In short
AST-004 is an investigational chemical substance being studied as a cerebroprotective treatment for people with mild complicated traumatic brain injury (TBI). It is given intravenously as a solution for injection and has no marketing authorisation, meaning it is not yet an approved medicine. A single Phase 2 trial (Cosmos Trial, 2026-525554-11-00) is authorised and plans to enrol 48 participants to assess safety and physiological effects.
Key points
- AST-004 is an investigational chemical substance with no marketing authorisation.
- It is being studied in a single authorised Phase 2 trial for mild complicated traumatic brain injury.
- The trial plans to enrol 48 participants and is called the Cosmos Trial.
- AST-004 is given intravenously as a solution for injection.
- The main measures are safety and the change in plasma GFAP, a marker of brain injury.
- The trial is sponsored by Astrocyte Pharmaceuticals Inc.
What is AST-004?
AST-004 is an investigational chemical substance, meaning it is a small molecule that has been synthesised chemically rather than produced from living cells. It is being developed by Astrocyte Pharmaceuticals Inc. as a cerebroprotective treatment, which means it is intended to protect brain tissue from damage. The substance appears in the EU Clinical Trials Information System (CTIS) under the name AST-004 and has no marketing authorisation in the product records, so it cannot yet be prescribed outside of a clinical trial.
How does AST-004 work?
The registry record describes AST-004 as having a cerebroprotective effect, meaning it is intended to reduce or limit injury to brain cells after a traumatic event. The trial measures the change in plasma GFAP (glial fibrillary acidic protein), a protein that is released into the blood when brain cells called astrocytes are damaged. A reduction in GFAP levels would suggest that AST-004 is protecting brain tissue from further harm. The precise molecular target of AST-004 is not described in the available registry data.
Which conditions is it studied for?
- Traumatic brain injury — specifically mild complicated traumatic brain injury, a form of head injury where there is evidence of damage on brain imaging but the person remains conscious. The Cosmos Trial is evaluating AST-004 as a cerebroprotective treatment for this condition.
How is AST-004 given?
AST-004 is given intravenously as a solution for injection. In the Cosmos Trial, the intervention is described as a dose of 1210 mg administered intravenously. The trial also includes a comparison arm that is identical to the test product except without the active substance, meaning a placebo.
What do the clinical trials show?
One clinical trial of AST-004 is registered in the EU Clinical Trials Information System. The Cosmos Trial (2026-525554-11-00) is a Phase 2 interventional study that is currently authorised, meaning it has received regulatory approval to begin but may not yet be recruiting participants. It plans to enrol 48 participants with mild complicated traumatic brain injury.
The trial has two primary measures. The first is safety, assessed by the incidence, frequency and severity of treatment-emergent adverse events (TEAEs) occurring through Day 7. The second is a pharmacodynamic measure: the change in plasma GFAP from baseline through dose completion, 24 hours, Day 7, Day 14 and Day 30, with the effect at 24 hours being of primary interest.
The study compares different doses of AST-004 against a placebo, which is identical to the test product except without the active substance. The trial is sponsored by Astrocyte Pharmaceuticals Inc., and AST-004 appears as the test substance in this single trial record.
What side effects are known?
The registry record does not list any known side effects for AST-004. The primary safety endpoint of the Cosmos Trial is specifically designed to collect information on treatment-emergent adverse events, so safety data will only become available once the trial has been completed and its results reported.