Drug in early trial appears to reduce harmful protein buildup in heart

An early-stage drug for a heart disease called ATTR-CM showed potential to reverse disease progression, opening up a new way of attacking the condition as existing drugs have been designed to only slow or stall the disease.

In a small Phase 1 trial, the drug, a monoclonal antibody named NI006, appeared to reduce harmful protein buildup in the heart based on imaging, according to the study, published Saturday in the New England Journal of Medicine.

The medication was initially developed by Neurimmune and has been licensed to Alexion, the rare disease unit of AstraZeneca. Given the results, researchers plan to advance the drug directly into a Phase 3 trial, said Martin Cowie, clinical vice president of cardiovascular and heart failure R&D at AstraZeneca.