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Prilenia Therapeutics About Pridopidine banner image



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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. 

Prilenia Therapeutics ALS Compound
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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.  

Activation of the S1R by pridopidine enhances the clearance of toxic proteins, enhances energy production, and reduces cellular stress and inflammation.

These mechanisms are crucial for a neuron’s function and survival.

Prilenia Therapeutics Pridopidine results

Studies in animal models and human cells show that pridopidine prevents neuronal cell death, and strengthens and enhances the connections between neurons.

Prilenia Therapeutics Prodopidine effects
Prilenia Therapeutics pridopidine fact

Pridopidine has an extensive safety and tolerability profile and was already evaluated in >1300 people including long term evaluation for >5 years.

Prilenia Therapeutics Propidine on HD and ALS

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.

Prilenia holds Orphan Drug Designation for pridopidine in both Huntington’s Disease (HD) and ALS in the U.S. and Europe. In addition, pridopidine recently received Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of HD. Drugs that receive Fast Track designation may be eligible for more frequent communications with the FDA and may also qualify for accelerated approval and priority review of new drug applications.

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