Positive Phase III results have been presented on eplontersen, a ligand-conjugated antisense therapy from US RNA-targeted drug developer Ionis Pharmaceuticals (Nasdaq: IONS) and Anglo-Swedish pharma major AstraZeneca (LSE: AZN).
In a planned 35-week interim analysis of the NEURO-TTRansform study in hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), eplontersen showed a statistically-significant and clinically-meaningful change from baseline for co-primary and secondary endpoints compared to the external placebo group.
"A positive impact on disease progression and improved quality of life in a substantial number of patients"Eplontersen also demonstrated a favorable safety and tolerability profile. The study results were presented at the International Symposium on Amyloidosis in Heidelberg, Germany, on Wednesday, when shares in Ionis closed 4% higher.
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