UPDATE: Travere Therapeutics Presents Long-Term Safety And Efficacy Data For Pegtibatinase In Classical Homocystinuria

Presentation details long-term safety and efficacy data on pegtibatinase as a potential treatment for classical HCU

Travere Therapeutics, Inc., (NASDAQ:TVTX) today announced that the Company shared two oral presentations in classical homocystinuria (HCU) at the International Congress of Inborn Errors of Metabolism, taking place September 2-6 in Kyoto, Japan.

New long-term data from Cohort 6 of the Phase 1/2 COMPOSE open-label extension (OLE) Study demonstrated that at the target dose of 2.5 mg/kg twice weekly, participants treated with pegtibatinase in the OLE maintained significant reductions in disease-related metabolite levels, including a 53.5% relative reduction in total homocysteine and a 67.1% relative reduction in methionine over 50 weeks of treatment. Importantly, within the observation period, homocysteine remained significantly below the clinical guidelines of 100 µM. These reductions are clinically meaningful, as the accumulation of these metabolites is associated with toxicity and disease progression. Pegtibatinase was generally well-tolerated, and no new safety signals were observed.