A first-in-class small molecule NCE for neuroplasticity-mediated disorders with social behavior and pro-cognitive properties.
Initially focused on the treatment of fragile X syndrome (FXS), the most common inherited cause of intellectual disability and considered a major unmet need. FXS is caused by a mutation in the X chromosome on the fragile X mental retardation 1 (FMR1) gene. The full gene mutation can cause significant intellectual disability, greatly reduce functional abilities, educational achievement and social-emotional skills.
Excellent preclinical efficacy results: CTH120 rescues the dendritic pathology of FXS, clearly improving cognitive ability and completely restoring the social abnormalities linked to the disorder.
Different approach from other FXS candidates: CTH120 is a multi-target drug with pleiotropic effects.
FXS is a genetic condition that causes a range of developmental problems including learning disabilities and cognitive impairment. It occurs in approximately 1 in 4,000 males and 1 in 8,000 females.
Affected individuals usually exhibit delayed development of speech and language by age 2. Most males with FXS have mild to moderate intellectual disability, while about one-third of affected females are intellectually disabled. Children with FXS may also have anxiety and hyperactive behavior such as fidgeting or impulsive actions. They may have attention deficit disorder (ADD), which includes an impaired ability to maintain attention and difficulty focusing on specific tasks. About one-third of individuals with FXS have features of autism spectrum disorder that affect communication and social interaction. Seizures occur in about 15% of males and about 5% of females with FXS.