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Emalex Biosciences Announces Completion of Patient Enrollment in Phase 2b Clinical Trial Evaluating Ecopipam (EBS-101) for Pediatric Patients with Tourette Syndrome

CHICAGO, June 22, 2021 /PRNewswire/ -- Emalex Biosciences, Inc. ("Emalex"), a clinical-stage biopharmaceutical company focused on developing treatments for central nervous system movement disorders and fluency disorders, announced today that its Phase 2b clinical trial evaluating the efficacy and safety of ecopipam (EBS-101) for the treatment of pediatric patients with Tourette Syndrome has reached 100% percent enrollment. The clinical trial, known as the D1AMOND Study (NCT04007991), is a 12-week randomized, double-blind, placebo controlled parallel-group study that enrolled 153 patients globally. 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.

100% enrollment in Emalex Biosciences' Phase 2b clinical trial for pediatric patients with Tourette Syndrome
Tourette Syndrome is a neurological disorder characterized by motor or vocal tics that begin in childhood and persist over time. These tics are brief, spontaneous and have irregular patterns. The only approved therapy for Tourette Syndrome in the U.S. acts at D2 dopamine receptors. Ecopipam is an investigational first-in-class drug designed to selectively block the actions of the neurotransmitter dopamine at the D1 receptor and may result in a different side effect profile. Ecopipam has been granted U.S. Food & Drug Administration Orphan Drug designation and Fast Track designation for the treatment of Tourette Syndrome in pediatric patients.

"Being able to reach complete patient enrollment, even with the challenges imposed by the COVID-19 pandemic, is a testament to the great need for a therapy for pediatric patients living with Tourette's," said Atul R. Mahableshwarkar, M.D., chief medical officer and senior vice president of drug development for Emalex. "This milestone moves us one step forward in our journey for a new potential treatment option specifically for Tourette Syndrome in pediatrics."

Emalex, a portfolio company of Paragon Biosciences, is also conducting a Phase 2 clinical trial to evaluate the efficacy and safety of ecopipam in adult patients with childhood-onset fluency disorder, commonly referred to as 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. The trial is being conducted in nine U.S. sites and is currently enrolling patients. The study recently reached its 50% patient enrollment milestone (34 of 68 patients) and is expected to complete enrollment by the end of the year. More on the Speak Freely Study for potential patients can be found at https://clinicaltrials.gov/ct2/show/NCT04492956.

With a dynamic portfolio of seven companies, Paragon Biosciences is a global life science leader that identifies unmet needs and uses biology to advance innovation to solve some of society's most challenging problems.

"The work at Emalex supports Paragon Biosciences' overarching goal of accelerating scientific breakthroughs to help people and improve the world," said Emalex Founder and Chairman Jeff Aronin, who is also the founder, chairman and chief executive officer of Paragon Biosciences. "It is incredibly rewarding to be able to support therapeutic endeavors that may result in an effective treatment for Tourette's in pediatric patients and stuttering in adult patients, which have limited or no treatment options, respectively."

About Ecopipam
Ecopipam is an investigational first-in-class drug being evaluated in pediatric patients for the treatment of Tourette Syndrome (TS) and for 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. The only approved therapy for the treatment of TS acts at D2 dopamine receptors.

Ecopipam has been shown to be generally well tolerated in clinical trials conducted to date, including in adult and pediatric 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.

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. Headquartered in Chicago, Emalex is a portfolio company of Paragon Biosciences. For more information, visit: EmalexBiosciences.com.

About Paragon Biosciences
Paragon is a global life science leader that creates, invests in and funds innovative biology-based companies in three key areas: cell and gene therapy, synthetic biology and advanced biotechnology. Paragon identifies unmet needs and uses biology to accelerate scientific breakthroughs to solve some of society's most challenging problems, enabling us to transform the way people live. 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/

SOURCE Emalex Biosciences, Inc.

Posted: June 2021

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