Neuroinflammation underpins symptomology in several common diseases
Diseases featuring overlapping mechanisms of GABAA overactivation, cognitive dysfunction and debilitating fatigue, where golexanolone could have an ameliorating effect, include, e.g., Parkinson’s disease, schizophrenia, multiple sclerosis, amyotrophic lateral sclerosis, Huntington’s disease, traumatic brain injury, and chronic inflammatory demyelinating polyneuropathy (CIDP). This offers a further scope for indication expansion.
Golexanolone has shown potential in Parkinson’s disease
Umecrine Cognition has already shown promising results in nonclinical research demonstrating golexanolone’s potential therapeutic effect in Parkinson’s disease (PD), including improvements in both motor and non-motor symptoms. The company’s progress in PD has received significant external validation in the form of a research grant from The Michael J. Fox Foundation to support advanced preclinical evaluation and preparation for clinical studies in humans.
What is Parkinson’s disease?
Parkinson’s disease (PD) is a chronic neurodegenerative disorder in which the nerve cells in the brain that produces the signal molecule dopamine gradually die. The loss of dopamine causes movement problems and the concurrent neuro¬inflammation perturbs GABAA signaling, leading to a wide range of non-motor symptoms, including cognitive impairments and sleep disturbances. Neuroinflammation plays a central role in driving this deteriorating process.
PD is most often diagnosed after the age of 60. Among all PD patients, roughly half develop fatigue, and about one-third experience central fatigue – a severe form of fatigue that reduces motivation, slows thinking and makes everyday tasks overwhelming. For those affected, this neurological alteration causes a marked drop in quality of life.
Current treatments and therapeutic opportunities
Today, an estimated 2.6 million people live with Parkinson’s disease in the seven largest pharmaceutical markets (7MM; United States, France, Germany, Italy, Spain, the UK and Japan), including about 1 million in the United States alone. There is no curative treatment for PD. Standard medications – such as levodopa, dopamine agonists and MAO-B inhibitors – are effective for motor symptoms but do not meaningfully improve fatigue or cognitive difficulties.
By normalizing GABAA signaling, golexanolone has the potential to become the first pharmaceutical treatment to address cognitive symptoms and central fatigue in PD patients.