Emalex Biosciences Publishes Phase 2b Results for Tourette Syndrome Candidate in Pediatrics

Study data shows investigational new drug, ecopipam, reduced total number of tics by 30% compared to placebo

CHICAGO, Jan. 11, 2023 /PRNewswire/ -- A Phase 2b clinical trial involving more than 150 children and adolescents with Tourette syndrome found ecopipam significantly reduced the total number of tics compared to placebo, according to a study published today in the journal Pediatrics.

The study, known as the D1AMOND trial, was an international, multicentered randomized, placebo-controlled trial of ecopipam in Tourette syndrome.  The primary outcome was the mean change in Yale Global Tourette Severity Total Tic Score.   Statistically significant results were achieved on the primary endpoint (p= 0.01) for the ecopipam group.  This represented a 30% reduction from baseline to week 12.  The key secondary outcome of Clinical Global Impression of Tourette Syndrome Severity also showed improvement in the ecopipam group (p= 0.03).

Patients in the trial did not have observable evidence of adverse movements, excessive weight gain, or metabolic side effects commonly reported with antipsychotic agents, which are often used to treat patients with Tourette syndrome. The most frequent adverse events related to ecopipam in the study were headache (15.8%), insomnia (14.5%), fatigue (7.9%), and somnolence (7.9%).

Ecopipam is the lead candidate for Emalex Biosciences, a clinical-stage biopharmaceutical company founded by Paragon Biosciences. In November, Emalex announced an upsized and oversubscribed $250 million Series D funding round that will fund a Phase 3 clinical trial and potential commercialization of ecopipam.

Ecopipam is a first-in-class dopamine-1 receptor antagonist. The antipsychotic agents currently approved to treat Tourette syndrome target the dopamine-2 receptor. The planned ecopipam Phase 3 trial is expected to enroll more than 220 patients across approximately 90 sites, making it the largest clinical trial ever conducted in North America for this serious central nervous system disorder.

Tourette syndrome is a chronic, childhood-onset neurodevelopmental disorder characterized by motor and vocal tics. The condition is associated with both increased mortality and significant morbidity.  In the majority of individuals, Tourette  syndrome substantially impacts day-to-day physical and social function.

"Most people don't realize that tics can cause pain and injury as well as significant social stigma and emotional distress. We see many children and teens whose symptoms of Tourette syndrome interfere with their daily activities, including their schoolwork," said lead author Donald Gilbert, MD, a movement disorders specialist at the Cincinnati Children's Hospital and principal investigator for the D1AMOND study.  "There is an urgent need for safer and more effective treatments."

Editor's Note: This study was funded by Emalex Biosciences Inc.

Emalex's first-in-class development candidate ecopipam, a novel investigational compound that is being studied as a potential treatment for certain central nervous system disorders, blocks the actions of the neurotransmitter dopamine at the D1 receptor. Dopamine is a neurotransmitter in the central nervous system, and its receptors have been classified into two "families" based on their genetic structure: "D1" (including subtypes D1 and D5) and "D2" (including subtypes D2, D3, and D4). D1 receptor super-sensitivity may be a mechanism for the repetitive and compulsive behaviors associated with Tourette syndrome. Currently approved therapies for the treatment of Tourette syndrome act at D2 receptors.  Ecopipam has been shown to be generally well tolerated in clinical trials conducted to date and has received Orphan Drug and Fast Track designation from the FDA for the treatment of patients with Tourette syndrome. Adverse events affecting primarily the central nervous system have been reported in clinical trials conducted to date, including headache, fatigue, somnolence, insomnia, restlessness, anxiety, depression and rarely, suicidal ideation.

Emalex Biosciences was created by Paragon Biosciences to develop new treatments for central nervous system disorders. Emalex is in late-stage development of a new class of drug targeting the D1 receptor for patients with Tourette syndrome and other conditions that have limited treatment options. Visit https://emalexbiosciences.com  to learn more.

Paragon Biosciences is a global life science leader that creates, builds and funds innovative biology-based companies in three key areas: cell and gene therapy, biology engineering and advanced biotechnology. Our portfolio companies use biology to accelerate scientific breakthroughs that solve some of society's most challenging problems. Learn more at https://www.paragonbiosci.com.

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SOURCE Emalex Biosciences