A novel and safe approach for treating cognitive disorders
Umecrine Cognition is developing pharmaceuticals against neurological disorders in the central nervous system, CNS that are caused by endogenous CNS-active steroids (neurosteroids). The company's primary focus is on the development and commercialization of novel therapeutics to treat CNS related disorders in patients with liver disease, such as Hepatic Encephalopathy (HE) in patients with liver cirrhosis.
Liver disease and its complications account for a growing and substantial disease burden worldwide. Hepatic Encephalopathy is a syndrome of episodically impaired brain function which remains the most common reason for readmission of patients hospitalized with decompensated cirrhosis. Symptoms of HE range from impaired cognitive function (covert HE) to clinically evident impairment (overt HE) manifested by a spectrum of abnormalities ranging from confusion to depressed consciousness and coma. Impaired cognitive function is associated with an increased risk of overt HE and there are today no treatments available that directly target the brain abnormalities believed to responsible for HE.
The company's therapy represents a new target class. The lead compound, golexanolone (GR3027), in clinical development for HE, is a GABAA receptor modulating steroid antagonist (GAMSA) designed to antagonize GABAA receptor activation and neuroinhibition by endogenous neuroactive steroids. Golexanolone has been shown to restore different types of neurological impairments including cognitive and sleep alternations in experimental models. The drug candidate enters the CNS and reverses the inhibitory effects of the neurosteroid allopregnanolone on brain function in humans. Collectively, these findings strongly implicate that golexanolone is a promising novel treatment for a wide range of cognitive disorders.