We are a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system, or CNS, disorders characterized by neuronal imbalance. Normal brain function requires a delicate balance of excitation and inhibition in neuronal circuits, which, when dysregulated, can lead to abnormal function and disease. We are applying insights into the genetic mutations that drive excitation-inhibition imbalance in diseases to select biological targets for severe pediatric epilepsies and more broadly for prevalent psychiatric diseases and neurologic disorders.

We have three product candidates currently in clinical development:

*PRAX-114, an extrasynaptic-preferring GABA_A receptor positive allosteric modulator, or PAM, to treat major depressive disorder, or MDD, and perimenopausal depression, or PMD. 

*PRAX-944, a potentially differentiated selective small molecule inhibitor of T-type calcium channels, to treat Essential Tremor, or ET.

*PRAX-562, our lead rare disease product candidate and third clinical program, is the first selective, persistent sodium current blocker in development to treat a broad range of rare devastating CNS disorders such as severe pediatric epilepsy and adult cephalgia.