− Achieved Second Quarter 2026 Global Net Product Revenues of$1,172 Million(74% Growth Compared with Q2 2025), Driven Primarily by Total TTR Revenues of$1,030 Million(89% Growth Compared with Q2 2025) –
− Revises Full-Year 2026 TTR Net Product Revenue Guidance from$4,400to$4,700 Millionto$4,200to$4,500 Million(75% Growth Compared with 2025 at Revised Midpoint) –
− Presented New Data from HELIOS-B at Heart Failure 2026 Demonstrating Vutrisiran's Consistent Clinical Benefit Across Patient Populations –
− Advanced Pipeline with Phase 2 Initiations of ALN-6400 in Von Willebrand Disease and Mivelsiran in Down Syndrome-Associated Alzheimer's Disease; Results from Phase 1 Trial of ALN-HTT02 in Patients withHuntington'sDisease to be Presented at EHDN –
− Accelerated Integration of AI Across Alnylam by Establishing Strategic Collaborations with Inceptive to Transform RNAi Discovery and aLarge Health Care SysteminCaliforniato Support Earlier Identification of ATTR-CM in Routine Care, as well asExpanding PartnershipwithKomodo Healthto Scale Commercial Intelligence –
− Entered Into an Exclusive Agreement with BeOne Medicines for Commercialization of AMVUTTRA inChina–
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jul. 30, 2026-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the second quarter endedJune 30, 2026, and reviewed recent business highlights.
“During the first half of 2026, we continued to meaningfully advance our business, generating over$1 billionin quarterly product revenues for the first time in our history during the first quarter and, building on that momentum, over$1 billionin TTR revenues during the second quarter. These results underscore the growing leadership and global impact of our TTR franchise in transforming outcomes for patients with ATTR amyloidosis, with AMVUTTRA being the only product approved for the full spectrum of the disease. We have lowered our TTR product sales guidance for full-year 2026 to reflect learnings from the initial phase of our launch in the evolving ATTR-CM market, in particular the normalization of growth in second line volume after satisfying pent-up demand from patients waiting for a new therapy. Given the strong foundation we have established and continued growth in ATTR-CM diagnosis and treatment, we remain confident in the trajectory of our ongoing ATTR-CM launch and are continuing to invest robustly in this franchise, as we bring AMVUTTRA to more patients and establish it as a foundational therapy,” saidYvonne Greenstreet, M.D., Chief Executive Officer ofAlnylam. “During the second quarter, we also continued to advance our high-value pipeline with the initiation of two Phase 2 studies, ALN-6400 in von Willebrand disease and mivelsiran in Down syndrome-associated Alzheimer’s disease, while progressing multiple additional programs toward important clinical readouts later this year. Together, these achievements demonstrate our continued progress against our Alnylam2030 strategy and our commitment to creating long-term value through scientific innovation and patient impact.”
Second Quarter 2026 and Recent Significant Business Highlights
Total TTR: AMVUTTRA® (vutrisiran) & ONPATTRO® (patisiran)
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Achieved global net product revenues for AMVUTTRA and ONPATTRO for the second quarter of$1,012 million and $18 million, respectively, together representing $1,030 million in total TTR net product revenues and 89% total TTR growth compared to Q2 2025.
U.S. TTR net product revenues increased $106 million compared with Q1 2026 with the growth driven by a $129 million increase in demand, partially offset by approximately $20 million in inventory impact and a modest reduction in net price. The growth in demand in the quarter was more than double the growth in demand in Q1 2026 compared with Q4 2025.
International TTR net product revenues increased $14 million compared with Q1 2026 driven primarily by increased demand in both hATTR-PN and ATTR-CM across international markets.
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Announced a collaboration with a large health care system in California to support their study, DETECT-ATTR, evaluating Invision Precision Cardiac Amyloid, an AI-enabled, FDA-cleared, echocardiography-based screening approach for the detection of cardiac amyloidosis. DETECT-ATTR will be conducted within one of the nation's largest integrated healthcare systems, serving more than 4.5 million members, with the intention of addressing underdiagnosis and improving disease recognition in clinical practice.
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Presented new analyses from the HELIOS-B Phase 3 clinical trial of vutrisiran in patients with ATTR-CM at Heart Failure 2026, the annual congress of the Heart Failure Association of the European Society of Cardiology:
Reductions in all-cause mortality and recurrent cardiovascular events were maintained across key subgroups of patients taking a broad range of heart failure therapies.
A pooled analysis of over 25,000 patient-years of experience with TTR-silencing RNAi therapies shows a consistent safety profile, including no clinical meaningful ocular effects of vitamin A lowering.
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Shared the design and rationale of the DemonsTTRate study, a global, prospective, observational study evaluating real-world outcomes in patients with ATTR-CM. The study is expected to enroll more than 2,000 patients and follow them for up to five years, generating longitudinal data on clinical outcomes, treatment patterns and healthcare utilization across routine clinical practice.
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Continued to expand the global reach of AMVUTTRA with a recent launch in Spain, and a new commercial partnership with BeOne Medicines to distribute AMVUTTRA in mainland China and Macao, subject to AMVUTTRA receiving marketing authorization.
Total Rare: GIVLAARI® (givosiran) & OXLUMO® (lumasiran)
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Achieved global net product revenues for GIVLAARI and OXLUMO for the second quarter of $90 million and $52 million , respectively, together representing $142 million in total Rare net product revenues and 11% total Rare growth compared to Q2 2025.
Other Highlights
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Initiated a Phase 2 clinical trial of ALN-6400, an investigational RNAi therapeutic targeting plasminogen, in adult and adolescent female patients with von Willebrand disease and heavy menstrual bleeding.
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Initiated a Phase 1 clinical trial of ALN-6222, an investigational RNAi therapeutic targeting inhibin E (INHBE), in adult patients with obesity.
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Advanced mivelsiran, an investigational RNAi therapeutic targeting amyloid precursor protein (APP) for the treatment of cerebral amyloid angiopathy (CAA) and Alzheimer’s disease.
Completed enrollment in the cAPPricorn-1 Phase 2 clinical trial in patients with CAA.
Initiated a Phase 2 clinical trial in patients with Down syndrome-associated Alzheimer's disease.
Shared additional Phase 1 data in early-onset Alzheimer's disease at the Alzheimer's
Association International Conference
(AAIC) 2026. An analysis of safety data from single- and multiple-doses of mivelsiran showed no evidence of increased risk of amyloid-related imaging abnormality (ARIA) events. Results also showed robust, durable reductions in cerebrospinal fluid (CSF) soluble amyloid beta precursor protein (sAPPβ) and amyloid beta 42 (Aβ42), with up to 30 months of treatment exposure. The most common adverse events (AEs) were procedural pain and procedural headache, and no serious or severe AEs were deemed related to study drug.
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Presented preclinical data and Phase 1 design details at AAIC 2026 for ALN-5288, an investigational RNAi therapeutic targeting microtubule-associated protein tau (MAPT) for Alzheimer's disease and tauopathies.
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Our collaboration partner, Regeneron Pharmaceuticals, Inc., announced that the U.S. Food and Drug Administration(FDA) and theEuropean Medicines Agency (EMA) have accepted regulatory applications for cemdisiran to treat adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. Regeneron indicated that the FDA will review the New Drug Application (NDA) under Priority Review with a target action date in November 2026, following use of a Priority Review Voucher, and that a decision from the European Commission is anticipated in the second half of 2027.
Additional Business Updates
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Announced a strategic collaboration agreement with Inceptive Nucleics, Inc. designed to increase the pace of RNAi therapeutic innovation. The alliance pairs Alnylam's RNAi platform and 20+ years of proprietary data with Inceptive's foundation models and AI expertise to catalyze progress beyond rational drug design.
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Expanded the Company's strategic partnership with Komodo Health to scale Marmot, Komodo's analytics AI platform, across key enterprise functions at Alnylam.
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Appointed Benjamin Franklin Cravatt III, Ph.D., to Alnylam's Board of Directors.
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Published 2025 Corporate Responsibility Report.
Key Upcoming Events
Alnylamannounces today that it will present initial results from the Phase 1 clinical trial of ALN-HTT02 in patients withHuntington'sdisease at the European Huntington'sDisease Network (EHDN) Clinical Research CongressonFriday, October 23, 2026, in Kraków,Poland.
The Company continues to host its 10th RNAi Roundtable series this year, during which Alnylam R&D leaders, as well as medical thought leaders, will discuss the progress and opportunity across key pipeline programs of investigational RNAi therapeutics. Upcoming RNAi Roundtables include:
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Zilebesiran: Targeting Angiotensinogen to Achieve Continuous Control of Blood Pressure Thursday, September 17,10:30 am ET
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ALN-HTT02: Targeting Exon 1 of the Huntington Gene to Reduce Progression of Huntington's Disease Monday, October 26 ,10:00 am ET
In the second half of 2026,Alnylamexpects to announce clinical data from additional pipeline programs, including:
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Results from Phase 1 and Phase 2 clinical trials of ALN-6400 in healthy volunteers and patients with hereditary hemorrhagic telangiectasia (HHT), respectively.
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Results from a Phase 1 clinical trial of ALN-2232 in obesity and weight management.
Second Quarter 2026 Financial Results
For an explanation of our use of non-GAAP financial measures, refer to the “Use of Non-GAAP Financial Measures” section later in this press release and for a reconciliation of each non-GAAP financial measure to the most comparable GAAP measure, see the tables at the end of this press release.
Revenue Summary
Total Net Product Revenues
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Total net product revenues increased 74%, both at actual currency and at CER during the three months ended June 30, 2026, compared to the same period in 2025. The increase was primarily due to growth from AMVUTTRA revenues driven by increased patient demand, mainly in patients with ATTR-CM in the U.S., and growth from an increased number of patients on GIVLAARI and OXLUMO, which was partially offset by a decreased number of patients on ONPATTRO.
Net Revenues from Collaborations
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Net revenues from collaborations decreased during the three months ended June 30, 2026, as compared to the same period in 2025, due to lower revenue recognized under our Regeneron collaboration, partially offset by increased revenue under our Roche collaboration driven by higher reimbursable development activities related to the ZENITH Phase 3 clinical trial of zilebesiran.
Royalty Revenue
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Royalty revenue increased during the three months ended June 30, 2026, as compared to the same period in 2025, due to increased volume and rate of royalties earned from global net sales of Leqvio by Novartis.
Other Financial Highlights Interest expense
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Interest expense for the three months ended June 30, 2026 of $82 million included interest of $53 million attributed to the liability related to the sale of future Leqvio royalties and $26 million attributed to the liabilities related to the vutrisiran and zilebesiran development funding.
Provision for income taxes
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During the three months ended June 30, 2026, we recorded a provision for income taxes of $13 million, primarily related toU.S. state income taxes, utilization of Switzerland net deferred tax assets, as well as taxable income from jurisdictions in which we are subject to tax. We will utilize deferred tax assets in Switzerland to offset current cash tax liabilities and will continue to maintain a full valuation allowance against our net deferred tax assets in the U.S . and certain deferred tax assets in Switzerland.
Financial position
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Cash, cash equivalents and marketable securities were $3.3 billion as of June 30, 2026 , as compared to $2.9 billion as of December 31, 2025 , with the increase primarily driven by net cash inflows from operating activities. Net cash provided by operating activities for the three months ended June 30, 2026 included $26 million of payments associated with the liability related to the sale of future Leqvio royalties recorded to interest expense, as well as $33 million of payments associated with the liabilities related to vutrisiran and zilebesiran development funding recorded to interest expense.
A reconciliation of our GAAP to non-GAAP financial results is included in the tables at the end of this press release.
2026 Financial Guidance
Full-year 2026 financial guidance is updated and consists of the following:
The change in the Company’s TTR net product revenue guidance reflects an updated outlook for AMVUTTRA in the second line segment of the U.S. market based on learnings as the ATTR-CM launch has progressed. Specifically, growth in second line demand for AMVUTTRA moderated in early 2026 to what the Company now believes is a normalized level, following an early launch period that, with hindsight, benefited from pent-up demand from patients progressing on stabilizers who had been waiting for a new treatment option.
About AMVUTTRA® (vutrisiran)
AMVUTTRA® (vutrisiran) is a transthyretin (TTR) silencer that delivers rapid knockdown of TTR at the source to address the underlying cause of transthyretin amyloidosis (ATTR). In a clinical study, AMVUTTRA rapidly knocked down TTR in as early as six weeks and decreased TTR levels by 87% with two and a half years of treatment. It is approved as a treatment for the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and for the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults in various countries, globally. Administered quarterly via subcutaneous injection, AMVUTTRA is the first and only silencer approved for the treatment of ATTR-CM and hATTR-PN. For more information about AMVUTTRA, including the fullU.S. Prescribing Information, visit AMVUTTRA.com.
About ONPATTRO® (patisiran)
ONPATTRO is an RNAi therapeutic that is approved inthe United StatesandCanadafor the treatment of adults with hATTR amyloidosis with polyneuropathy. ONPATTRO is also approved in the European Union,SwitzerlandandBrazilfor the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and inJapanfor the treatment of hATTR amyloidosis with polyneuropathy. ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR). It is designed to target and silence TTR messenger RNA, thereby reducing the production of TTR protein before it is made. Reducing the pathogenic protein leads to a reduction in amyloid deposits in tissues. For more information about ONPATTRO, including full Prescribing Information, visit ONPATTRO.com.
About GIVLAARI® (givosiran)
GIVLAARI (givosiran) is an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) approved inthe United StatesandBrazilfor the treatment of adults with acute hepatic porphyria (AHP). GIVLAARI is also approved in the European Union for the treatment of AHP in adults and adolescents aged 12 years and older. In the pivotal trial, GIVLAARI was shown to significantly reduce the rate of porphyria attacks that required hospitalizations, urgent healthcare visits or intravenous hemin administration at home compared to placebo. GIVLAARI is Alnylam’s first commercially available therapeutic based on its Enhanced Stabilization Chemistry ESC-GalNAc conjugate technology to increase potency and durability. GIVLAARI is administered via subcutaneous injection once monthly at a dose based on actual body weight and should be administered by a healthcare professional. GIVLAARI works by specifically reducing elevated levels of ALAS1 messenger RNA (mRNA), leading to reduction of toxins associated with attacks and other disease manifestations of AHP. For more information about GIVLAARI, including the fullU.S. Prescribing Information, visit GIVLAARI.com.
About OXLUMO® (lumasiran)
OXLUMO (lumasiran) is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1). HAO1 encodes glycolate oxidase (GO). Thus, by silencing HAO1 and depleting the GO enzyme, OXLUMO inhibits production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1. OXLUMO utilizes Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. OXLUMO has received regulatory approvals from theU.S. Food and Drug Administration(FDA) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pediatric and adult patients and from theEuropean Medicines Agency(EMA) for the treatment of PH1 in all age groups. In the pivotal ILLUMINATE-A trial, OXLUMO was shown to significantly reduce levels of urinary oxalate relative to placebo, with the majority of patients reaching normal or near-normal levels. In the ILLUMINATE-B pediatric Phase 3 trial, OXLUMO demonstrated an efficacy and safety profile consistent to that observed in ILLUMINATE-A. In the ILLUMINATE-C trial, OXLUMO resulted in substantial reductions in plasma oxalate in patients with advanced PH1. Across all three studies, injection site reactions (ISRs) were the most common drug-related adverse reaction. OXLUMO is administered via subcutaneous injection once monthly for three months, then once quarterly beginning one month after the last loading dose at a dose based on actual body weight. For patients who weigh less than 10 kg, ongoing dosing remains monthly. OXLUMO should be administered by a healthcare professional. For more information about OXLUMO, including the fullU.S. Prescribing Information, visit OXLUMO.com.
About LNP Technology
Alnylamhas licenses to Arbutus Biopharma lipid nanoparticle (LNP) intellectual property for use in RNAi therapeutic products using LNP technology.
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.