NLS Pharma has just announced new data which further supports the mechanism of action of Mazindol. Mazindol is the active ingredient in Quilience, a treatment used for narcolepsy. This therapy is an agonist to the Orexom-2 receptor (OX2R). As narcolepsy is the result of an orexin deficiency, Mazindol can help treat it.
This therapy has been used off-label for years as a treatment for narcolepsy through compassionate use programs. However, more research on the therapy’s nuances was needed. The treatment has also been given Orphan Drug Designation for narcolepsy in both the United States and Europe.
That is what this team of researchers is working on.
This data comes from a pre-clinical study in mice led by the University of Lausanne. Mice either did not express OX2R or were perfectly healthy. Mazindol was administered to both groups.
This study found that the mice which did not express OX2R were 70% less sensitive to the treatment. This confirmed that Mazindol was an effective agonist.
The researchers also documented the pathway of the mechanism of this therapy.
The company is continuing its research into this therapy to extend our understanding of Mazindol. However, each study is a critical component of the ongoing research portfolio.
This study was important for confirming the unique mechanism of action of this therapy. It can not only block monoamines within the central nervous system, but it can also have psychostimulant effects by activating OX2R.
This therapy is particularly important for narcolepsy patients as it is the first and currently the only treatment which targets OX2R. This also means that this therapy is able to ease the primary symptoms of the condition: cataplexy attacks as well as excessive sleepiness in the daytime.
Mazindol could be an extremely beneficial therapy for both Type 1 Narcolepsy and Type 2 Narcolepsy.
Mazindol has also been studied in a Phase 2 investigation as a treatment for ADHD. This study also showed extremely positive effects of the treatment, and it met all primary endpoints and secondary endpoints. The therapy was well-tolerated with minimal adverse events.
Researchers also suspect it may benefit other sleep-wake disorders.
Ultimately, this study has confirmed Mazindol’s potential to effectively treat the root cause of narcolepsy.
You can read more about this study and Mazindol as a therapy here.