Actions of AstraZeneca fell as much as 9% after a late-stage clinical trial of a heart disease drug failed to meet its goal.
The medicine, Wainua, did does not achieve its main objective to reduce deaths and recurrent cardiac emergencies over 140 weeks compared to a placebo by adding the drug to a patient’s current treatment plan, the British pharmaceutical maker said in a press release Thursday morning.
The treatment was for a rare and life-threatening heart condition called transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).
Jefferies analysts said the result did not undermine the company’s $80 billion sales target by 2030, but noted that AstraZeneca “had been very confident about the primary endpoint and its ability to achieve combined use.”
“The biggest problem is probably some degree of loss of credibility, with management very confident in the trial’s ability to meet the primary endpoint as well as its ability to demonstrate utility in addition to background treatment,” the analysts added.
AstraZeneca has confirmed that its existing license for Wainua is not affected by the results of these trials. The drug is already approved to treat conditions in which misfolded proteins build up, causing nerve damage. It is sold in Europe under the name Wainzua.
This study looked at a specific type of disease in which misfolded proteins build up in the heart muscle, stiffening it and making it difficult to pump blood, ultimately leading to heart failure. It is estimated that around half a million people are living with this disease.
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AstraZeneca shares listed in London for the last 12 months.
The stock last fell 8.8% in London, on course for its worst day since March 2020, at the start of the Covid-19 outbreak. Shares listed on the NYSE were down 8% in premarket trading.
Actions of Ionis Pharmaceuticalswhich co-develops Wainua in the United States, fell 12.5% in premarket trading.
In the study cohort, the majority of patients were already taking a stabilizer that prevents the protein from misfolding. Since the patients were already receiving treatment for this, adding Wainua – a so-called genetic silencer – on top of standard care did not provide significant additional benefit for the group as a whole.
For patients not taking a stabilizer at baseline, Wainua showed a “nominally significant” reduction in the risk of death and cardiac events compared to placebo, AstraZeneca said.
Despite this, Citi analysts said it was unlikely AstraZeneca would be able to seek additional approvals for Wainua given the failure of the primary endpoint, as reported by the Nasdaq-listed company. Alnylam Amvuttra already has a treatment for ATTR-CM on the market.
Shares of Alnylam Amvuttra rose 16% in premarket trading.
“Although the trial did not meet its primary endpoint, we believe the results support a better scientific understanding of therapeutic approaches for the hundreds of thousands of patients worldwide suffering from this progressive and often fatal disease,” said Sharon Barr, AstraZeneca’s executive vice president of biopharmaceutical research and development.
The full data will be presented to the European Society of Cardiology in August.
