Right now, there are no available therapies for people living with Fragile X syndrome (FXS). However, clinical-stage biotechnology company Connecta Therapeutics (“Connecta”) is working to change that. In a recent press release, Connecta announced that the company was evaluating its lead candidate CTH120 in an upcoming Phase 1 clinical study.
Connecta describes CTH120 as a first-in-class small molecule (NCE) designed to modulate neuroplasticity (the brain’s ability to change and adapt based on stimuli). Outside of FXS, CTH120 is being developed for Rett syndrome, Down syndrome, and autism spectrum disorder (ASD). Preclinical data found CTH120 to improve cognitive abilities and address social abnormalities related to these conditions.
The Phase 1 trial will be held at Barcelona’s Hospital del Mar Research Institute. 70 participants will enroll. During the study, the research team hopes to evaluate CTH120’s safety and tolerability. Data from the study will be available at some point next year.
About Fragile X Syndrome (FXS)
Fragile X syndrome is the most commonly inherited developmental and intellectual disability. This rare condition results from a lack of fragile X mental retardation 1 protein (FMRP). Normally, this tissue shapes how well you learn and retain memory. FXS tends to impact males more than females. Symptoms appear in infancy and early childhood, often beginning with developmental delays followed by:
- Learning disabilities
- Anxiety, depression, or hyperactivity
- Language difficulties
- A large head with protruding ears, forehead, and chin
- Flat feet
- Problems with coordination
- Sensory issues
- Stuttering or other language difficulties
- Enlarged testicles (after puberty)
There are no cures or disease-specific FXS treatments, which means there is a huge unmet need within this community. This is why research is so crucial: to advance drug development in this sphere and provide those affected with the necessary tools, resources, and therapies. Ultimately, this will help to improve the lives of those living with FXS and their families.