Eplontersen Phase 3 ATTR-CM Trial Falls Short of Main Endpoint but Shows Benefit in Certain Patients
Results from the Phase 3 CARDIO-TTRansform study found that eplontersen did not significantly reduce the combined risk of cardiovascular events and cardiovascular-related deaths compared with placebo in the overall population of patients with transthyretin amyloid cardiomyopathy (ATTR-CM). As a result, the trial did not achieve its primary endpoint.
However, a closer look at the data revealed that patients who were not taking transthyretin (TTR) stabilizer therapies experienced a meaningful reduction in the risk of heart-related complications and death when treated with eplontersen. Researchers believe the widespread use of approved TTR stabilizers among study participants may have limited the ability to detect additional benefits from eplontersen in the broader trial population.