ESC 2026: full Wainua data cast doubt on silencer-plus-stabilizer approach

Full results from CARDIO-TTRansform, presented at the European Society of Cardiology congress in Munich in August, have raised doubts about adding a gene silencer to stabilizer therapy for ATTR-CM.

The trial recorded 381 primary endpoint events in the eplontersen arm and 392 on placebo, a rate ratio of 0.89 that was not statistically significant. By the end of the study just over 80% of patients were on a stabilizer.

AstraZeneca's Mina Makar concluded that adding a silencing mechanism to a stabilizer did not provide additional benefit, Fierce Pharma reported. Jefferies analysts called the July result an outright failure. William Blair said the data could have wider implications for both stabilizers and silencers, including Alnylam's Amvuttra.

STAT reported that the post-mortem raised broader questions about the silencer class, and that analysts saw the result as strengthening the case for oral stabilizers.

Coverage of the congress by EU Reporter framed the market around whether silencers can meaningfully improve on established stabilizers.