PAN1195

PAN1195 is an orally administered, once-daily, CNS-optimized biguanide small molecule under development by Pano Therapeutics. It is designed to target neurodevelopmental disorders, with Fragile X Syndrome as its lead indication, and planned expansion into Phelan-McDermid Syndrome and Tuberous Sclerosis Complex. Mechanistically, PAN1195 acts as a partial inhibitor of mitochondrial Complex I, which indirectly activates AMP-activated protein kinase (AMPK). This cascade acts upstream of the mTORC1/ERK/eIF4E signaling axis, normalizing dysregulated synaptic translation and downstream disease-linked targets such as matrix metalloproteinase-9 (MMP-9). By optimizing transporter-guided design (profiling across organic cation transporter, MATE, and PMAT families), PAN1195 achieves enhanced blood-brain barrier penetration and CNS exposure compared to traditional biguanides like metformin.