Alnylam Pharmaceuticals presented new HELIOS-B analyses at Heart Failure 2026 showing vutrisiran delivered consistent clinical benefits across complex ATTR-CM patient populations, including patients with atrial fibrillation, low systolic blood pressure, and those receiving multiple background heart failure therapies.
Written by: Chikkula Pavan Kumar, PharmD
Reviewed By: Pharmacally Editorial Team
Alnylam Pharmaceuticals announced new analyses from the Phase 3 HELIOS-B study (NCT04153149) at Heart Failure 2026, demonstrating consistent clinical benefits of vutrisiran across diverse transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM) patient populations, including patients with high disease burden and complex clinical profiles. The Phase 3 HELIOS-B study previously demonstrated that vutrisiran significantly reduced all-cause mortality and recurrent cardiovascular events in patients with ATTR-CM, including both wild-type and hereditary forms of the disease.
The newly presented analyses showed that vutrisiran demonstrated consistent clinical benefits across several clinically important subgroups, including reductions in all-cause mortality and recurrent cardiovascular events. Benefits were observed among patients with atrial fibrillation, low systolic blood pressure, and those receiving standard background heart failure therapies, including SGLT2 inhibitors, beta-blockers, mineralocorticoid receptor antagonists, ACE inhibitors, ARBs, ARNIs, and transthyretin stabilizers such as tafamidis.
According to the findings, nearly 65% of patients enrolled in HELIOS-B had atrial fibrillation, a population commonly associated with more advanced ATTR-CM and poorer clinical outcomes. Vutrisiran demonstrated meaningful clinical benefit compared with placebo in these patients. Consistent treatment effects were also observed among patients with low systolic blood pressure, another population associated with more severe disease, with vutrisiran slowing the progressive decline in systolic blood pressure observed over time. Clinical benefits were also maintained regardless of comorbidity burden or concomitant use of disease-modifying therapies. Consistent effects were similarly observed in women, a historically underrepresented population in ATTR-CM clinical trials.
“These analyses reinforce the consistency and clinical relevance of vutrisiran across complex ATTR-CM patient populations encountered in real-world practice,” said Pushkal Garg, M.D., Chief Medical Officer at Alnylam Pharmaceuticals. “The findings continue to support vutrisiran as a potential first-line treatment option for a broad range of patients living with ATTR-CM.”
Alnylam Pharmaceuticals also presented pooled safety data representing more than 25,000 patient-years of experience with transthyretin-silencing RNA interference (RNAi) therapeutics. The analyses demonstrated a well-characterized and consistent safety profile, with rates of ocular adverse events potentially associated with vitamin A deficiency remaining low and comparable to placebo. No clinically meaningful vitamin A deficiency was observed.
In addition, the company highlighted progress from the ongoing DemonsTTRate study, a large global real-world evidence initiative evaluating outcomes in more than 2,000 patients with ATTR-CM. The study is designed to provide additional insights into disease progression, treatment patterns, healthcare utilization, and long-term clinical outcomes in routine clinical practice.
ATTR-CM is a progressive and often underdiagnosed disease caused by the accumulation of misfolded transthyretin protein in the heart, leading to worsening cardiac dysfunction and heart failure. Vutrisiran is an RNAi therapeutic designed to silence hepatic transthyretin production through RNA interference, enabling rapid and sustained reduction of transthyretin levels.
The updated HELIOS-B analyses further strengthen the growing body of evidence supporting vutrisiran’s efficacy, durability, and safety, highlighting its potential as a foundational therapy for patients living with ATTR-CM across diverse clinical settings.
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About the Writer
Chikkula Pavan Kumar (LinkedIn), PharmD is a Doctor of Pharmacy with a keen interest in clinical pharmacy, pharmacovigilance, and evidence-based practice. In his words, he is passionate about patient safety and translating complex medical information into clear, research-driven communication.