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.
“We are pleased to see sustained and clinically meaningful reductions in toxic homocysteine and methionine levels over nearly a year of treatment with pegtibatinase, particularly within the context of an open-label study,” said Jula Inrig, M.D., chief medical officer of Travere Therapeutics. “People living with classical HCU continue to face complications from this rare disease with limited therapeutic options, and we look forward to furthering our development of pegtibatinase as the potential first disease-modifying therapy to address these unmet needs.”
In the double-blind treatment period of COMPOSE, participants in Cohort 6 received placebo (n=1) or the target dose of 2.5 mg/kg twice weekly (n=4) over a 12-week period. In the OLE portion of the trial, the patient on placebo was titrated up to the target dose and one patient discontinued after entering the OLE.
The Company remains on track to restart enrollment in the Phase 3 HARMONY Study evaluating pegtibatinase for the treatment of classical HCU in 2026.
International Congress of Inborn Errors of Metabolism Presentations
Long-Term Safety and Efficacy of Pegtibatinase for Treatment of Classical Homocystinuria (HCU): Data from the Phase 1/2 COMPOSE Open-Label Extension Study
Presentation #: OP12-5
Date: September 3, 2025
Location: Room 3 (Kyoto International Conference Center)
Time: 16:00-17:30 JST
An Open-Label, Prospective, Interventional Study to Determine the Optimal Treatment of Classical Homocystinuria (HCU) in Infants Identified through Newborn Screening in Qatar
Presentation #: OP14-5
Date: September 3, 2025
Location: Room 3 (Kyoto International Conference Center)
Time: 16:00-17:30 JST
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