Positive results for Ionis and AstraZeneca's rare disease candidate

21 June 2022

AstraZeneca (LSE: AZN) has announced the positive high-level results from the Phase III NEURO-TTRansform Phase III trial of eplontersen.

The British company is working with US RNA-targeted drug developer Ionis Pharmaceuticals (Nasdaq: IONS) to develop the candidate for people with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), a rare disease.

The top-line results show the trial met both of its co-primary efficacy endpoints at a planned interim analysis after 35 weeks.

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