Phase 3 data for PCSK9 inhibitor wows

  title={Phase 3 data for PCSK9 inhibitor wows},
  author={Cormac Sheridan},
  journal={Nature Biotechnology},
  • C. Sheridan
  • Published 1 December 2013
  • Biology
  • Nature Biotechnology
Pfizer’s decision to move its proprotein convertase subtilisin kexin 9 (PCSK9) inhibitor bococizumab into phase 3 trials, which it disclosed on October 29, adds further momentum to the emergence of a highly anticipated new class of cardiovascular disease drugs that dramatically lowers levels of low-density lipoprotein cholesterol (LDL-c). A couple of weeks previously, Regeneron, of Tarrytown, New York, and Paris-based Sanofi, who are codeveloping a rival antibody called alirocumab, reported the… 
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Low Density Lipoprotein Receptor and its molecular properties are involved in lipid homeostasis, so potentially sets the therapeutic goals in cardiovascular patients, which is usually debated.
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Association of Rare Loss-Of-Function Alleles in HAL, Serum Histidine: Levels and Incident Coronary Heart Disease.
Three LoF mutations in HAL were associated with increased histidine levels, which in turn were shown to be inversely related to the risk of CHD among both African Americans and European Americans.
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Immunoprecipitation and immunoblotting of plasma for PCSK9 provided direct evidence that the serine protease is present in the circulation and identified the first known individual who has no immunodetectable circulating PC SK9, demonstrating that PCSK 9 plays a major role in determining plasma levels of LDL-C and provides an attractive target for LDL-lowering therapy.
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Low LDL cholesterol in individuals of African descent resulting from frequent nonsense mutations in PCSK9
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