Phase 3 data show delayed time to relapse and maintained tic improvement with investigational, first-in-class mechanism targeting dopamine pathway

Phase 3 results published in JAMA Neurology showed that ecopipam, an investigational, first-in-class dopamine-1 (D1) receptor antagonist, delayed time to relapse compared to placebo and maintained clinically meaningful tic improvement in subjects with Tourette syndrome for up to 24 weeks.

Emalex Biosciences is developing ecopipam, which targets the D1 receptor, a pathway implicated in tic expression. The mechanism represents a new class of investigational therapies for Tourette syndrome.

In the randomized withdrawal Phase 3 trial, ecopipam significantly delayed the time to relapse compared with placebo in pediatric participants and in the overall study population and maintained clinically meaningful improvements in tic severity for up to 24 weeks of treatment. The results of the study showed that ecopipam significantly delayed the time to relapse compared to placebo in both the pediatric population (HR = 0.5, P = 0.0084) and a combined population of both pediatric and adults (HR = 0.5, P = 0.0050).

“Tourette syndrome can affect nearly every part of a person’s life, yet treatment options remain limited,” said Frederick E. Munschauer, MD, Chief Medical Officer of Emalex Biosciences. “These Phase 3 data show that targeting the D1 receptor may offer a new approach to achieve and maintain meaningful tic improvement in people living with Tourette syndrome.”

Across the study, the most commonly reported adverse events with ecopipam were somnolence (11.1%), anxiety (9.7%), headache (9.7%), insomnia (8.8%), tic (7.9%), and fatigue (6.5%). The study reported no clinically meaningful effects on weight, metabolic parameters, or drug-induced movement disorders.

Ecopipam is an investigational therapy and has not been approved by the U.S. Food and Drug Administration or any other regulatory authority. Ecopipam has received Orphan Drug and Fast Track designation from the FDA for the treatment of pediatric patients with Tourette syndrome.

Ecopipam is available through an Expanded Access Program in the United States for eligible patients with Tourette syndrome who have exhausted available treatment options. Access is provided outside of formal clinical trials and is subject to physician request, and institutional review board approval.

Tourette syndrome is a chronic neurodevelopmental disorder characterized by motor and vocal tics and associated with substantial functional burden.

The full study, authored by Donald L. Gilbert, MD, MS, of Cincinnati Children's Hospital Medical Center, and colleagues, appears at jamanetwork.com. The trial is registered at ClinicalTrials.gov (NCT05615220).

About Ecopipam

Ecopipam is a first-in-class investigational compound that is being studied as a potential treatment for certain central nervous system (CNS) disorders. It 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 pediatric 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.

About Emalex Biosciences

Emalex Biosciences was created by Paragon Biosciences to develop new treatments for central nervous system disorders. The company is advancing a new class of therapy for patients with Tourette syndrome and other conditions with limited treatment options. Learn more at https://emalexbiosciences.com.

About Paragon Biosciences

Paragon Biosciences, founded by Jeff Aronin, creates, builds and funds innovative biology-based companies. Its portfolio companies advance scientific breakthroughs aimed at addressing significant unmet medical needs. Learn more at https://www.paragonbiosci.com.

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“These Phase 3 data show that targeting the D1 receptor may offer a new approach to achieve and maintain meaningful tic improvement in people living with Tourette syndrome," said Frederick Munschauer, MD, Chief Medical Officer of Emalex Biosciences.