Topline data from Phase 3 study in patients with Tourette syndrome shows statistical significance between ecopipam and placebo for both the primary efficacy endpoint in pediatric subjects (p = 0.0084) and secondary efficacy endpoint in pediatric and adult subjects (p=0.0050)
Emalex Biosciences announced positive topline data from its Phase 3 registrational study of ecopipam, a novel dopamine-1 receptor antagonist, in subjects with Tourette syndrome.
The company will meet with the Food and Drug Administration and other global health authorities to discuss the submission later this year of a new drug application for ecopipam for the treatment of patients with Tourette syndrome.
A total of 167 pediatric subjects and 49 adult subjects with Tourette syndrome were enrolled at study sites in the US, Canada, and the European Union. Those who experienced clinically meaningful reductions in vocal and motor tics while receiving ecopipam during a 12-week open-label period were randomized to either continue on ecopipam or be switched to placebo in a 12-week double-blind withdrawal period.
The primary efficacy endpoint was time to relapse for pediatric subjects following randomization to ecopipam or placebo. The study found 41.9% of subjects randomized to ecopipam relapsed and 68.1% of subjects randomized to placebo relapsed. The results were statistically significant (p = 0.0084) with a hazard ratio of 0.5 (0.3-0.8).
The secondary efficacy endpoint was time to relapse for both pediatric and adult subjects following randomization to ecopipam or placebo. The study found 41.2% of subjects randomized to ecopipam relapsed and 67.9% of subjects randomized to placebo relapsed. The results were statistically significant (p = 0.0050) with a hazard ratio of 0.5 (0.3-0.8).
Ecopipam was generally well tolerated in the study and the most common adverse events related to ecopipam therapy were somnolence (10.2%), insomnia (7.4%), anxiety (6.0%), fatigue (5.6%), and headache (5.1%).
“These results strengthen our confidence in ecopipam as a potential first-in-class treatment for patients with Tourette syndrome,” said Frederick Munschauer, MD, Emalex Biosciences chief medical officer. “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.”
Ecopipam is the lead candidate for Emalex Biosciences, a clinical-stage biopharmaceutical company founded by Paragon Biosciences.
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.
“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, Emalex Biosciences CEO. “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.”
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