Emalex Biosciences is advancing toward possible FDA approval for its investigational drug, ecopipam, following positive outcomes in a Phase III clinical trial targeting Tourette syndrome.
The trial demonstrated that ecopipam effectively reduced both motor and vocal tics in pediatric and adult patients, achieving significant results in its primary and secondary endpoints.
Tourette syndrome is a chronic neurodevelopmental disorder characterized by involuntary motor and vocal tics, often leading to significant physical and social challenges. Current treatment options are limited, and many are associated with considerable side effects.
According to the CDC, about one in 333 children aged three to 17 in the US have been diagnosed with the condition. Of those, 44% experience moderate to severe Tourette syndrome, with boys being three times more likely to be affected than girls.
The Phase III DIAMOND study enrolled 167 pediatric and 49 adult participants with Tourette syndrome across the US, Canada and the European Union (EU).
Initially, all participants received ecopipam during a 12-week open-label phase. Those who exhibited notable improvements were then randomized to continue with ecopipam or switch to a placebo for an additional 12 weeks.
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In the pediatric cohort, 41.9% of those maintained on ecopipam experienced a relapse, compared to 68.1% in the placebo group. When considering both pediatric and adult subjects, 41.2% on ecopipam relapsed versus 67.9% on placebo.
In a previous Phase IIb trial, ecopipam reduced tics in pediatric patients by 30% compared to placebo.
Ecopipam was generally well-tolerated among participants. The most frequently reported adverse events included somnolence (drowsiness), insomnia, anxiety, fatigue and headache.
“These results strengthen our confidence in ecopipam as a potential first-in-class treatment for patients with Tourette syndrome,” said Emalex’s chief medical officer Frederick Munschauer, in the company’s news release.
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Unlike existing Tourette syndrome treatments that target dopamine D2 receptors, ecopipam is a first-in-class dopamine-1 (D1) receptor antagonist.
According to Emalex, D1 receptor supersensitivity may play a role in the repetitive and compulsive behaviors characteristic of Tourette syndrome. And existing Tourette syndrome treatments primarily target D2 receptors.
Although several drugs are available on the market, Otsuka Holdings’ Abilify (aripiprazole), a partial agonist of D2 receptors, remains the most commonly used antipsychotic therapy. It is projected to earn approximately $192 million by the end of 2025.
This novel mechanism may offer an alternative for patients who do not respond well to current therapies or who experience undesirable side effects.
With the positive Phase III results, Emalex plans to engage with the FDA and other global health authorities to discuss submitting a New Drug Application (NDA) for ecopipam later this year.
Emalex Biosciences was created by life sciences incubator Paragon Biosciences to develop innovative therapies for central nervous system (CNS) disorders.
In 2022, Emalex secured a $250 million Series D funding round to advance ecopipam into Phase III, marking a significant surge in investor interest compared to the earlier $35 million Series C.
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