Ionis Pharma Reports Positive 66-week Results Of Eplontersen Phase 3 Study In ATTRv-PN

(RTTNews) - Ionis Pharmaceuticals Inc. (IONS) announced positive results from the 66-week analysis of the Phase 3 NEURO-TTRansform study of Ionis and AstraZeneca's eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy or ATTRv-PN.

ATTRv-PN is a debilitating and potentially fatal disease that leads to peripheral nerve damage and motor disability.

Ionis noted that Eplontersen met co-primary endpoints demonstrating sustained reduction in TTR and benefits in neuropathy and quality of life through 66 weeks. TTR reductions were consistent with those reported at week 35.

Eplontersen continued to demonstrate a safety and tolerability profile consistent with that observed at 35 weeks.

As part of a global development and commercialization agreement, Ionis and AstraZeneca are seeking regulatory approval for eplontersen for the treatment of ATTRv-PN in the U.S. and plan to seek regulatory approval in Europe and other parts of the world.

Earlier this month, the U.S. Food and Drug Administration accepted a New Drug Application for eplontersen for the treatment of ATTRv-PN with a PDUFA action date of December 22, 2023. Eplontersen was granted Orphan Drug Designation in the U.S.

Eplontersen is currently being evaluated in the Phase 3 CARDIO-TTRansform study for transthyretin amyloid cardiomyopathy (ATTR-CM), a systemic, progressive and fatal condition that typically leads to progressive heart failure and often death within three to five years from disease onset.

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