Financial Analysis

12. COMMITMENTS AND CONTINGENCIES Technology License and Other Commitments We have licensed from third parties the rights to use certain technologies and information in our research processes as well as in any other products we may develop. In accordance with the related license or technology agreements, we are required to make certain fixed payments to the licensor or a designee of the licensor over various agreement terms. Many of these agreement terms are consistent with the remaining lives of the underlying intellectual property that we have licensed. As of June 30, 2026, our commitments over the next five years to make fixed and cancellable payments under existing license agreements were not material. Legal Matters From time to time, we may be a party to litigation, arbitration or other legal proceedings in the ordinary course of our business activities, including the following types of matters which are common to companies in our industry: Patent litigation, which typically involves challenges to the coverage and/or validity of patents on various products or product candidates, processes or dosage forms. An adverse outcome could result in loss of patent protection for a product or product candidates, a significant loss of revenues from a product or impairment of the value of associated assets. Product liability and other product-related litigation related to our products, which could include personal injury, consumer fraud, off-label promotion, securities, antitrust and …

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8. CONVERTIBLE DEBT AND OTHER FINANCING Convertible Senior Notes Due 2028 On September 8, 2025, we commenced a private offering of $575.0 million in aggregate principal amount of 0.00% convertible senior notes due 2028, or the Initial 2028 Notes. On September 10, 2025, the initial purchasers in such offering exercised their option to purchase an additional $86.3 million in aggregate principal amount of our 0.00% Convertible Senior Notes due 2028, or the Additional 2028 Notes, and together with the Initial 2028 Notes referred to as the 2028 Notes, bringing the total aggregate principal amount of the 2028 Notes issued and outstanding to $661.3 million. The 2028 Notes are our senior unsecured obligations. The 2028 Notes were issued pursuant to an indenture, dated September 12, 2025, or the 2025 Indenture, between us and The Bank of New York Mellon, as trustee. The 2025 Indenture includes customary covenants and sets forth certain events of default after which the 2028 Notes may be declared immediately due and payable and sets forth certain types of bankruptcy or insolvency events of default involving us after which the 2028 Notes become automatically due and payable. The 2028 Notes will mature on September 15, 2028, unless earlier converted, redeemed or repurchased. The 2028 Notes will not bear regular interest. Before June 15, 2028, noteholders will have the right to convert their 2028 Notes in certain circumstances and during specified periods: (1) during any calendar quarter …

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Net product revenues, classified based on the geographic region in which the product is sold and by franchise (TTR, which includes AMVUTTRA and ONPATTRO, and Rare, which includes GIVLAARI and OXLUMO) consisted of the following: Three Months Ended June 30, Six Months Ended June 30, (In thousands) 2026 2025 2026 2025 AMVUTTRA United States $ 809,079 $ 361,346 $ 1,511,633 $ 559,310 Europe 119,010 92,868 232,323 172,956 Rest of World 83,673 37,739 157,737 69,679 Total 1,011,762 491,953 1,901,693 801,945 ONPATTRO United States 9,863 22,053 20,029 37,625 Europe 5,508 21,246 13,073 47,787 Rest of World 3,090 9,239 5,840 16,615 Total 18,461 52,538 38,942 102,027 Total TTR 1,030,223 544,491 1,940,635 903,972 GIVLAARI United States 58,000 55,151 107,091 98,945 Europe 24,323 20,966 44,613 39,510 Rest of World 7,441 4,732 12,454 9,362 Total 89,764 80,849 164,158 147,817 OXLUMO United States 18,545 16,019 35,510 30,128 Europe 22,678 21,929 47,068 42,913 Rest of World 10,899 8,924 20,865 15,920 Total 52,122 46,872 103,443 88,961 Total Rare 141,886 127,721 267,601 236,778 Total net product revenues $ 1,172,109 $ 672,212 $ 2,208,236 $ 1,140,750 …

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10. STOCK-BASED COMPENSATION The following table summarizes stock-based compensation expense included in operating costs and expenses on our condensed consolidated statements of operations and comprehensive income (loss), and stock-based compensation charges included in additional paid-in capital on our condensed consolidated statements of stockholders' equity: Three Months Ended June 30, Six Months Ended June 30, (In thousands) 2026 2025 2026 2025 Research and development $ 35,894 $ 49,552 $ 66,006 $ 73,350 Selling, general and administrative 50,731 62,128 90,773 95,042 Total stock-based compensation expense 86,625 111,680 156,779 168,392 Capitalized stock-based compensation costs 1,105 1,127 2,210 2,255 Total stock-based compensation charges $ 87,730 $ 112,807 $ 158,989 $ 170,647

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5. FAIR VALUE MEASUREMENTS The following tables present information about our financial assets that are measured at fair value on a recurring basis and indicate the fair value hierarchy of the valuation techniques we utilized to determine such fair value: (In thousands) As of June 30, 2026 Quoted Prices in Active Markets (Level 1) Significant Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Financial assets Cash equivalents: Money market funds 141,609 141,609 Marketable debt securities: U.S. treasury securities 808,038 808,038 Corporate notes 437,947 437,947 U.S. government-sponsored enterprise securities 339,623 339,623 Commercial paper 14,221 14,221 Restricted cash (money market funds) 569 569 Total financial assets $ 1,742,007 $ 142,178 $ 1,599,829 $ (In thousands) As of December 31, 2025 Quoted Prices in Active Markets (Level 1) Significant Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Financial assets Cash equivalents: Money market funds $ 140,445 $ 140,445 $ $ U.S. treasury securities 18,952 18,952 U.S. government-sponsored enterprise securities 2,690 2,690 Commercial paper 1,995 1,995 Marketable debt securities: U.S. treasury securities 653,341 653,341 Corporate notes 333,241 333,241 U.S. government-sponsored enterprise securities 252,634 252,634 Commercial paper 7,017 7,017 Municipal securities 5,001 5,001 Restricted cash (money market funds) 917 917 Total financial assets $ 1,416,233 $ 141,362 $ 1,274,871 $ During the thre …

FairValueDisclosuresTextBlock · excerpt; the full note is in the filing

Recent Accounting Pronouncements In September 2025, the Financial Accounting Standards Board, or FASB, issued ASU 2025-06, Targeted Improvements to the Accounting for Internal-Use Software , which modernizes the guidance for internal-use software costs by increasing the operability of the recognition guidance considering different methods of software development. The standard will be effective for annual reporting periods beginning after December 15, 2027, as well as interim period reporting periods within those annual reporting periods, with early adoption permitted. The standard updates may be applied on a prospective, retrospective, or modified retrospective approach. We are currently evaluating the impact this guidance could have on our condensed consolidated financial statements and related disclosures. In November 2024, the FASB issued ASU 2024-03, Disaggregation of Income Statement Expenses , or ASU 2024-03, which is intended to improve disclosures by requiring additional information about specific expense categories in the notes to the financial statements on an annual and interim basis. Additionally, in January 2025, the FASB issued ASU 2025-01, clarifying that ASU 2024-03 will be effective for annual reporting periods beginning after December 15, 2026 and interim reporting periods beginning after December 15, 2027, with early adoption permitted. The standard updates may be applied on either a prospective or retrospective basis. We are currently evaluating the disclo …

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3. NET PRODUCT REVENUES Net product revenues, classified based on the geographic region in which the product is sold and by franchise (TTR, which includes AMVUTTRA and ONPATTRO, and Rare, which includes GIVLAARI and OXLUMO) consisted of the following: Three Months Ended June 30, Six Months Ended June 30, (In thousands) 2026 2025 2026 2025 AMVUTTRA United States $ 809,079 $ 361,346 $ 1,511,633 $ 559,310 Europe 119,010 92,868 232,323 172,956 Rest of World 83,673 37,739 157,737 69,679 Total 1,011,762 491,953 1,901,693 801,945 ONPATTRO United States 9,863 22,053 20,029 37,625 Europe 5,508 21,246 13,073 47,787 Rest of World 3,090 9,239 5,840 16,615 Total 18,461 52,538 38,942 102,027 Total TTR 1,030,223 544,491 1,940,635 903,972 GIVLAARI United States 58,000 55,151 107,091 98,945 Europe 24,323 20,966 44,613 39,510 Rest of World 7,441 4,732 12,454 9,362 Total 89,764 80,849 164,158 147,817 OXLUMO United States 18,545 16,019 35,510 30,128 Europe 22,678 21,929 47,068 42,913 Rest of World 10,899 8,924 20,865 15,920 Total 52,122 46,872 103,443 88,961 Total Rare 141,886 127,721 267,601 236,778 Total net product revenues $ 1,172,109 $ 672,212 $ 2,208,236 $ 1,140,750 As of June 30, 2026 and December 31, 2025, net product revenue-related receivables of $787.8 million and $669.5 million, respectively, were included in accounts receivable, net on our condensed consolidated balance sheets. The following table summarizes balances and activity in each product revenue allowance and reserve categor …

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13. SEGMENT INFORMATION We operate in a single segment dedicated to the discovery, development, manufacturing and commercialization of RNAi therapeutics. Consistent with our management reporting, results of our operations are reported on a consolidated basis for purposes of segment reporting. Our Chief Executive Officer, or CEO, as the chief operating decision maker, or CODM, evaluates performance and decides how to allocate resources based on consolidated net income (loss) that is reported on the condensed consolidated statements of operations and comprehensive income (loss). The measure of segment assets is reported on the condensed consolidated balance sheets as total assets. Please refer to the condensed consolidated financial statements for further information related to these measures of segment performance. In addition, research and development and selling, general and administrative expenses are significant segment expenses regularly provided to the CEO with the following categories: Research and Development Three Months Ended June 30, Six Months Ended June 30, (In thousands) 2026 2025 2026 2025 Clinical research and outside services $ 244,226 $ 140,692 $ 443,483 $ 268,035 Compensation and related 125,390 143,450 249,688 242,637 Occupancy and all other costs (1) 43,518 39,479 84,829 78,071 Total research and development expense $ 413,134 $ 323,621 $ 778,000 $ 588,743 Selling, General and Administrative Three Months Ended June 30, Six Months Ended June 30, (In thousand …

SegmentReportingDisclosureTextBlock · excerpt; the full note is in the filing