Travere Therapeutics Advances Phase 3 Study for Rare Genetic Disorder Treatment

Travere Therapeutics, Inc. ((TVTX)) announced an update on their ongoing clinical study.

Study Overview: Travere Therapeutics, Inc. is conducting a Phase 3 long-term extension study titled ENSEMBLE to assess the safety, tolerability, and efficacy of Pegtibatinase in participants aged 5 to 65 with classical homocystinuria (HCU) due to cystathionine beta synthase deficiency. This study aims to provide crucial data on the long-term effects of Pegtibatinase, a promising treatment for this rare genetic disorder.

Intervention/Treatment: The study is testing Pegtibatinase, an experimental drug designed to treat classical homocystinuria by targeting and reducing elevated levels of homocysteine in the body, which is the underlying cause of the condition.

Study Design: This interventional study follows a single-group model without masking, meaning all participants receive the treatment, and both researchers and participants are aware of the treatment being administered. The primary purpose is to evaluate the treatment’s effectiveness and safety over an extended period.

Study Timeline: The study began on May 8, 2024, with a primary completion date yet to be determined. The latest update was submitted on April 8, 2025. These dates are significant as they mark the study’s progress and provide a timeline for potential data availability.

Market Implications: This study update could positively influence Travere Therapeutics’ stock performance by reinforcing investor confidence in the company’s pipeline. Successful results could position Travere as a leader in treating rare metabolic disorders, potentially impacting competitors focusing on similar conditions.

The study is ongoing, with further details available on the ClinicalTrials portal.