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− Achieved First Quarter 2026 Global Net Product Revenues of $1,036 Million (121% Growth Compared with Q1 2025), Driven Primarily by Total TTR Revenues of $910 Million (153% Growth Compared with Q1 2025) – − Reiterates 2026 Financial Guidance, Including Combined Net Product Revenues of $4,900 Million to $5,300 Million, and Total TTR Net Product Revenues of $4,400 to $4,700 Million – − New Data Presented at ACC.26 Further Support Use of Vutrisiran as First-Line Treatment for ATTR-CM – − Established Collaborations with Viz.ai and AHA Focused on Earlier Diagnosis, Coordinated Care and Long-Term Patient Impact in ATTR-CM – − Deep Pipeline Progressing Toward Multiple Clinical Readouts During the Second Half of 2026 Expected to Support Long-Term Growth – CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter ended March 31, 2026, and reviewed recent business highlights. “2026 is off to a strong start for Alnylam with the end of the first quarter marking one year since the U.S. launch of AMVUTTRA for ATTR-CM, and also representing a significant financial milestone with the achievement of over $1 billion in quarterly product revenues for the first time in our history,” said Yvonne Greenstreet, M.D., Chief Executive Officer of Alnylam. “At the same time, we continued to advance our deep and high-value pipeline of RNAi therapeutics, progressing three ongoing Phase 3 trials, and initiating a Phase 1 study for our first adipose-targeted program for obesity and weight management. In addition, multiple programs across our pipeline are advancing toward key clinical readouts this year. Together, this represents important initial progress toward our Alnylam 2030 goals of achieving global TTR leadership, driving growth through innovation, and scaling with financial discipline as we work to deliver substantial patient impact and create long-term value.” First Quarter 2026 and Recent Significant Business Highlights Total TTR: AMVUTTRA ® (vutrisiran) & ONPATTRO ® (patisiran) Achieved global net product revenues for AMVUTTRA and ONPATTRO for the first quarter of $890 million and $20 million, respectively, together representing $910 million in total TTR net product revenues and 153% total TTR growth compared to Q1 2025. Today announced an update to the TRITON-CM Phase 3 study of nucresiran, an investigational next-generation TTR silencer, in patients with ATTR-CM. Enrollment in the study is proceeding faster than anticipated. Consequently, the Company has decided to utilize a pre-specified protocol option to expand target enrollment from 1,250 to approximately 1,750 patients. While Alnylam is increasing the number of patients who will be enrolled in the trial, given the rapid pace of enrollment, the Company still expects to launch nucresiran, assuming positive data and regulatory approval, in ATTR-CM by 2030. Presented new analyses from the HELIOS-B Phase 3 clinical trial of vutrisiran in patients with ATTR-CM at the American College of Cardiology's Annual Scientific Session and Expo. Vutrisiran improved health-related quality-of-life in patients with ATTR-CM relative to placebo, with the treatment effect corresponding to the difference observed in ATTR-CM patients more than 10 years apart in age. Consistent treatment effects of vutrisiran were observed across the ATTR-CM disease spectrum, including patients with the most advanced disease and diastolic dysfunction. Real-world data demonstrated high adherence to and persistence of quarterly HCP-administered dosing with vutrisiran. Announced new strategic efforts aimed at facilitating earlier diagnosis, coordinated care, and long-term patient impact in ATTR-CM. Alnylam is partnering with Viz.ai to develop an AI-enabled ATTR-CM care pathway designed to assist in the identification of patients earlier in the course of disease and guide appropriate diagnostic evaluation and referral. Alnylam is also supporting a national effort led by the American Heart Association which consists of a three-year initiative that will convene a 10-site cohort of multidisciplinary health systems in a national learning collaborative designed to identify gaps in care, share best practices, and scale effective models for diagnosing and managing ATTR-CM. Hosted a TTR Investor Webinar highlighting Alnylam's progress delivering for patients with ATTR-CM as well as the long-term growth and durability of the Company's flagship TTR franchise. A replay is available here . Total Rare: GIVLAARI ® (givosiran) & OXLUMO ® (lumasiran) Achieved global net product revenues for GIVLAARI and OXLUMO for the first quarter of $74 million and $51 million, respectively, together representing $126 million in total Rare net product revenues and 15% total Rare growth compared to Q1 2025. Other Highlights Presented additional Phase 2 results for zilebesiran , in development for cardiovascular risk reduction in hypertension patients, including assessing the ability to provide continuous control of blood pressure, at the American College of Cardiology's Annual Scientific Session and Expo. A comprehensive analysis of safety across the Phase 2 KARDIA program demonstrated an acceptable safety pro zilebesiran, both as a monotherapy and in combination with standard-of-care antihypertensives, across patients with mild-to-moderate hypertension, those at high CV risk, or with lower eGFR at baseline. Initiated a Phase 1 clinical trial of ALN-2232 , Alnylam's first adipose-targeted RNAi therapeutic, targeting ACVR1C for obesity and weight management. Alnylam's collaboration partner, Regeneron Pharmaceuticals, Inc., announced the submission of a New Drug Application (NDA) to the FDA for cemdisiran, an investigational RNAi therapeutic for adults with generalized myasthenia gravis. Additional global filings are planned for 2026. Additional Business Updates Entered into a research collaboration with Tenaya Therapeutics to discover novel human genetic targets for the potential development of disease-modifying treatments for cardiovascular diseases. Announced a new multi-year agreement with Helix to drive precision medicine development, in which Alnylam will gain access to Helix's deeply phenotyped GenoSphere TM cohorts consisting of comprehensive genomic and longitudinal clinical data, and medical and pharmacy claims that cover a wide range of disease areas. Key Upcoming Events Alnylam will host its 10 th RNAi Roundtable series later this year, at which Alnylam scientists and program leaders, as well as medical thought leaders, will discuss the progress and opportunity across key pipeline programs. A detailed schedule and registration information will be announced separately. In the first half of 2026, Alnylam expects to: Complete enrollment in the cAPPricorn-1 Phase 2 clinical trial of mivelsiran in patients with cerebral amyloid angiopathy. Initiate a Phase 2 trial of mivelsiran in patients with Alzheimer’s disease. Initiate a Phase 2 clinical trial of ALN-6400 in a second bleeding disorder. In the second half of 2026, Alnylam expects to announce clinical data from several pipeline programs, including: Results from Phase 1 and Phase 2 clinical trials of ALN-6400 in healthy volunteers and patients with hereditary hemorrhagic telangiectasia (HHT), respectively. Results from a Phase 1 clinical trial of ALN-HTT02 in patients with Huntington's disease. Results from a Phase 1 clinical trial of ALN-2232 in obesity and weight management. Financial Results for the Quarter Ended March 31, 2026 Three Months Ended March 31, % Change (In thousands, except per share amounts and percentages) 2026 2025 Total revenues $ 1,167,175 $ 594,189 96 % GAAP Income from operations $ 268,636 $ 18,077 * Non-GAAP Income from operations $ 338,790 $ 74,789 353 % GAAP Net income (loss) $ 205,991 $ (18,251) ** Non-GAAP Net income $ 273,038 $ 37,941 * GAAP Net income (loss) per common share — basic $ 1.55 $ (0.14) ** GAAP Net income (loss) per common share — diluted $ 1.51 $ (0.14) ** Non-GAAP Net income per common share — basic $ 2.05 $ 0.29 * Non-GAAP Net income per common share — diluted $ 1.99 $ 0.29 * * Indicates the percentage change period over period is greater than 500% ** Not meaningful 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 Three Months Ended March 31, % Change % Change at CER* (In thousands, except percentages) 2026 2025 Net product revenues: AMVUTTRA $ 889,931 $ 309,992 187 % 183 % ONPATTRO 20,481 49,489 (59 )% (61 )% Total TTR net product revenues 910,412 359,481 153 % 150 % GIVLAARI 74,394 66,968 11 % 7 % OXLUMO 51,321 42,089 22 % 13 % Total Rare net product revenues 125,715 109,057 15 % 10 % Total net product revenues 1,036,127 468,538 121 % 117 % Net revenues from collaborations: Roche 35,641 17,056 109 % 109 % Regeneron Pharmaceuticals 46,336 51,039 (9 )% (9 )% Other 98 31,090 (100 )% (100 )% Total net revenues from collaborations 82,075 99,185 (17 )% (17 )% Royalty revenue 48,973 26,466 85 % 85 % Total revenues $ 1,167,175 $ 594,189 96 % 93 % * Change at constant exchange rates, or CER, represents growth calculated as if exchange rates had remained unchanged from those used during the three months ended March 31, 2025. CER is a non-GAAP financial measure. Total Net Product Revenues Total net product revenues increased 121% and 117% at actual currency and CER, respectively, during the three months ended March 31, 2026, as compared to the same period in 2025, primarily due to growth from AMVUTTRA revenues driven by increased patient demand, mainly in patients with ATTR-CM in the U.S., which was partially offset by a decreased number of patients on ONPATTRO, and due to growth from an increased number of patients on GIVLAARI and OXLUMO. Net Revenues from Collaborations Net revenues from collaborations decreased during the three months ended March 31, 2026, as compared to the same period in 2025, primarily driven by recognition of a $30 million payment in connection with the amendment to our agreement with Vir Biotechnology, Inc. in March 2025. Royalty Revenue Royalty revenue increased during the three months ended March 31, 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. Operating Expense Summary Three Months Ended March 31, % Change (In thousands, except percentages) 2026 2025 Cost of goods sold $ 207,520 $ 70,183 196 % % of net product revenues 20.0 % 15.0 % Cost of collaborations and royalties $ 3,602 $ 858 320 % GAAP Research and development expenses $ 364,866 $ 265,122 38 % Non-GAAP Research and development expenses $ 334,754 $ 241,324 39 % GAAP Selling, general and administrative expenses $ 322,551 $ 239,949 34 % Non-GAAP Selling, general and administrative expenses $ 282,509 $ 207,035 36 % Cost of Goods Sold Cost of goods sold as a percentage of net product revenues increased to 20% during the three months ended March 31, 2026, as compared to the same period in 2025, primarily as a result of increased sales of AMVUTTRA and an associated increase in the blended royalty rate payable on net sales of AMVUTTRA. Research & Development (R&D) Expenses GAAP and non-GAAP R&D expenses for the three months ended March 31, 2026 increased as compared to the same period in 2025, primarily due to increased clinical trial expenses for the ZENITH Phase 3 clinical trial of zilebesiran, the TRITON-CM Phase 3 clinical trial of nucresiran in patients with ATTR-CM and the TRITON-PN Phase 3 clinical trial of nucresiran in patients with hATTR-PN. Selling, General & Administrative (SG&A) Expenses GAAP and non-GAAP SG&A expenses for the three months ended March 31, 2026 increased as compared to the same period in 2025, primarily due to higher employee compensation costs and increased marketing investment associated with the ongoing global commercial launch of AMVUTTRA in ATTR-CM. Other Financial Highlights Interest expense Interest expense for the three months ended March 31, 2026 of $69 million included interest of $40 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 During the three months ended March 31, 2026, we recorded a provision for income taxes of $16 million primarily related to U.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 Cash, cash equivalents and marketable securities were $3.0 billion as of March 31, 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 March 31, 2026 included $30 million of payments associated with the liability related to the sale of future Leqvio royalties recorded to interest expense, as well as $32 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 reiterated and consists of the following: Total TTR net product revenues (AMVUTTRA, ONPATTRO) 1 $4,400 million - $4,700 million Total Rare net product revenues (GIVLAARI, OXLUMO) 1 $500 million - $600 million Total net product revenues 1 $4,900 million - $5,300 million Net product revenues growth vs. 2025 at currency exchange rates as of December 31, 2025 2 64% to 77% Net product revenues growth vs. 2025 at constant exchange rates 2 64% to 77% Net revenues from collaborations and royalties $400 million - $500 million Non-GAAP R&D and SG&A expenses 3 $2,700 million - $2,800 million 1 Full-year 2026 guidance utilizing currency exchange rates as of December 31, 2025: 1 EUR = 1.17 USD and 1 USD = 157 JPY 2 Representing growth calculated as if the exchange rates had remained unchanged from those used in 2025, which is a non-GAAP financial measure 3 Excludes $300 million - $400 million of stock-based compensation expense from estimated GAAP R&D and SG&A expenses Use of Non-GAAP Financial Measures This press release contains non-GAAP financial measures, including expenses adjusted to exclude certain non-cash expenses and non-recurring gains or losses outside the ordinary course of the Company’s business. These measures are not in accordance with, or an alternative to, GAAP, and may be different from non-GAAP financial measures used by other companies. The items included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in this press release are stock-based compensation expenses, and realized and unrealized gains or losses on marketable equity securities. The Company has excluded the impact of stock-based compensation expense, which may fluctuate from period to period based on factors including the variability associated with performance-based grants for stock options and restricted stock units and changes in the Company’s stock price, which impacts the fair value of these awards. The Company has excluded the impact of the realized and unrealized gains or losses on marketable equity securities because the Company does not believe these adjustments accurately reflect the performance of the Company’s ongoing operations for the period in which such gains or losses are reported, as their sole purpose is to adjust amounts on the balance sheet. Percentage changes in revenue growth at CER are presented excluding the impact of changes in foreign currency exchange rates for investors to understand the underlying business performance. The current period’s foreign currency revenue values are converted into U.S. dollars using the average exchange rates from the prior period. The Company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding the Company’s financial condition and results of operations. When GAAP financial measures are viewed in conjunction with non-GAAP financial measures, investors are provided with a more meaningful understanding of the Company’s ongoing operating performance and are better able to compare the Company’s performance between periods. In addition, these non-GAAP financial measures are among those indicators the Company uses as a basis for evaluating performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between GAAP and non-GAAP measures is provided later in this press release. Conference Call Information Management will provide an update on the Company and discuss first quarter 2026 results as well as expectations for the future via conference call on Thursday, April 30, 2026, at 8:30 am ET. A live audio webcast of the call will be available on the Investors section of the Company’s website at . An archived webcast will be available on the Alnylam website approximately two hours after the event. 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 full U.S. Prescribing Information, visit AMVUTTRA.com . About ONPATTRO ® (patisiran) ONPATTRO is an RNAi therapeutic that is approved in the United States and Canada for the treatment of adults with hATTR amyloidosis with polyneuropathy. ONPATTRO is also approved in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and in Japan for 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 in the United States and Brazil for 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 full U.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 the U.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 the European 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 pro 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 full U.S. Prescribing Information, visit OXLUMO.com . About LNP Technology Alnylam has 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. Contacts Alnylam Pharmaceuticals, Inc. Christine Akinc (Investors and Media) 617-682-4340 Josh Brodsky (Investors) 617-551-8276 Read full story here