Ecopipam data at MDS 2026 show early and sustained tic reduction in pediatric Tourette syndrome

Nearly 70% of patients treated with the investigational dopamine D1 receptor antagonist ecopipam achieved a clinically meaningful reduction in tic severity within the first 8 weeks of therapy, according to a pooled post hoc analysis presented at the International Congress of Parkinson's Disease and Movement Disorders (MDS) in Seoul, South Korea.¹ The new analyses, which also include interim long-term open-label data and a subgroup analysis by psychiatric comorbidity, come as the FDA conducts a priority review of Teva's new drug application for ecopipam in pediatric Tourette syndrome.¹

The data address a practical problem in tic management. "Many children with Tourette syndrome do not receive treatment, and among those who do, treatment is often discontinued within a year because symptoms remain inadequately controlled or side effects become difficult to tolerate," said Donald L. Gilbert, MD, MS, a pediatric movement disorders and Tourette syndrome specialist in the Division of Neurology at Cincinnati Children's Hospital Medical Center, who was lead author of the published ecopipam trials.¹⁻³

Ecopipam early response, 18-month extension, and comorbidity data in Tourette syndrome

The pooled post hoc analysis included 292 patients from the phase 2b and phase 3 programs. Overall, 69.8% reached at least a 25% improvement in the Yale Global Tic Severity Scale Total Tic Score (YGTSS-TTS) within 8 weeks of starting ecopipam.¹ Somnolence and headache were the most common adverse events (AEs), and AEs clustered in the first 8 weeks of exposure.¹

An interim analysis of an ongoing 36-month open-label extension (OLE) enrolled 118 children, adolescents, and adults who had completed the phase 3 trial or the phase 2b OLE. Ecopipam was titrated over 3 to 4 weeks to a target maintenance dose, and median exposure was 14.9 months.¹ Mean YGTSS-TTS reduction was 45.6% through 18 months, and investigators reported no new safety signals.¹ The most common AEs were nasopharyngitis, upper respiratory tract infection, anxiety, diarrhea, influenza, pyrexia, and insomnia.¹

A third post hoc analysis compared 129 phase 3 participants with at least 1 co-occurring condition (ADHD, obsessive-compulsive disorder, anxiety, or depression) against 87 without. During the 12-week open-label period, YGTSS-TTS reductions and tolerability were similar between groups.¹

Phase 2b and phase 3 D1AMOND trial results for ecopipam in pediatric Tourette syndrome

The new analyses build on 2 controlled trials. In the 12-week, randomized, double-blind phase 2b D1AMOND trial of 153 pediatric patients, ecopipam produced a significantly greater reduction in YGTSS-TTS than placebo.³

The phase 3 trial used a randomized withdrawal design. Of 216 participants entering open-label stabilization, 104 responders (90 pediatric) were randomized to continue ecopipam or taper to placebo.² Ecopipam reduced pediatric relapse risk vs placebo (hazard ratio, 0.47; 95% CI, 0.26-0.84; P = .008), with a median time to relapse of 4.0 weeks on placebo that was not estimable on ecopipam.² The most common AEs across the study were somnolence (11.1%), anxiety (9.7%), and headache (9.7%).²

Tourette syndrome treatment options and the dopamine D1 receptor mechanism

Tourette syndrome is a chronic neurodevelopmental disorder defined by motor and vocal tics, typically emerging between ages 5 and 10 years.¹ Guidelines from the American Academy of Neurology recommend comprehensive behavioral intervention for tics as an initial option, with α₂-adrenergic agonists and dopamine-blocking antipsychotics among pharmacologic choices.⁴ Antipsychotics such as haloperidol, pimozide, and aripiprazole act largely through D2 receptor blockade and carry risks of weight gain, metabolic changes, and drug-induced movement disorders.⁴

Ecopipam instead selectively blocks the D1 receptor; Teva cites D1 receptor hypersensitivity as a possible contributor to repetitive behaviors in Tourette syndrome.¹ The drug holds Orphan Drug designation, and the company says that, if approved, it would be the first new Tourette syndrome therapy in more than a decade.¹

References

1. Teva Pharmaceuticals. Teva presents new efficacy and safety data with ecopipam, an investigational treatment for pediatric patients with Tourette syndrome. News release. October 2, 2026. Accessed October 2, 2026.

2. Gilbert DL, Atkinson SD, Kim DJB, et al. Efficacy and safety of ecopipam for Tourette syndrome: a phase 3 randomized clinical trial. JAMA Neurol. 2026;83(7):645-653. doi:10.1001/jamaneurol.2026.1431

3. Gilbert DL, Dubow JS, Cunniff TM, et al. Ecopipam for Tourette syndrome: a randomized trial. Pediatrics. 2023;151(2):e2022059574. doi:10.1542/peds.2022-059574

4. Pringsheim T, Okun MS, Müller-Vahl K, et al. Practice guideline recommendations summary: treatment of tics in people with Tourette syndrome and chronic tic disorders. Neurology. 2019;92(19):896-906. doi:10.1212/WNL.0000000000007466