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)
CHICAGO--(BUSINESS WIRE)--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.”
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, 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 CNS disorders. Emalex is in late-stage development of a new class of drug for patients with Tourette syndrome and other conditions that have limited treatment options. Visit https://emalexbiosciences.com to learn more.
About Paragon Biosciences, LLC
Paragon Biosciences is a global life science leader that creates, builds and funds innovative biology-based companies. Our portfolio companies accelerate scientific breakthroughs to solve some of society’s most challenging problems. Learn more at https://www.paragonbiosci.com.
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