On March 24, 2026, we hosted a TTR investor webinar to highlight Alnylam’s progress in delivering for patients with ATTR-CM, as well as the long-term growth and durability of the Company’s flagship TTR franchise. The event included presentations from Alnylam’s senior commercial and medical leaders.
To view the webcast, click here
To view the presentation, click here
Data on emerging assays measuring huntingtin (HTT) protein in human cerebrospinal fluid were presented at the CHDI Foundation’s 21st Annual Huntington’s Disease Therapeutics Conference in Palm Springs, CA. Additional presentations on nonclinical data supporting the tolerability of deep and sustained HTT-lowering in wild-type nonhuman primates and an overview of the ongoing Phase 1 study of HTT02 in adult patients with Huntington’s disease were also included. ALN-HTT02 is an investigational RNAi therapeutic targeting exon 1 of HTT for Huntington’s disease.
Bond et al. “Emerging Approaches to Improve the Quantification of Huntingtin Protein in Human Cerebrospinal Fluid”
Cantley et al. “Preclinical Characterization of ALN-HTT02, an Investigational RNAi Therapeutic Targeting Exon 1 of HTT”
Sloan et al. “ALN-HTT02, an Investigational RNAi Therapeutic Targeting Exon 1 of HTT in Phase 1 Development for Huntington’s Disease”
Rates of amyloid-related imaging abnormalities (ARIA) from an interim analysis of the ongoing Phase 1 study of mivelsiran in patients with early-onset Alzheimer’s disease (EOAD) were presented at the Clinical Trials on Alzheimer’s Disease (CTAD) Conference 2025 in San Diego, CA. Mivelsiran is an investigational CNS-targeting RNAi therapeutic designed to reduce amyloid precursor protein (APP) and downstream Aβ peptides.
New analyses highlight that vutrisiran improved measures of heart structure and function and reduced amyloid burden in some patients.
Further findings show vutrisiran preserved kidney function and reduced the risk of death and cardiovascular (CV) events in patients with advanced chronic kidney disease (CKD).
Results from Cohort B of the KARDIA-3 Phase 2 trial evaluating zilebesiran in patients with uncontrolled hypertension with established or at high risk for CV disease on two or more background medications were also presented as a late-breaking abstract.
Late-breaking abstract from the HELIOS-B Phase 3 study showed vutrisiran significantly lowered rates of gastrointestinal events, a common disease manifestation of ATTR-CM, by up to 49% in patients.
Additional HELIOS-B analyses validate the efficacy and safety of vutrisiran as a monotherapy in ATTR-CM and support the consistent benefit of vutrisiran treatment in ATTR-CM patients regardless of baseline health status and quality of life.
Presentations demonstrated the potential of RNAi therapeutics to transform the treatment of cardiovascular disease.
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.
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.
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.
