Promising Cholesterol-Lowering Drug Could Help Stave Off Alzheimer’s Disease

Credit: Adobe Stock

A new study suggests a cholesterol-lowering pill could do more than protect your heart — it might also help prevent Alzheimer’s disease.

The drug, obicetrapib, is currently being studied for high cholesterol and heart disease. But new data from the phase 3 BROADWAY trial show it may also slow early biological changes linked to Alzheimer’s — especially in people with a genetic risk factor called APOE4, which is present in about one in four people.

Researchers found that patients who took obicetrapib had significantly lower levels of p-tau217, a blood marker tied to Alzheimer’s, than those who took a placebo. The results were seen after 12 months of treatment.

“The ability to reduce pathological biomarker progression suggests a potential to alter disease trajectory in this population, thereby delaying or preventing the onset of symptoms. The established safety profile of obicetrapib, demonstrated across multiple large clinical trials, combined with its oral administration, may greatly facilitate clinical implementation,” said Philip Scheltens, M.D., Ph.D., a professor at Amsterdam University Medical Center who was involved in the trial, in a statement.

What the Study Looked At

The BROADWAY trial enrolled more than 2,500 people with heart disease or genetic high cholesterol. Many participants also carried the APOE4 gene, which raises Alzheimer’s risk. The researchers focused on how the drug affected Alzheimer’s-related blood markers, including p-tau217, neurofilament light chain (NFL), and GFAP.

Compared with placebo, obicetrapib significantly reduced the progression of these markers, especially p-tau217. This was true in both the full study group and the subgroup of patients with APOE4. These kinds of changes suggest the drug could be interfering with the processes that lead to Alzheimer’s, possibly delaying or preventing symptoms.

At the same time, the drug continued to show strong cardiovascular benefits. Participants saw an average 33% drop in LDL (“bad”) cholesterol, along with improvements in HDL, triglycerides, and other lipid levels. Obicetrapib was well tolerated, taken as a once-daily pill, and no major safety concerns were reported.

What’s Next

The full Alzheimer’s substudy results will be presented at the 2025 Alzheimer’s Association International Conference in Toronto this July. The findings are part of a growing body of research exploring how drugs targeting heart health may also benefit the brain.

Earlier trials of obicetrapib in smaller groups showed similar results, including reduced levels of harmful cholesterol in the brain and spinal fluid.

“These results are the culmination of over two decades of dedicated scientific research. Approximately two-thirds of patients with Alzheimer’s disease carry the APOE4 risk isoform that is associated with a much greater risk of developing [Alzheimer’s disease], and the data shared today support our belief that CETP inhibition and specifically raising small functional HDL particles offers a novel and targeted approach to reducing that risk,” John Kastelein, MD, PhD, FESC, chief scientific officer at NewAmsterdam Pharma, said in a statement.

If future results continue to support these findings, obicetrapib could become a powerful new option for people looking to protect both their cardiovascular and cognitive health with a single, easy-to-take medication.