We are a neuroscience-focused company with a unique approach to modulating well-validated targets for the treatment of life-threatening, rare CNS (Central Nervous System) disorders.
The precise and intricate instructions needed to accomplish these actions are created in a web of communication between large networks of brain cells, or neurons. Many thousands of neurons must communicate in a precise sequence to allow someone to dance a waltz, perform math calculations or remember where to find a parked car. Because many of these different activities need to happen at the same time, the brain uses a combination of excitatory connections, or synapses, to relay information between neurons and inhibitory connections to restrict the flow of information. A constant, delicate balance between excitatory and inhibitory neurotransmission allows the brain to multi-task without getting the various messages confused.
Our science is focused on using allosteric modulation of specific excitatory and inhibitory neurotransmitter systems to reshape this disturbed balance during seizure or other CNS disorders, thus restoring normal brain function. The allosteric modulator therapies being developed by SAGE modify the activity of critical neurotransmitter receptor systems, which we believe leads to a restoration of a normal balance between excitatory and inhibitory signals within the brain.
Our discovery and development efforts are centered on two critical nervous system receptor families: GABAA receptors, one of the key families of inhibitory neurotransmitter receptors, and NMDA receptors, one of the key families of excitatory neurotransmitter receptors.
Imbalances in GABAA and NMDA receptor activity have been implicated in a broad spectrum of disorders, including disorders of mood, seizure, cognition, anxiety, sleep, pain, epilepsy and movement. These receptor systems are widely regarded as validated drug targets for a variety of CNS disorders, with decades of research and multiple approved drugs targeting these receptor systems. However, many drugs approved to modulate these receptor systems have safety and efficacy limitations related to poor pharmaceutical properties and adverse side effect profiles.
By influencing a natural regulatory pathway within the brain, our therapies have the potential to have a profound effect while maintaining a strong safety profile, and may deliver new options to patients suffering from a number of diseases.