{"id":61276,"date":"2025-05-17T13:02:49","date_gmt":"2025-05-17T11:02:49","guid":{"rendered":"https:\/\/pharma-trend.com\/en\/amvuttra-vutrisiran-significantly-reduces-mortality-and-a-range-of-important-cardiovascular-events-in-patients-with-attr-amyloidosis-with-cardiomyopathy-additional-data-from-helios-b\/"},"modified":"2025-05-17T13:02:49","modified_gmt":"2025-05-17T11:02:49","slug":"amvuttra-vutrisiran-significantly-reduces-mortality-and-a-range-of-important-cardiovascular-events-in-patients-with-attr-amyloidosis-with-cardiomyopathy-additional-data-from-helios-b","status":"publish","type":"post","link":"https:\/\/pharma-trend.com\/en\/amvuttra-vutrisiran-significantly-reduces-mortality-and-a-range-of-important-cardiovascular-events-in-patients-with-attr-amyloidosis-with-cardiomyopathy-additional-data-from-helios-b\/","title":{"rendered":"AMVUTTRA\u00ae (vutrisiran) Significantly Reduces Mortality and a Range of Important Cardiovascular Events in Patients with ATTR Amyloidosis with Cardiomyopathy: Additional Data from HELIOS-B"},"content":{"rendered":"
\n

\n\u2013 Analysis Presented at the Heart Failure 2025 Congress Supports Primary Findings, Highlighting Impact of AMVUTTRA, which Delivers Rapid Knockdown of Transthyretin \u2013<\/i>\n<\/p>\n

\n\u2013 Additional 42-Month Data Reinforce Vutrisiran\u2019s Impact on the Risk of All-Cause Mortality and Further Underscore the Effect on Cardiovascular Mortality \u2013<\/i>\n<\/p>\n

\n\u2013 Vutrisiran Demonstrated Substantial Benefit Across a Range of Cardiovascular Events, Notably Reducing Urgent Heart Failure Visits by 46% in the Overall Patient Population During the Double-Blind Period \u2013<\/i>\n<\/p>\n

\n\u2013 Findings Simultaneously Published in JACC \u2013<\/i>\n<\/p>\n

\n\u2013 Details of TRITON-CM Phase 3 Study Evaluating Alnylam\u2019s Investigational Next-Generation TTR Silencer, Nucresiran, to be Presented at Congress \u2013<\/i>\n<\/p>\n

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc.<\/a> (Nasdaq: ALNY), the leading RNAi therapeutics company, today presented the most contemporary analysis of the HELIOS-B Phase 3 study of vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM) as a late-breaking abstract at the Heart Failure 2025 Congress, a scientific congress of the European Society of Cardiology, taking place May 17-20 in Belgrade, Serbia. The results demonstrate that vutrisiran, which rapidly knocks down transthyretin, reduces key cardiovascular (CV) events such as CV hospitalizations, and heart failure (HF) hospitalizations. Additionally, in the analysis, urgent HF visits were reduced by 46% (95% CI: 0.30, 0.98; p = 0.041) in the overall population during the double-blind period, compared to placebo. These CV events often precede all-cause mortality (ACM) and are key indicators of disease progression.\n<\/p>\n

<\/a>
<\/a><\/p>\n

\nImportantly, results from the November 2024 data cut, including further follow up through up to 42 months, reinforce the primary HELIOS-B analysis showing vutrisiran\u2019s effect on ACM, and further demonstrate that vutrisiran reduces CV mortality. Through 42 months, the risk of ACM was reduced by 36% (95% CI: 0.46, 0.88; p = 0.007) and the risk of CV mortality was reduced by 33% (95% CI: 0.47, 0.96; p = 0.038) in the overall population, compared to placebo. For both the primary analysis and the current analysis, vital status through 42 months was ascertained for over 99% of all randomized patients from the HELIOS-B study, underscoring the robustness of the results.\n<\/p>\n

\nThe study was conducted in a contemporary patient population with patients receiving robust background therapy, inclusive of treatment with a TTR stabilizer and SGLT2 inhibitors. The analysis of the HELIOS-B Phase 3 study, including mortality data through up to 42 months, was simultaneously published in <\/i>JACC<\/i><\/a>.\n<\/p>\n

\n\u201cFrom the primary analysis of HELIOS-B, we know that AMVUTTRA profoundly impacts all-cause mortality, while preserving patients\u2019 functional capacity and quality of life,\u201d said Pushkal Garg, M.D., Chief Medical Officer of Alnylam. \u201cThese new data\u2014including the impact on mortality, on cardiovascular events and on urgent heart failure visits, the latter of which was reduced by nearly half\u2014add to the story of consistency and magnitude of benefit. I remain impressed by the HELIOS-B results, which are noteworthy given the substantial use of heart failure treatments in the study population, and I believe they continue to reinforce AMVUTTRA as a clinically differentiated, first-line option for patients with ATTR-CM.\u201d\n<\/p>\n

\nThe results from the analysis underscore the rapid and sustained benefits of vutrisiran in treating ATTR-CM across key endpoints:\n<\/p>\n\n\n\n\n\n\n\n\n
\n

\nImpact of Vutrisiran Versus Placebo\n<\/p>\n<\/td>\n

\n

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

\n

\nReduction in Urgent HF Visits through 36 Months\n<\/p>\n<\/td>\n

\n

\n46%\n<\/p>\n

\nRR: 0.54\n<\/p>\n

\nP = 0.041\n<\/p>\n<\/td>\n<\/tr>\n

\n

\nReduction in HF Hospitalizations through 36 Months\n<\/p>\n<\/td>\n

\n

\n33%\n<\/p>\n

\nRR: 0.67\n<\/p>\n

\nP = 0.002\n<\/p>\n<\/td>\n<\/tr>\n

\n

\nReduction in Risk of CV Events through 36 Months\n<\/p>\n<\/td>\n

\n

\n27%\n<\/p>\n

\nRR: 0.73\n<\/p>\n

\nP = 0.001\n<\/p>\n<\/td>\n<\/tr>\n

\n

\nReduction in CV Hospitalizations through 36 Months\n<\/p>\n<\/td>\n

\n

\n25%\n<\/p>\n

\nRR: 0.75\n<\/p>\n

\nP = 0.004\n<\/p>\n<\/td>\n<\/tr>\n

\n

\nReduction in Risk of ACM through 42 Months\n<\/p>\n<\/td>\n

\n

\n36%\n<\/p>\n

\nHR: 0.64\n<\/p>\n

\nP = 0.007\n<\/p>\n<\/td>\n<\/tr>\n

\n

\nReduction in CV Mortality through 42 Months\n<\/p>\n<\/td>\n

\n

\n33%\n<\/p>\n

\nHR: 0.67\n<\/p>\n

\nP = 0.038\n<\/p>\n<\/td>\n<\/tr>\n<\/table>\n

\nIn a separate presentation on Tuesday, May 20, Alnylam will share findings from a subgroup analysis of HELIOS-B evaluating the impact of vutrisiran on ACM and recurrent CV events among patients identified by investigators as having experienced disease progression while being treated with tafamidis.\n<\/p>\n

\nAlso at the Heart Failure 2025 Congress, Alnylam will present the study design and rationale for TRITON-CM, a Phase 3, randomized, double-blind, study of nucresiran in patients with ATTR-CM. Nucresiran is an investigational next-generation RNAi therapeutic targeting TTR that has been shown to deliver rapid knockdown of TTR greater than 95% with twice-annual dosing in a Phase 1 study. TRITON-CM is an event-driven CV outcomes trial with a primary endpoint of composite ACM and CV events. The study is on track to initiate in the first half of 2025 and will enroll approximately 1,200 patients with wild-type or variant TTR and confirmed cardiomyopathy, including those receiving background stabilizer therapy. Additional details of the study\u2019s secondary endpoints and key inclusion and exclusion criteria will be shared on Monday, May 19.\n<\/p>\n

\nAMVUTTRA\u00ae <\/sup>(vutrisiran) was approved by the U.S. Food and Drug Administration (FDA) and the Brazilian Health Regulatory Agency (ANVISA) for treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of vutrisiran for the same indication. A formal regulatory decision by the European Commission of the EMA is expected by the third quarter of 2025. Vutrisiran is currently under review for the treatment of ATTR-CM by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). Alnylam remains on track to proceed with additional global regulatory submissions for vutrisiran in 2025 and beyond.\n<\/p>\n

\nFor additional information on Alnylam\u2019s presentations at the Heart Failure 2025 Congress, please visit Capella<\/a>.\n<\/p>\n

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

\nIndications Approved by the U.S. FDA
\n
<\/b>AMVUTTRA\u00ae<\/sup> (vutrisiran) is indicated for the treatment of the:\n<\/p>\n