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”

15 Jun, 2025 UK Biobank Analyses and Preclinical Data on Hepatic Plasminogen Lowering Presented at the 2025 European Hematology Association (EHA) Congress

Human genetics and proteomics data from UK Biobank as well as data from preclinical models relevant to ALN-6400—an investigational RNAi therapeutic targeting plasminogen for bleeding disorders—were presented at the 2025 European Hematology Association (EHA) Congress, June 12-15, Milan, Italy.

Ivanciu, et al. “Hepatic plasminogen lowering with RNA interference for the treatment of bleeding disorders is unlikely to pose thrombotic risk based on UK Biobank analyses and mouse models of provoked thrombosis.”

29 May, 2025 Detailed Results from the KARDIA-2 Phase 2 study of Zilebesiran Published in The Journal of the American Medical Association

Detailed results from the KARDIA-2 Phase 2 study supporting the safety and efficacy of zilebesiran, an investigational RNAi therapeutic for the treatment of hypertension, when used in combination with one of three standard classes of antihypertensive medications were published in The Journal of the American Medical Association.

Desai, et al. “Add-on Treatment with Zilebesiran for Inadequately Controlled Hypertension: The KARDIA-2 Randomized Clinical Trial

17 May, 2025 New Data Presented from Across the Transthyretin Amyloidosis Franchise at the Heart Failure 2025 Congress

The following presentations include the latest data from the HELIOS-B Phase 3 study and results from across the flagship transthyretin amyloidosis (TTR) franchise presented at Heart Failure 2025 Congress, a scientific congress of the European Society of Cardiology.

Fontana, et al. “Vutrisiran reduces all-cause mortality, cardiovascular mortality, and cardiovascular events in patients with transthyretin amyloid cardiomyopathy: Analysis from the HELIOS-B trial”

Morbach, et al. “Clinical Presentation and Treatment Landscape of Patients with Transthyretin Amyloidosis With Cardiomyopathy: A Real-word Study in Five European Countries and Japan”

Bondue, et al. “Utility of Genetic Testing For Diagnosing hATTR Patients: Results from a European and Middle East Genetic Testing Program”

Fontana, et al. “Design and Rationale of a Phase 3 Study to Evaluate Efficacy and Safety of Nucresiran (ALN-TTRsc04) in Patients with Transthyretin Amyloidosis with Cardiomyopathy”

Gonzalez-Costello, et al. “Vutrisiran In Patients With Transthyretin Amyloidosis with Cardiomyopathy In HELIOS-B Who Had Progressed On Tafamidis”

01 Apr, 2025 Additional Presentation at American College of Cardiology Scientific Session 2025

An Analysis of US Insurance Claims Data examining real-world treatment persistency among patients receiving tafamidis was presented at the American College of Cardiology Scientific Session 2025.

Patel, et al. “Real-World Persistency on Tafamidis: An Analysis of US Insurance Claims Data”

29 Mar, 2025 New Data from Analyses of the HELIOS-B Phase 3 Study of Vutrisiran in Patients with ATTR Amyloidosis with Cardiomyopathy Presented at American College of Cardiology Scientific Session 2025

The following analyses from the Phase 3 HELIOS-B study which evaluated vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy were presented at the American College of Cardiology Scientific Session 2025. Two analyses were also published in the Journal of American College of Cardiology.

Jering, et al. “The Relationship Between Cardiac Structure, Function, and Clinical Outcomes and the Impact of Vutrisiran from the HELIOS-B Trial”

Maurer, et al. “Impact of Baseline Heart Failure Severity on Efficacy of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy in the HELIOS-B Trial: A Subgroup Analysis”

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Sheikh, et al. “Maintenance or Improvement of Functional Capacity, Health Status, and Quality of Life with Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy: Data from the HELIOS-B Study”

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