiola) was $23.90 million, slightly below the estimated $24.03 million, reflecting a year-over-year decrease of 5.2% [4] - Total net product sales reached $73.55 million, surpassing the estimated $69.22 million [4] - Revenue from FILSPARI was $49.64 million, significantly exceeding the estimated $45.31 million, representing a remarkable year-over-year increase of 237.7% [4] - License and collaboration revenue was reported at $1.24 million, falling short of the average estimate of $4.77 million, indicating a year-over-year decline of 75.9% [4] Stock Performance - Over the past month, Travere's shares have returned +19.9%, outperforming the Zacks S&P 500 composite's +2.6% change [3] - The stock currently holds a Zacks Rank 3 (Hold), suggesting it may perform in line with the broader market in the near term [3]

Group 1 - Travere Therapeutics reported a quarterly loss of $0.73 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.58, representing an earnings surprise of -25.86% [1] - The company posted revenues of $74.79 million for the quarter ended December 2024, exceeding the Zacks Consensus Estimate by 1.84%, and showing a significant increase from $45.06 million in the same quarter last year [2] - Travere shares have increased approximately 33.9% since the beginning of the year, outperforming the S&P 500's gain of 4.5% [3] Group 2 - The current consensus EPS estimate for the upcoming quarter is -$0.54 on revenues of $70.16 million, and for the current fiscal year, it is -$1.40 on revenues of $345.91 million [7] - The Medical - Biomedical and Genetics industry, to which Travere belongs, is currently ranked in the top 27% of over 250 Zacks industries, indicating a favorable outlook compared to the bottom 50% [8]

Company to submit sNDA around the end of 1Q 2025 seeking traditional approval of FILSPARI® (sparsentan) for FSGS Net product sales of FILSPARI totaled $50 million in 4Q 2024; $132 million for full year 2024 Net product sales totaled $74 million in 4Q 2024; $227 million for full year 2024 SAN DIEGO, Feb. 20, 2025 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ: TVTX) today reported its fourth quarter and full year 2024 financial results and provided a corporate update. “Our strong execution in 2024 ma ...

Core Viewpoint - Travere Therapeutics, Inc. is set to report its fourth quarter and full year 2024 financial results on February 20, 2025, after the U.S. financial markets close, followed by a conference call and webcast to discuss the results and provide a business update [1] Company Information - Travere Therapeutics is a biopharmaceutical company focused on developing therapies for patients with rare diseases, emphasizing the urgent need for treatment options [3] - The company collaborates with the rare disease community to identify, develop, and deliver life-changing therapies, aiming to understand the diverse perspectives of rare patients [3]

Travere Therapeutics (TVTX) shares ended the last trading session 12.6% higher at $23.75. The jump came on an impressive volume with a higher-than-average number of shares changing hands in the session. This compares to the stock's 13.5% gain over the past four weeks.The sudden soaring of the stock price was observed following Travere's announcement that it has completed a Type C meeting with the FDA and intends to submit a supplemental new drug application (sNDA) for traditional approval of Filspari in tre ...