New Data Supports Benefits of Vutrisiran for ATTR-CM and Zilebesiran for Hypertension

Analyses Demonstrate Vutrisiran Improves Quality of Life and Outcomes Across ATTR-CM Spectrum, While Zilebesiran Shows Promising Safety Profile

Mar. 31, 2026 at 3:24am

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Alnylam Pharmaceuticals presented new data at the American College of Cardiology's Annual Scientific Session reinforcing the potential of its RNAi therapeutics to deliver differentiated and durable impact for patients with cardiovascular disease. The analyses highlighted the benefits of vutrisiran, the first and only transthyretin (TTR) silencer approved for ATTR-CM and hATTR-PN, as well as the safety profile of zilebesiran, an investigational RNAi therapy being evaluated for hypertension.

Why it matters

These findings provide important evidence to help inform treatment decisions for ATTR-CM patients, a rare and debilitating disease with limited treatment options. Additionally, the data on zilebesiran support its continued evaluation in a global Phase 3 trial for patients with hypertension and high cardiovascular risk, an area of significant unmet need.

The details

The new vutrisiran analyses expand on the HELIOS-B study, demonstrating improvements in patients' health-related quality of life, consistent efficacy across disease severity, and high real-world treatment adherence. Vutrisiran was shown to reduce the risk of progression to advanced disease and improve outcomes in those who did progress. Separately, pooled Phase 2 data reinforced the safety profile of zilebesiran, an RNAi therapy targeting angiotensinogen to provide continuous blood pressure control.

  • The HELIOS-B study and real-world data on vutrisiran were presented at ACC.26 in March 2026.
  • The ZENITH global Phase 3 trial evaluating zilebesiran in hypertension was initiated in September 2025.

The players

Alnylam Pharmaceuticals

The leading RNAi therapeutics company that developed vutrisiran and zilebesiran.

Ronald Witteles, M.D.

HELIOS-B Investigator and Professor of Medicine at Stanford University School of Medicine, Co-Director of the Stanford Amyloid Center.

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What they’re saying

“These new data present further evidence of the meaningful impact vutrisiran offers patients with ATTR‑CM and provide deeper insight into its benefits for patients with more advanced disease.”

— Ronald Witteles, M.D., HELIOS-B Investigator, Professor of Medicine at Stanford University School of Medicine and Co-Director of the Stanford Amyloid Center

What’s next

The judge in the ZENITH trial will decide in September 2026 whether to allow zilebesiran to advance to Phase 3 based on the Phase 2 safety data.

The takeaway

These findings highlight the potential of Alnylam's RNAi therapies to address significant unmet needs in cardiovascular disease, from improving quality of life and outcomes for ATTR-CM patients to providing a new approach to managing hypertension and reducing cardiovascular risk.