Study summary

A second TTR-silencing drug missed its main goal in a large heart trial

CARDIO-TTRansform trial · Phase 3 · Announced July 2026; presented at ESC 2026 and published in NEJM

What was tested

Eplontersen, a monthly antisense injection that lowers transthyretin, against placebo, for ATTR cardiomyopathy, most on background stabilizers.

What they found

• No significant difference in cardiovascular death and recurrent heart events through 140 weeks.
• People already on a stabilizer saw no added benefit; a monotherapy subgroup showed a nominal benefit.
• TTR levels fell substantially and safety was favorable.

What it means

Raises the question of whether silencers add much on top of stabilizers, and who should get which.