Ecopipam

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.

iStock, Cristina Gaidau Emalex is gearing up for a New Drug Application for ecopipam in Tourette syndrome later this year. Emalex Biosciences’ experimental dopamine blocker ecopipam cleared late-stage studies on Tuesday, paving the way for the Chicago-based biotech to submissions with the FDA. In the Phase III trial, 41.9% of pediatric Tourette syndrome patients relapsed after ecopipam treatment, as compared with 68.1% of placebo comparators—resulting in a 50% relapse reduction, a statistically significant result, as per Emalex’s news release. These results remained true when analysis was expanded to cover both adults and children. Emalex reported that ecopipam was well-tolerated and its common side effects were excessive sleepiness, insomnia, anxiety, fatigue and headaches. Emalex Chief Medical Officer Frederick Munschauer in a statement said these results highlight the efficacy of ecopipam to elicit and maintain “clinically meaningful reductions in vocal and motor tics” in children with Tourette syndrome—and “strengthen our confidence in ecopipam as a potential first-in-class treatment” in this indication. Emalex plans to meet with the FDA and other international health authorities to discuss the potential regulatory path for ecopipam. A New Drug Application is planned for “later this year,” as per Tuesday’s announcement. Tourette syndrome is a neurological disorder characterized by tics, or sudden and brief uncontrollable movements. In the most severe cases, tics can disrupt a patient’s communication, quality of life and daily functioning. The exact causes of Tourette syndrome are not known and currently the disease is managed through certain neuroleptic medications or with behavioral therapy. Designed to be taken orally, ecopipam differentiates itself from other Tourette treatments by acting on the D1 subtype of dopamine receptors, instead of on the D2 subtype. Dopamine is a critical neurotransmitter involved in controlling motion, and dysfunction in its signaling could lead to movement-related disorders. With ecopipam’s Phase III win on Tuesday—and impending regulatory filings—Emalex pulls ahead of many other biotechs advancing Tourette treatments. One of these is Relmada Therapeutics, which earlier this month acquired sepranolone from Asarina Pharma for €3 million. The asset, a first-in-class GABAA modulating steroid agonist, is currently ready to enter Phase IIb development. Also in the Tourette space are SciSparc , testing a synthetic form of tetrahydrocannabinol, and Noema Pharma , advancing a PDE10A inhibitor, likewise assessing their respective assets in mid-stage studies.