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Aug. 28, 2026

CARDIO-TTRansform

eplontersen misses its primary endpoint in ATTR cardiomyopathy, with only a nominal signal in patients not on a stabiliser

Fontana M, Maurer MS, et al.; CARDIO-TTRansform investigators - New England Journal of Medicine

The largest ATTR-CM trial to date (1,432 patients, 130 centres) randomised eplontersen 45 mg every four weeks against placebo. The primary composite of cardiovascular death and recurrent cardiovascular events through week 140 was not reduced (rate ratio 0.89, 95% CI 0.73 to 1.09, p=0.277). In the prespecified 43 percent without a baseline stabiliser the signal was nominally positive; on top of tafamidis or acoramidis there was no added benefit. Serum TTR was suppressed throughout. Presented in Hot Line 1 by Mathew Maurer and published in the NEJM.