Eplontersen for Transthyretin Amyloid Cardiomyopathy

Heart Failure · Boston University

Primary results appeared to be influenced by baseline stabiliser use with no clear benefit in patients receiving background stabiliser therapy, while fewer primary endpoint events occurred with eplontersen in patients not receiving a stabiliser.

Heart Failure · Boston University

Over the last decade, multiple advances and increased awareness of ATTR-CM have driven earlier diagnosis, improved management and better outcomes for patients. These data from the largest enrolled study of a contemporary ATTR-CM patient population provide important clarity for the field that will help inform future treatment decisions in ATTR-CM.

#ESCCongress 2026 Round Up: 18 articles were simultaneously published in NEJM and presented at this year’s @escardio Congress in Munich. Original Article: Eplontersen for Transthyretin Amyloid Cardiomyopathy (phase 3 CARDIO-TTRansform trial)

Heart Failure · Boston University

In CARDIO-TTRansform, the largest ATTR-CM trial to date, eplontersen achieved reductions in circulating serum TTR, although the trial did not demonstrate a significant reduction in the primary endpoint in the overall population. Baseline stabilizer use appeared to influence the primary results. No additional benefit was observed in patients receiving background stabilizer therapy, while fewer primary endpoint events were observed with eplontersen in patients not receiving a stabilizer. These findings will inform clinical practice and the evaluation of TTR-lowering treatment strategies in ATTR-CM.

🔬 CARDIO-TTRansform results presented by Dr @dr_pavia García-Pavía at #ESCCongress2026 highlight the importance of treatment context in ATTR-CM. Eplontersen showed benefit in patients without baseline TTR stabiliser therapy, but not in those already receiving it. Published in

Can silencers add benefit in ATTR-CM? Detailed data from Phase III CARDIO-TTRansform for eplontersen from AZ, Ionis raise more questions

IONS: 2 readouts today $IONS is down 2.75% to $60.71 today (-$1.72 vs. prior close of $62.43), according to BPIQ market data. • Eplontersen, Phase 3 CARDIO-TTRansform: The detailed ATTR-CM dataset confirmed the trial missed its overall primary endpoint. A nominally positive

@NEJM @MKIttlesonMD The topline miss in CARDIO-TTRansform isn’t “silencers don’t work.” It’s that adding eplontersen on top of already-high stabilizer use didn’t move the composite needle for the whole population. The signal in stabilizer-naïve and earlier-stage patients is the part that actually

This result makes it hard for us to see a path towards filing/approval.

🚨 HOT LINE TAKEAWAY | CARDIO-TTRansform A neutral trial, but not the end of the story. Dr Margot Davis explains the full ESC 2026 CARDIO-TTRansform results and what they reveal about: ✅ Eplontersen as monotherapy ✅ The role of background tafamidis therapy ✅ Signals

The CARDIO-TTRansform trial was designed to examine the role of Wainua, a gene silencer treatment, on top of today's standard of care in reducing recurring cardiovascular events and mortality. Although the trial did not meet its primary objective, we believe the results support greater scientific understanding of treatment approaches for the hundreds of thousands of patients worldwide suffering from this progressive and often fatal condition.

Results from cardioTTRansform. Eplontersen (ASO) did not improve survival in Amyloidosis (85%WT, 15%) already on tafamidis. #ESC Main Arena, Múnich

Cross-Cutting Cardiology · Ionis Pharmaceuticals (United States)

We believe these findings reflect the rapidly evolving treatment landscape, in which contemporary ATTR-CM patients are widely treated with stabilizers. Although we are disappointed that the study did not meet the primary endpoint, these results have the potential to guide the treatment landscape for ATTR-CM and contribute to advancing future care for patients.

The negative outcome raises the possibility that the incremental benefit of combining a silencer with a [stabilizer] is smaller than previously assumed as seen in HELIOS-B, rather than the study having been designed poorly.

Heart Failure · Brigham and Women's Hospital

The first Hot Line session kicking off at #ESC2026 A great line up of LBCTs (biased opinion!) First up is Cardio-TTRansform with Mat Maurer #CardioTwitter

CARDIO-TTRansform findings show no significant difference in CV mortality and recurrent events with eplontersen vs. placebo among patients w/ ATTR-CM. Primary results also appeared to be influenced by baseline stabiliser, said researchers. Read more from #ESCCongress: #ACCIntl

CARDIO-TTRansform results presented at #ESCCongres

ACC Anywhere TV Live from Munich! 📺 ACC Immediate Past President Dr. Christopher Kramer discusses some of the trials presented during Hot Line 1 at #ESCCongress 2026. In particular, Kramer was excited by the results of ACACIA-HCM, CMR Guide & CARDIO-TTransform. Find more coverage at: #ACCAnywhere #Cardiology #ACCIntl

Cedars-Sinai Medical Center

Even if you never care for patients with cardiac amyloidosis, the CARDIO-TTRansform trial offers lessons in looking beyond soundbite of a clinical trial to understand what the findings really mean for patient care. @NEJM

In a new NEJM Voices blog post, @MKittlesonMD discusses the CARDIO-TTRansform trial results, which offer a lesson in looking beyond topline results to understand what the findings really mean for patient care. Learn more and sign up for email alerts:

Heart Failure · Brigham and Women's Hospital

Mat Maurer presentating the results of the CARDIO-TTRansform trial. We are so thankful to our/all patients who enrolled in this trial. Lots of learnings here. Lots more to do. Simultaneously published in @NEJM #CardioTwitter #ESC2026 #ESCCongress2026

⚡️CARDIO-TTRansform = largest trial in patients with ATTR-CM to date 📌 Eplontersen + stabilizer therapy did not confer added CV benefit 📋 Baseline standard care may have influenced results More ⬇️ #ESCCongress #ESC365 @escardio

CARDIO-TTRansform at #ESCCongress: more TTR suppression is not necessarily better. The first trial of HOT LINE 1 tested eplontersen, an antisense oligonucleotide that suppresses hepatic TTR production, in patients with transthyretin amyloid cardiomyopathy. The biological effect

General · Cedars-Sinai Medical Center

🫀 #CARDIOTTRansform at #ESCCongress Can adding TTR silencing with eplontersen improve outcomes in patients with #ATTRCM in the era of contemporary therapy? 1,432 patients,90%Men Eplontersen vs placebo 57% on a TTR stabilizer at baseline ⏱️ 140 weeks: sustained TTR ⤵️ Primary

ATTR-CM Study (Cardio-TTransform): TTR stabilizer use hazard ratio: NO 0.71 YES 1.14 interaction p-value = ~0.02 This means silencers won't work as well when patients have been on stabilizers. Bearish for Nex-Z and NTLA (1/3)

$ALNY -5% this morning Sellside comments re: the 1.14 event rate ratio from the stabilizer (combo) arm of the CARDIO-TTRansform study: Paul Matteis, Stifel "While minimal benefit was largely expected for the eplontersen/tafamidis combo arm in CARDIO-TTRansform, it's an