Givosiran Sodium

− 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 $4,400 to $4,700 Million to $4,200 to $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 with Huntington's Disease to be Presented at EHDN – ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); − Accelerated Integration of AI Across Alnylam by Establishing Strategic Collaborations with Inceptive to Transform RNAi Discovery and a Large Health Care System in California to Support Earlier Identification of ATTR-CM in Routine Care, as well as Expanding Partnership with Komodo Health to Scale Commercial Intelligence – − Entered Into an Exclusive Agreement with BeOne Medicines for Commercialization of AMVUTTRA in China – CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the second quarter ended June 30, 2026, and reviewed recent business highlights. “During the first half of 2026, we continued to meaningfully advance our business, generating over $1 billion in quarterly product revenues for the first time in our history during the first quarter and, building on that momentum, over $1 billion in 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,” said Yvonne Greenstreet, M.D., Chief Executive Officer of Alnylam. “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 Alnylam 2030 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) 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. 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. 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. 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. 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) 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 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. Initiated a Phase 1 clinical trial of ALN-6222 , an investigational RNAi therapeutic targeting inhibin E (INHBE), in adult patients with obesity. 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. 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. Our collaboration partner, Regeneron Pharmaceuticals, Inc., announced that the U.S. Food and Drug Administration (FDA) and the European 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 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. Expanded the Company's strategic partnership with Komodo Health to scale Marmot, Komodo's analytics AI platform, across key enterprise functions at Alnylam. Appointed Benjamin Franklin Cravatt III, Ph.D., to Alnylam's Board of Directors. Published 2025 Corporate Responsibility Report. Key Upcoming Events Alnylam announces today that it will present initial results from the Phase 1 clinical trial of ALN-HTT02 in patients with Huntington's disease at the European Huntington's Disease Network (EHDN) Clinical Research Congress on Friday, October 23, 2026, in Kraków, Poland. The Company continues to host its 10 th 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: Zilebesiran : Targeting Angiotensinogen to Achieve Continuous Control of Blood Pressure Thursday, September 17, 10:30 am ET 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, Alnylam expects to announce clinical data from additional 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-2232 in obesity and weight management. Second Quarter 2026 Financial Results Three Months Ended June 30, % Change (In thousands, except per share amounts and percentages) 2026 2025 Total revenues $ 1,290,948 $ 773,689 67 % GAAP Income (loss) from operations $ 231,441 $ (16,199 ) ** Non-GAAP Income from operations $ 318,066 $ 95,481 233 % GAAP Net income (loss) $ 164,494 $ (72,228 ) ** Non-GAAP Net income $ 251,801 $ 38,171 * GAAP Net income (loss) per common share — basic $ 1.23 $ (0.55 ) ** GAAP Net income (loss) per common share — diluted $ 1.21 $ (0.55 ) ** Non-GAAP Net income per common share — basic $ 1.88 $ 0.29 * Non-GAAP Net income per common share — diluted $ 1.84 $ 0.28 * * 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 June 30, % Change % Change at CER* (In thousands, except percentages) 2026 2025 Net product revenues: AMVUTTRA $ 1,011,762 $ 491,953 106 % 106 % ONPATTRO 18,461 52,538 (65 )% (65 )% Total TTR net product revenues 1,030,223 544,491 89 % 89 % GIVLAARI 89,764 80,849 11 % 10 % OXLUMO 52,122 46,872 11 % 9 % Total Rare net product revenues 141,886 127,721 11 % 10 % Total net product revenues 1,172,109 672,212 74 % 74 % Net revenues from collaborations: Roche 41,888 18,267 129 % 129 % Regeneron Pharmaceuticals 5,020 32,542 (85 )% (85 )% Other 257 10,687 (98 )% (98 )% Total net revenues from collaborations 47,165 61,496 (23 )% (23 )% Royalty revenue 71,674 39,981 79 % 79 % Total revenues $ 1,290,948 $ 773,689 67 % 67 % * 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 June 30, 2025. CER is a non-GAAP financial measure. Total Net Product Revenues 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 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 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. Operating Expense Summary Three Months Ended June 30, % Change (In thousands, except percentages) 2026 2025 Cost of goods sold $ 298,261 $ 142,029 110 % % of net product revenues 25.4 % 21.1 % Cost of collaborations and royalties $ 190 $ 924 (79 )% GAAP Research and development expenses $ 413,134 $ 323,621 28 % Non-GAAP Research and development expenses $ 377,240 $ 274,069 38 % GAAP Selling, general and administrative expenses $ 347,922 $ 323,314 8 % Non-GAAP Selling, general and administrative expenses $ 297,191 $ 261,186 14 % Cost of Goods Sold Cost of goods sold as a percentage of net product revenues increased during the three months ended June 30, 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 June 30, 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 June 30, 2026 increased as compared to the same period in 2025, primarily due to 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 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 During the three months ended June 30, 2026, we recorded a provision for income taxes of $13 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 $3.3 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: Item Prior FY 2026 Guidance Updated FY 2026 Guidance Total TTR net product revenues (AMVUTTRA, ONPATTRO) 1 $4,400 million - $4,700 million $4,200 million - $4,500 million Total Rare net product revenues (GIVLAARI, OXLUMO) 1 $500 million - $600 million Reiterate Total net product revenues 1 $4,900 million - $5,300 million $4,700 million - $5,100 million Net product revenues growth vs. 2025 at currency exchange rates as of June 30, 2026 1 64% to 77% 57% to 71% Net product revenues growth vs. 2025 at constant exchange rates 2 64% to 77% 57% to 70% Net revenues from collaborations and royalties $400 million - $500 million $575 million - $625 million Non-GAAP R&D and SG&A expenses 3 $2,700 million - $2,800 million Reiterate 1 Full-year 2026 guidance utilizing currency exchange rates as of June 30, 2026: 1 EUR = 1.14 USD and 1 USD = 162 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 in the prior FY 2026 guidance and $330 million - $380 million of stock-based compensation expense in the updated FY 2026 guidance 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. 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 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 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 second quarter 2026 results as well as expectations for the future via conference call on Thursday, July 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. Contacts Alnylam Pharmaceuticals, Inc. Bo Piela (Media) 508-308-9783 Josh Brodsky (Investors) 617-551-8276 Read full story here

SAN DIEGO, June 8, 2026 /PRNewswire/ -- Protego Biopharma, Inc., a clinical-stage biotechnology company focused on developing small-molecule drugs that target protein misfolding diseases, today announced the appointment of Jae B. Kim, MD, as its Chief Medical Officer (CMO). Dr. Kim joins Protego after serving as Chief Medical Officer of Septerna since 2024. Continue Reading Jae B. Kim, MD Prior to Septerna, Dr. Kim served as Chief Medical Officer at Design Therapeutics, where he led the clinical advancement of the company's pipeline of small molecule genomic therapeutics. Previously, he was Chief Medical Officer at Avidity Biosciences, Vice President of Clinical Development at Alnylam Pharmaceuticals, and held roles of increasing responsibility in global development at MyoKardia and Amgen. He has contributed to the development and/or approval of multiple drugs for cardiovascular and rare diseases, including Givlaari (givosiran), Amvuttra (vutrisiran), Leqvio (inclisiran), Camzyos (mavacamten), Corlanor (ivabradine), and Repatha (evolcumab). "Dr. Kim is an accomplished healthcare executive who brings to Protego a wealth of experience in advancing novel therapies across multiple therapeutic areas in addition to cardiovascular diseases," said Brent J. Warner, CEO of Protego. "He joins Protego at an opportune time to help advance our lead program PROT-001, a small molecule pharmacological chaperone designed to treat light chain amyloidosis, into pivotal studies this year." A board-certified cardiologist, Dr. Kim's academic career has included serving on the Faculty of Medicine at Harvard Medical School and the Brigham and Women's Hospital. He earned his BA in Biological Sciences from Cornell University and his MD from Cornell University Medical College, completed a postdoctoral fellowship in genetics at Harvard Medical School, and completed clinical training in cardiovascular disease at the Brigham and Women's Hospital and Massachusetts General Hospital. "I am pleased to join the Protego team and lead the global registration program that will pursue accelerated and full approval for PROT-001 in light chain amyloidosis, and potentially change the standard of care by addressing the root cause of this disease," said Kim. "My excitement about chaperoning this innovative therapy through late-stage development reflects my passion for addressing rare diseases with serious unmet medical needs." About Protego Biopharma Protego Biopharma is a San Diego-based biotech company focused on developing small-molecule drugs that target protein misfolding pathways. By reprogramming cellular processes to restore proper protein function, Protego aims to treat rare and systemic diseases with precision and improved patient outcomes. For more information, visit . SOURCE Protego Biopharma 21% more press release views with Request a Demo