Ionis Pharmacompletes enrollment for Phase 3 CARDIO-TTRansform study

Ionis Pharmaceuticals (IONS) announced the completion of enrollment in the Phase 3 CARDIO-TTRansform cardiovascular outcomes study of eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy, or ATTR-CM, a large and growing patient population at risk of life-threatening cardiovascular, or CV, events. The study enrolled more than 1,400 patients. As part of a global development and commercialization agreement, Ionis and AstraZeneca (AZN) are seeking regulatory approval for eplontersen for the treatment of transthyretin-mediated amyloid polyneuropathy, or ATTRv-PN, in the U.S. and plan to seek regulatory approval in Europe and other parts of the world. The FDA accepted the 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.