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Selection of Aptamers for Amyloid β-Protein, the Causative Agent of Alzheimer's Disease
15:23

Selection of Aptamers for Amyloid β-Protein, the Causative Agent of Alzheimer's Disease

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The BACE-1 inhibitor CNP520 for prevention trials in Alzheimer's disease

Ulf Neumann1, Mike Ufer2, Laura H Jacobson3

  • 1Neuroscience, Novartis Institute for BioMedical Research, Basel, Switzerland ulf.neumann@novartis.com cristina.lopez_lopez@novartis.com.

EMBO Molecular Medicine
|September 19, 2018
PubMed

Insights

The BACE-1 inhibitor CNP520 shows promise for Alzheimer's disease prevention by reducing amyloid-beta. Early trials in healthy adults demonstrate safety and efficacy, leading to further studies.

Area of Science:

  • Neuroscience
  • Pharmacology

Background:

  • Alzheimer's disease (AD) involves amyloid-beta (Aβ) plaque deposition initiated by beta-site amyloid precursor protein cleaving enzyme-1 (BACE-1).
  • Clinical trials targeting later AD stages have faced challenges, highlighting the need for early, asymptomatic intervention.

Purpose of the Study:

  • To evaluate the safety, tolerability, and Aβ-lowering effects of the BACE-1 inhibitor CNP520 in healthy older adults.
  • To assess the suitability of CNP520 for Alzheimer's disease prevention studies.

Main Methods:

  • Pre-clinical studies in rats, dogs, and APP-transgenic mice to assess efficacy and toxicology.
  • A Phase I/II clinical study in healthy adults aged ≥60 years old to evaluate safety, tolerability, and cerebrospinal fluid (CSF) Aβ levels.

Main Results:

  • CNP520 demonstrated dose-dependent reduction of Aβ in CSF in human participants.
  • Animal studies showed reduced brain and CSF Aβ and plaque deposition, with no significant toxicological findings.
  • CNP520 was found to be safe and well-tolerated in healthy older adults.

Conclusions:

  • CNP520 exhibits a favorable safety profile and effectively reduces Aβ levels, supporting its advancement into pivotal prevention studies for Alzheimer's disease.
  • The Generation Program has initiated long-term studies based on these promising results.
Keywords:
Alzheimer's diseaseBACE‐1 inhibitordrug discoverypreventionβ‐amyloid

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