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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27 Feb, 2025 Design of Phase 1 Study of ALN-HTT02 in Adult Patients with Huntington’s Disease Presented at the CHDI Foundation’s Annual Huntington’s Disease Therapeutics Conference

The design of an ongoing Phase 1 study of ALN-HTT02—an investigational RNAi therapeutic targeting exon 1 of huntingtin (HTT) for Huntington’s disease—was presented at the CHDI Foundation’s 20th Annual Huntington’s Disease Therapeutics Conference in Palm Springs, CA. Nonclinical data supporting the tolerability of deep and sustained HTT-lowering in wild-type nonhuman primates after single and repeated intrathecal administration of ALN-HTT02—which employs Alnylam’s C16-siRNA CNS delivery platform—were also shared.

Sloan et al. “ALN-HTT02, an Investigational RNAi Therapeutic Targeting Exon 1 of HTT in Phase 1 Development for Huntington’s Disease”

25 Feb, 2025 Alnylam R&D Day 2025

On February 25, 2025, we hosted an R&D Day event showcasing Alnylam’s late-stage clinical efforts, next wave programs, and platform advances. The event included presentations from Alnylam senior leaders, as well as a leading expert in Huntington’s disease.

To view the webcast, click here
To view the presentation, click here

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06 Feb, 2025 Preclinical Data for Amyloid-Beta Precursor Protein (APP) siRNA in Cerebral Amyloid Angiopathy (CAA) Presented at the International Stroke Conference (ISC), 2025

Therapeutic rationale for APP-lowering siRNA and preclinical results from rodent models of CAA were presented at the International Stroke Conference (ISC), February 5-7, 2025, Los Angeles, United States.

Brown, et al. “Targeting Cerebral Amyloid Angiopathy at its Source: An Amyloid-beta Precursor Protein-Targeted siRNA Reduced Vascular Amyloid-beta and Hemorrhage in Rodent Models”

18 Nov, 2024 New Data Presented in ATTR Amyloidosis and Hypertension at the American Heart Association (AHA) Scientific Sessions 2024

The following studies were presented at the American Heart Association (AHA) Scientific Sessions 2024: a new analysis of the Phase 3 HELIOS-B study of vutrisiran, an investigational RNAi therapeutic for the treatment of ATTR cardiomyopathy, and a new analysis of the Phase 2 KARDIA-1 study of zilebesiran, an investigational RNAi therapeutic for the treatment of hypertension.

Fontana, et al. “Impact of Vutrisiran on Outpatient Worsening Heart Failure in Patients with Transthyretin Amyloidosis with Cardiomyopathy in the HELIOS-B Trial”

Krohn, et al. “Impact of Long-Term Blood Pressure Variability on Adverse Cardiovascular Outcomes in High- and Low-Risk Populations”

Zappe, et al. “Home Blood Pressure Reductions with Zilebesiran in Patients with Mild-to-Moderate Hypertension Are Consistent with Ambulatory and Office Blood Pressure Reductions in the KARDIA-1 Study”

17 Nov, 2024 New Results from an Interim Phase 1 Study Evaluating Nucresiran (ALN-TTRsc04) Presented at the American Heart Association (AHA) Scientific Sessions 2024

Results from the interim Phase 1 study of nucresiran (ALN-TTRsc04), an investigational next-generation RNAi therapeutic for the treatment of transthyretin amyloidosis, were presented at the American Heart Association (AHA) Scientific Sessions 2024.

Murad et al. “A Phase 1, Single Ascending Dose Study to Evaluate ALN-TTRsc04, a Next-Generation RNA Interference Therapeutic, in Healthy Participants for Potential Treatment of Transthyretin Amyloidosis”