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AstraZeneca – Ionis Eplontersen Achieves Positive Data In Rare Disease Trial

  • AstraZeneca plc AZN and partner Ionis Pharmaceuticals Inc IONS announced a positive interim analysis of eplontersen in hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
  • ATTRv-PN is a rare inherited condition characterized by an abnormal build-up of a protein called amyloid in the body’s organs and tissues.
  • In Phase 3 NEURO-TTRansform study, eplontersen met the main goals.
  • Read Next: Ionis Pharma – AstraZeneca Antisense Medicine Met Its Primary, Secondary Goals To Lower Cholesterol.
  • Eplontersen demonstrated a statistically significant and clinically meaningful change from baseline in serum transthyretin concentration and the modified Neuropathy Impairment Score +7, a measure of neuropathic disease progression, versus the historical placebo group. 
  • Eplontersen also showed significantly improved patient-reported quality of life versus the historical placebo group.
  • In the 36-week interim analysis, eplontersen demonstrated a favorable safety and tolerability profile with no specific concerns.
  • Based on the results, the companies plan to submit a marketing application to the FDA this year for ATTRv-PN.
  • Price Action: IONS shares closed at $35.26, and AZN stock closed at $61.09 on Friday.

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