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Getting the latest healthcare news for you
The biggest ATTR-CM trial yet delivers a surprising miss. The CARDIO-TTRansform trial — the largest ever in transthyretin amyloidosis with cardiomyopathy — found that adding eplontersen to standard care, including TTR stabilizers, did not significantly reduce cardiovascular death or recurrent CV events. However, patients not already on a stabilizer did show meaningful benefit from the drug.
The largest-ever trial in transthyretin amyloidosis with cardiomyopathy (ATTR-CM) has delivered a surprising result: eplontersen (Wainua), a once-monthly RNA-targeted silencer that dramatically lowers TTR levels, did not significantly reduce the risk of cardiovascular death or recurrent CV events when added to current standard-of-care therapy. Results from the CARDIO-TTRansform trial were presented at the European Society of Cardiology Congress and published simultaneously in the New England Journal of Medicine.
The key factor? Background therapy. More than half of the 1,432 trial participants were already on TTR stabilizers at enrollment, which experts believe blunted eplontersen's incremental impact. As monotherapy, eplontersen showed nominal significant effects on the primary endpoint — and patients not on a stabilizer at baseline saw a meaningful reduction in CV events.
Key Takeaways:
Why it matters: These findings challenge the assumption that combining TTR silencers and stabilizers offers additive benefit. Clinicians may need to rethink sequencing strategies — prioritizing eplontersen for stabilizer-naïve or early-stage patients rather than as an add-on therapy.
