ATTR-CM treatment options narrowed after AstraZeneca and Ionis revealed their gene-silencing drug Wainua (eplontersen) failed a landmark 1,432-patient Phase 3 trial — finding no benefit over placebo when most patients were already on a TTR stabilizer, leaving Alnylam's Amvuttra as the only approved RNA-targeting therapy for the fatal heart disease.
Gene-Silencing Drug Wainua Fails Landmark ATTR-CM Trial in AstraZeneca Setback
