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 ...

Core Insights - Travere Therapeutics has completed a Type C meeting with the FDA and plans to submit a supplemental New Drug Application (sNDA) for FILSPARI, targeting focal segmental glomerulosclerosis (FSGS) [1][2] - If approved, FILSPARI could be the first and only approved treatment for FSGS, a rare kidney disorder affecting over 40,000 patients in the U.S. [2][5] - The sNDA submission will rely on data from the Phase 3 DUPLEX and Phase 2 DUET studies, which have shown promising results in reducing proteinuria [1][6][7] Company Updates - The sNDA submission is expected around the end of the first quarter of 2025 [1] - The CEO of Travere Therapeutics expressed optimism about the sNDA submission and highlighted the urgent need for treatment options for FSGS [2] - A conference call and webcast will be held to discuss these updates, scheduled for February 11, 2025, at 8:30 a.m. ET [3][4] Study Insights - The Phase 3 DUPLEX study is noted as the largest interventional study in FSGS, achieving significant results in reducing proteinuria [6][7] - The Phase 2 DUET study demonstrated a greater than two-fold reduction in proteinuria compared to irbesartan, indicating the efficacy of FILSPARI [7] - Both studies support the importance of proteinuria reduction as a key endpoint in FSGS treatment, aligning with findings from the PARASOL workgroup [2][8] Disease Context - FSGS is characterized by progressive kidney scarring and is a leading cause of kidney failure, with no currently approved pharmacologic treatments [5] - The disorder affects both adults and children, highlighting the need for effective therapies [5]