Emalex's Ecopipam Shows Promise in Phase III Tourette Syndrome Trial, Paving Way for FDA Submission

Emalex Biosciences has announced positive results from its Phase III trial of ecopipam, a novel dopamine blocker for the treatment of Tourette syndrome. The Chicago-based biotech is now preparing to file a New Drug Application (NDA) with the FDA later this year, potentially bringing a first-in-class treatment to patients with this challenging neurological disorder.

Phase III Trial Results Demonstrate Efficacy and Safety

The late-stage study, which included both pediatric and adult patients, showed a significant reduction in relapse rates for those treated with ecopipam compared to placebo. In the pediatric population, 41.9% of patients receiving ecopipam experienced a relapse, defined as a 50% loss of initial benefit in their Yale Global Tic Severity Score. This was substantially lower than the 68.1% relapse rate observed in the placebo group, representing a 50% reduction in relapse risk.

The trial enrolled 167 children and 49 adults, employing a 12-week open-label period followed by a 12-week double-blind randomized phase. The study's primary endpoint focused on time to relapse in pediatric patients, while secondary endpoints included analysis of the combined adult and pediatric population.

Ecopipam was generally well-tolerated, with common side effects including excessive sleepiness, insomnia, anxiety, fatigue, and headaches. Approximately 10% of patients experienced drowsiness, while 7% reported insomnia.

Unique Mechanism of Action and Market Potential

Ecopipam differentiates itself from existing Tourette syndrome treatments by targeting the D1 subtype of dopamine receptors, rather than the D2 subtype targeted by current therapies. This novel mechanism of action could potentially offer improved efficacy and safety profiles for patients struggling with the debilitating effects of Tourette syndrome.

Frederick Munschauer, M.D., Emalex's Chief Medical Officer, expressed confidence in ecopipam's potential, stating, "These results strengthen our confidence in ecopipam as a potential first-in-class treatment for patients with Tourette syndrome." He added that the data "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."

The positive Phase III results follow Emalex's successful $250 million Series D funding round in 2022, which supported the advancement of ecopipam into late-stage clinical development. This substantial increase in investor interest, compared to the company's previous $35 million Series C round, underscores the growing excitement surrounding ecopipam's potential in the Tourette syndrome market.