AstraZeneca's Wainua fails primary endpoint in heart trial

AstraZeneca (NASDAQ: AZN) and Ionis announced that their Phase III CARDIO-TTRansform trial for Wainua (eplontersen) did not meet its primary efficacy endpoint in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).

The trial's primary endpoint was a composite outcome of cardiovascular mortality and recurrent cardiovascular clinical events measured over 140 weeks compared with placebo. The drug was generally well tolerated, with a safety profile consistent with previous results.

The trial enrolled patients receiving standard of care, with 57% of participants in each arm on a stabilizer treatment at baseline, and an additional 24% in each arm initiating a stabilizer during the trial. Adding Wainua to standard of care did not produce a statistically significant benefit on the composite outcome.

A prespecified subgroup analysis of patients treated with Wainua without a stabilizer showed fewer primary composite events compared to placebo, a result described as nominally significant. No treatment effect was observed in patients who were on stabilizer therapy at baseline.

Sharon Barr, Executive Vice President of BioPharmaceuticals R&D, said: "Although the trial did not meet its primary objective, we believe the results support greater scientific understanding of treatment approaches for the hundreds of thousands of patients worldwide suffering from this progressive and often fatal condition."

AstraZeneca and Ionis said they will analyze the full data set and present results at the European Society of Cardiology Congress in August 2026.

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