Travere Therapeutics, Inc. operates as a biopharmaceutical company.
The company focuses on identifying, developing and delivering life-changing therapies to people living with rare kidney and metabolic diseases. The company’s approach centers on advancing its pipeline with multiple late-stage clinical programs targeting rare diseases with significant unmet medical needs. In September 2024, the U.S. Food and Drug Administration (FDA) granted full approval to the company’s lead development progra...
Travere Therapeutics, Inc. operates as a biopharmaceutical company.
The company focuses on identifying, developing and delivering life-changing therapies to people living with rare kidney and metabolic diseases. The company’s approach centers on advancing its pipeline with multiple late-stage clinical programs targeting rare diseases with significant unmet medical needs. In September 2024, the U.S. Food and Drug Administration (FDA) granted full approval to the company’s lead development program, FILSPARI (sparsentan), which is indicated to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression.
The company also advancing pegtibatinase, a novel investigational enzyme replacement therapy for the treatment of HCU, a genetic disorder caused by a deficiency in a pivotal enzyme essential to the body. The company is conducting a pivotal Phase 3 study to support the potential approval of pegtibatinase as the first disease modifying therapy for HCU. In addition, it continues to evaluate potential opportunities to expand its pipeline and approved products through licenses and acquisitions of products in areas that will serve rare disease patients with serious unmet medical need.
Strategy
The company’s strategies are to focus on developing products to treat rare diseases characterized by severe unmet medical needs; leverage the company’s commercialization expertise to effectively deliver the therapies the company develops; develop a sustainable pipeline by employing disciplined decision criteria in the evaluation of potential in-licensing candidates; listen to patients; and support earlier diagnosis.
Pipeline and Approved Products
The company has a diversified pipeline designed to address areas of high unmet need in rare kidney and metabolic diseases.
FILSPARI (sparsentan)
On September 5, 2024, the FDA granted full approval of FILSPARI (sparsentan) to slow kidney function decline in adults with primary IgAN who are at risk of disease progression. FILSPARI had previously been granted accelerated approval in February 2023 based on the surrogate marker of proteinuria. Full approval was based on positive long-term confirmatory results from the PROTECT Study demonstrating that FILSPARI significantly slowed kidney function decline over two years compared to irbesartan.
In April 2024, the company and its partner CSL Vifor announced that the European Commission has granted conditional marketing authorization (‘CMA’) for FILSPARI (sparsentan) for the treatment of adults with primary IgAN with a urine protein excretion =1.0 g/day (or urine protein-to-creatinine ratio =0.75 g/g).
In January 2024, the company announced its entry into an exclusive licensing agreement with Renalys Pharma, Inc. (Renalys), to bring sparsentan, for the treatment of IgAN, to patients in Japan and other countries in Asia.
Clinical-Stage Programs:
Sparsentan for the treatment of FSGS
Sparsentan has been granted Orphan Drug Designation for the treatment of FSGS in the U.S. and the EEA.
FSGS is a leading cause of kidney failure and nephrotic syndrome. Every year approximately 5,400 patients are diagnosed with FSGS, and the company estimates that there are more than 40,000 FSGS patients in the United States and a similar number in Europe with approximately half of them being candidates for sparsentan.
In 2016, the company generated positive data from its Phase 2 DUET study in FSGS. In 2018, the company announced the initiation of the Phase 3 clinical trial designed to serve as the basis for an NDA and MAA filing for sparsentan for the treatment of FSGS (the ‘DUPLEX Study’). The DUPLEX Study is a global, randomized, multicenter, double-blind, parallel-arm, active-controlled clinical trial evaluating the safety and efficacy of sparsentan in 371 patients. The DUPLEX Study protocol provided for an unblinded analysis of at least 190 patients to be performed after 36 weeks of treatment to evaluate the interim efficacy endpoint the proportion of patients achieving a FSGS partial remission of proteinuria endpoint (FPRE), which is defined as urine protein-to-creatinine ratio (UPCR) =1.5 g/g and a >40% reduction in UPCR from baseline, at week 36.
In February 2021, the company announced that the ongoing Phase 3 DUPLEX Study achieved its pre-specified interim FSGS partial remission of proteinuria endpoint following the 36-week interim period. After 36 weeks of treatment, 42.0 percent of patients receiving sparsentan achieved FPRE, compared to 26.0 percent of irbesartan-treated patients (p=0.0094). Following engagement with the FDA on the interim proteinuria analysis and a subsequent eGFR data-cut, the company elected to forego the previously planned submission for accelerated approval and pursue a potential traditional approval upon completion of the DUPLEX Study.
In May 2023, the company announced topline primary efficacy results from the pivotal Phase 3 DUPLEX Study of sparsentan in FSGS.
In December 2023, the company announced that it completed a planned Type C meeting with the FDA to discuss results from the Phase 3 DUPLEX Study of sparsentan in FSGS. In February 2025, the company announced that it completed a Type C meeting with the FDA and that the company plans to submit an sNDA around the end of the first quarter of 2025 seeking traditional approval of FILSPARI for FSGS. The sNDA will be based on existing data from the Phase 3 DUPLEX and Phase 2 DUET studies of FILSPARI.
Pegtibatinase
Pegtibatinase is a novel investigational human enzyme replacement candidate being evaluated for the treatment of classical homocystinuria (HCU). Classical HCU is a rare metabolic disorder characterized by elevated levels of plasma homocysteine that can lead to vision, skeletal, circulatory and central nervous system complications. Pegtibatinase has been granted Rare Pediatric Disease, Fast Track and Breakthrough Therapy designations by the FDA, as well as orphan drug designation in the United States and European Union.
In December 2021, the company announced positive topline results from the Phase 1/2 COMPOSE Study, a double blind, randomized, placebo-controlled dose escalation study to assess its safety, tolerability, pharmacokinetics, pharmacodynamics and clinical effects in patients with classical HCU.
In May 2023, the company announced positive topline results from the sixth cohort of the Phase 1/2 COMPOSE Study, which was initiated to inform and refine formulation work for future development and commercial purposes and to further evaluate the dose response curve for pegtibatinase, and to further inform its pivotal development program to ultimately support potential approval of pegtibatinase for the treatment of HCU.
In December 2023, the company initiated the pivotal Phase 3 HARMONY Study to support the potential approval of pegtibatinase for the treatment of classical HCU. The HARMONY Study is a global, randomized, multi-center, double-blind, placebo-controlled Phase 3 clinical trial designed to evaluate the efficacy and safety of pegtibatinase as a novel treatment to reduce total homocysteine (tHcy) levels. In the beginning of 2024, the first patients were dosed in the HARMONY Study.
In September 2024, the company announced a voluntary pause of enrollment in the Phase 3 HARMONY Study.
Preclinical Program:
The company is a party to a collaboration agreement with PharmaKrysto Limited and their early-stage cystinuria discovery program, whereby it is responsible for funding all research and development expenses for the pre-clinical activities associated with the cystinuria program.
Other Commercial Products:
Thiola and Thiola EC (tiopronin)
Thiola and Thiola EC are approved by the FDA for the treatment of cystinuria, a rare genetic cystine transport disorder that causes high cystine levels in the urine and the formation of recurring kidney stones. Due to the larger stone size, cystine stones may be more difficult to pass, often requiring surgical procedures to remove. More than 80 percent of people with cystinuria develop their first stone by the age of 20. More than 25 percent will develop cystine stones by the age of 10. Recurring stone formation can cause loss of kidney function in addition to substantial pain and loss of productivity associated with renal colic and stone passage.
In May 2021, a generic option for the 100mg version of the original formulation of Thiola (tiopronin tablets) became available and in June 2022, a second option for the 100mg version of the original formulation of Thiola (tiopronin tablets) was approved. These generic versions of the original formulation of Thiola have impacted its sales, and these or additional generic versions of either formulation could have a material adverse impact on sales. As of December 31, 2024, several generic options for the 100mg and 300mg versions of Thiola EC have been approved by the FDA and become available. Accordingly, Thiola EC is subject to generic competition.
Sale of Bile Acid Product Portfolio
On July 16, 2023, the company entered into an Asset Purchase Agreement (the ‘Purchase Agreement’) with Mirum Pharmaceuticals, Inc. (‘Mirum Pharmaceuticals’ or ‘Mirum’), pursuant to which Mirum agreed to purchase substantially all of the assets primarily related to its business of development, manufacture (including synthesis, formulation, finishing or packaging) and commercialization of Chenodal and Cholbam (also known as Kolbam, and together with Chenodal, the ‘Products’), collectively, the ‘bile acid business’. This transaction was consummated on August 31, 2023.
Licenses and Royalties
Ligand License Agreement
In 2012, the company entered into a license agreement with Ligand Pharmaceuticals, Inc. (‘Ligand’), granting a worldwide sublicense for the development, manufacture and commercialization of FILSPARI (sparsentan). Under the license agreement, Ligand granted the company a sublicense under certain of its patents and other intellectual property in connection with the development and commercialization of sparsentan. Under the license agreement, Ligand is obligated to transfer to its certain information, records, regulatory filings, materials and inventory controlled by Ligand and relating to or useful for developing sparsentan.
Intellectual Property
FILSPARI
As of February 1, 2025, the company’s patent portfolio for FILSPARI (sparsentan) was consists of six distinct patent families, one of which is exclusively licensed from Ligand (the ‘Ligand patent family’). The other five patent families are owned by Travere (the ‘Travere patent families’).
The first Travere patent family is directed to methods of using sparsentan in the treatment of various kidney diseases, including focal segmental glomerulosclerosis (FSGS) and IgAN, e.g., by achieving a specified urine protein-to-creatinine ratio. As of December 31, 2024, this patent family was consisted of a granted U.S. patent (U.S. Patent No. 10,864,197, which it refers to herein as the 197 patent) and pending patent applications in the U.S., Australia, Brazil, Canada, China, the European Patent Office, Hong Kong, Japan, Korea, New Zealand and South Africa.
The second Travere patent family is directed to methods of using sparsentan for treating hearing loss associated with Alport syndrome. As of December 31, 2024, this patent family was consisted of a granted U.S. patent (U.S. Patent No. 11,207,299) and pending patent applications in the U.S., Australia Brazil, Canada, China, the European Patent Office, Hong Kong, Israel, Japan, Korea, Mexico, New Zealand and South Africa.
The third Travere patent family consists of a pending international patent application, related to methods of treating a kidney disease or disorder consists of sparsentan, and a SGLT2 inhibitor.
The fourth Travere patent family consists of a pending international patent application, related to methods of treating IgA mediated diseases or disorders consists of sparsentan.
The fifth Travere patent family consists of a pending international patent application, related to methods of treating IgA mediated kidney diseases or disorders consists of sparsentan.
The sixth Travere patent family consists of a pending provisional patent application directed to additional methods of use of sparsentan for treating FSGS and IgAN.
Pegtibatinase
As of December 31, 2024, the company’s patent portfolio for pegtibatinase consisted of six distinct patent families, which the company obtained upon its acquisition of Orphan Technologies Limited (now Travere Therapeutics Switzerland GmbH). Orphan Technologies obtained the rights to the first five patent families under an exclusive license agreement with the University of Colorado, while the sixth patent family was owned by Orphan Technologies.
The first three patent families are owned by the University of Colorado and exclusively licensed to Travere Therapeutics Switzerland GmbH (the ‘CU patent families’). The first CU patent family is directed to human cystathionine ß-synthase variants and methods for their production, the second CU patent family is directed to methods of purifying human cystathionine ß-synthase variants, and the third CU patent family is directed to compositions consists of human cystathionine ß-synthase variants and methods of treating homocystinuria. The next two families are co-owned by the University of Colorado and Travere Therapeutics Switzerland GmbH, with the University of Colorado’s interest exclusively licensed to Travere Therapeutics Switzerland GmbH (the ‘co-owned patent families’).
The first co-owned patent family is directed to methods of pegylating human cystathionine ß-synthase variants, while the second co-owned patent family is directed to pharmaceutical formulations consists of pegylated human cystathionine ß-synthase variants and their use in treating homocystinuria. Lastly, the sixth patent family is owned by Travere Therapeutics Switzerland GmbH (the ‘Travere patent family’). The Travere patent family is directed to methods for treating human cystathionine ß-synthase deficiency in patients with elevated homocysteine levels.
Thiola
The company’s patent portfolio for Thiola consists of a patent family which is exclusively licensed from Mission Pharmacal (the ‘Mission patent family’). The Mission patent family is directed to a new formulation of Thiola, known as Thiola EC. As of December 31, 2024, this patent family included a granted U.S. patent (U.S. Patent No. 11,458,104, which it refers to herein as the ‘104 patent) and a pending U.S. patent application. The '104 patent claims a method for treating cystinuria by administering a formulation of tiopronin with food.
Trademarks
The company’s trademark portfolio includes both Travere-owned and Travere-licensed trademarks and consists of various U.S. and foreign registered trademarks and pending trademark applications relating to its company name, its commercial products (FILSPARI, Thiola, and Thiola EC), and sparsentan.
More specifically, as of December 31, 2024, the company’s trademark portfolio included registered U.S. and foreign trademarks for the wordmark ‘Travere Therapeutics’ and its logo, registered U.S. and foreign trademarks relating to FILSPARI (sparsentan), registered U.S. trademarks for both the wordmark ‘TOTALCARE’, and its logo, a registered trademark for both the wordmark ‘TOTAL CARE HUB’ and its logo, and a registered trademark for ‘In Rare for Life’. In addition, under the company’s license agreement with Mission it has an exclusive license to use Mission’s trademarks related to Thiola and Thiola EC, including three registered U.S. trademarks and one registered Canadian trademark for the mark ‘THIOLA’, and one registered U.S. trademark for the mark ‘Thiola EC’, in the United States and Canada.
Manufacturing
STA Pharmaceutical Hong Kong Limited manufactures the active pharmaceutical ingredient for FILSPARI. Catalent Pharma Solutions manufactures FILSPARI and performs primary packaging. PCI Pharma Services performs secondary packaging and serialization for FILSPARI. Mission Pharmacal manufactures Thiola and Thiola EC.
Sales, Marketing and Distribution
In 2024, the company continued to utilize its specialty sales force to market its FDA-approved products in the U.S. Through the company’s deep understanding of patient and healthcare provider needs, the company is able to serve patients living with rare disease that have limited treatment options; drive optimum performance of its marketed products; educate and train healthcare providers about the company’s products and the diseases for which they are approved to treat; support access to and reimbursement coverage for the company’s products without significant restrictions; support compliant use by providing patients with support services and disease education, to the extent and in the manner permitted under applicable laws, to help them utilize the company’s products in a manner consistent with the label and maximize the benefits of treatment; and successfully launch new treatment options once approved.
The company’s U.S. commercial initiatives are designed to support patients living with rare diseases and clinicians treating these patients. The company commercializes its products in the United States with a relatively small specialty sales force. Nephrologists are the primary call point for FILSPARI. The primary call points for Thiola and Thiola EC include urologists and nephrologists.
The company’s sales force is differentiated by its high level of experience, averaging more than 20 years in pharmaceutical sales, including over five years of experience in rare disease. The company’s commercial management and operations team also has an average of more than 20 years of pharmaceutical experience focused on specialty and rare disease.
The company’s marketing and patient access teams, supported by third-party agencies with rare disease experience, drive its commercialization and disease awareness efforts in the United States. Specifically, the company implements a variety of industry accepted programs to educate physicians, including direct-to-physician contact by sales representatives, peer-to-peer educational programs, and participation in targeted medical convention programs.
The company distributes FILSPARI in the United States through two direct to patient pharmacies, and operate Travere TotalCare, pursuant to which the company provides its comprehensive patient support services. This patient support program for FILSPARI in the United States provides services, assistance and resources that help patients understand IgAN, manage the insurance process, fill their prescriptions and initiate treatment.
The company distributes its other products, Thiola and Thiola EC, through one direct to patient pharmacy, Eversana, who also provides its comprehensive patient support services in the United States. This patient support program includes a case-managed approach to patient education, insurance verification and reimbursement support, co-pay and other financial assistance for eligible patients, monitoring and support of adherence, and 24/7 access to pharmacist counseling.
In April 2024, the company and CSL Vifor, with whom the company entered into a license and collaboration agreement (‘License Agreement’) in September 2021, announced that the European Commission granted conditional marketing authorization (CMA) for FILSPARI (sparsentan) for the treatment of adults. CSL Vifor is responsible for all commercialization activities in such licensed territories. The company remains responsible for the clinical development of sparsentan and will retain all rights to sparsentan in the United States and rest of world outside of the licensed territories, provided that CSL Vifor has a right of negotiation to expand the licensed territories into Canada, China, Brazil and/or Mexico.
In January 2024, the company announced its entry into an exclusive licensing agreement with Renalys, to bring sparsentan for the treatment of IgAN to patients in Japan and other countries in Asia. Renalys will hold regional rights to sparsentan for Japan, South Korea, Taiwan, Brunei, Cambodia, Indonesia, Laos, Malaysia, Myanmar, the Philippines, Singapore, Thailand, and Vietnam. Following successful meetings with the Pharmaceuticals and Medical Devices Agency (PMDA) in 2023, Renalys plans to initiate an open label registration study of sparsentan in Japan in the second quarter of 2024 to support potential approval of sparsentan in Japan. Results from the urine protein/creatinine ratio (UP/C) endpoint in the study are expected in the second half of 2025 to support a submission for approval to PMDA. Under the terms of the agreement, Renalys will be responsible for development, regulatory matters, and commercialization in the licensed territories.
Medical Affairs
The company has a global medical affairs team located in the United States and Europe which supports data dissemination, education, external stakeholder engagement and data generation in therapeutic areas relevant to the company’s pipeline and commercial assets. The responsibilities of the company’s medical affairs personnel include execution of real-world evidence studies, scientific exchange with external stakeholders, medical communication through scientific publications and presentations at medical congresses, providing medical information support to HCPs and patients related to the company’s pipeline and its post-approval clinical commitments, organizing medical advisory boards to obtain input from experts and practitioners, and supporting grants for investigator sponsored research and independent medical education programs on medical topics relevant to the company’s products and diseases.
Government Regulation
A portion of the company’s product demand for its approved therapies comes from patients covered under Medicaid, Medicare and other federal and state government-related programs such as TRICARE and the Department of Veterans Affairs, or the VA. As required by Federal regulations, the company provides rebates and discounts in connection with these programs.
The U.S. Foreign Corrupt Practices Act, and similar worldwide anti-bribery laws generally prohibit companies and their intermediaries from making improper payments to government officials for the purpose of obtaining or retaining business. The company’s policies mandate compliance with these anti-bribery laws.
History
The company was incorporated in 2008. It was formerly known as Retrophin, Inc. and changed its name to Travere Therapeutics, Inc. in 2020.
