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

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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