Evaluation of pridopidine in the transgenic YAC128 mouse model of Huntington disease

By January 12, 2017July 17th, 2020News


Pridopidine, a small molecule in mid-stage clinical development for Huntington disease, demonstrated improvement of motor symptoms in the clinic, as well as in preclinical models, and was originally characterized as a dopaminergic stabilizer. However, the mechanism of action of pridopidine cannot be solely attributed to dopamine modulation, and recent in vitro binding studies showed highest affinity of pridopidine to the sigma-1 receptor. These new preclinical observations in conjunction with efficacy and tolerability results from the recently completed large phase II, 52-week, hypothesis-generating study (PRIDE-HD) provide new insights into the therapeutic potential of pridopidine, not limited to modulation of dopaminergic pathways.