New data from the Phase 3 NEURO-TTRansform study evaluating WAINUA™ (eplontersen) to be presented at the 2024 International Symposium on Amyloidosis (ISA)

Core Insights - Ionis Pharmaceuticals announced new subgroup analyses from the Phase 3 NEURO-TTRansform study of WAINUA™ (eplontersen), showing consistent benefits in neuropathy impairment and improved quality of life across different patient segments [1] - WAINUA was approved by the FDA in December 2023 for treating hereditary transthyretin-mediated amyloidosis polyneuropathy (hATTR-PN) in adults [1][4] - AstraZeneca and Ionis are collaborating on the commercialization of WAINUA in the U.S. and are pursuing regulatory approval in Europe and other regions [2] Study Details - The NEURO-TTRansform study is a global, open-label, randomized trial assessing the efficacy and safety of eplontersen in patients with ATTRv-PN, with evaluations at weeks 35, 66, and 85 [10] - Eplontersen is also being evaluated in the Phase 3 CARDIO-TTRansform study for patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), which is fully enrolled with over 1,400 patients, making it the largest study in this patient population [3] Product Information - WAINUA™ (eplontersen) is a LIgand-Conjugated Antisense (LICA) medicine designed to inhibit the production of transthyretin (TTR) protein, specifically indicated for treating polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults [4][5] - The most common adverse reactions reported in WAINUA-treated patients include decreased serum vitamin A levels (15%) and vomiting (9%) [7] Disease Context - Hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) is characterized by the accumulation of misfolded TTR protein in peripheral nerves, leading to debilitating nerve damage and progressive loss of motor functions, with a typical disability onset within five years of diagnosis [11]