Travere Therapeutics Inc. (TVTX) announced a voluntary pause in enrollment for the Phase 3 HARMONY Study evaluating pegtibatinase for the treatment of classical homocystinuria (HCU). This decision follows the Company's determination that the desired drug substance profile was not achieved during the recent scale-up process.
The company is currently notifying all study investigators about the pause in enrollment of new patients until additional material becomes available.
According to the company, the voluntary enrollment pause enables the Company to work to address necessary process improvements in manufacturing scale-up to support commercial scale manufacturing as well as full enrollment in the HARMONY Study. Patients currently enrolled in pegtibatinase studies continue to receive study medication from small scale batches which are unaffected by the scale-up process. Currently enrolled patients will be able to continue on study medication as scheduled for the duration of the trials they are participating in.
The company plans to further assess the necessary commercial process improvements to facilitate the continuation of the Phase 3 program, with the earliest anticipated restart of enrollment in the Phase 3 HARMONY Study expected in 2026. Investments related to clinical enrollment in HARMONY and large-scale production are also expected to be delayed beyond 2025. Alongside a projected decline in costs for the development of sparsentan as the Phase 3 programs progress, the company now estimates that research and development expenses may be reduced by over $30 million in 2025 compared to 2024.
Additionally, the company continues to expect that its cash, cash equivalents, and marketable securities, totaling $325.4 million as of June 30, 2024, will support operations through 2028.
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