Ionis Pharmaceuticals: How will CARDIO-TTRansform transform the ATTR-CM space?

Research evidence excerpt

Ionis Pharmaceuticals: How will CARDIO-TTRansform transform the ATTR-CM space?

Equity Research

U.S. Small & Mid Cap Biotechnology

25 June 2026

Ionis Pharmaceuticals

How will CARDIO-TTRansform

transform the ATTR-CM space?

We preview into CARDIO-TTRansform data we think could IONS OVERWEIGHT

POSITIVEcome earlier in 2H. We think the trial is likely to work, but the U.S.BiotechnologySmall & Mid Cap

key question is stat sig in the taf combo subgroup - see inside Price Target USD 115.00

for our analysis of the trial designs across the space, Price (24-Jun-26) USD 76.52 Potential Upside/Downside +50.3%

Source: Bloomberg, Barclays Researchbaselines, powering, and key supportive reasons/risks.

Ultimately we think there is a large market in ATTR-CM with room for many players. We U.S. Small & Mid Cap Biotechnology

preview our thoughts into the CARDIO-TTRansform Ph3 topline readout of IONS/AZN's Wainua Eliana Merle, CFA

+1 212 526 9445in ATTR-CM expected in 2H (we think based on 140 weeks from enrollment completion this

eliana.merle@barclays.com

could be early in 2H, potentially July/August - see more details in the report). We expect the trial

BCI, US

to work (and we think most investors do too) on the primary endpoint. The key debate is

whether Wainua can reach statistical significance on the secondary endpoint in the tafamidis Jasmine Fels, PhD

+1 212 526 0865combination subgroup. This would be key evidence to support the utility of the stabilizer/

jasmine.fels@barclays.com

silencer combination, and we think would be taken as a read to the classes broadly. Although

we think it's a longer shot, if we do see stat sig in the combo subgroup, we see upside for IONS

shares. We think investors are mixed on whether the taf subgroup hits (and we think IONS and Tejas Wein