What's Happening?
Travere Therapeutics has presented new data on pegtibatinase at the International Congress of Inborn Errors of Metabolism in Kyoto, Japan. The data from the Phase 1/2 COMPOSE open-label extension study shows significant reductions in disease-related metabolite levels in patients with classical homocystinuria (HCU). Pegtibatinase demonstrated a 53.5% reduction in total homocysteine and a 67.1% reduction in methionine over 50 weeks, maintaining levels below clinical guidelines. The treatment was generally well-tolerated, with no new safety signals observed. Travere plans to restart enrollment in the Phase 3 HARMONY Study in 2026, aiming to establish pegtibatinase as the first disease-modifying therapy for HCU.
Why It's Important?
Classical homocystinuria is a rare genetic disorder with limited treatment options, primarily involving dietary restrictions and supplements. Pegtibatinase offers a potential breakthrough as a disease-modifying therapy, addressing the underlying cause of HCU. This development could significantly improve the quality of life for patients by reducing toxic metabolite levels associated with severe complications. The FDA has granted pegtibatinase several designations, including Breakthrough Therapy and Orphan Drug status, highlighting its potential impact on rare disease treatment.
What's Next?
Travere Therapeutics is on track to restart enrollment for the Phase 3 HARMONY Study in 2026, which will further evaluate the efficacy and safety of pegtibatinase. The study's success could lead to regulatory approval, providing a new therapeutic option for HCU patients. The company continues to collaborate with the rare disease community to advance treatment options and improve patient outcomes.
