New Positive 12-Month Extension Data: Ecopipam for Tourette Syndrome in Pediatric Patients

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Ecopipam for Tourette syndrome (TS) was found to be safe and effective in a 12-month, open-label extension (OLE) of a phase 2b study. Ecopipam 1.8 mg/kg/day reduced tic severity and improved quality of life in children and adolescents with TS, with no evidence of tachyphylaxis. This is the largest study to date that examines the long-term safety and effectiveness of ecopipam in a pediatric population with TS.¹

Ecopipam is a novel dopamine-1 receptor antagonist and first-in-class investigational compound that is being studied as a potential treatment for central nervous system disorders, including TS in pediatric patients, for which is has received Orphan Drug and Fast Track designation from the US Food and Drug Administration (FDA).²

"If approved by regulatory agencies, ecopipam could reduce pain and social stigma associated with moderate to severe TS in children and adolescents by reducing frequency and intensity of tics," Donald L. Gilbert, MD, MS, FAAN, FAAP, FCNS, the study's lead author, told Psychiatric Times. Gilbert is a pediatric movement disorders and Tourette syndrome specialist, as well as a professor in the Department of Pediatrics at the University of Cincinnati. "Evidence to date suggests that with ecopipam, tic reduction in children and adolescents with TS can occur and be maintained over time without worsening symptoms of other commonly cooccurring conditions in TS like ADHD, OCD, anxiety, or depression."

Study investigators enrolled 121 participants (74% male) aged 6-18 years (68% aged 12-18 years) diagnosed with TS who completed a phase 2b randomized, placebo-controlled, 12-week trial. Of that 121, 80 (66%) completed the study. Ecopipam was titrated over 4 weeks to achieve a once-daily target oral dose of 1.8 mg/kg/day. Study visits occurred at baseline, monthly for 12 months, and 7 and 14 days after last dose.

Ecopipam was well tolerated. The most common adverse events were nasopharyngitis (14.0%) and anxiety (9.1%). No new adverse events were detected. At month 12, there were no significant changes from baseline in body mass index Z-score (mean [standard deviation] change, 0.05 [0.43]; P = 0.35), glycated hemoglobin (0.03% [0.31]; P = 0.60), or total cholesterol (0.2 mmol/L [0.7]; P = 0.14). No notable changes in scales assessing akathisia, movement disorders, anxiety, or depression occurred. At all time points, investigators observed significant improvements (P < 0.001 vs baseline) in both the Yale Global Tic Severity Scale Total Tic Score and the Gilles de la Tourette Syndrome Quality of Life Scale for Children and Adolescents total score.

Study limitations included the lack of a control arm, the exclusion of adults with TS, and limited racial and ethnic diversity within the study population. Additionally, only a third of participants enrolled in the trial were aged 6 to 11 years; this could be due to lower tic severity and hesitation to place younger patients on investigational medications.

Overall, this data suggests ecopipam may be a safe and effective alternative to currently available pharmacotherapies for treatment of tics in pediatric patients with TS.

Earlier this year, topline data from the phase 3 D1AMOND study showed that ecopipam significantly reduced relapse rates in patients with TS. A total of 167 pediatric participants and 49 adult participants with TS were enrolled at study sites in the US, Canada, and the European Union. Those who experienced clinically meaningful reductions in vocal and motor tics while receiving ecopipam during a 12-week open-label period were randomized to either continue on ecopipam or be switched to placebo in a 12-week double-blind withdrawal period.³

The primary efficacy endpoint was time to relapse for pediatric participants following randomization to ecopipam or placebo. The study found 41.9% of participants randomized to ecopipam relapsed and 68.1% of participants randomized to placebo relapsed. The results were statistically significant (P = 0.0084) with a hazard ratio of 0.5 (0.3-0.8). The secondary efficacy endpoint was time to relapse for both pediatric and adult participants following randomization to ecopipam or placebo. Investigators found 41.2% of participants randomized to ecopipam relapsed and 67.9% of participants randomized to placebo relapsed. The results were statistically significant (P = 0.0050) with a hazard ratio of 0.5 (0.3-0.8).

“These results strengthen our confidence in ecopipam as a potential first-in-class treatment for patients with Tourette syndrome,” said Frederick Munschauer, MD, chief medical officer of Emalex Biosciences. “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 with placebo.”

The most common adverse events related to ecopipam therapy were somnolence (10.2%), insomnia (7.4%), anxiety (6.0%), fatigue (5.6%), and headache (5.1%).

“The Emalex team worked closely with physician investigators and patient advocates throughout the drug development process and we are hopeful that ecopipam can provide symptomatic relief from the tics suffered by patients with Tourette syndrome,” said Eric Messner, the CEO of Emalex Biosciences. “We are entering a new era of progress for people with central nervous system conditions with limited or no treatment options and we look forward to developing innovative new options for them.”

References

1. Gilbert Dl, Kim DJB, Miller MM, et al. Safety and effect of 12-month ecopipam treatment in pediatric patients with Tourette syndrome. Mov Disord Clin Pract. 2025. Online ahead of print.

2. Emalex Biosciences receives FDA Fast Track Designation for ecopipam for the treatment of patients with Tourette syndrome. News release. August 28, 2019. Accessed June 17, 2025. https://emalexbiosciences.com/news/emalex-biosciences-receives-fda-fast-track-designation-for-ecopipam-for-the-treatment-of-patients-with-tourette-syndrome/

3. Emalex Biosciences’ lead candidate meets primary and secondary endpoints in phase 3 Tourette syndrome study. News release. February 25, 2025. Accessed June 17, 2025. https://emalexbiosciences.com/news/emalex-biosciences-lead-candidate-meets-primary-and-secondary-endpoints-in-phase-3-tourette-syndrome-study/