How well does Wainua(Eplontersen) work?

WAINUA (eplontersen) is a transthyretin-directed antisense oligonucleotide that significantly reduces serum TTR protein levels, effectively treating the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults by demonstrating statistically significant improvements in neurological impairment and quality of life compared to placebo.

Therapeutic Efficacy of Eplontersen in hATTR Amyloidosis

Eplontersen functions as an antisense oligonucleotide-GalNAc conjugate that binds to TTR mRNA, causing degradation of both mutant and wild-type TTR mRNA. This mechanism results in substantial reduction of serum TTR protein levels and subsequent reduction of TTR protein deposits in tissues. 

In clinical studies, eplontersen demonstrated an 81% reduction in serum TTR from baseline by week 35, with consistent efficacy across various patient subgroups including those with different TTR variants.