Novel Dopamine-1 Receptor Antagonist Shows Promise for Tourette Syndrome

Topline data were announced from a phase 3 trial evaluating ecopipam, a dopamine-1 receptor antagonist, in pediatric and adult patients with Tourette syndrome.

Tourette syndrome is a chronic neurodevelopmental disorder characterized by motor and vocal tics, both of which significantly impact physical and social functions. By blocking dopamine-1 receptors, ecopipam is expected to reduce the repetitive and compulsive behaviors seen in Tourette syndrome.

The phase 3 D1AMOND trial (ClinicalTrials.gov Identifier: NCT05615220) evaluated the safety, tolerability, and efficacy of ecopipam in pediatric and adult participants with Tourette syndrome. The study included an initial 12 week open-label period followed by a 12 week double-blind withdrawal period.

Participants who experienced clinically meaningful reductions in vocal and motor tics in the open-label portion were randomly assigned to continue on ecopipam treatment or switch to placebo. The primary endpoint was time to relapse in the pediatric population.

Findings showed 41.9% of ecopipam-treated patients relapsed compared with 68.1% of placebo-treated patients (hazard ratio [HR], 0.5; P =.0084). For the secondary endpoint, time to relapse for both pediatric and adult patients, 41.2% of participants taking ecopipam relapsed vs 67.9% of those taking placebo (HR, 0.5; P =.0050).

Ecopipam was well tolerated, with the most common adverse events reported with treatment being somnolence, insomnia, anxiety, fatigue, and headache. 

“These results strengthen our confidence in ecopipam as a potential first-in-class treatment for patients with Tourette syndrome,” said Frederick Munschauer, MD, Emalex Biosciences chief medical officer. “The topline data from our large, multi-national, randomized withdrawal study show a statistically significant benefit for ecopipam in maintaining clinically meaningful reductions in vocal and motor tics for pediatric subjects with Tourette syndrome as compared to placebo.”

This article originally appeared on MPR