‘Worse than expected’ Wainua data muddies water for TTR silencers
AstraZeneca and Ionis' Wainua failed a Phase 3 trial for ATTR-CM, with a hazard ratio of 1.14, worse than expected. Analysts question its approval path, but see potential in monotherapy. Alnylam's Amvuttra showed better results in a similar trial. Stifel and Oppenheimer analysts commented on the data.
How this was made

The 30-second read
Why it matters
The trial failure undermines the commercial outlook for both AstraZeneca and Ionis, while offering a comparative advantage to Alnylam's approved therapy.
Market read
Negative trial data likely triggers sell‑offs in AZN and IONS, with modest sector ripple effects.
What to watch
Potential for the data to spur Alnylam's Amvuttra demand and shift market focus.
Background
The article reports fresh Phase 3 data for Wainua (eplontersen) presented at the ESC meeting, highlighting a negative hazard ratio versus standard of care.
Ticker impact
AstraZeneca's Phase 3 Wainua trial failed with a hazard ratio of 1.14, reducing approval prospects.
Downward pressure over the next few days.
Phase 3 failure for a key cardiovascular asset removes a potential revenue stream and raises R&D risk.
Ionis Pharmaceuticals co‑developed Wainua, which missed its primary endpoint in the ATTR‑CM study.
Sharp short‑term decline.
Ionis loses a major partnership and future sales opportunity from the failed antisense therapy.
Market effects
ATTR‑CM therapeutic space faces heightened scrutiny; competitors may benefit.
European biotech indices could see modest pullback.
Limited to biotech sector; broader markets largely unaffected.
Counterpoint
Some investors may view the partial monotherapy benefit as a catalyst for a pivot to a different indication.
Key entities
- companyAstraZeneca
Co‑developer of Wainua, a TTR silencer.
- companyIonis Pharmaceuticals
Partner in Wainua development.
- companyAlnylam Pharmaceuticals
Maker of Amvuttra, referenced as a comparator.



