Ionis’ ATTR-CM miss: Were overlapping mechanisms or the modality to blame?

A Phase III miss for one of the most highly anticipated clinical trials of the year doesn’t just set Ionis and partner AstraZeneca back billions of dollars in peak revenue; it also highlights a vulnerability of one of the modalities that was expected to transform the management of cardiovascular disease.

Ionis Pharmaceuticals Inc. (NASDAQ:IONS) announced Thursday that Wainua eplontersen failed to improve cardiovascular outcomes in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) — a major market expansion opportunity for the antisense therapy that’s already approved for the less common ATTR with polyneuropathy...