AstraZeneca announced that its CARDIO-TTRansform Phase III trial evaluating Wainua (eplontersen) in adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) did not meet its primary endpoint of reducing cardiovascular mortality and recurrent cardiovascular events compared with placebo. The study evaluated Wainua, an RNA-targeted gene silencing therapy developed with Ionis Pharmaceuticals, in patients receiving standard-of-care treatment, including transthyretin stabilizers. While the overall trial did not demonstrate a statistically significant benefit, a prespecified subgroup of patients receiving Wainua as a standalone therapy showed a nominally significant reduction in the primary composite outcome, and AstraZeneca and Ionis plan further analysis of the data.