Travere Therapeutics has voluntarily paused subject enrolment in the Phase III HARMONY Study of pegtibatinase to treat classical homocystinuria (HCU), a rare genetic metabolic disorder.
This decision will facilitate the company to implement crucial process improvements for manufacturing scale-up, aimed at facilitating commercial-scale production and full study enrolment.
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Subjects currently enrolled in pegtibatinase studies will continue to receive their medication from small-scale batches, which remain unimpacted by the scale-up challenges.
These patients will be able to maintain their treatment schedules throughout their respective trials.
The pause in subject enrolment was initiated after the company identified that the desired drug substance profile was not achieved during the recent scale-up process.
Travere is currently informing all study investigators about the decision to halt the enrolment of new subjects until additional material becomes available.
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By GlobalDataThe company is evaluating the required commercial process improvements to resume the Phase III programme, with the earliest expected date for restarting enrolment in the HARMONY Study being 2026.
Consequently, investments previously planned for clinical enrolment in HARMONY and large-scale production are now anticipated to be postponed beyond 2025.
In line with the expected decline in costs associated with the development of sparsentan as its Phase III programmes near completion, Travere Therapeutics projects that research and development expenses may decrease by over $30m in 2025 against 2024.
The Phase III HARMONY Study was initiated in December last year.
This randomised, multi-centre, double-blind, placebo-controlled trial is designed to assess the efficacy and safety of pegtibatinase in HCU patients.
Travere Therapeutics research and development senior vice-president Bill Rote said: “We believe the potential for pegtibatinase to become the first disease-modifying therapy for classical HCU remains unchanged. Our internal team and external manufacturing partners have extensive biologics expertise, and we are confident that we will be able to make the necessary manufacturing process improvements to continue with this pivotal programme.
“We remain committed to supporting the patients currently enrolled in our clinical trials while working with our manufacturing partners to optimise our commercial-scale manufacturing process.”
