AU403, a new drug that targets brain receptors linked to Alzheimer's disease
Official title: Evaluation of a specific LXR/PPAR agonist for treatment of Alzheimer's disease
Funded at Auburn University at Auburn · NIH-11307621 · Data updated 2026-11-12
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 type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Auburn University at Auburn NIH-funded |
| Lab location | 1 site (Auburn, UNITED STATES) |
| Project start | March 1, 2023 |
| Project end | February 28, 2027 |
| Funding year | 2026 |
| Award (this year) | $344,798 |
| NIH project number | 5R33NS126618-03 |
| Project ID | NIH-11307621 on NIH RePORTER |
Who could benefit from this research
✓Could help
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.
✕Less likely to help
People with very advanced Alzheimer’s, non‑Alzheimer’s dementias, or serious liver disease may be unlikely to benefit from this treatment.
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.
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 gone: 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 University at Auburn
Auburn, United States Active
Researchers
- Principal investigator: Amin, Rajesh H — Auburn University at Auburn
- Study coordinator: Amin, Rajesh H
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.