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Travere Looks Ahead To Phase III In Homocystinuria

Opportunity Bolstered By Competitor’s Recent HCU Failure

 
• By Joseph Haas

The latest data from a Phase I/II study shows that pegtibatinase can yield a rapid, sustained reduction in homocysteine, the protein behind the underlying cause of the rare disease.

Diet
HCU patients are hoping for a therapy that can loosen dietary restrictions • Source: Shutterstock

Travere Therapeutics, Inc. thinks it is ready to move its second pipeline asset into a pivotal Phase III program, after reporting data from a Phase I/II study of pegtibatinase (TVT-058) that showed rapid and sustained reductions of homocysteine in homocystinuria (HCU) patients. The San Diego-based company hopes its drug can be the first disease-modifying therapy for a rare genetic disease that can result in life-threatening thrombotic events.

The data readout comes from the sixth cohort of a placebo-controlled, dose-escalation study of pegtibatinase – a pegylated recombinant enzyme replacement therapy (ERT) – that previously yielded proof-of-concept data in 2021 with lower doses. It also follows a negative readout from a direct competitor’s program: Aeglea BioTherapeutics, Inc. suspended development of its AGLE-177 (pegtarviliase) in April after reviewing interim data from a Phase I/II study in HCU patients

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