Jul 08, 2021 01:58 AM EDT By Patrick Jones, UniversityHerald Reporter

Effective treatments for Tourette Syndrome in pediatrics and childhood-onset fluency disorder, also known as stuttering, have remained elusive. However, thanks to the efforts of Paragon Biosciences, which creates, invests in and funds innovative bio-based companies, help may be on the way in the form of a medication that can treat these conditions.

Clinical studies are being conducted by Emalex Biosciences, a biopharmaceutical company that focuses on first-in-class drugs for central nervous system movement disorders and fluency disorders.  Three Phase 2 clinical trials are currently underway to evaluate the safety and effectiveness of a product called ecopipam (EBS-101) as a suitable therapy for Tourette Syndrome in pediatric patients and stuttering in adults. 

Tourette Syndrome Study for Pediatric Patients

Briefly defined, Tourette syndrome is a serious neurological disorder that can severely impair daily function and seriously interfere with the overall quality of life. It is characterized by brief, spontaneous, and irregular motor or vocal tics that typically begin in childhood and persist into adulthood.

Emalex's Tourette Syndrome trial, called D1AMOND, began in July 2019. It recently achieved full study patient enrollment of 150 children or teens between the ages of 6 and 18. Over a 13-week period, participants will be given either ecopipam hydrochloride or a matching placebo. 

An interim futility analysis conducted in March 2021 by an Independent Data Monitoring Committee on the first 75 patients enrolled recommended that the trial continue as planned, as it appeared to be adequately powered and no safety signal was evident that would cause a study modification.  Full trial results are expected by the end of the year.    

The FDA has officially granted ecopipam Orphan Drug designation and Fast Track status for the treatment of pediatric Tourette Syndrome.  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.

Childhood-Onset Fluency Disorder (Stuttering)

More than 70 million people around the world have some form of stuttering, but currently there are no FDA-approved treatments available. Emalex's Speak Freely trial is for adults 18 years of age and older have stuttered since childhood. Participants must be willing to take the medicine or a placebo for up to 13 weeks and be evaluated before, during, or after the trial to assess the status of their stutter. The first participant received dosing in December 2020. Trials are taking place in nine communities around the country and volunteers are still welcome to join. The study recently reached 50% patient enrollment and expects full patient enrollment by year-end 2021.

"Emalex's continued progress toward a much-needed treatment for people living with Tourette syndrome and stuttering is encouraging," said Emalex Founder and Chairman Jeff Aronin, who is also the founder, CEO and chairman of Paragon Biosciences. "Its achievements embody the spirit of Paragon's mission and dedication to areas of high unmet need throughout our diverse portfolio of companies."

Emalex Biosciences is a portfolio company of Paragon Biosciences, a global life science leader.  To assist and support the efforts of these trials and future research for Emalex Biosciences, Paragon recently led a $35 million in Series C Preferred Stock funding. Valor Equity Partners and Fidelity Management and Research Company joined in the raise. The funding will help the company continue the clinical trials for ecopipam as well as other organizational operations.   

In addition to Emalex, Paragon's portfolio companies include: Castle Creek Biosciences, CiRC Biosciences, Harmony Biosciences, Evozyne, Qlarity Imaging, and Skyline Biosciences. Beyond the neurological and biopharmaceutical research taking place at Emalex Biosciences, Paragon portfolio companies explore everything from cell and gene therapy to synthetic biology and advanced biotechnology. 

Since 2017, Paragon Biosciences and various partners have invested more than $1 billion to build life science companies and expects ongoing investment of up to $500 million annually.    

About Jeff Aronin

Jeff Aronin is a leading entrepreneur and philanthropist who has a portfolio of successful, ground-breaking companies that are driving transformation across the biological and life sciences. As founder and co-chairman of MATTER - a health-technology startup incubator - Jeff has led one of Chicago's most remarkable, philanthropic venture-innovation successes, with more than 200 healthcare companies at any one time, more than 60 corporate sponsors, and more than $1.7 billion in deals since 2015. As a believer in education as a change agent, Jeff serves as a mentor to students and young professionals and is a frequent lecturer at the University of Chicago's Booth School of Business and Northwestern University's Kellogg School of Business. He has received a variety of local and national honors and serves on the boards of Discover Financial Services and The Aspen Institute.

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