STAT+: In autopsy of failed heart disease study, AstraZeneca raises broader questions about silencer drugs

Andrew covers the biopharma industry, scientific research, and public health across the continent. You can reach Andrew confidentially on Signal at drewqjoseph.71.AstraZeneca on Friday detailed the surprise failure of a closely watched heart disease clinical trial, laying out how its experimental drug offered no additional benefits for patients who were largely already on another type of medicine — the latest twist in a multi-billion dollar market. The study indicated that patients with a progressive condition known as ATTR-CM who received AstraZeneca’s drug — what’s called a silencer — saw no improvements if they were already taking another type of drug called a stabilizer, an effect so powerful that it tanked the whole Phase 3 study. Most participants were on a stabilizer at the outset of the trial. The trial’s miss has had implications not just for AstraZeneca and its development partner Ionis Pharmaceuticals, but for other companies that market ATTR-CM drugs, including Alnylam Pharmaceuticals, which sells another silencer, and BridgeBio, the maker of a new stabilizer. Analysts have said the study’s outcome bolstered the case that stabilizers, which are oral drugs, were perhaps superior to injectable silencers. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus daily coverage and analysis of the pharma industry — by subscribing to STAT+. Already have an account? Log in View All Plans To read the rest of this story subscribe to STAT+. Subscribe