AstraZeneca-Ionis Partnered Rare Disease Drug Data Shows Sustained Benefit; Drug Under FDA Review

  • Ionis Pharmaceuticals Inc IONS released topline results from the 66-week analysis of Phase 3 NEURO-TTRansform study of Ionis and AstraZeneca Plc's AZN eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) that leads to peripheral nerve damage and motor disability.
  • At 66 weeks, patients treated with eplontersen demonstrated a statistically significant and clinically meaningful change from baseline versus an external placebo group on the co-primary endpoints of modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression and Quality of life.
  • The study also met its third co-primary endpoint, demonstrating a statistically significant reduction in serum TTR concentration versus an external placebo group. 
  • TTR reductions were consistent with those reported at week 35.
  • Eplontersen exhibited a safety and tolerability profile consistent with that observed at 35 weeks.
  • Data from the 35 and 66-week analyses will be presented as an Emerging Science presentation at the April American Academy of Neurology Annual Meeting. 
  • Earlier this month, FDA accepted a marketing application for eplontersen with a PDUFA action date of Dec. 22, 2023. Eplontersen was granted Orphan Drug Designation in the U.S.
  • Eplontersen is currently being evaluated in the Phase 3 CARDIO-TTRansform study for transthyretin amyloid cardiomyopathy.
  • Price Action: IONS shares are up 0.32% at $34.06 on the last check Monday.
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