1Currently ongoing clinical Phase 1b/2a trial in patients. Recruitment ongoing.
2Currently on hold. Phase 2 date guiding hepatology development.
3Currently evaluating clinical feasibility.
Primary Biliary Cholangitis (PBC) – Phase 1B/2A
- Golexanolone is being developed as a novel therapy for PBC patients who exhibit debilitating central fatigue, i.e., severe fatigue with cognitive impairment.
- In 2023, golexanolone received FDA Orphan Drug Designation for PBC.
- Ongoing randomized, double-blind, placebo-controlled Phase 1b/2a clinical study evaluating golexanolone in 84 patients with primary biliary cholangitis (PBC) who experience central fatigue.
- Participants receive 40 mg or 80 mg golexanolone, or placebo, twice daily for 28 days.
- Primary outcome includes evaluation of treatment efficacy on cognitive symptoms, including fatigue and daytime sleepiness.
Hepatic Encephalopathy (HE) – Phase 2 (On pause)
- Results from an internationally acclaimed Phase 2 clinical study in hepatic encephalopathy patients show that golexanolone is safe and well tolerated, and that golexanolone produces significantly improved sleepiness, brain activity, and vigilance.
- Based on these important insights, the study’s results support the continued development of golexanolone in hepatological indications, such as PBC.
- The development of golexanolone in hepatic encephalopathy is currently on pause; Phase 2 clinical data insights are guiding the company’s other projects.
Parkinson’s Disease (PD) – Pre-clinical
- Peer-reviewed nonclinical data show that golexanolone improves both movement problems, like trouble walking or coordination, and non-movement symptoms, including fatigue, anxiety, and memory issues.
- Golexanolone reduces neuroinflammation, recovers dopamine production, and lowers levels of harmful proteins linked to PD in disease models.
- Golexanolone exerts its effects without causing common side effects like involuntary movements seen with some currently approved treatments.
- The Michael J. Fox Foundation grant funds the continued preclinical research of golexanolone, with the goal of preparing the drug for future testing in humans.
Broad Potential Indications (Neurology) – Pre-clinical
- Neuroinflammation underpins cognitive symptoms and disease progression in several large disease areas.
- Umecrine Cognition’s first-in-class drug candidates have documented potential to address cognitive impairment and central fatigue.
- Umecrine Cognition continuously evaluates the possibilities to expand into disease areas that overlap with current projects, where synergies and previous insights create new strategic development opportunities. Such areas include, among others, Parkinson’s disease, Alzheimer’s disease, and multiple sclerosis.