pridopidine

Pridopidine is an orally administered, investigational small molecule drug developed primarily for the treatment of neurodegenerative diseases such as Huntington's disease (HD) and amyotrophic lateral sclerosis (ALS). It acts as a highly selective and potent agonist of the sigma-1 receptor (S1R), a molecular chaperone located at the mitochondria-associated membrane of the endoplasmic reticulum. Activation of S1R by pridopidine regulates key cellular processes crucial to neuronal health, including calcium signaling, mitochondrial function, synaptic plasticity, and neurotrophic factor transport. Pridopidine demonstrates neuroprotective effects in preclinical models by alleviating ER stress, restoring synaptic function, enhancing BDNF expression and secretion, reducing toxic protein accumulation, and improving neuron-muscle connectivity. Although initially thought to target dopamine D2/D3 receptors as a stabilizer or antagonist with some dopaminergic activity in early studies, subsequent research has shown its primary mechanism is through S1R activation with much higher affinity than for other targets[5][6][7][8].

Brand names

huntexil

Other names

pridopidine4-[3-(Methylsulfonyl)phenyl]-1-propylpiperidine4-(3-(Methylsulfonyl)phenyl)-1-propylpiperidine4-(3-Methylsulfonylphenyl)-1-propylpiperidine