United States Patent and Trademark Office (USPTO) granted a patent (US11974992B2) covering the use of NLX-101, a clinical phase drug candidate, for treatment of Fragile X syndrome (FXS). FXS is a rare genetic autism spectrum disorder which affects about 1 in 5000 individuals and is a leading cause of inherited intellectual disability. There are no approved treatments for Fragile X syndrome. Neurolixis has already shown that NLX-101 has robust activity in transgenic models of another autism spectrum disorder (Rett syndrome) and the new patent expands the company's presence in the orphan autism disorder indication space.
Fragile X syndrome is a genetic disorder caused by a mutation in the FMR1 gene. It is the most common form of inherited intellectual disability and the leading cause of autism-like symptoms in males. Symptoms include intellectual disability, physical features, and behavioral symptoms. Genetic testing and physical examination are used for diagnosis. Supportive care and medications can help manage symptoms but there is no cure or approved treatment. The incidence of Fragile X syndrome is approximately 1 in 4,000 males and 1 in 8,000 females; in the United States 60 to 80 thousand individuals are affected.
Read the full press release.