The first patient has been dosed in a trial for an investigational Tourette syndrome therapy. 

Biopharmaceutical company Emalex Biosciences announced that the first patient was dosed in its expanded access programme for ecopipam, an investigational first-in-class D1 receptor antagonist in development for the treatment of Tourette syndrome.  

Ecopipam is a first-in-class investigational small-molecule therapy that selectively blocks dopamine-1 (D1) receptors. The therapy, which is not antipsychotic, differs from currently approved Tourette syndrome treatments, which primarily target dopamine-2 (D2) receptors. 

The programme 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, Chief Medical Officer of Emalex Biosciences.  

“We established this expanded access programme to help address that gap while ecopipam continues to advance through development.” 

Expanded access programmes provide a regulatory pathway for patients with serious conditions to receive investigational medicines outside of clinical trials. In this trial, Emalex aims to enrol approximately 200 patients at sites across the US.