Our Therapeutic Focus
Tourette Syndrome (in pediatrics)
- Tourette syndrome (TS) is a neurological disorder characterized by motor or vocal tics that begin in childhood and persist over time. The tics are brief, spontaneous and irregular.
Childhood-Onset Fluency Disorder (in adults)
- Childhood-onset fluency disorder (COFD), also known as stuttering, is a common multifactorial speech disorder that begins in childhood and persists in adults. It is normally seen with recurrent prolongations, reverberations, or blocks of sounds, syllables, phrases or words.
Emalex’s first-in-class development candidate is ecopipam (EBS-101), a novel investigational compound that has been studied as a potential treatment for other central nervous system (CNS) disorders. We are conducting clinical trials to evaluate the safety and efficacy of ecopipam in pediatric patients with TS and COFD in adults. These studies are regulated by the U.S. Food and Drug Administration (FDA) and by similar regulatory agencies in Canada and Europe.
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 patients with TS. Adverse events affecting primarily the central nervous system [CNS] (e.g., sedation, insomnia, psychiatric changes) and the gastrointestinal system (e.g., nausea and vomiting) are the most frequently reported side effects.
Further information and background on clinical trials may be found on www.clinicaltrials.gov, a service of the U.S. National Institutes of Health (NIH).
Emalex Biosciences is a biopharmaceutical company dedicated to the development of new treatments for rare and orphan neurological conditions. Ecopipam (EBS-101), its first development candidate, is a novel compound that has been studied as a potential treatment for many central nervous system (CNS) disorders, including pediatric Tourette Syndrome and Stuttering.
Ecopipam is an investigational first-in-class drug being evaluated in pediatric patients for the treatment of Tourette Syndrome (TS) and for childhood onset fluency disorder (Stuttering) in adults. Ecopipam selectively 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 dopamine receptors.