AstraZeneca Reports Phase 3 Results for Eplontersen in Hereditary Transthyretin Amyloidosis

AstraZeneca today announced results from the Phase 3 NEURO-TTRansform trial evaluating eplontersen in patients with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). The trial met its primary endpoints, demonstrating significant reductions in TTR protein levels and improvements in neurological impairment scores compared to placebo. Eplontersen is an antisense oligonucleotide that reduces TTR production.