Ionis Pharmaceuticals announces NEURO-TTRansform study met co-primary endpoints

Ionis Pharmaceuticals (IONS) announced topline results from the 66-week analysis of the Phase 3 NEURO-TTRansform study of Ionis and AstraZeneca’s (AZN) eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy, or ATTRv-PN, a debilitating and potentially fatal disease that leads to peripheral nerve damage and motor disability. 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, a measure of neuropathic disease progression, and Norfolk Quality of Life Questionnaire-Diabetic Neuropathy. 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. Data from both the 35 and 66-week analyses will be presented as an Emerging Science presentation at the American Academy of Neurology annual meeting in April. The initial results from the 35-week analysis were presented at the International Symposium on Amyloidosis meeting in September 2022. 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 FDA accepted a new drug application for eplontersen for the treatment of ATTRv-PN with a PDUFA action date of Dec. 22, 2023. Eplontersen was granted orphan drug designation in the U.S.