Emalex seeks FDA nod for Tourette therapy post-Phase III success

Emalex Biosciences is progressing towards obtaining approval for its Tourette syndrome treatment following the success of Phase III trials. The company's drug, ecopipam, a dopamine-1 receptor antagonist, demonstrated significant efficacy in both children and adults afflicted with the disorder, which is often characterized by involuntary tics and vocalizations. The trials revealed that 41.9% of children on ecopipam experienced a relapse compared to 68.1% in the placebo group.

In light of these promising results, Emalex Biosciences plans to engage with the US Food and Drug Administration (FDA) and international health authorities to discuss submitting a New Drug Application (NDA) in 2025. The DIAMOND study (NCT05615220) included 167 pediatric and 49 adult participants diagnosed with Tourette syndrome. During the 12-week open-label phase, children on ecopipam exhibited a significant reduction in both vocal and motor tics. Participants were subsequently assigned randomly to continue with the drug or switch to a placebo for a further 12-week double-blind phase.

In the adult cohort, 41.2% of those treated with ecopipam experienced some degree of relapse, compared to 67.9% in the placebo group. Frederick Munschauer, chief medical officer at Emalex Biosciences, expressed confidence in ecopipam as a pioneering treatment for Tourette syndrome, highlighting the statistically significant benefits in maintaining tic reduction among pediatric patients compared to placebo.

Market research by GlobalData indicated that the global market for Tourette syndrome therapies was approximately $248 million in 2024, with projections suggesting a decline to $84 million by 2030. Despite the availability of various drugs, Otsuka Holdings' Abilify (aripiprazole) remains the leading antipsychotic therapy, anticipated to generate $192 million by 2025. The Centers for Disease Control and Prevention (CDC) estimates that in the US, about 0.3% of children aged three to 17 have been diagnosed with Tourette syndrome, with boys being three times more likely to develop the condition than girls. Of these children, 44% are considered to have moderate or severe cases.

Ecopipam had previously shown promising results in a Phase IIb trial, reducing the number of tics by 30% in pediatric patients compared to a placebo. Eric Messner, CEO of Emalex Biosciences, emphasized the collaborative effort between the company, physician investigators, and patient advocates throughout the drug's development, expressing hope that ecopipam will offer symptomatic relief for Tourette syndrome patients. Messner highlighted the potential for ecopipam to usher in a new era of progress for individuals with central nervous system disorders that have few or no treatment options.

In related developments within the Tourette syndrome treatment landscape, SciSparc, an Israeli pharmaceutical firm, has obtained FDA approval to initiate a Phase IIb clinical trial of SCI-110 for adults with the condition. This move signifies continued exploration and innovation in the field of therapies for Tourette syndrome, addressing the significant unmet needs of patients suffering from this challenging disorder.