Ionis Pharma's Ligand-Conjugated Antisense Hits Primary Endpoint Goal In Hereditary Angioedema Study

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Ionis Pharmaceuticals Inc IONS has announced data from a Phase 2 study evaluating IONIS-PKK-LRx in patients with hereditary angioedema (HAE).

  • The study met its primary and secondary endpoints, achieving significant reductions in the number of attacks suffered by HAE patients compared to placebo.
  • Data demonstrated a mean reduction of 90% in the number of monthly HAE attacks in weeks one to 17 of the study and a mean decrease of 97% in the number of monthly HAE attacks in weeks five to 17
  • In weeks five to 17, 92% of patients treated with IONIS-PKK-LRx were attack-free compared to 0% in the placebo group.
  • On the safety front, most adverse events were mild with a frequency that was similar between groups.
  • The most common treatment-emergent adverse events were headaches and nausea, which were seen more frequently in the placebo arm than the active treatment arm.
  • The company will present a complete analysis of its Phase 2 study of IONIS-PKK-LRx at a medical conference later this year.
  • Price Action: IONS shares are trading 5.6% higher at $45.37 in market trading hours on the last check Monday.
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