AstraZeneca shares come under pressure after an unexpected stop from the clinical trial of one of the programs on which the market had high expectations. The failure of the advanced phase study on Wainua, a candidate for a rare heart disease, triggered a sharp reaction on the stock market and surprised analysts and investors, although, according to several experts, it does not change the group’s long-term financial targets.
THE STOCK MARKET CRASH
A heavy session for AstraZeneca on the London Stock Exchange, where the stock lost up to 9.9%, recording the biggest intraday drop since July 2017.
Since the beginning of the year, writes Bloomberg, the stock has fallen about 6%, while shares of the US-based Ionis Pharmaceuticals, partner in the drug’s development, dropped up to 15% in pre-market trading on Wall Street. The sales were triggered by the announcement of the failure to meet the primary endpoint of a phase 3 clinical trial on Wainua, intended for the treatment of a rare heart condition.
WHAT IS WAINUA AND WHICH DISEASE IS IT FOR
Wainua is a gene silencing therapy jointly developed by AstraZeneca and Ionis Pharmaceuticals. The drug works by suppressing the production of abnormal proteins in the liver that can accumulate in other tissues of the body.
It is already approved in over 20 countries for the treatment of polyneuropathy associated with hereditary transthyretin amyloidosis in adults, a rare progressive neurological disease. In the European Union, it is marketed under the name Wainzua.
The CARDIO-TTRansform study aimed to expand its use in transthyretin amyloid cardiomyopathy (ATTR-CM), a rare and progressive disease caused by the accumulation of misfolded proteins in the heart muscle, which can be fatal and, according to AstraZeneca’s estimates, affects between 300,000 and 500,000 people worldwide.
THE CLINICAL TRIAL RESULTS
According to what was announced by the pharmaceutical company, the phase 3 trial showed that Wainua “did not provide a statistically significant benefit” in reducing cardiovascular-related deaths when added to the existing standard therapy.
More specifically, the treatment failed to demonstrate a statistically significant reduction in deaths and recurrent cardiovascular emergencies over 140 weeks compared to placebo. The study evaluated the drug in combination with already available therapies to verify its ability to reduce recurrent cardiovascular events and mortality in patients with ATTR-CM.
THE IMPACT ON THE DRUG’S PROSPECTS
The trial failure significantly reduces the product’s commercial prospects. According to estimates reported by Jefferies, peak sales of Wainua are expected to be around 4 billion dollars, with about 2.5 billion dollars less in potential revenue compared to previous expectations.
The trial failure does not affect the drug’s already obtained approvals for its neurological indication, which last year, according to the Financial Times, generated revenues of 220 million dollars for AstraZeneca. Previously, analysts believed that a positive outcome could have added billions of dollars in sales, in a market for ATTR-CM therapies estimated at about 18 billion dollars by 2030.
ANALYSTS’ COMMENTS
Jefferies analysts believe that the trial failure does not compromise AstraZeneca’s strategic plan, which aims to reach revenues of 80 billion dollars by 2030, but warn that “the most significant issue is probably a certain loss of credibility,” recalling how management had expressed confidence in the study’s chances of success.
Experts from Bernstein, cited by the Sole 24 Ore, also described the negative results as “unexpected,” adding: “We just spoke with AstraZeneca, which is extremely disappointed and surprised by this news, and so are we.” According to the investment bank’s analysts, the company had recently expanded the trial to involve about 1,400 patients precisely to increase the likelihood of obtaining positive and statistically robust results on the primary endpoint.
