NNI-351

NNI-351 is an orally available, experimental small-molecule neurorestorative therapy being developed by Neuronascent to treat developmental neurocognitive disorders, particularly fragile X syndrome and other conditions associated with developmental delay. It is a patented new chemical entity discovered in Neuronascent’s phenotypic screens that promotes postnatal neurogenesis by stimulating proliferation and differentiation of neural progenitor cells in the hippocampus, thereby aiming to reverse neurodevelopmental and behavioral deficits rather than only alleviating symptoms.[1][5][13][9] Preclinical studies indicate that NNI-351 increases new neuron growth, reverses behavioral abnormalities in models of developmental disorders including fragile X syndrome and Down syndrome, and reduces hyperactivity in Down syndrome-model mice, although it does not consistently reverse memory deficits in aging and Down syndrome models.[5][6][13] The drug has received FDA Orphan Drug and Rare Pediatric Disease designations for fragile X syndrome and is being advanced towards early-stage clinical development as a first-in-class therapy targeting disease-related deficits in neurogenesis.[1][3][9][10][14][13]