AstraZeneca, Ionis Unveil Complete Phase 3 Data For ATTR Candidate, Says Drug Halts Disease Progression

  • Ionis Pharmaceuticals Inc IONS and AstraZeneca Plc AZN announced that the Phase 3 NEURO-TTRansform study for eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) met all co-primary endpoints and secondary endpoints at 66 weeks versus an external placebo group.
  • The companies released topline data in the last week of March.
  • Ionis and AstraZeneca seek regulatory approval for eplontersen for ATTRv-PN in the US, with a PDUFA action date of December 22.
  • Eplontersen halted patients' disease progression after 66 weeks in ATTRv-PN, a fatal neurodegenerative disease.
  • In a composite score of polyneuropathy clinical measurements like muscle strength and blood pressure, patients taking eplontersen recorded a 0.28-point average increase from baseline compared to a 25.06-point average increase in the placebo group.
  • The difference of 24.8 points between the groups was good for a p-value of p<0.0001. 
  • Two other co-primary endpoints showed eplontersen resulted in an 82% reduction of serum TTR concentration in active arm patients compared to 11% in the placebo group and a 19.7-point average increase in quality of life questionnaire.
  • Eplontersen is currently being evaluated in the Phase 3 CARDIO-TTRansform study for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a systemic, progressive, and fatal condition that typically leads to progressive heart failure and often death within three to five years from disease onset.
  • Price Action: IONS shares are down 3.55% at $35.62, and AZN shares are up 0.13% at $75.70 on the last check Monday.
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