Eplontersen fails primary endpoint in CARDIO‑TTRansform trial for transthyretin amyloid cardiomyopathy

The CARDIO‑TTRansform trial, evaluating the investigational agent eplontersen in patients with transthyretin‑mediated amyloid cardiomyopathy (ATTR‑CM), failed to achieve its primary efficacy endpoint, investigators reported during a Hot Line session at the European Society of Cardiology (ESC) Congress 2026. The multicenter, randomized study enrolled individuals with confirmed ATTR‑CM and compared eplontersen to standard of care, with the primary endpoint defined as a statistically significant improvement in functional capacity and cardiac biomarkers over a prespecified follow‑up period.

Although the trial did not meet its main objective, secondary analyses will be examined to assess any trends in safety, tolerability, and exploratory efficacy measures. The findings will inform the ongoing development program for eplontersen and contribute to the broader understanding of therapeutic strategies for ATTR‑CM, a progressive disease with limited treatment options. Further updates are expected as the sponsor prepares a detailed data package for regulatory review.