Analysts see opportunity for Alnylam’s RNAi therapy after AstraZeneca/Ionis ATTR-CM trial fails

AstraZeneca and its partner Ionis reported a disappointing Phase 3 result for the antisense drug Wainua in transthyretin amyloid cardiomyopathy (ATTR-CM). The unexpected outcome was a focal point of discussion at the recent European Society of Cardiology Congress.

Despite the setback, several analysts argue that the failure does not rule out the broader class of silencing therapies. They point to Alnylam’s approved RNAi drug Amvuttra, which continues to be used alongside standard stabilizer treatments, as evidence that the silencing approach can still have a role.

Alnylam presented new data supporting Amvuttra’s efficacy and safety, positioning the company to defend its market position as the field debates monotherapy versus a combination of silencer and stabilizer. The company’s next‑generation RNAi candidate, nucresiran, is also being watched as a potential future option.

The mixed signals underscore a shifting landscape in ATTR-CM treatment, where the failure of one platform may open space for others to demonstrate value in patients already receiving stabilizers.