Pridopidine (developmental code name PL-101) is an orally administrated small molecule investigational drug. Pridopidine is a selective and potent sigma-1 receptor agonist. It is being developed by Prilenia Therapeutics and is currently in late-stage clinical development for Huntington's disease (HD) and amyotrophic lateral sclerosis (ALS).
Pridopidine (developmental code name PL-101) is an orally administrated small molecule investigational drug. Pridopidine is a selective and potent sigma-1 receptor agonist. It is being developed by Prilenia Therapeutics and is currently in late-stage clinical development for Huntington's disease (HD) and amyotrophic lateral sclerosis (ALS).
== Mechanism of action == Pridopidine works by binding and activating an intracellular protein called the sigma-1 receptor (S1R) located at the mitochondria-associated membrane (MAM) of the endoplasmic reticulum. The S1R regulates key cellular processes crucial to neuronal health and survival. Selective activation of the S1R is a promising therapeutic target for treating neurodegenerative and neurodevelopmental disorders.
Discovered by embedding cosine similarity (sentence-transformers MiniLM, 384-dim).