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

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. 

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