Initial Phase 1b data for brezosiran, an investigational RNAi therapeutic in development for Huntington’s disease, were presented at the European Huntington’s Disease Network (EHDN) Clinical Research Congress 2026.
A new analysis from the open-label extension (OLE) period of the HELIOS-B Phase 3 study reinforced the clinical profile of vutrisiran across transthyretin amyloidosis with cardiomyopathy (ATTR-CM) patient populations, demonstrating that long-term, continuous treatment with vutrisiran was associated with lower rates of hospitalizations and urgent heart failure visits, with the greatest benefit observed in patients who were earlier in their disease course and received vutrisiran as first-line therapy. In the baseline tafamidis group, long-term treatment with vutrisiran was associated with a significant reduction in urgent heart failure visits, supporting the benefits of TTR silencing across a broad range of ATTR-CM patients. Results were simultaneously published in the Journal of Cardiac Failure.
Additionally, findings from a U.S. database analysis indicate that vutrisiran is being used across a wide spectrum of ATTR-CM patients in real-world clinical practice.
A review of the baseline characteristics of participants enrolled in the cAPPricorn-1 study, an ongoing phase 2, double-blind, placebo-controlled study of mivelsiran in individuals with cerebral amyloid angiopathy (CAA) were presented at the International CAA (ICAA) Conference in Banff, Canada. A review of ethnic and racial differences in the distribution of cerebral microbleeds among patients with lobar intracerebral hemorrhage enrolled in the Ethnic/Racial Variations of Intracerebral Hemorrhage (ERICH) study was also presented.
On September 17, 2026, we hosted an online RNAi Roundtable to review progress with zilebesiran, an investigational RNAi therapeutic for patients with hypertension and high cardiovascular risk.
Alnylam hosted an event at the European Society of Cardiology 2026 congress to discuss the Company’s leadership in TTR, including the strong ongoing launch of AMVUTTRA in ATTR-CM, and the underlying confidence in its next-generation RNAi-powered TTR silencer, nucresiran.
New analyses presented at ESC reinforced the clinical profile of vutrisiran across transthyretin amyloidosis (ATTR) patient populations, treatment settings, and manifestations of disease, and the potential application of RNAi to uncontrolled hypertension.
A late-breaking prespecified subgroup analysis of the HELIOS-B Phase 3 study demonstrated that vutrisiran provides consistent clinical benefit across all-cause mortality and recurrent cardiovascular events in patients, irrespective of baseline tafamidis use.
A subgroup analysis from the KARDIA-3 Phase 2 study highlights the potential for zilebesiran to provide durable blood pressure control, including at nighttime. The findings support ongoing evaluation in the Phase 3 cardiovascular outcomes trial (CVOT), ZENITH. Zilebesiran will be featured as part of Alnylam's 10th "RNAi Roundtable" series on September 17, 2026, at 10:30 a.m. ET.
Presentations highlight advances across Alnylam’s growing neuroscience portfolio and underscore the potential of RNAi therapeutics to address the needs of patients with debilitating neurological diseases. Presentations include the design and rationale of the global Phase 2 APPlauDS study of mivelsiran in people with Down syndrome-associated Alzheimer’s disease (DS-AD); updated Phase 1 data on mivelsiran in patients with early-onset Alzheimer’s disease, showing no evidence of increased risk of amyloid-related imaging abnormalities (ARIA); and the design of an ongoing first-in-human Phase 1 study of ALN-5288—an investigational tau-lowering RNAi therapeutic being developed in collaboration with Regeneron Pharmaceuticals—and supporting preclinical data.
Rafii, et al. “Design of APPlauDS, A Phase 2 Study of Mivelsiran in People With Down Syndrome and Early-Stage Alzheimer’s Disease”
Parikh, et al. “Amyloid-Related Imaging Abnormalities in a Phase 1 Study of Mivelsiran in Patients With Early-Onset Alzheimer’s Disease”
Sloan, et al. “ALN-5288, an Investigational RNA Interference Therapeutic Targeting MAPT in Phase 1 Development for Alzheimer’s Disease”
Farley, et al. “RNA Interference Knockdown of Microtubule-Associated Protein Tau (MAPT) as a Potential Therapeutic Strategy for Tauopathies”
In this analysis of UK Biobank data presented at ISTH, low plasminogen levels were not associated with an increased risk of myocardial infarction, ischaemic stroke, or arterial thrombosis. The findings support the continued development of ALN-6400—an investigational RNAi therapeutic targeting plasminogen—for the treatment of bleeding disorders.
On June 25, 2026, we hosted an online RNAi Roundtable to review progress with ALN-6400, an investigational RNAi therapeutic in development to address rare bleeding disorders.
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A post-hoc analysis of HELIOS-A demonstrated that vutrisiran and patisiran improved outcomes in patients with hATTR-PN regardless of baseline nerve conduction status, with those treated earlier in their disease course showing the greatest potential for disease reversal. Additionally, results of the NeuroFeeL study showed that neurofilament light chain (NfL) levels correlate with hATTR-PN disease severity, reinforcing NfL’s potential as a biomarker to support earlier diagnosis and clinical management.
