Americas Healthcare: Biotechnology: ALNY/IONS: Surprise CARDIOTTRansform miss results in status quo landscape

Research evidence excerpt

Americas Healthcare: Biotechnology: ALNY/IONS: Surprise CARDIOTTRansform miss results in status quo landscape

Equity Research

9 July 2026 | 9:46AM EDT

Americas Healthcare: Biotechnology: ALNY/IONS: Surprise

CARDIO-TTRansform miss results in status quo landscape

AZN and IONS announced that the Phase 3 CARDIO-TTRansform study (n= 1,432) of Salveen Richter, CFA

+1(212)934-4204 |

antisense oligonucleotide Wainua in ATTR-cardiomyopathy (CM) missed its primary salveen.richter@gs.com

Goldman Sachs & Co. LLC

endpoint of the composite outcome of cardiovascular (CV) mortality and recurrent

Tommie Reerink, CFA

CV clinical events assessed up to week 140 - while there was a nominally significant +1(212)357-2470 |

hazard ratio of 0.71 in the monotherapy arm, there was no evidence of benefit in tommie.reerink@gs.comGoldman Sachs & Co. LLC

combination with stabilizer therapy (e.g., PFE’s tafamidis), and tafamidis-treated Elizabeth Webster, Ph.D.

patients performed better-than-expected in the milder population. +1(212)357-9925elizabeth.webster@gs.com|

The update is a negative surprise, as success on the primary endpoint was expected Matt Dellatorre, Ph.D.

per our investor discussions and the key debate centered around whether the +1(212)855-0830matt.dellatorre@gs.com|

stabilizer combination group - 57% of patients with an additional 24% drop-in rate - Goldman Sachs & Co. LLC

Mishrawould achieve significance. We await full results at the European Society of Shrunatra | +1(332)245-7673

Cardiology (ESC) Congress in August, but believe that for IONS, the focus is now on shrunatra.mishra@gs.com Goldman Sachs India SPL

severe hypertriglyceridemia (SHTG) competitor ARWR’s Ph3 data this quarter, where

Lydia Erdman

its profile will be evaluated in the context of IONS’ asset Tryngolza.