KDventures

The survey was conducted with Newcastle University, PBC Foundation and MME/Indegene and included 250 patients with PBC, all receiving standard-of-care treatment. In total, 92 percent reported central fatigue and related cognitive symptoms, such as “brain fog”, and 44 percent were classified as having severe fatigue.

Patients with severe fatigue reported significantly worse quality of life than those with milder symptoms. Of these patients, 79 percent reported limitations in usual activities, compared with 23 percent and 12 percent among patients with moderate or mild fatigue, respectively. Among patients with severe fatigue, 44 percent reported being unable to work, working part-time or having retired because of their illness. Overall, 60 percent identified central fatigue as the PBC symptom most in need of treatment. In patients with severe fatigue, quality of life was largely independent of concomitant itch.

The findings highlight the substantial need for treatments targeting fatigue and cognitive symptoms in PBC, as current therapies primarily address the underlying liver disease. Umecrine Cognition is developing golexanolone to address these symptoms. The first part of its Phase 1b/2a study showed that the drug candidate was safe and well tolerated. Topline study results are expected in November 2026.

“The survey shows how much fatigue and cognitive symptoms affect patients’ daily lives despite ongoing standard-of-care treatment. This highlights the need for new treatment options. We look forward to new clinical data from our study of golexanolone,” says Viktor Drvota, CEO of KDventures and Umecrine Cognition.