P. HILLMANN,2 D. RAGEOT3, F. B. ILS2, P. HEBEISEN2, J.B. LANGLOIS3, C. BORSARI3, S. LEISER1, S. L. ROBERDS4, W. LOSCHER5, M. P. WYMANN3, D. FABBRO2


1PsychoGenics, Inc., Paramus, NJ, USA; 2PIQUR Therapeutics, Basel, Switzerland; 3Department of Biomedicine, Basel, Switzerland; 4TS Alliance, Silver Spring, MD, USA; 5University of Veterinary Medicine, Hannover, Germany.


Seizures are observed in 80 -90 % of TSC patients and are one of the disease manifestations that have the biggest impact on patients’ quality of life. Two ATP-competitive inhibitors of the mTOR pathway have been evaluated for their potential to reduce seizures and to extend life span in the Tsc1flox/flox-GFAP-Cre (TSC – GFAP [glial fibrillary acidic protein]) mouse model of TSC- induced epilepsy. POR530 is a potent, selective dual Pl31