Umecrine Cognition is currently running a randomized, double-blind, placebo-controlled phase 1b/2a study evaluating golexanolone in patients with primary biliary cholangitis, PBC, who experience clinically significant and debilitating fatigue and cognitive symptoms. The predefined sample-size re-estimation, which remains blinded to the company, was based on the cognitive domain of the clinically validated PBC-40 assessment tool. The analysis assessed the conditional power of the study, i.e., the likelihood of achieving statistically reliable results if the study proceeds according to the current protocol. The interim analysis was triggered by three-quarters of the intended study size having completed the treatment period in the study. The analysis concluded that no changes to the study design or target enrollment are required. The study will therefore continue as planned, with results expected in H2 2026.

The main objective in the ongoing second part of the study is to confirm the safety profile of golexanolone and evaluate the treatment effect of the drug candidate on several secondary efficacy endpoints, including the gold standard PBC-40 tool and the recently validated CGI-PBC tools, to guide the selection of primary efficacy endpoints for a phase 3 confirmatory study.

"We are happy to receive the news that the study of golexanolone is deemed to fulfill its purpose in its current design and are looking forward to its completion later in 2026. The drug candidate addresses a significant medical need in an area with limited treatment options, giving the compound strong commercial potential," says Viktor Drvota, CEO of KDventures and Umecrine Cognition.

About Umecrine Cognition

Umecrine Cognition is developing golexanolone, a drug candidate that acts on the central nervous system to alleviate fatigue and cognitive symptoms in chronic liver disease. Patient enrollment is currently underway at more than 30 European clinical sites, and results from the study are expected to be presented in the second half of 2026.