Document and Entity Information
Document and Entity Information - shares | 3 Months Ended | |
Mar. 31, 2024 | May 01, 2024 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Quarterly Report | true | |
Document Period End Date | Mar. 31, 2024 | |
Current Fiscal Year End Date | --12-31 | |
Document Fiscal Year Focus | 2024 | |
Document Fiscal Period Focus | Q1 | |
Document Transition Report | false | |
Entity File Number | 000-19125 | |
Entity Registrant Name | Ionis Pharmaceuticals, Inc. | |
Entity Central Index Key | 0000874015 | |
Entity Incorporation, State or Country Code | DE | |
Entity Tax Identification Number | 33-0336973 | |
Entity Address, Address Line One | 2855 Gazelle Court | |
Entity Address, City or Town | Carlsbad | |
Entity Address, State or Province | CA | |
Entity Address, Postal Zip Code | 92010 | |
City Area Code | 760 | |
Local Phone Number | 931-9200 | |
Title of 12(b) Security | Common Stock, $.001 Par Value | |
Trading Symbol | IONS | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 145,965,374 |
CONDENSED CONSOLIDATED BALANCE
CONDENSED CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Current assets: | ||
Cash and cash equivalents | $ 348,889 | $ 399,266 |
Short-term investments | 1,857,327 | 1,931,935 |
Contracts receivable | 5,140 | 97,778 |
Inventories | 30,259 | 28,425 |
Other current assets | 174,060 | 184,449 |
Total current assets | 2,415,675 | 2,641,853 |
Property, plant and equipment, net | 72,795 | 71,043 |
Right-of-use assets | 169,431 | 171,896 |
Deposits and other assets | 106,027 | 105,280 |
Total assets | 2,763,928 | 2,990,072 |
Current liabilities: | ||
Accounts payable | 13,082 | 26,027 |
Accrued compensation | 21,537 | 67,727 |
Accrued liabilities | 106,048 | 147,894 |
Income taxes payable | 2,210 | 2,151 |
0.125 percent convertible senior notes, net | 44,377 | 44,332 |
Current portion of deferred contract revenue | 130,406 | 151,128 |
Other current liabilities | 9,988 | 8,831 |
Total current liabilities | 327,648 | 448,090 |
Long-term deferred contract revenue | 215,088 | 241,184 |
Liability related to sale of future royalties, net | 525,072 | 513,736 |
Long-term lease liabilities | 168,674 | 170,875 |
Long-term obligations | 41,800 | 41,836 |
Total liabilities | 2,467,413 | 2,603,386 |
Stockholders' equity: | ||
Common stock, $0.001 par value; 300,000,000 shares authorized, 145,844,719 and 144,340,526 shares issued and outstanding at March 31, 2024 (unaudited) and December 31, 2023, respectively | 146 | 144 |
Additional paid-in capital | 2,270,047 | 2,215,098 |
Accumulated other comprehensive loss | (34,964) | (32,645) |
Accumulated deficit | (1,938,714) | (1,795,911) |
Total stockholders' equity | 296,515 | 386,686 |
Total liabilities and stockholders' equity | 2,763,928 | 2,990,072 |
1.75 Percent Convertible Senior Notes [Member] | ||
Current liabilities: | ||
Convertible senior notes, net | 562,964 | 562,285 |
0 Percent Convertible Senior Notes [Member] | ||
Current liabilities: | ||
Convertible senior notes, net | $ 626,167 | $ 625,380 |
CONDENSED CONSOLIDATED BALANC_2
CONDENSED CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares | Mar. 31, 2024 | Dec. 31, 2023 |
Stockholders' equity: | ||
Common stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Common stock, shares authorized (in shares) | 300,000,000 | 300,000,000 |
Common stock, shares issued (in shares) | 145,844,719 | 144,340,526 |
Common stock, shares outstanding (in shares) | 145,844,719 | 144,340,526 |
1.75 Percent Convertible Senior Notes [Member] | ||
LIABILITIES AND STOCKHOLDERS' EQUITY | ||
Interest rate on convertible senior notes | 1.75% | |
0 Percent Convertible Senior Notes [Member] | ||
LIABILITIES AND STOCKHOLDERS' EQUITY | ||
Interest rate on convertible senior notes | 0% | 0% |
0.125 Percent Convertible Senior Notes [Member] | ||
LIABILITIES AND STOCKHOLDERS' EQUITY | ||
Interest rate on convertible senior notes | 0.125% | 0.125% |
CONDENSED CONSOLIDATED STATEMEN
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS - USD ($) shares in Thousands, $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Revenue: | ||
Revenue | $ 119,497 | $ 130,524 |
Expenses: | ||
Cost of sales | 2,151 | 1,343 |
Research, development and patent | 214,215 | 197,813 |
Selling, general and administrative | 52,644 | 45,516 |
Total operating expenses | 269,010 | 244,672 |
Loss from operations | (149,513) | (114,148) |
Other income (expense): | ||
Investment income | 26,285 | 18,627 |
Interest expense | (4,151) | (1,608) |
Interest expense related to sale of future royalties | (17,959) | (15,515) |
Gain (loss) on investments | 2,333 | (529) |
Other income | 277 | 230 |
Loss before income tax expense | (142,728) | (112,943) |
Income tax expense | (75) | (11,380) |
Net loss | $ (142,803) | $ (124,323) |
Basic net loss per share (in dollars per share) | $ (0.98) | $ (0.87) |
Diluted net loss per share (in dollars per share) | $ (0.98) | $ (0.87) |
Shares used in computing basic net loss per share (in shares) | 145,538 | 142,735 |
Shares used in computing diluted net loss per share (in shares) | 145,538 | 142,735 |
Commercial Revenue [Member] | ||
Revenue: | ||
Revenue | $ 59,593 | $ 67,768 |
SPINRAZA Royalties [Member] | ||
Revenue: | ||
Revenue | 38,455 | 50,247 |
WAINUA Royalties [Member] | ||
Revenue: | ||
Revenue | 1,125 | 0 |
Other Commercial Revenue [Member] | ||
Revenue: | ||
Revenue | 20,013 | 17,521 |
Research and Development Revenue [Member] | ||
Revenue: | ||
Revenue | 59,904 | 62,756 |
Collaborative Agreement Revenue [Member] | ||
Revenue: | ||
Revenue | 49,345 | 38,334 |
WAINUA Joint Development Revenue [Member] | ||
Revenue: | ||
Revenue | $ 10,559 | $ 24,422 |
CONDENSED CONSOLIDATED STATEM_2
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS [Abstract] | ||
Net loss | $ (142,803) | $ (124,323) |
Unrealized losses on debt securities, net of tax | (2,205) | 8,393 |
Currency translation adjustment | (114) | 104 |
Comprehensive loss | $ (145,122) | $ (115,826) |
CONDENSED CONSOLIDATED STATEM_3
CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY - USD ($) shares in Thousands, $ in Thousands | Common Stock [Member] | Additional Paid in Capital [Member] | Accumulated Other Comprehensive Loss [Member] | Accumulated Deficit [Member] | Total |
Balance at Dec. 31, 2022 | $ 142 | $ 2,059,850 | $ (57,480) | $ (1,429,625) | $ 572,887 |
Balance (in shares) at Dec. 31, 2022 | 142,058 | ||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Net loss | $ 0 | 0 | 0 | (124,323) | (124,323) |
Change in unrealized losses, net of tax | 0 | 0 | 8,393 | 0 | 8,393 |
Foreign currency translation | 0 | 0 | 104 | 0 | 104 |
Issuance of common stock in connection with employee stock plans | $ 1 | 2,560 | 0 | 0 | 2,561 |
Issuance of common stock in connection with employee stock plans (in shares) | 965 | ||||
Stock-based compensation expense | $ 0 | 26,948 | 0 | 0 | 26,948 |
Balance at Mar. 31, 2023 | $ 143 | 2,089,358 | (48,983) | (1,553,948) | 486,570 |
Balance (in shares) at Mar. 31, 2023 | 143,023 | ||||
Balance at Dec. 31, 2023 | $ 144 | 2,215,098 | (32,645) | (1,795,911) | 386,686 |
Balance (in shares) at Dec. 31, 2023 | 144,341 | ||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||
Net loss | $ 0 | 0 | 0 | (142,803) | (142,803) |
Change in unrealized losses, net of tax | 0 | 0 | (2,205) | 0 | (2,205) |
Foreign currency translation | 0 | 0 | (114) | 0 | (114) |
Issuance of common stock in connection with employee stock plans | $ 2 | 23,609 | 0 | 0 | 23,611 |
Issuance of common stock in connection with employee stock plans (in shares) | 1,504 | ||||
Stock-based compensation expense | $ 0 | 31,340 | 0 | 0 | 31,340 |
Balance at Mar. 31, 2024 | $ 146 | $ 2,270,047 | $ (34,964) | $ (1,938,714) | $ 296,515 |
Balance (in shares) at Mar. 31, 2024 | 145,845 |
CONDENSED CONSOLIDATED STATEM_4
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | Dec. 31, 2023 | |
Operating activities: | |||
Net loss | $ (142,803) | $ (124,323) | |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Depreciation | 2,547 | 2,604 | |
Amortization of right-of-use operating lease assets | 2,465 | 2,395 | |
Amortization of other assets | 639 | 607 | |
Amortization of discount on investments, net | (9,257) | (5,042) | |
Amortization of debt issuance costs | 1,667 | 1,486 | |
Non-cash royalty revenue related to sale of royalties | (6,623) | 0 | |
Non-cash interest related to sale of future royalties | 17,806 | 15,363 | |
Stock-based compensation expense | 31,340 | 26,948 | |
Loss (gain) on investments | (2,332) | 529 | |
Non-cash losses related to other assets | 133 | 445 | |
Changes in operating assets and liabilities: | |||
Contracts receivable | 92,638 | 11,624 | |
Inventories | (1,834) | (167) | |
Other current and long-term assets | 13,381 | 5,312 | |
Income taxes payable | 59 | 11,037 | |
Accounts payable | (13,869) | (10,295) | |
Accrued compensation | (46,190) | (31,018) | |
Accrued liabilities and other current liabilities | (42,887) | (28,460) | |
Deferred contract revenue | (46,818) | (13,037) | |
Net cash used in operating activities | (149,938) | (133,992) | |
Investing activities: | |||
Purchases of short-term investments | (519,001) | (688,278) | |
Proceeds from sale of short-term investments | 600,836 | 374,363 | |
Purchases of property, plant and equipment | (4,493) | (10,472) | |
Acquisition of licenses and other assets, net | (1,237) | (1,253) | |
Net cash provided by (used in) investing activities | 76,105 | (325,640) | |
Financing activities: | |||
Proceeds from equity, net | 23,609 | 2,560 | |
Proceeds from sale of future royalties | 0 | 500,000 | |
Payments of transaction costs related to sale of future royalties | 0 | (10,434) | |
Principal payments on mortgage debt | (39) | (39) | |
Net cash provided by financing activities | 23,570 | 492,087 | |
Effects of exchange rates on cash | (114) | 104 | |
Net increase (decrease) in cash and cash equivalents | (50,377) | 32,559 | |
Cash and cash equivalents at beginning of period | 399,266 | 276,472 | $ 276,472 |
Cash and cash equivalents at end of period | 348,889 | 309,031 | $ 399,266 |
Supplemental disclosures of cash flow information: | |||
Interest paid | 95 | 89 | |
Income taxes paid | 13 | 293 | |
Supplemental disclosures of non-cash investing and financing activities: | |||
Amounts accrued for capital and patent expenditures | $ 924 | $ 3,058 |
Organization and Basis of Prese
Organization and Basis of Presentation | 3 Months Ended |
Mar. 31, 2024 | |
Organization and Basis of Presentation [Abstract] | |
Organization and Basis of Presentation | 1. Organization and Basis of Presentation Organization and Business Activity We incorporated in California on January 10, 1989. In conjunction with our initial public offering, we reorganized as a Delaware corporation in April 1991. We are a leader in the discovery and development of RNA-targeted therapeutics. Basis of Presentation We prepared the unaudited interim condensed consolidated financial statements for the three months ended March 31, 2024 and 2023 on the same basis as the audited financial statements for the year ended December 31, 2023. We included all normal recurring adjustments in the financial statements, which we considered necessary for a fair presentation of our financial position at such dates and our operating results and cash flows for those periods. Our operating results for the interim periods may not be indicative of what our operating results will be for the entire year. For more complete financial information, these financial statements, and notes thereto, should be read in conjunction with the audited financial statements for the year ended December 31, 2023 included in our Annual Report on Form 10-K filed with the Securities and Exchange Commission, or SEC. In our condensed consolidated financial statements, we included the accounts of Ionis Pharmaceuticals, Inc. and the consolidated results of our wholly owned subsidiary, Akcea Therapeutics, Inc. and its wholly owned subsidiaries (“we”, “us” or “our”). We operate as a single single Use of Estimates We prepare our condensed consolidated financial statements in conformity with accounting principles generally accepted in the United States, or |
Significant Accounting Policies
Significant Accounting Policies | 3 Months Ended |
Mar. 31, 2024 | |
Significant Accounting Policies [Abstract] | |
Significant Accounting Policies | 2. Significant Accounting Policies Our significant accounting policies have not changed substantially from those included in our Annual Report on Form 10-K for the year ended December 31, 2023. Recently Adopted Accounting Standards We do not expect any recently issued accounting standards to have a material impact to our financial results. |
Supplemental Financial Data
Supplemental Financial Data | 3 Months Ended |
Mar. 31, 2024 | |
Supplemental Financial Data [Abstract] | |
Supplemental Financial Data | 3. Supplemental Financial Data Inventories Our inventory consisted of the following (in thousands): March 31, 2024 December 31, 2023 Raw materials: Raw materials - clinical $ 23,317 $ 20,985 Raw materials - commercial 705 1,809 Total raw materials 24,022 22,794 Work in process 6,037 5,477 Finished goods 200 154 Total inventories $ 30,259 $ 28,425 Accrued Liabilities Our accrued liabilities consisted of the following (in thousands): March 31, 2024 December 31, 2023 Clinical development expenses $ 75,929 $ 105,967 In-licensing expenses 6,436 7,454 Commercial expenses 4,739 4,875 Other miscellaneous expenses 18,944 29,598 Total accrued liabilities $ 106,048 $ 147,894 |
Revenues
Revenues | 3 Months Ended |
Mar. 31, 2024 | |
Revenues [Abstract] | |
Revenues | 4. Revenues During the three months ended March 31, 2024 and 2023, our revenues were comprised of the following (in thousands): Three Months Ended March 31, 2024 2023 Revenue: Commercial revenue: SPINRAZA royalties $ 38,455 $ 50,247 WAINUA royalties 1,125 — Other commercial revenue: TEGSEDI and WAYLIVRA revenue, net 8,628 6,478 Licensing and other royalty revenue 11,385 11,043 Total other commercial revenue 20,013 17,521 Total commercial revenue 59,593 67,768 Research and development revenue: Collaborative agreement revenue 49,345 38,334 WAINUA joint development revenue 10,559 24,422 Total research and development revenue 59,904 62,756 Total revenue $ 119,497 $ 130,524 Revenue Sources The following are sources of revenue and when we typically recognize revenue. Commercial Revenue: SPINRAZA royalties and WAINUA royalties We earn commercial revenue primarily in the form of royalty payments on net sales of SPINRAZA. In 2024, we began earning royalties from WAINUA sales. Commercial Revenue: TEGSEDI and WAYLIVRA revenue, net We earn commercial revenue from TEGSEDI and WAYLIVRA sales under our distribution agreements with Sobi. In addition, we receive royalties from PTC Therapeutics International Limited, or PTC, for TEGSEDI and WAYLIVRA sales. Commercial Revenue: Licensing and other royalty revenue We also recognize as commercial revenue sales milestone payments and royalties we earn under our partnerships. For example, we earn royalty revenue on net sales of QALSODY, which is included in Licensing and other royalty revenue. Research and development revenue under collaboration agreements We enter into collaboration agreements to license and sell our technology on an exclusive or non-exclusive basis. Our collaboration agreements typically contain multiple elements, or performance obligations, including technology licenses or options to obtain technology licenses, research and development, or R&D, services and manufacturing services. Upfront payments: Milestone payments: We recognize milestone payments that relate to an ongoing performance obligation over our period of performance. For example, when we achieve a milestone payment from a partner for advancing a clinical study under a collaboration agreement, we add the milestone payment to the transaction price if the milestone relates to an ongoing R&D services performance obligation and recognize revenue related to the milestone payment over our estimated period of performance. If we have partially completed our performance obligation, then we record a cumulative-effect adjustment in the period we add the milestone payment to the transaction price. Conversely, we recognize in full those milestone payments that we earn based on our partners’ activities when our partner achieves the milestone event and we do not have a performance obligation. License fees: WAINUA (Eplontersen) Collaboration with AstraZeneca In 2021, we entered into a joint development and commercialization agreement with AstraZeneca to develop and commercialize WAINUA for the treatment of transthyretin amyloidosis, or ATTR. We jointly developed and are preparing to commercialize WAINUA with AstraZeneca in the U.S. We initially granted AstraZeneca exclusive rights to commercialize WAINUA outside the U.S., except for certain Latin American countries. In 2023, we expanded those rights to include Latin America. Under the terms of the agreement, we received a $200 million upfront payment in 2021. We evaluated our WAINUA collaboration under ASC 808 and identified four material components: (i) the license we granted to AstraZeneca in 2021, (ii) the co-development activities that we and AstraZeneca are performing, (iii) the co-commercialization activities that we and AstraZeneca are performing and (iv) the co-medical affairs activities that we and AstraZeneca are performing. We determined that we had a vendor-customer relationship within the scope of Accounting Standards Codification, or ASC, Topic 606, Revenue from Contracts with Customers We also concluded that the co-development activities, the co-commercialization activities and the co-medical affairs activities are within the scope of ASC Topic 808, Collaborative Arrangements |
Collaborative Arrangements and
Collaborative Arrangements and Licensing Agreements | 3 Months Ended |
Mar. 31, 2024 | |
Collaborative Arrangements and Licensing Agreements [Abstract] | |
Collaborative Arrangements and Licensing Agreements | 5. Collaborative Arrangements and Licensing Agreements Below, we have included our AstraZeneca and Biogen collaborations, which are the collaborations with substantive changes during 2024 from those included in Part IV, Item 15, Note 4, Collaborative Arrangements and Licensing Agreements , of our audited financial statements included in our Annual Report on Form 10-K for the year ended December 31, 2023. AstraZeneca We have collaborations with AstraZeneca, focused on the joint development and commercialization of WAINUA for the treatment of transthyretin amyloidosis, or ATTR, and focused on the treatment of cardiovascular, renal and metabolic diseases. From inception through , we have received nearly $ million from these collaborations. In January 2024, we and AstraZeneca launched WAINUA in the U.S. for the treatment of adults with polyneuropathy caused by hereditary transthyretin amyloidosis, or ATTRv-PN. As a result, we began earning royalties from WAINUA sales, which we recognize as commercial revenue in our condensed consolidated statements of operations. We will achieve the next payment of up to $30 million upon regulatory approval of WAINUA for ATTRv-PN in the European Union, or EU, under this collaboration. During the three months ended March 31, 2024 and 2023, we earned the following revenue from our relationship with AstraZeneca (in thousands, except percentage amounts): Three Months Ended March 31, 2024 2023 Revenue from our relationship with AstraZeneca $ 11,685 $ 24,425 Percentage of total revenue 10 % 19 % Our condensed consolidated balance sheet at March 31, 2024 included deferred contract revenue of $1.9 million from our relationship with AstraZeneca. We did not have any deferred contract revenue from our relationship with AstraZeneca at December 31, 2023. Biogen We have several strategic collaborations with Biogen focused on using antisense technology to advance the treatment of neurological disorders. We developed and licensed to Biogen SPINRAZA, our approved medicine to treat people with spinal muscular atrophy, or SMA. QALSODY, our medicine to treat patients with superoxide dismutase 1 amyotrophic lateral sclerosis, or SOD1-ALS, received accelerated approval in the U.S. in April 2023. In addition, we and Biogen are currently developing numerous other investigational medicines to treat neurodegenerative diseases, including medicines in development to treat people with amyotrophic lateral sclerosis, or ALS, SMA, Angelman Syndrome, or AS, Alzheimer’s disease, or AD, and Parkinson’s disease, or PD. In addition to these medicines, our collaborations with Biogen include a substantial research pipeline that addresses a broad range of neurological diseases. From inception through March 31, 2024, we have received more than $3.8 billion in payments from our Biogen collaborations, including payments to purchase our stock. During the three months ended March 31, 2024 and 2023, we earned the following revenue from our relationship with Biogen (in thousands, except percentage amounts): Three Months Ended March 31, 2024 2023 Revenue from our relationship with Biogen $ 59,236 $ 70,501 Percentage of total revenue 50 % 54 % Our condensed consolidated balance sheets at March 31, 2024 and December 31, 2023 |
Basic and Diluted Net Loss Per
Basic and Diluted Net Loss Per Share | 3 Months Ended |
Mar. 31, 2024 | |
Basic and Diluted Net Loss Per Share [Abstract] | |
Basic and Diluted Net Loss Per Share | 6. Basic and Diluted Net Loss Per Share Basic net loss per share We calculated our basic net loss per share for the three months ended March 31, 2024 and 2023 by dividing our net loss by our weighted-average number of common shares outstanding during the period. Diluted net loss per share For the three months ended March 31, 2024 and 2023, we incurred a net loss; therefore, we did not include dilutive common equivalent shares in the computation of diluted net loss per share because the effect would have been anti-dilutive. Common stock from the following would have had an anti-dilutive effect on net loss per share: ● 0 percent convertible senior notes, or 0% Notes; ● Note hedges related to the 0% Notes; ● 0.125 percent convertible senior notes, or 0.125% Notes; ● Note hedges related to the 0.125% Notes; ● Dilutive stock options; ● Unvested restricted stock units, or RSUs; ● Unvested performance restricted stock units, or PRSUs; and ● Employee Stock Purchase Plan, or ESPP. For the three months ended March 31, 2024, common stock underlying the 1.75 percent convertible senior notes, or Notes Additionally as of March 31, 2024 and 2023, we had warrants related to our 0% and 0.125% Notes outstanding. We will include the shares issuable under these warrants in our calculation of diluted earnings per share when the average market price per share of our common stock for the reporting period exceeds the strike price of the warrants. |
Investments
Investments | 3 Months Ended |
Mar. 31, 2024 | |
Investments [Abstract] | |
Investments | 7. Investments The following table summarizes the contract maturity of the available-for-sale securities we held as of March 31, 2024: One year 69 % After one year two years 26 % After two years three and a half years 5 % Total 100 % As illustrated above, at March 31, 2024, 95 percent of our available-for-sale securities had a maturity of less than two years. All of our available-for-sale debt securities are available to us for use in our current operations. As a result, we categorize all of these securities as current assets even though the stated maturity of some individual securities may be one year or more beyond the balance sheet date. We invest in debt securities At March 31, 2024, we had an equity ownership interest of less than 20 percent in seven private companies and three public companies with which we conduct business. The following is a summary of our investments (in thousands): Amortized Gross Unrealized Estimated March 31, 2024 Cost Gains Losses Fair Value Available-for-sale debt securities: Corporate debt securities (1) $ 529,593 $ 87 $ (1,990 ) $ 527,690 Debt securities issued by U.S. government agencies 179,632 24 (409 ) 179,247 Debt securities issued by the U.S. Treasury (1) 582,782 38 (1,372 ) 581,448 Debt securities issued by states of the U.S. and political subdivisions of the states 15,017 29 (77 ) 14,969 Total debt securities with a maturity of one year or less 1,307,024 178 (3,848 ) 1,303,354 Corporate debt securities 248,548 405 (890 ) 248,063 Debt securities issued by U.S. government agencies 115,152 134 (187 ) 115,099 Debt securities issued by the U.S. Treasury 204,535 133 (1,086 ) 203,582 Debt securities issued by states of the U.S. and political subdivisions of the states 1,420 — (3 ) 1,417 Total debt securities with a maturity of more than one year 569,655 672 (2,166 ) 568,161 Total available-for-sale debt securities $ 1,876,679 $ 850 $ (6,014 ) $ 1,871,515 Equity securities: Publicly traded equity securities included in other current assets (2) $ 11,897 $ 319 $ (3,756 ) $ 8,460 Privately held equity securities included in deposits and other assets (3) 23,115 25,001 (5,125 ) 42,991 Total equity securities 35,012 25,320 (8,881 ) 51,451 Total available-for-sale debt and equity securities $ 1,911,691 $ 26,170 $ (14,895 ) $ 1,922,966 Amortized Gross Unrealized Estimated December 31, 2023 Cost Gains Losses Fair Value Available-for-sale debt securities: Corporate debt securities (1) $ 559,967 $ 157 $ (2,625 ) $ 557,499 Debt securities issued by U.S. government agencies 224,711 64 (611 ) 224,164 Debt securities issued by the U.S. Treasury (1) 513,784 152 (1,889 ) 512,047 Debt securities issued by states of the U.S. and political subdivisions of the states 17,757 42 (113 ) 17,686 Total debt securities with a maturity of one year or less 1,316,219 415 (5,238 ) 1,311,396 Corporate debt securities 243,151 1,270 (692 ) 243,729 Debt securities issued by U.S. government agencies 110,138 547 (21 ) 110,664 Debt securities issued by the U.S. Treasury 294,873 1,239 (480 ) 295,632 Debt securities issued by states of the U.S. and political subdivisions of the states 3,466 7 (4 ) 3,469 Total debt securities with a maturity of more than one year 651,628 3,063 (1,197 ) 653,494 Total available-for-sale debt securities $ 1,967,847 $ 3,478 $ (6,435 ) $ 1,964,890 Equity securities: Publicly traded equity securities included in other current assets (2) $ 11,897 $ 236 $ (5,832 ) $ 6,301 Privately held equity securities included in deposits and other assets (3) 23,115 25,001 (5,125 ) 42,991 Total equity securities 35,012 25,237 (10,957 ) 49,292 Total available-for-sale debt and equity securities $ 2,002,859 $ 28,715 $ (17,392 ) $ 2,014,182 (1) Includes investments classified as cash equivalents in our condensed consolidated balance sheets. (2) Our publicly traded equity securities are included in other current assets. We recognize publicly traded equity securities at fair value. In the three months ended March 31, 2024, we recorded a $2.2 million net unrealized gain in our condensed consolidated statements of operations related to changes in the fair value of our investments in publicly traded companies. (3) Our privately held equity securities are included in deposits and other assets. We recognize our privately held equity securities at . In the , there were changes in the fair value of our investments in privately held companies. The following is a summary of our investments we consider to be temporarily impaired at March 31, 2024 (in thousands, except for number of investments): Less than 12 Months of Temporary Impairment More than 12 Months of Temporary Impairment Total Temporary Impairment Number of Investments Estimated Fair Value Unrealized Losses Estimated Fair Value Unrealized Losses Estimated Fair Value Unrealized Losses Corporate debt securities 392 $ 487,939 $ (1,361 ) $ 96,219 $ (1,519 ) $ 584,158 $ (2,880 ) Debt securities issued by U.S. government agencies 80 176,956 (360 ) 12,375 (236 ) 189,331 (596 ) Debt securities issued by the U.S. Treasury 64 588,958 (1,773 ) 86,772 (685 ) 675,730 (2,458 ) Debt securities issued by states of the U.S. and political subdivisions of the states 53 7,978 (22 ) 5,703 (58 ) 13,681 (80 ) Total temporarily impaired securities 589 $ 1,261,831 $ (3,516 ) $ 201,069 $ (2,498 ) $ 1,462,900 $ (6,014 ) We believe that the decline in value of these securities is temporary and is primarily related to the change in market interest rates since purchase rather than underlying credit deterioration for any of the issuers. We believe it is more likely than not that we will be able to hold our debt securities with declines in value to maturity. Therefore, we intend to hold these securities to maturity and anticipate full recovery of our debt securities’ amortized cost basis at maturity. |
Fair Value Measurements
Fair Value Measurements | 3 Months Ended |
Mar. 31, 2024 | |
Fair Value Measurements [Abstract] | |
Fair Value Measurements | 8. Fair Value Measurements The following tables present the major security types we held at March 31, 2024 and December 31, 2023 that we regularly measure and carry at fair value. The following tables segregate each security type by the level within the fair value hierarchy of the valuation techniques we utilized to determine the respective security’s fair value (in thousands): At March 31, 2024 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Cash equivalents (1) $ 263,639 $ 263,639 $ — Corporate debt securities (2) 775,753 — 775,753 Debt securities issued by U.S. government agencies (3) 294,346 — 294,346 Debt securities issued by the U.S. Treasury (3) 785,030 785,030 — Debt securities issued by states of the U.S. and political subdivisions of the states (3) 16,386 — 16,386 Publicly traded equity securities included in other current assets (4) 8,460 8,460 — Total $ 2,143,614 $ 1,057,129 $ 1,086,485 At December 31, 2023 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Cash equivalents (1) $ 185,424 $ 185,424 $ — Corporate debt securities (5) 801,228 — 801,228 Debt securities issued by U.S. government agencies (3) 334,828 — 334,828 Debt securities issued by the U.S. Treasury (3) 807,679 807,679 — Debt securities issued by states of the U.S. and political subdivisions of the states (3) 21,155 — 21,155 Publicly traded equity securities included in other current assets (4) 6,301 6,301 — Total $ 2,156,615 $ 999,404 $ 1,157,211 The following footnotes reference lines in our condensed consolidated balance sheets: (1) Included in cash and cash equivalents. (2) $14.2 million was included in cash and cash equivalents, with the difference included in short-term investments. (3) Included in short-term investments. (4) Included in other current assets. (5) $33.0 million was included in cash and cash equivalents, with the difference included in short-term investments. Convertible Notes Our 1.75% Notes, 0% Notes and 0.125% Notes had a fair value of $601.1 million, $627.8 million and $42.4 million at March 31, 2024, respectively. Our 1.75% Notes, 0% Notes and 0.125% Notes had a fair value of $661.1 million, $667.8 million and $42.4 million at December 31, 2023, respectively. We determine the fair value of our notes based on quoted market prices for these notes, which are Level 2 measurements because the notes do not trade regularly. |
Stock-based Compensation Expens
Stock-based Compensation Expense | 3 Months Ended |
Mar. 31, 2024 | |
Stock-based Compensation Expense [Abstract] | |
Stock-based Compensation Expense | 9. Stock-based Compensation Expense The following table summarizes stock-based compensation expense for the three months ended March 31, 2024 and 2023 (in thousands): Three Months Ended March 31, 2024 2023 Cost of sales $ 204 $ 119 Research, development and patent expense 22,225 19,567 Selling, general and administrative expense 8,911 7,262 Total $ 31,340 $ 26,948 As of March 31, 2024, total unrecognized estimated stock-based compensation expense related to non-vested stock options, RSUs and PRSUs was $71.1 million, $112.8 million and $16.8 million, respectively. Our actual expenses may differ from these estimates because we will adjust our unrecognized stock-based compensation expense for future forfeitures, including any PRSUs that do not vest. We expect to recognize the cost of stock-based compensation expense related to our non-vested stock options, RSUs and PRSUs over a weighted average amortization period of 1.4 years, 1.8 years and 2.2 years, respectively. Refer to Part IV, Item 15, Note 1, Organization and Significant Accounting Policies For the three months ended March 31, 2024 and 2023, we used the following weighted-average assumptions in our Black-Scholes calculations: Employee Stock Options: Three Months Ended March 31, 2024 2023 Risk-free interest rate 4.0 % 3.6 % Dividend yield 0.0 % 0.0 % Volatility 44.0 % 47.5 % Expected life 6.3 years 6.3 years ESPP: Three Months Ended March 31, 2024 2023 Risk-free interest rate 5.3 % 5.2 % Dividend yield 0.0 % 0.0 % Volatility 38.4 % 36.7 % Expected life 6 months 6 months RSUs: The weighted-average grant date fair value of RSUs granted to employees for the three months ended March 31, 2024 and 2023 was $53.54 and $39.85 per share, respectively. PRSUs: Under the terms of the PRSUs we granted in 2024 and 2023, 100 percent of the PRSUs may vest at the end of the three-year performance period based on our relative TSR as compared to a peer group of companies and as measured at the end of the performance period. Under the terms of the grants, no number of PRSUs is guaranteed to vest and the actual number of PRSUs that will vest at the end of each performance period may be anywhere from zero to 200 percent of the target number depending on our relative TSR. The weighted-average grant date fair value of PRSUs granted to our executive officers for the three months ended March 31, 2024 and 2023 was $78.41 and $58.99 per share, respectively. |
Income Taxes
Income Taxes | 3 Months Ended |
Mar. 31, 2024 | |
Income Taxes [Abstract] | |
Income Taxes | 10. Income Taxes We recorded income tax expense of $0.1 million for the three months ended March 31, 2024 compared to $11.4 million for the same period in 2023. The decrease in income tax expense relates primarily to the impact of the royalty purchase agreement with Royalty Pharma on income tax expense for the three months ended March 31, 2023. We reflected the Royalty Pharma transaction as a taxable sale, which required us to include the proceeds from the sale, net of currently deductible issuance costs, as taxable income in 2023. We continue to maintain a full valuation allowance on all of our net deferred tax assets. |
Liability Related to Sale of Fu
Liability Related to Sale of Future Royalties | 3 Months Ended |
Mar. 31, 2024 | |
Liability Related to Sale of Future Royalties [Abstract] | |
Liability Related to Sale of Future Royalties | 11. Liability Related to Sale of Future Royalties In January 2023, we entered into a royalty purchase agreement with Royalty Pharma to monetize a portion of our future SPINRAZA and pelacarsen royalties we are entitled to under our arrangements with Biogen and Novartis, respectively. As a result, we received an upfront payment of $500 million and we are eligible to receive up to $625 million in additional milestone payments. Under the terms of the agreement, Royalty Pharma will receive 25 percent of our SPINRAZA royalty payments from 2023 through 2027, increasing to 45 percent of royalty payments in 2028, on up to $1.5 billion in annual sales. In addition, Royalty Pharma will receive 25 percent of any future royalty payments on pelacarsen, our medicine in development to treat patients with elevated lipoprotein(a)-driven cardiovascular disease. Royalty Pharma’s royalty interest in SPINRAZA will revert to us after total SPINRAZA royalty payments to Royalty Pharma reach either $475 million or $550 million, depending on the timing and occurrence of FDA approval of pelacarsen. We recorded the upfront payment of $500 million as a liability related to the sale of future royalties, net of transaction costs of $10.4 million, which we are amortizing over the estimated life of the arrangement using the effective interest rate method. We recognize royalty revenue in the period in which the counterparty sells the related product and recognizes the related revenue. We record royalty payments made to Royalty Pharma as a reduction of the liability. We determine the effective interest rate used to record interest expense under this agreement based on an estimate of future royalty payments to Royalty Pharma. As of March 31, 2024, the estimated effective interest rate under the agreement was 13.5 percent. The following table sets forth information on our liability related to sale of future royalties (in thousands): Proceeds from sale of future royalties in January 2023 $ 500,000 Issuance costs related to sale of future royalties (10,434 ) Royalty payments to Royalty Pharma (44,628 ) Interest expense related to sale of future royalties 68,238 Amortization of issuance costs related to sale of future royalties 560 Net liability related to sale of future royalties as of December 31, 2023 513,736 Royalty payments to Royalty Pharma (6,623 ) Interest expense related to sale of future royalties 17,806 Amortization of issuance costs related to sale of future royalties 153 Net liability related to sale of future royalties as of March 31, 2024 $ 525,072 There are numerous factors, most of which are not within our control, that could materially impact the amount and timing of royalty payments from Biogen and Novartis, and result in changes to our estimate of future royalty payments to Royalty Pharma. Such factors include, but are not limited to, the commercial sales of SPINRAZA, the regulatory approval and commercial sales of pelacarsen, competing products or other significant events. |
Convertible Debt
Convertible Debt | 3 Months Ended |
Mar. 31, 2024 | |
Convertible Debt [Abstract] | |
Convertible Debt | 12. Convertible Debt 1.75 Percent Convertible Senior Notes In 2023, we completed a $575.0 million offering of convertible senior notes and used $488.2 million of the net proceeds from the issuance of the 1.75% Notes to repurchase $504.4 million in principal of our 0.125% Notes. We expect to use the remaining net proceeds to settle the 0.125% Notes that remain outstanding. At March 31, 2024, we had the following 1.75% Notes outstanding (in millions except interest rate and price per share data): 1.75% Notes Outstanding principal balance $ 575.0 Unamortized debt issuance costs $ 12.0 Maturity date June 2028 Interest rate 1.75 % Effective interest rate 2.3 % Conversion price per share $ 53.73 Total shares of common stock subject to conversion 10.7 0 Percent Convertible Senior Notes and Call Spread At March 31, 2024, we had the following 0% 0% Notes Outstanding principal balance $ 632.5 Unamortized debt issuance costs $ 6.3 Maturity date April 2026 Interest rate 0 % Effective interest rate 0.5 % Conversion price per share $ 57.84 Effective conversion price per share with call spread $ 76.39 Total shares of common stock subject to conversion 10.9 In conjunction with the 2021 offering, we entered into a call spread transaction, which was comprised of purchasing note hedges and selling warrants, to minimize the impact of potential economic dilution upon conversion of our 0% 0% 0% 0% 0% 0% 0% 0% We recorded the amount we paid for the note hedges and the amount we received for the warrants in additional paid-in capital in our condensed consolidated balance sheets. Refer to Part IV, Item 15, Note 1, Organization and Significant Accounting Policies 0.125 Percent Convertible Senior Notes and Call Spread As discussed above, in 2023, we repurchased $504.4 million of our 0.125% Notes. At March 31, 2024, we had the following 0.125% Notes outstanding with interest payable semi-annually (in millions except interest rate and price per share data): 0.125% Notes Outstanding principal balance $ 44.5 Unamortized debt issuance costs $ 0.1 Maturity date December 2024 Interest rate 0.125 % Effective interest rate 0.5 % Conversion price per share $ 83.28 Effective conversion price per share with call spread $ 123.38 Total shares of common stock subject to conversion, excluding shares related to 0.125 0.5 In conjunction with the issuance of our Notes in 2019, we entered into a call spread transaction, which was comprised of purchasing note hedges and selling warrants, to minimize the impact of potential economic dilution upon conversion of our . We increased our effective conversion price to $ with the same number of underlying shares as our Notes. The call spread cost us $ million, of which $ million was for the note hedge purchase, offset by $ million we received for selling the warrants. Similar to our , our note hedges are subject to adjustment. Additionally, our note hedges are exercisable upon conversion of the . The note hedges will expire upon maturity of the , or December 2024. The note hedges and warrants are separate transactions and are not part of the terms of our . The holders of the do not have any rights with respect to the note hedges and warrants. As of , the note hedges and warrants remain outstanding. We recorded the amount we paid for the note hedges and the amount we received for the warrants in additional paid-in capital in our condensed consolidated balance sheets. We reassess our ability to continue to classify the note hedges and warrants in shareholders’ equity at each reporting period. Other Terms of Convertible Senior Notes The , 0% and Notes are convertible under certain conditions, at the option of the note holders. We can settle conversions of the notes, at our election, in cash, shares of our common stock or a combination of both. We may not redeem the notes prior to maturity, and we do not have to provide a sinking fund for them. Holders of the notes may require us to purchase some or all of their notes upon the occurrence of certain fundamental changes, as set forth in the indentures governing the notes, at a purchase price equal to of the principal amount of the notes to be purchased, plus any accrued and unpaid interest. |
Legal Proceedings
Legal Proceedings | 3 Months Ended |
Mar. 31, 2024 | |
Legal Proceedings [Abstract] | |
Legal Proceedings | 13. Legal Proceedings From time to time, we are involved in legal proceedings arising in the ordinary course of our business. Periodically, we evaluate the status of each legal matter and assess our potential financial exposure. If we consider the potential loss from any legal proceeding to be probable and we can reasonably estimate the amount, we accrue a liability for the estimated loss. The outcome of any proceeding is not determinable in advance. Therefore, we are required to use significant judgment to determine the probability of a loss and whether the amount of the loss is reasonably estimable. Our assessment of a potential liability and the amount of accruals we recorded are based only on the information available to us at the time. As additional information becomes available, we reassess the potential liability related to the legal proceeding and may revise our estimates. There are no pending material legal proceedings to which we are a party or of which our property is the subject. |
Insider Trading Arrangements
Insider Trading Arrangements | 3 Months Ended |
Mar. 31, 2024 shares | |
Trading Arrangements, by Individual | |
Material Terms of Trading Arrangement | During the quarter ended March 31, 2024, our Section 16 officers and directors adopted or terminated contracts, instructions or written plans for the purchase or sale of our securities as noted in the table below. * Contract, instruction or written plan intended to satisfy the affirmative defense conditions of Rule 10b5-1(c) under the Exchange Act. ** “Non-Rule 10b5-1 trading arrangement” as defined in item 408(c) of Regulation S-K under the Exchange Act. Action Date Trading Arrangement Total Shares to Be Sold Expiration Date Rule 10b5-1* Non-Rule 10b5-1** B. Lynne Parshall Board Member Termination January 3, 2024 X 122,638 Upon the execution of all instructions provided in the plan Joseph Klein, III Board Member Adoption January 3, 2024 X 82,000 The earlier to occur of (i) December 31, 2025 Joseph Baroldi EVP, Chief Business Officer Adoption January 5, 2024 X 40,000 The earlier to occur of (i) March 31, 2025 Eugene Schneider EVP, Chief Clinical Development and Operations Officer Adoption January 12, 2024 X 40,633 The earlier to occur of (i) July 29, 2025 Elizabeth Hougen EVP, Finance & Chief Financial Officer Adoption January 12, 2024 X 178,200 The earlier to occur of (i) October 11, 2025 Patrick O'Neil Chief Legal Officer & General Counsel Termination February 14, 2024 X 170,830 Upon the execution of all instructions provided in the plan B. Lynne Parshall Board Member Adoption February 21, 2024 X 145,568 The earlier to occur of (i) June 30, 2025 Patrick O'Neil Chief Legal Officer & General Counsel Adoption February 21, 2024 X 252,747 The earlier to occur of (i) May 31, 2025 |
B. Lynne Parshall [Member] | |
Trading Arrangements, by Individual | |
Name | B. Lynne Parshall |
Title | Board Member |
Rule 10b5-1 Arrangement Adopted | true |
Non-Rule 10b5-1 Arrangement Adopted | false |
Adoption Date | February 21, 2024 |
Rule 10b5-1 Arrangement Terminated | true |
Non-Rule 10b5-1 Arrangement Terminated | false |
Termination Date | January 3, 2024 |
Joseph Klein, III [Member] | |
Trading Arrangements, by Individual | |
Name | Joseph Klein, III |
Title | Board Member |
Rule 10b5-1 Arrangement Adopted | true |
Non-Rule 10b5-1 Arrangement Adopted | false |
Adoption Date | January 3, 2024 |
Arrangement Duration | 729 days |
Aggregate Available | 82,000 |
Joseph Baroldi [Member] | |
Trading Arrangements, by Individual | |
Name | Joseph Baroldi |
Title | EVP, Chief Business Officer |
Rule 10b5-1 Arrangement Adopted | true |
Non-Rule 10b5-1 Arrangement Adopted | false |
Adoption Date | January 5, 2024 |
Arrangement Duration | 452 days |
Aggregate Available | 40,000 |
Eugene Schneider [Member] | |
Trading Arrangements, by Individual | |
Name | Eugene Schneider |
Title | EVP, Chief Clinical Development and Operations Officer |
Rule 10b5-1 Arrangement Adopted | true |
Non-Rule 10b5-1 Arrangement Adopted | false |
Adoption Date | January 12, 2024 |
Arrangement Duration | 565 days |
Aggregate Available | 40,633 |
Elizabeth Hougen [Member] | |
Trading Arrangements, by Individual | |
Name | Elizabeth Hougen |
Title | EVP, Finance & Chief Financial Officer |
Rule 10b5-1 Arrangement Adopted | true |
Non-Rule 10b5-1 Arrangement Adopted | false |
Adoption Date | January 12, 2024 |
Arrangement Duration | 639 days |
Aggregate Available | 178,200 |
Patrick O'Neil [Member] | |
Trading Arrangements, by Individual | |
Name | Patrick O'Neil |
Title | Chief Legal Officer & General Counsel |
Rule 10b5-1 Arrangement Adopted | true |
Non-Rule 10b5-1 Arrangement Adopted | false |
Adoption Date | February 21, 2024 |
Rule 10b5-1 Arrangement Terminated | true |
Non-Rule 10b5-1 Arrangement Terminated | false |
Termination Date | February 14, 2024 |
Termination [Member] | B. Lynne Parshall [Member] | |
Trading Arrangements, by Individual | |
Arrangement Duration | |
Aggregate Available | 122,638 |
Termination [Member] | Patrick O'Neil [Member] | |
Trading Arrangements, by Individual | |
Arrangement Duration | |
Aggregate Available | 170,830 |
Adoption [Member] | B. Lynne Parshall [Member] | |
Trading Arrangements, by Individual | |
Arrangement Duration | 496 days |
Aggregate Available | 145,568 |
Adoption [Member] | Patrick O'Neil [Member] | |
Trading Arrangements, by Individual | |
Arrangement Duration | 466 days |
Aggregate Available | 252,747 |
Organization and Basis of Pre_2
Organization and Basis of Presentation (Policies) | 3 Months Ended |
Mar. 31, 2024 | |
Organization and Basis of Presentation [Abstract] | |
Basis of Presentation | We prepared the unaudited interim condensed consolidated financial statements for the three months ended March 31, 2024 and 2023 on the same basis as the audited financial statements for the year ended December 31, 2023. We included all normal recurring adjustments in the financial statements, which we considered necessary for a fair presentation of our financial position at such dates and our operating results and cash flows for those periods. Our operating results for the interim periods may not be indicative of what our operating results will be for the entire year. For more complete financial information, these financial statements, and notes thereto, should be read in conjunction with the audited financial statements for the year ended December 31, 2023 included in our Annual Report on Form 10-K filed with the Securities and Exchange Commission, or SEC. |
Consolidation | In our condensed consolidated financial statements, we included the accounts of Ionis Pharmaceuticals, Inc. and the consolidated results of our wholly owned subsidiary, Akcea Therapeutics, Inc. and its wholly owned subsidiaries (“we”, “us” or “our”). |
Segment Information | We operate as a single single |
Use of Estimates | Use of Estimates We prepare our condensed consolidated financial statements in conformity with accounting principles generally accepted in the United States, or |
Significant Accounting Polici_2
Significant Accounting Policies (Policies) | 3 Months Ended |
Mar. 31, 2024 | |
Significant Accounting Policies [Abstract] | |
Recently Adopted Accounting Standards | Recently Adopted Accounting Standards We do not expect any recently issued accounting standards to have a material impact to our financial results. |
Basic and Diluted Net Loss Pe_2
Basic and Diluted Net Loss Per Share (Policies) | 3 Months Ended |
Mar. 31, 2024 | |
Basic and Diluted Net Loss Per Share [Abstract] | |
Basic and Diluted Net Loss Per Share | Basic net loss per share We calculated our basic net loss per share for the three months ended March 31, 2024 and 2023 by dividing our net loss by our weighted-average number of common shares outstanding during the period. Diluted net loss per share For the three months ended March 31, 2024 and 2023, we incurred a net loss; therefore, we did not include dilutive common equivalent shares in the computation of diluted net loss per share because the effect would have been anti-dilutive. Common stock from the following would have had an anti-dilutive effect on net loss per share: ● 0 percent convertible senior notes, or 0% Notes; ● Note hedges related to the 0% Notes; ● 0.125 percent convertible senior notes, or 0.125% Notes; ● Note hedges related to the 0.125% Notes; ● Dilutive stock options; ● Unvested restricted stock units, or RSUs; ● Unvested performance restricted stock units, or PRSUs; and ● Employee Stock Purchase Plan, or ESPP. For the three months ended March 31, 2024, common stock underlying the 1.75 percent convertible senior notes, or Notes Additionally as of March 31, 2024 and 2023, we had warrants related to our 0% and 0.125% Notes outstanding. We will include the shares issuable under these warrants in our calculation of diluted earnings per share when the average market price per share of our common stock for the reporting period exceeds the strike price of the warrants. |
Supplemental Financial Data (Ta
Supplemental Financial Data (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Supplemental Financial Data [Abstract] | |
Inventories | Inventories Our inventory consisted of the following (in thousands): March 31, 2024 December 31, 2023 Raw materials: Raw materials - clinical $ 23,317 $ 20,985 Raw materials - commercial 705 1,809 Total raw materials 24,022 22,794 Work in process 6,037 5,477 Finished goods 200 154 Total inventories $ 30,259 $ 28,425 |
Accrued Liabilities | Accrued Liabilities Our accrued liabilities consisted of the following (in thousands): March 31, 2024 December 31, 2023 Clinical development expenses $ 75,929 $ 105,967 In-licensing expenses 6,436 7,454 Commercial expenses 4,739 4,875 Other miscellaneous expenses 18,944 29,598 Total accrued liabilities $ 106,048 $ 147,894 |
Revenues (Tables)
Revenues (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Revenues [Abstract] | |
Revenues | During the three months ended March 31, 2024 and 2023, our revenues were comprised of the following (in thousands): Three Months Ended March 31, 2024 2023 Revenue: Commercial revenue: SPINRAZA royalties $ 38,455 $ 50,247 WAINUA royalties 1,125 — Other commercial revenue: TEGSEDI and WAYLIVRA revenue, net 8,628 6,478 Licensing and other royalty revenue 11,385 11,043 Total other commercial revenue 20,013 17,521 Total commercial revenue 59,593 67,768 Research and development revenue: Collaborative agreement revenue 49,345 38,334 WAINUA joint development revenue 10,559 24,422 Total research and development revenue 59,904 62,756 Total revenue $ 119,497 $ 130,524 |
Collaborative Arrangements an_2
Collaborative Arrangements and Licensing Agreements (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
AstraZeneca [Member] | |
Collaborative Arrangements and Licensing Agreements [Abstract] | |
Revenue from Collaborative Relationship | During the three months ended March 31, 2024 and 2023, we earned the following revenue from our relationship with AstraZeneca (in thousands, except percentage amounts): Three Months Ended March 31, 2024 2023 Revenue from our relationship with AstraZeneca $ 11,685 $ 24,425 Percentage of total revenue 10 % 19 % |
Biogen [Member] | |
Collaborative Arrangements and Licensing Agreements [Abstract] | |
Revenue from Collaborative Relationship | During the three months ended March 31, 2024 and 2023, we earned the following revenue from our relationship with Biogen (in thousands, except percentage amounts): Three Months Ended March 31, 2024 2023 Revenue from our relationship with Biogen $ 59,236 $ 70,501 Percentage of total revenue 50 % 54 % |
Investments (Tables)
Investments (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Investments [Abstract] | |
Contract Maturity of Available-for-Sale Securities | The following table summarizes the contract maturity of the available-for-sale securities we held as of March 31, 2024: One year 69 % After one year two years 26 % After two years three and a half years 5 % Total 100 % |
Summary of Investments | The following is a summary of our investments (in thousands): Amortized Gross Unrealized Estimated March 31, 2024 Cost Gains Losses Fair Value Available-for-sale debt securities: Corporate debt securities (1) $ 529,593 $ 87 $ (1,990 ) $ 527,690 Debt securities issued by U.S. government agencies 179,632 24 (409 ) 179,247 Debt securities issued by the U.S. Treasury (1) 582,782 38 (1,372 ) 581,448 Debt securities issued by states of the U.S. and political subdivisions of the states 15,017 29 (77 ) 14,969 Total debt securities with a maturity of one year or less 1,307,024 178 (3,848 ) 1,303,354 Corporate debt securities 248,548 405 (890 ) 248,063 Debt securities issued by U.S. government agencies 115,152 134 (187 ) 115,099 Debt securities issued by the U.S. Treasury 204,535 133 (1,086 ) 203,582 Debt securities issued by states of the U.S. and political subdivisions of the states 1,420 — (3 ) 1,417 Total debt securities with a maturity of more than one year 569,655 672 (2,166 ) 568,161 Total available-for-sale debt securities $ 1,876,679 $ 850 $ (6,014 ) $ 1,871,515 Equity securities: Publicly traded equity securities included in other current assets (2) $ 11,897 $ 319 $ (3,756 ) $ 8,460 Privately held equity securities included in deposits and other assets (3) 23,115 25,001 (5,125 ) 42,991 Total equity securities 35,012 25,320 (8,881 ) 51,451 Total available-for-sale debt and equity securities $ 1,911,691 $ 26,170 $ (14,895 ) $ 1,922,966 Amortized Gross Unrealized Estimated December 31, 2023 Cost Gains Losses Fair Value Available-for-sale debt securities: Corporate debt securities (1) $ 559,967 $ 157 $ (2,625 ) $ 557,499 Debt securities issued by U.S. government agencies 224,711 64 (611 ) 224,164 Debt securities issued by the U.S. Treasury (1) 513,784 152 (1,889 ) 512,047 Debt securities issued by states of the U.S. and political subdivisions of the states 17,757 42 (113 ) 17,686 Total debt securities with a maturity of one year or less 1,316,219 415 (5,238 ) 1,311,396 Corporate debt securities 243,151 1,270 (692 ) 243,729 Debt securities issued by U.S. government agencies 110,138 547 (21 ) 110,664 Debt securities issued by the U.S. Treasury 294,873 1,239 (480 ) 295,632 Debt securities issued by states of the U.S. and political subdivisions of the states 3,466 7 (4 ) 3,469 Total debt securities with a maturity of more than one year 651,628 3,063 (1,197 ) 653,494 Total available-for-sale debt securities $ 1,967,847 $ 3,478 $ (6,435 ) $ 1,964,890 Equity securities: Publicly traded equity securities included in other current assets (2) $ 11,897 $ 236 $ (5,832 ) $ 6,301 Privately held equity securities included in deposits and other assets (3) 23,115 25,001 (5,125 ) 42,991 Total equity securities 35,012 25,237 (10,957 ) 49,292 Total available-for-sale debt and equity securities $ 2,002,859 $ 28,715 $ (17,392 ) $ 2,014,182 (1) Includes investments classified as cash equivalents in our condensed consolidated balance sheets. (2) Our publicly traded equity securities are included in other current assets. We recognize publicly traded equity securities at fair value. In the three months ended March 31, 2024, we recorded a $2.2 million net unrealized gain in our condensed consolidated statements of operations related to changes in the fair value of our investments in publicly traded companies. (3) Our privately held equity securities are included in deposits and other assets. We recognize our privately held equity securities at . In the , there were changes in the fair value of our investments in privately held companies. |
Temporarily Impaired Investments | The following is a summary of our investments we consider to be temporarily impaired at March 31, 2024 (in thousands, except for number of investments): Less than 12 Months of Temporary Impairment More than 12 Months of Temporary Impairment Total Temporary Impairment Number of Investments Estimated Fair Value Unrealized Losses Estimated Fair Value Unrealized Losses Estimated Fair Value Unrealized Losses Corporate debt securities 392 $ 487,939 $ (1,361 ) $ 96,219 $ (1,519 ) $ 584,158 $ (2,880 ) Debt securities issued by U.S. government agencies 80 176,956 (360 ) 12,375 (236 ) 189,331 (596 ) Debt securities issued by the U.S. Treasury 64 588,958 (1,773 ) 86,772 (685 ) 675,730 (2,458 ) Debt securities issued by states of the U.S. and political subdivisions of the states 53 7,978 (22 ) 5,703 (58 ) 13,681 (80 ) Total temporarily impaired securities 589 $ 1,261,831 $ (3,516 ) $ 201,069 $ (2,498 ) $ 1,462,900 $ (6,014 ) |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Fair Value Measurements [Abstract] | |
Assets Measured at Fair Value on a Recurring Basis | The following tables present the major security types we held at March 31, 2024 and December 31, 2023 that we regularly measure and carry at fair value. The following tables segregate each security type by the level within the fair value hierarchy of the valuation techniques we utilized to determine the respective security’s fair value (in thousands): At March 31, 2024 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Cash equivalents (1) $ 263,639 $ 263,639 $ — Corporate debt securities (2) 775,753 — 775,753 Debt securities issued by U.S. government agencies (3) 294,346 — 294,346 Debt securities issued by the U.S. Treasury (3) 785,030 785,030 — Debt securities issued by states of the U.S. and political subdivisions of the states (3) 16,386 — 16,386 Publicly traded equity securities included in other current assets (4) 8,460 8,460 — Total $ 2,143,614 $ 1,057,129 $ 1,086,485 At December 31, 2023 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Cash equivalents (1) $ 185,424 $ 185,424 $ — Corporate debt securities (5) 801,228 — 801,228 Debt securities issued by U.S. government agencies (3) 334,828 — 334,828 Debt securities issued by the U.S. Treasury (3) 807,679 807,679 — Debt securities issued by states of the U.S. and political subdivisions of the states (3) 21,155 — 21,155 Publicly traded equity securities included in other current assets (4) 6,301 6,301 — Total $ 2,156,615 $ 999,404 $ 1,157,211 The following footnotes reference lines in our condensed consolidated balance sheets: (1) Included in cash and cash equivalents. (2) $14.2 million was included in cash and cash equivalents, with the difference included in short-term investments. (3) Included in short-term investments. (4) Included in other current assets. (5) $33.0 million was included in cash and cash equivalents, with the difference included in short-term investments. |
Stock-based Compensation Expe_2
Stock-based Compensation Expense (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Stock-based Compensation Expense [Abstract] | |
Stock-Based Compensation Expense | The following table summarizes stock-based compensation expense for the three months ended March 31, 2024 and 2023 (in thousands): Three Months Ended March 31, 2024 2023 Cost of sales $ 204 $ 119 Research, development and patent expense 22,225 19,567 Selling, general and administrative expense 8,911 7,262 Total $ 31,340 $ 26,948 |
Weighted-Average Assumptions for Stock Options | Employee Stock Options: Three Months Ended March 31, 2024 2023 Risk-free interest rate 4.0 % 3.6 % Dividend yield 0.0 % 0.0 % Volatility 44.0 % 47.5 % Expected life 6.3 years 6.3 years |
Weighted-Average Assumptions for ESPP | ESPP: Three Months Ended March 31, 2024 2023 Risk-free interest rate 5.3 % 5.2 % Dividend yield 0.0 % 0.0 % Volatility 38.4 % 36.7 % Expected life 6 months 6 months |
Liability Related to Sale of _2
Liability Related to Sale of Future Royalties (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Liability Related to Sale of Future Royalties [Abstract] | |
Liability Related to Sale of Future Royalties | The following table sets forth information on our liability related to sale of future royalties (in thousands): Proceeds from sale of future royalties in January 2023 $ 500,000 Issuance costs related to sale of future royalties (10,434 ) Royalty payments to Royalty Pharma (44,628 ) Interest expense related to sale of future royalties 68,238 Amortization of issuance costs related to sale of future royalties 560 Net liability related to sale of future royalties as of December 31, 2023 513,736 Royalty payments to Royalty Pharma (6,623 ) Interest expense related to sale of future royalties 17,806 Amortization of issuance costs related to sale of future royalties 153 Net liability related to sale of future royalties as of March 31, 2024 $ 525,072 |
Convertible Debt (Tables)
Convertible Debt (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
1.75% Notes [Member] | |
Convertible Debt [Abstract] | |
Convertible Senior Notes | At March 31, 2024, we had the following 1.75% Notes outstanding (in millions except interest rate and price per share data): 1.75% Notes Outstanding principal balance $ 575.0 Unamortized debt issuance costs $ 12.0 Maturity date June 2028 Interest rate 1.75 % Effective interest rate 2.3 % Conversion price per share $ 53.73 Total shares of common stock subject to conversion 10.7 |
0% Notes [Member] | |
Convertible Debt [Abstract] | |
Convertible Senior Notes | At March 31, 2024, we had the following 0% 0% Notes Outstanding principal balance $ 632.5 Unamortized debt issuance costs $ 6.3 Maturity date April 2026 Interest rate 0 % Effective interest rate 0.5 % Conversion price per share $ 57.84 Effective conversion price per share with call spread $ 76.39 Total shares of common stock subject to conversion 10.9 |
0.125% Notes [Member] | |
Convertible Debt [Abstract] | |
Convertible Senior Notes | At March 31, 2024, we had the following 0.125% Notes outstanding with interest payable semi-annually (in millions except interest rate and price per share data): 0.125% Notes Outstanding principal balance $ 44.5 Unamortized debt issuance costs $ 0.1 Maturity date December 2024 Interest rate 0.125 % Effective interest rate 0.5 % Conversion price per share $ 83.28 Effective conversion price per share with call spread $ 123.38 Total shares of common stock subject to conversion, excluding shares related to 0.125 0.5 |
Organization and Basis of Pre_3
Organization and Basis of Presentation (Details) | 3 Months Ended |
Mar. 31, 2024 Segment | |
Organization and Basis of Presentation [Abstract] | |
Number of operating segments | 1 |
Supplemental Financial Data, In
Supplemental Financial Data, Inventories (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Inventory [Abstract] | ||
Raw materials | $ 24,022 | $ 22,794 |
Work in process | 6,037 | 5,477 |
Finished goods | 200 | 154 |
Total inventories | 30,259 | 28,425 |
Clinical [Member] | ||
Inventory [Abstract] | ||
Raw materials | 23,317 | 20,985 |
Commercial [Member] | ||
Inventory [Abstract] | ||
Raw materials | $ 705 | $ 1,809 |
Supplemental Financial Data, Ac
Supplemental Financial Data, Accrued Liabilities (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Accrued Liabilities [Abstract] | ||
Clinical development expenses | $ 75,929 | $ 105,967 |
In-licensing expenses | 6,436 | 7,454 |
Commercial expenses | 4,739 | 4,875 |
Other miscellaneous expenses | 18,944 | 29,598 |
Total accrued liabilities | $ 106,048 | $ 147,894 |
Revenues (Details)
Revenues (Details) $ in Thousands | 3 Months Ended | 12 Months Ended | |
Mar. 31, 2024 USD ($) | Mar. 31, 2023 USD ($) | Dec. 31, 2021 USD ($) PerformanceObligation Component | |
Revenues [Abstract] | |||
Revenue | $ 119,497 | $ 130,524 | |
WAINUA (Eplontersen) Collaboration with AstraZeneca [Member] | |||
Revenues [Abstract] | |||
Upfront payment received | $ 200,000 | ||
Number of material components | Component | 4 | ||
Number of performance obligations | PerformanceObligation | 1 | ||
Transaction price | $ 200,000 | ||
Percentage of costs associated with ongoing global Phase 3 development program paid by AstraZeneca | 55% | ||
Commercial Revenue [Member] | |||
Revenues [Abstract] | |||
Revenue | $ 59,593 | 67,768 | |
SPINRAZA Royalties [Member] | |||
Revenues [Abstract] | |||
Revenue | 38,455 | 50,247 | |
WAINUA Royalties [Member] | |||
Revenues [Abstract] | |||
Revenue | 1,125 | 0 | |
Other Commercial Revenue [Member] | |||
Revenues [Abstract] | |||
Revenue | 20,013 | 17,521 | |
TEGSEDI and WAYLIVRA Revenue, Net [Member] | |||
Revenues [Abstract] | |||
Revenue | 8,628 | 6,478 | |
Licensing and Other Royalty Revenue [Member] | |||
Revenues [Abstract] | |||
Revenue | 11,385 | 11,043 | |
Research and Development Revenue [Member] | |||
Revenues [Abstract] | |||
Revenue | 59,904 | 62,756 | |
Collaborative Agreement Revenue [Member] | |||
Revenues [Abstract] | |||
Revenue | 49,345 | 38,334 | |
WAINUA Joint Development Revenue [Member] | |||
Revenues [Abstract] | |||
Revenue | $ 10,559 | $ 24,422 |
Collaborative Arrangements an_3
Collaborative Arrangements and Licensing Agreements, AstraZeneca (Details) $ in Thousands | 3 Months Ended | ||
Mar. 31, 2024 USD ($) Agreement | Mar. 31, 2023 USD ($) | Dec. 31, 2023 USD ($) | |
Collaborative Arrangement and Licensing Agreement [Abstract] | |||
Revenue | $ 119,497 | $ 130,524 | |
AstraZeneca [Member] | |||
Collaborative Arrangement and Licensing Agreement [Abstract] | |||
Number of collaboration agreements | Agreement | 2 | ||
Revenue | $ 11,685 | $ 24,425 | |
Deferred contract revenue | $ 1,900 | $ 0 | |
AstraZeneca [Member] | Revenue [Member] | Strategic Partner [Member] | |||
Collaborative Arrangement and Licensing Agreement [Abstract] | |||
Concentration percentage | 10% | 19% | |
AstraZeneca [Member] | Maximum [Member] | |||
Collaborative Arrangement and Licensing Agreement [Abstract] | |||
Cumulative payments received | $ 910,000 | ||
WAINUA [Member] | |||
Collaborative Arrangement and Licensing Agreement [Abstract] | |||
Number of collaboration agreements | Agreement | 1 | ||
WAINUA [Member] | Maximum [Member] | |||
Collaborative Arrangement and Licensing Agreement [Abstract] | |||
Next payment to be achieved | $ 30,000 | ||
Cardiovascular, Renal and Metabolic Diseases [Member] | |||
Collaborative Arrangement and Licensing Agreement [Abstract] | |||
Number of collaboration agreements | Agreement | 1 |
Collaborative Arrangements an_4
Collaborative Arrangements and Licensing Agreements, Biogen (Details) - USD ($) $ in Thousands | 3 Months Ended | ||
Mar. 31, 2024 | Mar. 31, 2023 | Dec. 31, 2023 | |
Collaborative Arrangement and Licensing Agreement [Abstract] | |||
Revenue | $ 119,497 | $ 130,524 | |
Biogen [Member] | |||
Collaborative Arrangement and Licensing Agreement [Abstract] | |||
Revenue | 59,236 | $ 70,501 | |
Deferred contract revenue | $ 287,200 | $ 307,400 | |
Biogen [Member] | Revenue [Member] | Strategic Partner [Member] | |||
Collaborative Arrangement and Licensing Agreement [Abstract] | |||
Concentration percentage | 50% | 54% | |
Biogen [Member] | Minimum [Member] | |||
Collaborative Arrangement and Licensing Agreement [Abstract] | |||
Cumulative payments received | $ 3,800,000 |
Basic and Diluted Net Loss Pe_3
Basic and Diluted Net Loss Per Share (Details) | Mar. 31, 2024 | Dec. 31, 2023 | Mar. 31, 2023 |
0% Notes [Member] | |||
Diluted Net Loss per Share [Abstract] | |||
Interest rate on convertible senior notes | 0% | 0% | 0% |
0.125% Notes [Member] | |||
Diluted Net Loss per Share [Abstract] | |||
Interest rate on convertible senior notes | 0.125% | 0.125% | 0.125% |
1.75% Notes [Member] | |||
Diluted Net Loss per Share [Abstract] | |||
Interest rate on convertible senior notes | 1.75% |
Investments, Contract Maturity
Investments, Contract Maturity of Available-for-Sale Securities (Details) | 3 Months Ended |
Mar. 31, 2024 | |
Contract Maturity of Available-for-Sale Securities [Abstract] | |
One year or less | 69% |
After one year but within two years | 26% |
After two years but within three and a half years | 5% |
Total | 100% |
Maximum contract maturity period, range 1 | 1 year |
Maximum contract maturity period, range 2 | 2 years |
Maximum contract maturity period, range 3 | 3 years 6 months |
Percentage of available-for-sale securities with a maturity of less than two years | 95% |
Investments, Summary of Investm
Investments, Summary of Investments (Details) $ in Thousands | 3 Months Ended | ||
Mar. 31, 2024 USD ($) Company | Dec. 31, 2023 USD ($) | ||
Ownership Interests in Private and Public Companies [Abstract] | |||
Number of private companies in which there is an equity ownership interest of less than 20% | Company | 7 | ||
Number of public companies in which there is an equity ownership interest of less than 20% | Company | 3 | ||
Available-for-sale Debt Securities with Maturity of One Year or Less [Abstract] | |||
Amortized cost | $ 1,307,024 | $ 1,316,219 | |
Gross unrealized gains | 178 | 415 | |
Gross unrealized losses | (3,848) | (5,238) | |
Estimated fair value | 1,303,354 | 1,311,396 | |
Available-for-sale Debt Securities with Maturity of More Than One Year [Abstract] | |||
Amortized cost | 569,655 | 651,628 | |
Gross unrealized gains | 672 | 3,063 | |
Gross unrealized losses | (2,166) | (1,197) | |
Estimated fair value | 568,161 | 653,494 | |
Available-for-sale Debt Securities [Abstract] | |||
Amortized cost | 1,876,679 | 1,967,847 | |
Gross unrealized gains | 850 | 3,478 | |
Gross unrealized losses | (6,014) | (6,435) | |
Estimated fair value | 1,871,515 | 1,964,890 | |
Equity Securities [Abstract] | |||
Amortized cost | 35,012 | 35,012 | |
Gross unrealized gains | 25,320 | 25,237 | |
Gross unrealized losses | (8,881) | (10,957) | |
Estimated fair value | 51,451 | 49,292 | |
Available-for-sale Debt and Equity Securities [Abstract] | |||
Amortized cost | 1,911,691 | 2,002,859 | |
Gross unrealized gains | 26,170 | 28,715 | |
Gross unrealized losses | (14,895) | (17,392) | |
Estimated fair value | 1,922,966 | 2,014,182 | |
Corporate Debt Securities [Member] | |||
Available-for-sale Debt Securities with Maturity of One Year or Less [Abstract] | |||
Amortized cost | [1] | 529,593 | 559,967 |
Gross unrealized gains | 87 | 157 | |
Gross unrealized losses | (1,990) | (2,625) | |
Estimated fair value | 527,690 | 557,499 | |
Available-for-sale Debt Securities with Maturity of More Than One Year [Abstract] | |||
Amortized cost | 248,548 | 243,151 | |
Gross unrealized gains | 405 | 1,270 | |
Gross unrealized losses | (890) | (692) | |
Estimated fair value | 248,063 | 243,729 | |
Debt Securities Issued by U.S. Government Agencies [Member] | |||
Available-for-sale Debt Securities with Maturity of One Year or Less [Abstract] | |||
Amortized cost | 179,632 | 224,711 | |
Gross unrealized gains | 24 | 64 | |
Gross unrealized losses | (409) | (611) | |
Estimated fair value | 179,247 | 224,164 | |
Available-for-sale Debt Securities with Maturity of More Than One Year [Abstract] | |||
Amortized cost | 115,152 | 110,138 | |
Gross unrealized gains | 134 | 547 | |
Gross unrealized losses | (187) | (21) | |
Estimated fair value | 115,099 | 110,664 | |
Debt Securities Issued by the U.S. Treasury [Member] | |||
Available-for-sale Debt Securities with Maturity of One Year or Less [Abstract] | |||
Amortized cost | [1] | 582,782 | 513,784 |
Gross unrealized gains | 38 | 152 | |
Gross unrealized losses | (1,372) | (1,889) | |
Estimated fair value | 581,448 | 512,047 | |
Available-for-sale Debt Securities with Maturity of More Than One Year [Abstract] | |||
Amortized cost | 204,535 | 294,873 | |
Gross unrealized gains | 133 | 1,239 | |
Gross unrealized losses | (1,086) | (480) | |
Estimated fair value | 203,582 | 295,632 | |
Debt Securities Issued by States of the U.S. and Political Subdivisions of the States [Member] | |||
Available-for-sale Debt Securities with Maturity of One Year or Less [Abstract] | |||
Amortized cost | 15,017 | 17,757 | |
Gross unrealized gains | 29 | 42 | |
Gross unrealized losses | (77) | (113) | |
Estimated fair value | 14,969 | 17,686 | |
Available-for-sale Debt Securities with Maturity of More Than One Year [Abstract] | |||
Amortized cost | 1,420 | 3,466 | |
Gross unrealized gains | 0 | 7 | |
Gross unrealized losses | (3) | (4) | |
Estimated fair value | 1,417 | 3,469 | |
Publicly Traded Equity Securities [Member] | |||
Equity Securities [Abstract] | |||
Amortized cost | [2] | 11,897 | 11,897 |
Gross unrealized gains | 319 | 236 | |
Gross unrealized losses | (3,756) | (5,832) | |
Estimated fair value | 8,460 | 6,301 | |
Available-for-sale Debt and Equity Securities [Abstract] | |||
Unrealized gain on equity securities | 2,200 | ||
Privately Held Equity Securities [Member] | |||
Equity Securities [Abstract] | |||
Amortized cost | [3] | 23,115 | 23,115 |
Gross unrealized gains | 25,001 | 25,001 | |
Gross unrealized losses | (5,125) | (5,125) | |
Estimated fair value | 42,991 | $ 42,991 | |
Available-for-sale Debt and Equity Securities [Abstract] | |||
Unrealized gain on equity securities | $ 0 | ||
[1]Includes investments classified as cash equivalents in our condensed consolidated balance sheets.[2]Our publicly traded equity securities are included in other current assets. We recognize publicly traded equity securities at fair value. In the three months ended March 31, 2024, we recorded a $2.2 million net unrealized gain in our condensed consolidated statements of operations related to changes in the fair value of our investments in publicly traded companies.[3] Our privately held equity securities are included in deposits and other assets. We recognize our privately held equity securities at . In the , there were changes in the fair value of our investments in privately held companies. |
Investments, Investments Tempor
Investments, Investments Temporarily Impaired (Details) $ in Thousands | Mar. 31, 2024 USD ($) Investment |
Temporarily Impaired Investments [Abstract] | |
Number of investments | Investment | 589 |
Estimated Fair Value [Abstract] | |
Less than 12 months of temporary impairment | $ 1,261,831 |
More than 12 months of temporary impairment | 201,069 |
Total temporary impairment | 1,462,900 |
Unrealized Losses [Abstract] | |
Less than 12 months of temporary impairment | (3,516) |
More than 12 months of temporary impairment | (2,498) |
Total temporary impairment | $ (6,014) |
Corporate Debt Securities [Member] | |
Temporarily Impaired Investments [Abstract] | |
Number of investments | Investment | 392 |
Estimated Fair Value [Abstract] | |
Less than 12 months of temporary impairment | $ 487,939 |
More than 12 months of temporary impairment | 96,219 |
Total temporary impairment | 584,158 |
Unrealized Losses [Abstract] | |
Less than 12 months of temporary impairment | (1,361) |
More than 12 months of temporary impairment | (1,519) |
Total temporary impairment | $ (2,880) |
Debt Securities Issued by U.S. Government Agencies [Member] | |
Temporarily Impaired Investments [Abstract] | |
Number of investments | Investment | 80 |
Estimated Fair Value [Abstract] | |
Less than 12 months of temporary impairment | $ 176,956 |
More than 12 months of temporary impairment | 12,375 |
Total temporary impairment | 189,331 |
Unrealized Losses [Abstract] | |
Less than 12 months of temporary impairment | (360) |
More than 12 months of temporary impairment | (236) |
Total temporary impairment | $ (596) |
Debt Securities Issued by the U.S. Treasury [Member] | |
Temporarily Impaired Investments [Abstract] | |
Number of investments | Investment | 64 |
Estimated Fair Value [Abstract] | |
Less than 12 months of temporary impairment | $ 588,958 |
More than 12 months of temporary impairment | 86,772 |
Total temporary impairment | 675,730 |
Unrealized Losses [Abstract] | |
Less than 12 months of temporary impairment | (1,773) |
More than 12 months of temporary impairment | (685) |
Total temporary impairment | $ (2,458) |
Debt Securities Issued by States of the U.S. and Political Subdivisions of the States [Member] | |
Temporarily Impaired Investments [Abstract] | |
Number of investments | Investment | 53 |
Estimated Fair Value [Abstract] | |
Less than 12 months of temporary impairment | $ 7,978 |
More than 12 months of temporary impairment | 5,703 |
Total temporary impairment | 13,681 |
Unrealized Losses [Abstract] | |
Less than 12 months of temporary impairment | (22) |
More than 12 months of temporary impairment | (58) |
Total temporary impairment | $ (80) |
Fair Value Measurements (Detail
Fair Value Measurements (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 | Mar. 31, 2023 | |||
Fair Value Measurements [Abstract] | ||||||
Available-for-sale securities | $ 1,871,515 | $ 1,964,890 | ||||
Equity securities | $ 51,451 | $ 49,292 | ||||
1.75% Notes [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Interest rate on convertible senior notes | 1.75% | |||||
0% Notes [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Interest rate on convertible senior notes | 0% | 0% | 0% | |||
0.125% Notes [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Interest rate on convertible senior notes | 0.125% | 0.125% | 0.125% | |||
Publicly Traded Equity Securities [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Equity securities | $ 8,460 | $ 6,301 | ||||
Recurring Basis [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Cash equivalents | [1] | 263,639 | 185,424 | |||
Total | 2,143,614 | 2,156,615 | ||||
Recurring Basis [Member] | Corporate Debt Securities [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Available-for-sale securities | 775,753 | [2] | 801,228 | [3] | ||
Recurring Basis [Member] | Corporate Debt Securities [Member] | Cash and Cash Equivalents [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Available-for-sale securities | 14,200 | 33,000 | ||||
Recurring Basis [Member] | Debt Securities Issued by U.S. Government Agencies [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Available-for-sale securities | [4] | 294,346 | 334,828 | |||
Recurring Basis [Member] | Debt Securities Issued by the U.S. Treasury [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Available-for-sale securities | [4] | 785,030 | 807,679 | |||
Recurring Basis [Member] | Debt Securities Issued by States of the U.S. and Political Subdivisions of the States [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Available-for-sale securities | [4] | 16,386 | 21,155 | |||
Recurring Basis [Member] | Publicly Traded Equity Securities [Member] | Other Current Assets [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Equity securities | [5] | 8,460 | 6,301 | |||
Recurring Basis [Member] | Quoted Prices in Active Markets (Level 1) [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Cash equivalents | 263,639 | 185,424 | ||||
Total | 1,057,129 | 999,404 | ||||
Recurring Basis [Member] | Quoted Prices in Active Markets (Level 1) [Member] | Corporate Debt Securities [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Available-for-sale securities | 0 | 0 | ||||
Recurring Basis [Member] | Quoted Prices in Active Markets (Level 1) [Member] | Debt Securities Issued by U.S. Government Agencies [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Available-for-sale securities | 0 | 0 | ||||
Recurring Basis [Member] | Quoted Prices in Active Markets (Level 1) [Member] | Debt Securities Issued by the U.S. Treasury [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Available-for-sale securities | 785,030 | 807,679 | ||||
Recurring Basis [Member] | Quoted Prices in Active Markets (Level 1) [Member] | Debt Securities Issued by States of the U.S. and Political Subdivisions of the States [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Available-for-sale securities | 0 | 0 | ||||
Recurring Basis [Member] | Quoted Prices in Active Markets (Level 1) [Member] | Publicly Traded Equity Securities [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Equity securities | 8,460 | 6,301 | ||||
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Cash equivalents | 0 | 0 | ||||
Total | 1,086,485 | 1,157,211 | ||||
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | 1.75% Notes [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Convertible notes | 601,100 | 661,100 | ||||
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | 0% Notes [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Convertible notes | 627,800 | 667,800 | ||||
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | 0.125% Notes [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Convertible notes | 42,400 | 42,400 | ||||
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | Corporate Debt Securities [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Available-for-sale securities | 775,753 | 801,228 | ||||
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | Debt Securities Issued by U.S. Government Agencies [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Available-for-sale securities | 294,346 | 334,828 | ||||
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | Debt Securities Issued by the U.S. Treasury [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Available-for-sale securities | 0 | 0 | ||||
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | Debt Securities Issued by States of the U.S. and Political Subdivisions of the States [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Available-for-sale securities | 16,386 | 21,155 | ||||
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | Publicly Traded Equity Securities [Member] | ||||||
Fair Value Measurements [Abstract] | ||||||
Equity securities | $ 0 | $ 0 | ||||
[1]Included in cash and cash equivalents.[2]$14.2 million was included in cash and cash equivalents, with the difference included in short-term investments.[3]$33.0 million was included in cash and cash equivalents, with the difference included in short-term investments.[4]Included in short-term investments.[5]Included in other current assets. |
Stock-based Compensation Expe_3
Stock-based Compensation Expense (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Stock-based Compensation Expense [Abstract] | ||
Stock-based compensation expense | $ 31,340 | $ 26,948 |
Cost of Sales [Member] | ||
Stock-based Compensation Expense [Abstract] | ||
Stock-based compensation expense | 204 | 119 |
Research, Development and Patent Expense [Member] | ||
Stock-based Compensation Expense [Abstract] | ||
Stock-based compensation expense | 22,225 | 19,567 |
Selling, General and Administrative Expense [Member] | ||
Stock-based Compensation Expense [Abstract] | ||
Stock-based compensation expense | 8,911 | $ 7,262 |
Stock Options [Member] | ||
Unrecognized Compensation Expense [Abstract] | ||
Unrecognized compensation expense related to non-vested stock options | $ 71,100 | |
Weighted average period for recognition | 1 year 4 months 24 days | |
Stock Options [Member] | Employees [Member] | ||
Weighted-Average Assumptions [Abstract] | ||
Risk-free interest rate | 4% | 3.60% |
Dividend yield | 0% | 0% |
Volatility | 44% | 47.50% |
Expected life | 6 years 3 months 18 days | 6 years 3 months 18 days |
ESPP [Member] | ||
Weighted-Average Assumptions [Abstract] | ||
Risk-free interest rate | 5.30% | 5.20% |
Dividend yield | 0% | 0% |
Volatility | 38.40% | 36.70% |
Expected life | 6 months | 6 months |
RSUs [Member] | ||
Unrecognized Compensation Expense [Abstract] | ||
Unrecognized compensation cost related to non-vested units | $ 112,800 | |
Weighted average period for recognition | 1 year 9 months 18 days | |
RSUs [Member] | Employees [Member] | ||
RSUs and PRSUs [Abstract] | ||
Weighted-average grant date fair value (in dollars per share) | $ 53.54 | $ 39.85 |
PRSUs [Member] | ||
Unrecognized Compensation Expense [Abstract] | ||
Unrecognized compensation cost related to non-vested units | $ 16,800 | |
Weighted average period for recognition | 2 years 2 months 12 days | |
PRSUs [Member] | Executive Officers [Member] | ||
RSUs and PRSUs [Abstract] | ||
Weighted-average grant date fair value (in dollars per share) | $ 78.41 | $ 58.99 |
PRSUs [Member] | Granted in 2024 [Member] | ||
RSUs and PRSUs [Abstract] | ||
Vesting percentage | 100% | |
Vesting period | 3 years | |
Number of units guaranteed to vest (in shares) | 0 | |
PRSUs [Member] | Granted in 2024 [Member] | Minimum [Member] | ||
RSUs and PRSUs [Abstract] | ||
Percentage of units guaranteed to vest | 0% | |
PRSUs [Member] | Granted in 2024 [Member] | Maximum [Member] | ||
RSUs and PRSUs [Abstract] | ||
Percentage of units guaranteed to vest | 200% | |
PRSUs [Member] | Granted in 2023 [Member] | Executive Officers [Member] | ||
RSUs and PRSUs [Abstract] | ||
Vesting percentage | 100% | |
Vesting period | 3 years | |
Number of units guaranteed to vest (in shares) | 0 | |
PRSUs [Member] | Granted in 2023 [Member] | Executive Officers [Member] | Minimum [Member] | ||
RSUs and PRSUs [Abstract] | ||
Percentage of units guaranteed to vest | 0% | |
PRSUs [Member] | Granted in 2023 [Member] | Executive Officers [Member] | Maximum [Member] | ||
RSUs and PRSUs [Abstract] | ||
Percentage of units guaranteed to vest | 200% |
Income Taxes (Details)
Income Taxes (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Income Taxes [Abstract] | ||
Income tax expense | $ 75 | $ 11,380 |
Liability Related to Sale of _3
Liability Related to Sale of Future Royalties (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | |
Jan. 31, 2023 | Mar. 31, 2024 | Mar. 31, 2023 | Dec. 31, 2023 | |
Royalty Revenue Monetization [Abstract] | ||||
Proceeds from sale of future royalties in January 2023 | $ 0 | $ 500,000 | ||
Issuance costs related to sale of future royalties | 0 | (10,434) | ||
Royalty payments to Royalty Pharma | (6,623) | 0 | ||
Interest expense related to sale of future royalties | 17,806 | $ 15,363 | ||
Net liability related to sale of future royalties | 525,072 | $ 513,736 | ||
Royalty Purchase Agreement [Member] | ||||
Royalty Revenue Monetization [Abstract] | ||||
Upfront payment received | $ 500,000 | |||
Maximum amount of payments receivable for additional milestones | $ 625,000 | |||
Effective interest rate | 13.50% | |||
Proceeds from sale of future royalties in January 2023 | $ 500,000 | |||
Issuance costs related to sale of future royalties | (10,434) | |||
Royalty payments to Royalty Pharma | $ (6,623) | (44,628) | ||
Interest expense related to sale of future royalties | 17,806 | 68,238 | ||
Amortization of issuance costs related to sale of future royalties | 153 | 560 | ||
Net liability related to sale of future royalties | 525,072 | $ 513,736 | ||
Royalty Purchase Agreement [Member] | SPINRAZA [Member] | ||||
Royalty Revenue Monetization [Abstract] | ||||
Maximum amount of annual sales on which royalty payments are paid | $ 1,500,000 | |||
Royalty Purchase Agreement [Member] | SPINRAZA [Member] | Minimum [Member] | ||||
Royalty Revenue Monetization [Abstract] | ||||
Percentage of royalty payments paid on annual sales of medicine | 25% | |||
Maximum royalty payments made before royalty interest reverts back | $ 475,000 | |||
Royalty Purchase Agreement [Member] | SPINRAZA [Member] | Maximum [Member] | ||||
Royalty Revenue Monetization [Abstract] | ||||
Percentage of royalty payments paid on annual sales of medicine | 45% | |||
Maximum royalty payments made before royalty interest reverts back | $ 550,000 | |||
Royalty Purchase Agreement [Member] | Pelacarsen [Member] | ||||
Royalty Revenue Monetization [Abstract] | ||||
Percentage of royalty payments paid on annual sales of medicine | 25% |
Convertible Debt (Details)
Convertible Debt (Details) - USD ($) $ / shares in Units, shares in Millions, $ in Millions | 3 Months Ended | 12 Months Ended | |||
Mar. 31, 2024 | Dec. 31, 2023 | Dec. 31, 2021 | Dec. 31, 2019 | Mar. 31, 2023 | |
1.75% Notes [Member] | |||||
Convertible Debt [Abstract] | |||||
Face amount of offering | $ 575 | ||||
Repurchase of convertible senior notes | $ 488.2 | ||||
Outstanding principal balance | $ 575 | ||||
Unamortized debt issuance costs | $ 12 | ||||
Maturity date | Jun. 30, 2028 | ||||
Interest rate | 1.75% | ||||
Effective interest rate | 2.30% | ||||
Conversion price per share (in dollars per share) | $ 53.73 | ||||
Total shares of common stock subject to conversion (in shares) | 10.7 | ||||
Percentage of principal amount used as purchase price upon occurrence of fundamental change | 100% | ||||
0% Notes [Member] | |||||
Convertible Debt [Abstract] | |||||
Outstanding principal balance | $ 632.5 | ||||
Unamortized debt issuance costs | $ 6.3 | ||||
Maturity date | Apr. 30, 2026 | ||||
Interest rate | 0% | 0% | 0% | ||
Effective interest rate | 0.50% | ||||
Conversion price per share (in dollars per share) | $ 57.84 | ||||
Effective conversion price per share with call spread (in dollars per share) | $ 76.39 | ||||
Total shares of common stock subject to conversion (in shares) | 10.9 | ||||
Cost of call spread | $ 46.9 | ||||
Purchase of note hedges | 136.7 | ||||
Proceeds from issuance of warrants | $ 89.8 | ||||
Percentage of principal amount used as purchase price upon occurrence of fundamental change | 100% | ||||
0.125% Notes [Member] | |||||
Convertible Debt [Abstract] | |||||
Repurchase of convertible senior notes | $ 504.4 | ||||
Outstanding principal balance | $ 44.5 | ||||
Unamortized debt issuance costs | $ 0.1 | ||||
Maturity date | Dec. 31, 2024 | ||||
Interest rate | 0.125% | 0.125% | 0.125% | ||
Effective interest rate | 0.50% | ||||
Conversion price per share (in dollars per share) | $ 83.28 | ||||
Effective conversion price per share with call spread (in dollars per share) | $ 123.38 | ||||
Total shares of common stock subject to conversion (in shares) | 0.5 | ||||
Cost of call spread | $ 52.6 | ||||
Purchase of note hedges | 108.7 | ||||
Proceeds from issuance of warrants | $ 56.1 | ||||
Percentage of principal amount used as purchase price upon occurrence of fundamental change | 100% |