Eplontersen halted ATTRv-PN disease progression and improved neuropathy impairment and quality of life in Phase 3 study through 66 weeks

NEURO-TTRansform study met all co-primary and secondary endpoints Positive results to be presented today at AAN 2023 demonstrate eplontersen efficacy, safety and administration profile may provide an important new treatment option in this fatal disease with significant unmet need Ionis to host webcast on Tuesday, April 25 at 1 p.m. ET CARLSBAD, Calif., April 24, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the Phase 3 NEURO-TTRansform study for AstraZeneca and Ionis' 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 positive results are being presented today in an Emerging Science Session at the American Academy of Neurology (AAN) 2023 Annual Meeting in Boston. ATTRv-PN is a debilitating disease driven by the progressive accumulation of TTR amyloid deposits, which causes progressive nerve damage and leads to organ failure and eventually death.

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