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2017 Part the Cloud: Translational Research Funding for Alzheimer’s Disease (PTC)

Phase 1 study with NPT088, a fusion protein to treat Alzheimer's disease

This Phase I clinical trial will determine if the drug, NPT088, is safe and can reduce abnormal protein clumps that accumulate in the brain during Alzheimer’s disease

Franz Hefti, Ph.D.
Proclara Biosciences, Inc.
Cambridge, MA - United States


During Alzheimer’s disease, the build-up of beta-amyloid protein into “plaques” and abnormal tau protein into “tangles” in the brain may interfere with nerve cell function and contribute to cognitive decline. Most drugs being developed are designed to target beta-amyloid or tau, but not both at once. It is possible that targeting beta-amyloid and tau simultaneously could produce synergistic effects resulting in a more effective treatment.

NPT088 is a “fusion protein” which has been shown to prevent the abnormal accumulation of protein clumps including beta-amyloid and tau. NPT088 has a portion that helps it bind to abnormal proteins “fused” to another portion that helps dissolve the clump. In previous studies, Dr. Franz Hefti, Ph.D., and colleagues found that NPT088 reduced both beta-amyloid and abnormal tau in the brains of Alzheimer’s-like mice. The animals also showed improvements in cognitive function following the treatment.

Research Plan

Dr. Hefti and colleagues have already started a Phase 1a clinical trial in healthy volunteers to determine the safety of a single dose of NPT088. For their current study, they will conduct a Phase 1b clinical trial to assess the safety of multiple doses of NPT088 given over 6 months to people with mild to moderate Alzheimer’s disease. The study participants will also have brain scans to measure if the drug can effectively reduce amyloid plaques and tau tangles.


This study is essential for establishing the safety of NPT088 and will help determine if the drug can simultaneously reduce the abnormal build-up of beta-amyloid and tau protein in the brain. If successful, the results of this work could help advance NPT088 to larger-scale clinical trials to further examine the drug’s safety and determine if it can improve cognitive function in individuals with Alzheimer’s disease.

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