FRAX486

FRAX486 is a small molecule inhibitor of group I p21-activated kinases (PAKs), specifically targeting PAK1, PAK2, and PAK3. Developed by Afraxis, Inc., the compound is designed to treat Fragile X Syndrome and other cognitive and neurological disorders by modulating actin cytoskeleton dynamics within dendritic spines. In Fragile X Syndrome, an overabundance of immature dendritic spines leads to impaired neuronal signaling; FRAX486 works by inhibiting PAK activity to normalize spine density and morphology. Research led by scientific co-founder Susumu Tonegawa at MIT demonstrated that FRAX486 can cross the blood-brain barrier and rescue behavioral abnormalities, including hyperactivity, repetitive movements, and seizures, in Fmr1 knockout mouse models, even when administered to adult animals.

Other names

PAK inhibitorp21-activated kinase inhibitorp-21-activated kinase inhibitorp 21-activated kinase inhibitorGroup I PAK inhibitor