Travere Therapeutics (TVTX) has announced a voluntary pause in enrollment for its Phase 3 HARMONY study, which is evaluating pegtibatinase for the treatment of classical homocystinuria (HCU). The decision, revealed on September 26, 2024, stems from challenges encountered during the manufacturing scale-up process. The company determined that the desired drug substance profile was not achieved, prompting the enrollment pause to address necessary process improvements to support commercial-scale manufacturing and full enrollment in the HARMONY study.
The HARMONY study is a Phase 3 clinical trial assessing the safety and efficacy of pegtibatinase in patients with classical HCU, a rare inherited metabolic disorder. The trial aims to evaluate the drug's ability to lower homocysteine levels, a key indicator of disease severity. The voluntary enrollment pause will allow Travere to refine its manufacturing processes to ensure consistent product quality and meet the demands of a larger patient population.
Following the announcement, Travere's stock price experienced a decline of $1.03 per share, or 6.85%, closing at $14.01 per share on September 27, 2024. This market reaction has prompted scrutiny from investor rights law firms. Bronstein, Gewirtz & Grossman, LLC, a nationally recognized firm, has initiated an investigation into potential claims on behalf of purchasers of Travere Therapeutics, Inc. securities.
Classical homocystinuria is a rare genetic disorder affecting the metabolism of methionine, an essential amino acid. The condition leads to an accumulation of homocysteine in the blood and urine, causing a range of health problems, including developmental delays, vision problems, blood clots, and skeletal abnormalities. Current treatment options include dietary restrictions, vitamin supplementation, and enzyme replacement therapy, but there remains an unmet need for more effective and convenient therapies.
Pegtibatinase is an investigational enzyme replacement therapy designed to address the underlying cause of HCU by breaking down homocysteine. The Phase 3 HARMONY study is a crucial step in evaluating the potential of pegtibatinase to improve outcomes for patients with this debilitating condition. The company is working to resolve the manufacturing issues and resume enrollment in the HARMONY study as soon as possible.
