Ionis, AstraZeneca report positive outcomes from eplontersen trial
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Ionis Pharmaceuticals (NASDAQ:IONS) on Monday reported positive topline results from the 66-week analysis of the Phase 3 NEURO-TTRansform study of eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
Eplontersen is an investigational medicine designed to reduce the production of transthyretin protein to treat both hereditary and non-hereditary forms of ATTR amyloidosis. Ionis (IONS) is partnered with AstraZeneca (NASDAQ:AZN) on the candidate.
The medicine showed sustained benefit in Phase III trial for ATTRv-PN through 66 weeks, meeting its co-primary endpoints. The trial also met its third co-primary endpoint demonstrating a statistically significant reduction in serum TTR concentration compared to an external placebo group.
The results were consistent with the positive 35-week findings announced in June 2022.
Ionis said: "We are encouraged by the sustained benefit demonstrated by eplontersen and what a self-administered treatment could mean for patients and families affected by ATTRv-PN. Together, with our partner AstraZeneca, we look forward to sharing detailed results from this study at the upcoming American Academy of Neurology Annual Meeting."
AstraZeneca (AZN) and Ionis (IONS) are seeking regulatory approval for eplontersen for the treatment of ATTRv-PN in the U.S. under a global development and commercialization agreement.

In early March, Ionis received FDA acceptance of new drug application for eplontersen.