Eplontersen trial fails to meet primary endpoint in transthyretin-mediated amyloid cardiomyopathy

The CARDIO-TTRansform trial investigating eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) did not meet its primary efficacy endpoint, according to results presented at ESC Congress 2026. ATTR-CM, a progressive and fatal disease characterized by misfolded transthyretin (TTR) amyloid deposits in the heart muscle, affects an estimated 300,000 to 500,000 people worldwide.

Eplontersen, a once-monthly RNA-targeted silencer, is approved for the treatment of hereditary/variant TTR amyloid polyneuropathy. The trial, a double-blind Phase III study conducted at 130 centers in 20 countries, enrolled 1,432 patients with ATTR-CM who were receiving standard care. While eplontersen suppressed circulating serum TTR, the trial did not demonstrate a significant reduction in the primary endpoint of cardiovascular mortality and recurrent cardiovascular events in the overall population.

However, among patients not receiving stabilizer therapy at baseline, eplontersen monotherapy showed a treatment effect that reached nominal statistical significance.

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