Umecrine raises $2.5M to advance trial testing golexanolone for PBC
First-in-class drug candidate targets cognitive problems, fatigue in patients
Umecrine Cognition has raised around $2.5 million to support an ongoing Phase 1b/2a clinical trial testing its investigational therapy golexanolone for cognitive problems and fatigue in people with primary biliary cholangitis (PBC).
“Golexanolone is a first-in-class drug candidate that has the potential to deliver a clinically meaningful impact,” Viktor Drvota, MD, PhD, Umecrine’s CEO, said in a company press release. “Based on this capital raise, we advance the ongoing clinical Phase 1b/2a study and move closer toward a new potential therapy for primary biliary patients.”
The funding — totaling 24.6 million Swedish kroner — was raised through a convertible loan with attached share options directed to a consortium of long-term shareholders and investors that includes Umecrine’s majority shareholder, Karolinska Development.
The proceeds from the loan will mainly be used to advance patient inclusion and cover operational expenses related to the Europe-based Phase 1b/2a trial (EudraCT2022-000422-16). The study is now in its second part and is expected to be completed in the first half of 2026.
It aims to recruit 84 adults with PBC, who have clinically significant cognitive difficulties and fatigue, across more than 30 sites in eight countries. Eligible participants cannot have any form of irreversible liver scarring, or cirrhosis, that substantially affects liver function.
Golexanolone designed to counteract effects of elevated levels of molecule
PBC is a chronic form of cholangitis, wherein the ducts that carry the digestive fluid bile from the liver to the small intestine become inflamed. Bile can toxically accumulate in the liver and bloodstream, leading to substantial liver damage and related symptoms.
More than half of people with PBC also experience behavioral symptoms, including fatigue and cognitive problems, that are not directly related to the severity of liver disease, but which can substantially impair life quality.
The exact cause of these symptoms is not completely understood, but they are associated with elevated levels of allopregnanolone, a molecule that activates GABA-A receptor proteins on nerve cells. Overactivation of GABA-A receptors have also been linked to cognitive problems.
Golexanolone is designed to counteract the harmful effects of elevated allopregnanolone by blocking its activity at GABA-A receptors. It is designed to modulate GABA-A receptor activity in ways that avoid serious side effects, like seizures or unconsciousness, which can occur when these receptors are entirely suppressed.
In a previous Phase 2 trial (EudraCT2016-003651-30), the oral therapy was found to safely improve cognitive function in people with hepatic encephalopathy, a condition where liver disease causes neurological problems.
Trial resumes after delay due to issues with oral capsules’ production
The ongoing Phase 1b/2a trial is now testing the therapy’s safety and efficacy in people with PBC who have fatigue and cognitive issues. The study was delayed earlier this year due to technical issues related to production of the oral capsules, but resumed last month.
The trial’s Phase 1b part tested the safety, tolerability, and pharmacological properties of golexanolone oral capsules (40 mg), taken twice daily for five days.
Interim results showed the therapy was generally safe and well tolerated, with all reported side effects being mild in severity. Golexanolone also showed favorable pharmacological properties and early signs of reducing anxiety and depression, according to the company.
In the ongoing Phase 2a part, enrolled participants are receiving golexanolone twice daily at two dose levels for 28 days, or about a month. In addition to safety, this part of the study will assess the treatment’s efficacy, including its impact on fatigue, cognitive function, life quality, and other efficacy indicators.
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