Travere Therapeutics Pauses Phase 3 HARMONY Study Due to Manufacturing Scale-Up Challenges

Pause in Enrollment:
Travere Therapeutics has voluntarily paused enrollment in the Phase 3 HARMONY Study of pegtibatinase, a treatment for classical homocystinuria (HCU), due to issues in achieving the desired drug substance profile during the manufacturing scale-up process.

Reason for Pause:
The pause was initiated after the company identified that the desired drug substance profile was not met during the scale-up process, necessitating improvements in commercial manufacturing.

Expected Restart:
The company anticipates that the earliest date to restart enrollment in the Phase 3 HARMONY Study will be in 2026, following necessary manufacturing process improvements.

Impact on Costs:
The pause and delay in large-scale production are expected to reduce research and development expenses, particularly as the Phase 3 programs for sparsentan advance towards completion.

Background on Pegtibatinase:
Pegtibatinase is an investigational PEGylated, recombinant enzyme replacement therapy designed to address the underlying cause of classical HCU. It has shown promising results in preclinical studies and has been granted Breakthrough Therapy designation, Rare Pediatric Disease and Fast Track designations by the FDA, as well as Orphan Drug designation in the U.S. and Europe.