CASTRES, France - ColoradoDesk -- A research team specialized in the study of nervous system disorders has found that NLX-112, a serotonin 5-HT_1A receptor agonist developed by Neurolixis, reversed motor deficits in a transgenic model of Spinocerebellar ataxia3 (SCA3), a rare genetic orphan disease. The team led by Prof. Patricia Maciel, at the University of Minho, Portugal, reported online in the journal, Neurobiology of Disease, that NLX-112 restored motor function in nematode worms that express the same gene mutation as that seen in SCA3 patients.

Prof. Maciel commented: "SCA3, also known as Machado-Joseph disease, is an inherited condition. Symptoms including clumsiness, loss of strength, and tremor in the arms and legs that worsen over time, eventually leading to very severe disability. Our new data suggest that NLX-112 may improve the condition of patients suffering from this debilitating disorder."

Neurolixis is now collaborating with Prof. Maciel's team to test NLX-112 in transgenic SCA3 mice. The project, supported by the US Dept. of Defense, will determine whether extended NLX-112 administration attenuates SCA3 symptoms and slows down their development.

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Adrian Newman-Tancredi, CEO of Neurolixis commented: "SCA3 is incurable and there is no approved treatment. If the results seen in the transgenic nematode worm model are translated to transgenic SCA3 mice, Neurolixis will seek to develop NLX-112 in clinical trials for SCA3 patients. NLX-112 has previously been tested in over 500 patients for other indications so we know it is safe and well tolerated."

NLX-112 is currently undergoing a clinical Phase 2A study for treatment of L-DOPA-induced dyskinesia in Parkinson's disease patients and the new SCA3 data suggests that it may also have utility for treatment of additional movement disorders.

Full publication – Open Access
Identification of the 5-HT_1A serotonin receptor as a novel therapeutic target in a C. elegans model of Machado-Joseph disease.
Pereira-Sousa et al., Neurobiology of Disease, 28 Jan 2021 (Online ahead of print)
doi: 10.1016/j.nbd.2021.105278, PMID: 33516872

About Neurolixis
Neurolixis is a privately held biotechnology company developing therapies for central nervous system disorders. It has two lead compounds, NLX‑112 (Phase 2) which targets treatment of motor disorders, and NLX-101 (Phase 1),  a potential rapid-acting anti-depressant, with utility for treating neuro-developmental disorders including Rett syndrome. In addition, Neurolixis conducts a drug discovery program targeting chronic pain (non-opioid mechanism) and depression.

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About the University of Minho
The mission of University of Minho, founded in 1973, is to create, spread and convert knowledge into value-generating applications. The Life and Health Sciences Research Institute (ICVS) is the Biomedicine R&D Unit of the School of Medicine cluster of the University. The cluster includes  2CA-Braga, a clinical research site that currently runs 1/3 of all clinical trials in Portugal; P5, that with the ICVS conceives and develops new digital medicine products and provides medical services with a digital interface; and B.ACIS, a knowledge transfer interface fostering developments in biomedical research resulting in marketed products that bring clinical benefits to patients.