Travere Therapeutics Announces Positive Topline Results from Cohort 6 in the Phase 1/2 COMPOSE Study of Pegtibatinase in Classical Homocystinuria

Data from the four patients treated with the highest dose of pegtibatinase showed a clinically meaningful 67.1% mean relative reduction in total homocysteine from baseline

Pegtibatinase has been generally well-tolerated to date

The Company is engaging with regulators and expects to initiate a pivotal Phase 3 Study by year-end 2023

Company to host conference call and webcast today at 8.30 a.m. ET

SAN DIEGO, May 31, 2023 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced positive results from cohort 6 in the placebo-controlled Phase 1/2 COMPOSE Study of pegtibatinase, a novel investigational enzyme replacement therapy being evaluated for the treatment of classical homocystinuria (HCU). In this cohort, five patients were randomized in a blinded fashion to receive 2.5 mg/kg of lyophilized pegtibatinase or placebo twice weekly (BIW), with four patients assigned to the treatment group. In this highest dose cohort to date, treatment with pegtibatinase resulted in rapid and sustained reductions in total homocysteine (tHcy), with a 67.1% mean relative reduction in tHcy from baseline, as well as maintenance of mean tHcy below the clinically meaningful threshold of 100 μM, over weeks 6 to 12. To date in the study, pegtibatinase has been generally well-tolerated.

“The results from cohort 6 in the Phase 1/2 COMPOSE Study build upon the dose dependent response observed in cohorts 1-5 and continue to demonstrate pegtibatinase has the potential to improve overall metabolic function in patients living with classical homocystinuria, a rare and devastating disease,” said William Rote, Ph.D., senior vice president of research and development at Travere Therapeutics. “Our goal is to deliver pegtibatinase as the first disease-modifying treatment option for the HCU community and today we are one step closer. We look forward to continuing to engage with regulators to further refine our plans for a potential pivotal Phase 3 study which is expected to initiate by year-end.”

“The current standard of care used to reduce homocysteine levels in individuals living with classical homocystinuria is a highly restrictive diet combined with vitamin B6 and betaine – and this presents significant challenges in maintaining metabolic control of homocysteine levels. As a result, many of these individuals continue to be at a high risk of having toxic levels of homocysteine in their bodies that often results in serious and long-term complications,” said Can Ficicioglu M.D., Ph.D., Clinical Director of the Metabolic Disease Program at Children’s Hospital of Philadelphia. “The impressive reduction of total homocysteine observed with the highest dose of pegtibatinase in the Phase 1/2 COMPOSE Study is very encouraging and offers hope for a potential novel approach to managing this devastating rare disease.”