Long-term safety testing of CMS121 for people with mild to moderate Alzheimer's disease
Official title: Long-term, non-clinical toxicology for advancing CMS121 to Phase 2 trials for AD
Funded at Virogenics, INC. · NIH-11196206 · Data updated 2026-11-12
This project is testing whether the new pill CMS121 can be safely given every day for a long time to adults with mild to moderate Alzheimer's disease.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Virogenics, INC. NIH-funded |
| Lab location | 1 site (Del Mar, United States) |
| Project start | September 24, 2024 |
| Project end | August 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $1,562,187 |
| NIH project number | 5U01AG084549-02 |
| Project ID | NIH-11196206 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for future clinical testing would be adults diagnosed with mild to moderate Alzheimer's disease who meet trial eligibility and safety criteria for daily oral medication.
✕Less likely to help
People with severe Alzheimer's, non‑Alzheimer dementia, or medical conditions that make long-term medication unsafe are unlikely to benefit from this program.
What this research studies
From a patient point of view, the team is doing long-term safety work in labs and animals to look for side effects if CMS121 were taken daily by people with mild to moderate Alzheimer's. CMS121 is a small molecule that helped in multiple mouse Alzheimer models and works differently than current drugs that target amyloid plaques. The researchers will run detailed toxicology experiments to find any organ, metabolic, or other safety problems that could appear after months of use. Those safety results are needed before CMS121 can move into Phase 2 human testing for cognitive benefit.
Why it matters
Potential benefit: If successful, this work could enable human trials of CMS121 and eventually lead to a new treatment that protects the brain and improves thinking and memory for people with Alzheimer's.
How similar studies have gone: CMS121 showed promising results in multiple mouse models, but similar approaches have not yet proven long-term cognitive benefit in humans.
Where this research is happening
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Virogenics, INC.
Del Mar, United States Active
Researchers
- Principal investigator: Raschke, William C — Virogenics, INC.
- Study coordinator: Raschke, William C
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.