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”

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”

12 May, 2026 New Analyses from Vutrisiran Program Presented at Heart Failure 2026

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.

Abovich, et al. “Influence of Disease-Modifying Therapy on the Effectiveness of Vutrisiran in Transthyretin Cardiac Amyloidosis”

Birkhoelzer, et al. “Comorbidity Burden in Transthyretin Amyloidosis With Cardiomyopathy: Insights From the HELIOS-B Trial”

Mansell, et al. “Consistent Efficacy of Vutrisiran Across Sexes in ATTR-CM, HELIOS-B Trial – Prespecified Sex Analysis”

Sheikh, et al. “Influence of Vutrisiran on Systolic Blood Pressure in ATTR-CM: Insights From HELIOS-B”

Wang, et al. “Effect of Vutrisiran in Patients with and without Atrial Fibrillation or Flutter: Analysis from HELIOS-B”

Algalarrondo, et al. “Vutrisiran-Mediated Knockdown of Transthyretin in Patients with Transthyretin Amyloidosis”

Maurer, et al. “Treatment with Transthyretin-Lowering RNA Interference Therapeutics is Not Associated with Ocular or Other Clinical Events Due to Vitamin A Reduction: Pooled Analysis of Vutrisiran and Patisiran Data”

Garcia-Pavia, et al. “Design and Rationale of DemonsTTRate: A Global, Long-Term Observational Study to Evaluate Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy”

20 Apr, 2026 TRITON-PN Phase 3 Study Design and Rationale Presented at the 2026 Annual American Academy of Neurology (AAN) Meeting

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.

Obici, et al. “Design and Rationale of TRITON-PN, a Phase 3 Study to Evaluate the Efficacy of Nucresiran in Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy.”

30 Mar, 2026 New Analyses from Amyloidosis and Hypertension Programs Presented at The American College of Cardiology (ACC) Scientific Sessions 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.

Cuddy, et al. “Post Hoc Analysis of Diastolic Dysfunction in the HELIOS-B Study of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy”

DiDomenico, et al. “Polypharmacy and Potential for Drug–Drug Interactions Among Patients With ATTR-CM Initiating Tafamidis”

Hamatani, et al. “Impact of Vutrisiran on the Components of Quality of Life in Transthyretin Cardiomyopathy (ATTR-CM)”

Hefferman, et al. “Real-World Adherence and Persistence With Vutrisiran, an RNA Interference Therapeutic for the Treatment of Transthyretin Amyloidosis”

Witteles, et al. “Vutrisiran Reduces the Risk of Developing Advanced Disease and Demonstrates Benefit in Patients Who Do Develop Advanced Disease in ATTR-CM: Analysis From the HELIOS-B Study”

Desai, et al. “Pooled Safety Analysis of Zilebesiran, an Investigational Long-Acting RNA Interference, From Phase 2 Studies in Patients With Hypertension”

24 Mar, 2026 Alnylam TTR Investor Webinar 2026

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.

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26 Feb, 2026 Biomarker Approach, Preclinical Data, and Phase 1 Study Overview of ALN-HTT02 in Adult Patients with Huntington’s Disease, Presented at the CHDI Foundation’s Annual Huntington’s Disease Therapeutics Conference

Data on emerging assays measuring huntingtin (HTT) protein in human cerebrospinal fluid were presented at the CHDI Foundation’s 21^st 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”