Ecopipam Demonstrates Promise in Patients with Tourette Syndrome

Image Credit: Adobe Stock Images/jitendra jadhav

Emalex Biosciences announced positive topline results from a Phase III registrational study of its lead candidate ecopipam, a dopamine-1 receptor antagonist, for Tourette syndrome (TS). According to the company, a meeting with the FDA and other regulatory bodies will be scheduled for later this year to discuss a New Drug Application.¹

“These results strengthen our confidence in ecopipam as a potential first-in-class treatment for patients with Tourette syndrome,” said Frederick Munschauer, MD, chief medical officer, Emalex Biosciences, in a press release. “The topline data from our large, multi-national, randomized withdrawal study show a statistically significant benefit for ecopipam in maintaining clinically meaningful reductions in vocal and motor tics for pediatric subjects with Tourette syndrome as compared to placebo.”

The registrational study evaluated ecopipam’s ability to maintain reductions in vocal and motor tics in 167 pediatric and 49 adult patients with TS across the United States, Canada, and the EU. The primary endpoint of the study was time to relapse for pediatric subjects following randomization to ecopipam or placebo. The secondary endpoint was time to relapse for both pediatric and adult subjects following randomization to ecopipam or placebo.

Results found that 41.9% of pediatric patients on ecopipam relapsed compared to 68.1% on placebo, whereas for the secondary endpoint, 41.2% of patients receiving ecopipam relapsed compared to 67.9% patients receiving placebo.

In terms of safety, ecopipam was generally well tolerated, with common adverse events that included somnolence, insomnia, anxiety, fatigue, and headache.¹

According to the Centers for Disease Control and Prevention (CDC), approximately one out of every 162 children in the United States have TS. Additionally, a CDC study that included parent-reported data found that around one in every 333 children between three and 17 years of age have received a TS diagnosis in the United States, indicating that at least half of children with TS remain undiagnosed.

In children diagnosed between 2016 and 2017, 44% were reported to have moderate to severe TS. Boys were found to be three times more likely to be diagnosed. However, children from all racial and ethnic backgrounds were reported to have similar prevalence rates. Adolescents were twice as likely to have a positive diagnosis than children between six and 11 years of age.

According to the CDC, many children with TS also have other disorders. This includes 61% experiencing anxiety; 52% with attention-deficit/hyperactivity disorder; 34% with behavioral problems and learning disabilities; 26% with developmental delays; 21% with autism spectrum disorder; 20% with depression; 15% with speech or language issues; and 8% with intellectual disabilities. Additionally, over one-third of people with TS also have obsessive-compulsive disorder. Further, children with TS are more likely to require special healthcare and have at least one chronic medical condition.²

According to Medline Plus, the global prevalence of TS is anywhere from one to 10 in every 100,000 children, but the exact rate is unknown.³

“The Emalex team worked closely with physician investigators and patient advocates throughout the drug development process, and we are hopeful that ecopipam can provide symptomatic relief from the tics suffered by patients with Tourette syndrome,” said Eric Messner, CEO, Emalex Biosciences, in the press release. “We’re entering a new era of progress for people with central nervous system conditions with limited or no treatment options and we look forward to developing innovative new options for them.”

References

1. Emalex Biosciences’ Lead Candidate Meets Primary and Secondary Endpoints in Phase 3 Tourette Syndrome Study. Emalex. February 25, 2025. Accessed February 27, 2025. https://emalexbiosciences.com/news/emalex-biosciences-lead-candidate-meets-primary-and-secondary-endpoints-in-phase-3-tourette-syndrome-study/

2. Data and Statistics on Tourette Syndrome. CDC. Accessed February 27, 2025. https://www.cdc.gov/tourette-syndrome/data/index.html

3. Tourette syndrome. Medline Plus. Accessed February 27, 2025. https://medlineplus.gov/genetics/condition/tourette-syndrome/#inheritance