Teva Pharmaceuticals has announced new data for ecopipam, a first-in-class investigational selective D1 (dopamine) receptor antagonist for the treatment of pediatric patients with Tourette Syndrome.

Presented at the International Congress of Parkinson’s Disease (PD) and Movement Disorders (MDs) in Seoul, South Korea, the data include a post hoc analysis of safety and efficacy during the initial 8 weeks of treatment with ecopipam, long-term efficacy and safety from an interim analysis of an ongoing Open-Label Extension (OLE) study, post hoc subgroup analyses and preclinical receptor selectivity findings.

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

A pooled post hoc analysis of 292 patients across phase 2b and phase 3 trials found that 69.8 percent of participants experienced a clinically meaningful reduction in tic severity, defined as a ≥25 percent improvement in the Yale Global Tic Severity Scale Total Tic Score (YGTSS-TTS), within the first 8 weeks of treatment with ecopipam. The most commonly reported Adverse Events (AEs) were somnolence and headache, with cross-trial AEs most often occurring during the first 8 weeks of ecopipam exposure.

An interim analysis of an ongoing 36-month Open-Label Extension (OLE) study found that ecopipam maintained clinically meaningful tic suppression through 18 months. The analysis included 118 children, adolescents and adults with Tourette Syndrome who had received at least 1 dose of ecopipam, with a median treatment exposure of 14.9 months. Patients received ecopipam titrated over 3 to 4 weeks before being maintained at a target dose for up to 36 months. The most commonly reported AEs were nasopharyngitis, upper Respiratory Tract Infection (RTI), anxiety, diarrhea, influenza, pyrexia and insomnia.

A post hoc analysis of 216 participants aged 6 years and older from the phase 3 trial evaluated whether co-occurring psychiatric conditions affected ecopipam’s efficacy or safety. Among the participants, 129 had at least 1 common co-occurring condition, including Attention Deficit Hyperactivity Disorder (ADHD), Obsessive-Compulsive Disorder (OCD), anxiety or depression, while 87 did not.

During the 12-week open-label period, co-occurring psychiatric conditions did not negatively affect reductions in tic severity, with mean YGTSS-TTS reductions remaining consistent between participants with and without these conditions. Overall safety and tolerability profiles were also similar between the 2 groups.

Donald L Gilbert, MD, MS, Pediatric Movement Disorders and Tourette Syndrome Specialist, Division of Neurology, Cincinnati Children’s Hospital Medical Center, said, “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. 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.”