zatolmilast

Zatolmilast is an investigational small molecule drug developed for the treatment of Fragile X syndrome, a leading cause of inherited intellectual disability and autism spectrum disorder. It acts as a selective, brain-penetrant allosteric modulator and partial inhibitor of phosphodiesterase 4D (PDE4D), prolonging cyclic AMP (cAMP) signaling in neurons. This mechanism is believed to promote maturation and stabilization of synapses, potentially improving cognitive function in patients with Fragile X syndrome. Zatolmilast was originally discovered by Tetra Therapeutics, which was acquired by Shionogi in 2020. The drug has received rare pediatric disease designation from the FDA for neurodevelopmental disorders and orphan drug status for Fragile X syndrome. Clinical trials have shown improvements in cognition and daily functioning with a favorable safety profile[1][2][3][5][6][7].

Other names

zatolmilastBPN14770BPN-14770BPN 147702-(4-((2-(3-chlorophenyl)-6-(trifluoromethyl)pyridin-4-yl)methyl)phenyl)acetic acid