Travere Therapeutics is a biotechnology company focused on the discovery, development, and delivery of therapies for rare diseases. The company works with the rare disease community to advance potential treatment options and address unmet medical needs. Its current focus includes developing therapies for rare conditions, including Homocystinuria due to cystathionine beta-synthase deficiency (HCU).

The HARMONY study is a large study exploring whether pegtibatinase can be used to treat classical HCU.

• Total time in study: About 9 months
• Goal: To learn more about classical homocystinuria (HCU), and how safe and effective pegtibatinase may be in patients with HCU
• Phase: This is a Phase 3 study that follows a Phase 1/Phase 2 study called COMPOSE

The study will look at:

• Whether the study drug works
• Any side effects of the study drug.

Learn more: https://hcuconnection.com/trials/3?locale=en