Pridopidine is an oral drug currently in development for the treatment of Huntington’s disease and ALS. It is administered in a small easy-to-swallow capsule twice a day.
Multiple clinical studies have been conducted providing important understanding about pridopidine’s safety, mechanism of action, and efficacy.
Recent imaging studies in humans show that pridopidine enters the brain and spinal cord, where it activates a protein called the sigma-1 receptor (S1R).
The S1R is highly expressed in the brain where it regulates several cellular mechanisms common to neurodegenerative diseases such as HD and ALS.
Pridopidine has an extensive safety and tolerability profile and was already evaluated in >1300 people including long term evaluation for >5 years.
Pridopidine dose of 45 mg twice daily, the dose currently used in the clinical trials for HD and ALS, is safe and tolerable, with side effects similar to those seen with placebo.