XPC-A

XPC-A is a selective, potent small molecule enhancer of NaV1.1 (SCN1A) voltage-gated sodium channels being developed by Xenon Pharmaceuticals for the treatment of Dravet Syndrome. It acts as a positive allosteric modulator by selectively disrupting the inactivated state of NaV1.1 channels, demonstrating over 100-fold selectivity over other sodium channel isoforms such as NaV1.2, NaV1.5, and NaV1.6. In preclinical models of Dravet Syndrome (Scn1a+/- mice), XPC-A has been shown to increase the firing of fast-spiking inhibitory interneurons, rebalance the excitation/inhibition ratio, suppress spontaneous seizures, prevent SUDEP (Sudden Unexpected Death in Epilepsy), and improve cognitive and motor functions. The compound is designed for oral administration and is capable of crossing the blood-brain barrier.