Capella

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

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”

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

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”