FRAgile X syndrome Clinical trial: Unravelling the science behind this Rare disease in Europe

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CONNECTA Therapeutics is a clinical biotech company developing new treatments for unmet Central Nervous System medical needs associated with neuroplasticity dysfunction, such as Fragile X Syndrome (FXS), 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. 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 FXS paediatric and adult patients, including women with childbearing potential. CONNECTA’s technology expands to other neurodevelopmental diseases, such as Rett syndrome, Down syndrome, and autism spectrum disorder, currently under preclinical testing by CONNECTA. Therefore, the success of the FRAXCURE project will boost the development of medicines for neurodevelopmental diseases for children and adults in Europe. Likewise, FRAXCURE will position CONNECTA as a leading biotech in the already highly successful research-based European pharmaceutical sector while reinforcing the competitiveness of the European Union in a key strategic technological field.