Endogenous and synthetic neuroactive steroids (NASs) or neurosteroids have been shown to be effective modulators of neuronal signaling pathways, most notably, the γ-aminobutyric acid A (GABAA) and the N-methyl-D-aspartate (NMDA) receptor systems. These receptors play a major role in inhibitory and excitatory signaling pathways within the central nervous systems and have been the target for many drug discovery efforts over the past decades, although mostly focusing on multiple receptors sites not associated with allosteric modulation. Proposed endogenous ligands have been reported for the allosteric modulatory sites in both receptors and there is growing evidence suggesting that synthetic modulators may have an impact on disease states rooted in the dysregulation of these receptors. The significant unmet medical need for treatment of CNS disorders has increased the interest for these types of novel drugs. This presentation will highlight recent progress in the drug discovery and early clinical development of NAS drug candidates at Sage Therapeutics, which has focused on treating various GABAA and NMDA based disorders.

Advances in neurosteroid drug discovery: GABA and NMDA modulators

2019 Fall National Meeting

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