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PRAX-114
- Development stage
- Phase 3
- Lead developer
- Praxis Precision Medicines
- Modality
- Allosteric Modulators → Classical Binding Small Molecules → Small Molecules
- Administration
- Oral
01
Overview
PRAX-114 is an orally administered neurosteroid and small molecule that acts as a positive allosteric modulator of the GABA A receptor, with a preference for extrasynaptic over synaptic subtypes. It was developed primarily for the treatment of major depressive disorder (MDD), essential tremor, depressive disorders, epilepsy, and previously post-traumatic stress disorder (PTSD). The drug demonstrated antidepressant-like and anxiolytic-like effects in preclinical studies with improved tolerability compared to similar agents due to its selectivity. Clinical development in MDD failed to meet efficacy endpoints in phase 2/3 trials, leading to discontinuation of psychiatric indications; development has also been discontinued for PTSD. As of early 2023, clinical activity focused on movement disorders and epilepsy[1][3][4][9].
- Other names
- PRAX 114PRAX114PRAX-114
02
Targets
GABAA (Gamma-aminobutyric acid receptor)
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