AU403, a new drug that targets brain receptors linked to Alzheimer's disease

Evaluation of a specific LXR/PPAR agonist for treatment of Alzheimer's disease

NIH-funded research Auburn University at Auburn · NIH-11307621

Researchers aim to find out if a new medicine called AU403 that activates specific brain receptors (LXRβ and PPARγ) can reduce Alzheimer’s-related brain changes in people with Alzheimer's disease.

Quick facts

Grant typeNIH-funded research
Study typeNIH-funded research
Funding institutionAuburn University at Auburn NIH-funded
Lab location1 site (Auburn, UNITED STATES)
Project IDNIH-11307621 on NIH RePORTER

What this research studies

The team will test AU403 in lab and animal experiments to measure how strongly it activates the target receptors and what doses produce effects versus harm. They will study how the drug is absorbed and distributed after oral or IV dosing and measure drug levels in the brain using sensitive chemical tests (LC-MS). Safety testing includes checking effects on human liver cells and looking for changes in liver enzyme activity, plus animal safety studies. The data gathered will be used to prepare AU403 for early human testing or commercial development.

Who could benefit from this research

Good fit: Ideal candidates for future clinical testing would be people with early Alzheimer's disease or mild cognitive impairment due to AD, including those who carry the APOE ε4 gene.

Not a fit: People with very advanced Alzheimer’s, non‑Alzheimer’s dementias, or serious liver disease may be unlikely to benefit from this treatment.

Why it matters

Potential benefit: If successful, AU403 could reduce harmful protein buildup, protect brain cells, and slow memory decline in people with Alzheimer's disease.

How similar studies have performed: Drugs targeting LXR and PPAR pathways have shown benefits in animal models of Alzheimer’s but have not yet been proven effective in humans.

Where this research is happening

Auburn, UNITED STATES

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.