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.
