Ionis, AstraZeneca's eplontersen meets goals in phase 3 ATTRv-PN trial
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Eplontersen, a late-stage candidate from Ionis Pharmaceuticals (NASDAQ:IONS) and AstraZeneca (NASDAQ:AZN) for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), met all primary and secondary endpoints in a phase 3 trial at 66 weeks.
For one of the co-primary endpoints, those in the eplontersen group achieved a least squares (LS) mean reduction of 82% in serum TTR concentration from baseline, compared to an 11% reduction for those on placebo.
A second co-primary endpoint, the candidate halted disease progression based on the modified Neuropathy Impairment Score +7 (mNIS+7), leading to a 0.28 point LS mean increase compared to a 25.06 point increase in the placebo group.
For the third co-primary endpoint in the NEURO-TTRansform trial, 47% of treated patients demonstrated improvements in neuropathy at 66 weeks compared to baseline versus 17% in the placebo group.
ATTRv-PN is caused by the accumulation of misfolded mutated TTR protein in the peripheral nerves that leads to nerve damage.
Eplontersen is also in phase 3 in CARDIO-TTRansform study for transthyretin-mediated amyloid cardiomyopathy. 
Results from the NEURO-TTRansform trial will be presented Monday at American Academy of Neurology (AAN) 2023 Annual Meeting.
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