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 file regulatory 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.
