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.
Travere Looks Ahead To Phase III In Homocystinuria
Opportunity Bolstered By Competitor’s Recent HCU Failure
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.
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