Ionis Reports 35 Week Interim Analysis of Eplontersen in P-III (NEURO-TTRansform) Study for Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
- The P-III (NEURO-TTRansform) study evaluates the efficacy & safety of eplontersen vs PBO in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN)
- The results showed a significant & clinical change from baseline for the co-1EPs of percent change in serum TTR concentration & mNIS+7. The therapy also met its 2EPs of change from baseline in Norfolk QoL-DN which showed an improvement in patient-reported QoL
- The therapy has a favorable safety & tolerability profile with no specific concerns & the results were consistent with the clinical profile across other LICA programs. Ionis & AstraZeneca are expected to file an NDA with the US FDA in 2022 & will seek regulatory approval for eplontersen in ATTRv-PN
Ref: PRNewswire | Image: Ionis
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