Industry Spotlight: Astrogen

About Astrogen, inc

Astrogen, inc. is a South Korea-based pharmaceutical company specializing in the research and development of innovative small molecule therapies for neurological and rare diseases. Founded in 2017 by a pediatric neurologist, Astrogen leverages its clinical expertise and cutting-edge research to address unmet medical needs in conditions such as Autism Spectrum Disorder (ASD), Rett syndrome, ADHD, Parkinson’s disease, and more.

Astrogen’s lead program, AST-001, is a proprietary oral liquid formulation that crosses the blood-brain barrier and modulates NMDA receptor activity by indirectly downregulating SK channel function, thereby restoring neuronal excitability and normalizing the excitation/inhibition (E/I) balance in prefrontal cortex neurons.

AST-001 is safe and well-tolerated with exposure in more than 400 subjects in clinical trials setting conducted in South Korea to date. In a Phase 2 study (KCT0007519), AST-001 demonstrated statistically significant improvements in ASD symptoms, especially in communication and motor skills, in a pediatric population.

Ongoing Clinical Trial

Phase 3 (NCT06333964) study will enroll 160 subjects of the age of 2 to 7 years old in a double-blind, placebo-controlled, multicenter, 12-week randomized clinical trial to evaluate the efficacy and safety of AST-001 followed by a 12-week open-label extension in ASD.

Assessment of AST-001’s effect on the deficits in social communication and interactions will be analyzed using Korean Vineland Adaptive Behavior Scales II (K-VABS-II), CGI-S, CGI-I and other measurements.