PQR626

PQR626 is an orally available, brain-penetrant, small-molecule inhibitor of the mechanistic target of rapamycin (mTOR) kinase, targeting both mTORC1 and mTORC2 complexes. Originally discovered by researchers at the University of Basel and Piqur Therapeutics, the asset is currently being developed by Torqur (a subsidiary of Swiss Rockets) for the treatment of neurological disorders, including tuberous sclerosis complex (TSC) and epilepsy. PQR626 was designed to overcome the metabolic liabilities of its predecessor, PQR620, demonstrating improved stability in human hepatocytes and excellent blood-brain barrier penetration. In preclinical models of TSC and epilepsy, PQR626 significantly reduced mortality and attenuated epileptic seizures without inducing the systemic metabolic side effects, such as hyperinsulinemia, typically associated with pan-PI3K inhibitors.

Other names

4-(difluoromethyl)-5-(4-((3R,5S)-3,5-dimethylmorpholino)-6-((R)-3-methylmorpholino)-1,3,5-triazin-2-yl)pyridin-2-amine