Ecopipam Reduces Tic Relapse in Patients with Tourette Syndrome
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Reviewed & Translated by Dat Tien Nguyen, B.A, ScM.
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Posted on June 19th, 2026
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Behavioral therapy remains the first-line treatment for Tourette syndrome. When behavioral interventions fail to adequately control symptoms, medications such as α2-adrenergic receptor agonists or dopamine D2 receptor modulators may be prescribed. However, their use can be limited by adverse effects, including sedation, weight gain, and extrapyramidal symptoms. To address this unmet need, a recent study published in the Journal of the American Medical Association evaluated ecopipam, a selective dopamine D1 receptor antagonist, as a treatment for Tourette syndrome.
Funding Source(s): Emalex Biosciences Inc.
The phase 3 clinical trial enrolled 216 participants who met DSM-5 diagnostic criteria for Tourette syndrome. At baseline, the cohort had an average Yale Global Tic Severity Scale Total Tic Score (YGTSS-TTS) of approximately 34, indicating substantial tic burden. During an initial open-label treatment phase, all participants received ecopipam at a daily dose of 1.8 mg per kilogram of body weight. Among them, 104 patients achieved at least a 25% reduction in YGTSS-TTS and were subsequently randomized to continue ecopipam or switch to placebo.
After 12 weeks of follow-up, patients who continued ecopipam treatment had a 53% lower risk of symptom worsening compared with those switched to placebo. Disease exacerbation was defined as the loss of at least half of the improvement achieved during the initial treatment phase. The benefit of continued ecopipam therapy was observed in both pediatric and adult patients. These findings suggest that ecopipam may be effective in maintaining symptom control among patients who initially respond to treatment. However, the study design has important limitations. Only participants who demonstrated a favorable response during the open-label phase were included in the randomized portion of the trial. As a result, the findings primarily reflect the drug’s ability to maintain improvement rather than its overall effectiveness in the broader Tourette syndrome population. Future studies should evaluate outcomes in treatment-naïve patients and in those who do not initially respond to ecopipam.
Funding Source(s): Emalex Biosciences Inc.
The phase 3 clinical trial enrolled 216 participants who met DSM-5 diagnostic criteria for Tourette syndrome. At baseline, the cohort had an average Yale Global Tic Severity Scale Total Tic Score (YGTSS-TTS) of approximately 34, indicating substantial tic burden. During an initial open-label treatment phase, all participants received ecopipam at a daily dose of 1.8 mg per kilogram of body weight. Among them, 104 patients achieved at least a 25% reduction in YGTSS-TTS and were subsequently randomized to continue ecopipam or switch to placebo.
After 12 weeks of follow-up, patients who continued ecopipam treatment had a 53% lower risk of symptom worsening compared with those switched to placebo. Disease exacerbation was defined as the loss of at least half of the improvement achieved during the initial treatment phase. The benefit of continued ecopipam therapy was observed in both pediatric and adult patients. These findings suggest that ecopipam may be effective in maintaining symptom control among patients who initially respond to treatment. However, the study design has important limitations. Only participants who demonstrated a favorable response during the open-label phase were included in the randomized portion of the trial. As a result, the findings primarily reflect the drug’s ability to maintain improvement rather than its overall effectiveness in the broader Tourette syndrome population. Future studies should evaluate outcomes in treatment-naïve patients and in those who do not initially respond to ecopipam.