Ecopipam is a first-in-class selective dopamine-1 receptor antagonist in Phase 3 clinical development for children and adolescents with Tourette Syndrome (TS) (ages 6 to <18).  In a 12-week phase 2b, randomized, double-blind, placebo-controlled trial (D1AMOND), ecopipam-HCl at a dose of 2 mg/kg/day improved the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) by 30% (p=0.01).

Dose-dependent, ecopipam-related effects included clinical signs, decreased body weight/body weight gain, food consumption, and increased latency times in the Morris Maze (high dose).  There were no adverse effects on other behavioral assessments, sexual maturation, mating/fertility, ophthalmic examinations, clinical pathology, macroscopic or microscopic pathology.  Systemic exposures increased with dose levels.  At the end of the recovery period, ecopipam-related effects demonstrated partial or complete recovery (clinical signs, body weight/body weight gain, food consumption).