Travere Therapeutics Inc. (TVTX) has announced a temporary halt in patient enrollment for its Phase 3 HARMONY Study, which is investigating the use of pegtibatinase for treating classical homocystinuria (HCU). This pause in the study follows the company's assessment that the drug substance profile did not meet expectations during a recent scale-up process.
The company is in the process of informing all study investigators regarding the suspension of new patient enrollments until additional material is available.
Travere Therapeutics emphasizes that this voluntary enrollment pause will allow the company to address critical process improvements in the manufacturing scale-up, which are essential for supporting large-scale commercial production and full patient enrollment in the HARMONY Study. Importantly, patients currently participating in pegtibatinase studies will continue receiving the medication from small-scale batches that remain unaffected by the scale-up issues. These patients will proceed with their scheduled treatments for the duration of their respective trials.
The company aims to evaluate and implement necessary commercial process improvements to resume the Phase 3 program, with a tentative restart for patient enrollment in the HARMONY Study projected for 2026. As a result, investments related to clinical enrollment in HARMONY and large-scale production are expected to be deferred beyond 2025. Furthermore, due to a projected reduction in costs associated with the development of sparsentan as the Phase 3 program advances, Travere Therapeutics forecasts a reduction in research and development expenses by over $30 million in 2025 compared to 2024.
The company also anticipates that its cash reserves, cash equivalents, and marketable securities—totaling $325.4 million as of June 30, 2024—will sustain operations through 2028.
