Ionis Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved Ionis and AstraZeneca's WAINUA (eplontersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN. WAINUA is the only approved medicine for the treatment of ATTRv-PN that can be self-administered via an auto-injector. The approval was based on the positive 35-week interim analysis from the Phase 3 NEURO-TTRansform study which showed patients treated with WAINUA demonstrated consistent and sustained benefit on the co-primary endpoints of serum transthyretin (TTR) concentration and neuropathy impairment measured by modified Neuropathy Impairment Score +7 (mNIS+7), and key secondary endpoint of quality of life (QoL) on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Nor Norfolk QoL-DN).

Positive results from the Phase 3 NEURo-TTRansform study were published in The Journal of the American Medical Association (JAMA) further demonstrating the benefit of WAINUA across the spectrum of ATTRv-PN at 35, 66 and 85-we weeks. ATTRv-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade. WAINUA is a ligand-conjugatedantis oligonucleotide (LICA) medicine designed to reduce the production of TTR protein at its source.