Travere Therapeutics to Present Abstracts at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2024

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]