The SLEEP 2020 Annual Meeting will be held virtually due to the COVID-19 pandemic, but that is not stopping Axsome Therapeutics from presenting the results of their Phase 2 CONCERT trial, which evaluated AXS-12 as a treatment for narcolepsy.
Narcolepsy is a neurological disorder that stops the brain from regulating one’s sleep-wake cycle. It is characterized by instantly falling asleep for a few minutes or longer, depending on the severity of one’s symptoms. Some people experience hallucinations while asleep or directly after waking up. People also often have cataplexy, which is the sudden loss of muscle control, along with narcolepsy, but this is not the case for everybody. When people have both disorders it is known as type one narcolepsy. Cataplexy is triggered by a strong emotion, such as fear or anger, and it manifests as uncontrollable muscle weakness or paralysis. Those who have cataplexy without narcolepsy are often misdiagnosed, as doctors think it is a seizure disorder. Depending on severity, cataplexy can be slight eyelid drooping or the inability to remain standing. One is usually awake during a cataplectic attack but are unable to move. As cataplexy is often related to narcolepsy, people fall asleep after an attack.
The most common cause of an attack is laughter. The destruction of a neurotransmitter hypocretin, which regulates wakefulness, is the root of cataplexy. The cause of narcolepsy type two, which is narcolepsy without cataplexy, is unknown. It is believed that genetics play a part in the disorder, but it is not often passed down from parent to child. Symptoms of type two narcolepsy include sleep paralysis, hallucinations, and changes in rapid eye movement (REM) sleep. In terms of treatment, there are drugs available to treat cataplexy. Xyrem, which was approved in 2002 by the FDA, is used to treat narcolepsy itself, but due to its high potential for abuse it is tightly regulated. Besides drugs, people with narcolepsy often take regularly scheduled naps and do not drink alcohol or caffeine before bed.
AXS-12 for Narcolepsy
AXS-12 is an oral potent norepinephrine reuptake inhibitor. It is able to regulate noradrenergic activity, which then helps with wakefulness, enhances cognition, and maintains muscle tone. The FDA has already granted two designations to this treatment: the Breakthrough Therapy and Orphan Drug Designations.
It has recently been studied in the Phase 2 CONCERT trial, which evaluated safety and efficacy. The results will be presented at the 34th Annual SLEEP Meeting of the Associated Professional Sleep Societies. This meeting is the joint effort of the Sleep Research Society and American Academy of Sleep Medicine.
You can read more about the meeting and treatment here.