New heart drug from AstraZeneca and Ionis falls short in clinical trial
Eplontersen, a once-monthly medication from AstraZeneca and Ionis Pharmaceuticals, failed to meet a primary endpoint of a randomized trial exploring its ability to treat transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). CARDIO-TTRansform included data from nearly 1,500 patients randomized to receive eplontersen or a placebo every four weeks. The primary endpoint was a composite of cardiovascular mortality and recurrent cardiovascular clinical events after 140 weeks of treatment.
AstraZeneca and Ionis Pharmaceuticals are jointly developing and commercializing eplontersen in the United States to be sold under the brand name Wainua. The two companies have emphasized that the drug was “generally well tolerated, with a safety profile consistent with previous results.” Also, there was evidence that the drug may help select ATTR-CM patients who are not already on any sort of stabilizer therapy. However, the drug’s overall clinical impact was not significant.
The full results of CARDIO-TTRansform are expected to be shared with the public at the European Society of Cardiology Congress meeting in August 2026. For now, AstraZeneca and Ionis Pharmaceuticals are reviewing the data to gain a better understanding of what they mean for the companies going forward.
“The CARDIO-TTRansform trial was designed to examine the role of Wainua, a gene silencer treatment, on top of today’s standard of care in reducing recurring cardiovascular events and mortality,” Sharon Barr, executive vice president of biopharmaceuticals R&D for AstraZeneca, said in a statement. “Although the trial did not meet its primary objective, we believe the results support greater scientific understanding of treatment approaches for the hundreds of thousands of patients worldwide suffering from this progressive and often fatal condition.”
“Although we are disappointed that the study did not meet the primary endpoint, these results have the potential to guide the treatment landscape for ATTR-CM and contribute to advancing future care for patients,” added Brett P. Monia, PhD, CEO of Ionis Pharmaceuticals, in a separate statement. “We extend our sincere gratitude to the patients, families and investigators whose participation helped progress the science of ATTR-CM treatment.”
Michael has more than 19 years of experience as a professional writer and editor. He has written at length about cardiology, radiology, artificial intelligence and other key healthcare topics.