Capella

11 Oct, 2026 New Vutrisiran Data Presented at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 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.

Cuddy, et al. "Long-term Vutrisiran Reduces Cardiovascular Events and Hospitalizations in Patients with Transthyretin Cardiac Amyloidosis in the HELIOS-B One-Year Open Label Extension"

Mitter, et al. "Characteristics and Treatment Patterns in Patients with ATTR-CM Treated with Vutrisiran in Clinical Practice"

22 Sep, 2026 Baseline Characteristics for cAPPricorn-1 Phase 2 Study Presented at the International Cerebral Amyloid Angiopathy (ICAA) Conference

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.

Parikh, et al. “Baseline Characteristics in cAPPricorn-1, a Phase 2 Study in Cerebral Amyloid Angiopathy”

Parikh, et al. “Ethnic/Racial Differences in Microbleed Distribution in Lobar ICH: An Analysis of the ERICH Study”
 

17 Sep, 2026 RNAi Roundtable: Zilebesiran, An Investigational RNAi Therapeutic for Patients with Hypertension and High Cardiovascular Risk

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. 

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30 Aug, 2026 The Power of RNAi for ATTR-CM

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.

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29 Aug, 2026 New Data from ATTR and Hypertension Programs Presented at the European Society of Cardiology (ESC) Congress 2026 Demonstrate Strength of RNAi-Powered Silencing

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. 

Hamatani, et al. "Effect of Vutrisiran According to Baseline Tafamidis Use in Transthyretin Amyloidosis with Cardiomyopathy”

Damy, et al. "Amylo'ExTTRa: Exploring Extra-Cardiac Manifestations in Patients with ATTR-CM Using a French National Health Data System"

Lairez, et al. "Vutrisiran is Associated with Preservation of Intrinsic Capacity and Healthy Ageing in Patients with Transthyretin Amyloidosis with Cardiomyopathy: Post Hoc Analysis of HELIOS-B"

Schwarting, et al. "Fewer Adverse Events Reported in ATTR-CM Patients Treated with Vutrisiran Versus Placebo: Post Hoc Safety Analysis from HELIOS-B"

Zaroui, et al. "Cardiac Presentation and Treatment Response by Sex in Patients with Transthyretin Amyloidosis: Pooled Analysis of Vutrisiran and Patisiran Phase 3 Studies"

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.

Pagidipati, et al. "Zilebesiran in Combination with a Diuretic in Patients with Uncontrolled Hypertension: A Subgroup Analysis from the Phase 2 KARDIA-3 Trial"

15 Jul, 2026 Alnylam Showcases Progress with Neuroscience Programs at Alzheimer’s Association International Conference (AAIC) 2026

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”

13 Jul, 2026 New Analysis of UK Biobank Data on Plasminogen Levels Presented at International Society on Thrombosis and Haemostasis (ISTH) 2026

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.

Krohn, et al. “Low plasminogen levels are not associated with an increased risk of arterial thrombosis”

25 Jun, 2026 RNAi Roundtable: ALN-6400, An Investigational RNAi Therapeutic Targeting Plasminogen to Address Rare 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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16 Jun, 2026 New Data Presented at the 2026 Peripheral Nerve Society (PNS) Annual Meeting

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.

Plante-Bordeneuve, et al. “RNAi efficacy in large and small fiber-predominant polyneuropathy of hereditary transthyretin amyloidosis: HELIOS-A post-hoc analysis”

Cauquil, et al. “Neurofilament light chain as a biomarker in patients with hereditary transthyretin amyloidosis with polyneuropathy”

11 Jun, 2026 New Pooled Analysis from Zilebesiran Program Presented at the 63rd European Renal Association (ERA) Congress

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.

Wheeler, et al. “Zilebesiran as Add-on Treatment to RAAS Inhibitors for Patients with Hypertension: Data from KARDIA-2 and KARDIA-3 Phase 2 Trials”