By Raechel Thankam Job
July 9 (Reuters) – AstraZeneca shares tumbled on Thursday after the company said its nerve disease drug Wainua, made in partnership with Ionis, failed a pivotal heart-disease trial and cast doubt on the company’s trial designs.
The setback dims what some analysts predicted could be a $2 billion peak-sales opportunity for the drug, and is another blow for AstraZeneca following a U.S. regulatory panel’s rejection of its breast cancer drug camizestrant on trial design grounds.
AstraZeneca, which is counting on up to 20 new medicine launches to help generate $80 billion in annual revenue by 2030, said Wainua did not meet the main goal of reducing cardiovascular deaths and recurring heart problems in a late-stage trial for patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).
The company’s shares fell nearly 10% to be the biggest loser on the FTSE 100 index. If losses as of 0822 GMT hold, it would be the stock’s worst performance since March 2020.
Shares of U.S.-listed partner Ionis Pharmaceuticals fell 13.8% in premarket trade while U.S. competitors Alnylam Pharmaceuticals and BridgeBio, which have approved drugs for ATTR-CM, climbed between 11% and 16%.
TRIAL DESIGN MASKS BENEFITS
AstraZeneca said the trial showed that adding Wainua to standard care did not provide a statistically significant benefit. Taking it as a standalone therapy without stabilizers showed “nominally significant” benefit, it said.
Analysts criticised the 140-week trial’s design where 57% of patients were already taking a stabilizer and a design flaw allowing a further 24% to add the stabilizer during the study, which made it difficult to assess any benefit from the drug.
Analysts at Jefferies said that could dent management credibility after executives expressed strong confidence in the trial’s prospects.
“(AstraZeneca) is meant to be able to have exceptionally good trial design ability,” Jefferies said, adding that the trial miss should not derail AstraZeneca’s long-term revenue target.
FUTURE APPROACHES
Wainua was being tested in 1,432 patients for the condition that lets protein build-up in the heart disrupt blood pumping and possibly cause heart failure. It affects 300,000 to 500,000 people globally, according to AstraZeneca.
The trial was keenly followed by analysts and investors as a key step in AstraZeneca accessing an underpenetrated ATTR market and building on growth beyond its dominant cancer medicines portfolio.
“Although the trial did not meet its primary objective, we believe the results support greater scientific understanding of treatment approaches,” said Sharon Barr, executive vice president of AstraZeneca’s biopharmaceuticals R&D.
Barclays analysts said that they do not expect AstraZeneca to fund a new monotherapy trial for Wainua, as that would be unlikely to get an approval this decade and would be too far behind a well-established rival drug from Alnylam.
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.
(Reporting by Raechel Thankam Job in Bengaluru; Editing by Janane Venkatraman and Elaine Hardcastle)
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