Pipeline &
Clinical Studies
Pipeline
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 has been submitted for marketing approval for the treatment of Huntington’s disease (HD) in Europe and is in late-stage development for the treatment of HD in the U.S./RoW and for amyotrophic lateral sclerosis (ALS), with plans to initiate a global Phase 3 study in ALS shortly.
Additionally, clinical and preclinical evaluation of pridopidine in various other neurodegenerative diseases and neurodevelopmental disorders is ongoing.
Indication
Preclinical
Phase 1
Phase 2
Phase 3
Registration
Vanishing white matter disease*
Wolfram syndrome
Rett syndrome
Fragile X syndrome
Neurodegenerative eye disease
Alzheimer’s disease
Parkinson’s disease
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).