AstraZeneca (LSE:AZN) and Ionis have announced that the Phase III CARDIO-TTRansform study evaluating Wainua (eplontersen) in adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) did not achieve its primary objective. The treatment failed to deliver a statistically significant reduction in cardiovascular deaths and recurrent cardiovascular events when used alongside standard stabiliser therapy. Wainua was generally well tolerated, with a safety profile consistent with previous clinical findings, and the companies intend to present the complete dataset at the upcoming European Society of Cardiology Congress.
A predefined analysis identified a potential benefit among patients treated with Wainua as a standalone therapy, where the number of primary composite cardiovascular events was lower than in the placebo group, producing a nominally significant result. However, no meaningful treatment benefit was observed in participants who were already receiving stabiliser therapy when the trial began. The findings represent a setback for AstraZeneca’s plans to broaden Wainua’s use beyond its current approvals for hereditary transthyretin-mediated amyloid polyneuropathy, underlining the growing challenge of demonstrating additional clinical benefit as existing ATTR-CM therapies become more widely established.
Despite the disappointing trial outcome, AstraZeneca continues to be supported by solid business fundamentals, including strong profitability, healthy margins and attractive returns. The company has also maintained its financial guidance while delivering encouraging pipeline progress. These strengths are partially offset by softer technical market momentum, a moderate valuation and short-term pressure on cash flow and debt as investment spending and payment obligations increase.
More about AstraZeneca
AstraZeneca is a global biopharmaceutical company focused on researching, developing and commercialising prescription medicines across Oncology, Rare Disease and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism and Respiratory & Immunology. Headquartered in Cambridge, UK, the company markets innovative medicines in more than 125 countries, with its Cardiovascular, Renal & Metabolism business representing an important long-term growth platform centred on protecting organs and slowing disease progression.
The company’s Cardiovascular, Renal & Metabolism portfolio targets diseases affecting the heart, kidneys, liver and pancreas through therapies designed to address their underlying biological mechanisms. AstraZeneca aims to improve patient outcomes by enabling earlier diagnosis and delivering more effective treatments to millions of people around the world.
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