Potential neuroprotective effect in neurodegenerative diseases

and a favorable safety profile.

Pridopidine is an orally bioavailable small molecule investigational drug exhibiting potential neuroprotective effect in multiple neurodegenerative diseases with a favorable safety profile.

Pridopidine is the most selective high affinity Sigma-1-receptor (S1R) agonist. The S1R regulates key cellular processes relevant to neurodegenerative diseases, such as calcium homeostasis, cytoskeleton dynamics, restoring mitochondrial health and neurotrophic factor release. S1R is implicated in cellular differentiation, neuroplasticity, neuroprotection, and cognitive functioning of the brain.

Pridopidine positively influences S1R regulated pathways across neurodegenerative and neurodevelopmental indications, including protection against axonal and neuronal injury, restoring spine impairments, enhancing BDNF secretion and restoring mitochondrial function. These mechanisms have been validated in multiple disease models including Huntington Disease (HD), ALS, Neurodegenerative eye disease, Parkinson’s Diseases (PD), Rett Syndrome (RTT), Fragile X and Alzheimer Disease (AD).  Pridopidine’s occupancy of the S1R (full occupancy at low doses) has been validated using PET imaging in humans.

Pridopidine is the first drug to show statistically significant effect on maintenance of functional capacity in HD, as measured by Total Functional Capacity (TFC). This effect was most prominent in early HD patients. This clinical validation further supports the potential effect of Pridopidine on maintenance of functional capacity in HD and other neurodegenerative diseases.


While at low doses, Pridopidine affects S1R, at higher doses it interacts with other targets including dopamine D3, adrenergic α2C and serotoninergic 5-HT1A, which are established therapeutic targets for Parkinson’s Disease Levodopa Induced Dyskinesia (PD-LID). This is the likely mechanism for the clear effect seen in the “gold standard” non-human primate model for PD-LID – the MPTP-Lesioned Macaques. This model is considered highly translatable to success in phase 2 clinical trials.

Pridopidine’s favorable safety profile has been established in clinical trials in 1260 people, exposed to various doses for a total of ~1000 patient years. This extensive safety evaluation supports a favorable safety and tolerability profile which has been well defined.

Pridopidine is currently in Phase 2 clinical development for the treatment of patients suffering from PD-LID, and we are planning to initiate a phase 3 trial in Huntington Disease in the near future. Additional clinical studies in other indications are also under consideration.



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