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AstraZeneca's Phase III CARDIO-TTRansform trial for Wainua (eplontersen) failed to meet its primary efficacy endpoint
AZNCFAstrazeneca PLC
AstraZeneca's Phase III CARDIO-TTRansform trial for Wainua (eplontersen) failed to meet its primary efficacy endpoint. The study evaluated the drug in 1,432 adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) to determine if it reduced the composite outcome of cardiovascular mortality and recurrent cardiovascular clinical events compared to a placebo. While a prespecified subgroup analysis of patients on Wainua monotherapy showed nominally significant results, no treatment effect was observed in patients receiving stabilizer therapy at baseline. The drug was generally well tolerated with a safety profile consistent with previous findings. AstraZeneca and Ionis will analyze the full dataset for presentation at the August 2026 European Society of Cardiology Congress. AstraZeneca is a global biopharmaceutical company that discovers, develops, and commercializes prescription medicines.