CTN1101

CTN1101 is a first-in-class small molecule neuroplasticity modulator developed by Connecta Therapeutics for the treatment of Fragile X syndrome (FXS). The compound targets the cofilin pathway, a critical regulator of actin cytoskeleton dynamics in dendritic spines. In Fragile X syndrome, the absence of the FMRP protein leads to the overactivation of cofilin, which results in the formation of immature, abnormally thin dendritic spines and impaired synaptic plasticity. CTN1101 is designed to restore normal cofilin activity, thereby promoting dendritic spine maturation and improving cognitive and behavioral outcomes. It has received Orphan Drug Designation from both the FDA and EMA and has successfully completed Phase 1 clinical trials, demonstrating a favorable safety profile, and is currently progressing into Phase 2 development.

Other names

2-[4-[3-(methylamino)-1-phenylpropoxy]phenyl]ethanol hydrochloride2-(4-(3-(methylamino)-1-phenylpropoxy)phenyl)ethan-1-ol hydrochloride