SAN DIEGO, Aug. 22, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced that the Company will present two posters in classical homocystinuria (HCU) at the Society for the Study of Inborn Errors of Metabolism (SSIEM) annual symposium in Porto, Portugal, September 3-6, 2024.
At SSIEM, the Company will present the trial designs of the pivotal Phase 3 HARMONY Study and ENSEMBLE long-term extension study of pegtibatinase, a novel investigational enzyme replacement therapy for the treatment of classical HCU. Additionally, the Company will share the trial design of cohort 7 in the Phase 1/2 COMPOSE Study of pegtibatinase in pediatric participants with classical HCU.
“We believe that pegtibatinase has the potential to effectively replace the deficient CBS enzyme activity, and that it could ultimately become the first disease-modifying therapy for people living with classical HCU,” said Jula Inrig, M.D., chief medical officer of Travere Therapeutics. “We look forward to sharing the study designs of our innovative clinical programs in this rare metabolic disorder at the SSIEM symposium this year.”
SSIEM Poster PresentationsRosa Mota Pavilion, Porto, Portugal
Pegtibatinase, an Investigational Enzyme Replacement Therapy for ClassicalHomocystinuria (HCU): design of the HARMONY and ENSEMBLE Phase 3 studies Poster: EP-013Abstract Category: Clinical studies, patient-reported outcome measures Wednesday, September 4, 2024, 6:15 – 8:15 p.m. GMT+1
COMPOSE Phase 1/2, Dose-Escalation Cohort 7 Design: safety of pegtibatinase inchildren aged ≥5–