Emalex Biosciences Announces First Patient Dosed in Phase 3 Trial of Ecopipam for Tourette Syndrome

– Enrolled patients will receive medication for up to 24 weeks; 90 sites planned for North America and Europe – 

CHICAGO, March 1, 2023 – Emalex Biosciences announced that the first patient was dosed in its Phase 3 clinical trial evaluating ecopipam for the treatment of Tourette Syndrome. Participants in the trial receive ecopipam for 12 weeks in the open-label phase of the study. Those with at least a 25% reduction in the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) at both week 8 and 12 will be randomized to continue on ecopipam or placebo in the double-blind phase of the study until they relapse, up to an additional 12 weeks. Efficacy will be assessed as the difference in time-to-relapse between groups.

“Tourette Syndrome is a serious condition for which there is significant unmet need,” said Amanda Talty, Tourette Association of America president and CEO. “Currently, the two classes of medicines commonly used to treat Tourette can have a number of concerning side effects. We are happy to see the progress of additional studies being conducted to test new treatment alternatives for our community.”

Emalex closed an upsized and oversubscribed $250 million Series D round led by Bain Capital Life Sciences in November to fund the Phase 3 trial and potential commercialization of ecopipam. The trial is expected to enroll more than 200 patients across 90 sites in North America and Europe.

Ecopipam is a first-in-class dopamine-1 receptor antagonist. The antipsychotic agents currently approved to treat Tourette syndrome target the dopamine-2 receptor and are associated with serious side effects. Patients enrolled in the Phase 2b D1AMOND study did not have observable evidence of adverse movements, excessive weight gain, or the metabolic side effects commonly reported with antipsychotic agents. The most frequent ecopipam-related adverse events in the study were headache (9.2%), fatigue (6.6%), somnolence (6.6%), insomnia (5.3%), and restlessness (5.3%).

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.

Principal investigator Donald Gilbert, MD, a movement disorders specialist at the Cincinnati Children’s Hospital, noted patients’ tics often cause pain and injury in addition to social stigma and emotional distress.

“My patients have an urgent need for safer and more effective treatments. Tourette interferes with daily activities and commonly has negative impacts on mental and emotional health. For children and teens, Tourette can affect school performance and social development, which has lifelong consequences,” Dr. Gilbert said.

About Ecopipam
Emalex’s first-in-class development candidate, ecopipam, is a novel investigational compound that is being studied as a potential treatment for certain central nervous system disorders. Ecopipam 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. Adverse events affecting primarily the central nervous system have been reported in clinical trials conducted to date include headache, fatigue, somnolence, insomnia, restlessness, anxiety, depression and rarely, suicidal ideation. Further information can be found on

About Emalex Biosciences
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 to learn more.

About Paragon Biosciences, LLC
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

Media Contact:
Sheridan Chaney