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”

09 Nov, 2024 Design of Phase 1 Study of ALN-HTT02 in Adult Patients with Huntington’s Disease Presented at the Annual Meeting of the Huntington Study Group (HSG)

The design of a Phase 1 study of ALN-HTT02—an investigational RNAi therapeutic targeting huntingtin (HTT) for Huntington’s disease—was presented at the 31st Annual Meeting of the Huntington Study Group (HSG) in Cincinnati, Ohio.

Sloan, et al. “ALN-HTT02, a Novel C16-siRNA Conjugate for HTT-lowering in the CNS”

31 Oct, 2024 Overview of cAPPricorn-1 Trial of Mivelsiran Presented at CTAD Conference 2024

Trial design and rationale for the global Phase 2 cAPPricorn-1 study of mivelsiran (ALN-APP) in patients with cerebral amyloid angiopathy (CAA), were presented at the Clinical Trials on Alzheimer’s Disease (CTAD) Conference 2024.

Lee, et al. “Design and Rationale of cAPPricorn-1, A Phase 2 Study of Mivelsiran in Patients with Cerebral Amyloid Angiopathy”

09 Oct, 2024 Alnylam TTR Investor Day 2024

On October 9, 2024, we hosted a TTR Investor Day to discuss Alnylam’s potential for market leadership in ATTR amyloidosis. The event included presentations from Alnylam’s senior commercial leaders, as well as a leading expert in ATTR amyloidosis.

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

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29 Sep, 2024 Additional Results from the HELIOS-B Phase 3 study of Vutrisiran in Patients with ATTR Amyloidosis with Cardiomyopathy Presented at the Heart Failure Society of America Annual Scientific Meeting 2024

Additional results from the HELIOS-B Phase 3 study of vutrisiran, an RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), were presented in the Late Breaking Clinical Research Session 1 at the Heart Failure Society of America Annual Scientific Meeting 2024.

Jering, et al. “Effects of Vutrisiran on Echocardiographic Cardiac Structure and Function: The HELIOS-B Trial”

Maurer, et al. “Exploratory Biomarker Analyses from HELIOS-B, a Phase 3 Study of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy”

24 Sep, 2024 New Results for Acute Hepatic Porphyria (AHP) Program Presented at ICPP 2024

New results from a post hoc subgroup analysis of the ENVISION Phase 3 study evaluating long-term outcomes in patients with acute hepatic porphyria (AHP) who were not attack-free after the first six months of GIVLAARI^® (givosiran) treatment were presented at The International Congress of Porphyrins and Porphyrias (ICPP 2024). Additional data presented at ICPP 2024 included demographic and clinical characteristics of patients with AHP enrolled in ELEVATE, an international, prospective, observational registry.

Ventura, et al. “Long-term clinical outcomes of patients with acute hepatic porphyria who were not attack-free after 6 months of givosiran treatment: a subgroup analysis of the phase 3 ENVISION study”

Sardh, et al. “Patient demographics and clinical characteristics at enrolment in ELEVATE, an international registry of acute hepatic porphyria”

13 Sep, 2024 Design of Phase 1 Study of ALN-HTT02 in Adult Patients with Huntington’s Disease Presented at the EHDN & Enroll-HD 2024 Congress

The design of a Phase 1 study of ALN-HTT02—an investigational RNAi therapeutic targeting huntingtin (HTT) for Huntington’s disease—was presented at the EHDN & Enroll-HD 2024 Congress in Strasbourg, France. 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, a Novel C16-siRNA Conjugate for HTT-lowering in the CNS”

Cantley, et al. “Tolerability of HTT-Lowering: Lessons Learned from Nonhuman Primates”

01 Sep, 2024 New Data from the KARDIA-2 Phase 2 Study of Zilebesiran and the APOLLO-B Phase 3 and APOLLO-OLE Studies of Patisiran Presented at the European Society of Cardiology (ESC) Congress 2024

Findings from a subgroup analysis of the KARDIA-2 Phase 2 study of zilebesiran, in development for the treatment of hypertension, and new data from post-hoc analyses of the APOLLO-B Phase 3 and APOLLO-OLE studies of patisiran in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM) were presented at the European Society of Cardiology Congress (ESC) 2024.

Saxena, et al. “Subgroup Results from KARDIA-2: Impact of Demographic and Baseline Disease Characteristics on Zilebesiran Response in Patients with Hypertension Uncontrolled by a Standard Oral Antihypertensive”

Lairez, et al. “Long-term Effects of Patisiran on Survival and Cardiac Parameters in Patients with Transthyretin-Mediated Cardiac Amyloidosis: Post-hoc Analyses of APOLLO-B and Cardiac Subpopulation of APOLLO-OLE”

30 Aug, 2024 Detailed Results from the HELIOS-B Phase 3 study of Vutrisiran in Patients with ATTR Amyloidosis with Cardiomyopathy Presented at the ESC Congress 2024 and Published in The New England Journal of Medicine

Detailed results from the Phase 3 HELIOS-B study of vutrisiran, an RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), were presented in a Hot Line session at the European Society of Cardiology Congress (ESC) 2024 and simultaneously published in The New England Journal of Medicine.

Fontana, et al. “Primary Results from HELIOS-B, a Phase 3 Study of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy”

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