CARDIO‑TTRansform's Size & Design Boosts Confidence In Positive Outcome

Research evidence excerpt

CARDIO‑TTRansform's Size & Design Boosts Confidence In Positive Outcome

Biotechnology

Ionis Pharmaceuticals

TD SECURITIES (USA) LLC QUICK TAKE: COMPANY UPDATE

May 11, 2026

Price: $75.71 (05/8/2026) CARDIO-TTRansform's Size & Design Boosts

Price Target: $108.00 Confidence In Positive Outcome

BUY (1)

ESG SCORE: 58/100

Yaron Werber, M.D. THE TD COWEN INSIGHT

646 562 1415

CARDIO-TTRansform's size increases chance of positive outcome & powering is conservative,

yaron.werber@tdsecurities.com

likely based on improved SOC/background tx. 57% of patients (n=819) are on background

Steven Ionov, Ph.D. stabilizer vs ~260 in HELIOS-B & combo efficacy is tested as key secondary endpoint: unlimited

646 562 1478 drop-in may increase combo cohort size further. We like the risk/reward going into the H2:26

steven.ionov@tdsecurities.com

readout.

Jaena Han, Ph.D.

646 562 1428 Our Take: CARDIO-TTRansform's Conservative Powering Is Favorable – 57% Stabilizer Use &

jaena.han@tdsecurities.com Unlimited Drop-In Raise Chance Of Stat Sign Combo Signal

Sarah Cai, Ph.D. At ESC Heart Failure Congress on Saturday, and published tandemly in Circulation: Heart

646 562 1447

Failure, AstraZeneca/Ionis presented/published baseline characteristics and design of the

sarah.cai@tdsecurities.com

pivotal Ph3 CARDIO-TTRansform study of Wainua (eplontersen) in ATTR-CM. With 1,432

Key Data patients, the trial is the largest ATTR-CM outcomes study conducted to date and enrolled a

Symbol NASDAQ: IONS population broadly comparable to Amvuttra’s HELIOS-B, but with higher enrollment of sicker

Market Cap $12.5B patients, and allowing modern day stabilizer use, thereby increasing the real world relevance

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