Neuroplasticity modulation addressed by the network pharmacology approach is the core of CONNECTA’s technology. Particularly, CTH120, an excellent neuroplasticity modulator, is a new investigational medicinal product designed as an effective cure for both adults and children. This patented and proprietary technology has already succeeded in Phase I clinical trial demonstrating safety, tolerability and pharmacokinetics in humans, and its efficacy will be validated through the FRAXCURE project in the corresponding Phase II clinical trials targeting Fragile X Syndrome (FXS) paediatric and adult patients, including women with childbearing potential.
FXS is a neurodevelopmental rare disease without a cure affecting around 1.8 M patients worldwide. FXS is responsible for 30% of intellectual disability and cognitive deterioration in both males and females, starting at paediatric age and chronically persisting for life, impacting in learning abilities, attention problems, hyperactivity, autism and social impairments. Early intervention in children is crucial to provide the greatest chance of developing a full range of neurodevelopmental skills, and more opportunities for their social inclusion.
The FRAXCURE project embraces all the activities to confirm CTH120 as a breakthrough disease-modifying treatment for the FXS paediatric population.
Funded by the European Union under the call HORIZON-EIC-2024-ACCELERATOR-02— EIC Accelerator Open 2024, “FRAXCURE: FRAgile X syndrome Clinical trial: Unravelling the science behind this Rare disease in Europe” with grant agreement number GA 101217931. Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union or European Innovation Council and SMEs Executive Agency (EISMEA). Neither the European Union nor the granting authority can be held responsible for them.