Emalex Biosciences Phase 2b Tourette Syndrome Clinical Trial Reaches Enrollment Milestone


– Half of participants are now enrolled in study evaluating ecopipam (EBS-101) as a potential first-in-class treatment for pediatric patients with Tourette Syndrome –

Chicago – September 1, 2020 – Emalex Biosciences, Inc. (“Emalex”), a clinical-stage biopharmaceutical company focused on developing treatments for central nervous system movement disorders and fluency disorders, announced today that its Phase 2b clinical trial evaluating the efficacy and safety of ecopipam (EBS-101) for the treatment of pediatric patients with Tourette Syndrome has reached 50 percent (75 of 150 total patients) enrollment. Tourette Syndrome is a neurological disorder characterized by motor or vocal tics that begin in childhood and persist over time. These tics are brief, spontaneous and have irregular patterns.

Ecopipam is an investigational first-in-class drug designed to selectively block the actions of the neurotransmitter dopamine at the D1 receptor. Ecopipam has been granted Orphan Drug designation and Fast Track designation by the U.S. Food & Drug Administration for the treatment of Tourette Syndrome in pediatric patients.

“Currently available therapies for Tourette Syndrome act at D2 dopamine receptors. We believe that ecopipam, which acts at D1 dopamine receptors, may have a different side effect profile,” said Atul R. Mahableshwarkar, M.D., chief medical officer and senior vice president of drug development for Emalex. “We are encouraged by having randomized half of our target study enrollment, which gives us an opportunity to advance this potential treatment option for a disease that can be socially ostracizing for those afflicted.”

Emalex, a portfolio company of Paragon Biosciences, is also conducting a Phase 2b clinical trial to evaluate the efficacy and safety of ecopipam in adult patients with childhood-onset fluency disorder, commonly referred to as stuttering.

“Emalex is leading the fight to get novel medicines approved for patients with Tourette’s to improve their quality of life and help restore their confidence,” said Jeff Aronin, founder, chairman and chief executive officer of Paragon Biosciences. “Paragon supports the important work of Emalex to advance therapies for these patients who have limited treatment options available.”

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.

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 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 Emalex Biosciences

Emalex Biosciences is a clinical-stage biopharmaceutical company focused on developing treatments for central nervous system movement disorders and fluency disorders with limited or no treatment options. Headquartered in Chicago, Emalex is a portfolio company of Paragon Biosciences. For more information, visit: EmalexBiosciences.com.

About Paragon Biosciences

Paragon is a life science innovator that creates, invests in and builds life science companies in artificial intelligence, cell and gene therapy, synthetic biology and biopharmaceuticals. The company’s current portfolio includes Castle Creek Biosciences, Emalex Biosciences, Evozyne, Harmony Biosciences, Qlarity Imaging, Skyline Biosciences, and a consistent flow of incubating companies created and supported by the replicable Paragon Innovation Capital™ model. Paragon stands at the intersection of human need, life science, and company creation. For more information, please visit https://paragonbiosci.com/.

Media Contacts:

Evelyn M. O’Connor
Paragon Biosciences
312-847-1335
eo@paragonbiosci.com

Adam Daley
Berry & Company Public Relations
212-253-8881
adaley@berrypr.com

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