Welcome to Capella, Alnylam’s destination for updates on our work translating the breakthrough discovery of RNA interference (RNAi) into an innovative new class of medicines. We’ve been pioneering RNAi therapeutics since 2002 and are excited to share our ongoing scientific progress.
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
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.
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.
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”
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.
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.
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.
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”
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.
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 84th 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.
