Ionis, AstraZeneca Report Amyloid Cardiomyopathy Treatment Failed to Meet Primary Endpoint in Phase 3
Ionis Pharmaceuticals (IONS) and partner AstraZeneca (AZN) said a phase 3 trial for eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy, or ATTR-CM, failed to meet the primary efficacy endpoint. The endpoint was a composite outcome of cardiovascular mortality and recurrent cardiovascular clinical events up to week 140 compared with placebo, according to a statement early Thursday. "In this contemporary patient population treated with standard of care, including a majority on a stabilizer, adding eplontersen did not provide a statistically significant benefit," the statement said. Ionis and AstraZeneca plan to continue analyzing the full data and share results at the European Society of Cardiology Congress in August 2026. ATTR-CM is a fatal disease that predominantly affects the heart and is an underrecognized cause of heart failure.
Ionis Pharmaceuticals (IONS) and partner AstraZeneca (AZN) said a phase 3 trial for eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy, or ATTR-CM, failed to meet the primary efficacy endpoint.
The endpoint was a composite outcome of cardiovascular mortality and recurrent cardiovascular clinical events up to week 140 compared with placebo, according to a statement early Thursday. "In this contemporary patient population treated with standard of care, including a majority on a stabilizer, adding eplontersen did not provide a statistically significant benefit," the statement said.
Ionis and AstraZeneca plan to continue analyzing the full data and share results at the European Society of Cardiology Congress in August 2026.
ATTR-CM is a fatal disease that predominantly affects the heart and is an underrecognized cause of heart failure.