Following the presentation of detailed Phase III data last week, Alnylam Pharmaceuticals Inc.’s Amvuttra seems likely to become the first gene silencer approved for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). But perhaps not the last: Ionis Pharmaceuticals, Inc. and AstraZeneca PLC are awaiting pivotal data on Wainua (eplontersen) in the disease, and their decision to enroll huge numbers of patients could give their product an edge.
Key Takeaways
- In 2026 the Phase III trial of AstraZeneca and Ionis’s Wainua in ATTR-CM will read out, giving an idea of whether the product is better than Alnylam’s similar product Amvuttra.
- The study is twice the size of Amvuttra’s, which might allow the partners to show a bigger treatment effect, particularly in subgroups
“Ideally, we’ll establish compelling evidence for the silencer class to be in first line, and eplontersen within that as the preferred silencer,” Mina Makar, senior vice president of AstraZeneca’s
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