Eplontersen Phase 3 results published in JAMA show consistent and sustained benefit

Key Facts

• • Publication of NEURO-TTRansform study in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed eplontersen halted disease progression and continuously improved quality of life at 35-, 66- and 85-week analyses * Data demonstrates consistent and sustained benefit on all co-primary and secondary endpoints , /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that The Journal of the American Medical Association (JAMA) published positive results from the Phase 3 NEURO-TTRansform study of eplontersen, an investigational treatment for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
• Results from the week 66 primary analysis showed that eplontersen-treated patients demonstrated improvement across all co-primary and secondary endpoints, including serum transthyretin (TTR) concentration, neuropathy impairment and quality of life, compared to the external placebo group.
• In the NEURO-TTRansform Phase 3 study, patients treated with eplontersen demonstrated consistent and sustained benefit on the three co-primary endpoints of serum transthyretin (TTR) concentration, neuropathy impairment measured by modified Neuropathy Impairment Score +7 (mNIS+7) and quality of life (QoL) on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN): * Eplontersen achieved a least squares (LS) mean reduction of 82% in TTR serum concentration from baseline at 65 weeks compared to an 11% reduction from baseline in the external placebo group (p<0.001).
• • In addition, eplontersen improved QoL (on Norfolk QoL-DN), demonstrating a 5.5 point LS mean decrease at 66 weeks (improvement) on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN), compared to a 14.2 point increase (worsening) in the external placebo group (19.7 point LS mean improvement; p<0.001).

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