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Ionis Presents Positive Results From Phase 3 Study Of Eplontersen In Neuropathy Impairment

Ionis Pharmaceuticals Inc. (IONS) presented positive results from a planned 35-week interim analysis of the Phase 3 NEURO-TTRansform study of Ionis and AstraZeneca's eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The study results were presented at the International Symposium on Amyloidosis or ISA in Heidelberg, Germany.

In the study, eplontersen demonstrated a statistically significant and clinically meaningful change from baseline for co-primary and secondary endpoints at 35 weeks compared to the external placebo group. Eplontersen achieved an 81.2% reduction in the co-primary endpoint of serum transthyretin (TTR) concentration from baseline, demonstrating reduced TTR protein production. Eplontersen demonstrated a favorable safety and tolerability profile.

"Eplontersen showed clinically meaningful improvement in neuropathy impairment and quality of life measures relative to baseline..," said Teresa Coelho, a neurologist and neurophysiologist at Hospital Santo António, Centro Hospitalar Universitário do Porto, Portugal and an investigator for the NEURO-TTRansform study.

Ionis said that, based on the study results, the companies will seek regulatory approval for eplontersen for ATTRv-PN and plan to file a new drug application with the U.S. Food and Drug Administration this year.

Hereditary transthyretin amyloidosis (ATTRv) is a severe, progressive, and life-threatening disease caused by the abnormal formation of the TTR protein and aggregation of TTR amyloid deposits in various tissues and organs throughout the body, including in peripheral nerves, the heart and intestinal tract.

The progressive accumulation of TTR amyloid deposits in these organs often leads to intractable peripheral sensorimotor neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations.

Hereditary transthyretin amyloidosis causes significant morbidity and progressive decline in quality of life, severely impacting activities of daily living. The disease often progresses rapidly and can lead to premature death. The median survival is 4.7 years following diagnosis.

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