SAGE’s proprietary chemistry platform focuses on two key, validated nervous system targets: GABAA and NMDA receptors, the primary inhibitory and excitatory receptors that mediate “fast” synaptic transmission in the brain. These targets are critically important and are now better understood than ever before due to decades of research and SAGE’s own pioneering work.
Allosteric modulation of a receptor represents a more regulated approach to impacting the levels of neurotransmission rather than directly acting as a full “on or off switch.” SAGE’s data suggest this approach may avoid intrinsic compensatory mechanisms that hamper the activity and safety of many currently available medications.
Identifying selective neuroactive molecules to modulate these receptors without the potential downside of direct agonism or antagonism may allow for improved therapeutic options over the well-established compounds in this class.
Our focus is on the development of compounds initially for acute and orphan indications where preclinical validation and translatable biomarkers support both high confidence for the target and allow rapid entry into human clinical trials. Once established in the acute and orphan settings, opportunities for broader application across similar disease areas exist.