AstraZeneca said its heart disease drug Wainua, developed with Ionis Pharmaceuticals, failed to outperform placebo in a pivotal trial in transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM). The result missed endpoints tied to cardiovascular death and clinical events. The setback increases pressure on AstraZeneca’s cardiovascular pipeline and shifts competitive expectations for RNA-targeting and other therapies in ATTR-CM. Provided coverage also notes immediate market reaction, with shares down in premarket trading. For the field, the failure reinforces how difficult it remains to demonstrate incremental clinical benefit in ATTR-CM, even as biologic and antisense approaches aim to slow disease progression.
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