May 23, 2024MenuSECTIONS
Golexanolone Phase 2 Trial for PBC Begins With First Patient Randomizations
Avatar photoJuliana Campos, PhD | May 23, 2024
Umecrine Cognition has announced the randomization of the first 2 patients into the second part of its ongoing phase 1b/2 clinical study of golexanolone. The initial randomizations occurred at the Nottingham University Hospital.
This phase 2 study aims to assess the early treatment efficacy of golexanolone on cognitive symptoms in patients with primary biliary cholangitis (PBC), including fatigue, daytime sleepiness, and cognitive function.
“Around one-third of all patients with PBC suffer from severe fatigue and impaired cognition as part of their disease which is very disabilitating [sic] in their daily life. In this study, we evaluate golexanolone’s effect on patients with these symptoms and our hope is that with this study we will find a new and effective treatment that provides the opportunity for a better life for these patients,” said Stephen Ryder, BM BS, DM, FRCP, professor at Nottingham University and director of the NIHR Nottingham Clinical Research Facility.
The study plans to enroll 84 patients across more than 30 clinical research centers in Europe. Participants will be assigned to receive either 40 or 80 mg of golexanolone, or a placebo, twice daily for 28 days in a randomized, double-blind, placebo-controlled trial.
Read more about PBC experimental therapies
The initiation of this phase follows approval from Umecrine Cognition’s internal safety review committee.
Umecrine Cognition plans to conduct an interim analysis once 75% of the participants complete the 28-day treatment to potentially reassess the sample size. Moreover, the company expects to release top-line results in the first half of 2025.
“There are still no treatment options available that address cognitive symptoms in PBC. We are therefore very glad to have initiated phase 2 of our clinical study, which aims to, apart from further documenting the safety profile of golexanolone, assess the therapeutic effect of our lead drug candidate on life-disrupting cognitive symptoms in PBC. We look forward to communicating further milestone achievements in the clinical evaluation of golexanolone as the study progresses,” said Anders Karlsson, CEO of Umecrine Cognition.
The company aims to provide new treatment options that improve both liver function and cognitive symptoms in patients with PBC.
Reference
Umecrine Cognition’s Phase 2 study of golexanolone in Primary Biliary Cholangitis has randomized its first two patients. News release. Umecrine Cognition; May 8, 2024.
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