A new drug treatment for Tourette syndrome from Emalex Biosciences, a company known for developing solutions for rare and orphan neurological conditions as well as central nervous system disorders, is making waves in the healthcare investment world based on its stellar results from targeting a different part of the central nervous system than other approved drugs on the market. Instead of acting at the D2 receptor, as do antipsychotic medicines Orap, Haldol, and Risperdal, Emalex’s ecopipam blocks dopamine at the D1 receptor, and so far the drug has performed exceptionally well in clinical trials.
Chicago-based Emalex’s planned phase-3 trial for this, its only clinical-stage drug, has attracted Series D financing totaling $250 million in a round led by Bain Capital Life Sciences. Other contributors included Paragon Biosciences, Valor Equity Partners, and Fidelity Management & Research Company. Ecopipam is also in the early development stages for possible treatment of restless leg syndrome with augmentation — meaning that sometimes after treatment with dopamine-dropping drugs, the condition in question has either returned or worsened. For now, though, the on-track phase-3 study will bring in over 220 patients across 90 sites.
The drug’s original clinical trial evaluated results from just shy of 150 Tourette’s patients aged 6 to 17 randomly placed in study drug or placebo groups. Those in the drug group saw a 30% reduction in motor and vocal tic severity in three months, while the placebo group on average achieved just 19%. Side effects found in trial participants included insomnia, fatigue, and anxiety.