Goldman Sachs: Americas Biotechnology — ALNY/IONS: Surprise CARDIO-TTRansform Miss Results in Status Quo Landscape
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AstraZeneca and Ionis Pharmaceuticals announced that the Phase 3 CARDIO-TTRansform trial evaluating Wainua in ATTR cardiomyopathy failed to reach its primary endpoint. The composite outcome of cardiovascular death and recurrent cardiovascular events did not show significant improvement through week 140. While a nominally significant hazard ratio of 0.71 was observed in the monotherapy subgroup, no treatment benefit was detected among the 57% of patients on baseline stabilizers, plus an additional 24% drop-in rate.
This outcome surprised the market, which generally expected the primary endpoint to succeed despite the central debate over the stabilizer combination cohort. Ionis noted that the tafamidis arm performed better than anticipated. Following the announcement, Alnylam shares rose as the competitive threat in ATTR-CM diminished.
Goldman Sachs expects the market to scrutinize detailed data at the ESC Congress in August 2026 to assess implications for future Phase 3 studies, including Alnylam's event-driven TRITON-CM trial (n~1,750). Success in TRITON-CM could support an approximate 2030 approval.
The broker maintains a Buy rating on Alnylam with a 12-month target of $550 and a Neutral on Ionis with a reduced target of $68, down from $75. For Ionis, investor focus shifts toward severe hypertriglyceridemia, with Arrowhead's competing Phase 3 data expected this quarter.
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