AstraZeneca NYSE:AZN, a UK drugmaker known for its cancer medicines, faced a rare clinical setback after Wainua, a gene-silencing heart drug it is developing with Ionis Pharmaceuticals, a California-based biotechnology company, failed to reduce cardiovascular events or heart-related deaths in a late-stage trial. The result surprised investors who had become used to positive readouts from AstraZeneca, especially as analysts noted that management communication before Thursday's data had appeared relatively confident. The setback sent AstraZeneca shares down as much as 9.9% in London trading, marking the company's biggest intraday decline since July 2017, while Ionis shares fell as much as 15% in premarket trading.
Jefferies analyst Michael Leuchten suggested the market reaction may reflect more than the loss of possible revenue from Wainua. He noted that AstraZeneca is generally expected to design clinical trials that are mostly watertight, meaning investors may view the failed readout as a credibility issue as well as a commercial disappointment. While Leuchten said the result does not threaten AstraZeneca's $80 billion sales target for 2030, the failure still appears to weaken confidence around one of the company's potential growth opportunities beyond its established cancer franchise.
Wainua is already approved for patients with conditions where proteins misfold and build up in the body, but the failed trial focused on transthyretin-mediated amyloid cardiomyopathy, a progressive and potentially fatal disease where misfolded proteins accumulate in the heart muscle. AstraZeneca estimates the condition affects as many as 500,000 people worldwide, while the broader therapy market is expected to reach $18 billion by 2030. Bloomberg Intelligence's John Murphy said AstraZeneca's ambition for more than $5 billion in Wainua sales now looks unlikely, as expansion into the larger cardiomyopathy indication appears challenging and the path to approval without additional studies may be difficult.