What is hepatic encephalopathy?
Hepatic encephalopathy (HE) is a brain disorder caused by liver dysfunction, most commonly seen in people with cirrhosis or advanced liver disease. Due to liver dysfunction, toxins like ammonia accumulate in the bloodstream and impair brain function, in part by inducing neuroinflammation. This causes symptoms such as confusion, sleeping difficulties, concentration and memory impairments, and mood swings. In severe cases, HE may lead to recurrent episodes of mental confusion, severe disorientation, drowsiness and, in the worst cases, coma.
Severe episodes of HE often requires intensive in-hospital care. In the US alone, hepatic encephalopathy causes about 100,000 hospital admissions per year, with an estimated mortality rate of 14-16%. The cost of HE hospital care in the US is estimated to be USD 11.9 billion.
Current treatments and therapeutic opportunities
There are currently no curative treatments for HE, and only few alternatives address cognitive symptoms associated with the disease. Attempts have been made to prevent recurrent episodes by reducing the intestinal production of ammonium. However, the effects are limited, and the risk of new episodes is high.
Xifaxan, an antibiotic, is the only approved drug for the prevention of HE episodes, recording sales of over USD 1 billion in the US alone. However, Xifaxan is not approved to treat the cognitive impairment that occurs during new episodes of hepatic encephalopathy.
Phase 2 results support golexanolone in liver disease
Results from an internationally acclaimed Phase 2 clinical study show that golexanolone improves the quality of sleep and attention and improves brain signaling in patients with severe liver disease (hepatic encephalopathy). This suggests that the drug candidate can affect patients’ cognition positively.
Golexanolone is well-studied in HE, providing important scientific and clinical support to the continued development of Umecrine Cognition’s drug candidates in liver diseases that show neurological involvement. The clinical development of golexanolone in HE is currently pause, with Phase 2 clinical data insights guiding the company’s other projects.