
AstraZeneca's Wainua fails to reduce cardiovascular deaths in late-stage trial, shares fall 9%
AstraZeneca and Ionis Pharmaceuticals' gene silencer Wainua did not meet its primary objective in a phase 3 trial for transthyretin amyloid cardiomyopathy, a rare heart disease, the company said on Thursday. Shares of the UK's largest pharmaceutical group fell as much as 9% in early London trading.
Trial results
AstraZeneca announced on Thursday that the phase 3 trial of Wainua in transthyretin amyloid cardiomyopathy (ATTR-CM) did not meet its primary endpoint of reducing cardiovascular-related deaths. The company said the gene silencer, developed in partnership with Ionis Pharmaceuticals, "did not provide a statistically significant benefit" in the study.
The phase 3 trial found that Wainua did not provide a statistically significant benefit in reducing cardiovascular-related deaths.
ATTR-CM is a rare and progressive heart condition caused by misfolded transthyretin proteins building up in the heart muscle, leading to heart failure.
Market reaction
Shares of AstraZeneca fell 9% in early trading on the London Stock Exchange following the announcement. The decline reflects investor disappointment, as Wainua was seen as a potential treatment for a condition with limited options.
Drug background
Wainua is already approved in several markets for other conditions involving misfolded proteins. The drug works by silencing the gene that produces transthyretin, reducing the accumulation of toxic protein deposits. The partnership between AstraZeneca and Ionis combined the former's commercial reach with Ionis's antisense technology.

