Travere Therapeutics announces anticipated 2024 milestones

FILSPARI is an endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy at risk of rapid disease progression, generally a UPCR greater than or equal to1.5 g/g. In 2024, the Company anticipates conversion of FILSPARI from accelerated approval to full approval for IgAN as well as a potential approval for the treatment of IgAN in Europe. The Company is also generating additional data that is expected to support the clinical profile of FILSPARI and it anticipates updates to treatment guidelines for IgAN to include FILSPARI. FILSPARI – IgAN: In the fourth quarter of 2023, the Company received 459 new patient start forms, reflecting continued strength in physician demand. Preliminary net product sales of FILSPARI in the fourth quarter of 2023 were $15 million; $30 million since the beginning of commercial launch in February 2023. The Company is on track to submit a supplemental New Drug Application (sNDA) in the first quarter of 2024 for conversion of the existing U.S. accelerated approval of FILSPARI to full approval. Following submission of the two-year results from the PROTECT Study of FILSPARI in IgAN and a corresponding procedural review clock-stop, the Company and its collaborator CSL Vifor anticipate a review opinion by the Committee for Medicinal Products for Human Use on the potential approval of the Conditional Marketing Authorization application for sparsentan for the treatment of IgAN in Europe in the first quarter of 2024. If approved, sparsentan would receive CMA in all member states of the European Union, as well as in Iceland, Liechtenstein, and Norway. In 2024, the Company expects additional data from its ongoing open-label studies evaluating the safety and efficacy of sparsentan in combination with sodium glucose cotransporter-2 inhibitors as well as from the ongoing SPARTAN Study evaluating the potential effect of FILSPARI as a first-line therapy in patients with newly diagnosed IgAN. In 2024, the Company anticipates inclusion of FILSPARI into the Kidney Disease Improving Global Outcomes Clinical Practice Guideline for the Management of Glomerular Diseases. Sparsentan – Focal Segmental Glomerulosclerosis: In 2024, the Company is conducting additional analyses of FSGS data and will engage with regulators to evaluate potential regulatory pathways for a sparsentan FSGS indication. Pegtibatinase – Classical HCU: The Company is advancing pegtibatinase, a novel investigational enzyme replacement therapy with the potential to become the first and only disease-modifying therapy for people living with classical homocystinuria: 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 levels. The trial is expected to enroll approximately 70 patients with a diagnosis of classical HCU and tHcy levels greater than or equal to50 muM while maintaining their standard-of-care treatment. Participants will be randomized 1:1 to receive 2.5 mg/kg of pegtibatinase or placebo, administered subcutaneously, for a 24-week blinded treatment duration. The primary endpoint is relative geometric mean change in plasma tHcy levels from baseline compared to weeks 6 through 12. Durability of treatment response through 24 weeks of treatment will also be measured as a secondary endpoint. Topline results from the HARMONY Study are expected in 2026. The Company will also be initiating the ENSEMBLE Study, a Phase 3b, open-label, long-term extension, that will evaluate the ongoing efficacy and long-term safety of pegtibatinase in participants with HCU following their completion of the COMPOSE Study or the HARMONY Study. ENSEMBLE will include an optional protein tolerance modification sub-study that will evaluate if patients can increase their natural dietary protein intake and maintain an acceptable level of metabolic control while receiving pegtibatinase.