Paragon’s holding company, Emalex Biosciences, announces the first positive results of the phase 2b clinical study evaluating ecopipam for the treatment of pediatric Tourette syndrome


CHICAGO, November 10, 2021 / PRNewswire / – Emalex Biosciences, Inc. (“Emalex”), a biopharmaceutical company founded by Paragon Biosciences to develop treatments for central nervous system movement disorders and fluidity disorders, today announced the first positive results of its phase 2b clinical study (D1AMOND study) evaluating the efficacy and safety of ecopipam (EBS-101), an experimental, first-in-class dopamine-1 (D1) receptor antagonist, for the treatment of pediatric patients with Gilles de la Tourette syndrome.

“The strong results of our ecopipam study are very encouraging, giving us the momentum to continue our important work towards a safe and effective therapy for pediatric patients with Gilles de la Tourette syndrome,” said Atul R. Mahableshwarkar MD., Chief Medical Officer and Senior Vice President of Drug Development. “Behind the data lies a strong team of scientists, clinicians, neurologists, psychiatrists and industry experts committed to developing new treatment options for people with this disease, as well as patients and caregivers. dedicated families who participated in the study. “

The D1AMOND study was a multicenter, randomized, double-blind, placebo-controlled, parallel group study evaluating the efficacy and safety of ecopipam in 153 children and adolescents (aged 6 to Canada and Europe. Patients were randomized to receive either ecopipam tablets at a dose of 2 mg / kg / day or placebo tablets. The study drug was titrated to the target dose over four weeks and then held for an eight week treatment period. The primary efficacy endpoint was moving from baseline to week 12 in the Yale Global Tic Severity Score-Total Tic Score (YGTSS-TTS). Statistically significant and clinically significant results were obtained on this primary endpoint at all time points from week 4 to week 12. The key secondary endpoint, the overall clinical impression of the severity of the syndrome Gilles de la Tourette (CGI-TS-S), was also statistically significant at all time points from week 6 to week 12.

The most common adverse events (≥ 5% and greater than placebo) were headache, fatigue, drowsiness and restlessness. For less frequently reported adverse events, a slightly higher proportion of patients treated with ecopipam experienced depression, compared to placebo, and several patients treated with ecopipam reported anxiety. No clinically significant difference between ecopipam and placebo was noted in labs, vital signs, ECGs, and in a scale commonly used to assess suicidal ideation and behavior.

“After decades of research in pediatric neurology and psychiatry, it is extremely gratifying to obtain positive and meaningful study results for a group of patients whose unmet medical needs are so high,” said the principal investigator. Donald gilbert, MD, MS, specialist in pediatric movement disorders and Tourette’s syndrome and professor of neurology, Cincinnati Children’s Hospital Medical Center. “We are one step closer to life changing therapy for a group of patients who inspires and fuels our efforts.”

Emalex is preparing to meet with the United States Food and Drug Administration and other global regulatory bodies in the coming months to discuss next steps related to the drug approval process. The full study data, including the specifics of the primary efficacy and safety data set, will be presented at an upcoming scientific conference and submitted for peer-reviewed scientific publication.

“I am delighted for the pediatric patients with Gilles de la Tourette syndrome who now have hope for a potentially effective treatment,” said Jeff Aronin, founder, CEO of Paragon Biosciences. “These results are a testament to the extraordinary talents of the Paragon and Emalex teams. Their work, focused on people living with conditions with high unmet need, reflects Paragon’s mission where we use biology to accelerate scientific breakthroughs for transform the way people live. “

About Ecopipam
Ecopipam is an investigational first-class drug being evaluated for the treatment of Gilles de la Tourette syndrome (TMS) in pediatric patients and pediatric fluidity disorder (stuttering) in adults. Ecopipam selectively blocks the actions of the neurotransmitter dopamine at the D1 receptor. Dopamine is a neurotransmitter of the central nervous system and its receptors have been classified into two “families” according to their genetic structure: “D1” (including the D1 and D5 subtypes) and “D2” (including the sub-types. -types D2, D3 and D4). D1 receptor super-sensitivity may be a mechanism of repetitive and compulsive behaviors associated with TS.

Ecopipam has been shown to be generally well tolerated in clinical trials to date and has received orphan and fast track designation from the United States Food and Drug Administration for the treatment of patients with TS. Adverse reactions mainly affecting the central nervous system [CNS] (eg, sedation, insomnia, psychiatric changes) and the gastrointestinal system (eg, nausea and vomiting) are the side effects most frequently reported in clinical studies.

About Emalex Biosciences
Emalex Biosciences is a biopharmaceutical company focused on developing treatments for central nervous system movement disorders and fluidity disorders with limited or no treatment options. In keeping with its mission, the company is evaluating treatments for childhood fluidity disorder, or stuttering, and for children and adolescents with Gilles de la Tourette syndrome. Based at Chicago, Emalex is a holding company of Paragon Biosciences. For more information visit:

About Paragon Biosciences
Paragon is a global leader in life sciences that creates, builds and finances innovative biology-based companies in the fields of cell and gene therapy, adaptive biology and advanced biotechnology. The company’s current portfolio includes Castle Creek Biosciences, CiRC Biosciences, Emalex Biosciences, Evozyne, Harmony Biosciences, Qlarity Imaging, Skyline Biosciences and a constant flow of incubating companies created and supported by the Paragon Innovation Capital â„¢ model. Paragon sits at the intersection of human need, life sciences and business creation. For more information, please visit

Media contact:

Evelyn M. O’Connor
Paragon Biosciences
[email protected]

SOURCE Emalex Biosciences, Inc.

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