New data from researchers at the University of California at Riverside show that the novel chemical entity, NLX-101, potently reverses sensory overload in a transgenic mouse model of Fragile X syndrome. The study, published in the peer-reviewed journal Neuroscience, examined the effects of NLX-101 on audiogenic seizures (i.e., seizures that are triggered by a loud noise) in mice that lack expression of Fragile X Messenger RibonucleoProtein (FMRP), thus mimicking human Fragile X syndrome. NLX-101, a selective 5-HT1A receptor biased agonist, potently and almost completely protected the mice from seizures, an effect that was maintained upon repeated treatment and reversed by co-administration of selective 5-HT1A receptor antagonists.
The results, generated in collaboration with Drs. Khaleel Razak and Xin Tao, suggest that NLX-101 can attenuate the damaging sensory hypersensitivity seen in Fragile X syndrome patients. Fragile X syndrome, for which there are no approved treatments, is characterized by social impairment and communication difficulties, and is the most common Autism Spectrum Disorder, affecting approximately 50 000 patients in the US and a similar number in Europe. NLX-101 has previously shown robust activity in rodent models of cognition, mood deficits and Rett syndrome, and has begun Phase 1 human study .
Adrian Newman-Tancredi, CEO of Neurolixis, commented: “Autism Spectrum Disorders, such as Fragile X syndrome, cumulatively affect an estimated 1 in 100 children, causing a substantial suffering as well as a medical and social burden. The present data support further development of NLX-101 for treatment of Fragile X syndrome and related clinical indications."
Acute and Repeated Administration of NLX-101, a Selective Serotonin-1A Receptor Biased Agonist, Reduces Audiogenic Seizures in Developing Fmr1 Knockout Mice.
Neuroscience. 2022 Nov 21:S0306-4522(22)00570-X. doi: 10.1016/j.neuroscience.2022.11.014, PMID: 36410632
