Chicago – December 15, 2020 – Emalex Biosciences, Inc. (“Emalex”), a clinical-stage biopharmaceutical company focused on developing treatments for central nervous system movement disorders and fluency disorders, announced that the first adult patient has been dosed in its Phase 2 clinical trial evaluating ecopipam, the company’s investigational medicine, for the treatment of childhood-onset fluency disorder, or “stuttering.”
The Speak Freely Study is for adults who were diagnosed with a stuttering disorder as a child and who still struggle with speaking clearly. It examines the safety of ecopipam and whether it may help improve a person’s ability to speak without stuttering. The trial is being conducted in eight U.S. sites and is seeking volunteers. Enrollment is planned to continue through April 2021.
“Stuttering is a disorder that has significant impact on patients’ quality of life, and there are currently no FDA-approved treatments available,” said Atul Mahableshwarkar, M.D., Chief Medical Officer and Senior Vice President of Drug Development at Emalex. “Having the first patient dosed in this trial represents a significant milestone for 70 million people worldwide living with stuttering. We’re proud to be evaluating this medication and are encouraged by the transformative potential it holds for patients.”
Principal investigator Gerald Maguire, M.D., Psychiatrist and Professor and Chair of Psychiatry and Neuroscience at the University of California, Riverside, has spent much of his professional career seeking an effective treatment for stuttering. His early research with ecopipam in patients who stutter showed encouraging results and has helped to advance the work being conducted at Emalex today.
“This is a tremendous opportunity for such an underserved patient population,” said Dr. Maguire, who also stutters. “Knowing first-hand how stuttering can be detrimental to a person’s overall well-being, I am excited to be part of this journey and the meaningful work being done by Emalex. Many people are unaware that research shows stuttering may be treatable; so there is a significant educational opportunity here that will serve those who stutter and their families.”
Emalex, founded by Paragon Biosciences, is unceasing in its work to develop innovative treatments for central nervous system movement and fluency disorders, especially for patient populations with high unmet needs.
Ecopipam is an investigational first-in-class drug being evaluated for the treatment of childhood-onset fluency disorder (stuttering) in adults and Tourette syndrome (TS) in pediatric patients. 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.
Childhood-onset fluency disorder, also known as stuttering or stammering, is a common multifactorial speech disorder. It is normally seen with recurrent prolongations, reverberations, or blocks of sounds, syllables, phrases or words. It is estimated that more than three million people in America and over 70 million people worldwide stutter, with males affected more frequently than females. While there is no one cause of stuttering, possible causes may include family history and neurological differences in the way one’s brain works during speech. There are currently no U.S. Food and Drug Administration approved medications for the treatment of stuttering.
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. 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: EmalexBiosciences.com.
About Paragon Biosciences
Paragon is a life science innovator that creates, invests in and builds life science companies in biopharmaceuticals, cell and gene therapy and synthetic biology utilizing artificial intelligence. 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 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/.