Wainua was generally well tolerated but did not beat placebo on the main efficacy measure in transthyretin amyloid cardiomyopathy, while analysts said the result could benefit BridgeBio’s Attruby and leave Alnylam’s Amvuttra as the only ATTR-CM gene silencer.
Ionis Pharmaceuticals and AstraZeneca said the Phase 3 CARDIO-TTRansform trial of eplontersen, marketed as Wainua, in transthyretin-mediated amyloid cardiomyopathy, or ATTR-CM, failed to meet its primary endpoint, as the drug did not significantly improve the composite of cardiovascular mortality and recurrent cardiovascular clinical events through Week 140 versus placebo. The companies said Wainua was generally well tolerated, with a safety profile consistent with previous results, and reported that secondary, imaging and biomarker measures favored eplontersen overall, with large and sustained reductions in transthyretin, or TTR. In a prespecified subgroup, patients receiving eplontersen monotherapy showed a nominally significant hazard ratio of 0.71 on the composite outcome, while no treatment effect was seen in patients already on stabilizer therapy at baseline. Following the result, Jefferies analysts said the setback could be a clearing event for BridgeBio Pharma’s Attruby and would leave Alnylam Pharmaceuticals’ Amvuttra as the only gene silencer for ATTR-CM; BridgeBio shares rose 15% and Alnylam rose 2.4% after paring earlier gains.