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Drug intelligence / Profile preview
DSP-0378
- Development stage
- Unknown
- Lead developer
- Sumitomo Pharma
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
DSP-0378 is an investigational small molecule developed by Sumitomo Pharma that acts as a positive allosteric modulator (PAM) of the gamma-aminobutyric acid (GABA) A receptor. It is distinguished from conventional GABAergic potentiators, such as benzodiazepines and neurosteroids, by its ability to modulate a broad range of GABA_A receptor subtypes expressed in both synaptic and extrasynaptic regions. This distinct mechanism of action is intended to provide broad-spectrum antiepileptic activity while potentially avoiding some of the limitations of traditional therapies. DSP-0378 is currently in Phase 1 clinical development as an oral treatment for rare and severe epilepsy syndromes, including Progressive Myoclonic Epilepsy (PME) and Developmental Epileptic Encephalopathy (DEE).
02
Targets
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