Speragen

AST-001 (Speragen) is a first-in-class oral syrup formulation of L-serine being developed by Astrogen for the treatment of core symptoms of Autism Spectrum Disorder (ASD) in pediatric patients. The drug functions by modulating small conductance calcium-activated potassium (SK) channels, which are involved in regulating the excitability of neurons. By targeting these channels, AST-001 aims to restore normal dopamine neuron activity and excitability in brain regions critical for social behavior. The treatment is administered as a weight-tiered syrup twice daily. It has completed Phase 3 clinical trials in South Korea and has been submitted for regulatory approval (NDA) to the Korea Ministry of Food and Drug Safety (MFDS), having previously received orphan drug designation.