Rare DiseaseAstraZeneca
AstraZeneca and Ionis Report Positive Phase 3 Results for Eplontersen in hATTR Amyloidosis
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Biotechnology Media Insights
Editorial Team · June 25, 2025
AstraZeneca and Ionis announce positive Phase 3 NEURO-TTRansform trial results showing eplontersen significantly reduces TTR protein and improves neurological outcomes.
AstraZeneca and Ionis Pharmaceuticals today announced positive results from the Phase 3 NEURO-TTRansform trial evaluating eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN). The trial demonstrated significant reductions in serum TTR protein levels and improvements in neurological impairment scores.
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