JAMA Neurology Publishes Phase 3 Data on D1 Receptor Antagonist Ecopipam in Tourette Syndrome
Phase 3 data show delayed time to relapse and maintained tic improvement with investigational, first-in-class mechanism targeting dopamine pathway
Phase 3 results published in JAMA Neurology showed that ecopipam, an investigational, first-in-class dopamine-1 (D1) receptor antagonist, delayed time to relapse compared to placebo and maintained clinically meaningful tic improvement in subjects with Tourette syndrome for up to 24 weeks.
Emalex Biosciences is developing ecopipam, which targets the D1 receptor, a pathway implicated in tic expression. The mechanism represents a new class of investigational therapies for Tourette syndrome.
In the randomized withdrawal Phase 3 trial, ecopipam significantly delayed the time to relapse compared with placebo in pediatric participants and in the overall study population and maintained clinically meaningful improvements in tic severity for up to 24 weeks of treatment. The results of the study showed that ecopipam significantly delayed the time to relapse compared to placebo in both the pediatric population (HR = 0.5, P = 0.0084) and a combined population of both pediatric and adults (HR = 0.5, P = 0.0050).
