What is it

PBC is a chronic liver disease resulting from progressive destruction of the bile ducts in the liver. The disease is characterised by inflammation of the liver, which gradually attacks all liver tissue, and over a period of years may cause scarring which leads to cirrhosis and as a consequence impaired liver function. Common symptoms are fatigue and itching.  


Fatigue is the most common symptom in PBC patients affecting 50% of patients with PBC and has severely disabling effects in approximately 20%.  Is the first symptom noticed by majority of PBC patients. Fatigue is associated with memory changes, difficulty concentrating, sleep disturbances, mood changes, and impaired physical activity. Because fatigue can limit a person’s ability to work and perform their daily activities and affects their mood it has a strong negative impact on patients’ quality of life.  

Fatigue is not related to severity of the underlying liver damage, and, to the frustration of patients it has not been shown to improve with current treatments of PBC.   

Cognitive dysfunction, which is described by patients as “brain fog” – difficulty in concentrating and processing information and short-term memory lapses – is also common in PBC patients. 33% of PBC patients have symptoms of both fatigue and significant cognitive deficits. This is the patient population that Umecrine Cognition is focused on for treatment with the drug candidate golexanolone.   

There are no good therapies which improve the fatigue and cognitive dysfunction associated with PBC.  Effective treatment of these symptoms is identified as a high priority of patients with PBC and their relatives. 

Therapeutic goals

The desired purpose of treatment with golexanolone is to improve fatigue and the cognitive deficits in patients with PBC and to maintain a good quality of life.  


There are approximately 190,000 diagnosed cases of PBC in 7 major pharmaceutical markets (7MM: France, Germany, Italy, Japan, Spain, United Kingdom and United States). 90-95% of these patients are women and the majority of those diagnosed are usually in their 40s or 50s.  

The target patient population for golexanolone in PBC is initially those 33% of patients who present with symptoms of both fatigue and significant cognitive deficits. This equates to 63,000 patients in 7MM. In the key commercial market, the US, there are an estimated 35,000 patients. 

Current treatments

There is no cure, but medications such as ursodeoxycholic acid (UDCA) and obeticholic acid (OCA) may help to slow the progression of liver damage. In most patients, they prevent liver failure.  However, UDCA and OCA do not relieve fatigue or cognitive impairment. There are no other drugs currently available and, as far as we know, no drug under development for the treatment of fatigue and cognitive deficits in patients with PBC.