Policy & Regulation
Ionis Pharmaceuticals announces positive topline results from eplontersen Phase three NEURO-TTRansform study
27 March 2023 -

Ionis Pharmaceuticals, Inc. (Nasdaq:IONS), a United States-based biotechnology company, announced on Monday positive topline results from the 66-week evaluation of the Phase three NEURO-TTRansform study of Ionis and AstraZeneca's eplontersen in subjects with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).

The company said that at 66 weeks, patients treated with eplontersen continued to demonstrate a statistically significant and clinically meaningful change from baseline versus an external placebo group on the co-primary endpoints of modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression, and Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN). The study also met its third co-primary endpoint demonstrating a statistically significant reduction in serum TTR concentration versus an external placebo group. 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.

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.



Related Headlines