Alnylam: Phase 3 Trial Shows AMVUTTRA Delivers Long-Term Heart Benefit In ATTR-CM

Alnylam Pharmaceuticals

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(RTTNews) - Alnylam Pharmaceuticals Inc. (ALNY) announced that new data from the HELIOS-B Phase 3 study, presented at the ESC Congress 2025, demonstrated the long-term cardiovascular benefit of AMVUTTRA (vutrisiran) in adults with cardiomyopathy due to wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM).

The data reflect outcomes of treatment through up to 48 months, including the initial double-blind period of 33-36 months, and highlight the ongoing clinical benefit of vutrisiran, which causes rapid knockdown of the disease-causing transthyretin (TTR) protein, including a 37% risk reduction in the composite endpoint of all-cause mortality (ACM) or first cardiovascular (CV) event in the overall population and a 42% risk reduction in the monotherapy group, reinforcing vutrisiran's potential as a first-line treatment for patients with ATTR-CM. Vutrisiran also reduced the risk of ACM alone by 37% in the overall population and 39% in the monotherapy group during this same period.

The findings from the ongoing HELIOS-B study showed that the clinical benefits seen during the double-blind period of 33-36 months across key clinical measures of disease progression, including quality of life (QoL) as measured by the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) and cardiac biomarkers, were maintained with continued vutrisiran treatment during the 12-month OLE period. The long-term safety and tolerability profile during the OLE period was consistent with the established profile of vutrisiran.

Additionally, a post hoc analysis of the HELIOS-B trial presented during a poster session highlighted reductions in days lost to death and/or hospitalization (DLDH) and improvements in functional and QoL outcomes compared to placebo. Vutrisiran was associated with a reduction in mean DLDH of more than one month versus placebo over a three-year period, with a greater effect observed when accounting for impaired function and quality of life.

The data from the HELIOS-B study supported the recent approvals of AMVUTTRA for the treatment of the cardiomyopathy of wild-type or hereditary ATTR-CM in adults in the United States (US), Brazil, European Union (EU), Japan, United Arab Emirates (UAE) and United Kingdom (UK).

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