Alnylam to Present Five-Year HELIOS-B Data and Expanding Evidence on the Benefits of RNAi-Powered TTR Silencing with AMVUTTRA® (vutrisiran) at AHA and ISA 2026
The upcoming analyses address long-term outcomes, exercise performance and neuropathy-related quality of life with vutrisiran.
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– Late-Breaking Presentation at AHA Will Feature Long-Term Results from the HELIOS-B Phase 3 Study, Including Two Years of Open-Label Extension Data in Patients with ATTR-CM –
– HELIOS-B Post Hoc Analysis Demonstrates Positive Impact of AMVUTTRA in Preserving Exercise Capacity and Cardiopulmonary Performance –
“Alnylam continues to pioneer TTR silencing through RNAi innovation, with an expanding evidence base that reinforces the potential of AMVUTTRA to transform ATTR care,” said Yvonne Greenstreet, M.D., Chief Executive Officer of Alnylam. “The landmark HELIOS-B study established a strong foundation for AMVUTTRA, and the research being presented at AHA and ISA reflects our determination to build on this progress and drive the next chapter in the treatment of this progressive disease.”
A highlight of the AHA program will be a late-breaking presentation of five-year results from the HELIOS-B Phase 3 study, including two years of open-label extension data evaluating long-term outcomes with vutrisiran in patients with the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM). The findings will provide additional insights into the long-term clinical effects of RNAi-powered TTR silencing, including outcomes among patients receiving continuous vutrisiran treatment and those who initially received placebo before switching to vutrisiran.
A second late-breaking presentation of cardiopulmonary exercise testing (CPET) with imaging of 48 patients enrolled in HELIOS-B will show that treatment with vutrisiran preserved exercise capacity and cardiopulmonary performance during exercise over two years compared with placebo, providing mechanistic insight into the functional benefits of TTR silencing in ATTR-CM. An additional real-world evidence analysis of sex-related differences in ATTR may inform efforts to improve earlier diagnosis.
At ISA, a post hoc analysis of HELIOS-B will highlight statistically significant benefits of vutrisiran versus placebo on neuropathy-related quality-of-life measures among patients with ATTR-CM and evidence of polyneuropathy, further strengthening the body of data supporting TTR silencing across the multisystemic manifestations of ATTR amyloidosis in both hereditary and wild-type ATTR. Separately, real-world data from more than 1,000 patients initiating vutrisiran will demonstrate a higher prevalence of heart failure and greater comorbidity burden among second-line versus first-line initiators, supporting the rationale for initiating vutrisiran earlier in the treatment pathway. In addition, results from an indirect treatment comparison of vutrisiran versus acoramidis in ATTR-CM will be presented.
The AHA Scientific Sessions 2026 regular / non-late breaking abstracts are available here and ISA 2026 abstracts are available here. Alnylam will make the data presentations available on its Capella site.
AHA Scientific Sessions 2026 Presentation Details
Presentation Title |
Presenting Author |
Session Information
|
2-Year Results From the Open-Label Extension Period of the Phase 3 HELIOS-B Study of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy |
Sarah Cuddy |
Featured Science
|
The Effect of Vutrisiran on Exercise Capacity and Ventilatory Efficiency: Insights From the HELIOS-B CPET and Stress Echocardiography Subgroup Analysis |
Brinda Thillainathan |
Featured Science
|
Sex Differences at Diagnosis Among Patients With Hereditary or Wild-Type Transthyretin Amyloidosis: Analysis From the ConTTRibute Study |
Zubair Shah |
Abstract Poster Board Session
10:30 am - 11:30 am CT
|
ISA 2026 Presentation Details
Presentation Title |
Presenting Author |
Session Information
|
Treatment Effect of Vutrisiran on Measures of Polyneuropathy in HELIOS-B |
Yoshiki Sekijima |
Unmoderated Poster #242
|
Real-World Characteristics and Treatment Patterns Among New Vutrisiran Patients With ATTR-CM |
Sumeet Mitter |
Unmoderated Poster #209
|
Efficacy and Safety of Vutrisiran in South American Patients With Transthyretin Amyloidosis With Cardiomyopathy: Post Hoc Analysis of HELIOS-B |
Juan Costabel |
Unmoderated Poster #368
|
Comparative Efficacy of Vutrisiran and Acoramidis in ATTR-CM: A Matching-Adjusted Indirect Comparison |
Ronald Witteles |
Unmoderated Poster #526
|
Real-World Adherence and Persistence of Eplontersen in Patients with ATTR |
Sumeet Mitter |
Unmoderated Poster #569
|
AMVUTTRA® (vutrisiran) INDICATIONS AND IMPORTANT SAFETY INFORMATION
Indications Approved by the
AMVUTTRA® (vutrisiran) is indicated for the treatment of the:
- cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits.
- polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.
Important Safety Information
Reduced Serum Vitamin A Levels and Recommended Supplementation
AMVUTTRA treatment leads to a decrease in serum vitamin A levels.
Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking AMVUTTRA. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with AMVUTTRA, as serum vitamin A levels do not reflect the total vitamin A in the body.
Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).
Adverse Reactions
In a study of patients with hATTR-PN, the most common adverse reactions that occurred in patients treated with AMVUTTRA were pain in extremity (
In a study of patients with ATTR-CM, no new safety issues were identified.
For additional information about AMVUTTRA, please see the full
The therapeutic indications described above are those approved by the
About AMVUTTRA® (vutrisiran)
AMVUTTRA® (vutrisiran) demonstrates strength in RNAi-powered transthyretin (TTR) silencing, delivering rapid knockdown of TTR at the source of disease to address the underlying cause of transthyretin amyloidosis (ATTR). As ATTR is a rapidly progressive disease, even when patients may appear clinically stable, early treatment intervention is critical. In the HELIOS-B Phase 3 study, AMVUTTRA reduced the risk of all-cause mortality and recurrent CV events compared to placebo in the overall and monotherapy populations by
About Transthyretin Amyloidosis (ATTR)
Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating, and fatal disease caused by pathogenic transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart, and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant. It is estimated that more than 500,000 people worldwide live with ATTR, with ~
About RNAi Technology
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.
About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) is a leading global biopharmaceutical company and the pioneer of the RNA interference (RNAi) revolution. The Company is focused on developing transformative therapies with the potential to prevent, halt, or reverse disease. For more than two decades, Alnylam has advanced the Nobel-Prize-winning science of RNAi, delivering critical breakthroughs and six approved medicines. Alnylam has medicines available in more than 70 countries and a rapidly expanding and robust pipeline, in addition to consistently being recognized as an exceptional workplace and socially responsible organization. The Company is executing on its Alnylam 2030 strategy to accelerate innovation and scale impact to transform human health. For more information, please visit www.alnylam.com or follow Alnylam on X, LinkedIn, Facebook, Instagram, or YouTube.
Alnylam Forward-Looking Statements
This press release contains forward-looking statements. Forward-looking statements include statements regarding Alnylam’s expectations, beliefs, goals, plans or prospects, including, without limitation, statements regarding: Alnylam’s ability to pioneer TTR silencing through RNAi innovation; the potential of AMVUTTRA to transform ATTR care; Alnylam’s ability to build on its progress and drive the next chapter in the treatment of ATTR; the potential for the real-world evidence analysis of sex-related differences in ATTR to inform efforts to improve earlier diagnosis; and Alnylam’s ability to achieve the goals in its Alnylam 2030 strategy. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: Alnylam’s ability to successfully execute on its Alnylam 2030 strategy; Alnylam’s ability to successfully launch, market and sell Alnylam’s approved products globally, including AMVUTTRA; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; delays, interruptions or failures in the manufacture and supply of Alnylam’s marketed products or its product candidates; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products; the outcome of litigation and government investigations; the risk of future litigation and government investigations; and unexpected expenditures; as well as those risks and uncertainties more fully discussed in the “Risk Factors” filed with Alnylam’s most recent periodic report (Quarterly Report on Form10-Q or Annual Report on Form 10-K) filed with the SEC and in its other SEC filings. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.
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Alnylam Pharmaceuticals, Inc.
Sarah D’Souza
(Media)
Media@alnylam.com
Josh Brodsky
(Investors)
Investors@alnylam.com
Source: Alnylam Pharmaceuticals, Inc.