London, July 9, 2026, 15:01 BST
- Wainua did not hit the primary endpoint in its Phase 3 study for heart disease, which enrolled 1,432 patients.
- AstraZeneca slid roughly 9% in London. Ionis shares dropped too. ATTR-CM competitors moved higher.
- The setback puts new doubts on the trial setup and AstraZeneca’s $80 billion growth plan for 2030.
AstraZeneca stock fell Thursday after its Wainua drug, co-developed with Ionis Pharmaceuticals, did not meet the main goal in a late-stage study for a rare heart disease. The setback deals a blow to one of AstraZeneca’s high-profile programs outside cancer.
AstraZeneca is looking for new drugs to push revenue to $80 billion by 2030, up from $45.8 billion last year. The company aims to launch 20 new medicines by the end of the decade. The timing isn’t great.
AstraZeneca said its Phase 3 CARDIO-TTRansform trial showed Wainua, or eplontersen, did not cut cardiovascular deaths or recurrent heart events by a significant amount in adults with ATTR-CM over 140 weeks, compared to placebo. The company said Wainua was generally well tolerated. The disease, called transthyretin-mediated amyloid cardiomyopathy, happens when misfolded transthyretin proteins gather in the heart and reduce its ability to pump blood.
The study enrolled 1,432 patients at 130 sites in 20 countries. In both treatment arms, 57% of patients were on a stabilizer—a drug meant to keep transthyretin proteins intact. Another 24% started on a stabilizer during the trial.
Ionis said a subgroup of patients who were only taking eplontersen had a nominally significant hazard ratio of 0.71 for the main composite endpoint. That means fewer events in that set of patients. Still, the overall trial was negative, with no benefit seen for patients already on stabilizers when the study started.
Sharon Barr, EVP of biopharma R&D at AstraZeneca, said the results still add to “greater scientific understanding” of how to treat the disease. Ionis CEO Brett P. Monia called it a sign of a “rapidly evolving treatment landscape.” Mathew Maurer, who led the trial at Columbia, said the data brings “important clarity for the field.” AstraZeneca
AstraZeneca dropped roughly 9% in London trade after missing forecasts, per the Financial Times. Reuters said the stock looked set for its sharpest one-day percentage fall in nine years, wiping out £23.3 billion in market value at the low.
Ionis slid hard in U.S. trading. AstraZeneca dropped 8.55% and Ionis lost 19.17% after the companies reported Wainua failed to hit its main goal, The Wall Street Journal said.
BofA analyst Sachin Jain told Reuters the data was a “surprise,” citing positive precedent from Alnylam’s Amvuttra, which is a rival drug. Jefferies said the result could hit confidence in AstraZeneca’s trial design, but said it shouldn’t hurt the company’s long-term revenue target. Reuters
The news gave a lift to rivals. Pfizer’s Vyndaqel is a stabilizer for ATTR-CM. Amvuttra and Wainua from Alnylam lower transthyretin protein levels. BridgeBio’s Attruby is also approved for ATTR-CM. Shares of Alnylam and BridgeBio traded higher after the Wainua data.
The final numbers may still prove important. AstraZeneca and Ionis plan to keep digging into the full data set and will share results at the European Society of Cardiology Congress in August. The monotherapy hint will be a talking point, but with the main endpoint missed and no impact in patients already on stabilizers, the regulatory route isn’t clear.
Wainua is still cleared in over 20 countries for polyneuropathy tied to hereditary transthyretin-mediated amyloidosis, a nerve disorder. But missing in heart disease narrows the drug’s outlook, putting focus back on AstraZeneca’s next late-stage data.
