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Travere Therapeutics Initiates Pivotal Phase 3 Clinical Trial of Pegtibatinase for the Treatment of Classical Homocystinuria (HCU)
Potential for pegtibatinase to become first disease-modifying treatment for classical HCU; topline data expected in 2026 SAN DIEGO, Dec. 14, 2023 (GLOBE
About this update from Travere Therapeutics, Inc.
[{"type":"text","content":"Potential for pegtibatinase to become first disease-modifying treatment for classical HCU; topline data expected in 2026\nSAN DIEGO, Dec. 14, 2023 (GLOBE NEWSWIRE) -- 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. “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. Patients and caregivers have limited treatment options, including adherence to highly restrictive diets that often are very challenging to follow and inadequate for maintaining metabolic control of homocysteine levels,” said William Rote, Ph.D., senior vice president of research and development at Travere Therapeutics. “The initiation of the HARMONY Study is an exciting step forward in addressing this significant unmet need and advancing our goal to deliver pegtibatinase as the first disease-modifying treatment option for the classical HCU community.” The HARMONY Study is a global, randomized, multi-center, double-blind, placebo-controlled Phase 3 clinical trial designed to evaluate the efficacy and safety of pegtibatinase as a novel treatment to reduce tHcy levels. The trial is expected to enroll approximately 70 patients (≥12 to ≤65 years of age at screening) with a diagnosis of classical HCU who continue to have tHcy levels ≥50 μM while maintaining their standard-of-care treatment. Participants will be randomized 1:1 to receive 2.5 mg/kg of pegtibatinase or placebo, administered subcutaneously, for a 24-week blinded treatment duration. The primary...