Interim data released by Umecrine Cognition have shown that patients with primary biliary cholangitis (PBC) treated with golexanolone—a gamma-aminobutyric acid type A (GABA-A) receptor-modulating steroid agonist—have demonstrated a favorable safety and tolerability profile.
These results support the successful completion by Umecrine Cognition of Part A of its ongoing phase 1b/2, randomized, placebo-controlled, double-blind study of golexanolone for patients with PBC. Based on these findings, the internal safety review committee of Umecrine Cognition has authorized initiating Part B of the analysis.
In Part B of the study, the researchers intend to assess the preliminary treatment efficacy signals of golexanolone in patients with noncirrhotic or Child-Pugh class A cirrhotic PBC who exhibit considerable fatigue and cognitive symptoms while receiving a stable background standard-of-care treatment for the disorder. Participants also will undergo additional evaluations on the safety/tolerability profile of the agent. Final results of Part B are anticipated to be available in mid-2025.
In Part A of the analysis, the participants were treated with orally administered golexanolone 40 mg or placebo twice daily for five days. Based on data regarding the pharmacokinetic profile of golexanolone that were derived from Part A of the study, Part B will continue without any dose adjustments.
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In Part B of the study, approximately 84 evaluable patients will be randomized in a 1:1:1 ratio to twice-daily golexanolone 40 mg, golexanolone 80 mg, or placebo for 28 consecutive days. Part B will be carried out at 35 different treatment centers under the management of Professor David Jones, BM, BCh, FRCP, PhD, OBE. Dr. Jones is the Professor of Liver Immunology, Newcastle University, and Honorary Consultant Hepatologist, Newcastle upon Tyne Hospitals National Health Service (NHS) Foundation Trust, both of which are located in the United Kingdom.
The first patient is anticipated to be enrolled in Part B of the study in the second quarter of 2024. A portion of the assessment also will involve an interim analysis for sample size reevaluation when three-quarters of the participants have completed day 28 of the study.
Umecrine Cognition AB is developing a completely new class of pharmaceutical agents that are active against neurologic disturbances in the brain that may be associated with several underlying diseases that have been linked to highly decreased cognitive functioning and wakefulness.
According to Andrew Karlsson, CEO of Umecrine Corporation, “We are delighted by the successful outcome of the first part of our phase 1/2 study and are now looking forward to progressing the clinical program based on the intriguing data generated so far.”