Clinical TrialJul 9, 2026, 06:22 AM
AstraZeneca's Wainua Phase III CARDIO-TTRansform Trial Fails Primary Endpoint
AI Summary
AstraZeneca announced that its CARDIO-TTRansform Phase III trial for Wainua (eplontersen) in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) did not meet its primary efficacy endpoint. The primary endpoint was a composite outcome of cardiovascular mortality and recurrent CV clinical events. While Wainua was generally well tolerated, a prespecified subgroup analysis showed a nominally significant benefit in patients on monotherapy, but no effect was observed in those on stabiliser therapy at baseline. AstraZeneca and Ionis will analyze the full data set and present it at the European Society of Cardiology Congress in August 2026.
Key Highlights
- CARDIO-TTRansform Phase III trial for Wainua (eplontersen) did not meet primary efficacy endpoint.
- Primary endpoint was composite of CV mortality and recurrent CV clinical events.
- Wainua was generally well tolerated with a safety profile consistent with previous results.
- Prespecified subgroup analysis showed nominally significant benefit for Wainua monotherapy.
- No treatment effect observed in patients on stabiliser therapy at baseline.
- Full data will be shared with the scientific community in August 2026.
- Trial enrolled 1,432 participants across 130 study sites in 20 countries.
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