Eplontersen Misses Primary Endpoint in Phase 3 ATTR-CM Trial

On July 9, 2026, AstraZeneca and Ionis reported that the phase 3 CARDIO-TTRansform trial of eplontersen (Wainua) did not meet its primary efficacy endpoint in adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).1

“The CARDIO-TTRansform trial was designed to examine the role of Wainua, a gene silencer treatment, on top of today’s standard of care in reducing recurring cardiovascular events and mortality,” Sharon Barr, executive vice president of BioPharmaceuticals R&D, said in a statement. “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.”