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.
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.
A pooled analysis from the KARDIA Phase 2 studies demonstrated zilebesiran’s manageable safety profile as an add-on treatment for patients with hypertension receiving treatment with a background renin-angiotensin-aldosterone system (RAAS) inhibitor. The findings support continued evaluation of zilebesiran in the Phase 3 cardiovascular outcomes (CVOT) trial, ZENITH.
Analyses presented at Heart Failure 2026 reinforce the consistent clinical profile of vutrisiran in patients with the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM), including across clinically complex populations with a high disease burden and in the context of concomitant therapies. Additional data further characterize transthyretin (TTR) knockdown and support its relevance in real-world clinical practice.
A pooled safety analysis evaluating vitamin A-related outcomes across clinical trial and real-world datasets shows no evidence of clinically meaningful safety concerns associated with TTR reduction. The design of DemonsTTRate, a global, long-term observational study evaluating real-world use of vutrisiran in ATTR-CM, was also presented.
The design and rationale for the TRITON-PN Phase 3 study evaluating our third-generation investigational transthyretin (TTR) silencer, nucresiran, in patients with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) was presented at AAN 2026.
Analyses presented at ACC reinforce the totality of data for vutrisiran in patients with ATTR-CM including the impact on cardiovascular outcomes across a range of patient subgroups, including those with most advanced disease and diastolic dysfunction and on health-related quality of life. Real-world evidence also shows high treatment adherence with quarterly dosing.
A pooled safety analysis from the Phase 2 KARDIA studies demonstrate zilebesiran’s acceptable safety profile across a broad hypertension population, supporting continued investigation in the Phase 3 ZENITH cardiovascular outcomes trial.
