Daegu Medical Company Astrogen Passes Phase 2 Clinical Trial for Autism Treatment Drug

Application for Phase 3 Trial Plan

'New Drug Development Still Distant'

Daegu City announced on the 28th that ‘Astrogen,’ a startup company developing treatments for intractable neurological diseases, has confirmed the efficacy and safety of AST-001 (development name), which is being developed as a treatment for autism spectrum disorder, in a domestic Phase 2 clinical trial, successfully completing the Phase 2 trial for improving core symptoms of autism spectrum disorder in children.

Astrogen, which has grown through customized support for startups such as the Star Venture Promotion Project, was selected as a Baby Unicorn by the Ministry of SMEs and Startups in 2020 and as a Pre-Star Company by Daegu City in 2021. It is a local company preparing for a KOSDAQ listing, leveraging Daegu City’s IPO (Initial Public Offering) support project as a stepping stone.

Its headquarters are located in the Daegu Dong-gu Advanced Medical Complex.

Astrogen has completed the submission of the Phase 2 clinical trial results report to the Ministry of Food and Drug Safety (hereinafter referred to as ‘MFDS’), and on the 16th, it applied for the Phase 3 clinical trial plan (IND), significantly increasing the likelihood that this treatment will be launched as a therapy for core symptoms of pediatric autism spectrum disorder.

Astrogen, a startup located in the Daegu Dong-gu Advanced Medical Complex.

The core symptoms of autism spectrum disorder include persistent deficits in social communication and interaction, as well as restricted and repetitive behaviors, and currently, there are no treatments that improve these core symptoms.

Despite the severe COVID-19 pandemic in the first half of 2021, Astrogen completed patient recruitment for the Phase 2 clinical trial in an unprecedented short period of seven months. The trial involved a total of 151 children aged 2 to 11 years diagnosed with autism spectrum disorder, conducted at major university hospitals nationwide to evaluate the efficacy and safety of the AST-001 treatment.

The Phase 2 clinical trial period lasted 1 year and 4 months, from February 2021 to May 2022, and the results report was submitted to the MFDS in December 2022.

In the Phase 2 clinical trial, statistically significant improvement was confirmed in the primary efficacy endpoint, the Vineland Adaptive Behavior Composite Score, compared to placebo (a control drug used to evaluate the effect of the test drug) (p=0.042). Among the subdomains of the Vineland Adaptive Behavior Composite Score, the communication and motor skills domains also showed significance compared to placebo.

Additionally, significance was confirmed in the Clinical Global Impression-Severity scale (a severity assessment by clinicians) and the Parenting Stress Index, successfully achieving the objectives of the Phase 2 clinical trial for autism spectrum disorder.

Ahn Jung-gon, Director of the Economic Bureau of Daegu City, said, “Daegu City is focusing on supporting related startups at each growth stage to lead knowledge- and technology-intensive future new industries such as healthcare, UAM, non-memory semiconductors, robotics, and ABB. We expect that this achievement by Astrogen will serve as a catalyst for the groundbreaking expansion of new drug development startups and the medical industry in the region.”

A Daegu City official stated, “It is very rare not only domestically but also globally for a specialized treatment for autism symptoms to pass Phase 2 clinical trials. However, to be developed as a new drug, it must pass Phase 3 clinical trials. Currently, only the Phase 3 application has been submitted, and it takes a considerable amount of time to receive approval from the MFDS and conduct the Phase 3 clinical trial. This process must be completed for successful new drug development.”