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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31 Jul, 2024 Additional Results From Ongoing Phase 1 Study of Mivelsiran (ALN-APP) Presented at the Alzheimer’s Association International Conference (AAIC) 2024

Additional interim results from the single ascending dose part of the ongoing Phase 1 study of mivelsiran (ALN-APP), an investigational RNAi therapeutic in development for Alzheimer’s disease and cerebral amyloid angiopathy, were presented at the Alzheimer’s Association International Conference (AAIC) 2024.

Cohen, et al. “Single ascending dose results from an ongoing Phase 1 study of mivelsiran (ALN-APP), the first investigational RNA interference therapeutic targeting amyloid precursor protein for Alzheimer’s disease”

29 Jul, 2024 Preclinical Data from Tau-Targeting Program in the Central Nervous System Presented at the Alzheimer’s Association International Conference (AAIC) 2024

Preclinical data from a Tau-targeting program were presented at the Alzheimer’s Association International Conference (AAIC) 2024. An RNAi therapeutic development candidate leveraging Alnylam’s C16-siRNA CNS delivery platform was shown to achieve durable Tau-lowering for at least four months after a single intrathecal dose with an encouraging tolerability profile.

Kevin Sloan, et al, “A New Approach to Tau Lowering Using C16-siRNA Conjugates”

09 Jul, 2024 Analysis of Six-minute Walk Test as a Clinical End Point in Cardiomyopathy Clinical Trials Published in Journal of Comparative Effectiveness Research

Results from an analysis of 41 studies that measured the six-minute walk test (6MWT) and rates of hospitalization and/or death in people with cardiomyopathy, including transthyretin amyloidosis with cardiomyopathy, were published in the Journal of Comparative Effectiveness Research.

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26 Jun, 2024 New Phase 1 Results for ALN-KHK Presented at the American Diabetes Association’s 84th Scientific Sessions

We presented additional positive interim results for an ongoing Phase 1/2 study of ALN-KHK, an investigational RNAi therapeutic targeting ketohexokinase in development for the treatment of type 2 diabetes, at the American Diabetes Association’s 84^th Scientific Sessions. Our investigational therapeutic showed an encouraging safety and tolerability profile and target engagement in the single ascending dose part of the study in healthy overweight to obese adults.

Maratos-Flier, et al. “ALN-KHK, an Investigational RNA Interference Therapeutic, Successfully Targets Hepatic Ketohexokinase in a Single Ascending Dose Study of Overweight to Obese Adults”

06 Jun, 2024 Updates From Our KARDIA Clinical Trial Program Presented at the 33rd European Meeting on Hypertension and Cardiovascular Protection

New results from the KARDIA-2 Phase 2 study of zilebesiran, an investigational RNAi therapeutic in development for the treatment of hypertension, when added to standard of care antihypertensive medications, were presented at the 33rd European Meeting on Hypertension and Cardiovascular Protection. Additional details were presented on the KARDIA-3 study design with zilebesiran as an add-on therapy in patients with high cardiovascular risk and hypertension inadequately controlled by standard of care antihypertensives.

Saxena, et al. “Zilebesiran as Add-On Therapy in Patients with Hypertension Inadequately Controlled with a Standard Antihypertensive Medication: Efficacy and Safety Results from the KARDIA-2 Study”

Havasi, et al. “Zilebesiran as Add-On Therapy in Patients with High Cardiovascular Risk and Hypertension Inadequately Controlled by Standard of Care Antihypertensive”