AstraZeneca shares tumbled on Thursday after the company said its nerve disease drug Wainua, made in partnership with U.S.-based Ionis, failed to meet the main goal of reducing cardiovascular deaths and recurring heart problems in a late-stage trial.
The setback dims what some analysts predicted could be a $2 billion peak-sales opportunity for Wainua, as the Anglo-Swedish drugmaker aims for $80 billion in annual revenue by 2030 by launching up to 20 new medicines.
The company's shares were down 9.5% by 0709 GMT and were the biggest losers on the FTSE 100 index
The trial was for patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a type of heart disease that lets protein build-up in the heart disrupt blood pumping and possibly cause heart failure. Adding Wainua to standard care did not provide a statistically significant benefit, the trial found.
The trial was seen by analysts as a key step for AstraZeneca in accessing an underpenetrated ATTR market as it seeks growth beyond its cancer medicines portfolio.
"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," said Sharon Barr, executive vice president of AstraZeneca's biopharmaceuticals R&D.
In patients who were already on stabilizer therapy when the trial started, representing 57% of participants, Wainua had no effect, though a subgroup taking the drug as a standalone therapy without stabilizers showed "nominally significant" benefit.
Wainua, which generated $212 million in product revenue for AstraZeneca in 2025, is already approved in more than 20 countries to treat patients with polyneuropathy, a life-shortening rare disease that causes nerve damage.
In the late-stage trial called CARDIO-TTRansform, Wainua was being tested in 1,432 patients compared to placebo over 140 weeks.
