Alnylam Pharmaceuticals announced results from new analyses of its HELIOS-B Phase 3 trial evaluating AMVUTTRA (vutrisiran) in adults with transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM). The data, presented at the European Society of Cardiology (ESC) Congress 2025 in Madrid, Spain, covered up to 48 months of treatment.
The study included a 12-month follow-up from the open-label extension (OLE) period. Researchers observed continued clinical benefits with vutrisiran treatment.
The analysis showed a substantial reduction in the risk of death or a first cardiovascular event. This benefit was consistent across the overall study population and within a subgroup receiving vutrisiran as monotherapy.
Positive effects were also sustained across other key measures. Quality of life, as measured by the Kansas City Cardiomyopathy Questionnaire, remained improved. Cardiac biomarkers also showed continued positive trends. The long-term safety profile remained consistent with previous findings.
John Vest, Alnylam‘s Senior Vice President and TTR Global Clinical Lead, highlighted the robust evidence supporting vutrisiran‘s clinical profile. He emphasized the sustained benefits across key endpoints, including survival rates, cardiovascular events, and quality of life, supporting its potential as a first-line therapy.
Pablo Garcia-Pavia, Head of the Inherited Cardiac Diseases and Heart Failure Unit at Hospital Universitario Puerta de Hierro in Madrid, noted the importance of the long-term data in managing ATTR-CM. He highlighted the irreversible nature of disease progression and the sustained benefits of vutrisiran, supporting early treatment initiation. He also observed that the study population included patients receiving standard-of-care therapies.
A post-hoc analysis further examined the impact of vutrisiran on days lost to death and/or hospitalization (DLDH). Vutrisiran was associated with significantly fewer DLDH days compared to placebo. This benefit was particularly notable when considering impaired well-being.
The results from HELIOS-B underpinned the recent approvals of AMVUTTRA for ATTR-CM in several countries, including the United States, Brazil, the European Union, Japan, the United Arab Emirates, and the United Kingdom. AMVUTTRA has accumulated a substantial amount of patient-treatment experience globally.
AMVUTTRA is the first RNAi therapeutic approved for both ATTR-CM and the polyneuropathy form of hereditary transthyretin-mediated amyloidosis (hATTR-PN). It’s administered quarterly via subcutaneous injection.
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