Travere Therapeutics Inc. announced the presentation of new clinical data at the 15th International Congress of Inborn Errors of Metabolism in Kyoto, Japan. The company shared results from two oral presentations on classical homocystinuria (HCU). Notably, long-term data from the Phase 1/2 COMPOSE open-label extension study demonstrated that participants receiving the target dose of 2.5 mg/kg of pegtibatinase twice weekly maintained significant reductions in metabolite levels, including a 53.5% reduction in total homocysteine and a 67.1% reduction in methionine over 50 weeks. The study found pegtibatinase to be generally well-tolerated with no new safety concerns observed. Travere Therapeutics plans to advance the development of pegtibatinase as a potential first disease-modifying therapy for classical HCU, with enrollment in the Phase 3 HARMONY Study slated to restart in 2026.