Shots: 

•  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