Emalex Biosciences Announces First Patient Enrolled in Phase 2b Clinical Study to Evaluate the Efficacy and Safety of Ecopipam Tablets for the Treatment of Pediatric Patients with Tourette Syndrome
- Category: Small Molecules
- Published on Wednesday, 14 August 2019 17:42
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CHICAGO, IL, USA I August 14, 2019 I Emalex Biosciences, Inc., a biopharmaceutical company developing innovative therapies for patients with neurological and psychiatric conditions, announced today that the first patient was recently enrolled in the company’s international Phase 2b study to evaluate the efficacy and safety of ecopipam HCl tablets in children and adolescents with Tourette Syndrome (TS). Ecopipam is an investigational drug that selectively blocks the actions of dopamine at the D1 receptor.
The Phase 2b clinical study of ecopipam in children and adolescent patients (aged >6 to <18 years) with TS is being conducted in multiple centers in the United States, Canada, and the European Union. The study is double-blind. Approximately 150 patients will be randomized to either ecopipam HCl 2 mg/kg/day or a matching placebo for 12 weeks. Those who complete the study will also be eligible to enroll in a one-year open-label safety extension study where all patients will receive ecopipam HCl 2 mg/kg/day.
“Enrollment of the first patient in this Phase 2b clinical trial of ecopipam, an investigational first-in-class drug, is a significant milestone in the compound's development program," said Atul Mahableshwarkar, MD, Senior Vice President of Drug Development at Emalex Biosciences. “The evaluation of ecopipam HCl 2 mg/kg/day will allow us to gain insights into the potential utility of this compound to reduce the frequency and severity of vocal and motor tics."
Additional information about the clinical trial, including eligibility criteria and the location of clinical trial sites, can be found at https://clinicaltrials.gov/ct2/show/NCT04007991.
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 TS. Currently approved therapies for the treatment of TS act at D2 dopamine receptors.
Ecopipam has been shown to be generally well tolerated in clinical trials conducted to date, including in adult and pediatric 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.
About Tourette Syndrome
Tourette Syndrome (TS) is a neurological disorder characterized by motor or vocal tics that begins during childhood and is not caused by medications, other medical reasons, or a confirmed neurological abnormality. Motor symptoms may include eye-blinking, facial grimacing, mouth movements, head jerks, shoulder shrugs, and arm/leg jerks. Vocal symptoms may include fast, meaningless sounds or noises such as sniffing, throat clearing, grunting, barks, and squeals. TS is a complicated disease that is often difficult to diagnose due to commonly associated co-morbid complications such as attention deficit/hyperactivity disorder (ADHD), obsessive-compulsive disorder (OCD), anxiety, and depression.
About Emalex Biosciences, Inc
Emalex Biosciences is a biopharmaceutical company dedicated to the development of new and novel treatments for patients with central nervous system disorders who have limited or no treatment options. Emalex is headquartered in Chicago, Illinois. For more information, visit: EmalexBiosciences.com.
SOURCE: Emalex Biosciences