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SAGE-689
- Development stage
- Discontinued
- Lead developer
- Sage Therapeutics
- Modality
- Allosteric Modulators → Classical Binding Small Molecules → Small Molecules, Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules
- Administration
- Intramuscular
01
Overview
SAGE-689 is a next-generation, small molecule neuroactive steroid developed by Sage Therapeutics. It acts as a potent and selective positive allosteric modulator (PAM) of the GABAA receptor, enhancing inhibitory neurotransmission in the central nervous system. The drug was designed for intramuscular administration and targeted acute disorders associated with GABA hypofunction, particularly status epilepticus and other central nervous system (CNS) disorders. Preclinical studies demonstrated that SAGE-689 significantly reduced seizure activity in rodent models of pharmacoresistant status epilepticus, outperforming standard therapies like phenytoin at tested doses. Despite initial promise, clinical development was discontinued before completion of Phase 1 trials for CNS disorders and preclinical development for status epilepticus[1][2][4][5][7].
- Other names
- SAGE 689SAGE689SAGE-689
02
Targets
GABRR (GABA-A receptor subunit rho)
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