Paragon’s Portfolio Company Emalex Biosciences Announces Positive Topline Results from Phase 2b Clinical Study Evaluating Ecopipam for Pediatric Tourette Syndrome

CHICAGO – Emalex Biosciences, Inc. (“Emalex”), a biopharmaceutical company founded by Paragon Biosciences to develop treatments for central nervous system movement disorders and fluency disorders, today announced positive topline results from its Phase 2b clinical study (D1AMOND Study) evaluating the efficacy and safety of ecopipam (EBS-101), an investigational, first-in-class, dopamine-1 (D1) receptor antagonist, for the treatment of pediatric patients with Tourette Syndrome.

“The robust results of our ecopipam study are very encouraging, giving us momentum to continue our important work towards a safe and effective therapy for pediatric patients living with Tourette Syndrome,” said Atul R. Mahableshwarkar M.D., Chief Medical Officer and Senior Vice President of Drug Development. “Behind the data is a strong team of scientists, clinicians, neurologists, psychiatrists and industry experts determined to develop new treatment options for those with this disease, as well as dedicated patients and 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 (ages 6 to < 18) conducted at 63 sites across the U.S., Canada and Europe. Patients were randomized to receive either ecopipam tablets at a dose of 2 mg/kg/day or placebo tablets. Study medication was titrated to the target dose over four weeks and then maintained for an eight-week treatment period. The primary efficacy endpoint was the change from Baseline to Week 12 in the Yale Global Tic Severity Score-Total Tic Score (YGTSS-TTS). Statistically significant and clinically meaningful results were obtained on this primary endpoint at all timepoints, from Week 4 to Week 12. The key secondary endpoint, the Clinical Global Impression of Tourette Syndrome Severity (CGI-TS-S), was also statistically significant at all timepoints from Week 6 to Week 12.

The most frequent adverse events (≥ 5% and higher than placebo) were headache, fatigue, somnolence, and restlessness. For less commonly reported adverse events, a slightly higher proportion of ecopipam-treated patients experienced depression, when compared to placebo, and several ecopipam-treated patients reported anxiety. No clinically significant differences between ecopipam and placebo were noted in labs, vital signs, EKGs and in a commonly used scale to assess suicidal ideation and behaviors.

“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,” said Lead Investigator Donald Gilbert, M.D., M.S., Pediatric Movement Disorders and Tourette Syndrome Specialist and Professor of Neurology, Cincinnati Children’s Hospital Medical Center. “We are one step closer to a potentially life-changing therapy for a patient group who inspires and fuels our efforts.”

Emalex is preparing to meet with the U.S. Food and Drug Administration and other global regulatory agencies in the coming months to discuss next steps related to the drug approval process. Full data from the study, including the specifics of the primary efficacy and safety dataset, will be presented at an upcoming scientific conference and submitted for scientific peer-review publication.

“I am thrilled for pediatric patients living with Tourette Syndrome who now have hope for a potentially effective treatment,” said Jeff Aronin, Founder, Chairman and CEO, Paragon Biosciences. “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.”

About Ecopipam

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 Emalex Biosciences

Emalex 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 evaluating treatments for childhood-onset fluency disorder, or stuttering, and for children and adolescents with Tourette Syndrome. Headquartered in Chicago, Emalex is a portfolio company of Paragon Biosciences. For more information, visit:

About Paragon Biosciences

Paragon is a global life science leader that creates, builds and funds innovative biology-based companies in 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 consistent flow of incubating companies created and supported by the Paragon Innovation Capital™ model. Paragon stands at the intersection of human need, life science, and company creation. For more information, please visit

Media Contact: 

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