Teva Pharmaceuticals has unveiled new clinical data for ecopipam, an investigational treatment for pediatric Tourette syndrome, with analyses showing meaningful reductions in tic severity within eight weeks and sustained benefits over more than a year.

 

The data, presented at the International Congress of Parkinson’s Disease and Movement Disorders in Seoul, add to the evidence behind ecopipam as the drug awaits a decision from the US Food and Drug Administration under Priority Review.

 

In a pooled post hoc analysis of 292 patients from Phase 2b and Phase 3 trials, 69.8% of participants experienced a clinically meaningful improvement in tic severity within the first eight weeks of treatment, defined as at least a 25% reduction in the Yale Global Tic Severity Scale Total Tic Score.

 

The most commonly reported adverse events included somnolence and headache, with many of the cross-trial adverse events occurring during the first eight weeks of exposure.

 

“Tourette syndrome is a complex neurodevelopmental disorder that presents significant daily challenges for children and their families,” said Eric Hughes, Executive Vice President, Global R&D and Chief Medical Officer of Teva. 

 

“These promising new data for ecopipam reinforce our confidence in its potential in pediatric Tourette syndrome care. If approved, ecopipam would be the first new therapy for Tourette syndrome in more than 10 years and the first with a novel mechanism of action in more than 50 years.”

 

Teva also reported interim results from an ongoing 36-month open-label extension study involving 118 children, adolescents and adults with Tourette syndrome.

 

Participants had received at least one dose of ecopipam, with a median treatment exposure of 14.9 months. The analysis found that ecopipam maintained clinically meaningful tic suppression through 18 months, with a 45.6% mean reduction in tic severity scores.

 

Reported adverse events included nasopharyngitis, upper respiratory tract infection, anxiety, diarrhea, influenza, fever and insomnia.

 

The findings also suggest that commonly occurring psychiatric conditions did not diminish the drug’s observed effects.

 

A separate post hoc analysis of 216 Phase 3 participants compared patients with conditions including ADHD, obsessive-compulsive disorder, anxiety and depression with those without such conditions. During the 12-week open-label period, reductions in tic severity were consistent between the groups, while overall safety and tolerability profiles were similarly consistent.

 

“Many children with Tourette syndrome do not receive treatment, and among those who do, treatment is often discontinued within a year because symptoms remain inadequately controlled or side effects become difficult to tolerate,” said Donald L. Gilbert, Pediatric Movement Disorders and Tourette Syndrome Specialist, Division of Neurology, Cincinnati Children’s Hospital Medical Center. 

 

“Children living with Tourette syndrome urgently need treatment options that work rapidly, remain effective over time and have a demonstrated tolerability profile. These findings are encouraging because they suggest that, if approved, ecopipam may offer patients and families a meaningful new option.”

 

Ecopipam is designed to block dopamine signaling at the D1 receptor, making it distinct from existing Tourette syndrome therapies that act through other dopamine pathways.

 

The drug has received FDA Priority Review and Orphan Drug designation for pediatric Tourette syndrome. Teva is seeking approval specifically for pediatric patients.

 

The company's Phase 2b D1AMOND trial enrolled 153 pediatric participants, while its subsequent Phase 3 trial enrolled 216 pediatric and adult participants during an open-label stabilization period. A total of 104 responders were randomized in the Phase 3 study.