golexanolone

Golexanolone is a neurosteroid medication under development primarily for the treatment of cognitive dysfunction and fatigue associated with chronic liver diseases, such as hepatic encephalopathy and primary biliary cholangitis (PBC), as well as idiopathic hypersomnia. It acts as a negative allosteric modulator of the GABA A receptor, selectively antagonizing the effects of inhibitory neurosteroids like allopregnanolone and tetrahydrodeoxycorticosterone (THDOC) without affecting normal GABAergic signaling. This mechanism allows it to prevent harmful neurosteroid-induced effects in the brain while avoiding side effects typical of traditional GABA A antagonists, such as seizures or unconsciousness. Preclinical studies have shown that golexanolone reduces glial activation, neuroinflammation, and improves cognitive and motor function in animal models of hyperammonemia, hepatic encephalopathy, and Parkinson’s disease. The drug has been granted Orphan Drug Designation by the FDA for PBC and is being developed by Umecrine Cognition[1][2][3][5][6][7][8].

Other names

3α-Ethynyl-3β-hydroxyandrostan-17 E-one oxime