Phase III trial miss for Wainua

The CARDIO-TTRansform Phase III trial for AstraZeneca (LSE: AZN) and Ionis Pharmaceuticals (Nasdaq: IONS), Wainua (eplontersen) in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) did not meet the primary efficacy endpoint of the composite outcome of cardiovascular (CV) mortality and recurrent CV clinical events up to 140 weeks compared with placebo.  Wainua was generally well tolerated, with a safety profile consistent with previous results.

In this contemporary patient population treated with standard of care, including a majority on a stabiliser, adding Wainua did not provide a statistically-significant benefit on the composite outcome of CV mortality and recurrent CV events. In a prespecified subgroup analysis of patients treated with Wainua monotherapy as compared to placebo, fewer primary composite events (CV mortality and recurrent CV events) were observed and this result was nominally significant. In patients who were on stabiliser therapy at baseline, no treatment effect was observed.