Psyadon Pharmaceuticals, a company focused on treatments for neurological and psychiatric disorders, has announced that its lead product—ecopipam—produced significant reductions in total tic severity scores in children (7-17 years) with Tourette’s Syndrome.
The study’s primary endpoint was a statistically significant reduction in the mean Yale Global Tic Severity Score Total Tic Score (YGTSS-TTS). This is a well-validated and standard measure of the clinical efficacy of drugs treating Tourette’s Syndrome. Ecopipam caused significant reductions in the YGTSS-TTS at both two- and four weeks after initiating treatment. Furthermore, the side effects seen in the subjects taking ecopipam were comparable to those seen in the placebo-treated subjects, including no clinically significant changes in standard clinical lab tests or ECG’s, and no increases in body weights.
“Ecopipam works by blocking the dopamine D1 receptor which differentiates it from any other drug either on the market or currently in development. Since there hasn’t been a mechanistically new drug for the treatment of Tourette’s in over 30 years, this could offer a better option for people who suffer with the disease,” said Richard Chipkin, Ph.D., president and chief executive officer of Psyadon.
Donald Gilbert, M.D., the study’s Principal Investigator and professor and director of the Movement Disorder and Tourette Syndrome clinics at Cincinnati Children's Hospital Medical Center said, “Too many individuals with Tourette’s Syndrome remain symptomatic despite treatment with available drugs, or experience side effects like weight gain or sedation that limit their use. These results demonstrate the efficacy of D1 antagonism, an entirely new approach that doctors and scientists have speculated about for years. As importantly, the subjects in the study tolerated ecopipam very well, with no weight gain, sedation, or heart problems.”
The phase IIb study called “Ecopipam Treatment of Tourette’s Syndrome in Subjects 7-17 Years (PSY302),” was a double-blind, randomized, placebo-controlled crossover study conducted to assess the efficacy and safety of ecopipam in children with Tourette’s Syndrome.
Patients were instructed to take the study medication (either ecopipam or placebo) each evening before bedtime over a four-week treatment period. Patients were evaluated in the clinic every other week with telephone contacts on the alternate weeks. The primary efficacy endpoint was the change in the Yale Global Tic Severity Score-Total Tic Score (YGTSS-TTS), a well-validated rating scale typically used in Tourette’s Syndrome trials. The study was fully enrolled at 40 subjects.