Travere Therapeutics initiates Phase 3 trial of pegtibatinase

Travere Therapeutics announced the Company has opened enrollment in the HARMONY Study, a global, randomized pivotal Phase 3 clinical trial of pegtibatinase, a novel investigational enzyme replacement therapy being evaluated for the treatment of classical homocystinuria. Classical HCU is a rare genetic metabolic disorder caused by a deficiency in the enzyme cystathionine beta synthase. The study is designed to determine the safety and efficacy of pegtibatinase in reducing plasma total homocysteine levels, a key treatment goal in classical HCU, compared to placebo in participants who are receiving standard of care. “Classical HCU is a devastating rare disease that often manifests in childhood and can lead to serious complications due to toxic levels of homocysteine. These complications include continuous risk of developing life-threatening thrombotic events, such as heart attack and stroke, skeletal abnormalities, cognitive developmental delays, and intellectual disabilities,” said William Rote, Ph.D., senior vice president of research and development at Travere Therapeutics.