Transthyretin amyloid depletion therapy: lessons from the phase 2 trial of coramitug.

Abstract

This focused review contextualizes the results of the phase 2 randomized clinical trial of the monoclonal antibody coramitug (formerly PRX004 and NNC6019) in transthyretin amyloid cardiomyopathy (ATTR-CM). Coramitug, a humanized monoclonal antibody directed against misfolded transthyretin, represents a novel amyloid-depleting approach aimed at promoting phagocytic clearance of fibrillar deposits. In the phase 2 trial enrolling 105 patients with ATTR-CM, treatment with 60 mg/kg coramitug significantly reduced N-terminal pro-B-type natriuretic peptide levels at 52 weeks compared with placebo, though no change was observed in the co-primary endpoint of six-minute walk distance or secondary endpoints. Coramitug was generally well tolerated. These results support continued evaluation in a larger phase 3 trial to determine its clinical efficacy and effect on myocardial function.