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A Promising Drug for an Inherited Heart Risk Flops, Stunning Doctors

  • On Saturday, researchers reported that Novartis drug pelacarsen failed to prevent cardiovascular events in a trial of more than 8,300 patients, despite reducing the genetic heart disease marker Lp by 78%.
  • Lp is a genetic risk factor for heart disease affecting roughly 20% of the global population, making it a major target for researchers seeking new cardiovascular prevention strategies.
  • Participants received a monthly 70-milligram dose of pelacarsen for a median of 4.7 years, though 20.6% stopped the medication due to adverse effects compared to 7.5% in the placebo group.
  • Dr. Steven Nissen of the Cleveland Clinic, who led the trial, called the results 'very disappointing' and an 'extreme surprise,' as experts had expected the drug to reduce cardiovascular risks.
  • Despite the trial's failure, experts emphasize that screening for Lp remains valuable, while experimental treatments from Amgen and Eli Lilly advance through development pipelines with results expected by 2029.
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The drugs lowered the measured blood values, but did not change what was actually happening in the arteries.

·Helsinki, Finland
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Center

By Brenda Goodman, CNN In the course of scientific discoveries, there are triumphant moments in which researchers present their findings to an absorbed audience who stand up to ovation them.This happened in May in a conference room full of doctors after it was learned that a new drug, Daraxonrasib, almost doubled the survival time of patients with pancreatic cancer.And then there is the reaction that Dr. Steven Nissen expects at the annual confe…

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NBC Dallas-Fort Worth broke the news in Fort Worth, United States on Saturday, November 7, 2026.
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