Study opens pathway to investigational therapy for patients with Tourette syndrome who have exhausted approved treatments
CHICAGO–(BUSINESS WIRE)–Emalex Biosciences today announced that the first patient was dosed in its Expanded Access Program for ecopipam, an investigational first-in-class D1 receptor antagonist in development for the treatment of Tourette syndrome.
The Expanded Access Program will enroll approximately 200 patients at sites across the United States. A current list of participating sites for the trial, NCT07093541, is available on ClinicalTrials.gov.
The program allows physicians to request ecopipam for patients who have been treated with an FDA-approved therapy for Tourette syndrome, including aripiprazole, haloperidol, pimozide or another D2 receptor antagonist, and who have experienced treatment failure, tolerability or safety concerns, or lack access to approved medications.
“Tourette syndrome can profoundly disrupt a child’s education, social development and quality of life, and many families struggle to find effective, well-tolerated treatments,” said Frederick Munschauer, MD, chief medical officer of Emalex Biosciences. “We established this Expanded Access Program to help address that gap while ecopipam continues to advance through development.”
Expanded Access Programs provide a regulatory pathway for patients with serious conditions to receive investigational medicines outside of clinical trials. Tourette syndrome is a chronic, childhood-onset neurodevelopmental disorder characterized by motor and vocal tics and is associated with significant morbidity that can disrupt school, work and social functioning.
Ecopipam is a first-in-class investigational small-molecule therapy that selectively blocks dopamine-1 (D1) receptors. It is not an antipsychotic. This mechanism differs from currently approved Tourette syndrome treatments, which primarily target dopamine-2 (D2) receptors.
Participation in the Expanded Access Program requires physician oversight, FDA authorization, and Institutional Review Board approval. Enrollment is limited, and eligibility is determined on a case-by-case basis.
Patients and clinicians with questions can contact Emalex Biosciences at [email protected]. Emalex’s Expanded Access Policy is available here.
Ecopipam is an investigational drug and has not been approved by the FDA for any indication. In clinical studies in pediatric patients with Tourette syndrome, ecopipam was generally well tolerated. The most common adverse events reported at a frequency of 5 percent or greater included headache, insomnia, fatigue, somnolence, anxiety, nausea, and restlessness.
About Ecopipam
Ecopipam is a first-in-class investigational compound being studied as a potential treatment for certain central nervous system disorders. It blocks dopamine activity at the D1 receptor. Dopamine receptors are grouped into two families based on genetic structure: D1, which includes D1 and D5 subtypes, and D2, which includes D2, D3, and D4 subtypes. D1 receptor supersensitivity may contribute to the repetitive and compulsive behaviors associated with Tourette syndrome. Currently approved therapies act at D2 receptors. Ecopipam has been generally well tolerated in clinical trials conducted to date and has received Orphan Drug and Fast Track designation from the FDA for the treatment of pediatric patients with Tourette syndrome. Adverse events reported in clinical trials have primarily affected the central nervous system and have included headache, fatigue, somnolence, insomnia, restlessness, anxiety, depression and, rarely, suicidal ideation.
About Emalex Biosciences
Emalex Biosciences was created by Paragon Biosciences to develop new treatments for central nervous system disorders. The company is advancing a new class of therapy for patients with Tourette syndrome and other conditions with limited treatment options. Learn more at https://emalexbiosciences.com.
About Paragon Biosciences
Paragon Biosciences, founded by Jeff Aronin, creates, builds and funds innovative biology-based companies. Its portfolio companies advance scientific breakthroughs aimed at addressing significant unmet medical needs. Learn more at https://www.paragonbiosci.com.
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