12 May, 2026 New Analyses from Vutrisiran Program Presented at Heart Failure 2026

Analyses presented at Heart Failure 2026 reinforce the consistent clinical profile of vutrisiran in patients with the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM), including across clinically complex populations with a high disease burden and in the context of concomitant therapies. Additional data further characterize transthyretin (TTR) knockdown and support its relevance in real-world clinical practice.

A pooled safety analysis evaluating vitamin A-related outcomes across clinical trial and real-world datasets shows no evidence of clinically meaningful safety concerns associated with TTR reduction. The design of DemonsTTRate, a global, long-term observational study evaluating real-world use of vutrisiran in ATTR-CM, was also presented.

Abovich, et al. “Influence of Disease-Modifying Therapy on the Effectiveness of Vutrisiran in Transthyretin Cardiac Amyloidosis”

Birkhoelzer, et al. “Comorbidity Burden in Transthyretin Amyloidosis With Cardiomyopathy: Insights From the HELIOS-B Trial”

Mansell, et al. “Consistent Efficacy of Vutrisiran Across Sexes in ATTR-CM, HELIOS-B Trial – Prespecified Sex Analysis”

Sheikh, et al. “Influence of Vutrisiran on Systolic Blood Pressure in ATTR-CM: Insights From HELIOS-B”

Wang, et al. “Effect of Vutrisiran in Patients with and without Atrial Fibrillation or Flutter: Analysis from HELIOS-B”

Algalarrondo, et al. “Vutrisiran-Mediated Knockdown of Transthyretin in Patients with Transthyretin Amyloidosis”

Maurer, et al. “Treatment with Transthyretin-Lowering RNA Interference Therapeutics is Not Associated with Ocular or Other Clinical Events Due to Vitamin A Reduction: Pooled Analysis of Vutrisiran and Patisiran Data”

Garcia-Pavia, et al. “Design and Rationale of DemonsTTRate: A Global, Long-Term Observational Study to Evaluate Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy”

20 Apr, 2026 TRITON-PN Phase 3 Study Design and Rationale Presented at the 2026 Annual American Academy of Neurology (AAN) Meeting

The design and rationale for the TRITON-PN Phase 3 study evaluating our third-generation investigational transthyretin (TTR) silencer, nucresiran, in patients with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) was presented at AAN 2026.

Obici, et al. “Design and Rationale of TRITON-PN, a Phase 3 Study to Evaluate the Efficacy of Nucresiran in Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy.”

30 Mar, 2026 New Analyses from Amyloidosis and Hypertension Programs Presented at The American College of Cardiology (ACC) Scientific Sessions 2026

Analyses presented at ACC reinforce the totality of data for vutrisiran in patients with ATTR-CM including the impact on cardiovascular outcomes across a range of patient subgroups, including those with most advanced disease and diastolic dysfunction and on health-related quality of life. Real-world evidence also shows high treatment adherence with quarterly dosing.

A pooled safety analysis from the Phase 2 KARDIA studies demonstrate zilebesiran’s acceptable safety profile across a broad hypertension population, supporting continued investigation in the Phase 3 ZENITH cardiovascular outcomes trial.

Cuddy, et al. “Post Hoc Analysis of Diastolic Dysfunction in the HELIOS-B Study of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy”

DiDomenico, et al. “Polypharmacy and Potential for Drug–Drug Interactions Among Patients With ATTR-CM Initiating Tafamidis”

Hamatani, et al. “Impact of Vutrisiran on the Components of Quality of Life in Transthyretin Cardiomyopathy (ATTR-CM)”

Hefferman, et al. “Real-World Adherence and Persistence With Vutrisiran, an RNA Interference Therapeutic for the Treatment of Transthyretin Amyloidosis”

Witteles, et al. “Vutrisiran Reduces the Risk of Developing Advanced Disease and Demonstrates Benefit in Patients Who Do Develop Advanced Disease in ATTR-CM: Analysis From the HELIOS-B Study”

Desai, et al. “Pooled Safety Analysis of Zilebesiran, an Investigational Long-Acting RNA Interference, From Phase 2 Studies in Patients With Hypertension”

24 Mar, 2026 Alnylam TTR Investor Webinar 2026

On March 24, 2026, we hosted a TTR investor webinar to highlight Alnylam’s progress in delivering for patients with ATTR-CM, as well as the long-term growth and durability of the Company’s flagship TTR franchise. The event included presentations from Alnylam’s senior commercial and medical leaders.

To view the webcast, click here
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26 Feb, 2026 Biomarker Approach, Preclinical Data, and Phase 1 Study Overview of ALN-HTT02 in Adult Patients with Huntington’s Disease, Presented at the CHDI Foundation’s Annual Huntington’s Disease Therapeutics Conference

Data on emerging assays measuring huntingtin (HTT) protein in human cerebrospinal fluid were presented at the CHDI Foundation’s 21^st Annual Huntington’s Disease Therapeutics Conference in Palm Springs, CA. Additional presentations on nonclinical data supporting the tolerability of deep and sustained HTT-lowering in wild-type nonhuman primates and an overview of the ongoing Phase 1 study of HTT02 in adult patients with Huntington’s disease were also included. ALN-HTT02 is an investigational RNAi therapeutic targeting exon 1 of HTT for Huntington’s disease.

Bond et al. “Emerging Approaches to Improve the Quantification of Huntingtin Protein in Human Cerebrospinal Fluid”
Cantley et al. “Preclinical Characterization of ALN-HTT02, an Investigational RNAi Therapeutic Targeting Exon 1 of HTT”
Sloan et al. “ALN-HTT02, an Investigational RNAi Therapeutic Targeting Exon 1 of HTT in Phase 1 Development for Huntington’s Disease”

04 Dec, 2025 New Data Evaluating Rates of ARIA from the Ongoing Phase 1 Study of Mivelsiran in Early-Onset Alzheimer’s Disease Presented at the Clinical Trials on Alzheimer’s Disease Conference 2025

Rates of amyloid-related imaging abnormalities (ARIA) from an interim analysis of the ongoing Phase 1 study of mivelsiran in patients with early-onset Alzheimer’s disease (EOAD) were presented at the Clinical Trials on Alzheimer’s Disease (CTAD) Conference 2025 in San Diego, CA. Mivelsiran is an investigational CNS-targeting RNAi therapeutic designed to reduce amyloid precursor protein (APP) and downstream Aβ peptides.

Parikh, et al. “Amyloid-Related Imaging Abnormalities in an Ongoing Phase 1 Study of Mivelsiran, an Investigational RNA Interference Therapeutic Targeting Amyloid Precursor Protein, in Patients with Alzheimer’s Disease”

08 Nov, 2025 New Analyses from Amyloidosis and Hypertension Programs Presented at the American Heart Association (AHA) Scientific Sessions 2025

New analyses highlight that vutrisiran improved measures of heart structure and function and reduced amyloid burden in some patients.

Further findings show vutrisiran preserved kidney function and reduced the risk of death and cardiovascular (CV) events in patients with advanced chronic kidney disease (CKD).

Results from Cohort B of the KARDIA-3 Phase 2 trial evaluating zilebesiran in patients with uncontrolled hypertension with established or at high risk for CV disease on two or more background medications were also presented as a late-breaking abstract.

Fontana, et al. “Effects of Vutrisiran on Measures of Cardiac Structure, Function and Amyloid Burden by Cardiovascular Magnetic Resonance from the HELIOS-B Trial”

Jering, et al. “Left Atrial Strain and Clinical Outcomes in Transthyretin Amyloid Cardiomyopathy: Insights from the HELIOS-B Trial on Vutrisiran Efficacy”

Manafi, et al. “Right Ventricular Free Wall Strain and Clinical Outcomes in Transthyretin Amyloid Cardiomyopathy and Effect of Vutrisiran: The HELIOS-B Study”

Sheikh, et al. “Vutrisiran Improved Outcomes Versus Placebo in Patients with Transthyretin Amyloidosis with Cardiomyopathy and Severe Chronic Kidney Disease: Post Hoc Analysis of HELIOS-B”

Pagidipati, et al. “KARDIA-3: A Randomized Trial of Zilebesiran Versus Placebo on Top of Standard Care for Patients with Hypertension and Established Cardiovascular Disease or High Cardiovascular Risk with or Without Chronic Kidney Disease”

28 Sep, 2025 New Data from HELIOS-B Phase 3 Study Presented at the Heart Failure Society Annual Scientific Meeting

Late-breaking abstract from the HELIOS-B Phase 3 study showed vutrisiran significantly lowered rates of gastrointestinal events, a common disease manifestation of ATTR-CM, by up to 49% in patients.

Additional HELIOS-B analyses validate the efficacy and safety of vutrisiran as a monotherapy in ATTR-CM and support the consistent benefit of vutrisiran treatment in ATTR-CM patients regardless of baseline health status and quality of life.

Urey, et al. “Evidence of Fewer Gastrointestinal Events in ATTR-CM Patients Treated with Vutrisiran Compared with Placebo: Analysis from HELIOS-B”

Witteles, et al. “Outcomes of the HELIOS-B Monotherapy Population: A Post Hoc Analysis Censoring Data Following Tafamidis Initiation”

Huttin, et al. “Efficacy of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy by Baseline Health Status and Quality of Life”

30 Aug, 2025 New Data from Hypertension and Amyloidosis Programs Presented at the European Society of Cardiology Congress 2025, Madrid

Presentations demonstrated the potential of RNAi therapeutics to transform the treatment of cardiovascular disease.

Pagidipati, et al. “KARDIA-3: Zilebesiran as Add-on Therapy in Adults With Hypertension Who Have Established Cardiovascular Disease or Are at High Cardiovascular Risk”

Weber, et al. “Impact of zilebesiran, an investigational RNA interference therapeutic targeting hepatic angiotensinogen, on renin–angiotensin system biomarkers in patients with mild-to-moderate hypertension”

Hanna, et al. “Vutrisiran reduces days lost to death and/or hospitalization versus placebo in patients with transthyretin amyloidosis with cardiomyopathy in the HELIOS-B trial”

Garcia-Pavia, et al. “HELIOS-B: 12-month results from the open-label extension period of vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy”

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29 Jul, 2025 New Data Supporting the Development of Mivelsiran, an Investigational CNS-targeting RNAi Therapeutic, Presented at the Alzheimer’s Association International Conference 2025

Cohen, et al. “Multiple-Dose Results from an Ongoing Phase 1 Study of Mivelsiran, an Investigational RNA Interference Therapeutic Targeting Amyloid-Beta Precursor Protein for Alzheimer’s Disease”

Taillie, et al. “Small Interfering RNA Targeting Amyloid-Beta Precursor Protein Reduces Alzheimer’s Disease Pathology in 5xFAD Mice”