Pipeline &
Clinical Studies


Pridopidine is a first-in-class, investigational, highly selective and potent sigma-1 receptor (S1R) agonist with neuroprotective properties. It is administered by mouth in a small, easy-to-swallow capsule twice daily. Pridopidine has been studied in more than 1700 people and long-term safety data are available from previous clinical studies, up to 7 years duration. In these investigational studies, pridopidine at the therapeutic dose has an observed safety and tolerability profile comparable to placebo.

Pridopidine is currently in late-stage development for the treatment of Huntington’s disease (HD) and amyotrophic lateral sclerosis (ALS).

Prilenia is continuing the clinical and preclinical evaluation of pridopidine in various neurodegenerative diseases and neurodevelopmental disorders and plans to initiate additional clinical studies in the future, including a global Phase 3 study in ALS.



Phase 1

Phase 2

Phase 3

Vanishing white matter disease*

Phase 1

Wolfram syndrome

Pre Clinical

Rett syndrome

Pre Clinical

Fragile X syndrome

Pre Clinical

Neurodegenerative eye disease

Pre Clinical

Alzheimer’s disease

Pre Clinical

Parkinson’s disease

Pre Clinical
*Investigator-sponsored expanded access

Pridopidine is an investigational product and has not been approved by the US Food & Drug Administration or other ex-US regulatory agencies. Its safety and efficacy have not yet been determined.

Expanded Access

We understand that, in some cases, patients with serious diseases may not be eligible to participate in a clinical study, and there may not be any alternative treatment options for their disease. We are deeply committed to the patient community, and under certain circumstances, we will consider making pridopidine available to individual patients through an expanded access program (also referred to as early access or compassionate use).