Emalex Biosciences plans to submit a New Drug Application (NDA) for its Tourette syndrome therapy, ecopipam later this year, as the Phase III trial evaluating the therapy met the primary endpoint.
The Chicago-based company, founded by Paragon Biosciences, announced positive topline data from its Phase III registrational trial showing that ecopipam reduced the time to relapse for pediatric subjects. Emalex intends to meet with the US Food and Drug Administration (FDA) and other global health authorities to discuss submitting an NDA for ecopipam later in 2025, as per a press release.
Ecopipam trial data
The Phase III study (NCT06021522) enrolled 167 pediatric and 49 adult participants with Tourette syndrome. After a 12-week open-label period, patients who experienced reductions in vocal and motor tics when given the drug were randomized to a double-blind withdrawal period where they either continued receiving the drug, called ecopipam, or switched to placebo for a further 12 weeks.
The study found 41.9% of participants in the treatment arm relapsed, compared to 68.1% of subjects in the placebo group. Additionally, the trial also met its secondary endpoint by reducing the time to relapse in both pediatric and adult participants, with 41.9% of the participants in ecopipam group suffering a relapse, compared to 68.1% in the placebo arm.
The most common treatment-related adverse events include somnolence (drowsiness), insomnia, anxiety, fatigue, and headache.
“The topline data from our large, multi-national, randomized withdrawal study show a statistically significant benefit for ecopipam in maintaining clinically meaningful reductions in vocal and motor tics for pediatric subjects with Tourette syndrome as compared to placebo,” said Frederick Munschauer, Emalex Biosciences chief medical officer.
Tourette syndrome landscape
Tourette syndrome is a hyperkinetic movement disorder, which is characterized by motor and phonic tics or sudden and brief uncontrollable movements. The exact causes of Tourette syndrome are not known. The disease is currently managed through certain neuroleptic medications or with behavioral therapy.
Ecopipam is a D1 dopamine receptor blocker, which acts by blocking the actions of the neurotransmitter dopamine at the D1 receptor. Emalex notes that D1 receptor super-sensitivity may be a mechanism for the repetitive and compulsive behaviors associated with Tourette syndrome. Ecopipam has received Orphan Drug and Fast Track designation from the FDA for the treatment of pediatric patients with Tourette syndrome.
Other Tourette syndrome therapies currently in development include SciSparc’s synthetic form of tetrahydrocannabinol, SCI-110, Noema Pharma’s PDE10A inhibitor (originally developed by Roche) along with Relmada Therapeutics’ sepranolone (originally developed by Asarina Pharma), GABAA modulating steroid therapy. All three of the therapies are currently being evaluated in Phase II clinical trials.
