Isis Pharmaceuticals Completes Final Trial for Cholesterol-Lowering Drug Mipomersen

Strategic Insights -

Isis Pharmaceuticals, a leader in the development of antisense technology, has successfully completed the fourth and final late-stage trial of its flagship cholesterol-lowering drug, mipomersen. Despite the successful trial outcome, shares of Isis Pharmaceuticals fell 5.6% on the day of the announcement, primarily due to concerns about liver toxicity. The company plans to file applications with European and American regulators in the first half of 2011 to market mipomersen, a drug that could revolutionize the treatment of heart disease.

Key Takeaways:

  • Mipomersen, Isis Pharmaceuticals' flagship cholesterol-lowering drug, has completed its final late-stage trial with successful outcomes in reducing LDL (bad) cholesterol in patients with high cholesterol and high risk of heart disease.
  • Patients with severely high cholesterol experienced a 36% reduction in LDL cholesterol compared to a 13% increase for the placebo group.
  • Patients with high cholesterol and high risk of heart disease experienced a 37% reduction in LDL cholesterol compared to a 5% reduction for the placebo group.
  • Isis Pharmaceuticals and its marketing partner, Genzyme Corp., intend to file applications with European and American regulators in the first half of 2011 to sell mipomersen for treating homozygous familial hypercholesterolemia, an inherited disease causing extremely high levels of LDL.
  • The applications will also include plans for treating high cholesterol in the general population, a market valued at billions of dollars annually.
  • Stanley Crooke, Isis Pharmaceuticals' CEO, emphasized the effectiveness of mipomersen in reducing LDL cholesterol, which outweighs the concerns about liver toxicity.

Statistics:

  • Mipomersen reduced LDL cholesterol by 36% in patients with severely high cholesterol.
  • Mipomersen reduced LDL cholesterol by 37% in patients with high cholesterol and high risk of heart disease.
  • The placebo group experienced a 13% increase in LDL cholesterol in patients with severely high cholesterol.
  • The placebo group experienced a 5% reduction in LDL cholesterol in patients with high cholesterol and high risk of heart disease.
  • Liver toxicity was a concern in the trial, with elevated counts of liver enzymes, but the CEO emphasized the effectiveness of mipomersen in reducing LDL cholesterol.

Sources:

  • North County Times, "Isis Pharmaceuticals Completes Final Trial for Cholesterol-Lowering Drug"
  • McClatchy-Tribune Information Services