Ecopipam Provides Clinically Meaningful Reduction in Tic Severity for Children With Tourette Syndrome

Children with Tourette syndrome treated with ecopipam (Emalex Biosciences, Chicago, IL) vs placebo had statistically significant and clinically meaningful reductions in tic severity during the phase 2b D1AMOND study (NCT04007991). Tic severity was measured by change from baseline at all timepoints, from week 4 to 12 in the Yale Global Tic Severity Score-Total Tic Score (YGTSS-TTS). Statistically significant improvements in the Clinical Global Impression of Tourette Syndrome Severity (CGI-TS-S) were also observed with ecopipam vs placebo from week 6 to 12.

"After decades of research in pediatric neurology and psychiatry, it is extremely gratifying to achieve positive and meaningful study results for a patient group that has such a high unmet medical need," said Donald Gilbert, MD, MS, pediatric movement disorders and Tourette Syndrome specialist, and professor of neurology, Cincinnati Children's Hospital Medical Center. "We are one step closer to a potentially life-changing therapy for a patient group who inspires and fuels our efforts." 

The most frequent adverse events (≥ 5% and higher than placebo) were headache, fatigue, somnolence, and restlessness. For less commonly reported adverse events, a slightly higher proportion of ecopipam-treated patients experienced depression when compared with placebo, and several children treated with ecopipam reported anxiety.

The D1AMOND study was a randomized, placebo-controlled trial in which participants (n= 153, age 6 to <18 years) were randomly assigned to receive either ecopipam   2 mg/kg/day or placebo. Study medication was titrated to the target dose over 4 weeks and then maintained for an 8-week treatment period. Ecopipam is an investigational, first-in-class drug that selectively blocks the actions of the neurotransmitter dopamine at the D1 receptor.