SAN DIEGO, April 04, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced that the Company will present eight posters in classical homocystinuria (HCU) at Society for Inherited Metabolic Disorders (SIMD) in Charlotte, North Carolina from April 14-17, 2024, and Genetic Metabolic Dieticians International (GMDI) in Charlotte, North Carolina from April 17-20, 2024.

圣地亚哥，2024年4月4日（环球通讯社）--Travere Therapeutics，Inc.（纳斯达克：TVTX）今天宣布，该公司将于2024年4月14日至17日在北卡罗来纳州夏洛特的遗传代谢紊乱学会（SIMD）和2024年4月17日至20日在北卡罗来纳州夏洛特的遗传代谢营养师国际（GMDI）上展示八幅经典高半胱氨酸尿症（HCU）海报。

At SIMD and GMDI, the Company will present the trial design of the pivotal Phase 3 HARMONY Study of pegtibatinase, a novel investigational enzyme replacement therapy for the treatment of classical HCU. Additionally, the Company will share insights on the development of an innovative tool used for dietary management and monitoring in the Phase 3 HARMONY Study and open-label extension ENSEMBLE Study, and the positive results from cohort 6 in the placebo-controlled Phase 1/2 COMPOSE Study of pegtibatinase in classical HCU.

在SIMD和GMDI上，该公司将介绍关键的3期pegtibatinase和谐研究的试验设计，这是一种用于治疗经典HCU的新型研究性酶替代疗法。此外，该公司将分享在第三阶段和谐研究和开放标签扩展集成研究中开发用于饮食管理和监测的创新工具的见解，以及在安慰剂对照的1/2期COMPOSE研究中队列6的积极结果经典HCU中的pegtibatinase。

“Classical HCU is an isolating and devastating rare disorder with very limited treatment options, including adherence to highly restrictive diets, leaving patients and their caregivers with immense challenges,” said Jula Inrig, M.D., chief medical officer of Travere Therapeutics. “We look forward to participating at both of these prestigious metabolic medical meetings, and we are excited about our work pioneering the first potential disease-modifying treatment for the thousands of people affected by classical HCU.” SIMD Poster Presentations Latest Results From the COMPOSE Phase 1/2 Trial For the Treatment of Classical Homocystinuria (HCU) Using Pegtibatinase, a Novel Investigational Enzyme Replacement TherapyPoster: 139Abstract Category: Innovative TherapiesSymphony Ballrooms 4-7; April 15, 2024, 8-9 p.m.

Travere Therapeutics首席医疗官Jula Inrig医学博士说：“经典HCU是一种孤立且毁灭性的罕见疾病，治疗选择非常有限，包括坚持高度限制性饮食，给患者及其护理人员带来巨大挑战。”。“我们期待着参加这两次久负盛名的代谢医学会议，我们为开创数千名受经典HCU影响的人的第一种潜在疾病缓解治疗的工作感到兴奋。”SIMD海报介绍了使用新型研究性酶替代疗法Pegtibatinase治疗经典高半胱氨酸尿症（HCU）的COMPOSE 1/2期试验的最新结果。Poster:139摘要类别：创新疗法Symphony Ballrooms 4-7；2024年4月15日下午8-9点。

ET Pegtibatinase, an Investigational Enzyme Replacement Ther.

ET Pegtibatinase，一种研究性酶替代疗法。