Ionis, AstraZeneca Pivotal Heart Disease Drug Flunks

Ionis and AstraZeneca reported that their Phase 3 CARDIO-TTRansform study of eplontersen in transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) failed to meet the primary efficacy endpoint. The topline trial failure is a major setback to the program’s clinical prospects and likely weighs on near-term expectations for both companies’ ATTR-CM pipeline.

Analysis

Ionis and AstraZeneca reported that their Phase 3 CARDIO-TTRansform study of eplontersen in transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) failed to meet the primary efficacy endpoint. The topline trial failure is a major setback to the program’s clinical prospects and likely weighs on near-term expectations for both companies’ ATTR-CM pipeline.