Travere Therapeutics to Present Abstracts at the Society for Inherited Metabolic Disorders and Genetic Metabolic Dieticians International

Posters to be presented highlighting research investigating pegtibatinase as the first potential disease-modifying treatment for classical homocystinuria

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.

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.

“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 Therapy
Poster: 139
Abstract Category: Innovative Therapies
Symphony Ballrooms 4-7; April 15, 2024, 8-9 p.m. ET

Pegtibatinase, an Investigational Enzyme Replacement Therapy, For The Treatment of Classical Homocystinuria (HCU): Design of the HARMONY Phase 3 Study
Poster: 147
Abstract Category: Innovative Therapies
Symphony Ballrooms 4-7; April 15, 2024, 8-9 p.m. ET

Economic Burden of Classical Homocystinuria in the United States
Poster: 48
Abstract Category: Clinical Care/Research
Symphony Ballrooms 4-7; April 15, 2024, 7-8 p.m. ET

Clinical Burden of Classical Homocystinuria in the United States: A Retrospective Claims Analysis
Poster: 64
Abstract Category: Clinical Care/Research
Symphony Ballrooms 4-7; April 15, 2024, 7-8 p.m. ET

Association Between Homocysteine and Clinical Outcomes in Patients with Classical Homocystinuria: A Systematic Literature Review