CTH120

CTH120 is a first-in-class small molecule neuroplasticity modulator developed by Connecta Therapeutics for the treatment of Fragile X syndrome (FXS). The drug is designed to address the underlying neurobiological deficits of FXS by modulating the Cdc42 pathway, which is critical for regulating actin cytoskeleton dynamics and dendritic spine morphology. By restoring synaptic plasticity and connectivity, CTH120 aims to improve cognitive and behavioral symptoms in patients with this genetic condition. The compound has completed a Phase I first-in-human clinical trial (NCT05358496) evaluating safety, tolerability, and pharmacokinetics in healthy adult volunteers. The development program, supported by the European Innovation Council (EIC) Accelerator under the FRAXCURE project, is advancing into Phase II clinical trials for both pediatric and adult populations.