Travere Starts Phase 3 Trial of Pegtibatinase for Treatment of Classical Homocystinuria

  Travere Therapeutics, Inc. (Nasdaq: TVTX) today 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 (HCU). Classical HCU is a rare genetic metabolic disorder caused by a deficiency in the enzyme cystathionine beta synthase (CBS). The study is designed to determine the safety and efficacy of pegtibatinase in reducing plasma total homocysteine (tHcy) levels, a key treatment goal in classical HCU, compared to placebo in participants who are receiving standard of care.

https://www.globenewswire.com/news-release/2023/12/14/2796174/0/en/Travere-Therapeutics-Initiates-Pivotal-Phase-3-Clinical-Trial-of-Pegtibatinase-for-the-Treatment-of-Classical-Homocystinuria-HCU.html