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Travere Therapeutics Announces Positive Topline Results from the Ongoing Phase 1/2 COMPOSE Study of Pegtibatinase in Classical Homocystinuria
Treatment with highest dose pegtibatinase led to a clinically meaningful 55% mean reduction in total homocysteine from baseline and was generally
About this update from Travere Therapeutics, Inc.
[{"type":"text","content":"Treatment with highest dose pegtibatinase led to a clinically meaningful 55% mean reduction in total homocysteine from baseline and was generally well-tolerated after 12 weeks of treatment Results provide clinical proof of concept for first potential therapy targeting the underlying enzyme deficiency in classical homocystinuria Company to host conference call and webcast today at 8:30 a.m. ET SAN DIEGO, Dec. 15, 2021 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced positive topline results from the ongoing Phase 1/2 COMPOSE Study of pegtibatinase, a novel investigational enzyme replacement therapy being evaluated for the treatment of classical homocystinuria (HCU). In the highest dose cohort to date evaluating 1.5mg/kg of pegtibatinase twice weekly (BIW), treatment with pegtibatinase resulted in rapid and sustained reductions in total homocysteine (tHcy) through 12 weeks of treatment, including a 55.1% mean relative reduction in tHcy from baseline as well as maintenance of tHcy below a clinically meaningful threshold of 100 μmol. To date in the study, pegtibatinase has been generally well-tolerated. “These promising topline results from the ongoing COMPOSE Study show that pegtibatinase has the potential to improve overall metabolic function and provide clear proof of concept for pegtibatinase as a potential novel therapy for patients living with HCU,” said Bill Rote, Ph.D., senior vice president of research and development at Travere Therapeutics. “With the strength of these data, we are furthering efforts to refine our formulation and explore pegtibatinase dosing, while in parallel engaging with regulators to establish next steps for a pivotal development program designed to ultimately support potential approvals of pegtibatinase for the treatment of HCU.” “Most patients with HCU are at high risk of developing long term and often devastating complications from HCU because they cannot sustain low levels of toxic homocysteine with currently available treatment options,” said Harvey Levy, M.D., senior physician in medicine/genetics, Division of Genetics and Genomics, Boston Children's Hospital, professor of pediatrics, Harvard Medical School. “The data from the ongoing COMPOSE Study are highly encouraging and provide promise for a potentially novel approach to targeting the underlying enzymatic defect ...