Emalex Clears Phase 3 for Tourette Syndrome, Plans FDA Approval

Emalex Biosciences has announced promising results from a Phase 3 clinical trial of its experimental drug, ecopipam, which aims to reduce relapses in both children and adults with Tourette syndrome. The company now intends to seek regulatory approval for the drug following these positive outcomes.

The clinical trial involved an initial 12-week open-label period where all participants received ecopipam to assess its effectiveness in reducing vocal and motor tics. Participants who showed significant improvement were then randomized into a double-blind, 12-week withdrawal phase, receiving either ecopipam or a placebo. The study included 167 pediatric participants and 49 adults from the United States, Canada, and the European Union.

The trial's main focus was on pediatric participants, where 41.9% of those taking ecopipam experienced a relapse, compared to a higher relapse rate of 68.1% among those on placebo. A secondary endpoint also evaluated both adult and pediatric participants, revealing similar results: 41.2% relapse rate for those on ecopipam versus 67.9% for those on placebo. These findings were deemed statistically significant by Emalex.

Regarding safety, the drug was reportedly "generally well tolerated" by participants. Common side effects included somnolence (drowsiness), insomnia, anxiety, fatigue, and headache.

Based in Chicago, Emalex Biosciences plans to engage with the FDA and other international health authorities to submit a new drug application for ecopipam later this year. CEO Eric Messner expressed optimism about the potential of ecopipam in providing relief from the tics associated with Tourette syndrome.

Tourette syndrome is a neurodevelopmental disorder that typically begins in childhood, characterized by chronic vocal and motor tics. While various companies, such as Neurocrine and Noema Pharma, have explored treatments for the condition, Emalex is hopeful that ecopipam may offer a new avenue of relief. Noema Pharma is pursuing a different approach currently in Phase 2b testing.

Emalex acquired ecopipam in 2018 from Psyadon, which had advanced the drug through a Phase 2b trial. The mechanism of ecopipam involves blocking dopamine at the D1 receptor, which could potentially mitigate the symptoms of Tourette syndrome.

In 2022, Emalex garnered substantial financial backing from notable investors to support the registrational trial. Bain Capital Life Sciences led a $250 million Series D funding round, alongside Paragon Biosciences, Valor Equity Partners, Fidelity, and several family offices. This funding was intended to carry the company through the completion of the Phase 3 trial and assist in manufacturing and pre-commercialization efforts.

Emalex was founded in 2018 by Paragon Biosciences, a Midwest biotech incubator and investor led by former Marathon Pharmaceuticals executive Jeff Aronin. Paragon is known for supporting various innovative biotech companies, including Castle Creek Biosciences, Harmony Biosciences, Evozyne, and CiRC Biosciences, which focus on different areas of medical research and treatment.

With these promising results, Emalex is poised to take significant steps towards bringing a potentially effective treatment for Tourette syndrome to patients in need, pending regulatory approval.