Actions of AstraZeneca fell as much as 9% after a late-stage clinical trial for a heart disease drug failed to meet its goal.
The Wainua drug fell short of its primary goal of reducing deaths and recurrent cardiac emergencies over 140 weeks compared to a placebo when adding the drug to a patient's current treatment plan, the British manufacturer said in a news release Thursday morning.
The treatment was for a rare, life-threatening heart condition called transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).
Jefferies analysts said the result did not jeopardize the company's $80 billion sales target by 2030, but noted that AstraZeneca “had been very confident in the primary endpoint and the ability to achieve combination use.”
“The most important issue is probably some degree of loss of credibility, as management is very confident in the trial's ability to meet the primary endpoint, as well as its ability to show utility beyond background therapy,” the analysts added.
AstraZeneca confirmed that its existing license for Wainua was not affected by the results of these trials. The drug is already approved to treat conditions in which misfolded proteins build up and cause nerve damage. It is sold in Europe as Wainzua.
This study looked at a specific type of condition in which misfolded proteins build up in the heart muscle, stiffening it and making it difficult to pump blood and eventually leading to heart failure. It is estimated that around half a million people live with this condition.
AstraZeneca shares listed in London over the last 12 months.
The stock last fell 8.8% in London, on track for its worst day since March 2020, at the start of the Covid-19 outbreak. Shares listed on the New York Stock Exchange fell 8% in premarket trading.
Actions of Ionis Pharmaceuticalswhich Wainua is co-developing in the US, fell 12.5% in pre-market trading.
In the study cohort, most patients were already taking a stabilizer that prevents the protein from misfolding in the first place. Because patients were already receiving treatment for this, adding Wainua, the so-called gene silencer, on top of standard care showed no significant additional benefit for the group as a whole.
For patients not taking a stabilizer at the start of the study, Wainua showed a “nominally significant” risk reduction of deaths and cardiac events compared with placebo, AstraZeneca said.
Still, Citi analysts said it was unlikely AstraZeneca would be able to seek additional approvals for Wainua given the primary endpoint failure, as the Nasdaq-listed exchange Alnylam Amvuttra There is already a treatment for ATTR-CM on the market.
Alnylam Amvuttra shares rose 16% in pre-market trading.
“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 around the world suffering from this progressive and often fatal disease,” said AstraZeneca executive vice president of biopharmaceuticals R&D Sharon Barr.
Full data will be presented at the European Society of Cardiology in August.
