Ecopipam Shows Early and Sustained Tic Reduction in New Pediatric Tourette Syndrome Analyses

Teva Pharmaceuticals has presented new efficacy and safety analyses of ecopipam, an investigational selective dopamine D1 receptor antagonist for pediatric patients with Tourette syndrome, including findings suggesting clinically meaningful reductions in tic severity within the first 8 weeks of treatment and sustained benefit through 18 months of treatment.¹

The data, presented at the International Congress of Parkinson’s Disease and Movement Disorders (MDS) in Seoul, South Korea, included a pooled post hoc analysis of phase 2b and phase 3 data, an interim analysis of an ongoing open-label extension (OLE) study, and analyses examining the impact of common psychiatric comorbidities on treatment outcomes. The findings add to the clinical evidence supporting ecopipam, which is currently under FDA priority review for pediatric Tourette syndrome.¹

In the pooled post hoc analysis, 69.8% of 292 participants across the phase 2b and phase 3 trials achieved a clinically meaningful improvement in the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS), defined as at least a 25% reduction from baseline, within the first 8 weeks of ecopipam treatment. The most commonly reported adverse events (AEs) during this period were somnolence and headache, with the company reporting that cross-trial AEs were most frequentlyobserved during the initial8 weeks of exposure.¹

“Tourette syndrome is a complex neurodevelopmental disorder that presents significant daily challenges for children and their families,” said Eric Hughes, MD, PhD, executive vice president, global R&D and chief medical officer of Teva, in a statement.¹ “These promising new data for ecopipam reinforce our confidence in its potential in pediatric Tourette syndrome care. If approved, ecopipam would be the first new therapy for Tourette syndrome in more than 10 years and the first with a novel mechanism of action in more than 50 years.”

Short-Term and Long-Term Findings

The findings build on results from the 12-week, randomized, double-blind, placebo-controlled phase 2b D1AMOND trial, which enrolled 153 pediatric participants across 68 sites in North America and Europe. In that study, ecopipam produced a statistically significant and clinically meaningful reduction in YGTSS-TTS compared with placebo at week 12 (P = .01). A subsequent 12-month OLE involving 121 pediatric participants suggested that tic control could be maintained over longer-term treatment.²

Additional evidence of maintenance of effect came from the phase 3 D1AMOND randomized-withdrawal study (NCT05615220), which enrolled 216 pediatric and adult participants. After a 12-week open-label stabilization period, responders were randomly assigned to continue ecopipam or switch to placebo for an additional 12 weeks. Among pediatric participants, 41.9% of those who continued ecopipam experienced relapse compared with 68.1% of those switched to placebo, corresponding to a 53% relative reduction in relapse risk (HR, 0.50; P = .008).³

An interim analysis from an ongoing 36-month OLE further evaluated the durability of ecopipam treatment. The analysis included 118 children, adolescents, and adults with Tourette syndrome who received at least 1 dose of ecopipam, with a median treatment exposure of 14.9 months. Participants underwent a 3- to 4-week titration period before being maintained at the target dose.

At 18 months, ecopipam was associated with a mean 45.6% reduction in YGTSS-TTS, with the company reporting that clinically meaningful tic suppression was maintained through the 18-month assessment. No new safety signals were identified in the interim analysis.The most frequently reported AEs in the longer-term analysis included nasopharyngitis, upper respiratory tract infection, anxiety, diarrhea, influenza, pyrexia, and insomnia.

The longer-term findings are particularly relevant given the chronic nature of Tourette syndrome and the need to maintain tic control over time. In the earlier phase 2b OLE, investigators likewise reported sustained tic control through 12 months, providing a longer-term extension of the efficacy findings observed in the randomized portion of the study.

Psychiatric Comorbidities

A separate post hoc analysis evaluated whether commonly occurring psychiatric conditions affected ecopipam's efficacy or safety. The analysis included all 216 participants from the phase 3 trial and compared 129 participants with at least 1 co-occurring condition with 87 participants without these conditions.The comorbidities examined included attention-deficit/hyperactivity disorder (ADHD), obsessive-compulsive disorder (OCD), anxiety, and depression.¹

During the 12-week open-label treatment period, reductions in YGTSS-TTS were consistent between participants with and without co-occurring psychiatric conditions. The safety and tolerability profiles were also generally consistent between the groups.

These findings complement previous analyses from the ecopipam program examining psychiatric symptoms in pediatric patients. Earlier data found no significant differences between ecopipam and placebo on measures of ADHD, anxiety, OCD, or depression, despite the high prevalence of these conditions among children with Tourette syndrome.

A Different Dopaminergic Target

Ecopipam is designed to selectively block dopamine signaling at the D1 receptor, representing a different pharmacologic approach from commonly used dopamine D2 receptor antagonists.¹

Tourette syndrome is a chronic neurodevelopmental disorder characterized by involuntary motor and vocal tics, with symptoms typically emerging during childhood. Current pharmacologic treatment options include D2 receptor antagonists such as haloperidol, pimozide, and aripiprazole, although treatment can be limited by adverse effects including sedation, extrapyramidal symptoms, and metabolic effects.

The rationale for ecopipam is based in part on the proposed role of altered dopaminergic signaling in the basal ganglia in Tourette syndrome. By selectively antagonizing D1 receptors, ecopipam is being investigated as a potential means of modulating dopaminergic activity through a mechanism distinct from D2-targeting therapies.

Across the phase 2b and phase 3 clinical program, investigators have not identified clinically meaningful changes in body weight, body mass index, vital signs, laboratory or ECG parameters, movement-disorder scales, or measures of psychiatric comorbidity. Common AEs reported across the studies have included headache, insomnia, fatigue, somnolence, tics, anxiety, nausea, and restlessness.¹

Regulatory Status

The new data come as ecopipam remains under FDA priority review for pediatric patients with Tourette syndrome. The FDA accepted Teva's new drug application (NDA) in August 2026 and established a Prescription Drug User Fee Act target action date for late in the first quarter of 2027.⁴

The NDA is supported by data from the phase 2b and phase 3 D1AMOND program. Although the phase 3 trial included both pediatric and adult participants, the NDA and proposed indication are limited to pediatric patients.⁴

If approved, ecopipam would represent a new pharmacologic approach to tic management through selective D1 receptor antagonism. Teva has characterized the therapy as potentially the first new treatment for Tourette syndrome in more than a decade, although ecopipam remains investigational and has not yet been approved for this indication.

REFERENCES
1. Teva presents new efficacy and safety data with ecopipam, an investigational treatment for pediatric patients with Tourette syndrome. Teva Pharmaceuticals. News release. October 2, 2026. Accessed October 2, 2026. https://www.globenewswire.com/news-release/2026/10/02/3373765/0/en/teva-presents-new-efficacy-and-safety-data-with-ecopipam-an-investigational-treatment-for-pediatric-patients-with-tourette-syndrome.html 
2. Gilbert DL, Dubow JS, Cunniff TM, et al. Ecopipam for Tourette syndrome: a randomized trial. Pediatrics. 2023;151(2). doi:10.1542/peds.2022-059574.
3. Gilbert DL, Atkinson SD, Kim DJB. 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.
4. U.S. FDA accepts Teva's new drug application (NDA) and grants priority review for ecopipam, a first-in-class investigational therapy for pediatric patients with Tourette syndrome. Teva Pharmaceuticals. News release. August 19, 2026. Accessed October 2, 2026. https://ir.tevapharm.com/news-and-events/press-releases/press-release-details/2026/U-S--FDA-Accepts-Tevas-New-Drug-Application-NDA-and-Grants-Priority-Review-for-Ecopipam-a-First-in-Class-Investigational-Therapy-for-Pediatric-Patients-with-Tourette-Syndrome/default.aspx