Travere Posts Topline Data For Pegtibatinase In patients With Rare Metabolic Disorder

Travere Therapeutics Inc TVTX reported topline results from the ongoing Phase 1/2 COMPOSE Study of pegtibatinase for classical homocystinuria (HCU). 

• There were no discontinuations due to treatment-related adverse events. 
• One serious adverse event, moderate acute urticaria (hives), was categorized to be likely related to pegtibatinase treatment but did not lead to treatment discontinuation and resolved following a single dose interruption.
• Pegtibatinase demonstrated dose-dependent reductions in tHcy during the 12 weeks of treatment.
• At the two highest doses, pegtibatinase appeared to reduce tHcy regardless of starting baseline tHcy levels or background therapy.
• In the highest dose cohort to date of 1.5mg/kg BIW, pegtibatinase resulted in rapid and sustained reductions in tHcy, resulting in a maintenance of tHcy below a clinically meaningful threshold of 100 μmol from week 2 through week 12 of treatment.
• In the 1.5mg/kg BIW dose cohort, treatment with pegtibatinase resulted in a mean relative reduction from baseline of 55.1%, compared to a decrease of 4.8% for all patients receiving placebo in the study.
• Related Link: See Why Travere Therapeutics Stock Moving Higher On Tuesday.
• Price Action: TVTX shares are down 4.17% at $25.25 during the market session on the last check Wednesday.