Ecopipam

CHICAGO--(BUSINESS WIRE)--Emalex Biosciences today announced that the U.S. Food and Drug Administration has authorized the company’s Expanded Access Program (EAP) for ecopipam, an investigational therapy in development for the treatment of Tourette syndrome. “The FDA’s authorization of our Expanded Access Program marks a significant milestone as we advance toward NDA submission,” said Frederick Munschauer, MD, Emalex Biosciences chief medical officer. The program (ClinicalTrials.gov Identifier: NCT07093541) allows physicians to request access to ecopipam for eligible patients who have been treated with an FDA-approved treatment for Tourette syndrome (aripiprazole, haloperidol, or pimozide) or any other D2r antagonist and have experienced treatment failure, tolerability or safety issues or do not have access to any of these approved medications. Emalex plans to submit a New Drug Application (NDA) to the FDA in late 2025. “The FDA’s authorization of our Expanded Access Program marks a significant milestone as we advance toward NDA submission,” said Frederick Munschauer, MD, Emalex Biosciences chief medical officer. “This pathway offers a potential option for patients and their physicians while development continues.” Expanded Access Programs provide a regulatory pathway for patients with serious conditions to access investigational medicines outside of clinical trials. Tourette syndrome is a chronic, childhood-onset neurodevelopmental disorder characterized by motor and vocal tics. The condition is associated with increased mortality and significant morbidity. In the majority of individuals, Tourette syndrome substantially impacts day-to-day physical and social function. Ecopipam is an investigational, first-in-class small-molecule drug that selectively blocks dopamine-1 (D1) receptors. It is not an antipsychotic. Investigators are evaluating ecopipam as a potential therapy for Tourette syndrome. This mechanism represents a novel approach, distinct from currently approved therapies that 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. 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 and the most common (≥ 5%) adverse events were headache, insomnia, fatigue, somnolence, anxiety, nausea, and restlessness. About Ecopipam Ecopipam is a first-in-class investigational compound that is being studied as a potential treatment for certain central nervous system (CNS) disorders. It 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 Tourette syndrome. Currently approved therapies for the treatment of Tourette syndrome act at D2 receptors. 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 FDA for the treatment of pediatric patients with Tourette syndrome. Adverse events affecting primarily the central nervous system have been reported in clinical trials conducted to date, including 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 CNS disorders. Emalex is in late-stage development of a new class of drug for patients with Tourette syndrome and other conditions that have limited treatment options. Visit https://emalexbiosciences.com to learn more. About Paragon Biosciences Paragon Biosciences, founded by Jeff Aronin, is a global life science leader that creates, builds and funds innovative biology-based companies. Our portfolio companies accelerate scientific breakthroughs to solve some of society’s most challenging problems. Learn more at https://www.paragonbiosci.com.

Among the 49 adult patients recruited for the trial, 41.2% of patients dosed with Ecopipam experienced some form of relapse. Credit: Shutterstock / Studio Romantic Emalex Biosciences is eyeing approval for its Tourette syndrome therapy after it achieved its primary and secondary endpoints in a Phase III study. The US company’s central nervous system (CNS) disorder drug ecopipam, a dopamine-1 receptor antagonist treatment, met both primary and secondary endpoints in both paediatric and adult patients living with the condition that can lead, in some cases, to involuntary tics and vocalisations. The drug achieved a 41.9% of paediatric patients relapse against the placebo group’s 68.1%. Following the positive data, Emalex Biosciences said it will be meeting with the US Food and Drug Administration (FDA) and other global health authorities to discuss the submission of a New Drug Application (NDA) later in 2025. The DIAMOND registrational study (NCT05615220) examined a total of 167 paediatric subjects and 49 adult subjects with Tourette syndrome. The trial saw a clinically meaningful reduction in vocal and motor tics in paediatric patients receiving ecopipam during a 12-week open-label period. Patients were then randomised to remain on the study drug or be moved to a placebo for a 12-week double-blind withdrawal period. Among the 49 adult patients recruited for the trial, 41.2% of patients dosed with ecopipam experienced some form of relapse against the placebo arm’s 67.9% of subjects. Frederick Munschauer, chief medical officer for Emalex Biosciences, said: “These results strengthen our confidence in ecopipam as a potential first-in-class treatment for patients with Tourette syndrome. “The topline data from our large, multi-national, randomised withdrawal study show a statistically significant benefit for ecopipam in maintaining clinically meaningful reductions in vocal and motor tics for paediatric subjects with Tourette syndrome as compared to placebo.” Research by GlobalData uncovered that in 2024 the global market for Tourette syndrome therapies stood at approximately $248m, with that figure expected to plummet down to just $84m by the end of 2030. Whilst there are many marketed drugs, Otsuka Holdings’ Abilify (aripiprazole) is the most commonly used antipsychotic therapy and is expected to bring in around $192m by the end of 2025. GlobalData is the parent company of Clinical Trials Arena. The US Centers for Disease Control (CDC) estimates that across the US one out of every 333 (0.3%) children between the ages of three and 17 has been diagnosed with the condition. Additionally, 44% of those children have been diagnosed with moderate or severe Tourette syndrome, with boys three times more likely to live with the condition than girls. Previously in results taken from a Phase IIb trial , ecopipam was able to reduce the number of tics experienced by a paediatric patient by 30% compared with placebo. Eric Messner, Emalex Biosciences’ CEO, added: “The Emalex team worked closely with physician investigators and patient advocates throughout the drug development process and we are hopeful that ecopipam can provide symptomatic relief from the tics suffered by patients with Tourette syndrome. We’re entering a new era of progress for people with central nervous system conditions with limited or no treatment options.” Elsewhere in the market of Tourette therapies, Israeli pharmaceutical company SciSparc has received approval from the US Food and Drug Administration (FDA) to begin a Phase IIb clinical trial of SCI-110 in adults living with the condition.