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”

05 Jun, 2024 New Results from ENVISION Phase 3 Study of GIVLAARI® (givosiran) Presented at EASL Congress 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 who were not attack-free after the first six months of GIVLAARI^® (givosiran) treatment were presented at the European Association for the Study of the Liver (EASL) Congress 2024.

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

23 May, 2024 Final Results from Phase 2 Open-Label Extension Study of OXLUMO® (lumasiran) Presented at ERA Congress

New 54-month results from the Phase 2 open-label extension study evaluating long-term treatment with OXLUMO^® (lumasiran) in patients with primary hyperoxaluria type 1 were presented at the 61st European Renal Association (ERA) Congress.

Frishberg, et al. “Long-term Treatment with Lumasiran: Final Results from the Phase 2 Open-Label Extension Study”

07 Apr, 2024 New Analysis From KARDIA-1 Phase 2 Study Presented at the 2024 American College of Cardiology Annual Scientific Session

A new subgroup analysis from the KARDIA-1 Phase 2 study of zilebesiran, an investigational RNAi therapeutic in development for the treatment of hypertension, was presented at the 2024 American College of Cardiology (ACC) Annual Scientific Session.

Saxena, et al. “Consistent Antihypertensive Efficacy of the RNA Interference Therapeutic Zilebesiran: Subgroup Results from the KARDIA-1 Phase 2 Study in Patients with Hypertension”

07 Apr, 2024 New Results From KARDIA-2 Phase 2 Study Presented at the 2024 American College of Cardiology Annual Scientific Session

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 2024 American College of Cardiology (ACC) Annual Scientific Session.

Bakris, et al. “Zilebesiran In Combination With A Standard-of-care Antihypertensive In Patients With Inadequately Controlled Hypertension – Primary Results From The Phase 2 KARDIA-2 Study”

06 Apr, 2024 New Results from APOLLO-B Phase 3 Study Presented at the 2024 American College of Cardiology (ACC) Annual Scientific Session

New results from a post-hoc analysis of outpatient heart failure worsening in the APOLLO-B Phase 3 study of patisiran were presented at the 2024 ACC Annual Scientific Session. Additional data were also presented from a meta-regression analysis of the association between change in 6-minute walk distance and survival in transthyretin-mediated amyloidosis with cardiomyopathy.

Fontana, et al. “Outpatient Heart Failure Worsening in Patients with Cardiac Transthyretin Amyloidosis: Results from the APOLLO-B Trial”
Kumar, et al. “Meta-Regression Analysis of the Association Between Change in Six Minute Walk Distance and Survival in Transthyretin-Mediated Amyloidosis With Cardiomyopathy”

20 Mar, 2024 Non-clinical Data for APP-targeting siRNA Presented at the 2024 International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD) and Related Neurological Disorders

Non-clinical data for APP-targeting siRNA showing reduced beta-CTF and corrected endosomal abnormalities in cell models of Alzheimer’s Disease were presented at AD/PD 2024, in Lisbon, Portugal.

Dang, et al. “An RNAi Therapeutic Targeting APP Reduced Beta-CTF and Corrected Endosomal Abnormalities in Multiple Human Alzheimer’s Disease iPSC Lines”

29 Feb, 2024 ALN-HTT02 Updates Presented at the CHDI Foundation’s Annual Huntington’s Disease Therapeutics Conference

New preclinical data for ALN-HTT02—an RNAi therapeutic development candidate targeting huntingtin (HTT) for Huntington’s disease (HD)—were presented during the CHDI Foundation’s 19th Annual Huntington’s Disease Therapeutics Conference in Palm Springs, CA . Additional data were presented on the emerging tolerability profile of the underlying C16-siRNA central nervous system delivery platform, highlighting potential differences with antisense oligonucleotides (ASOs) in development for HD.

Cantley, et al. “A New Approach to HTT-Lowering Using C16-siRNA Conjugates”
De Angelis, et al. “Emerging Tolerability Profiles of C16-siRNA Conjugates for CNS Delivery”