Research & Development

Therapeutic Focus

Emalex is focused on central nervous system movement disorders and fluency disorders. Our current focus is developing ecopipam, an investigational medicine, for Tourette Syndrome and also for Stuttering.

Drug Pipeline

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.

Paragon Pipeline Chart

Central Nervous System

Tourette Syndrome

  • 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 in duration, occur spontaneously, and do not show a regular temporal pattern.

Stuttering

  • Childhood-onset fluency disorder, also known as Stuttering or stammering, is a common multifactorial speech disorder. It is normally seen with recurrent prolongations, reverberations, or blocks of sounds, syllables, phrases or words. 

About Ecopipam

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.

Clinical Trials

Ecopipam has been shown to be generally well tolerated in clinical trials conducted to date, including in adult and pediatric patients with Tourette syndrome. 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. Emalex is conducting two Phase 2 clinical trials to evaluate the efficacy and safety in patients with Tourette syndrome and stuttering.

For more information on the current clinical trial, please visit: https://clinicaltrials.gov/ct2/show/NCT04007991.