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Ionis and AstraZeneca Present P-III (NEURO-TTRansform) Study Results of Eplontersen for Hereditary Transthyretin-Mediated Amyloid Polyneuropathy at ISA 2022

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Ionis and AstraZeneca Present P-III (NEURO-TTRansform) Study Results of Eplontersen for Hereditary Transthyretin-Mediated Amyloid Polyneuropathy at ISA 2022

Shots:

  • The P-III (NEURO-TTRansform) study evaluating eplontersen vs PBO in patients with ATTRv-PN
  • The results showed a significant & clinical change from baseline for its co-primary & secondary efficacy EPs @35wks., 81.2% mean reduction in co-primary EPs of serum TTR concentration which showed reduced TTR protein production, the treatment effect was observed on the co-primary EPs of mNIS+7. The study also met its 2EPs i.e., change from baseline in Norfolk QoL-DN which showed an improvement in patient-reported QoL
  • The therapy showed an improvement in neuropathy impairment & has a favorable safety & tolerability profile, the rate of TEAEs was low or similar to PBO. The companies plan to submit an NDA to the US FDA in 2022

Ref: PRNewswire | Image: Ionis 

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Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.

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