Experimental heart drug misses trial goal but protects some participants

The injectable therapy eplontersen failed to show a significant benefit over a placebo at reducing cardiovascular events and related deaths in a Phase 3 clinical trial of adults with transthyretin amyloid cardiomyopathy (ATTR-CM).

While the trial, called CARDIO-TTRansform (NCT04136171), missed its primary goal in the overall patient group, the therapy was associated with a significantly lower risk of heart-related events and death among participants who were not already taking standard-of-care medications called TTR stabilizers.