Financial Data and Key Metrics Changes - Net product sales for Q3 2024 reached $61 million, an increase of approximately 80% compared to $33.9 million in Q3 2023, driven by strong performance in the U.S. launch of FILSPARI for IgA nephropathy [60] - Total revenue for Q3 2024 was $62.9 million, up from $37.1 million in the same period last year [61] - Net loss for Q3 2024 was $54.8 million or $0.70 per share, compared to a net income of $150.7 million or $1.97 per share in Q3 2023 [65] Business Line Data and Key Metrics Changes - FILSPARI's net sales grew by more than 30% quarter-over-quarter, with over 500 new patient start forms added during Q3 2024 [8][49] - The company continues to outperform previous rare nephrology launches on all core metrics and is on track to exceed benchmarks for net revenue in the first full year of launch [50] Market Data and Key Metrics Changes - FILSPARI has launched in two key European markets, Germany and Austria, and received temporary marketing approval in Switzerland [16] - The recent draft KDIGO guidelines are expected to drive nephrologists to treat patients more aggressively, which could further enhance FILSPARI's market penetration [55] Company Strategy and Development Direction - The company aims for FILSPARI to become foundational care in IgA nephropathy, leveraging full approval and updated guidelines to educate the nephrology community [9][51] - There is a focus on expanding access to FILSPARI outside the U.S. and preparing for potential sNDA submissions for FSGS indications [15][28] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing demand for FILSPARI, particularly following full approval and the positive reception of new clinical data [48][59] - The company anticipates a decline in cash used over time, supported by increasing FILSPARI sales and expected milestone payments from partners [66] Other Important Information - The company has submitted an sNDA to modify liver monitoring frequency in the REMS, which, if approved, would improve patient access while maintaining safety [11][29] - The PARASOL initiative has provided new hope for the FSGS community, with potential regulatory pathways being discussed with the FDA [41][42] Q&A Session Summary Question: Insights on PSF metrics and trends in Q4 - Management noted that the minor dip in metrics was attributed to seasonal factors and training, but they are encouraged by the strong growth trajectory post-approval [71][72] Question: Focus of discussions with the FDA regarding PARASOL recommendations - Management indicated that while proteinuria can be a validated surrogate for approval, the totality of data will be important in discussions with the FDA [74][76] Question: Additional data on liver monitoring for the submission - The submission includes data from ongoing clinical trials and commercial experience, showing consistent low levels of LFT elevation [79][80] Question: Expectations for the Type C meeting with the FDA - The company plans to propose specific proteinuria thresholds and discuss the overall data package with the FDA [81][83] Question: Inclusion of open-label extension data in the FDA submission - Management confirmed that data from open-label extensions will be included in the submission to the FDA [85] Question: Efforts to help patients get diagnosed and treated earlier - The company is actively educating payers and physicians about the urgency of treating patients earlier based on new guidelines [88] Question: Broader opportunity for FILSPARI in FSGS - Management expressed excitement about the potential market for FILSPARI in FSGS, noting the high unmet need and established prescriber relationships [92][94] Question: Potential updates to FSGS treatment guidelines - Management anticipates that if full approval for Sparsentan is achieved, it could prompt updates to treatment guidelines [96] Question: Compliance metrics for FILSPARI and expected changes with reduced monitoring - High compliance and persistency rates for FILSPARI were reported, with expectations that reduced monitoring frequency will maintain patient engagement [98][100] Question: Payer willingness to reimburse for patients starting below 1 gram of proteinuria - Management noted positive progress with payers recognizing the progressive nature of IgA nephropathy and updating authorization criteria accordingly [102][104] Question: Current expectations for LOE in IgAN - The company expects LOE for Sparsentan to extend into 2033, with additional orphan drug exclusivity extending to September 2031 [106]

or UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 _________________________________ FORM 10-Q _________________________________ ☑ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2024 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________ to ___________ Commission File Number: 001-36257 TRAVERE THERAPEUTICS, INC. (Exact na ...

Exhibit 99.1 Contact: Investors: Media: 888-969-7879 888-969-7879 IR@travere.com mediarelations@travere.com Travere Therapeutics Reports Third Quarter 2024 Financial Results ® FILSPARI (sparsentan) received full FDA approval as the only non-immunosuppressive treatment that significantly slows kidney function decline in IgAN Type C meeting scheduled with FDA to discuss potential sNDA submission for FILSPARI in FSGS sNDA requesting modification of liver monitoring for FILSPARI submitted to FDA Net product sal ...

Company Overview - Travere Therapeutics, Inc. (NASDAQ: TVTX) focuses on treatments for rare kidney and metabolic diseases [1] - The company's portfolio includes FDA-approved drugs such as Filspari (Sparsentan) for IgA Nephropathy (IgAN) and Thiola (Tiopronin) for cystinuria [1] Recent Developments - Filspari received full FDA approval in September 2024, which expanded its label and increased patient access [1]

SAN DIEGO, Sept. 26, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced a voluntary pause of enrollment in the Phase 3 HARMONY Study evaluating pegtibatinase for the treatment of classical homocystinuria (HCU). The voluntary enrollment pause enables the Company to work to address necessary process improvements in manufacturing scale-up to support commercial scale manufacturing as well as full enrollment in the HARMONY Study. Patients currently enrolled in pegtibatinase studi ...

Travere Therapeutics (TVTX) shares rallied 5.9% in the last trading session to close at $14.27. This move can be attributable to notable volume with a higher number of shares being traded than in a typical session. This compares to the stock's 55.3% gain over the past four weeks. The company markets Filspari (sparsentan), an oral non-immunosuppressive drug approved for treating IgA nephropathy (IgAN), a rare kidney disease. Filspari has seen strong market demand, generating $47 million in sales in the first ...

Core Viewpoint - Travere Therapeutics received full FDA approval for its oral nonimmunosuppressive drug Filspari (sparsentan) for the treatment of IgA nephropathy (IgAN), marking it as the only nonimmunosuppressive medication in this space [1] Company Summary - Filspari was initially granted accelerated approval in February 2023 to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression [2] - Following the full approval, Filspari's label expansion allows treatment for a broader patient population, including those at lower risk of disease progression [3] - Travere's shares increased nearly 11% after the announcement, with a year-to-date stock increase of 22.1%, contrasting with the industry's 2.8% decline [3] Clinical Study Insights - The approval was based on the pivotal phase III PROTECT study, which showed that Filspari preserved long-term kidney function compared to irbesartan [5] - The modified intention to treat (ITT) analysis included all patients, even those who stopped treatment, which was favored by the FDA [6] - The PROTECT study achieved a mean eGFR slope of -3.0 mL/min/1.73 m/year for Filspari compared to -4.2 mL/min/1.73 m/year for irbesartan [8] Regulatory and Safety Considerations - Filspari is included in the FDA's Risk Evaluation and Mitigation Strategies (REMS) program, requiring monitoring of liver enzymes before and during treatment [9] - The drug carries a boxed warning for severe birth defects if taken during pregnancy [10] Competitive Landscape - Calliditas Therapeutics dominates the IgAN space with its drug Tarpeyo, which received full FDA approval in December 2023 [11] - Novartis entered the IgAN market with the accelerated approval of Fabhalta for reducing proteinuria, marking its first FDA approval in the renal pipeline [13] - Novartis is also developing additional candidates in the IgAN indication, including atrasentan and zigakibart, following its acquisition of Chinook Therapeutics [14]

FDA approves expanded indication making FILSPARI available to patients with IgA nephropathy (IgAN) at risk of progression; updated label includes data showing long-term durable benefit on proteinuria and kidney function preservation that accrued over two years Conversion to full approval based on results from the PROTECT Study, where FILSPARI delivered superior long-term kidney function preservation compared to the active comparator irbesartan in the only Phase 3 headto-head trial conducted in IgAN As an or ...

Chris Ryan/OJO Images via Getty Images Investment Overview - Background To Upcoming PDUFA - Failed Confirmatory Study Hammers Share Price The last time I covered Travere Therapeutics (NASDAQ:TVTX) it was September 22, 2023, and the San Diego-based biotech's stock price was in free-fall. Earlier that day, the company shared data from a Phase 3 clinical study of its lead asset Filspari (sparsentan), showing that the drug had failed to meet a key primary endpoint, unable to demonstrate a statistically signific ...

Core Insights - Travere Therapeutics will present two posters on classical homocystinuria (HCU) at the SSIEM annual symposium in Porto, Portugal, from September 3-6, 2024 [1][2] - The presentations will include trial designs for the pivotal Phase 3 HARMONY Study and the ENSEMBLE long-term extension study of pegtibatinase, an investigational enzyme replacement therapy for classical HCU [2][3] Company Overview - Travere Therapeutics is focused on developing therapies for rare diseases, particularly classical HCU, which is caused by a deficiency in the cystathionine beta synthase (CBS) enzyme [4][6] - The company aims to provide innovative treatment options for patients with rare diseases, emphasizing the urgency of addressing treatment needs [6] Product Information - Pegtibatinase is a PEGylated, recombinant enzyme replacement therapy designed to address the underlying cause of classical HCU [5] - The HARMONY Study is a global, randomized, multi-center, double-blind, placebo-controlled Phase 3 clinical trial aimed at evaluating the efficacy and safety of pegtibatinase [5] - Preliminary data from the Phase 1/2 COMPOSE Study indicated a 67.1% mean relative reduction in total homocysteine levels from baseline after 12 weeks of treatment with the highest dose of pegtibatinase [5] Clinical Studies - The HARMONY Study and ENSEMBLE long-term extension study will be presented as part of the SSIEM symposium, highlighting their significance in the treatment landscape for classical HCU [2][3] - The COMPOSE Study will also present findings on the safety of pegtibatinase in pediatric participants aged 5 to under 12 years with classical HCU [2][3]