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www.retrophin.comTravere Therapeutics is well-positioned for sustainable growth with the recent approvals and strong sales performance of FILSPARI.
The Rare Group Problem Management Plus (PM+) clinical trial, sponsored by Children's National Research Institute, is expected to complete on June 30, 2025. The study evaluates a group-based psychoeducational intervention for parents or guardians of children with rare conditions, aiming to address practical and emotional challenges. The intervention consists of five weekly sessions delivered via telehealth, with a total enrollment of 30 families. The trial is not associated with a drug or device product and is categorized as a behavioral health intervention.
trial completionTravere Therapeutics is conducting a global Phase 3 randomized, placebo-controlled trial of Pegtibatinase in patients aged 12-65 with classical homocystinuria due to cystathionine beta synthase deficiency. The study, enrolling approximately 70 participants, is designed to assess efficacy and safety over a 38-week period. Trial completion is anticipated on January 1, 2026. Pegtibatinase is an investigational enzyme replacement therapy targeting elevated homocysteine levels in this rare metabolic disorder.
trial completionTravere Therapeutics is expected to complete its Phase 3 clinical trial of sparsentan in patients with primary focal segmental glomerulosclerosis (FSGS) by February 1, 2026. The global, randomized, double-blind study compares sparsentan, a dual-acting angiotensin and endothelin receptor antagonist, to irbesartan. The trial's primary completion date refers to the end of the double-blind phase, with an open-label extension to follow. Sparsentan is being developed as a potential treatment for FSGS, a rare kidney disorder.
trial completionTravere Therapeutics is conducting a global Phase 3 clinical trial (PROTECT, NCT03762850) evaluating sparsentan, a dual angiotensin receptor blocker and endothelin receptor antagonist, in patients with IgA Nephropathy at high risk of disease progression. The study compares sparsentan to irbesartan, the current standard of care, with primary endpoints including change in proteinuria and estimated glomerular filtration rate. The trial, which includes an open-label extension and a sub-study with dapagliflozin, is expected to complete on July 1, 2026. Sparsentan is being developed as a potential first-in-class treatment for IgA Nephropathy.
trial completionTravere Therapeutics is conducting the ACAPPELLA natural history study (NCT02998710) to characterize the clinical course of homocystinuria caused by cystathionine beta-synthase deficiency in patients aged 1 to 65. The trial, which began in January 2017 and is currently recruiting, is expected to complete by August 1, 2026. Homocystinuria is a rare inherited metabolic disorder, and the study aims to inform future therapeutic development and clinical management.
trial completionTravere Therapeutics is conducting a Phase 1/2 clinical trial of Pegtibatinase (TVT-058), an enzyme replacement therapy for homocystinuria caused by cystathionine beta-synthase deficiency. The trial, known as COMPOSE (NCT03406611), is expected to complete on December 1, 2026. Pegtibatinase is designed to reduce elevated homocysteine levels in patients with this rare metabolic disorder, potentially offering an alternative to restrictive dietary management. The study has enrolled 32 participants and is currently active but not recruiting.
trial completionTravere Therapeutics is conducting the Phase 3 ENSEMBLE long-term extension study (NCT06431893) to evaluate the long-term safety and efficacy of Pegtibatinase in patients aged 5 to 65 with classical homocystinuria (HCU). The study, which began in April 2024, is expected to complete on January 1, 2027. Pegtibatinase is an investigational enzyme replacement therapy designed to lower homocysteine levels in HCU, a rare metabolic disorder. The trial is currently enrolling by invitation and will provide key data on the drug’s long-term profile.
trial completionTravere Therapeutics is conducting a Phase 2 multicenter, open-label clinical trial evaluating Sparsentan in approximately 67 pediatric patients with various proteinuric glomerular diseases, including FSGS, MCD, IgAN, IgAV, and Alport syndrome. The study aims to assess long-term safety, tolerability, and efficacy, with completion expected on April 12, 2027. Sparsentan is a dual endothelin and angiotensin receptor antagonist being developed for rare kidney disorders.
trial completionThe Phase 2 SPARVASC clinical trial (NCT05630612), sponsored by the University of Edinburgh and involving Travere Therapeutics (TVTX), is expected to complete on September 1, 2027. The study evaluates the efficacy of sparsentan, an endothelin-A and angiotensin-1 receptor antagonist, versus irbesartan in patients with ANCA-associated vasculitis in long-term remission. The trial aims to assess whether sparsentan improves endothelial function and reduces cardiovascular risk in this patient population. Sparsentan is an investigational dual endothelin and angiotensin receptor antagonist being developed for rare kidney and vascular diseases.
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