Jeff Aronin, Founder, Chairman, and CEO of Paragon Biosciences, is encouraged by the promising topline results from a clinical study of ecopipam for pediatric Tourette Syndrome. The study was sponsored by Emalex, a portfolio company of Paragon founded by Aronin, that focuses on developing treatments for nervous system disorders.
Speaking on the study, Aronin said, “I am thrilled for pediatric patients living with Tourette Syndrome who now have hope for a potentially effective treatment. These results are a testament to the extraordinary talents of the Paragon and Emalex teams. Their work, centered on people living with conditions with high unmet needs, mirrors the mission of Paragon where we use biology to accelerate scientific breakthroughs to transform the way people live.”
On November 10, 2021, Emalex reported topline results for its D1AMOND Study, a Phase 2b clinical study evaluating ecopipam for pediatric patients with Tourette Syndrome. The study enrolled 153 children (ages 6 to 17) and randomized them to receive either placebo or ecopipam. The group receiving ecopipam took oral tablets daily, with the dose scaling from initiation to week 4. They continued to receive the same dose from week 4 until the end of the investigational period at 12 weeks.
The research team evaluated the efficacy of the medication using a primary efficacy endpoint—the Yale Global Tic Severity Score-Total Tic Score (YGTSS-TTS)). A key secondary endpoint was the Clinical Global Impression of Tourette Syndrome Severity (CGI-TS-S)). For both measures, participants taking the medication showed marked improvement throughout the study period.
Ecopipam was associated with some side effects. These were generally mild and included headache, fatigue, somnolence, and restlessness. There were no differences between the treatment group and the placebo group in any labs or vital sign measures.
Dr. Atul R. Mahableshwarker, Chief Medical Officer and Senior Vice President of Drug Development at Emalex said of the results, “The robust results of our ecopipam study are very encouraging, giving us momentum to continue our important work toward a safe and effective therapy for pediatric patients living with Tourette Syndrome.”
Dr. Donald Gilbert, Lead Investigator of the D1AMOND Study added, “After decades of research in pediatric neurology and psychiatry, it is extremely gratifying to achieve positive and meaningful study results for a patient group that has such a high unmet medical need.”
The Emalex team is preparing to meet with the U.S. Food and Drug Administration and other global regulatory agencies. Full data from the study is expected to be published in a peer-reviewed scientific publication and presented at an upcoming scientific conference.
About Emalex Biosciences
Emalex Biosciences, a portfolio company of Paragon Biosciences, is a biopharmaceutical company focused on developing treatments for central nervous system movement disorders and fluency disorders with limited or no treatment options. Aligned with its mission, the company is currently evaluating treatments for pediatric Tourette Syndrome and childhood-onset fluency disorder (stuttering) in adults. For more information, visit EmalexBiosciences.com.
Ecopipam is an investigational first-in-class drug being evaluated for the treatment of Tourette Syndrome (TS) in pediatric patients and 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.
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 U.S. Food and Drug Administration 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 from clinical studies.
About Paragon Biosciences
Paragon Biosciences is a global life science leader that creates, builds and funds innovative biology- based companies in cell and gene therapies, adaptive biology, and advanced biotechnology. Paragon’s portfolio includes Castle Creek Biosciences, CiRC Biosciences, Emalex Biosciences, Evozyne, Harmony Biosciences, Qlarity Imaging, and Skyline Biosciences. The Paragon Innovation Capital™ model supports a consistent flow of incubating companies. For more information, visit paragonbiosci.com.
About Jeff Aronin
Jeff Aronin is the founder, chairman and CEO of Paragon Biosciences. He has decades of experience in the life sciences and biotechnology industries with a focus on identifying innovative technologies that have the potential to solve some of the world’s greatest health and societal challenges. His companies have received FDA approval for numerous medications and technologies.
Jeff has received many distinctions and honors for his work spanning more than three decades. In 2020, he was named one of the Top 10 Inspiring CEOs by Insight Success magazine. In 2018, he was awarded Innovator of the Year by Best in Biz Awards. He’s received the Weizmann Leadership Award and was inducted into the Chicago Entrepreneur Hall of Fame. He was named one of the 20 Most Influential Leaders in Healthcare by Insights Care. He received an EY Entrepreneur of the Year Award.
Outside of Paragon, Jeff devotes his time and talents to philanthropy. He is the founder and chairman of MATTER, a technology incubator that supports healthcare innovation. MATTER has helped fund more than 200 healthcare companies, collaborating with more than 60 corporate sponsors to close more than $1.7 billion in deals.
Jeff is a member of The Commercial Club of Chicago and serves on the board Discover Financial Services, The Aspen Institute and International Board of the Weizmann Institute of Science. As a believer of education as a change agent, he often mentors students and young professionals. He is a regular guest lecturer at the University of Chicago’s Booth School of Business as well as Northwestern University’s Kellogg School of Business.