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AstraZeneca receives 'positive' results from trial on amyloid polyneuropathy drug

By Iain Gilbert

Date: Tuesday 21 Jun 2022

AstraZeneca receives 'positive' results from trial on amyloid polyneuropathy drug

(Sharecast News) - Drugmaker AstraZeneca has received positive high-level results from an interim analysis of its NEURO-TTRansform Phase III trial on eplontersen, the group's hereditary transthyretin-mediated amyloid polyneuropathy drug candidate.
AstraZeneca said on Tuesday that analysis of the study had revealed eplontersen had met its co-primary and secondary endpoints, reaching a "statistically significant and clinically meaningful" improvement in the percentage change of serum transthyretin concentration, and also met its co-primary endpoint of change in the modified neuropathy impairment score - a measure of neuropathic disease progression.

The FTSE 100-listed firm added that the high-level results also showed the trial had reached its secondary endpoint of change from baseline in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy, indicating that treatment with eplontersen had "significantly" improved patient-reported quality of life.

"These promising results show eplontersen has the potential to be a new and much-needed treatment where limited options exist and significant unmet medical need remains," said AZN.

Based on the 35-week interim trial results, AZN and its partner Ionis will seek regulatory approval for eplontersen and plan to file a new drug application with the Food and Drug Administration in 2022.

As of 0845 BST, AstraZeneca shares were up 0.14% at 10,068.0p.

Reporting by Iain Gilbert at


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