{"id":1018918,"date":"2026-11-09T10:55:43","date_gmt":"2026-11-09T15:55:43","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/alnylam-announces-five-year-helios-b-data-demonstrating-sustained-benefit-of-amvuttra-vutrisiran-in-attr-cm\/"},"modified":"2026-11-09T10:55:43","modified_gmt":"2026-11-09T15:55:43","slug":"alnylam-announces-five-year-helios-b-data-demonstrating-sustained-benefit-of-amvuttra-vutrisiran-in-attr-cm","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/alnylam-announces-five-year-helios-b-data-demonstrating-sustained-benefit-of-amvuttra-vutrisiran-in-attr-cm\/","title":{"rendered":"Alnylam Announces Five-Year HELIOS-B Data Demonstrating Sustained Benefit of AMVUTTRA\u00ae (vutrisiran) in ATTR-CM"},"content":{"rendered":"

<\/p>\n

Alnylam Announces Five-Year HELIOS-B Data Demonstrating Sustained Benefit of AMVUTTRA\u00ae (vutrisiran) in ATTR-CM<\/b><\/p>\n

\u2013 AMVUTTRA Provided Sustained Benefit Across Mortality, Cardiovascular Events, Quality of Life and Cardiac Biomarkers Over 60 Months, with a Consistent Safety Profile \u2013<\/i><\/p>\n

\u2013 Benefit of Earlier TTR Silencing Compared to Delayed Start in Placebo-Treated Patients Underscores Importance of Early Treatment Initiation \u2013<\/i><\/p>\n

\u2013 Cardiopulmonary Exercise Test Results Demonstrate AMVUTTRA Preserved Exercise Capacity and Cardiac and Ventilatory Function \u2013<\/i><\/p>\n

CAMBRIDGE, Mass.–(BUSINESS WIRE<\/a>)–Alnylam Pharmaceuticals, Inc<\/a>. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced new data at the American Heart Association (AHA) Scientific Sessions 2026, adding to the robust body of evidence demonstrating the strength of RNAi-powered TTR silencing with AMVUTTRA\u00ae<\/sup> (vutrisiran). The analyses, including data from the HELIOS-B Phase 3 two-year open-label extension (OLE) period, show that patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) treated continuously with AMVUTTRA had lower rates of the composite of all-cause mortality and recurrent cardiovascular events, better-preserved quality of life and greater stability of cardiac biomarkers through five years than patients who initiated treatment with AMVUTTRA approximately three years later. A separate post hoc analysis of HELIOS-B provides new physiologic insight into the effects of AMVUTTRA during exercise, demonstrating preserved exercise capacity and favorable effects across multiple components of the cardiopulmonary response to exercise compared with placebo.<\/p>\n

\n\u201cThe sustained treatment effects observed with AMVUTTRA across multiple measures of cardiovascular health during the five years of HELIOS-B strengthen the evidence that early and continuous TTR silencing at the source can change the course of a rapidly progressive disease. Together with new insights into the effects of AMVUTTRA on the cardiopulmonary response to exercise, these findings underscore the importance of treating patients early, when preserving cardiac function and slowing disease progression may have the greatest impact,\u201d said Pushkal Garg, M.D., Chief Research and Development Officer at Alnylam. \u201cThese data reinforce the strength of TTR silencing as a foundational approach to ATTR-CM care, and show the potential of RNAi therapeutics to translate deep, sustained target suppression into meaningful long-term outcomes for patients.\u201d<\/p>\n

Vutrisiran Demonstrated Significant Sustained Cardiovascular and Quality of Life Benefits through Five Years<\/b><\/p>\n

\nIn the HELIOS-B Phase 3 study, patients initially randomized to vutrisiran in the double-blind period were eligible to continue treatment during the OLE period, and patients initially randomized to placebo were eligible to transition to vutrisiran, enabling assessment of outcomes with continuous versus delayed treatment initiation through 60 months. Pre-specified analyses were conducted in both the monotherapy population not receiving tafamidis at baseline and the overall study population.<\/p>\n

\nAcross the monotherapy and overall populations, patients initially randomized to vutrisiran experienced more favorable long-term outcomes than patients who initiated vutrisiran during the OLE period, reinforcing the importance of initiating TTR silencing early in the course of disease. Continuous vutrisiran treatment was associated with significant and durable reductions in the composite of all-cause mortality (ACM) and recurrent cardiovascular (CV) events compared with those who initially received placebo and transitioned to vutrisiran during the OLE period, with a 48% lower risk in the monotherapy population and a 41% lower risk in the overall population. The risk of recurrent CV events was also significantly lower with continuous treatment with vutrisiran, decreasing by 50% in the monotherapy population and 41% in the overall population. Patients who initiated vutrisiran earlier also maintained better quality of life, as measured by the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) score, and showed greater stability of cardiac biomarker levels, including NT-proBNP and troponin I, compared with delayed treatment initiation.<\/p>\n\n\n\n\n\n\n\n
\n

\nEndpoint<\/p>\n<\/td>\n

\n

\nMonotherapy Population (n = 395)<\/p>\n

\n\u00a0<\/p>\n<\/td>\n

\n

\nOverall Population (n = 654)<\/p>\n<\/td>\n<\/tr>\n

\n

\nRisk of ACM and<\/p>\n

\nRecurrent CV<\/p>\n

\nEvents<\/p>\n<\/td>\n

\n

\nRelative reduction: 48%<\/p>\n

\nHR: 0.515 (95% CI: 0.368, 0.722)<\/p>\n

\np=0.0001<\/p>\n

\n\u00a0<\/p>\n<\/td>\n

\n

\nRelative reduction: 41%<\/p>\n

\nHR: 0.589 (95% CI: 0.459, 0.756)<\/p>\n

\np<0.0001<\/p>\n<\/td>\n<\/tr>\n

\n

\nRisk of ACM or<\/p>\n

\nFirst CV Event<\/p>\n<\/td>\n

\n

\nRelative reduction: 48%<\/p>\n

\nHR: 0.523 (95% CI: 0.401, 0.681)<\/p>\n

\np<0.0001<\/p>\n

\n\u00a0<\/p>\n<\/td>\n

\n

\nRelative reduction: 39%<\/p>\n

\nHR: 0.614 (95% CI: 0.498, 0.756)<\/p>\n

\np<0.0001<\/p>\n<\/td>\n<\/tr>\n

\n

\nRisk of Recurrent<\/p>\n

\nCV Events<\/p>\n<\/td>\n

\n

\nRelative reduction: 50%<\/p>\n

\nRR: 0.501 (95% CI: 0.410, 0.611)<\/p>\n

\np<0.0001<\/p>\n

\n\u00a0<\/p>\n<\/td>\n

\n

\nRelative reduction: 41%<\/p>\n

\nRR: 0.587 (95% CI: 0.502, 0.685)<\/p>\n

\np<0.0001<\/p>\n

\n\u00a0<\/p>\n<\/td>\n<\/tr>\n

\n

\nLS Mean Difference<\/p>\n

\nin Change from<\/p>\n

\nBaseline in KCCQ-OS<\/p>\n

\nScore<\/p>\n<\/td>\n

\n

\nLS mean difference: 11.66 (SEM 2.65) points<\/p>\n

\np<0.0001<\/p>\n

\n\u00a0<\/p>\n<\/td>\n

\n

\nLS mean difference: 9.68 (SEM 1.96) points<\/p>\n

\np<0.0001<\/p>\n<\/td>\n<\/tr>\n

\n

P values are nominal and are presented without adjustment for multiplicity.<\/i><\/p>\n<\/td>\n<\/tr>\n<\/table>\n

\nAmong patients receiving tafamidis at baseline, continuous treatment with vutrisiran and tafamidis was associated with a 23% reduction in recurrent CV events (RR: 0.766; 95% CI: 0.597, 0.984; p=0.0366) relative to patients who initially received tafamidis alone and initiated vutrisiran during the OLE. The safety profile of vutrisiran through 60 months was consistent with its known safety profile, with no new safety signals identified.<\/p>\n

\n\u201cATTR-CM is a progressive disease, making it critical to intervene before patients experience further irreversible decline. These long-term HELIOS-B data are particularly compelling because they demonstrate that patients treated continuously with vutrisiran experienced more favorable outcomes than those who initiated treatment later,\u201d said Sarah Cuddy, M.D., Brigham and Women’s Hospital and Harvard Medical School. \u201cThese findings reinforce the importance of considering TTR silencing early in the course of disease to preserve patients\u2019 health and quality of life for as long as possible.\u201d<\/p>\n

HELIOS-B Post Hoc Analysis Provides New Insight into the Cardiopulmonary Response to Exercise<\/b><\/p>\n

\nExercise intolerance is a hallmark of ATTR-CM and can progressively limit patients\u2019 daily function as the disease advances. A post hoc, single-center analysis of 48 patients enrolled in HELIOS-B who underwent cardiopulmonary exercise testing (CPET) with concurrent stress echocardiography at the National Amyloidosis Centre in London showed that vutrisiran preserved multiple components of the cardiopulmonary response to exercise over two years compared with placebo. Vutrisiran preserved exercise capacity, as measured by peak oxygen uptake (VO\u2082), with an LS mean difference of 1.57 mL\/kg\/min versus placebo (95% CI: 0.14 to 3.00; p=0.031). Vutrisiran also preserved chronotropic response and improved ventilatory efficiency, while stress echocardiography showed favorable changes in measures of right ventricular systolic reserve and left ventricular diastolic reserve. No patients received other ATTR-CM disease-modifying therapies during the study period. Together, these findings provide new physiologic insight into how TTR silencing with vutrisiran may help preserve the cardiovascular system\u2019s ability to respond to the demands of exercise in patients with ATTR-CM.<\/p>\n

\nTo view Alnylam\u2019s AHA Scientific Sessions 2026 presentations, please visit Capella<\/a>.<\/p>\n

Indications and Important Safety Information<\/b><\/p>\n

Indications Approved by the U.S. FDA<\/b><\/p>\n

\nAMVUTTRA\u00ae <\/sup>(vutrisiran) is indicated for the treatment of the:<\/p>\n