NLX-112, a serotonin 5-HT1A 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.
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 Phase 2A clinical trial 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.
See Full publication – Open Access:
Identification of the 5-HT1A 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
