Alnylam defends its heart failure silencers after AstraZeneca's ATTR-CM trial miss

AstraZeneca reported that its experimental drug Wainua failed to meet its primary endpoints in a study of transthyretin amyloid cardiomyopathy (ATTR-CM). The setback raised concerns about the therapeutic class of gene silencers that target the same disease pathway.

Some market analysts interpreted the miss as a signal that silencers may not add benefit for patients already receiving stabilizer drugs, suggesting stabilizers could be the preferred approach.

Alnylam responded by emphasizing that its approved silencer, Amvuttra, and its pipeline candidate nucresiran remain valuable, arguing that the AstraZeneca trial had a different design and that its own data continue to support the efficacy of its products.