Astrocyte Pharmaceuticals Inc., a Groton-based clinical-stage biotechnology company, received a Phase I Small Business Technology Transfer (STTR) grant of approximately $400,000 from the National Institute on Drug Abuse (NIDA), part of the National Institutes of Health (NIH), to evaluate its lead drug candidate, AST-004, as a potential treatment for substance use disorders.

The award, made jointly with the Oklahoma Medical Research Foundation (OMRF) and The University of Texas at San Antonio, will support a collaborative preclinical research program evaluating oral AST-004 in rodent models of psychostimulant addiction.  

Substance use disorders remain a significant public health challenge; the 2023 National Survey on Drug Use and Health found approximately 48.5 million Americans aged 12 and older met criteria for a substance use disorder in the previous year. Despite decades of research, there are currently no FDA-approved medications for stimulant use disorders such as cocaine and methamphetamine addiction.

AST-004 enhances astrocyte mitochondrial energy production through activation of adenosine A1/A3 receptors preferentially expressed on astrocytes. Previous research has demonstrated efficacy across multiple animal models of ischemic stroke and traumatic brain injury, and the new program will test whether enhancing astrocyte energy metabolism can favorably modify behaviors associated with psychostimulant use and relapse. The project will evaluate whether oral AST-004 reduces cocaine and methamphetamine self-administration, drug seeking, and relapse-like behaviors in rat and mouse models while preserving normal reward-related function.

William Korinek, Ph.D., Chief Executive Officer of Astrocyte Pharmaceuticals, said, "This award builds on the tremendous momentum our company has achieved over the past year, including the successful completion of our Series B financing and continued advancement of our clinical development programs. We are building an increasingly broad body of evidence around AST-004, from our Phase 2 program in sports-related concussion, to planned clinical development in complicated mild TBI and acute ischemic stroke, and now promising research in substance use disorders."

AST-004 has completed two Phase 1 clinical safety studies and is currently in a Phase 2 clinical trial for sports-related concussion in Australia, with additional programs advancing in complicated mild TBI and acute ischemic stroke.