Ionis Highlighted P-III Study (NEURO-TTRansform) Results of Eplontersen for the Treatment of THereditary Transthyretin-Mediated Amyloid Polyneuropathy
- The P-III study evaluating eplontersen in patients with ATTRv-PN at 35, 66, and 85wk. In the 66wk. primary analysis, eplontersen showed an improvement across all co-1EPs & 2EPs incl. serum transthyretin (TTR) concentration with LS mean reduction of 82% vs 11%
- Improvement in neuropathy (47% vs 17%), and QoL (58% vs 20%), benefits on mNIS+7 and Norfolk QoL-DN at 35wks. further improved at 66wks. Eplontersen halted disease progression. The results were published in the JAMA
- Improvements in all 2EPs through 66wks. and showed a favorable safety and tolerability profile, rate of TEAEs was comparable & no AEs led to study drug discontinuation. In the end-of-treatment analysis, eplontersen continued to sustain improvements through 85wks.
Ref: PR Newswire | Image: Ionis
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