RegulationPriority Review (United States), Breakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (Australia)

Structure/Sequence

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Sequence Code 29732340

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Sequence Code 29732483

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Clinical Trials associated with Patisiran sodium

ISRCTN13564930

/ Active, not recruitingPhase 1

A phase I monotherapy study to evaluate the safety, tolerability and immunogenicity of a candidate next generation lipid nanoparticle (LNP) mRNA vaccine against SARS-CoV-2 (NeomiVac) in UK healthy adult volunteers

Start Date15 May 2024

Sponsor / Collaborator

Neovac Ltd.

NCT05023889

/ CompletedEarly Phase 1

Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy

To evaluate the efficacy and safety of patisiran in patients with wtATTR amyloidosis and symptomatic polyneuropathy by evaluating the effect on neurologic impairment and quality of life.

Start Date03 Aug 2022

Sponsor / Collaborator

Austin Neuromuscular Center

[+1]

NCT04201418

/ CompletedNot Applicable

A Phase 4 Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of Hereditary Transthyretin-Mediated (ATTRv) Amyloidosis With a V122I or T60A Mutation

To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.

Start Date18 Dec 2019

Sponsor / Collaborator

Alnylam Pharmaceuticals, Inc.

100 Clinical Results associated with Patisiran sodium

100 Translational Medicine associated with Patisiran sodium

100 Patents (Medical) associated with Patisiran sodium

217

Literatures (Medical) associated with Patisiran sodium

01 Aug 2026·HEART

Ribonucleic acid and gene therapies in cardiovascular disease: clinical applications, delivery challenges and emerging precision tools

Review

Author: Andrew Baker ; Neil Craig ; David E Newby ; Marc Richard Dweck ; Neeraj Dhaun ; Craig Balmforth ; Alex J Jordan

Cardiovascular diseases remain a leading cause of global mortality despite advancements in pharmacotherapies, with current treatments facing challenges related to efficacy, tolerability and patient adherence. In response, advanced therapies, such as RNA and gene therapies, have emerged as a promising alternative for addressing both acquired and monogenic cardiovascular conditions. This review explores the current landscape of RNA and gene therapies for cardiovascular disease, focusing on RNA-based therapeutics such as small-interfering RNAs (siRNAs), antisense oligonucleotides and clustered regularly interspaced short palindromic repeats and associated Cas9 endonuclease (CRISPR-Cas9)-based gene editing systems. Recent European Medicines Agency and Food and Drug Administration-approved RNA therapies, including patisiran, vutrisiran and inclisiran, which employ lipid nanoparticle delivery systems, highlight the clinical potential of siRNAs for targeting hepatic molecular pathways. Emerging CRISPR-Cas9 technologies are poised to address genetic mutations at their source, offering permanent correction of pathogenic variants and the potential to treat a broad range of hereditary cardiovascular conditions. Together, these therapies represent a major leap forward in precision medicine, offering long-lasting therapeutic effects and improved patient care and adherence. However, many challenges remain, particularly in targeting such therapies to cardiac tissues and optimising delivery systems. This review discusses the current state of the art in cardiovascular RNA and gene therapies, including current evidence, delivery challenges and the current landscape of gene and RNA therapies in phase I clinical trials and beyond.

03 Jul 2026·CURRENT GENE THERAPY

Advancing siRNA Therapeutics with Pharmacometrics: A Review of Modeling Approaches and Clinical Applications

Article

Author: Jeong, Hyunsuk ; Yim, Hyeonwoo ; Han, Seunghoon ; Han, Sungpil ; Kang, Jueun ; Choi, Suein

Small interfering RNA (siRNA) therapeutics represent a paradigm shift in targeting previously undruggable diseases through specific gene silencing. Since the FDA's first approval of patisiran in 2018, the field has expanded rapidly, with eight approved drugs and numerous clinical candidates. However, the atypical pharmacokinetic-pharmacodynamic (PK-PD) profile of siRNAs, characterized by rapid plasma elimination, prolonged tissue retention, and temporal dissociation between exposure and effect, has created distinct obstacles for conventional dose-finding strategies. This review examines how model-informed drug development, particularly pharmacometric modeling, has emerged as an essential tool in the advancement of siRNA therapeutics. Population PK-PD models for all FDA-approved siRNA drugs are systematically described, demonstrating that mechanistic and semi-mechanistic approaches inform preclinical-to-clinical translation, optimize dosing regimens, and support regulatory decisions. Key modeling frameworks include: (1) mechanistic models incorporating asialoglycoprotein receptor (ASGPR)-mediated uptake, RNA-induced silencing complex (RISC) loading, and mRNA degradation kinetics; (2) minimal physiologically-based PK-PD models for interspecies scaling; and (3) exposure-response models addressing the disconnect between plasma PK and pharmacological activity. This review also discusses how quantitative pharmacology addresses developmental challenges, including first-in-human dose selection, optimal dosing intervals, organ impairment effects, and interindividual variability. This pharmacometric perspective provides a quantitative framework for rational development of safe and effective siRNA therapeutics across diverse patient populations.

01 Jun 2026·Revista espanola de cardiologia (English ed.)

Multicenter cohort analysis of cardiac amyloidosis patients treated with heart transplant

Article

Author: Allinovi, Marco ; Garofalo, Manuel ; Sheikh, Awais ; Sinigiani, Giulio ; Limongelli, Giuseppe ; Argirò, Alessia ; Aloisio, Alessio ; Fontana, Marianna ; de Frutos, Fernando ; Porcari, Aldostefano ; Tini, Giacomo ; García-Pavía, Pablo ; Alqabani, Mohammad ; Colio, Federica ; Saturi, Giulia ; Gillmore, Julian ; Cattadori, Barbara ; Potena, Luciano ; Palladini, Giovanni ; Palmiero, Giuseppe ; Gasperetti, Alessio ; Urey, Marcus Anthony ; Pieroni, Maurizio ; Musumeci, Beatrice ; Longhi, Simone ; Perfetto, Federico ; Milani, Paolo ; Merlo, Marco ; Chen, Liang ; Gerosa, Gino ; Bui, Quan M ; Masri, Ahmad ; Chen, Zhongli ; González-Costello, José ; Di Nora, Concetta ; Cipriani, Alberto ; Sinagra, Gianfranco ; Gómez Bueno, Manuel ; Imazio, Massimo ; Vendramin, Igor ; Thornton, Caitlin ; Moody, William E ; Menale, Silvia ; Cappelli, Francesco ; Herrador, Lorena

INTRODUCTION AND OBJECTIVES:

To describe the clinical characteristics of patients with cardiac amyloidosis who have undergone heart transplantation (HT).

METHODS:

This retrospective multicenter study involving 14 referral centers included 113 patients with cardiac amyloidosis who underwent HT: 57 with transthyretin amyloidosis (ATTR) (22 with wild-type ATTR and 35 with variant ATTR) and 56 with light chain amyloidosis (AL).

RESULTS:

Compared with patients with ATTR, patients with AL amyloidosis showed more severe hemodynamic compromise before HT, with a lower cardiac index (1.7 L/min/m² [interquartile range, 1.4-2.0 L/min/m²] vs 1.9 L/min/m² [interquartile range, 1.6-2.4 L/min/m²]; P = .027) and higher right atrial pressure. Post-transplant infections occurred in 23 patients with ATTR (40%) and 24 with AL (43%). New-onset neuropathy occurred in 11% of patients, while neuropathy progression was more frequent in ATTR compared with AL (23% vs 7.1%; P = .044). Median follow-up was 4.6 years in ATTR and 5.4 years in AL. Five-year survival was similar in both groups (77% in AL vs 83% in ATTR). Sepsis was the leading cause of death (n = 11, 29% of deaths). Among patients with ATTR, 9 received tafamidis before HT and 7 after HT. Eight patients with ATTR received patisiran post-HT (1 pre-HT), and 3 received diflunisal post-HT. In AL, 70% received bortezomib-based therapy while 28% received daratumumab-based therapy. In AL, hematologic responses improved after HT, with the complete response rate increasing from 42% before HT to 54% after HT.

CONCLUSIONS:

HT is an effective treatment for carefully selected patients with cardiac amyloidosis, with comparable unadjusted survival between AL and ATTR. These findings warrant confirmation in prospective studies.

371

News (Medical) associated with Patisiran sodium

14 Sep 2026

·www.biospace.com

Slate Medicines Appoints Kenneth Koblan, PhD, as Chief Scientific Officer

RALEIGH, N.C., Sept. 14, 2026 (GLOBE NEWSWIRE) -- Slate Medicines Inc. (“Slate”), a clinical-stage biotechnology company committed to advancing innovative new therapies that may broaden the treatment paradigm and potentially improve outcomes for people with migraine and other headache disorders, today announced the appointment of Kenneth Koblan, PhD, as Chief Scientific Officer. Dr. Koblan brings more than three decades of experience in drug discovery and development, with deep expertise in neuroscience, translational medicine and novel therapeutics spanning multiple modalities. “We are pleased to welcome Dr. Koblan as Chief Scientific Officer as we continue to expand our leadership team,” said Gregory Oakes, MBA, Chief Executive Officer of Slate Medicines. “Ken’s ability to bridge discovery innovation and development excellence across multiple successful products and novel platforms demonstrates his deep understanding of what it takes to build and advance a world-class pipeline. His expertise in neuroscience and translational medicine will be particularly valuable as we advance SLTE-1009 and build our pipeline of innovative therapies for patients with migraine and other headache disorders.” Dr. Koblan is a distinguished biopharmaceutical R&D leader with more than 30 years of experience across drug discovery and development. Prior to joining Slate, he served as Head of Research and Development at Vesalius Therapeutics. Previously, he spent 13 years at Sumitomo Pharma America (formerly Sunovion), where he served as Chief Scientific Officer and Head of Development Operations. There, he pioneered the use of induced pluripotent stem cells (iPSCs) for rare disease modeling and cell-based therapeutics and led development programs for novel central nervous system medicines. Earlier in his career, Dr. Koblan held senior scientific leadership positions at Merck Research Laboratories and Alnylam Pharmaceuticals. At Merck, his work contributed to the development of important medicines including GARDASIL ® , JANUVIA ® and BELSOMRA ® , the first approved orexin receptor antagonist. He also led the discovery of telcagepant and its backup compounds UBRELVY ® and QULIPTA ® , establishing clinical proof of concept for oral CGRP antagonism and helping pioneer the gepant class for migraine. At Alnylam, where he served as Chief Scientific Officer, he led the company’s “5x15” rare disease initiative and contributed to the development of ONPATTRO ® , the first approved RNA-interference medicine. Dr. Koblan was elected to the American College of Neuropsychopharmacology in 2022 and has authored more than 100 peer-reviewed publications and 192 patent applications. He holds a PhD from Johns Hopkins University and a BS from the Massachusetts Institute of Technology. “I’ve spent my career advancing new therapeutic approaches from emerging biology toward medicines that can meaningfully change patient care,” said Dr. Koblan. “Slate’s work targeting the PACAP/VIP pathway represents an exciting opportunity to build on our understanding of neuropeptide biology and potentially advance the treatment of migraine beyond what is possible today. I look forward to working with the team to advance SLTE-1009 and build a pipeline of innovative medicines for patients with migraine and other headache disorders.” About SLTE-1009 SLTE-1009 is a potential best-in-class monoclonal antibody in development for the preventive treatment of migraine. SLTE-1009 is designed to bind both PACAP and VIP, two neuropeptides with foundational roles in migraine pathophysiology, which may offer the potential for enhanced efficacy relative to PACAP-only targeting therapeutics through more complete neutralization of the PACAP/VIP pathway. SLTE-1009 was engineered with half-life extension to enable subcutaneous dosing and the potential for quarterly administration. About Slate Medicines, Inc. Slate Medicines is a clinical-stage biotechnology company committed to advancing innovative new therapies that may broaden the treatment paradigm and potentially improve outcomes for people with migraine and other headache disorders. Slate is advancing a portfolio of potentially best-in-class therapeutics, led by SLTE-1009, a clinical-stage anti-PACAP/VIP monoclonal antibody designed to enable subcutaneous, infrequent dosing. For more information, please visit the Slate Medicines website. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “anticipates,” “believes,” “expects,” “intends,” “projects,” “plans,” and “future” or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of SLTE-1009 and Slate’s other product candidates to expand treatment options for migraine and other headache disorders and Slate’s research, development and regulatory plans for its product candidates. Forward-looking statements are based on management’s current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation, risks with respect to: the ability of Slate to retain, attract and hire key personnel; the potential failure to identify additional product candidates and develop or commercialize marketable products; the early stage of Slate’s development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the regulatory approval process; interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; Slate’s product candidates may cause serious adverse side effects; inability to maintain existing or future collaborations, or the failure of these collaborations; Slate’s reliance on third parties, including for the manufacture of materials for research programs, preclinical and clinical studies; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; securities class action litigation; the impact of general economic conditions on their respective business and operations, including Slate’s preclinical studies and clinical trials; the possibility of system failures or security breaches; and risks relating to intellectual property. These forward-looking statements speak only as of the date hereof, and Slate disclaims any obligation to update these statements except as may be required by law. Media and Investor Contacts ICR Healthcare slate@icrhealthcare.com

Executive ChangeDrug Approval

30 Aug 2026

·www.businesswire.com

Alnylam Presents New Data at ESC Congress 2026 Reinforcing Strength in RNAi-Powered TTR Silencing Across ATTR-CM Patient Populations and Treatment Settings

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced new data at the European Society of Cardiology (ESC) Congress 2026 demonstrating the strength of RNAi-powered silencing for cardiovascular disease. The findings further reinforce the clinical profile of AMVUTTRA® (vutrisiran) across transthyretin amyloidosis (ATTR) patient populations, treatment settings, and manifestations of disease. Additionally, the data expand the potential application of RNAi to uncontrolled hypertension, the world’s leading cause of cardiovascular disease. “With the power of our RNAi therapeutics platform, we have the potential to make a transformational impact on cardiovascular care,” said Pushkal Garg, M.D., Chief Research and Development Officer at Alnylam. “The data presented at ESC demonstrate the consistency of clinical outcomes achieved by RNAi-powered TTR silencing, reinforcing our conviction in AMVUTTRA as a first-line treatment option for ATTR-CM. With zilebesiran, we have the potential to extend the precision and durability of RNAi to uncontrolled hypertension. Together, these programs reflect our ambition to change the course of cardiovascular disease for patients with high unmet need.” Vutrisiran Analyses HELIOS-B Prespecified Subgroup Analysis Demonstrates Consistent Clinical Benefit with Vutrisiran Across Contemporary ATTR-CM Treatment Settings A late-breaking oral presentation featured a prespecified subgroup analysis of the HELIOS-B Phase 3 clinical trial evaluating the treatment effect of vutrisiran according to baseline tafamidis use. The results were simultaneously published in the Journal of the American College of Cardiology. Among 654 randomized and treated patients in HELIOS-B, 259 patients (40%) were receiving tafamidis at baseline. The treatment effect for vutrisiran on the primary composite endpoint of all-cause mortality and recurrent cardiovascular events through 33-36 months was consistent irrespective of baseline tafamidis use, suggesting clinical benefits across broad patient populations, including those receiving stabilizers. All-cause mortality and additional cardiovascular outcomes showed a similar benefit among patients who were receiving tafamidis at baseline (“combination population”) and those who were not (“monotherapy population”). Across both groups, treatment with vutrisiran preserved functional capacity versus placebo, as measured by the Six-Minute Walk Test. Improvement in health status by vutrisiran versus placebo, as measured by the Kansas City Cardiomyopathy Questionnaire-overall summary score, was observed in both the monotherapy and combination populations with an attenuated effect seen among patients receiving tafamidis at baseline. Safety outcomes were generally similar between combination vutrisiran and tafamidis versus tafamidis alone, and between vutrisiran monotherapy versus placebo. HELIOS-B was not powered to establish the benefit of vutrisiran specifically in the population of patients receiving background tafamidis at baseline. These findings reinforce the impact of vutrisiran across contemporary ATTR-CM treatment settings and warrant further evaluation of TTR silencing and stabilization combination strategies. Additional HELIOS-B Analyses Highlight the Potential Impact of Vutrisiran on the Multisystemic Burden of ATTR-CM Additional analyses presented at ESC further underscore the multisystemic burden of ATTR-CM and the importance of evaluating measures beyond traditional cardiac endpoints. Real-world evidence from the French National Health Data System showed that patients with ATTR-CM had a significantly higher burden of extra-cardiac manifestations across multiple organ systems compared with matched controls, and multiple manifestations were recorded years before ATTR-CM identification and tended to accumulate over time, suggesting a prolonged pre-diagnostic phase with evolving multisystem involvement. A post hoc analysis of HELIOS-B evaluated the impact of treatment with vutrisiran on intrinsic capacity, a composite measure encompassing locomotion, cognition, vitality, psychological well-being and sensory function aligned with the World Health Organization Integrated Care for Older People framework. In the overall study population, compared with placebo, patients treated with vutrisiran demonstrated 25% less decline from baseline intrinsic capacity score and a 52% reduction in the risk of decline, suggesting that treatment with vutrisiran may help preserve functional reserve and support healthy aging in patients with ATTR-CM. A separate post hoc safety analysis of HELIOS-B showed that patients treated with vutrisiran had fewer adverse events overall compared with placebo across the overall study population, monotherapy population and combination population. Among the most frequent system organ classes in the overall population, the lowest adverse event rate ratios were observed for gastrointestinal disorders and nervous system disorders, with 42% and 41% lower adverse event rates, respectively, with vutrisiran compared with placebo; eye disorders showed a 46% lower event rate with vutrisiran compared with placebo. Pooled Phase 3 Data Reinforce Consistent Treatment Effects of RNAi-Powered TTR Silencing Across Sexes A pooled analysis of 1,402 patients (203 females, 1,199 males) across four Phase 3 studies of vutrisiran and patisiran further reinforces the clinical benefits of RNAi-mediated TTR silencing across sexes. Despite sex-specific baseline differences in disease presentation, treatment effects were consistent between females and males across both ATTR-CM and the polyneuropathy of hereditary ATTR (hATTR-PN), including clinical, biomarker, functional, health status and echocardiographic measures. “These data add to the deep and consistent evidence base supporting RNAi-mediated TTR silencing in ATTR-CM,” said Teresa Trenkwalder, M.D., Senior Physician, TUM University Hospital German Heart Center. “Across patient populations, treatment settings, and manifestations of disease, the analyses of vutrisiran demonstrate the clinical benefit that can be achieved by reducing TTR production at its source.” Zilebesiran Analysis The KARDIA-3 Phase 2 study evaluated zilebesiran, an investigational RNAi therapeutic with the potential to provide continuous control of blood pressure (BP) with biannual dosing, in patients with uncontrolled hypertension with high cardiovascular (CV) risk treated with two or more background antihypertensives. In patients who were receiving a background diuretic with an office systolic BP (SBP) ≥140 mmHg at baseline, zilebesiran achieved greater reductions in mean office and 24-hour ambulatory SBP than in the overall study population. Furthermore, patients treated with zilebesiran experienced SBP reductions across the diurnal cycle, including at nighttime. Similar findings were observed in patients who had impaired nocturnal dipping at baseline. These findings are potentially important given the association between elevated nighttime BP and CV risk. The safety profile in this post hoc subgroup was consistent with the broader zilebesiran Phase 2 program. These findings further support the evaluation of zilebesiran in the ongoing global Phase 3 CV outcomes trial, ZENITH. Zilebesiran will be featured as part of Alnylam's 10th “RNAi Roundtable” series on September 17, 2026, at 10:30 a.m. ET. To view Alnylam’s ESC Congress 2026 presentations, please visit Capella. Alnylam may share additional data and information during the Congress through its Investors website and/or Capella. 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. ONPATTRO® (patisiran) INDICATION AND IMPORTANT SAFETY INFORMATION Indication In the EU, ONPATTRO® (patisiran) is indicated for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage 1 or stage 2 polyneuropathy. Important Safety Information Reduced Serum Vitamin A Levels and Recommended Supplementation Patisiran 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 The most common adverse reactions that occurred in patients treated with patisiran were peripheral oedema (30%) and infusion-related reactions (19%). For additional information about patisiran, 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 28.2% and 32.8%, respectively, through 36 months. It is the only TTR silencer approved for both the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) and cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in countries globally. AMVUTTRA is administered once quarterly via subcutaneous injection. 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 ~80% remaining undiagnosed. 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 80% of all patients, and up to one-third of treated patients, do not reach and maintain blood pressure (BP) targets. Even when BP appears well-managed, continuous control of BP may remain suboptimal, leading to variability in BP during the 24-hour period and in the long-term, putting patients at greater risk of cardiovascular events and end organ damage. 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. Forward-looking statements include statements regarding Alnylam’s expectations, beliefs, goals, plans or prospects including, without limitation, statements regarding the potential efficacy or safety of vutrisiran for the treatment of ATTR CM, including in combination with a stabilizer; the potential clinical benefit that can be achieved by reducing TTR production at its source across patient populations, treatment settings and manifestations of disease; the potential for AMVUTTRA to be a first-line treatment for ATTR-CM; the potential for zilebesiran to extend the precision and durability of RNAi, and to provide continuous control of blood pressure with biannual dosing, in patients with uncontrolled hypertension; Alnylam’s ability to make a transformational impact on cardiovascular care and to change the course of cardiovascular disease for patients with high unmet need; and Alnylam’s ability to execute on its Alnylam 2030 strategy to accelerate innovation and scale impact to transform human health. 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 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. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

Clinical ResultPhase 3Phase 2Drug Approval

24 Aug 2026

·www.biospace.com

Alnylam to Present New Data Reinforcing Strength in RNAi-Powered TTR Silencing and Continued Progress in Hypertension at ESC Congress 2026

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc . (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that it will present new clinical and real-world data across its transthyretin-mediated amyloidosis (ATTR) and hypertension programs at the European Society of Cardiology (ESC) Congress 2026, taking place August 28-31, 2026, in Munich, Germany. These presentations add to the existing and growing body of evidence demonstrating strength in RNAi-powered transthyretin (TTR) silencing with AMVUTTRA ® (vutrisiran) and underscore the potential of zilebesiran as an innovative treatment approach for hypertension, the leading preventable risk factor for cardiovascular disease (CVD) worldwide. “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, France 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, France 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, Germany 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, U.S. Room: Ashgabat (Hall A3) 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, France 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, U.S. 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 28.2% and 32.8%, respectively, through 36 months. It is the only TTR silencer approved for both the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) and cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in countries globally. AMVUTTRA is administered once quarterly via subcutaneous injection. 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 ~80% remaining undiagnosed. 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 80% of all patients, and up to one-third of treated patients, do not reach and maintain blood pressure (BP) targets. Even when BP appears well-managed, continuous control of BP may remain suboptimal, leading to variability in BP during the 24-hour period and in the long-term, putting patients at greater risk of cardiovascular events and end organ damage. 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 . 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. Contacts Alnylam Pharmaceuticals, Inc. Sarah D’Souza (Media) Media@alnylam.com Josh Brodsky (Investors) Investors@alnylam.com

Phase 3Clinical ResultDrug ApprovalPhase 2

100 Deals associated with Patisiran sodium

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KEGG	Wiki	ATC	Drug Bank
D10794	Patisiran sodium	N07XX12	DB14582

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Polyneuropathies	European Union	Alnylam Netherlands BV	27 Aug 2018
Polyneuropathies	Iceland	Alnylam Netherlands BV	27 Aug 2018
Polyneuropathies	Liechtenstein	Alnylam Netherlands BV	27 Aug 2018
Polyneuropathies	Norway	Alnylam Netherlands BV	27 Aug 2018
Amyloidosis, Hereditary, Transthyretin-Related	United States	Alnylam Pharmaceuticals, Inc.	10 Aug 2018

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Transthyretin Amyloid Cardiomyopathy	NDA/BLA	United States	Alnylam Pharmaceuticals, Inc.	21 Feb 2023
Amyloid Neuropathies, Familial	Phase 3	United States	Alnylam Pharmaceuticals, Inc.	16 Jul 2015
Amyloid Neuropathies, Familial	Phase 3	Japan	Alnylam Pharmaceuticals, Inc.	16 Jul 2015
Amyloid Neuropathies, Familial	Phase 3	Argentina	Alnylam Pharmaceuticals, Inc.	16 Jul 2015
Amyloid Neuropathies, Familial	Phase 3	Australia	Alnylam Pharmaceuticals, Inc.	16 Jul 2015
Amyloid Neuropathies, Familial	Phase 3	Brazil	Alnylam Pharmaceuticals, Inc.	16 Jul 2015
Amyloid Neuropathies, Familial	Phase 3	Bulgaria	Alnylam Pharmaceuticals, Inc.	16 Jul 2015
Amyloid Neuropathies, Familial	Phase 3	Canada	Alnylam Pharmaceuticals, Inc.	16 Jul 2015
Amyloid Neuropathies, Familial	Phase 3	Cyprus	Alnylam Pharmaceuticals, Inc.	16 Jul 2015
Amyloid Neuropathies, Familial	Phase 3	France	Alnylam Pharmaceuticals, Inc.	16 Jul 2015

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ESC2026	Not Applicable	Transthyretin Amyloid Cardiomyopathy	2,494	Vutrisiran	iozsighoma(pgsbblqsdo) = bhbziwitdg ifotqylxbj (ntotvkpozu, 0.24 - 0.82) View more	Positive	30 Aug 2026
				Tafamidis	ryhqrqmfkn(qwcitvqqes) = avfmezftrf isbfstserb (hlljfnmkcd, 0.43 - 0.82) View more
ESC2026	Not Applicable	Amyloid Neuropathies, Familial	37	Tafamidis 20 mg received before TTR silencer therapy	cfbxdazwvt(gyzepbpbhn) = diaksbpszr bujcrmjojj (xoqrzcgplf ) View more	Positive	11 May 2026
NCT01960348 (APOLLO, Pubmed \| J Formos Med Assoc)	Phase 3	Amyloidosis, Hereditary, Transthyretin-Related A97S gene variant	18	Patisiran 0.3 mg/kg	ihgwvjofnj(uaklomxrum) = qcncwnibtx mvvnzywebi (uwijbvseri, -45.5 to -7.5)	Positive	01 Sep 2024
NCT02510261 (APOLLO, CTgov)	Phase 3	Amyloid Neuropathies, Familial \| Transthyretin-mediated amyloidosis	211	Patisiran (Prior Placebo Group of Study 004)	dufsgssngq = czprwppvna dcdewbdyid (lqgidwrwhb, ainiypjaca - zwvokmehgw) View more	-	06 Dec 2023
				Patisiran (Prior Patisiran Group of Study 004)	dufsgssngq = bqkwiyrjnk dcdewbdyid (lqgidwrwhb, pzwbdbtjnj - ucsntptrjz) View more
NCT03997383 (APOLLO-B, Pubmed \| N Engl J Med)	Phase 3	Transthyretin Amyloid Cardiomyopathy hereditary \| variant \| wild-type	360	Patisiran 0.3 mg/kg	scubjlnnsy(frwkwpqyry) = ijlxozqbls eyqykjaits (lmdglsykwb, lower)	Positive	26 Oct 2023
				Placebo	scubjlnnsy(frwkwpqyry) = hqjsaoduha eyqykjaits (lmdglsykwb, higher)
NCT03997383 (APOLLO-B, Biospace) Manual	Phase 3	Transthyretin Amyloid Cardiomyopathy	360	Patisiran 0.3 mg/kg	juavfzktom(tfqfqhfhte) = gosjzounmu xyroaoaevi (orpnuxlcrt, 0.69-28.69 - P=0.02) Met View more	Positive	25 Oct 2023
NCT03997383 (APOLLO-B, CTgov)	Phase 3	Transthyretin Amyloid Cardiomyopathy	360	Placebo+Patisiran (OLE) (Placebo (DB)/Patisiran (OLE))	xdgvlbrzmq(pcphghcfjt) = fdgynsgzzo bsdqvtrrop (eslscptqod, cmgdiqyunj - xzdqosyxuj) View more	-	18 Oct 2023
				OLE+Patisiran (Patisiran (DB+OLE))	xdgvlbrzmq(pcphghcfjt) = cmbxkjvnly bsdqvtrrop (eslscptqod, vbdvkzovlo - jcswryyizn) View more
NCT03997383 (APOLLO-B, ESC2023)	Phase 3	Amyloidosis transthyretin (TTR) amyloid \| N-terminal prohormone B-type natriuretic peptide \| troponin I ... View more	360	Patisiran	otdmhfjamk(rqwxzrrecl): OR = 1.58 (95% CI, 1.03 - 2.42) View more	-	26 Aug 2023
				Placebo
NCT03997383 (APOLLO-B, Corporate Publications) Manual	Phase 3	Amyloidosis	359	Patisiran sodium	gcisvxkcau(huqbepkrup) = Patisiran demonstrated a benefit or trend toward benefit in change from baseline of most echocardiographic parameters compared with placebo at Month 12 tujijcldrb (zqzemonkgy )	Positive	30 Sep 2022
				Placebo
NCT01960348 \| NCT03759379 (APOLLO, ANA2022)	Phase 3	Polyneuropathies \| Amyloidosis NfL	-	Patisiran	eufdfsndpo(yqaifwdmzz) = fhnayqynkl cfcutumfaa (yjykgqfeet )	Positive	14 Sep 2022
				Vutrisiran	eufdfsndpo(yqaifwdmzz) = zgdkurmjgs cfcutumfaa (yjykgqfeet )

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