Ecopipam significantly reduces motor, vocal tics in Tourette syndrome

Ecopipam, a novel investigational compound for central nervous system disorders significantly reduced symptoms of Tourette syndrome in a cohort of pediatric patients, according to the manufacturer.

Results of an open-label extension study were presented at the International Congress of Parkinson’s Disease and Movement Disorder Society in Copenhagen in August.

According to a release from Emalex Biosciences, the clinical trial included 121 young patients with the disorder and evaluated the safety profile of ecopipam, a first-in-class dopamine-1 receptor antagonist, for up to 12 months. The study found no new safety concerns with respect to adverse events, physical exams, vital signs, laboratory results and safety scales, the company said in the release.

Further, a durable effect was observed over the course of the study based on 3-month evaluations of participants’ motor and vocal tics, per the release.

All participants previously completed a phase 2b study that included more than 150 children and adolescents that found ecopipam significantly reduced the total number of motor and vocal tics compared to placebo and was generally well tolerated with few adverse events recorded.

On the heels of the positive OLE results, Emalex is conducting a phase 3 clinical trial of ecopipam at more than 90 sites across North America and Europe.

“The fact that most patients who completed the Phase 2b study asked to participate in the open-label extension points to the unmet medical needs for people with Tourette syndrome,” Emalex Biosciences Chief Medical Officer Frederick Munschauer, MD, said in the release. “The 12-month data supports the continued development of ecopipam as a first-in-class novel therapy for children and adolescents with Tourette syndrome.”

Editor's Note: The story was updated to clarify that ecopipam is a dopamine-1 receptor antagonist.