Alnylam to Present New Data Reinforcing Strength in RNAi-Powered TTR Silencing and Continued Progress in Hypertension at ESC Congress 2026
Key Terms
rna interference technical
sirna technical
mrna technical
renin-angiotensin-aldosterone system medical
“We're excited to share new data at this year's ESC Congress that reinforce our leadership in transthyretin-mediated amyloidosis and advance our hypertension program," said Yvonne Greenstreet, M.D., Chief Executive Officer of Alnylam. "HELIOS-B remains the foundation of our approach to ATTR-CM, and the multiple presentations we're sharing this week reflect the continued depth of that program alongside the progress we're making in hypertension."
The data being presented at ESC further characterize treatment outcomes with AMVUTTRA across ATTR manifestations, patient populations and treatment settings. Highlights include a Late-Breaking Science presentation of pre-specified analyses from the HELIOS-B Phase 3 study of vutrisiran evaluating treatment outcomes in patients on background stabilizer therapy. Additional HELIOS-B analyses examine healthy aging and functional capacity, safety and treatment outcomes by sex. Amylo'ExTTRa, a large real-world analysis from the French National Health Data System, further characterizes the multisystem burden of ATTR-CM beyond cardiac manifestations.
The Company will also present new data from its hypertension program, including a subgroup analysis of the KARDIA-3 Phase 2 study evaluating zilebesiran in combination with a diuretic in patients with uncontrolled hypertension. Zilebesiran is an investigational RNAi therapeutic with the potential to provide continuous control of blood pressure with biannual dosing and is being evaluated in the Phase 3 cardiovascular (CV) outcomes trial, ZENITH.
The ESC abstracts are available here. Alnylam may share additional data and information during the Congress through its Investors website and/or its Capella site.
ESC Congress Presentation Details
ATTR Abstracts
Amylo'ExTTRa: Exploring Extra-Cardiac Manifestations in Patients with ATTR-CM Using a French National Health Data System
Abstract Session: Aetiology, Comorbidities, and Outcomes in Heart Failure
Time: Friday, August 28, 2026, 1:27 – 1:39 pm (CEST), 7:27 – 7:39 am (EDT)
Presenting Author: Thibaud Damy,
Room: Science Box 3 – Research Gateway Hall A1
Vutrisiran is Associated with Preservation of Intrinsic Capacity and Healthy Ageing in Patients with Transthyretin Amyloidosis with Cardiomyopathy: Post Hoc Analysis of HELIOS-B
Abstract Session: Approaching Heart Failure Therapy From all Angles
Time: Sunday, August 30, 2026, 8:15 – 8:25 am (CEST), 2:15 – 2:25 am (EDT)
Presenting Author: Olivier Lairez,
Room: Science Box 3 – Research Gateway Hall A1
Fewer Adverse Events Reported in ATTR-CM Patients Treated with Vutrisiran Versus Placebo: Post Hoc Safety Analysis from HELIOS-B
Moderated Poster: Heart Failure Redefined: Hypertrophic Cardiomyopathy and Transthyretin Amyloid Cardiomyopathy Precision Treatments
Time: Sunday, August 30, 2026, 9:15 – 9:21 am (CEST), 3:15 – 3:21 am (EDT)
Presenting Author: Stéphanie Schwarting,
Room: Station 5 – Research Gateway Hall A1
Effect of Vutrisiran According to Baseline Tafamidis Use in Transthyretin Amyloidosis with Cardiomyopathy
Late-Breaking Clinical Science: Cardiac Amyloidosis
Time: Sunday, August 30, 2026, 2:15 – 2:30 pm (CEST), 8:15 – 8:30 am (EDT)
Presenting Author: Yasuhiro Hamatani,
Room:
Cardiac Presentation and Treatment Response by Sex in Patients with Transthyretin Amyloidosis: Pooled Analysis of Vutrisiran and Patisiran Phase 3 Studies
Abstract Session: Improving Care in Cardiac Amyloidosis
Time: Monday, August 31, 2026, 10:00 – 10:10 am (CEST), 4:00 – 4:10 am (EDT)
Presenting Author: Amira Zaroui,
Room: Science Box 3 – Research Gateway Hall A1
Hypertension Abstract
Zilebesiran in Combination with a Diuretic in Patients with Uncontrolled Hypertension: A Subgroup Analysis from the Phase 2 KARDIA-3 Trial
Abstract Session: Treatment of Hypertension
Time: Sunday, August 30, 2026, 8:15 – 8:25 am (CEST), 2:15 – 2:25 am (EDT)
Presenting Author: Neha Pagidipati,
Room: Science Box 4 – Research Gateway Hall A1
AMVUTTRA® (vutrisiran) INDICATIONS AND IMPORTANT SAFETY INFORMATION
Indications
In the EU, AMVUTTRA® (vutrisiran) is indicated for the treatment of:
- hereditary transthyretin amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy (hATTR-PN).
- wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
Availability across the EU is subject to local reimbursement timelines.
Important Safety Information
Reduced Serum Vitamin A Levels and Recommended Supplementation
Vutrisiran treatment can lower serum vitamin A levels, therefore supplementation of approximately, but not exceeding, 2500 IU to 3000 IU vitamin A per day is advised for patients.
Adverse Reactions
Commonly reported adverse reactions with vutrisiran were injection site reactions and increase in blood alkaline phosphatase and alanine transaminase.
For additional information about vutrisiran, please see the full Summary of Product Characteristics.
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). 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 Zilebesiran
Zilebesiran is an investigational, subcutaneously administered RNAi therapeutic in development for cardiovascular (CV) risk reduction in hypertensive patients at high risk or with established CVD. Zilebesiran targets angiotensinogen (AGT), the most upstream precursor in the renin-angiotensin-aldosterone system (RAAS), which plays a role in blood pressure (BP) regulation and impacts CV and renal health. Clinical trial results have shown the potential for zilebesiran to provide continuous control of BP with biannual dosing in a broad population of patients with hypertension. Zilebesiran is being evaluated in a Phase 3 CV outcomes trial, ZENITH, which will assess its ability to reduce the risk of CV death, nonfatal myocardial infarction, nonfatal stroke, or heart failure events in patients with hypertension and established or at high risk of CVD, despite the use of at least two or more antihypertensives. The safety and efficacy of zilebesiran have not been established or evaluated by the FDA, EMA, or any other health authority. Zilebesiran is being co-developed and co-commercialized by Alnylam and Roche.
About Cardiovascular Disease and Hypertension
Cardiovascular disease (CVD) is a global health crisis and a leading cause of death worldwide, responsible for approximately 20 million deaths annually. Hypertension is the primary cause of and number one modifiable risk factor for CVD. An estimated one in three adults worldwide have hypertension, and despite wide availability of antihypertensives, up to
About RNAi
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. Alnylam routinely posts information that may be important to investors in the “Investors” section of its website at https://investors.alnylam.com/. Investors and potential investors are encouraged to consult the Alnylam website regularly.
Alnylam Forward-Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects including, without limitation, statements regarding the potential for zilebesiran to provide continuous control of blood pressure with biannual dosing and to be an innovative treatment approach for hypertension; and Alnylam’s ability to execute on its Alnylam 2030 strategy to accelerate innovation and scale impact to transform human health, should be considered forward-looking statements. 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; 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 2025 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. 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)
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Josh Brodsky
(Investors)
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Source: Alnylam Pharmaceuticals, Inc.