AstraZeneca - Ionis Eplontersen Achieves Positive Data In Rare Disease Trial - AstraZeneca (NASDAQ:AZN), AstraZeneca (OTC:AZNCF), Ionis Pharmaceuticals (NASDAQ:IONS)

AstraZeneca plc AZN and partner Ionis Pharmaceuticals Inc IONS announced a positive interim analysis of eplontersen in hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
ATTRv-PN is a rare inherited condition characterized by an abnormal build-up of a protein called amyloid in the body's organs and tissues.
In Phase 3 NEURO-TTRansform study, eplontersen met the main goals.
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Eplontersen demonstrated a statistically significant and clinically meaningful change from baseline in serum transthyretin concentration and the modified Neuropathy Impairment Score +7, a measure of neuropathic disease progression, versus the historical placebo group.
Eplontersen also showed significantly improved patient-reported quality of life versus the historical placebo group.
In the 36-week interim analysis, eplontersen demonstrated a favorable safety and tolerability profile with no specific concerns.
Based on the results, the companies plan to submit a marketing application to the FDA this year for ATTRv-PN.
Price Action: IONS shares closed at $35.26, and AZN stock closed at $61.09 on Friday.