Ionis Pharma - AstraZeneca Antisense Medicine Met Its Primary, Secondary Goals To Lower Cholesterol

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Ionis Pharmaceuticals Inc IONS and its partner, AstraZeneca Plc AZN, have announced data from the ETESIAN Phase 2b study of ION449 (AZD8233).

  • The drug is an investigational antisense medicine designed to reduce blood cholesterol levels in patients with hypercholesterolemia by targeting proprotein convertase subtilisin/kexin type 9 (PCSK9). 
  • The study met its primary endpoint of reducing serum LDL-C levels by up to 79%. 
  • Both the 50mg and 90mg doses reached statistically significant and clinically meaningful reductions in LDL-C levels from baseline of 73% and 79%, respectively, versus 2% for placebo. 
  • Read Next: Ionis-Biogen Partnered Amyotrophic Lateral Sclerosis Trial Disappoints.
  • The trial also met the secondary endpoints, including significantly reducing PCSK9 levels by 94%. The 50mg and 90mg doses achieved reductions in PCSK9 levels of 89% and 94%, respectively, versus 5% for placebo.
  • The reductions in LDL-C and PCSK9 were maintained until week 14 (6 weeks after the last dose).
  • ION449 was generally well tolerated and demonstrated a potential efficacy profile for a self-administered subcutaneous monthly dose regimen.
  • Price Action: IONS shares are trading lower by 2.47% at $37.94, and AZN is down 0.42% at $66.53 on Monday's last check.
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