Quick Take: Ph3 CARDIO-TTRansform Study Unexpectedly Disappoints

Research evidence excerpt

Quick Take: Ph3 CARDIO-TTRansform Study Unexpectedly Disappoints

Update

July 9, 2026 10:56 AM GMT

Morgan Stanley & Co. LLCMBiotechnology | North America Michael E Ulz

Equity Analyst

Quick Take: Ph3 CARDIO- Mike.Ulz@morganstanley.comAvraham Novick +1 212 761-4650

Research Associate

Avi.Novick@morganstanley.com +1 212 761-2231

TTRansform Study Rohit Bhasin

Rohit.Bhasin@morganstanley.com +1 212 761-0534

Unexpectedly Disappoints

Biotechnology

North America

Key Takeaways Industry View Attractive

Ph3 CARDIO-TTRansform study of eplontersen in TTR-CM did not meet its

primary endpoint, likely driven by high stabilizer use.

IONS: Expect shares to be under pressure given expectations for a positive study.

ALNY: Expect shares to react positively given removal of a key competitor.

Ph3 CARDIO-TTRansform study misses primary endpoint. IONS and partner AZN

(covered by Sarita Kapila; see her note here) announced (PR here) the Ph3 CARDIO-

TTRansform study of eplontersen in patients with TTR-CM did not meet its primary

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

CV clinical events up to Week 140 compared with placebo. We had a chance to catch

up with mgt. and the miss was attributed to the contemporary patient population

where the standard of care (tafamidis) performed better than expected and the

background use was high (>80%; 57% at baseline and 24% added during the study)

making it challenging of eplontersen to demonstrate a benefit as no treatment

effect was observed. However, in patients treated with monotherapy a nominal

statistically significant benefit (HR=0.71) compared to placebo was observed. Of

note, eplontersen resulted in robust TTR knockdown, while favorable benefits were