NLX-112: Designated Orphan Drug for Spinocerebellar Ataxia Type 3
NLX-112, also known as befiradol, is a novel compound developed by Neurolixis, primarily aimed at treating L-DOPA-induced dyskinesia in Parkinson's disease. It functions as a highly selective full agonist at serotonin 5-HT1A receptors, boasting over 1000-fold selectivity compared to other receptor types.
This compound has shown remarkable potential in reducing abnormal movements in animal models of Parkinson's disease and has successfully completed Phase 2 clinical trials.
In a significant advancement, NLX-112 has been granted Orphan Medicinal Product Designation by the European Commission for the treatment of Spinocerebellar Ataxia (SCA). This designation underscores its potential to mitigate motor dysfunction in preclinical models of SCA type 3 (Machado-Joseph Disease).
A noteworthy aspect of NLX-112's development is its initial testing in Caenorhabditis elegans. Researchers at the University of Minho, led by Prof. Patricia Maciel, utilized a C. elegans model expressing the gene mutation associated with SCA3. Their studies demonstrated that NLX-112 could restore motor function in these transgenic nematodes. This foundational research was critical in supporting further development and subsequent testing in more complex models, such as transgenic mice, ultimately contributing to its designation as an orphan drug for SCA3.
The journey of NLX-112 exemplifies the value of simple model organisms like C. elegans in accelerating drug discovery and development, showcasing how foundational research can lead to significant therapeutic advancements.
Pereira-Sousa et al., Neurobiology of Disease, 28 Jan 2021 (Online ahead of print) doi: 10.1016/j.nbd.2021.105278, PMID: 33516872