Recent studies suggest that extracellular signal-regulated kinase (ERK) plays a critical role in synaptic plasticity (the ability of certain synapses to strengthen or weaken over time) and neurodevelopment. AMO-01 is an inhibitor of the Ras-ERK pathway. In pre-clinical efficacy studies, AMO-01 has been shown to rescue the neuronal phenotype of the multiple knockout mouse models of intellectual disability. This provides strong scientific evidence to support the conclusion that inhibition of the Ras-ERK cascade may have therapeutic benefit in the treatment of intellectual disabilities in humans.
Wang, Zhu et al. 2012. J. Neurochemistry. 212(121), 672-679.