AstraZeneca and Ionis Pharmaceuticals said their drug Wainua failed to meet the primary endpoint of a Phase 3 trial in cardiovascular disease. The companies did not disclose detailed efficacy or safety data. Full results will be presented at an upcoming medical meeting.
AstraZeneca and Ionis Pharmaceuticals said their drug Wainua failed to meet the main goal of a Phase 3 trial testing it in cardiovascular disease, a setback for efforts to expand the drug beyond its approved use in a rare nerve condition. The companies did not disclose detailed data or say whether they would continue development in the indication.
AstraZeneca said Thursday that Wainua, developed in partnership with Ionis, did not reduce cardiovascular deaths and recurring heart problems compared with placebo in the late-stage study. "Wainua did not meet the primary endpoint," the company said, without providing specific efficacy or safety data. Full results will be presented at an upcoming medical meeting.
The trial tested Wainua (eplontersen), an antisense oligonucleotide — a type of genetic medicine that interferes with the production of a specific protein. The drug targets transthyretin, a protein that can accumulate in tissues and cause organ damage. Wainua is already approved for transthyretin-mediated amyloidosis polyneuropathy, a rare condition affecting the nerves, where it slows disease progression. The cardiovascular indication represented a potential expansion into a much larger patient population, as transthyretin buildup can also affect the heart muscle, leading to transthyretin cardiac amyloidosis.
Under the partnership, Ionis discovered the drug using its antisense technology platform and receives royalties on sales, while AstraZeneca holds commercialization rights outside the US. The failed trial removes a potential growth avenue for the Wainua franchise beyond its approved niche in ATTR polyneuropathy. Ionis, which relies on its pipeline for valuation as a pure-play biotechnology company with limited commercial revenue, faces greater downside risk from the setback. AstraZeneca, which generated more than $50 billion in total revenue last year, may redirect resources to other pipeline programs. Alnylam Pharmaceuticals, whose rival RNA-interference drug Amvuttra competes with Wainua in ATTR amyloidosis, could benefit from the development. Investors will watch for whether the companies pursue a narrower patient population or discontinue the cardiovascular program entirely.
This article is for informational purposes only and does not constitute investment advice.
