Cover Page
Cover Page - USD ($) | 12 Months Ended | ||
Dec. 31, 2022 | Feb. 17, 2023 | Jun. 30, 2022 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Document Annual Report | true | ||
Document Period End Date | Dec. 31, 2022 | ||
Current Fiscal Year End Date | --12-31 | ||
Document Transition Report | false | ||
Entity File Number | 001-36407 | ||
Entity Registrant Name | ALNYLAM PHARMACEUTICALS, INC. | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Tax Identification Number | 77-0602661 | ||
Entity Address, Address Line One | 675 West Kendall Street | ||
Entity Address, Address Line Two | Henri A. Termeer Square | ||
Entity Address, City or Town | Cambridge | ||
Entity Address, State or Province | MA | ||
Entity Address, Postal Zip Code | 02142 | ||
City Area Code | 617 | ||
Local Phone Number | 551-8200 | ||
Title of 12(b) Security | Common Stock, $0.01 par value per share | ||
Trading Symbol | ALNY | ||
Security Exchange Name | NASDAQ | ||
Entity Well-known Seasoned Issuer | Yes | ||
Entity Voluntary Filers | No | ||
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 | ||
ICFR Auditor Attestation Flag | true | ||
Entity Shell Company | false | ||
Entity Public Float | $ 17,596,044,220 | ||
Entity Common Stock, Shares Outstanding | 124,130,988 | ||
Documents Incorporated by Reference | Portions of the registrant’s definitive proxy statement for its 2023 annual meeting of stockholders, which the registrant intends to file pursuant to Regulation 14A with the Securities and Exchange Commission not later than 120 days after the registrant’s fiscal year end of December 31, 2022, are incorporated by reference into Part II, Item 5 and Part III of this Form 10-K. | ||
Amendment Flag | false | ||
Document Fiscal Year Focus | 2022 | ||
Document Fiscal Period Focus | FY | ||
Entity Central Index Key | 0001178670 |
Audit Information
Audit Information | 12 Months Ended |
Dec. 31, 2022 | |
Audit Information [Abstract] | |
Auditor Firm ID | 238 |
Auditor Name | PricewaterhouseCoopers LLP |
Auditor Location | Boston, Massachusetts |
CONSOLIDATED BALANCE SHEETS
CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Current assets: | ||
Cash and cash equivalents | $ 866,394 | $ 819,975 |
Marketable debt securities | 1,297,890 | 1,548,617 |
Marketable equity securities | 28,122 | 66,972 |
Accounts receivable, net | 237,963 | 198,571 |
Inventory | 128,962 | 86,363 |
Prepaid expenses and other current assets | 132,916 | 88,078 |
Total current assets | 2,692,247 | 2,808,576 |
Property, plant and equipment, net | 523,494 | 501,958 |
Operating lease right-of-use assets | 215,136 | 231,675 |
Restricted investments | 49,390 | 40,891 |
Other assets | 66,092 | 60,204 |
Total assets | 3,546,359 | 3,643,304 |
Current liabilities: | ||
Accounts payable | 98,094 | 73,426 |
Accrued expenses | 545,460 | 395,174 |
Operating lease liability | 41,967 | 40,548 |
Deferred revenue | 42,105 | 149,483 |
Liability related to the sale of future royalties | 40,289 | 37,079 |
Total current liabilities | 767,915 | 695,710 |
Operating lease liability, net of current portion | 261,339 | 281,347 |
Deferred revenue, net of current portion | 193,791 | 152,360 |
Convertible debt | 1,016,942 | 0 |
Long-term debt, net | 0 | 675,697 |
Liability related to the sale of future royalties, net of current portion | 1,252,015 | 1,151,024 |
Other liabilities | 212,580 | 98,963 |
Total liabilities | 3,704,582 | 3,055,101 |
Commitments and contingencies (Note 14) | ||
Stockholders’ (deficit) equity: | ||
Preferred stock, $0.01 par value per share, 5,000 shares authorized and no shares issued and outstanding as of December 31, 2022 and December 31, 2021 | 0 | 0 |
Common stock, $0.01 par value per share, 250,000 shares authorized as of December 31, 2022 and December 31, 2021, respectively; 123,925 shares issued and outstanding as of December 31, 2022; 120,182 shares issued and outstanding as of December 31, 2021 | 1,240 | 1,202 |
Additional paid-in capital | 6,454,540 | 6,058,453 |
Accumulated other comprehensive loss | (44,654) | (33,259) |
Accumulated deficit | (6,569,349) | (5,438,193) |
Total stockholders’ (deficit) equity | (158,223) | 588,203 |
Total liabilities and stockholders’ (deficit) equity | $ 3,546,359 | $ 3,643,304 |
CONSOLIDATED BALANCE SHEETS (Pa
CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares | Dec. 31, 2022 | Dec. 31, 2021 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value (in dollars per share) | $ 0.01 | $ 0.01 |
Preferred stock, shares authorized (in shares) | 5,000,000 | 5,000,000 |
Preferred stock, shares issued (in shares) | 0 | 0 |
Preferred stock, shares outstanding (in shares) | 0 | 0 |
Common stock, par value (in dollars per share) | $ 0.01 | $ 0.01 |
Common stock, shares authorized (in shares) | 250,000,000 | 250,000,000 |
Common stock, shares issued (in shares) | 123,925,000 | 120,182,000 |
Common stock, shares outstanding (in shares) | 123,925,000 | 120,182,000 |
CONSOLIDATED STATEMENTS OF OPER
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Revenues: | |||
Total revenues | $ 1,037,418 | $ 844,287 | $ 492,853 |
Operating costs and expenses: | |||
Cost of goods sold | 140,174 | 115,005 | 74,185 |
Cost of collaborations and royalties | 28,643 | 25,139 | 3,867 |
Research and development | 883,015 | 792,156 | 654,819 |
Selling, general and administrative | 770,658 | 620,639 | 588,420 |
Total operating costs and expenses | 1,822,490 | 1,552,939 | 1,321,291 |
Loss from operations | (785,072) | (708,652) | (828,438) |
Other (expense) income: | |||
Interest expense | (155,968) | (143,021) | (84,496) |
Other (expense) income, net | (109,367) | (471) | 57,334 |
Loss on the extinguishment of debt | (76,586) | 0 | 0 |
Total other expense, net | (341,921) | (143,492) | (27,162) |
Loss before income taxes | (1,126,993) | (852,144) | (855,600) |
Provision for income taxes | (4,163) | (680) | (2,681) |
Net loss | $ (1,131,156) | $ (852,824) | $ (858,281) |
Net loss per share, basic (in dollars per share) | $ (9.30) | $ (7.20) | $ (7.46) |
Net loss per share, diluted (in dollars per share) | $ (9.30) | $ (7.20) | $ (7.46) |
Weighted average common shares, basic (in shares) | 121,689,000 | 118,451,000 | 114,986,000 |
Weighted average common shares, diluted (in shares) | 121,689,000 | 118,451,000 | 114,986,000 |
Statements of Comprehensive Loss | |||
Net loss | $ (1,131,156) | $ (852,824) | $ (858,281) |
Other comprehensive (loss) income: | |||
Unrealized (loss) gain on marketable securities | (7,840) | (1,978) | 211 |
Foreign currency translation (loss) gain | (5,274) | 11,398 | (7,081) |
Defined benefit pension plans, net of tax | 1,719 | 943 | (234) |
Total other comprehensive (loss) income | (11,395) | 10,363 | (7,104) |
Comprehensive loss | (1,142,551) | (842,461) | (865,385) |
Net revenues from collaborations | |||
Revenues: | |||
Total revenues | 134,912 | 180,953 | 131,333 |
Net product revenues | |||
Revenues: | |||
Total revenues | 894,329 | 662,138 | 361,520 |
Royalty revenue | |||
Revenues: | |||
Total revenues | $ 8,177 | $ 1,196 | $ 0 |
CONSOLIDATED STATEMENTS OF STOC
CONSOLIDATED STATEMENTS OF STOCKHOLDERS' (DEFICIT) EQUITY - USD ($) shares in Thousands, $ in Thousands | Total | Convertible Senior Notes Due 2027 | Common Stock | Additional Paid-in Capital | Accumulated Other Comprehensive (Loss) Income | Accumulated Deficit |
Beginning balance (in shares) at Dec. 31, 2019 | 112,188 | |||||
Beginning balance at Dec. 31, 2019 | $ 1,438,692 | $ 1,122 | $ 5,201,176 | $ (36,518) | $ (3,727,088) | |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Exercise of common stock options, net of tax withholdings (in shares) | 2,926 | |||||
Exercise of common stock options, net of tax withholdings | 189,371 | $ 28 | 189,343 | |||
Issuance of common stock under equity plans (in shares) | 350 | |||||
Issuance of common stock under equity plans | 11,083 | $ 4 | 11,079 | |||
Issuance of common stock to strategic partners, net of closing costs (in shares) | 963 | |||||
Issuance of common stock to strategic partners, net of closing costs | 99,498 | $ 10 | 99,488 | |||
Stock-based compensation expense | 142,988 | 142,988 | ||||
Purchases of capped calls related to convertible debt | 0 | |||||
Other comprehensive gain (loss) | (7,104) | (7,104) | ||||
Net loss | (858,281) | (858,281) | ||||
Ending balance (in shares) at Dec. 31, 2020 | 116,427 | |||||
Ending balance at Dec. 31, 2020 | 1,016,247 | $ 1,164 | 5,644,074 | (43,622) | (4,585,369) | |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Exercise of common stock options, net of tax withholdings (in shares) | 2,978 | |||||
Exercise of common stock options, net of tax withholdings | 232,486 | $ 30 | 232,456 | |||
Issuance of common stock under equity plans (in shares) | 777 | |||||
Issuance of common stock under equity plans | 13,631 | $ 8 | 13,623 | |||
Stock-based compensation expense | 168,300 | 168,300 | ||||
Purchases of capped calls related to convertible debt | 0 | |||||
Other comprehensive gain (loss) | 10,363 | 10,363 | ||||
Net loss | $ (852,824) | (852,824) | ||||
Ending balance (in shares) at Dec. 31, 2021 | 120,182 | 120,182 | ||||
Ending balance at Dec. 31, 2021 | $ 588,203 | $ 1,202 | 6,058,453 | (33,259) | (5,438,193) | |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Exercise of common stock options, net of tax withholdings (in shares) | 3,103 | |||||
Exercise of common stock options, net of tax withholdings | 263,612 | $ 32 | 263,580 | |||
Issuance of common stock under equity plans (in shares) | 640 | |||||
Issuance of common stock under equity plans | 13,725 | $ 6 | 13,719 | |||
Stock-based compensation expense | 237,399 | 237,399 | ||||
Purchases of capped calls related to convertible debt | (118,611) | $ (118,611) | ||||
Other comprehensive gain (loss) | (11,395) | (11,395) | ||||
Net loss | $ (1,131,156) | (1,131,156) | ||||
Ending balance (in shares) at Dec. 31, 2022 | 123,925 | 123,925 | ||||
Ending balance at Dec. 31, 2022 | $ (158,223) | $ 1,240 | $ 6,454,540 | $ (44,654) | $ (6,569,349) |
CONSOLIDATED STATEMENTS OF CASH
CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Cash flows from operating activities: | |||
Net loss | $ (1,131,156) | $ (852,824) | $ (858,281) |
Non-cash adjustments to reconcile net loss to net cash used in operating activities: | |||
Depreciation and amortization | 44,468 | 47,567 | 34,772 |
Amortization and interest accretion related to operating leases | 41,082 | 42,127 | 39,663 |
Non-cash interest expense on liability related to the sale of future royalties | 104,200 | 116,562 | 84,496 |
Stock-based compensation | 230,649 | 165,717 | 139,873 |
Realized and unrealized loss (gain) on marketable equity securities | 33,312 | (55,695) | (54,042) |
Loss on extinguishment of debt | 76,586 | 0 | 0 |
Change in fair value of development derivative liability | 94,659 | 38,433 | 17,185 |
Other | 479 | 19,243 | (5,926) |
Changes in operating assets and liabilities: | |||
Accounts receivable, net | (45,597) | (101,799) | (56,236) |
Inventory | (34,136) | (26,415) | (35,426) |
Prepaid expenses and other assets | (38,507) | (32,093) | 13,017 |
Accounts payable, accrued expenses and other liabilities | 191,769 | 88,240 | 143,732 |
Operating lease liability | (43,171) | (40,352) | (33,823) |
Deferred revenue | (65,911) | (50,404) | (43,965) |
Net cash used in operating activities | (541,274) | (641,693) | (614,961) |
Cash flows from investing activities: | |||
Purchases of property, plant and equipment | (72,059) | (76,372) | (70,361) |
Purchases of marketable securities | (1,976,961) | (1,656,114) | (2,025,626) |
Sales and maturities of marketable securities | 2,231,568 | 1,463,550 | 1,691,669 |
Proceeds from maturity of restricted investments | 89,951 | 41,975 | 0 |
Purchases of restricted investments | (98,451) | (42,141) | (25,900) |
Other investing activities | (4,694) | (4,198) | (5,300) |
Net cash provided by (used in) investing activities | 169,354 | (273,300) | (435,518) |
Cash flows from financing activities: | |||
Proceeds from exercise of stock options and other types of equity, net | 259,360 | 246,268 | 200,484 |
Proceeds from convertible debt, net | 1,016,111 | 0 | 0 |
Repayment of term loan | (762,107) | 0 | 0 |
Purchases of capped calls related to convertible debt | (118,611) | 0 | 0 |
Proceeds from the sale of future royalties | 0 | 500,000 | 500,000 |
Proceeds from development derivative | 31,000 | 19,600 | 8,400 |
Proceeds from term loan facility | 0 | 500,000 | 200,000 |
Proceeds from issuance of common stock to strategic partners, net of closing costs | 0 | 0 | 99,498 |
Other financing activities | 0 | (18,750) | (13,403) |
Net cash provided by financing activities | 425,753 | 1,247,118 | 994,979 |
Effect of exchange rate changes on cash, cash equivalents and restricted cash | (7,430) | (9,018) | 4,918 |
Net increase (decrease) in cash, cash equivalents and restricted cash | 46,403 | 323,107 | (50,582) |
Cash, cash equivalents and restricted cash, beginning of period | 822,153 | 499,046 | 549,628 |
Cash, cash equivalents and restricted cash, end of period | 868,556 | 822,153 | 499,046 |
Supplemental disclosure of cash flows: | |||
Cash paid for interest | 45,235 | 24,657 | 0 |
Supplemental disclosure of noncash investing and financing activities: | |||
Capital expenditures included in accounts payable and accrued expenses | 5,213 | 13,599 | 14,518 |
Lease liabilities arising from obtaining right-of-use assets | 1,013 | 7,932 | 34,435 |
Receivable and liability related to the sale of future royalties | $ 0 | $ 0 | $ 500,000 |
NATURE OF BUSINESS
NATURE OF BUSINESS | 12 Months Ended |
Dec. 31, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
NATURE OF BUSINESS | NATURE OF BUSINESS Alnylam Pharmaceuticals, Inc. (also referred to as Alnylam, we, our or us) commenced operations on June 14, 2002 as a biopharmaceutical company seeking to develop and commercialize novel therapeutics based on ribonucleic acid interference, or RNAi. We are committed to the advancement of our company strategy of building a multi-product, global, commercial biopharmaceutical company with a deep and sustainable clinical pipeline of RNAi therapeutics for future growth and a robust, organic research engine for sustainable innovation and great potential for patient impact. Since inception, we have focused on discovering, developing and commercializing RNAi therapeutics by establishing and maintaining a strong intellectual property position in the RNAi field, establishing strategic alliances with leading pharmaceutical and life sciences companies, generating revenues through licensing agreements, and ultimately developing and commercializing RNAi therapeutics globally, either independently or with our strategic partners. We have devoted substantially all of our efforts to business planning, research, development, manufacturing and early commercial efforts, acquiring, filing and expanding intellectual property rights, recruiting management and technical staff, and raising capital. In early 2021, we launched our Alnylam P 5 x25 strategy, which focuses on our planned transition to a top-tier biotech company by the end of 2025. With Alnylam P 5 x25 , we aim to deliver transformative rare and prevalent disease medicines for patients around the world through sustainable innovation, delivering exceptional financial performance and driving profitability. As of December 31, 2022, we have five products that have received marketing approval, including one partnered product, and multiple late-stage investigational programs advancing towards potential commercialization. We currently generate worldwide product revenues from four commercialized products, ONPATTRO, AMVUTTRA, GIVLAARI and OXLUMO, primarily in the United States, or U.S., Europe and Japan. |
SUMMARY OF SIGNIFICANT ACCOUNTI
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | 12 Months Ended |
Dec. 31, 2022 | |
Accounting Policies [Abstract] | |
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES Basis of Presentation and Principles of Consolidation The accompanying consolidated financial statements reflect the operations of Alnylam and our wholly-owned subsidiaries. All intercompany accounts and transactions have been eliminated. Use of Estimates The preparation of financial statements in conformity with accounting principles generally accepted in the United States of America, or GAAP, requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the consolidated financial statements and the reported amounts of revenues and expenses during the reporting period. In our consolidated financial statements, we use estimates and assumptions related to our inventory valuation and related reserves, liability related to the sale of future royalties, development derivative liability, income taxes, revenue recognition, research and development expenses, and stock-based compensation. We base our estimates on historical experience and on various other assumptions that we believe to be reasonable. Actual results could differ from those estimates. Liquidity Based on our current operating plan, we believe that our cash, cash equivalents and marketable securities as of December 31, 2022, together with the cash we expect to generate from product sales and under our current alliances, will be sufficient to enable us to advance our Alnylam P 5 x25 strategy for at least the next 12 months from the filing of this Annual Report on Form 10-K. Concentrations of Credit Risk and Significant Customers Financial instruments that potentially expose us to concentrations of credit risk primarily consist of cash, cash equivalents and marketable securities. As of December 31, 2022 and 2021, substantially all of our cash, cash equivalents and marketable securities were invested in money market funds, certificates of deposit, commercial paper, corporate notes, U.S. government-sponsored enterprise securities and U.S. treasury securities through highly rated financial institutions. Corporate notes may also include foreign bonds denominated in U.S. dollars. Investments are restricted, in accordance with our investment policy, to a concentration limit per issuer. During the years ended December 31, 2022, 2021 and 2020, our revenues were generated primarily from product sales to distributors and collaborations with strategic partners. For the years ended December 31, 2022, 2021 and 2020, our gross accounts receivable balance was comprised of payments primarily due from distributors for product sales and our strategic partners. The following table summarizes customers that represent 10% or greater of our consolidated total gross revenues: Year Ended December 31, 2022 2021 2020 Distributor A 33 % 27 % 31 % Regeneron Pharmaceuticals * 11% 12% __________________________________________ * Represents less than 10% The following table summarizes customers with amounts due that represent 10% or greater of our consolidated gross accounts receivable balance: As of December 31, 2022 2021 Distributor A 32 % 14 % Distributor B 10 % 10 % Novartis AG * 20 % Regeneron Pharmaceuticals * 12 % __________________________________________ * Represents less than 10% Fair Value Measurements The fair value is the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date. In general, fair values determined by Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities. Fair values determined by Level 2 inputs utilize data points that are observable, such as quoted prices (adjusted), interest rates and yield curves. Fair values determined by Level 3 inputs utilize unobservable data points for the asset or liability, and include situations where there is little, if any, market activity for the asset or liability. The fair value hierarchy level is determined by the lowest level of significant input. Investments in Marketable Securities and Cash Equivalents We invest our excess cash balances in marketable debt securities and classify our investments as either held-to-maturity or available-for-sale based on facts and circumstances present at the time we purchased the securities. At each balance sheet date presented, we classified all of our investments in debt securities as available-for-sale and as current assets as they represent the investment of funds available for current operations. We report available-for-sale debt securities at fair value at each balance sheet date and include any unrealized holding gains and losses (the adjustment to fair value) in accumulated other comprehensive (loss) income, a component of stockholders’ (deficit) equity. Realized gains and losses are determined using the specific identification method and are included in other income (expense). If any adjustment to fair value reflects a decline in the value of the marketable debt securities, we consider all available evidence to evaluate if an impairment loss exists, and if so, mark the investment to market through a charge to our consolidated statements of operations and comprehensive loss. We did not record any impairment charges related to our marketable debt securities during the years ended December 31, 2022, 2021 or 2020. Our marketable debt securities are classified as cash equivalents if the original maturity, from the date of purchase, is 90 days or less, and as marketable debt securities if the original maturity, from the date of purchase, is in excess of 90 days. Our cash equivalents are generally composed of commercial paper, corporate notes, U.S. government-sponsored enterprise securities, U.S. treasury securities and money market funds. We measure marketable equity investments (except those accounted for under the equity method of accounting or those that result in consolidation of an investee), which have readily available prices, at fair value with changes in fair value recognized in other income (expense) on our consolidated statements of operations and comprehensive loss. We obtain fair value measurement data for our marketable debt securities from independent pricing services. We perform validation procedures to ensure the reasonableness of this data. This includes discussing with the independent pricing services to understand the methods and data sources used. For our marketable debt securities, we perform our own review of prices received from the independent pricing services by comparing these prices to other sources and for our marketable equity securities, we confirm those securities are trading in active markets. Accounts Receivable We record accounts receivable net of customer allowances for distribution services, prompt payment discounts and chargebacks based on contractual terms. As of December 31, 2022 and 2021, based on our estimation of expected write-offs, we determined an allowance for doubtful accounts was not material. We have standard payment terms that generally require payment within approximately 30 to 90 days. Accounts receivable, net on our consolidated balance sheets also includes billed and unbilled collaboration receivables. Inventory Inventory is measured at the lower of cost or estimated net realizable value and classified based on the anticipation of when it will be consumed either within our normal operating cycle (short-term) or beyond (long-term). We use a standard cost basis, which approximates cost determined on a first-in, first-out basis. Inventory costs include all raw materials, direct conversion costs and overhead. Raw and intermediate materials that may be used for either research and development or commercial purposes are classified as inventory until the material is consumed or otherwise allocated for research and development. If the material is used for research and development, it is expensed as research and development once that determination is made. We capitalize inventory costs that are expected to be sold commercially once we determine it is probable that the inventory costs will be recovered through commercial sale based on the review of several factors, including (i) the likelihood that all required regulatory approvals will be received, considering any special filing status, (ii) the expected timing of validation (if not yet completed) of manufacturing processes in the associated facility, (iii) the expected expiration of the inventory, (iv) logistical or commercial constraints that may impede the timely distribution and sale of the product, including transport requirements and reimbursement status, (v) current market factors, including competitive landscape and pricing, (vi) threatened or anticipated litigation challenges, (vii) history of approvals of similar products or formulations, and (viii) FDA (or other appropriate regulatory agencies) correspondence regarding the safety and efficacy of the product. Prior to the capitalization of inventory costs, we record such costs as research and development expenses on our consolidated statements of operations and comprehensive loss. We reduce our inventory to net realizable value for potentially excess, dated or obsolete inventory based on our quarterly assessment of the recoverability of our capitalized inventory. We periodically review inventory levels to identify what may expire prior to expected sale or has a cost basis in excess of its estimated realizable value and write-down such inventories as appropriate. Property, Plant and Equipment Property, plant and equipment are stated at cost, net of accumulated depreciation. Depreciation expense is recorded on a straight-line basis over the estimated useful life of the asset. Construction in progress reflects amounts incurred for construction or improvements of property, plant or equipment that have not been placed in service. Costs of construction of certain long-lived assets include capitalized interest, which is amortized over the estimated useful life of the related asset. The cost and accumulated depreciation of assets retired or sold are removed from the respective asset category, and any gain or loss is recognized in our consolidated statements of operations and comprehensive loss. During the years ended December 31, 2022, 2021 and 2020, we recorded $39.1 million, $36.8 million and $30.2 million, respectively, of depreciation expense related to our property, plant and equipment. The estimated useful lives of property, plant and equipment are as follows: Asset Category Useful Life Laboratory equipment 5 Computer equipment and software 3-10 years Furniture and fixtures 5 Leasehold improvements Shorter of asset life or lease term Manufacturing Equipment 7-15 years Buildings 40 years Leases We determine if an arrangement is a lease at contract inception based on the facts and circumstances present in the arrangement. All of our leases are classified as operating leases. W e record operating lease assets and lease liabilities in our consolidated balance sheets. Operating lease assets represent our right to use an underlying asset for the lease term and operating lease liabilities represent our obligation to make lease payments arising from the leasing arrangement. Operating lease assets and operating lease liabilities are recognized at commencement date based on the present value of lease payments over the lease term. As most of our leases do not provide an implicit rate, in determining the operating lease liabilities, we use an estimate of our incremental borrowing rate based on the information available at commencement. Lease expense for lease payments is recognized on a straight-line basis over the lease term. Short-term leases, or leases that have a lease term of 12 months or less at commencement date, are excluded from this treatment and are recognized on a straight-line basis over the term of the lease. Clinical Accruals We record accrued liabilities related to products we have received or services that we have incurred, specifically related to ongoing pre-clinical studies and clinical trials, for which service providers have not yet billed us, or when billing terms under these contracts do not coincide with the timing of when the work is performed, as of our period-end. These costs primarily relate to third-party clinical management costs, laboratory and analysis costs, toxicology studies and investigator fees. The assessment of these costs is a subjective process, requiring judgment based on our knowledge of the research and development programs, services performed for the period, experience with related activities and the expected duration of the third-party service contract, where applicable. Upon settlement, these costs may differ materially from the amounts accrued in our consolidated financial statements. Our historical accrual estimates have not been materially different from our actual costs. Revenue Recognition We recognize revenue when control of promised goods or services is transferred to a customer at an amount that reflects the consideration to which we expect to be entitled in exchange for those goods or services. To determine revenue recognition, we perform the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price, including variable consideration, if any; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) we satisfy a performance obligation. We only apply the five-step model to contracts when collectability of the consideration to which we are entitled in exchange for the goods or services we transfer to the customer is determined to be probable. At contract inception, once the contract is determined to be within the scope of Accounting Standards Codification Topic 606, Revenue from Contracts with Customers, or ASC 606, we assess whether the goods or services promised within each contract are distinct and, therefore, represent a separate performance obligation. Goods and services that are determined not to be distinct are combined with other promised goods and services until a distinct bundle is identified. We then allocate the transaction price (the amount of consideration we expect to be entitled to from a customer in exchange for the promised goods or services) to each performance obligation and recognize the associated revenue when (or as) each performance obligation is satisfied. Our estimate of the transaction price for each contract includes all variable consideration to which we expect to be entitled. Amounts are recorded as accounts receivable when our right to consideration is unconditional. We do not assess whether a contract has a significant financing component if the expectation at contract inception is that the period between payment by the customer and the transfer of the promised goods or services to the customer will be one year or less. We expense incremental costs of obtaining a contract as and when incurred if the expected amortization period of the asset that we would have recognized is one year or less or the amount is immaterial. As of December 31, 2022 and 2021, we had not capitalized any costs to obtain any of our contracts. Net Product Revenues Our net product revenues are recognized, net of variable consideration related to certain allowances and accruals, at the time the customer obtains control of our product. We use the expected value method, which is the sum of probability-weighted amounts in a range of possible consideration amounts, or the most likely amount method, which is the single most likely amount in a range of possible considerations, to estimate variable consideration related to our product sales. We use the expected value method to estimate variable consideration for certain rebates, chargebacks, product returns, and other incentives and we use the most likely amount method for certain rebates and trade discounts and allowances. We record reserves, based on contractual terms, for components related to product sold during the reporting period, as well as our estimate of product that remains in the distribution channel inventory at the end of the reporting period that we expect will be sold to qualified healthcare providers. On a quarterly basis, we update our estimates and record any needed adjustments in the period we identify the adjustments. The following are the components of variable consideration related to product revenues: Chargebacks : We estimate obligations resulting from contractual commitments with the government and other entities to sell products to qualified healthcare providers at prices lower than the list prices charged to the customer who directly purchases from us. The customer charges us for the difference between what it pays to us for the product and the selling price to the qualified healthcare providers. Rebates : We are subject to discount obligations under government programs, including Medicaid in the U.S. and similar programs in certain other countries, including countries in which we are accruing for estimated rebates because final pricing has not yet been negotiated. We are also subject to potential rebates in connection with our value-based agreements with certain commercial payors. We record reserves for rebates in the same period the related product revenue is recognized, resulting in a reduction of product revenues and a current liability that is included in accrued expenses on our consolidated balance sheet. Our estimate for rebates is based on statutory discount rates, expected utilization or an estimated number of patients on treatment, as applicable. Trade discounts and allowances : We provide customary invoice discounts on product sales to our customers for prompt payment and we pay fees for distribution services, such as fees for certain data that customers provide to us. We estimate our customers will earn these discounts and fees, and deduct these discounts and fees in full from gross product revenues and accounts receivable at the time we recognize the related revenues. Product returns: We offer customers product return rights if products are damaged, defective or expired, with “expired” defined within each customer agreement. We estimate the amount of product that will be returned using a probability-weighted estimate based on our sales history. Other incentives: Other incentives include co-payment assistance we provide to patients with commercial insurance that have coverage and reside in states that allow co-payment assistance. We estimate the average co-payment assistance amounts for our products based on expected customer demographics and record any such amounts within accrued expenses on our consolidated balance sheet. Net Revenues from Collaborations We earn revenue in connection with collaboration agreements which allow our collaboration partners to utilize our technology platforms and develop product candidates. Our collaboration agreements are detailed in Note 4, Net Revenues from Collaborations. For each collaboration partner, we discuss our revenue recognition, including our significant performance obligations under each agreement. At contract inception, we assess whether the collaboration arrangements are within the scope of ASC Topic 808, Collaborative Arrangements, or ASC 808, to determine whether such arrangements involve joint operating activities performed by parties that are both active participants in the activities and exposed to significant risks and rewards dependent on the commercial success of such activities. This assessment is performed based on the responsibilities of all parties in the arrangement. For collaboration arrangements within the scope of ASC 808 that contain multiple elements, we first determine which elements of the arrangement are within the scope of ASC 808 and which elements are within the scope of ASC 606. For elements of collaboration arrangements that are accounted for pursuant to ASC 808, an appropriate recognition method is determined and applied consistently, either by analogy to authoritative accounting literature or by applying a reasonable and rational policy election. For elements of collaboration arrangements that are accounted for pursuant to ASC 606, we identify the performance obligations and allocate the total consideration we expect to receive on a relative standalone selling price basis to each performance obligation. Variable consideration such as performance-based milestones will be included in the total consideration if we expect to receive such consideration and if it is probable that the inclusion of the variable consideration will not result in a significant reversal in the cumulative amount of revenue recognized under the arrangement. Our estimate of the total consideration we expect to receive under each collaboration arrangement is updated for each reporting period, and any adjustments to revenue are recorded on a cumulative catch-up basis. We exclude sales-based royalty and milestone payments from the total consideration we expect to receive until the underlying sales occur because the license to our intellectual property is deemed to be the predominant item to which the royalties or milestones relate as it is the primary driver of value in our collaboration arrangements. Key assumptions to determine the standalone selling price may include forecasted revenues, development timelines, reimbursement rates for personnel costs, discount rates and probabilities of technical and regulatory success. We recognize revenue associated with each performance obligation as the control over the promised goods or services transfer to our collaboration partner which occurs either at a point in time or over time. If control transfers over time, revenue is recognized by using a method of measuring progress that best depicts the transfer of goods or services. We evaluate the measure of progress and related inputs each reporting period and any resulting adjustments to revenue are recorded on a cumulative catch-up basis. Consideration received that does not meet the requirements to satisfy ASC 808 or ASC 606 revenue recognition criteria is recorded as deferred revenue in the accompanying consolidated balance sheets, classified as either short-term (less than 12 months) or long-term (more than 12 months) deferred revenue based on our best estimate of when such revenue will be recognized. Cost of Goods Sold Cost of goods sold includes the cost of producing and distributing inventories that are related to product revenues during the respective period (including salary-related and stock-based compensation expenses for employees involved with production and distribution, freight and indirect overhead costs), third-party royalties payable on our net product revenues, amortization of intangible assets associated with the sale of our products and costs related to sales of product supply under our collaboration agreements. Cost of goods sold may also include costs related to excess or obsolete inventory adjustment charges, abnormal costs, unabsorbed manufacturing and overhead costs, and manufacturing variances. Cost of Collaborations and Royalties Cost of collaborations and royalties includes costs we incur in connection with providing commercial drug supplies, such as GalNAc material, to collaborators, in addition to royalties we owe to third parties on the net sales of licensed products by Novartis. Income Taxes We account for income taxes under the asset and liability method. Under this method, deferred tax assets and liabilities are recognized for the estimated future tax consequences attributable to differences between financial statement carrying amounts of existing assets and liabilities and their respective tax basis. Deferred tax assets and liabilities are measured using enacted rates in effect for the year in which these temporary differences are expected to be recovered or settled. Valuation allowances are provided if, based on the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. Uncertain tax positions, for which management's assessment is that there is a more than 50% probability of sustaining the position upon challenge by a taxing authority based upon its technical merits, are subject to certain recognition and measurement criteria. The nature of the uncertain tax positions is often very complex and subject to change, and the amounts at issue can be substantial. We develop our cumulative probability assessment of the measurement of uncertain tax positions using internal experience, judgment and assistance from professional advisors. We re-evaluate these uncertain tax positions on a quarterly basis based on a number of factors including, but not limited to, changes in facts or circumstances, changes in tax law, and effectively settled issues under audit and new audit activity. Any change in these factors could result in the recognition of a tax benefit or an additional charge to the tax provision. Research and Development Expenses We record research and development expenses as incurred. Included in research and development expenses are wages, stock-based compensation expenses, benefits and other operating costs, facilities, supplies, external services, clinical trial and manufacturing costs, certain costs related to our collaboration arrangements, and overhead directly related to our research and development operations, as well as costs to acquire technology licenses. We have entered into several license agreements for rights to utilize certain technologies. The terms of the licenses may provide for upfront payments, annual maintenance payments, milestone payments based upon certain specified events being achieved and royalties on product sales. We charge costs to acquire and maintain licensed technology that has not reached technological feasibility and does not have alternative future use to research and development expense as incurred. During the years ended December 31, 2022, 2021 and 2020, we charged to research and development expense costs associated with license fees of $7.3 million, $16.8 million and $2.8 million, respectively. Stock-Based Compensation We recognize stock-based compensation expense for grants under our stock incentive plans and employee stock purchase plan. We account for all stock-based awards granted to employees at their fair value and recognize compensation expense over the vesting period of the award. Determining the amount of stock-based compensation to be recorded requires us to develop estimates of fair values of stock options as of the grant date. We calculate the grant date fair values of stock options using the Black-Scholes valuation model, which requires the input of subjective assumptions, including but not limited to expected stock price volatility over the term of the awards and the expected term of stock options. The fair value of restricted stock awards granted to employees is based upon the quoted closing market price per share on the date of grant. We have performance conditions included in certain of our restricted stock awards that are based upon the achievement of pre-specified clinical development, regulatory, commercial and/or financial performance events. As the outcome of each event has inherent risk and uncertainties, and a positive outcome may not be known until the event is achieved, we begin to recognize the value of the performance-based restricted stock awards when we determine the achievement of each performance condition is deemed probable, a determination which requires significant judgment by management. At the probable date, we record a cumulative expense catch-up, with remaining expense amortized over the remaining service period. Liability Related to the Sale of Future Royalties We account for the liability related to the sale of future royalties as a debt financing, as we have significant continuing involvement in the generation of the cash flows. Interest on the liability related to the sale of future royalties will be recognized using the effective interest rate method over the life of the related royalty stream. The liability related to the sale of future royalties and the related interest expense are based on our current estimates of future royalties and commercial milestones expected to be paid over the life of the arrangement, which we determine by using third-party forecasts of Leqvio’s global net revenue. We will periodically assess the expected payments and to the extent the amount or timing of our future estimated payments is materially different than our previous estimates, we will account for any such change by adjusting the liability related to the sale of future royalties and prospectively recognizing the related non-cash interest expense. Development Derivative Liability Development derivative liability is recorded at fair value based on the probability weighted present value of the estimated cash flows pursuant to contractual terms of the funding agreement. The liability is remeasured quarterly with any change in fair value recorded in other income (expense) on the consolidated statements of operations and comprehensive loss. Comprehensive Loss Comprehensive loss is comprised of net loss and certain changes in stockholders’ (deficit) equity that are excluded from net loss. We include unrealized gains and losses on certain marketable securities in other comprehensive loss, including changes in the value of our marketable debt securities. We include foreign currency translation adjustments in other comprehensive loss if the functional currency is not the U.S. dollar. We include certain changes in the fair value of the plan assets and projected benefit obligation attributed to our defined benefit pension plan in other comprehensive loss. Net Loss per Common Share We compute basic net loss per common share by dividing net loss by the weighted-average number of common shares outstanding. We compute diluted net loss per common share by dividing net loss by the weighted-average number of common shares and dilutive potential common share equivalents then outstanding during the period. In the diluted net loss per share calculation, net loss would be adjusted for the elimination of interest expense on the Convertible debt. Potential common shares consist of shares issuable upon the exercise of stock options (the proceeds of which are then assumed to have been used to repurchase outstanding shares using the treasury stock method) or upon conversion of the convertible debt outstanding during the period (calculated using the if-converted method assuming the conversion of the convertible debt as of the earliest period reported or at the date of issuance, if later). Because the inclusion of potential common shares would be anti-dilutive for all periods presented, diluted net loss per common share is the same as basic net loss per common share. The following table sets forth the potential common shares (prior to consideration of the treasury stock or if-converted methods) excluded from the calculation of net loss per common share because their inclusion would be anti-dilutive: As of December 31, (In thousands) 2022 2021 2020 Options to purchase common stock, inclusive of performance-based stock options 8,424 10,015 11,692 Unvested restricted common stock, inclusive of performance-based restricted common stock 1,487 1,210 1,160 Convertible debt 3,616 — — Total 13,527 11,225 12,852 Segment Information We operate in a single reporting segment, the discovery, development and commercialization of RNAi therapeutics. Consistent with our management reporting, results of our operations are reported on a consolidated basis for purposes of segment reporting. As of December 31, 2022 and 2021, substantially all of our consolidated property, plant and equipment, net, was from U.S. operations. For the years ended December 31, 2022, 2021 and 2020, net revenues from collaborations were attributed to the U.S. Please read Note 3 for information regarding our net product sales by geography. Recently Adopted Accounting Pronouncements In August 2020, the Financial Accounting Standards Board, or FASB, issued Accounting Standards Update, or ASU, 2020-06, Debt—Debt with Conversion and Other Options (Subtopic 470-20) and Derivatives and Hedging—Contracts in Entity’s Own Equity (Subtopic 815-40): Accounting for Convertible Instruments and Contracts in an Entity’s Own Equity, which simplifies |
NET PRODUCT REVENUES
NET PRODUCT REVENUES | 12 Months Ended |
Dec. 31, 2022 | |
Revenue from Contract with Customer [Abstract] | |
NET PRODUCT REVENUES | NET PRODUCT REVENUES Net product revenues consist of the following: Year Ended December 31, (In thousands) 2022 2021 2020 ONPATTRO United States $ 246,748 $ 213,210 $ 151,574 Europe 224,063 190,435 107,755 Rest of World 86,797 71,092 46,752 Total 557,608 474,737 306,081 AMVUTTRA United States 82,521 — — Europe 4,214 — — Rest of World 7,060 — — Total 93,795 — — GIVLAARI United States 115,659 92,747 42,797 Europe 48,670 30,895 12,000 Rest of World 8,815 4,173 309 Total 173,144 127,815 55,106 OXLUMO United States 27,698 18,876 — Europe 37,915 38,949 333 Rest of World 4,169 1,761 — Total 69,782 59,586 333 Total net product revenues $ 894,329 $ 662,138 $ 361,520 As of December 31, 2022 and 2021, net product revenue-related receivables of $203.8 million and $124.9 million , respectively, were included in “Accounts receivable, net.” The following table summarizes balances and activity in each product revenue allowance and reserve category: As of December 31, 2022 (In thousands) Chargebacks and Rebates Trade Discounts and Allowances Returns Reserve and Other Incentives Total Beginning balance $ 120,682 $ 522 $ 10,112 $ 131,316 Provision related to current period sales 245,236 13,085 15,249 273,570 Credit or payments made during the period for current year sales (108,185) (10,310) (9,850) (128,345) Credit or payments made during the period for prior year sales (65,961) (847) (735) (67,543) Total $ 191,772 $ 2,450 $ 14,776 $ 208,998 As of December 31, 2021 (In thousands) Chargebacks and Rebates Trade Discounts and Allowances Returns Reserve and Other Incentives Total Beginning balance $ 90,705 $ 639 $ 3,763 $ 95,107 Provision related to current period sales 167,691 8,064 12,223 187,978 Credit or payments made during the period for current year sales (75,073) (7,665) (5,190) (87,928) Credit or payments made during the period for prior period sales (62,641) (516) (684) (63,841) Total $ 120,682 $ 522 $ 10,112 $ 131,316 During the years ended December 31, 2022 and 2021 , we paid $44.3 million and $56.4 million, respectively, attributed to the pricing and reimbursement for the sale of our commercial products in France. |
NET REVENUES FROM COLLABORATION
NET REVENUES FROM COLLABORATIONS | 12 Months Ended |
Dec. 31, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
NET REVENUES FROM COLLABORATIONS | NET REVENUES FROM COLLABORATIONS Net revenues from collaborations consist of the following: Year Ended December 31, (In thousands) 2022 2021 2020 Regeneron Pharmaceuticals $ 87,844 $ 113,226 $ 74,072 Novartis AG 43,159 49,120 22,208 Vir Biotechnology 1,755 16,897 31,396 Other 2,154 1,710 3,657 Total $ 134,912 $ 180,953 $ 131,333 The following table presents the balance of our receivables and contract liabilities related to our collaboration agreements: As of December 31, (In thousands) 2022 2021 Receivables included in "Accounts receivable, net" $ 32,342 $ 73,266 Contract liabilities included in "Deferred revenue" $ 235,528 $ 88,627 We recognized revenue of $55.5 million and $62.9 million in the years ended December 31, 2022 and 2021, respectively, that was included in the contract liability balance at the beginning of the period. In order to determine revenue recognized in the period from contract liabilities, we first allocate revenue to the individual contract liability balance outstanding at the beginning of the period until the revenue exceeds that balance. If additional consideration is received on those contracts in subsequent periods, we assume all revenue recognized in the reporting period first applies to the beginning contract liability as opposed to a portion applying to the new consideration for the period. The following table provides research and development expenses incurred by type, for which we recognize net revenue, that are directly attributable to our collaboration agreements, by collaboration partner: Year Ended December 31, 2022 2021 2020 (In thousands) Clinical Trial and Manufacturing External Services Other Clinical Trial and Manufacturing External Services Other Clinical Trial and Manufacturing External Services Other Regeneron $ 12,926 $ 2,141 $ 27,935 $ 24,989 $ 840 $ 47,582 $ 13,302 $ 171 $ 44,360 Other 156 679 337 10,311 775 4,489 20,113 765 14,422 Total $ 13,082 $ 2,820 $ 28,272 $ 35,300 $ 1,615 $ 52,071 $ 33,415 $ 936 $ 58,782 The research and development expenses incurred for the agreements included in the table above consist of costs incurred for (i) clinical expenses, including manufacturing of clinical product, (ii) external services including consulting services and lab supplies and services, and (iii) other expenses, including professional services, facilities and overhead allocations, and a reasonable estimate of compensation and related costs as billed to our counterparties, for which we recognize net revenues from collaborations. For the years ended December 31, 2022, 2021 and 2020, we did not incur material selling, general and administrative expenses related to our collaboration agreements. In addition, we recognized a reduction to our research and development expenses of $16.9 million, $17.1 million, and $11.1 million for the years ended December 31, 2022, 2021 and 2020, respectively, from cost reimbursement due under certain of our collaboration agreements with Regeneron Pharmaceuticals, Inc., or Regeneron, accounted for under Accounting Standards Codification, or ASC, Topic 808, Collaborative Arrangements, or ASC 808. Product Alliances Regeneron Pharmaceuticals, Inc. In April 2019, we entered into a global, strategic collaboration with Regeneron to discover, develop and commercialize RNAi therapeutics for a broad range of diseases by addressing therapeutic targets expressed in the eye and central nervous system, or CNS, in addition to a select number of targets expressed in the liver, which we refer to as the Regeneron Collaboration. The Regeneron Collaboration is governed by a Master Agreement, referred to as the Regeneron Master Agreement, which became effective on May 21, 2019. In connection with the Regeneron Master Agreement, we and Regeneron entered into (i) a binding co-co collaboration term sheet covering the continued development of cemdisiran, our C5 small interfering RNA, or siRNA, currently in Phase 2 development for C5 complement-mediated diseases, as a monotherapy and (ii) a binding license term sheet to evaluate anti-C5 antibody-siRNA combinations for C5 complement-mediated diseases including evaluating the combination of Regeneron’s pozelimab (REGN3918), currently in Phase 3 development, and cemdisiran. The C5 co-co collaboration and license agreements were executed in August 2019. Under the terms of the Regeneron Collaboration, we are working exclusively with Regeneron to discover RNAi therapeutics for eye and CNS diseases for an initial research period of approximately five years, which we refer to as the Initial Research Term. Regeneron has an option to extend the Initial Research Term (referred to as the Research Term Extension Period, and together with the Initial Research Term, the Research Term) for up to an additional five years, for a research term extension fee of up to $400.0 million. The Regeneron Collaboration also covers a select number of RNAi therapeutic programs designed to target genes expressed in the liver, including our previously announced collaboration with Regeneron to identify RNAi therapeutics for the chronic liver disease nonalcoholic steatohepatitis. We retain broad global rights to all of our other unpartnered liver-directed clinical and pre-clinical pipeline programs. The Regeneron Collaboration is governed by a joint steering committee that is comprised of an equal number of representatives from each party. Regeneron leads development and commercialization for all programs targeting eye diseases (subject to limited exceptions), entitling us to certain potential milestone and royalty payments pursuant to the terms of a license agreement, the form of which has been agreed upon by the parties. We and Regeneron are alternating leadership on CNS and liver programs covered by the Regeneron Collaboration, with the lead party retaining global development and commercial responsibility. For such CNS and liver programs, both we and Regeneron have the option at lead candidate selection to enter into a co-co collaboration agreement, the form of which has been agreed upon by the parties, whereby both companies will share equally all costs of, and profits from, all development and commercialization activities under the program. If the non-lead party elects to not enter into a co-co collaboration agreement with respect to a given CNS or liver program, we and Regeneron will enter into a license agreement with respect to such program and the lead party will be the “Licensee” for the purposes of the license agreement. If the lead party for a CNS or liver program elects to not enter into the co-co collaboration agreement, then we and Regeneron will enter into a license agreement with respect to such program and leadership of the program will transfer to the other party and the former non-lead party will be the “Licensee” for the purposes of the license agreement. With respect to the programs directed to C5 complement-mediated diseases, we retain control of cemdisiran monotherapy development, and Regeneron is leading combination product development. Pursuant to the C5 co-co collaboration agreement, Regeneron notified us in November 2022 of its decision to exercise its right to opt-out of the further development and commercialization of cemdisiran monotherapy. As a result, Regeneron no longer shares costs and potential future profits on any monotherapy program with us. We continue to perform our obligations under the agreement and we are solely responsible for all development and commercialization costs. Regeneron will be eligible to receive tiered double-digit royalties on net sales. Under the C5 license agreement, for cemdisiran to be used as part of a combination product, Regeneron is solely responsible for all development and commercialization costs and we will receive low double-digit royalties and commercial milestones of up to $325.0 million on any potential combination product sales. The C5 co-co collaboration agreement, the C5 license agreement, and the Master Agreement have been combined for accounting purposes and treated as a single agreement. In connection with the Regeneron Master Agreement, Regeneron made an upfront payment of $400.0 million. We are also eligible to receive up to an additional $200.0 million in milestone payments upon achievement of certain criteria during early clinical development for eye and CNS programs. We and Regeneron plan to advance programs directed to up to 30 targets in the first five years under the Regeneron Collaboration during the Initial Research Term. For each program, Regeneron will provide us with $2.5 million in funding at program initiation and an additional $2.5 million at lead candidate identification, with the potential for approximately $30.0 million in annual discovery funding to us as the Regeneron Collaboration reaches steady state. Regeneron has the right to terminate the Regeneron Master Agreement for convenience upon ninety days’ notice. The termination of the Regeneron Master Agreement does not affect the term of any license agreement or co-co collaboration agreement then in effect. In addition, either party may terminate the Regeneron Master Agreement for a material breach by, or insolvency of, the other party. Unless earlier terminated pursuant to its terms, the Regeneron Master Agreement will remain in effect with respect to each program until (a) such program becomes a terminated program or (b) the parties enter into a license agreement or co-co collaboration agreement with respect to such program. The Regeneron Master Agreement includes various representations, warranties, covenants, dispute escalation and resolution mechanisms, indemnities and other provisions customary for transactions of this nature. For any license agreement subsequently entered into, the licensee will generally be responsible for its own costs and expenses incurred in connection with the development and commercialization of the collaboration products. The licensee will pay to the licensor certain development and/or commercialization milestone payments totaling up to $150.0 million for each collaboration product. In addition, following the first commercial sale of the applicable collaboration product under a license agreement, the licensee is required to make certain tiered royalty payments, ranging from low double-digits up to 20%, to the licensor based on the aggregate annual net sales of the collaboration product, subject to customary reductions. For any co-co collaboration agreement subsequently entered into, we and Regeneron will share equally all costs of, and profits from, development and commercialization activities. Reimbursement of our share of costs will be recognized as a reduction to research and development expense in the consolidated statements of operations and comprehensive loss. In the event that a party exercises its opt-out right, the lead party will be responsible for all costs and expenses incurred in connection with the development and commercialization of the collaboration products under the applicable co-co collaboration agreement, subject to continued sharing of costs through defined points. If a party exercises its opt-out right, following the first commercial sale of the applicable collaboration product under a co-co collaboration agreement, the lead party is required to make certain tiered royalty payments, ranging from low double-digits up to 20%, to the other party based on the aggregate annual net sales of the collaboration product and the timing of the exercise of the opt-out right, subject to customary reductions and a reduction for opt-out transition costs. Due to the uncertainty of pharmaceutical development and the high historical failure rates generally associated with drug development, we may not receive any milestone or royalty payments from Regeneron under the Regeneron Master Agreement, the C5 license agreement, or any future license agreement, or under any co-co collaboration agreement in the event we exercise our opt-out right. Our obligations under the Regeneron Collaboration include: (i) a research license and research services, collectively referred to as the Research Services Obligation; (ii) a worldwide license to cemdisiran for combination therapies, and manufacturing and supply and development service obligations, collectively referred to as the C5 License Obligation; and (iii) development, manufacturing and commercialization activities for cemdisiran monotherapies, referred to as the C5 Co-Co Obligation. The research license is not distinct from the research services primarily as a result of Regeneron being unable to benefit on its own or with other resources reasonably available, as the license is providing access to specialized expertise, particularly as it relates to RNAi technology that is not available in the marketplace. Similarly, the worldwide license to cemdisiran for combination therapies is not distinct from the manufacturing and supply and development service obligations, as Regeneron cannot benefit on its own from the value of the license without receipt of supply. Separately, prior to Regeneron’s decision in November 2022 to exercise its right to opt-out of the further development and commercialization of cemdisiran monotherapy, the cemdisiran monotherapy co-co collaboration agreement was under the scope of ASC 808 as we and Regeneron were both active participants in the development and manufacturing activities and were exposed to significant risks and rewards that were dependent on commercial success of the activities of the arrangement. Regeneron’s decision to exercise its right to opt-out of the arrangement caused a change in the role of Regeneron and its exposure to significant risks and rewards under the arrangement. As a result, we determined that the arrangement no longer represents a collaborative arrangement. The arrangement now represents a vendor-customer relationship under ASC 606 as we perform our obligation to provide development and manufacturing activities under the arrangement. The transaction price allocated to the C5 Co-Co obligation unit of account will be recognized over time using an input method based on cost incurred relative to the total estimated costs for the identified performance obligation by determining the proportion of effort incurred as a percentage of total effort we expect to expend. The total transaction price is comprised of the $400.0 million upfront payment and additional variable consideration related to research, development, manufacturing and supply activities related to the Research Services Obligation and the C5 License Obligation. We utilized the expected value method to determine the amount of reimbursement for these activities. We determined that any variable consideration related to sales-based royalties and milestones related to the worldwide license to cemdisiran for combination therapies is deemed to be constrained and therefore has been excluded from the transaction price. In addition, we are eligible to receive future milestones upon the achievement of certain criteria during early clinical development for the eye and CNS programs. We are also eligible to receive royalties on future commercial sales for certain eye, CNS or liver targets, if any; however, these amounts are excluded from variable consideration under the Regeneron Collaboration as we are only eligible to receive such amounts if, after a drug candidate is identified, the form of license agreement is subsequently executed resulting in a license that is granted to Regeneron. Any such subsequently granted license would represent a separate transaction under ASC 606. We allocated the initial transaction price to each unit of account based on the applicable accounting guidance as follows, in thousands: Performance Obligations Standalone Selling Price Transaction Price Allocated Accounting Guidance Research Services Obligation $ 130,700 $ 183,100 ASC 606 C5 License Obligation $ 97,600 92,500 ASC 606 C5 Co-Co Obligation $ 364,600 246,000 ASC 606 $ 521,600 The transaction price was allocated to the obligations based on the relative estimated standalone selling prices of each obligation, over which management has applied significant judgment. We developed the estimated standalone selling price for the licenses included in the Research Services Obligation and the C5 License Obligation primarily based on the probability-weighted present value of expected future cash flows associated with each license related to each specific program. In developing such estimate, we applied judgment in the determination of the forecasted revenues, taking into consideration the applicable market conditions and relevant entity-specific factors, the expected number of targets or indications expected to be pursued under each license, the probability of success, the time needed to develop a product candidate pursuant to the associated license and the discount rate. We developed the estimated standalone selling price for the services and/or manufacturing and supply included in each of the obligations, as applicable, primarily based on the nature of the services to be performed and/or goods to be manufactured and estimates of the associated costs. The estimated standalone selling price of the C5 Co-Co Obligation was developed by estimating the present value of expected future cash flows that Regeneron is entitled to receive. In developing such estimate, we applied judgment in determining the indications that will be pursued, the forecasted revenues for such indications, the probability of success and the discount rate. For the Research Services Obligation and the C5 License Obligation accounted for under ASC 606, we measure proportional performance over time using an input method based on cost incurred relative to the total estimated costs for each of the identified obligations, on a quarterly basis, by determining the proportion of effort incurred as a percentage of total effort we expect to expend. This ratio is applied to the transaction price allocated to each obligation. Management has applied significant judgment in the process of developing our estimates. Any changes to these estimates will be recognized in the period in which they change as a cumulative catch up. We re-evaluate the transaction price as of the end of each reporting period and as of December 31, 2022, the total transaction price was determined to be $558.9 million, an increase of $20.5 million from December 31, 2021. As of December 31, 2022, the transaction price is comprised of the upfront payment and variable consideration related to development, manufacture and supply activities. For the C5 Co-Co Obligation accounted for under ASC 808, the transaction price allocated to this obligation is recognized using a proportional performance method. Revenue recognized under this agreement, inclusive of the amount allocated to the C5 Co-Co Obligation, is accounted for as collaboration revenue. The following tables provide a summary of the transaction price allocated to each unit of account based on the applicable accounting guidance, in addition to revenue activity during the period, in thousands: Transaction Price Allocated Deferred Revenue Performance Obligations As of December 31, 2022 As of December 31, 2022 As of December 31, 2021 Accounting Guidance Research Services Obligation $ 215,680 $ 26,200 $ 42,300 ASC 606 C5 License Obligation 97,200 7,000 26,900 ASC 606 C5 Co-Co Obligation 246,000 193,600 212,500 ASC 606 Total $ 558,880 $ 226,800 $ 281,700 Revenue Recognized During Performance Obligations Year Ended December 31, 2022 Year Ended December 31, 2021 Year Ended December 31, 2020 Accounting Guidance Research Services Obligation $ 28,600 $ 37,600 $ 44,800 ASC 606 C5 License Obligation 32,500 44,600 7,100 ASC 606 C5 Co-Co Obligation 20,080 18,900 11,700 ASC 606 Total $ 81,180 $ 101,100 $ 63,600 As of December 31, 2022, the aggregate amount of the transaction price allocated to the remaining Research Services Obligation, C5 License Obligation and C5 Co-Co Obligation that was unsatisfied was $289.1 million, which is expected to be recognized through the term of the Regeneron Collaboration as the services are performed. Deferred revenue related to the Regeneron Collaboration is classified as either current or non-current in the consolidated balance sheets based on the period the revenue is expected to be recognized. Novartis AG 2013 Collaboration with The Medicines Company In February 2013, we and The Medicines Company, or MDCO, entered into a license and collaboration agreement pursuant to which we granted to MDCO an exclusive, worldwide license to develop, manufacture and commercialize RNAi therapeutics targeting proprotein convertase subtilisin/kexin type 9, or PCSK9, for the treatment of hypercholesterolemia and other human diseases, including inclisiran. We refer to this agreement, as amended through the date hereof, as the MDCO License Agreement. On January 6, 2020, Novartis AG, or Novartis, completed its acquisition of MDCO and assumed all rights and obligations under the MDCO License Agreement. As of December 31, 2022, we have earned $80.0 million of milestones and upon achievement of certain events, we will be entitled to receive additional milestones, up to an aggregate of $100.0 million, including $90.0 million in specified commercialization milestones and $10.0 million in other specified regulatory milestones. In addition, we are entitled to royalties ranging from 10% up to 20% based on annual worldwide net sales of licensed products by Novartis, its affiliates and sublicensees, subject to reduction under specified circumstances. Due to the uncertainty of pharmaceutical development and the high historical failure rates generally associated with drug development, we may not receive any additional milestone payments under the MDCO License Agreement and future royalty payments may be less than anticipated. Unless terminated earlier in accordance with the terms of the agreement, the MDCO License Agreement expires on a licensed product-by-licensed product and country-by-country basis upon expiration of the last royalty term for any licensed product in any country, where a royalty term is defined as the latest to occur of (1) the expiration of the last valid claim of patent rights covering a licensed product, (2) the expiration of the Regulatory Exclusivity, as defined in the MDCO License Agreement, and (3) the twelfth anniversary of the first commercial sale of the licensed product in such country. We estimate that our core technology patents covering licensed products under the MDCO License Agreement will expire in most countries by 2029. We also estimate that our Leqvio (inclisiran) product-specific patents covering licensed products under the MDCO License Agreement will expire in the U.S. and Europe between 2027 and 2036, inclusive of any patent term extensions and/or any supplementary protection certificates extending such terms due to regulatory delay in those countries where such extensions are available. In addition, more patent filings relating to the collaboration may be made in the future. Either party may terminate the MDCO License Agreement in the event the other party fails to cure a material breach or upon patent-related challenges by the other party. In addition, Novartis has the right to terminate the agreement without cause at any time upon four months’ prior written notice. During the term of the MDCO License Agreement, neither party will, alone or with an affiliate or third party, research, develop or commercialize, or grant a license to any third party to research, develop or commercialize, in any country, any product (for Alnylam) and any siRNA product (for Novartis) directed to the PCSK9 gene, other than a licensed product, without the prior written agreement of the other party, subject to the terms of the MDCO License Agreement. We evaluated the MDCO License Agreement and concluded that Novartis meets the definition of a customer and that the MDCO License Agreement is a contract. We determined the transaction price, identified the performance obligations and allocated the transaction price to each performance obligation. We also determined that substantially all of our performance obligations are within the scope of the revenue standard as they relate to the delivery of goods and services to a customer for that customer’s use in monetizing an asset. Specifically, we concluded that Novartis meets the definition of a customer as we are delivering intellectual property and know-how rights as well as research and development activities. In addition, we determined that the MDCO License Agreement met the requirements to be accounted for as a contract, including that it is probable that we will collect the consideration to which we are entitled in exchange for the goods or services that will be delivered to Novartis. We determined that, pursuant to ASC 606, the performance obligations were not separately identifiable and were not distinct (and did not have standalone value) due to the specialized nature of the services to be provided by us and the dependent relationship between the performance obligations. Given this fact pattern, we have concluded the MDCO License Agreement has a single identified or combined performance obligation. None of the unearned milestones are included in the transaction price, as all unearned milestone amounts are not considered likely of achievement and therefore constrained. We considered several factors, including that achievement of the milestones is outside our control and contingent upon success in regulatory decisions. Any consideration related to sales-based royalties (including milestones) will be recognized when the related sales occur as they were determined to relate predominantly to the license granted to MDCO and as a result have also been excluded from the transaction price. During 2018, we completed the performance obligations identified in the MDCO License Agreement, including the supply and technical transfer agreement, however, we continue to receive additional orders for supply of certain material. We consider such orders as promised goods to be distinct from the other performance obligations since Novartis now has the ability to manufacture on its own through its own vendors. Such orders will be treated as separate agreements and any associated revenue will be recognized upon transfer of control. Novartis License Agreement In December 2021, we and Novartis entered into a collaboration and license agreement, or the Novartis License Agreement, pursuant to which we granted to Novartis an exclusive, worldwide license to develop, manufacture and commercialize siRNAs targeting end-stage liver disease, or ESLD, potentially leading to the development of a treatment designed to promote the regrowth of functional liver cells and to provide an alternative to transplantation for patients with liver failure. Pursuant to the Novartis License Agreement, we received an upfront fee of $12.5 million. We may also receive milestone payments upon the achievement of certain development, regulatory and commercial milestones, as well as royalties on the net sales of licensed products ranging from high-single-digit to sub-teen double-digit percentages. Due to the uncertainty of pharmaceutical development and the high historical failure rates generally associated with drug development, we may not receive any milestone or royalty payments under the Novartis License Agreement. Under the Novartis License Agreement, we are developing and testing potential siRNAs using target-specific assays developed by Novartis pursuant to an agreed upon research plan for a specified period referred to as the Collaboration Term. Novartis will reimburse us for the cost of our activities under the research plan, referred to as the DC Workplan, subject to an agreed upon cap. Once a lead candidate is identified, further development and clinical research will be conducted by Novartis. The collaboration is governed by a joint steering committee comprised of an equal number of representatives from each party. Unless terminated earlier in accordance with the terms of the Novartis License Agreement, the Collaboration Term expires at the earlier of (1) 180 days after completion of the development activities assigned to us as agreed upon between the parties, or (2) December 17, 2024. Either party may terminate the Novartis License Agreement in the event the other party fails to cure a material breach or upon patent-related challenges by the other party. In addition, Novartis has the right to terminate the agreement without cause at any time upon three months’ prior written notice. During the term of the Novartis License Agreement, neither party will, alone or with an affiliate or third party, research, develop or commercialize, or grant a license to any third party to research, develop or commercialize, in any country, any siRNA product directed to a liver target identified by Novartis, other than a licensed product, without the prior written agreement of the other party, subject to the terms of the Novartis License Agreement. We identified one performance obligation under the Novartis License Agreement comprised of: i) the exclusive license to develop, manufacture and commercialize siRNAs targeting ESLD; and ii) the obligation to perform work under the DC Workplan. The license is not distinct from the services, including the obligation to deliver development candidates, as Novartis cannot benefit on its own from the value of the license without receipt of such services. We measure proportional performance over time using an input method based on cost incurred relative to the total estimated costs for the identified performance obligation, on a quarterly basis, by determining the proportion of effort incurred as a percentage of total effort we expect to expend. This ratio is applied to the total transaction price. Management has applied significant judgment in the process of developing our estimates. Any changes to these estimates will be recognized in the period in which they change as a cumulative catch up. As of December 31, 2022, the total transaction price was determined to be approximately $16.0 million, comprised of the $12.5 million upfront payment and estimated variable consideration attributed to work to be performed under the DC Workplan. We utilized the expected value method to determine the amount of reimbursement for these activities. The total transaction price is allocated entirely to the single performance obligation. We determined any variable consideration related to sales-based royalties and milestones related to the exclusive license to be constrained and therefore excluded such consideration from the transaction price. As of December 31, 2022, the aggregate amount of the transaction price allocated to the performance obligation that was unsatisfied was $11.0 million, which is expected to be recognized through the term of the DC Workplan as the services are performed. Vir Biotechnology, Inc. In October 2017, we and Vir Biotechnology, Inc., or Vir, entered into a collaboration and license agreement, or the Vir Agreement, for the development and commercialization of RNAi therapeutics for infectious diseases, including chronic hepatitis B virus, or HBV, infection. Pursuant to the Vir Agreement, we granted to Vir an exclusive license to develop, manufacture and commercialize ALN-HBV02 (VIR-2218), for all uses and purposes other than certain excluded fields, as set forth in the Vir Agreement. In addition, we granted Vir an exclusive option for up to four additional RNAi therapeutic programs for the treatment of infectious diseases. Under the terms of the Vir Agreement, for each product arising from the HBV program, including ALN-HBV02, we retained the right to opt into a profit-sharing arrangement prior to the start of a Phase 3 clinical trial. In addition, we have the right on a product-by-product basis with respect to each additional infectious disease program that Vir elects to pursue, to opt into a profit-sharing arrangement for each such product at any time during a specified period prior to the initiation of a Phase 3 clinical trial for each such product. Under the Vir Agreement, we have earned and received certain upfront and development milestone payments in the form of cash ad Vir common stock, as well as sublicense revenue. We may receive additional milestone payments upon the achievement of certain development, regulatory and commercial milestones, as well as royalties on the net sales of licensed products ranging from high-single-digit to sub-teen double-digit percen |
LIABILITY RELATED TO THE SALE O
LIABILITY RELATED TO THE SALE OF FUTURE ROYALTIES | 12 Months Ended |
Dec. 31, 2022 | |
Related Party Transactions [Abstract] | |
LIABILITY RELATED TO THE SALE OF FUTURE ROYALTIES | LIABILITY RELATED TO THE SALE OF FUTURE ROYALTIES In April 2020, we entered into a purchase and sale agreement, or Purchase Agreement, with BX Bodyguard Royalties L.P. (an affiliate of The Blackstone Group Inc.), or Blackstone Royalties, under which Blackstone Royalties acquired 50% of royalties payable, or Royalty Interest, with respect to net sales by MDCO, its affiliates or sublicensees of inclisiran (or the branded drug product, Leqvio) and any other licensed products under the MDCO License Agreement, and 75% of the commercial milestone payments payable under the MDCO License Agreement, together with the Royalty Interest, the Purchased Interest. If Blackstone Royalties does not receive payments in respect of the Royalty Interest by December 31, 2029, equaling at least $1.00 billion, Blackstone Royalties will receive 55% of the Royalty Interest beginning on January 1, 2030. In consideration for the sale of the Purchased Interest, Blackstone Royalties paid us $1.00 billion. We continue to own or control all inclisiran intellectual property rights and are responsible for certain ongoing manufacturing and supply obligations related to the generation of the Purchased Interest. Due to our continuing involvement, we will continue to account for any royalties and commercial milestones due to us under the MDCO License Agreement as revenue on our consolidated statement of operations and comprehensive loss and record the proceeds from this transaction as a liability, net of closing costs, on our consolidated balance sheet. In order to determine the amortization of the liability related to the sale of future royalties, we are required to estimate the total amount of future payments to Blackstone Royalties over the life of the Purchase Agreement. The $1.00 billion liability, recorded at execution of the agreement, will be accreted to the total of these royalty and commercial milestone payments as interest expense over the life of the Purchase Agreement. As of December 31, 2022, our estimate of this total interest expense resulted in an effective annual interest rate of 8%. These estimates contain assumptions that impact both the amount recorded at execution and the interest expense that will be recognized in future periods. As payments are made to Blackstone Royalties, the balance of the liability will be effectively repaid over the life of the Purchase Agreement. The exact timing and amount of repayment is likely to change each reporting period. A significant increase or decrease in Leqvio global net revenue will materially impact the liability related to the sale of future royalties, interest expense and the time period for repayment. We will periodically assess the expected payments to Blackstone Royalties and to the extent the amount or timing of such payments is materially different than our initial estimates, we will prospectively adjust the amortization of the liability related to the sale of future royalties and the related interest expense. As of December 31, 2022, the carrying value of the liability related to the sale of future royalties was $1.29 billion, net of closing costs of $10.8 million. The carrying value of the liability related to the sale of future royalties approximates fair value as of December 31, 2022 and is based on our current estimates of future royalties and commercial milestones expected to be paid to Blackstone Royalties over the life of the arrangement, which are considered Level 3 inputs. The following table shows the activity with respect to the liability related to the sale of future royalties, in thousands: Carrying value as of December 31, 2020 $ 1,071,541 Interest expense recognized 116,940 Payments (378) Carrying value as of December 31, 2021 1,188,103 Interest expense recognized 107,601 Payments (3,400) Carrying value as of December 31, 2022 $ 1,292,304 |
FAIR VALUE MEASUREMENTS
FAIR VALUE MEASUREMENTS | 12 Months Ended |
Dec. 31, 2022 | |
Fair Value Disclosures [Abstract] | |
FAIR VALUE MEASUREMENTS | FAIR VALUE MEASUREMENTS The following tables present information about our financial assets and liabilities that are measured at fair value on a recurring basis and indicate the fair value hierarchy of the valuation techniques we utilized to determine such fair value: (In thousands) As of December 31, Quoted Prices in Active Markets Significant Observable Inputs Significant Unobservable Inputs Financial assets Cash equivalents: Money market funds $ 270,394 $ 270,394 $ — $ — U.S. treasury securities 44,817 — 44,817 — U.S. government-sponsored enterprise securities 41,763 — 41,763 — Commercial paper 22,350 — 22,350 — Certificates of deposit 3,289 — 3,289 — Corporate notes 1,024 — 1,024 — Marketable debt securities: U.S. treasury securities 820,913 — 820,913 — U.S. government-sponsored enterprise securities 230,770 — 230,770 — Corporate notes 208,284 — 208,284 — Commercial paper 36,793 — 36,793 — Certificates of deposit 1,130 — 1,130 — Marketable equity securities 28,122 28,122 — — Restricted cash (money market funds) 1,197 1,197 — — Total financial assets $ 1,710,846 $ 299,713 $ 1,411,133 $ — Financial liabilities Development derivative liability $ 209,277 $ — $ — $ 209,277 (In thousands) As of December 31, Quoted Prices in Active Markets Significant Observable Inputs Significant Unobservable Inputs Financial assets Cash equivalents: Money market funds $ 255,869 $ 255,869 $ — $ — U.S. treasury securities 54,998 — 54,998 — Marketable debt securities: U.S. treasury securities 1,030,578 — 1,030,578 — Corporate notes 253,239 — 253,239 — U.S. government-sponsored enterprise securities 177,741 — 177,741 — Commercial paper 78,543 — 78,543 — Certificates of deposit 7,501 — 7,501 — Municipal securities 1,015 — 1,015 — Marketable equity securities 66,972 66,972 — — Restricted cash (money market funds) 1,195 1,195 — — Total financial assets $ 1,927,651 $ 324,036 $ 1,603,615 $ — Financial liabilities Development derivative liability $ 83,618 $ — $ — $ 83,618 |
MARKETABLE DEBT SECURITIES
MARKETABLE DEBT SECURITIES | 12 Months Ended |
Dec. 31, 2022 | |
Investments, Debt and Equity Securities [Abstract] | |
MARKETABLE DEBT SECURITIES | MARKETABLE DEBT SECURITIES The following tables summarize our marketable debt securities: As of December 31, 2022 (In thousands) Amortized Gross Gross Fair Value U.S. treasury securities $ 870,033 $ 79 $ (4,382) $ 865,730 U.S. government-sponsored enterprise securities 275,610 24 (3,101) 272,533 Corporate notes 211,398 16 (2,106) 209,308 Commercial paper 59,143 — — 59,143 Certificates of deposit 4,419 — — 4,419 Total $ 1,420,603 $ 119 $ (9,589) $ 1,411,133 As of December 31, 2021 (In thousands) Amortized Gross Gross Fair Value U.S. treasury securities $ 1,086,232 $ 6 $ (662) $ 1,085,576 Corporate notes 253,926 1 (688) 253,239 U.S. government-sponsored enterprise securities 178,027 2 (288) 177,741 Commercial paper 78,543 — — 78,543 Certificates of deposit 7,501 — — 7,501 Municipal securities 1,016 — (1) 1,015 Total $ 1,605,245 $ 9 $ (1,639) $ 1,603,615 The fair values of our marketable debt securities by classification in the consolidated balance sheets were as follows: (In thousands) December 31, 2022 December 31, 2021 Cash and cash equivalents $ 113,243 $ 54,998 Marketable debt securities 1,297,890 1,548,617 Total $ 1,411,133 $ 1,603,615 |
OTHER BALANCE SHEET DETAILS
OTHER BALANCE SHEET DETAILS | 12 Months Ended |
Dec. 31, 2022 | |
Balance Sheet Related Disclosures [Abstract] | |
OTHER BALANCE SHEET DETAILS | OTHER BALANCE SHEET DETAILS Inventory The components of inventory are summarized as follows: As of December 31, (In thousands) 2022 2021 Raw materials $ 22,315 $ 14,754 Work in process 113,783 100,942 Finished goods 25,606 7,005 Total inventory $ 161,704 $ 122,701 As of December 31, 2022 and 2021, we had $32.7 million and $36.3 million of long-term inventory, respectively, included within other assets in our consolidated balance sheet as we anticipate it being consumed beyond our normal operating cycle. As of December 31, 2022 and 2021, we had $0.0 million and $7.1 million, respectively, of capitalized inventory produced for commercial sale for products awaiting regulatory approval. Property, Plant and Equipment, Net Property, plant and equipment, net consist of the following: As of December 31, (In thousands) 2022 2021 Buildings $ 269,322 $ 262,637 Leasehold improvements 230,848 152,045 Construction in progress 14,595 80,753 Laboratory equipment 82,586 61,351 Manufacturing equipment 45,311 43,739 Computer equipment and software 33,370 21,885 Furniture and fixtures 11,832 10,883 Land 9,080 9,080 696,944 642,373 Less: accumulated depreciation (173,450) (140,415) Total $ 523,494 $ 501,958 Accrued Expenses Accrued expenses consist of the following: As of December 31, (In thousands) 2022 2021 Product rebates and discounts $ 208,998 $ 131,279 Compensation and related 130,690 93,583 Pre-clinical, clinical trial and manufacturing 94,702 83,534 Licensing and collaboration agreements 31,680 22,843 Consulting and professional services 19,848 17,784 Other 59,542 46,151 Total $ 545,460 $ 395,174 Cash, Cash Equivalents and Restricted Cash The following table provides a reconciliation of cash, cash equivalents and restricted cash reported within our consolidated balance sheets that sum to the total of these amounts shown in the consolidated statements of cash flows: As of December 31, (In thousands) 2022 2021 2020 Cash and cash equivalents $ 866,394 $ 819,975 $ 496,580 Total restricted cash included in other assets 2,162 2,178 2,466 Total cash, cash equivalents, and restricted cash shown in the consolidated statements of cash flows $ 868,556 $ 822,153 $ 499,046 Accumulated Other Comprehensive (Loss) Income The following table summarizes the changes in accumulated other comprehensive (loss) income, by component: (In thousands) Loss on Investment in Joint Venture Defined Benefit Pension Unrealized Gains (Losses) from Debt Securities Foreign Currency Translation Total Accumulated Other Comprehensive (Loss) Income Balance as of December 31, 2020 $ (32,792) $ (3,754) $ 348 $ (7,424) $ (43,622) Other comprehensive loss before reclassifications — 899 — 11,398 12,297 Amounts reclassified from other comprehensive income — 44 (1,978) — (1,934) Net other comprehensive income (loss) — 943 (1,978) 11,398 10,363 Balance as of December 31, 2021 (32,792) (2,811) (1,630) 3,974 (33,259) Other comprehensive income before reclassifications — — 11 (5,274) (5,263) Amounts reclassified from other comprehensive income — 1,719 (7,851) — (6,132) Net other comprehensive income (loss) — 1,719 (7,840) (5,274) (11,395) Balance as of December 31, 2022 $ (32,792) $ (1,092) $ (9,470) $ (1,300) $ (44,654) |
CREDIT AGREEMENT
CREDIT AGREEMENT | 12 Months Ended |
Dec. 31, 2022 | |
Line of Credit Facility [Abstract] | |
CREDIT AGREEMENT | CREDIT AGREEMENT In April 2020, we entered into a credit agreement, or Credit Agreement, among us, certain of our subsidiaries (such subsidiaries, together with us, the Loan Parties), funds or accounts managed or advised by GSO Capital Partners LP (now Blackstone Alternative Credit Advisors LP) and certain other affiliates of T he Blackstone Group Inc. , and the other lenders from time to time parties thereto, collectively, the Lenders, and Wilmington Trust, National Association, as the administrative agent for the Lenders. The Credit Agreement provided for a senior secured delayed draw term loan facility, referred to as the Term Loans, which consisted of three tranches providing funding of $700.0 million. In September 2022, we extinguished the Term Loans and paid all outstanding balances, including principal of $700.0 million and prepayment premiums of $62.1 million and terminated the Credit Agreement. Upon termination, we recorded a $76.6 million loss on the extinguishment of the debt. The Term Loans were set to mature in December 2027. During the period the Term Loans were outstanding, we had elected a LIBOR Rate plus 7%, and paid $17.5 million in total funding fees in connection with such Term Loans. Our interest rate was 9% and 8% as of June 30, 2022 and December 31, 2021, respectively. Convertible Senior Notes Due 2027 On September 12, 2022, we commenced a private offering of $900.0 million in aggregate principal amount of 1% Convertible Senior Notes due 2027, or the Initial Notes. On September 13, 2022, the initial purchasers in such offering exercised their option to purchase an additional $135.0 million in aggregate principal amount of our 1% Convertible Senior Notes due 2027, or the Additional Notes, and together with the Initial Notes collectively referred to as the Notes, bringing the total aggregate principal amount of the Notes to $1.04 billion. The Notes were issued pursuant to an indenture, dated September 15, 2022, or the Indenture. The Indenture includes customary covenants and sets forth certain events of default after which the Notes may be declared immediately due and payable and sets forth certain types of bankruptcy or insolvency events of default involving the Company after which the Notes become automatically due and payable. The Notes will mature on September 15, 2027, unless earlier converted, redeemed or repurchased. The Notes will bear interest from September 15, 2022 at a rate of 1% per year payable semiannually in arrears on March 15 and September 15 of each year, beginning on March 15, 2023. The Notes are convertible at the option of the noteholder on or after June 15, 2027. Prior to June 15, 2027, the Notes are convertible only under the following circumstances: (1) During any calendar quarter commencing after the calendar quarter ending on December 31, 2022 (and only during such calendar quarter), if the last reported sale price of our common stock for at least 20 trading days (whether or not consecutive) during a period of 30 consecutive trading days ending on, and including, the last trading day of the immediately preceding calendar quarter is greater than or equal to 130% of the conversion price on each applicable trading day; (2) During the five business day period after any ten consecutive trading day period in which the trading price per $1,000 principal amount of the Notes for each trading day of that ten consecutive trading day period was less than 98% of the product of the last reported sale price of our common stock and the conversion rate of the Notes on such trading day; (3) If we call any or all of the Notes for redemption; or (4) Upon the occurrence of specific corporate events as set forth in the Indenture governing the Notes. We will settle any conversions of Notes by paying or delivering, as applicable, cash, shares of our common stock, or a combination of cash and shares of common stock, at our election. The conversion rate for the Notes will initially be 3.4941 shares of common stock per $1,000 principal amount of Notes, which is equivalent to an initial conversion price of approximately $286.20 per share of common stock. The initial conversion price of the Notes represents a premium of approximately 35% over the $212.00 per share last reported sale price of common stock on September 12, 2022. The conversion rate is subject to adjustment under certain circumstances in accordance with the terms of the Indenture. We may not redeem the Notes prior to September 20, 2025. We may redeem for cash equal to 100% of the principal amount of the Notes being redeemed plus accrued and unpaid interest all or any portion of the Notes, at our option, on or after September 20, 2025, if the last reported sales price of our common stock has been at least 130% of the conversion price then in effect for at least 20 trading days (whether or not consecutive) during any 30 consecutive trading day period. No sinking fund is provided for the Notes and therefore we are not required to redeem or retire the Notes periodically. If we undergo a fundamental change, as defined in the indenture agreement, then subject to certain conditions, holders may require us to repurchase for cash all or any portion of their Notes at a fundamental change repurchase price equal to 100% of the principal amount of the Notes to be repurchased plus accrued and unpaid interest. In addition, if specific corporate events occur prior to the maturity date or if we issue a notice of redemption, we will increase the conversion rate by pre-defined amounts for holders who elect to convert their notes in connection with such a corporate event. The conditions allowing holders of the Notes to convert were not met this quarter. As of December 31, 2022, the Notes are classified as a long-term liability, net of issuance costs of $19.2 million, on the consolidated balance sheets. As of December 31, 2022, the estimated fair value of the Notes was approximately $1.12 billion. The fair value was determined based on the last actively traded price per $100 of the Notes for the period ended December 31, 2022 (Level 2). Interest expense recognized related to the Notes for the twelve months ended December 31, 2022 was $3.0 million. The Notes were issued at par and costs associated with the issuance of the Notes are amortized to interest expense over the contractual term of the Notes. As of December 31, 2022, the effective interest rate of the Notes is 1%. Capped Call Transactions In September 2022, in connection with the pricing of the Initial Notes and the initial purchasers’ exercise of their option to purchase the Additional Notes, we entered into privately negotiated capped call transactions, or Capped Call Transactions. The Capped Call Transactions initially cover, subject to customary anti-dilution adjustments, the number of shares of common stock that underlie the Notes. The cap price of the Capped Call Transactions is initially $424.00 per share, which represents a premium of 100% over the last reported sale price of common stock of $212.00 per share on September 12, 2022, and is subject to certain adjustments under the terms of the capped call transactions. We used approximately $118.6 million of the proceeds from the offering of Notes to pay the cost of the Capped Call Transactions. |
CONVERTIBLE DEBT
CONVERTIBLE DEBT | 12 Months Ended |
Dec. 31, 2022 | |
Debt Disclosure [Abstract] | |
CONVERTIBLE DEBT | CREDIT AGREEMENT In April 2020, we entered into a credit agreement, or Credit Agreement, among us, certain of our subsidiaries (such subsidiaries, together with us, the Loan Parties), funds or accounts managed or advised by GSO Capital Partners LP (now Blackstone Alternative Credit Advisors LP) and certain other affiliates of T he Blackstone Group Inc. , and the other lenders from time to time parties thereto, collectively, the Lenders, and Wilmington Trust, National Association, as the administrative agent for the Lenders. The Credit Agreement provided for a senior secured delayed draw term loan facility, referred to as the Term Loans, which consisted of three tranches providing funding of $700.0 million. In September 2022, we extinguished the Term Loans and paid all outstanding balances, including principal of $700.0 million and prepayment premiums of $62.1 million and terminated the Credit Agreement. Upon termination, we recorded a $76.6 million loss on the extinguishment of the debt. The Term Loans were set to mature in December 2027. During the period the Term Loans were outstanding, we had elected a LIBOR Rate plus 7%, and paid $17.5 million in total funding fees in connection with such Term Loans. Our interest rate was 9% and 8% as of June 30, 2022 and December 31, 2021, respectively. Convertible Senior Notes Due 2027 On September 12, 2022, we commenced a private offering of $900.0 million in aggregate principal amount of 1% Convertible Senior Notes due 2027, or the Initial Notes. On September 13, 2022, the initial purchasers in such offering exercised their option to purchase an additional $135.0 million in aggregate principal amount of our 1% Convertible Senior Notes due 2027, or the Additional Notes, and together with the Initial Notes collectively referred to as the Notes, bringing the total aggregate principal amount of the Notes to $1.04 billion. The Notes were issued pursuant to an indenture, dated September 15, 2022, or the Indenture. The Indenture includes customary covenants and sets forth certain events of default after which the Notes may be declared immediately due and payable and sets forth certain types of bankruptcy or insolvency events of default involving the Company after which the Notes become automatically due and payable. The Notes will mature on September 15, 2027, unless earlier converted, redeemed or repurchased. The Notes will bear interest from September 15, 2022 at a rate of 1% per year payable semiannually in arrears on March 15 and September 15 of each year, beginning on March 15, 2023. The Notes are convertible at the option of the noteholder on or after June 15, 2027. Prior to June 15, 2027, the Notes are convertible only under the following circumstances: (1) During any calendar quarter commencing after the calendar quarter ending on December 31, 2022 (and only during such calendar quarter), if the last reported sale price of our common stock for at least 20 trading days (whether or not consecutive) during a period of 30 consecutive trading days ending on, and including, the last trading day of the immediately preceding calendar quarter is greater than or equal to 130% of the conversion price on each applicable trading day; (2) During the five business day period after any ten consecutive trading day period in which the trading price per $1,000 principal amount of the Notes for each trading day of that ten consecutive trading day period was less than 98% of the product of the last reported sale price of our common stock and the conversion rate of the Notes on such trading day; (3) If we call any or all of the Notes for redemption; or (4) Upon the occurrence of specific corporate events as set forth in the Indenture governing the Notes. We will settle any conversions of Notes by paying or delivering, as applicable, cash, shares of our common stock, or a combination of cash and shares of common stock, at our election. The conversion rate for the Notes will initially be 3.4941 shares of common stock per $1,000 principal amount of Notes, which is equivalent to an initial conversion price of approximately $286.20 per share of common stock. The initial conversion price of the Notes represents a premium of approximately 35% over the $212.00 per share last reported sale price of common stock on September 12, 2022. The conversion rate is subject to adjustment under certain circumstances in accordance with the terms of the Indenture. We may not redeem the Notes prior to September 20, 2025. We may redeem for cash equal to 100% of the principal amount of the Notes being redeemed plus accrued and unpaid interest all or any portion of the Notes, at our option, on or after September 20, 2025, if the last reported sales price of our common stock has been at least 130% of the conversion price then in effect for at least 20 trading days (whether or not consecutive) during any 30 consecutive trading day period. No sinking fund is provided for the Notes and therefore we are not required to redeem or retire the Notes periodically. If we undergo a fundamental change, as defined in the indenture agreement, then subject to certain conditions, holders may require us to repurchase for cash all or any portion of their Notes at a fundamental change repurchase price equal to 100% of the principal amount of the Notes to be repurchased plus accrued and unpaid interest. In addition, if specific corporate events occur prior to the maturity date or if we issue a notice of redemption, we will increase the conversion rate by pre-defined amounts for holders who elect to convert their notes in connection with such a corporate event. The conditions allowing holders of the Notes to convert were not met this quarter. As of December 31, 2022, the Notes are classified as a long-term liability, net of issuance costs of $19.2 million, on the consolidated balance sheets. As of December 31, 2022, the estimated fair value of the Notes was approximately $1.12 billion. The fair value was determined based on the last actively traded price per $100 of the Notes for the period ended December 31, 2022 (Level 2). Interest expense recognized related to the Notes for the twelve months ended December 31, 2022 was $3.0 million. The Notes were issued at par and costs associated with the issuance of the Notes are amortized to interest expense over the contractual term of the Notes. As of December 31, 2022, the effective interest rate of the Notes is 1%. Capped Call Transactions In September 2022, in connection with the pricing of the Initial Notes and the initial purchasers’ exercise of their option to purchase the Additional Notes, we entered into privately negotiated capped call transactions, or Capped Call Transactions. The Capped Call Transactions initially cover, subject to customary anti-dilution adjustments, the number of shares of common stock that underlie the Notes. The cap price of the Capped Call Transactions is initially $424.00 per share, which represents a premium of 100% over the last reported sale price of common stock of $212.00 per share on September 12, 2022, and is subject to certain adjustments under the terms of the capped call transactions. We used approximately $118.6 million of the proceeds from the offering of Notes to pay the cost of the Capped Call Transactions. |
DEVELOPMENT DERIVATIVE LIABILIT
DEVELOPMENT DERIVATIVE LIABILITY | 12 Months Ended |
Dec. 31, 2022 | |
Derivative Instruments and Hedging Activities Disclosure [Abstract] | |
DEVELOPMENT DERIVATIVE LIABILITY | DEVELOPMENT DERIVATIVE LIABILITY In August 2020, we entered into a co-development agreement, referred to as the Funding Agreement, with BXLS V Bodyguard – PCP L.P. and BXLS Family Investment Partnership V – ESC L.P., collectively referred to as Blackstone Life Sciences, pursuant to which Blackstone Life Sciences will provide up to $150.0 million in funding for the clinical development of vutrisiran and zilebesiran, two of our cardiometabolic programs. With respect to vutrisiran, Blackstone Life Sciences has committed to provide up to $70.0 million to fund development costs related to the HELIOS-B Phase 3 clinical trial. In November 2021, Blackstone Life Sciences opted in to Phase 2 clinical trial funding of zilebesiran, committing to fund, upon meeting certain patient enrollment thresholds, up to $26.0 million. Furthermore, Blackstone Life Sciences has the right, but is not obligated, to fund up to $54.0 million for development costs related to a Phase 3 clinical trial of zilebesiran. The amount of funding ultimately provided by Blackstone Life Sciences is dependent on us achieving specified development milestones with respect to each clinical trial. We retain sole responsibility for the development and commercialization of both vutrisiran and zilebesiran. As consideration for Blackstone Life Sciences’ funding for vutrisiran clinical development costs, we have agreed to pay Blackstone Life Sciences a 1% royalty on net sales of AMVUTTRA (vutrisiran) for a 10-year term beginning upon the first commercial sale following regulatory approval of vutrisiran for ATTR-cardiomyopathy, as well as fixed payments of up to 2.5 times their investment over a two-year period upon regulatory approval of vutrisiran for ATTR-cardiomyopathy in specified countries, unless it is later withdrawn from the market following a mandatory recall. As consideration for Blackstone Life Sciences’ funding for Phase 2 clinical development costs of zilebesiran, we have agreed to pay Blackstone Life Sciences fixed payments of up to 3.25 times their Phase 2 investment over a four-year period upon the successful completion of the zilebesiran Phase 2 clinical trial, unless certain regulatory events affecting the continued development of zilebesiran occur. As consideration for Blackstone Life Sciences’ funding for Phase 3 clinical development costs of zilebesiran, we have agreed to pay Blackstone Life Sciences fixed payments of up to 4.5 times their Phase 3 investment over a four-year period upon regulatory approval of zilebesiran in specified countries, unless it is later withdrawn from the market following a mandatory recall. Our payment obligations under the Funding Agreement will be secured, subject to certain exceptions, by security interests in intellectual property owned by us relating to vutrisiran and zilebesiran, as well as in our bank account in which the funding deposits will be made. We and Blackstone Life Sciences each have the right to terminate the Funding Agreement in its entirety in the event of the other party’s bankruptcy or similar proceedings. We and Blackstone Life Sciences may each terminate the Funding Agreement in its entirety or with respect to either product in the event of an uncured material breach by the other party, or with respect to a product for certain patient health and safety reasons, or if regulatory approval in specified major market countries is not obtained for the product following the completion of clinical trials for the product. In addition, Blackstone Life Sciences has the right to terminate the Funding Agreement in its entirety upon the occurrence of certain events affecting our ability to make payments under the agreement or to develop or commercialize the products, or upon a change of control of us. Blackstone Life Sciences may also terminate the Funding Agreement with respect to a product if the joint steering committee elects to terminate the development program for that product in its entirety, if certain clinical endpoints are not achieved for that product or, with respect to vutrisiran only, if our right to develop or commercialize vutrisiran is enjoined in a specified major market as a result of an alleged patent infringement. In certain termination circumstances, we will be obligated to pay Blackstone Life Sciences an amount that is equal to, or a multiplier of, the development funding received from Blackstone Life Sciences, and we may remain obligated under certain circumstances to make the payments to Blackstone Life Sciences described above, or the royalty described above in the case of AMVUTTRA, should we obtain regulatory approval for zilebesiran or for vutrisiran for ATTR-cardiomyopathy following termination. We account for the Funding Agreement under ASC Topic 815, Derivatives and Hedging, as a derivative liability, measured at fair value, within other liabilities on our consolidated balance sheets. The change in fair value due to the remeasurement of the development derivative liability is recorded as other expense on our consolidated statements of operations and comprehensive loss. As of December 31, 2022 and 2021, the derivative liability is classified as a Level 3 financial liability in the fair value hierarchy. The valuation method incorporates certain unobservable Level 3 key inputs including (i) the probability and timing of achieving stated development milestones to receive payments from Blackstone Life Sciences, (ii) the probability and timing of achieving regulatory approval and payments to Blackstone Life Sciences, (iii) an estimate of the amount and timing of the royalty payable on net sales of AMVUTTRA, assuming regulatory approval for ATTR-cardiomyopathy, (iv) our cost of borrowing (12%), and (v) Blackstone Life Sciences' cost of borrowing (4%). The following table presents the activity with respect to the development derivative liability, in thousands: Carrying value as of December 31, 2020 $ 25,585 Amount received under the Funding Agreement 19,600 Loss recorded from remeasurement 38,433 Carrying value as of December 31, 2021 83,618 Amount received under the Funding Agreement 31,000 Loss recorded from remeasurement 94,659 Carrying value as of December 31, 2022 $ 209,277 |
STOCK-BASED COMPENSATION
STOCK-BASED COMPENSATION | 12 Months Ended |
Dec. 31, 2022 | |
Share-Based Payment Arrangement [Abstract] | |
STOCK-BASED COMPENSATION | STOCK-BASED COMPENSATION Stock Plans In May 2020, our stockholders approved a second amendment to our 2018 Stock Incentive Plan, as amended, to increase the number of shares authorized for issuance thereunder by 7,000,000 shares, and in May 2022, our stockholders approved the amendment and restatement of our 2018 Stock Incentive Plan, as amended, or the Amended and Restated 2018 Plan, which increased the number of shares authorized for issuance thereunder by 6,000,000 shares. The Amended and Restated 2018 Plan provides for the granting of stock options, restricted stock and restricted stock units (together, restricted stock awards), stock appreciation rights and other stock-based awards, and has a fungible share pool. Any award that is not a full value award is counted against the authorized share limits specified as one share for each share of common stock subject to the award, and all full value awards, defined as restricted stock awards or other stock-based awards, are counted as one and a half shares for each one share of common stock subject to such full value award. As of December 31, 2022, an aggregate of 20,711,629 shares of common stock were reserved for issuance under our stock plans, including outstanding stock options to purchase 8,423,552 shares of common stock, 1,487,394 outstanding restricted stock units, 10,083,911 of common stock available for additional equity awards and 716,772 shares available for future grant under our Amended and Restated 2004 Employee Stock Purchase Plan, as amended, or the Amended and Restated ESPP. Each stock option shall expire within 10 years of issuance. Time-based stock options granted to employees generally vest as to 25% of the shares on the first anniversary of the grant date and 6.25% of the shares at the end of each successive three-month period thereafter until fully vested. Stock-Based Compensation The following table summarizes stock-based compensation expenses included in operating costs and expenses: Year Ended December 31, (In thousands) 2022 2021 2020 Research and development $ 92,161 $ 68,415 $ 60,464 Selling, general and administrative 138,488 97,302 79,409 Total $ 230,649 $ 165,717 $ 139,873 The following table summarizes stock-based compensation expense: Year Ended December 31, (In thousands) 2022 2021 2020 Stock-based compensation expense by type of award: Time-based stock options $ 114,901 $ 118,635 $ 112,971 Time-based restricted stock units 12,791 4,231 6,909 Performance-based restricted stock units 102,925 39,943 11,162 Other equity programs 4,057 6,235 9,402 Less: Stock-based compensation expense capitalized to inventory (4,025) (3,327) (571) Total $ 230,649 $ 165,717 $ 139,873 The following table summarizes our unrecognized stock-based compensation expense, net of estimated forfeitures, by type of awards, and the weighted-average period over which that expense is expected to be recognized: As of December 31, 2022 Unrecognized Expense, Net of Estimated Forfeitures (in thousands) Weighted-average Recognition Period (in years) Type of award: Time-based stock options $ 187,534 2.47 Time-based restricted stock units $ 30,676 2.09 Performance-based restricted stock units * $ 7,695 0.58 Other equity programs $ 4,225 0.45 __________________________________________ * Excludes performance-based restricted stock units for which the associated vesting events are not yet determined to be probable. Valuation Assumptions for Stock Options The fair value of stock options, at date of grant, based on the following assumptions, was estimated using the Black-Scholes option-pricing model. Our expected stock-price volatility assumption is based on the historical volatility of our publicly traded stock. The expected life assumption is based on our historical data. The dividend yield assumption is based on the fact that we have never paid cash dividends and have no present intention to pay cash dividends. The risk-free interest rate used for each grant is equal to the zero coupon rate for instruments with a similar expected life. The following table summarizes the Black-Scholes valuation assumption inputs for employee stock options granted: Year Ended December 31, 2022 2021 2020 Risk-free interest rate 1.3 - 4.2% 0.4 - 1.4% 0.3 - 1.7% Expected dividend yield — — — Expected option life 5.1 - 7.0 years 5.4 - 6.8 years 5.4 - 7.2 years Expected volatility 50 - 60% 58 - 63% 61 - 63% Stock Option Activity The following table summarizes the activity of our stock option plans, excluding performance-based stock options: Number of Options (in thousands) Weighted-average Exercise Price (per share) Weighted-average Remaining Contractual Term (in years) Aggregate Intrinsic Value (in thousands) Outstanding as of December 31, 2021 8,840 $ 103.87 Granted 1,874 $ 153.18 Exercised (2,490) $ 84.48 Cancelled (356) $ 129.47 Outstanding as of December 31, 2022 7,868 $ 120.59 6.53 $ 921,016 Exercisable as of December 31, 2022 4,450 $ 102.17 5.22 $ 602,969 Vested or expected to vest as of December 31, 2022 7,537 $ 119.24 6.44 $ 892,463 The weighted-average fair value of stock options granted was $80.65, $82.59 and $66.28 per share for the years ended December 31, 2022, 2021 and 2020, respectively. The intrinsic value of stock options exercised was $289.3 million, $247.8 million and $177.8 million for the years ended December 31, 2022, 2021 and 2020, respectively. We satisfy stock option exercises with newly issued shares of our common stock. Performance-Based Stock Options The following table summarizes the activity of our performance-based stock options granted under our equity plans: Number of Options (in thousands) Weighted-average Exercise Price (per share) Weighted-average Remaining Contractual Term (in years) Aggregate Intrinsic Value (in thousands) Outstanding as of December 31, 2021 1,175 $ 92.31 Granted — $ — Exercised (619) $ 87.85 Cancelled — $ — Outstanding as of December 31, 2022 556 $ 97.28 3.81 $ 77,975 Exercisable as of December 31, 2022 556 $ 97.28 3.81 $ 77,975 During the years ended December 31, 2022, 2021 and 2020, there were 0, 197,102 and 0 performance-based stock options that vested, respectively. The intrinsic value of performance-based stock options exercised was $74.4 million, $40.2 million and $34.1 million for the years ended December 31, 2022, 2021 and 2020, respectively. We satisfy performance-based stock option exercises with newly issued shares of our common stock. Restricted Stock Units and Awards The following table summarizes the activity of our restricted stock units and awards granted under our equity plans, excluding performance-based restricted stock units: Number of Units (in thousands) Weighted-average Grant Date Fair Value (per share) Outstanding as of December 31, 2021 75 $ 158.87 Awarded 249 $ 165.32 Released (23) $ 152.98 Cancelled (9) $ 143.85 Outstanding as of December 31, 2022 292 $ 165.27 Performance-Based Restricted Stock Units The following table summarizes the activity of our performance-based restricted stock units granted under our equity plans: Number of Units (in thousands) Weighted-average Grant Date Fair Value (per share) Outstanding as of December 31, 2021 1,136 $ 143.13 Awarded 690 $ 147.47 Released (501) $ 142.72 Cancelled (130) $ 143.95 Outstanding as of December 31, 2022 1,195 $ 144.77 The performance-based restricted stock units granted in 2022 and 2021 will vest upon the later of the one-year anniversary of the date of grant and the achievement of specific clinical development, regulatory, commercial and/or financial performance events, as approved by our people, culture and compensation committee. Employee Stock Purchase Plan In 2004, we adopted the 2004 Employee Stock Purchase Plan and in 2017, our stockholders approved the Amended and Restated ESPP. In 2020, our stockholders approved an amendment to the Amended and Restated ESPP, to increase the number of shares authorized for issuance to 1,965,789 shares. Under the Amended and Restated ESPP, as amended, each offering period is six months, at the end of which employees may purchase shares of common stock through payroll deductions made over the term of the offering. The per-share purchase price at the end of each offering period is equal to the lesser of 85% of the closing price of our common stock at the beginning or end of the offering period. We issued 119,285 and 124,101 shares during the years ended December 31, 2022 and 2021, respectively. We estimate the fair value of shares to be issued under the Amended and Restated ESPP, as amended, using the Black-Scholes option-pricing model on the date of grant, or first day of the offering period, using the same methodology approach as the employee stock option grants. The following table summarizes the Black-Scholes valuation assumption inputs for stock purchase rights granted under the employee stock purchase plan: Year Ended December 31, 2022 2021 2020 Risk-free interest rate 1.4% - 4.5% 0.03% - 0.06% 0.1% - 0.1% Expected dividend yield — — — Expected option life 6 months 6 months 6 months Expected volatility 53% - 71% 41% - 46% 40% - 50% |
STOCKHOLDERS' (DEFICIT) EQUITY
STOCKHOLDERS' (DEFICIT) EQUITY | 12 Months Ended |
Dec. 31, 2022 | |
Equity [Abstract] | |
STOCKHOLDERS' (DEFICIT) EQUITY | STOCKHOLDERS’ (DEFICIT) EQUITY Preferred Stock We have authorized up to 5,000,000 shares of preferred stock, $0.01 par value per share, for issuance. The preferred stock will have such rights, preferences, privileges and restrictions, including voting rights, dividend rights, conversion rights, redemption privileges and liquidation preferences, as shall be determined by our board of directors upon its issuance. As of December 31, 2022 and 2021, there were no shares of preferred stock outstanding. Blackstone Equity Placement In April 2020, we entered into a stock purchase agreement, or Investors SPA, with certain affiliates of The Blackstone Group Inc., or Investors, pursuant to which we sold 963,486 shares of our common stock to the Investors for aggregate cash consideration of $100.0 million, or $103.79 per share, as part of the broad strategic financing collaboration with The Blackstone Group Inc. The Investors SPA contains customary representations, warranties, and covenants of each of the parties thereto. |
COMMITMENTS AND CONTINGENCIES
COMMITMENTS AND CONTINGENCIES | 12 Months Ended |
Dec. 31, 2022 | |
Commitments and Contingencies Disclosure [Abstract] | |
COMMITMENTS AND CONTINGENCIES | COMMITMENTS AND CONTINGENCIES Technology License and Other Commitments We have licensed from third parties the rights to use certain technologies and information in our research processes as well as in any other products we may develop. In accordance with the related license or technology agreements, we are required to make certain fixed payments to the licensor or a designee of the licensor over various agreement terms. Many of these agreement terms are consistent with the remaining lives of the underlying intellectual property that we have licensed. As of December 31, 2022, our commitments over the next five years to make fixed and cancellable payments under existing license agreements were not material. Legal Matters From time to time, we may be a party to litigation, arbitration or other legal proceedings in the course of our business, including the matters described below. The claims and legal proceedings in which we could be involved include challenges to the scope, validity or enforceability of patents relating to our products or product candidates, and challenges by us to the scope, validity or enforceability of the patents held by others. These include claims by third parties that we infringe their patents or breach our license or other agreements with such third parties. The outcome of any such legal proceedings, regardless of the merits, is inherently uncertain. In addition, litigation and related matters are costly and may divert the attention of our management and other resources that would otherwise be engaged in other activities. If we were unable to prevail in any such legal proceedings, our business, results of operations, liquidity and financial condition could be adversely affected. Our accounting policy for accrual of legal costs is to recognize such expenses as incurred. Government Investigation We have previously disclosed that, on or about April 9, 2021, we received a subpoena from the U.S. Department of Justice, U.S. Attorney’s Office for the District of Massachusetts, requiring production of documents pertaining to our marketing and promotion of ONPATTRO (patisiran) in the U.S. We are cooperating with the U.S. Attorney’s Office and producing documents in response to the subpoena. Current and former officers and employees also have received subpoenas in connection with the preservation and production of related materials. Given the ongoing nature of the investigation, it is possible that the U.S. Attorney’s Office for the District of Massachusetts or other government entities may request other information from, or issue other subpoenas, findings or similar documents to, us, our related entities and their respective directors, officers and employees. In light of the ongoing nature of the investigation, no determination has been made that a loss, if any, arising from this matter is probable or that the amount of any such loss, or range of loss, is reasonably estimable. We also previously disclosed that since learning of this federal government investigation, our nominating and corporate governance committee is directing our review of and response to the matter. Patent Infringement Lawsuits In March 2022, we filed separate lawsuits in the U.S. District Court for the District of Delaware against (1) Pfizer, Inc. and its subsidiary Pharmacia & Upjohn Co. LLC, collectively referred to as Pfizer, and (2) Moderna, Inc., and its subsidiaries ModernaTX, Inc., and Moderna US, Inc., collectively referred to as Moderna. The lawsuits seek damages for infringement of U.S. Patent No. 11,246,933, or ‘933 Patent, in Pfizer’s and Moderna’s manufacture and sale of their messenger RNA, or mRNA, COVID-19 vaccines. The patent relates to the Company’s biodegradable cationic lipids that are foundational to the success of the mRNA COVID-19 vaccines. We are seeking judgment that each of Pfizer and Moderna is infringing the ‘933 Patent, as well as damages adequate to compensate for the infringement, but in no event less than a reasonable royalty for the unlicensed uses made of our patented lipids by Pfizer and Moderna, together with interest and costs as may be awarded by the court. As stated in the filed complaints, we are not seeking injunctive relief in these lawsuits. On May 23, 2022, Moderna filed a partial motion to dismiss, asserting an affirmative defense under Section 1498(a). We responded on May 27, 2022, opposing their motion arguing Moderna had significant non-government sales and the government contract ended in April 2022. Moderna responded on June 13, 2022, requesting a partial motion to dismiss those claims for sales under 1498(a). This motion is fully briefed and pending before the court. On May 27, 2022, Pfizer filed an answer to our complaint, denying the allegations, and asserting invalidity and non-infringement defenses. In addition, Pfizer added BioNTech SE to the suit and added counter-claims seeking a declaratory judgment that our patent is invalid and a second claim alleging that our patent is invalid due to patent misuse. We believe their defenses and counter-claims have no merit and responded on June 10, 2022, with substantive arguments as to the validity of our claims and the lack of merit of their patent misuse claim. On July 12, 2022, we filed a new lawsuit against each of Pfizer and Moderna seeking damages for infringing our newly granted U.S. Patent No. 11,382,979 in Pfizer’s and Moderna’s manufacture and sale of their mRNA COVID-19 vaccines. The parties agreed to combine the two patents in one lawsuit, separately against each of Moderna and Pfizer/BioNTech. The court has set a trial date of November 12, 2024 for Alnylam v. Moderna and November 18, 2024 for Alnylam v. Pfizer/BioNTech. Indemnifications In connection with license agreements we may enter with companies to obtain rights to intellectual property, we may be required to indemnify such companies for certain damages arising in connection with the intellectual property rights licensed under the agreements. Under such agreements, we may be responsible for paying the costs of any litigation relating to the license agreements or the underlying intellectual property rights, including the costs associated with certain litigation regarding the licensed intellectual property. We are also a party to a number of agreements entered into in the ordinary course of business, which contain typical provisions that obligate us to indemnify the other parties to such agreements upon the occurrence of certain events, including litigation or other legal proceedings. In addition, we have agreed to indemnify our officers and directors for expenses, judgments, fines, penalties, excise taxes, and settlement amounts paid in connection with any threatened, pending or completed litigation proceedings, including, for example, the current government investigation, in which an officer or director was, is or will be involved as a party, on account of such person’s status as an officer or director, or by reason of any action taken by the officer or director while acting in such capacity, subject to certain limitations. These indemnification costs are charged to selling, general and administrative expense. Our maximum potential future liability under any such indemnification provisions is uncertain. We have determined that the estimated aggregate fair value of our potential liabilities under all such indemnification provisions is minimal and had not recorded any liability related to such indemnification provisions as of December 31, 2022 or 2021. |
LEASES
LEASES | 12 Months Ended |
Dec. 31, 2022 | |
Leases [Abstract] | |
LEASES | LEASES Overview of Significant Leases We lease three facilities for office and laboratory space in Cambridge, Massachusetts that represent substantially all of our significant lease obligations. An overview of these significant leases are as follows: 675 West Kendall Street We lease office and laboratory space located at 675 West Kendall Street, Cambridge, Massachusetts for our corporate headquarters from BMR-675 West Kendall Street, LLC, or BMR, under a non-cancelable real property lease. The lease commenced on May 1, 2018 and monthly rent payments became due commencing on February 1, 2019 upon substantial completion of the building improvements, and continue for 15 years, with options to renew for two five-year terms each. Exercise of these options was not determined to be reasonably certain and thus was not included in the operating lease liability on the consolidated balance sheet as of December 31, 2022. 300 Third Street We lease office and laboratory space located at 300 Third Street, Cambridge, Massachusetts under a non-cancelable real property lease agreement by and between us and ARE-MA Region No. 28, LLC, or ARE-MA, dated as of September 26, 2003, as amended. The term of the lease expires on January 31, 2034 with options to renew for two five-year terms each. Exercise of these options was not determined to be reasonably certain and thus was not included in the operating lease liability on the consolidated balance sheet as of December 31, 2022. 101 Main Street We lease office space on several floors at 101 Main Street, Cambridge, Massachusetts under non-cancelable real property lease agreements by and between us and RREEF America REIT II CORP. PPP, or RREEF, entered into in March 2015 and May 2015, as amended in September 2020, that will expire in March 2024 and June 2026, respectively, each with an option to renew for one five-year term. Exercise of these options was not determined to be reasonably certain and thus was not included in the operating lease liability on the consolidated balance sheet as of December 31, 2022. Other Lease Disclosures Our facility leases described above generally contain customary provisions allowing the landlords to terminate the leases if we fail to remedy a breach of any of our obligations under any such lease within specified time periods, or upon our bankruptcy or insolvency. The leases do not include any restrictions or covenants that had to be accounted for under the lease guidance. Total rent expense, including operating expenses, under all of our real property leases was $58.6 million, $59.5 million and $50.7 million for the years ended December 31, 2022, 2021 and 2020, respectively. The following table summarizes our costs included in operating expenses related to right of use lease assets we have entered into through December 31, 2022: (In thousands) Year Ended December 31, 2022 Year Ended December 31, 2021 Operating lease cost $ 45,789 $ 45,359 Variable lease cost 17,614 18,271 Total $ 63,403 $ 63,630 Short-term lease costs were not material for the years ended December 31, 2022 and 2021. Net cash paid for the amounts included in the measurement of the operating lease liability in our consolidated balance sheet and included in change in operating lease liability within operating activities in our consolidated statement of cash flow was $43.1 million and $41.9 million for the years ended December 31, 2022 and 2021, respectively. The weighted-average remaining lease term and weighted-average discount rate for all leases as of December 31, 2022 was 10 years and 8%, respectively, and as of December 31, 2021 was 11 years and 8%, respectively. Future lease payments for non-cancellable operating leases and a reconciliation to the carrying amount of the operating lease liability presented in the consolidated balance sheet as of December 31, 2022 were as follows, in thousands: Year Ending December 31 2023 $ 43,028 2024 46,486 2025 42,867 2026 40,376 2027 38,753 2028 and thereafter 248,622 Total undiscounted lease liability 460,132 Less imputed interest (156,826) Total discounted lease liability $ 303,306 Current operating lease liability $ 41,967 Non-current operating lease liability 261,339 Total $ 303,306 |
INCOME TAXES
INCOME TAXES | 12 Months Ended |
Dec. 31, 2022 | |
Income Tax Disclosure [Abstract] | |
INCOME TAXES | INCOME TAXES The domestic and foreign components of loss before income taxes are as follows: (In thousands) 2022 2021 2020 Domestic $ (1,148,604) $ (794,729) $ (682,859) Foreign 21,611 (57,415) (172,741) Loss before income taxes $ (1,126,993) $ (852,144) $ (855,600) The provision for income taxes consisted of the following: Year Ended December 31, (In thousands) 2022 2021 2020 Current provision: Domestic $ — $ 293 $ 61 Foreign 5,596 3,154 5,837 Total current provision 5,596 3,447 5,898 Deferred benefit: Domestic — — 393 Foreign (1,433) (2,767) (3,610) Total deferred benefit (1,433) (2,767) (3,217) Total provision for income taxes $ 4,163 $ 680 $ 2,681 During the year ended December 31, 2022, we recorded a net provision for income taxes of $4.2 million. This is primarily comprised of $5.6 million of foreign current provision offset by $1.4 million of foreign deferred provision. Deferred income taxes reflect the tax effects of temporary differences between the carrying amounts of assets and liabilities for financial reporting and income tax purposes. We establish a valuation allowance when uncertainty exists as to whether all or a portion of the net deferred tax assets will be realized. Components of the net deferred tax asset are as follows: As of December 31, (In thousands) 2022 2021 Deferred tax assets: Net operating loss carryforwards $ 803,251 $ 745,985 Research and development and other credit carryforwards 381,032 342,431 Sale of future royalties 326,183 302,217 Lease liability 67,242 71,859 Deferred revenue 59,437 76,612 Deferred compensation 52,989 59,349 Intangible assets 264,564 279,082 Capitalized research and development expenditures 206,727 23,039 Other 133,376 48,242 Total deferred tax assets 2,294,801 1,948,816 Deferred tax liabilities: Property, plant and equipment, net (12,786) (13,170) Unrealized gain on marketable securities (5,728) (16,693) Right of use assets (46,819) (50,562) Deferred revenue tax accounting method change (24,995) (50,380) Deferred tax asset valuation allowance (2,193,633) (1,808,992) Net deferred tax asset $ 10,840 $ 9,019 Our effective income tax rate differs from the statutory federal income tax rate, as follows: Year Ended December 31, (In thousands) 2022 2021 2020 At U.S. federal statutory rate 21.0 % 21.0 % 21.0 % State taxes, net of federal effect 6.0 5.2 4.5 Stock-based compensation 4.4 4.6 2.2 Tax credits 2.7 4.5 3.3 Other permanent items (1.5) (1.0) (1.5) Foreign rate differential (0.5) (1.7) (3.5) Internal reorganization of certain intellectual property rights — 20.1 12.3 Other (0.8) (0.1) (2.7) Revaluation of deferred due to rate change (0.4) 1.1 — Valuation allowance (31.3) (53.8) (35.9) Effective income tax rate (0.4) % (0.1) % (0.3) % We have evaluated the positive and negative evidence bearing upon the realizability of our deferred tax assets. We have concluded, in accordance with the applicable accounting standards, that it is more likely than not that we may not realize the benefit of all of our deferred tax assets, with the exception of the deferred assets related to certain foreign subsidiaries. Accordingly, we have recorded a valuation allowance against the deferred tax assets that management believes will not be realized. We re-evaluate the positive and negative evidence on a quarterly basis. The valuation allowance increased by $384.6 million, $459.3 million and $303.7 million for the years ended December 31, 2022, 2021 and 2020, respectively. The increase in our valuation allowance is primarily due to additional net operating losses for the years ended December 31, 2022 and 2020 and primarily due to the liability related to the sale of future royalties for the year ended December 31, 2021. On December 22, 2017, the Tax Cuts and Jobs Act, or TCJA, was signed into law. Under the TCJA provisions, effective with tax years beginning on or after January 1, 2022, taxpayers can no longer immediately expense qualified research and development expenditures, including all direct, indirect, overhead and software development costs. Taxpayers are now required to capitalize and amortize these costs over five years for research conducted within the United States or 15 years for research conducted abroad. As a result, the Company capitalized $870.6 million of research and development expenses for the year ended December 31, 2022. As of December 31, 2022, we had federal and state net operating loss carryforwards, or NOLs, of $2.9 billion and $3.0 billion, respectively, to reduce future taxable income. Federal NOLs of $1.1 billion, generated before 2018, will begin expiring in varying amounts through 2037 unless utilized. The remaining federal NOLs of $1.8 billion, generated after 2017, will be carried forward indefinitely and could be used to offset up to 100% of taxable income of each future tax year for tax years before January 1, 2021 and up to 80% of taxable income in all other future tax years. State NOLs will begin expiring in varying amounts through 2042 unless utilized. As of December 31, 2022, we had federal and state research and development, including Orphan Drug, and state investment tax credit carryforwards of $343.0 million and $62.3 million, respectively, available to reduce future tax liabilities that expire at various dates through 2042. We have a valuation allowance against the net operating loss and tax credit carryforwards as it is unlikely that we will realize these assets. Ownership changes, as defined in the Internal Revenue Code and similar state provisions, including those resulting from the issuance of common stock in connection with our public offerings, may limit the amount of federal and state net operating loss and tax credit carryforwards that can be utilized to offset future taxable income or tax liability. The amount of the limitation is determined in accordance with Section 382 of the Internal Revenue Code and similar state provisions. We have performed an analysis of ownership changes through December 31, 2022. Based on this analysis, we do not believe that any of our federal and state tax attributes will expire unutilized due to Section 382 limitations. As of December 31, 2022, we had foreign NOLs of $153.3 million to reduce future taxable income which will begin expiring in varying amounts through 2029 unless utilized. We have a valuation allowance against the foreign NOLs as it is unlikely that we will realize these assets. We apply the accounting guidance in ASC 740 related to accounting for uncertainty in income taxes. Our reserves related to income taxes are based on a determination of whether, and how much of, a tax benefit taken by us in our tax filings or positions is more likely than not to be realized and ultimately sustained upon challenge by a taxing authority based upon its technical merits and subject to certain recognition and measurement criteria. We recognize potential interest and penalties related to unrecognized tax benefits in our provision for income taxes. Our reserve related to income taxes, including potential interest and penalties, was not material as of December 31, 2022 and 2021. Our uncertain income tax positions do not impact our effective tax rate due to our full valuation allowance in the U.S. As of December 31, 2022, the unremitted earnings of our foreign subsidiaries are not material. We have not provided for U.S. income taxes or foreign withholding taxes on these earnings as it is our current intention to permanently reinvest these earnings outside the U.S. The tax liability on these earnings is also not material. Events that could trigger a tax liability include, but are not limited to, distributions, reorganizations or restructurings and/or tax law changes. The tax years 2019 through 2022 remain open to examination by major taxing jurisdictions, which are primarily in the U.S., although net operating loss and tax credit carryforwards generated prior to 2019 may still be adjusted upon examination by the Internal Revenue Service or state tax authorities if they have or will be used in a future period. |
EMPLOYEE BENEFIT PLANS
EMPLOYEE BENEFIT PLANS | 12 Months Ended |
Dec. 31, 2022 | |
Retirement Benefits [Abstract] | |
EMPLOYEE BENEFIT PLANS | EMPLOYEE BENEFIT PLANS We maintain a retirement saving plan under Section 401(k) of the Internal Revenue Code, in which eligible U.S. employees may defer compensation for income tax purposes. Contributions made by employees are limited to the maximum allowable for U.S. federal income tax purposes. The plan allows for a discretionary match in an amount up to 100% of each participant’s first 2% of compensation contributed plus 50% of each participant’s next 4% of compensation contributed. The expense related to our 401(k) Savings Plan primarily consists of our matching contributions. Furthermore, we maintain defined benefit plans for employees in certain countries outside the U.S., including retirement benefit plans required by applicable local law. The benefit obligation corresponds to the projected benefit obligations of which the discounted net present value is calculated based on years of employment, expected salary increases and pension adjustments. For the years ended December 31, 2022, 2021 and 2020 contributions and net periodic benefit costs to such plans generated a total expense of $16.5 million, $13.1 million and $12.0 million, respectively. |
SUMMARY OF SIGNIFICANT ACCOUN_2
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies) | 12 Months Ended |
Dec. 31, 2022 | |
Accounting Policies [Abstract] | |
Basis of Presentation and Principles of Consolidation | Basis of Presentation and Principles of Consolidation The accompanying consolidated financial statements reflect the operations of Alnylam and our wholly-owned subsidiaries. All intercompany accounts and transactions have been eliminated. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with accounting principles generally accepted in the United States of America, or GAAP, requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the consolidated financial statements and the reported amounts of revenues and expenses during the reporting period. In our consolidated financial statements, we use estimates and assumptions related to our inventory valuation and related reserves, liability related to the sale of future royalties, development derivative liability, income taxes, revenue recognition, research and development expenses, and stock-based compensation. We base our estimates on historical experience and on various other assumptions that we believe to be reasonable. Actual results could differ from those estimates. |
Liquidity | Liquidity Based on our current operating plan, we believe that our cash, cash equivalents and marketable securities as of December 31, 2022, together with the cash we expect to generate from product sales and under our current alliances, will be sufficient to enable us to advance our Alnylam P 5 x25 strategy for at least the next 12 months from the filing of this Annual Report on Form 10-K. |
Concentrations of Credit Risk and Significant Customers | Concentrations of Credit Risk and Significant Customers Financial instruments that potentially expose us to concentrations of credit risk primarily consist of cash, cash equivalents and marketable securities. As of December 31, 2022 and 2021, substantially all of our cash, cash equivalents and marketable securities were invested in money market funds, certificates of deposit, commercial paper, corporate notes, U.S. government-sponsored enterprise securities and U.S. treasury securities through highly rated financial institutions. Corporate notes may also include foreign bonds denominated in U.S. dollars. Investments are restricted, in accordance with our investment policy, to a concentration limit per issuer. |
Fair Value Measurements | Fair Value Measurements The fair value is the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date. In general, fair values determined by Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities. Fair values determined by Level 2 inputs utilize data points that are observable, such as quoted prices (adjusted), interest rates and yield curves. Fair values determined by Level 3 inputs utilize unobservable data points for the asset or liability, and include situations where there is little, if any, market activity for the asset or liability. The fair value hierarchy level is determined by the lowest level of significant input. |
Investments in Marketable Securities and Cash Equivalents | Investments in Marketable Securities and Cash Equivalents We invest our excess cash balances in marketable debt securities and classify our investments as either held-to-maturity or available-for-sale based on facts and circumstances present at the time we purchased the securities. At each balance sheet date presented, we classified all of our investments in debt securities as available-for-sale and as current assets as they represent the investment of funds available for current operations. We report available-for-sale debt securities at fair value at each balance sheet date and include any unrealized holding gains and losses (the adjustment to fair value) in accumulated other comprehensive (loss) income, a component of stockholders’ (deficit) equity. Realized gains and losses are determined using the specific identification method and are included in other income (expense). If any adjustment to fair value reflects a decline in the value of the marketable debt securities, we consider all available evidence to evaluate if an impairment loss exists, and if so, mark the investment to market through a charge to our consolidated statements of operations and comprehensive loss. We did not record any impairment charges related to our marketable debt securities during the years ended December 31, 2022, 2021 or 2020. Our marketable debt securities are classified as cash equivalents if the original maturity, from the date of purchase, is 90 days or less, and as marketable debt securities if the original maturity, from the date of purchase, is in excess of 90 days. Our cash equivalents are generally composed of commercial paper, corporate notes, U.S. government-sponsored enterprise securities, U.S. treasury securities and money market funds. |
Accounts Receivable | Accounts Receivable We record accounts receivable net of customer allowances for distribution services, prompt payment discounts and chargebacks based on contractual terms. As of December 31, 2022 and 2021, based on our estimation of expected write-offs, we determined an allowance for doubtful accounts was not material. We have standard payment terms that generally require |
Inventory | Inventory Inventory is measured at the lower of cost or estimated net realizable value and classified based on the anticipation of when it will be consumed either within our normal operating cycle (short-term) or beyond (long-term). We use a standard cost basis, which approximates cost determined on a first-in, first-out basis. Inventory costs include all raw materials, direct conversion costs and overhead. Raw and intermediate materials that may be used for either research and development or commercial purposes are classified as inventory until the material is consumed or otherwise allocated for research and development. If the material is used for research and development, it is expensed as research and development once that determination is made. We capitalize inventory costs that are expected to be sold commercially once we determine it is probable that the inventory costs will be recovered through commercial sale based on the review of several factors, including (i) the likelihood that all required regulatory approvals will be received, considering any special filing status, (ii) the expected timing of validation (if not yet completed) of manufacturing processes in the associated facility, (iii) the expected expiration of the inventory, (iv) logistical or commercial constraints that may impede the timely distribution and sale of the product, including transport requirements and reimbursement status, (v) current market factors, including competitive landscape and pricing, (vi) threatened or anticipated litigation challenges, (vii) history of approvals of similar products or formulations, and (viii) FDA (or other appropriate regulatory agencies) correspondence regarding the safety and efficacy of the product. Prior to the capitalization of inventory costs, we record such costs as research and development expenses on our consolidated statements of operations and comprehensive loss. We reduce our inventory to net realizable value for potentially excess, dated or obsolete inventory based on our quarterly assessment of the recoverability of our capitalized inventory. We periodically review inventory levels to identify what may expire prior to expected sale or has a cost basis in excess of its estimated realizable value and write-down such inventories as appropriate. |
Property, Plant and Equipment | Property, Plant and Equipment Property, plant and equipment are stated at cost, net of accumulated depreciation. Depreciation expense is recorded on a straight-line basis over the estimated useful life of the asset. Construction in progress reflects amounts incurred for construction or improvements of property, plant or equipment that have not been placed in service. Costs of construction of certain long-lived assets include capitalized interest, which is amortized over the estimated useful life of the related asset. The cost and accumulated depreciation of assets retired or sold are removed from the respective asset category, and any gain or loss is recognized in our consolidated statements of operations and comprehensive loss. During the years ended December 31, 2022, 2021 and 2020, we recorded $39.1 million, $36.8 million and $30.2 million, respectively, of depreciation expense related to our property, plant and equipment. The estimated useful lives of property, plant and equipment are as follows: Asset Category Useful Life Laboratory equipment 5 Computer equipment and software 3-10 years Furniture and fixtures 5 Leasehold improvements Shorter of asset life or lease term Manufacturing Equipment 7-15 years Buildings 40 years |
Leases | Leases We determine if an arrangement is a lease at contract inception based on the facts and circumstances present in the arrangement. All of our leases are classified as operating leases. W e record operating lease assets and lease liabilities in our consolidated balance sheets. Operating lease assets represent our right to use an underlying asset for the lease term and operating lease liabilities represent our obligation to make lease payments arising from the leasing arrangement. Operating lease assets and operating lease liabilities are recognized at commencement date based on the present value of lease payments over the lease term. As most of our leases do not provide an implicit rate, in determining the operating lease liabilities, we use an estimate of our incremental borrowing rate based on the information available at commencement. Lease expense for lease payments is recognized on a straight-line basis over the lease term. Short-term leases, or leases that have a lease term of 12 |
Clinical Accruals | Clinical Accruals We record accrued liabilities related to products we have received or services that we have incurred, specifically related to ongoing pre-clinical studies and clinical trials, for which service providers have not yet billed us, or when billing terms under these contracts do not coincide with the timing of when the work is performed, as of our period-end. These costs primarily relate to third-party clinical management costs, laboratory and analysis costs, toxicology studies and investigator fees. The assessment of these costs is a subjective process, requiring judgment based on our knowledge of the research and development programs, services performed for the period, experience with related activities and the expected duration of the third-party service contract, where applicable. Upon settlement, these costs may differ materially from the amounts accrued in our consolidated financial statements. Our historical accrual estimates have not been materially different from our actual costs. |
Revenue Recognition | Revenue Recognition We recognize revenue when control of promised goods or services is transferred to a customer at an amount that reflects the consideration to which we expect to be entitled in exchange for those goods or services. To determine revenue recognition, we perform the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price, including variable consideration, if any; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) we satisfy a performance obligation. We only apply the five-step model to contracts when collectability of the consideration to which we are entitled in exchange for the goods or services we transfer to the customer is determined to be probable. At contract inception, once the contract is determined to be within the scope of Accounting Standards Codification Topic 606, Revenue from Contracts with Customers, or ASC 606, we assess whether the goods or services promised within each contract are distinct and, therefore, represent a separate performance obligation. Goods and services that are determined not to be distinct are combined with other promised goods and services until a distinct bundle is identified. We then allocate the transaction price (the amount of consideration we expect to be entitled to from a customer in exchange for the promised goods or services) to each performance obligation and recognize the associated revenue when (or as) each performance obligation is satisfied. Our estimate of the transaction price for each contract includes all variable consideration to which we expect to be entitled. Amounts are recorded as accounts receivable when our right to consideration is unconditional. We do not assess whether a contract has a significant financing component if the expectation at contract inception is that the period between payment by the customer and the transfer of the promised goods or services to the customer will be one year or less. We expense incremental costs of obtaining a contract as and when incurred if the expected amortization period of the asset that we would have recognized is one year or less or the amount is immaterial. As of December 31, 2022 and 2021, we had not capitalized any costs to obtain any of our contracts. Net Product Revenues Our net product revenues are recognized, net of variable consideration related to certain allowances and accruals, at the time the customer obtains control of our product. We use the expected value method, which is the sum of probability-weighted amounts in a range of possible consideration amounts, or the most likely amount method, which is the single most likely amount in a range of possible considerations, to estimate variable consideration related to our product sales. We use the expected value method to estimate variable consideration for certain rebates, chargebacks, product returns, and other incentives and we use the most likely amount method for certain rebates and trade discounts and allowances. We record reserves, based on contractual terms, for components related to product sold during the reporting period, as well as our estimate of product that remains in the distribution channel inventory at the end of the reporting period that we expect will be sold to qualified healthcare providers. On a quarterly basis, we update our estimates and record any needed adjustments in the period we identify the adjustments. The following are the components of variable consideration related to product revenues: Chargebacks : We estimate obligations resulting from contractual commitments with the government and other entities to sell products to qualified healthcare providers at prices lower than the list prices charged to the customer who directly purchases from us. The customer charges us for the difference between what it pays to us for the product and the selling price to the qualified healthcare providers. Rebates : We are subject to discount obligations under government programs, including Medicaid in the U.S. and similar programs in certain other countries, including countries in which we are accruing for estimated rebates because final pricing has not yet been negotiated. We are also subject to potential rebates in connection with our value-based agreements with certain commercial payors. We record reserves for rebates in the same period the related product revenue is recognized, resulting in a reduction of product revenues and a current liability that is included in accrued expenses on our consolidated balance sheet. Our estimate for rebates is based on statutory discount rates, expected utilization or an estimated number of patients on treatment, as applicable. Trade discounts and allowances : We provide customary invoice discounts on product sales to our customers for prompt payment and we pay fees for distribution services, such as fees for certain data that customers provide to us. We estimate our customers will earn these discounts and fees, and deduct these discounts and fees in full from gross product revenues and accounts receivable at the time we recognize the related revenues. Product returns: We offer customers product return rights if products are damaged, defective or expired, with “expired” defined within each customer agreement. We estimate the amount of product that will be returned using a probability-weighted estimate based on our sales history. Other incentives: Other incentives include co-payment assistance we provide to patients with commercial insurance that have coverage and reside in states that allow co-payment assistance. We estimate the average co-payment assistance amounts for our products based on expected customer demographics and record any such amounts within accrued expenses on our consolidated balance sheet. Net Revenues from Collaborations We earn revenue in connection with collaboration agreements which allow our collaboration partners to utilize our technology platforms and develop product candidates. Our collaboration agreements are detailed in Note 4, Net Revenues from Collaborations. For each collaboration partner, we discuss our revenue recognition, including our significant performance obligations under each agreement. At contract inception, we assess whether the collaboration arrangements are within the scope of ASC Topic 808, Collaborative Arrangements, or ASC 808, to determine whether such arrangements involve joint operating activities performed by parties that are both active participants in the activities and exposed to significant risks and rewards dependent on the commercial success of such activities. This assessment is performed based on the responsibilities of all parties in the arrangement. For collaboration arrangements within the scope of ASC 808 that contain multiple elements, we first determine which elements of the arrangement are within the scope of ASC 808 and which elements are within the scope of ASC 606. For elements of collaboration arrangements that are accounted for pursuant to ASC 808, an appropriate recognition method is determined and applied consistently, either by analogy to authoritative accounting literature or by applying a reasonable and rational policy election. For elements of collaboration arrangements that are accounted for pursuant to ASC 606, we identify the performance obligations and allocate the total consideration we expect to receive on a relative standalone selling price basis to each performance obligation. Variable consideration such as performance-based milestones will be included in the total consideration if we expect to receive such consideration and if it is probable that the inclusion of the variable consideration will not result in a significant reversal in the cumulative amount of revenue recognized under the arrangement. Our estimate of the total consideration we expect to receive under each collaboration arrangement is updated for each reporting period, and any adjustments to revenue are recorded on a cumulative catch-up basis. We exclude sales-based royalty and milestone payments from the total consideration we expect to receive until the underlying sales occur because the license to our intellectual property is deemed to be the predominant item to which the royalties or milestones relate as it is the primary driver of value in our collaboration arrangements. Key assumptions to determine the standalone selling price may include forecasted revenues, development timelines, reimbursement rates for personnel costs, discount rates and probabilities of technical and regulatory success. We recognize revenue associated with each performance obligation as the control over the promised goods or services transfer to our collaboration partner which occurs either at a point in time or over time. If control transfers over time, revenue is recognized by using a method of measuring progress that best depicts the transfer of goods or services. We evaluate the measure of progress and related inputs each reporting period and any resulting adjustments to revenue are recorded on a cumulative catch-up basis. Consideration received that does not meet the requirements to satisfy ASC 808 or ASC 606 revenue recognition criteria is recorded as deferred revenue in the accompanying consolidated balance sheets, classified as either short-term (less than 12 months) or long-term (more than 12 months) deferred revenue based on our best estimate of when such revenue will be recognized. |
Cost of Goods Sold | Cost of Goods Sold Cost of goods sold includes the cost of producing and distributing inventories that are related to product revenues during the respective period (including salary-related and stock-based compensation expenses for employees involved with production and distribution, freight and indirect overhead costs), third-party royalties payable on our net product revenues, amortization of intangible assets associated with the sale of our products and costs related to sales of product supply under our collaboration agreements. Cost of goods sold may also include costs related to excess or obsolete inventory adjustment charges, abnormal costs, unabsorbed manufacturing and overhead costs, and manufacturing variances. |
Cost of Collaborations and Royalties | Cost of Collaborations and Royalties Cost of collaborations and royalties includes costs we incur in connection with providing commercial drug supplies, such as GalNAc material, to collaborators, in addition to royalties we owe to third parties on the net sales of licensed products by Novartis. |
Income Taxes | Income Taxes We account for income taxes under the asset and liability method. Under this method, deferred tax assets and liabilities are recognized for the estimated future tax consequences attributable to differences between financial statement carrying amounts of existing assets and liabilities and their respective tax basis. Deferred tax assets and liabilities are measured using enacted rates in effect for the year in which these temporary differences are expected to be recovered or settled. Valuation allowances are provided if, based on the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. Uncertain tax positions, for which management's assessment is that there is a more than 50% probability of sustaining the position upon challenge by a taxing authority based upon its technical merits, are subject to certain recognition and measurement criteria. The nature of the uncertain tax positions is often very complex and subject to change, and the amounts at issue can be substantial. We develop our cumulative probability assessment of the measurement of uncertain tax positions using internal experience, judgment and assistance from professional advisors. We re-evaluate these uncertain tax positions on a quarterly basis based on a number of factors including, but not limited to, changes in facts or circumstances, changes in tax law, and effectively settled issues under audit and new audit activity. Any change in these factors could result in the recognition of a tax benefit or an additional charge to the tax provision. |
Research and Development Expenses | Research and Development Expenses We record research and development expenses as incurred. Included in research and development expenses are wages, stock-based compensation expenses, benefits and other operating costs, facilities, supplies, external services, clinical trial and manufacturing costs, certain costs related to our collaboration arrangements, and overhead directly related to our research and development operations, as well as costs to acquire technology licenses. |
Stock-Based Compensation | Stock-Based Compensation We recognize stock-based compensation expense for grants under our stock incentive plans and employee stock purchase plan. We account for all stock-based awards granted to employees at their fair value and recognize compensation expense over the vesting period of the award. Determining the amount of stock-based compensation to be recorded requires us to develop estimates of fair values of stock options as of the grant date. We calculate the grant date fair values of stock options using the Black-Scholes valuation model, which requires the input of subjective assumptions, including but not limited to expected stock price volatility over the term of the awards and the expected term of stock options. The fair value of restricted stock awards granted to employees is based upon the quoted closing market price per share on the date of grant. We have performance conditions included in certain of our restricted stock awards that are based upon the achievement of pre-specified clinical development, regulatory, commercial and/or financial performance events. As the outcome of each event has inherent risk and uncertainties, and a positive outcome may not be known until the event is achieved, we begin to recognize the value of the performance-based restricted stock awards when we determine the achievement of each performance condition is deemed probable, a determination which requires significant judgment by management. At the probable date, we record a cumulative expense catch-up, with remaining expense amortized over the remaining service period. |
Liability Related to the Sale of Future Royalties | Liability Related to the Sale of Future Royalties We account for the liability related to the sale of future royalties as a debt financing, as we have significant continuing involvement in the generation of the cash flows. Interest on the liability related to the sale of future royalties will be recognized using the effective interest rate method over the life of the related royalty stream. The liability related to the sale of future royalties and the related interest expense are based on our current estimates of future royalties and commercial milestones expected to be paid over the life of the arrangement, which we determine by using third-party forecasts of Leqvio’s global net revenue. We will periodically assess the expected payments and to the extent the amount or timing of our future estimated payments is materially different than our previous estimates, we will account for any |
Development Derivative Liability | Development Derivative Liability Development derivative liability is recorded at fair value based on the probability weighted present value of the estimated cash flows pursuant to contractual terms of the funding agreement. The liability is remeasured quarterly with any change in fair value recorded in other income (expense) on the consolidated statements of operations and comprehensive loss. |
Comprehensive Loss | Comprehensive Loss Comprehensive loss is comprised of net loss and certain changes in stockholders’ (deficit) equity that are excluded from net loss. We include unrealized gains and losses on certain marketable securities in other comprehensive loss, including changes in the value of our marketable debt securities. We include foreign currency translation adjustments in other comprehensive loss if the functional currency is not the U.S. dollar. We include certain changes in the fair value of the plan assets and projected benefit obligation attributed to our defined benefit pension plan in other comprehensive loss. |
Net Loss per Common Share | Net Loss per Common Share We compute basic net loss per common share by dividing net loss by the weighted-average number of common shares outstanding. We compute diluted net loss per common share by dividing net loss by the weighted-average number of common shares and dilutive potential common share equivalents then outstanding during the period. In the diluted net loss per share calculation, net loss would be adjusted for the elimination of interest expense on the Convertible debt. Potential common shares consist of shares issuable upon the exercise of stock options (the proceeds of which are then assumed to have been used to repurchase outstanding shares using the treasury stock method) or upon conversion of the convertible debt outstanding during the period (calculated using the if-converted method assuming the conversion of the convertible debt as of the earliest period reported or at the date of issuance, if later). Because the inclusion of potential common shares would be anti-dilutive for all periods presented, diluted net loss per common share is the same as basic net loss per common share. |
Segment Information | Segment Information We operate in a single reporting segment, the discovery, development and commercialization of RNAi therapeutics. Consistent with our management reporting, results of our operations are reported on a consolidated basis for purposes of segment reporting. As of December 31, 2022 and 2021, substantially all of our consolidated property, plant and equipment, net, was from U.S. operations. For the years ended December 31, 2022, 2021 and 2020, net revenues from collaborations were attributed to the U.S. Please read Note 3 for information regarding our net product sales by geography. |
Recently Adopted Accounting Pronouncements | Recently Adopted Accounting Pronouncements In August 2020, the Financial Accounting Standards Board, or FASB, issued Accounting Standards Update, or ASU, 2020-06, Debt—Debt with Conversion and Other Options (Subtopic 470-20) and Derivatives and Hedging—Contracts in Entity’s Own Equity (Subtopic 815-40): Accounting for Convertible Instruments and Contracts in an Entity’s Own Equity, which simplifies the accounting for certain financial instruments with characteristics of liabilities and equity, including convertible instruments and contracts in an entity’s own equity. Specifically, ASU 2020-06 simplifies accounting for the issuance of convertible instruments by removing certain separation models required under existing guidance. In addition, the standard removes certain settlement conditions that are required for equity contracts to qualify for the derivative scope exception and requires use of the “if-converted” method when calculating the dilutive impact of convertible debt on earnings per share, or EPS. ASU 2020-06 is effective for financial statements issued for fiscal years beginning after December 15, 2021, and interim periods within those fiscal years, with early adoption permitted. We adopted the new standard on January 1, 2022 and there was no impact on the date of adoption. During the third quarter of 2022, we issued convertible notes as described in Note 10, Convertible Debt. |
SUMMARY OF SIGNIFICANT ACCOUN_3
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Accounting Policies [Abstract] | |
Concentrations of Credit Risk and Significant Customers | The following table summarizes customers that represent 10% or greater of our consolidated total gross revenues: Year Ended December 31, 2022 2021 2020 Distributor A 33 % 27 % 31 % Regeneron Pharmaceuticals * 11% 12% __________________________________________ * Represents less than 10% The following table summarizes customers with amounts due that represent 10% or greater of our consolidated gross accounts receivable balance: As of December 31, 2022 2021 Distributor A 32 % 14 % Distributor B 10 % 10 % Novartis AG * 20 % Regeneron Pharmaceuticals * 12 % __________________________________________ * Represents less than 10% |
Estimated Useful Lives of Property, Plant and Equipment | The estimated useful lives of property, plant and equipment are as follows: Asset Category Useful Life Laboratory equipment 5 Computer equipment and software 3-10 years Furniture and fixtures 5 Leasehold improvements Shorter of asset life or lease term Manufacturing Equipment 7-15 years Buildings 40 years |
Common Shares Excluded from the Calculation of Net Loss Per Common Share | The following table sets forth the potential common shares (prior to consideration of the treasury stock or if-converted methods) excluded from the calculation of net loss per common share because their inclusion would be anti-dilutive: As of December 31, (In thousands) 2022 2021 2020 Options to purchase common stock, inclusive of performance-based stock options 8,424 10,015 11,692 Unvested restricted common stock, inclusive of performance-based restricted common stock 1,487 1,210 1,160 Convertible debt 3,616 — — Total 13,527 11,225 12,852 |
NET PRODUCT REVENUES (Tables)
NET PRODUCT REVENUES (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Revenue from Contract with Customer [Abstract] | |
Net Product Revenues by Geography | Net product revenues consist of the following: Year Ended December 31, (In thousands) 2022 2021 2020 ONPATTRO United States $ 246,748 $ 213,210 $ 151,574 Europe 224,063 190,435 107,755 Rest of World 86,797 71,092 46,752 Total 557,608 474,737 306,081 AMVUTTRA United States 82,521 — — Europe 4,214 — — Rest of World 7,060 — — Total 93,795 — — GIVLAARI United States 115,659 92,747 42,797 Europe 48,670 30,895 12,000 Rest of World 8,815 4,173 309 Total 173,144 127,815 55,106 OXLUMO United States 27,698 18,876 — Europe 37,915 38,949 333 Rest of World 4,169 1,761 — Total 69,782 59,586 333 Total net product revenues $ 894,329 $ 662,138 $ 361,520 |
Summary of Balances and Activity in Each Product Revenue Allowance and Reserve Category | The following table summarizes balances and activity in each product revenue allowance and reserve category: As of December 31, 2022 (In thousands) Chargebacks and Rebates Trade Discounts and Allowances Returns Reserve and Other Incentives Total Beginning balance $ 120,682 $ 522 $ 10,112 $ 131,316 Provision related to current period sales 245,236 13,085 15,249 273,570 Credit or payments made during the period for current year sales (108,185) (10,310) (9,850) (128,345) Credit or payments made during the period for prior year sales (65,961) (847) (735) (67,543) Total $ 191,772 $ 2,450 $ 14,776 $ 208,998 As of December 31, 2021 (In thousands) Chargebacks and Rebates Trade Discounts and Allowances Returns Reserve and Other Incentives Total Beginning balance $ 90,705 $ 639 $ 3,763 $ 95,107 Provision related to current period sales 167,691 8,064 12,223 187,978 Credit or payments made during the period for current year sales (75,073) (7,665) (5,190) (87,928) Credit or payments made during the period for prior period sales (62,641) (516) (684) (63,841) Total $ 120,682 $ 522 $ 10,112 $ 131,316 |
NET REVENUES FROM COLLABORATI_2
NET REVENUES FROM COLLABORATIONS (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Revenue from Collaborators | Net revenues from collaborations consist of the following: Year Ended December 31, (In thousands) 2022 2021 2020 Regeneron Pharmaceuticals $ 87,844 $ 113,226 $ 74,072 Novartis AG 43,159 49,120 22,208 Vir Biotechnology 1,755 16,897 31,396 Other 2,154 1,710 3,657 Total $ 134,912 $ 180,953 $ 131,333 |
Balance and Change in Contract Liabilities Related to Collaboration Agreements | The following table presents the balance of our receivables and contract liabilities related to our collaboration agreements: As of December 31, (In thousands) 2022 2021 Receivables included in "Accounts receivable, net" $ 32,342 $ 73,266 Contract liabilities included in "Deferred revenue" $ 235,528 $ 88,627 |
Schedule of Research and Development Expenses Incurred by Type that are Directly Attributable to Collaboration Agreements | The following table provides research and development expenses incurred by type, for which we recognize net revenue, that are directly attributable to our collaboration agreements, by collaboration partner: Year Ended December 31, 2022 2021 2020 (In thousands) Clinical Trial and Manufacturing External Services Other Clinical Trial and Manufacturing External Services Other Clinical Trial and Manufacturing External Services Other Regeneron $ 12,926 $ 2,141 $ 27,935 $ 24,989 $ 840 $ 47,582 $ 13,302 $ 171 $ 44,360 Other 156 679 337 10,311 775 4,489 20,113 765 14,422 Total $ 13,082 $ 2,820 $ 28,272 $ 35,300 $ 1,615 $ 52,071 $ 33,415 $ 936 $ 58,782 |
Schedule of Allocated Transaction Price | We allocated the initial transaction price to each unit of account based on the applicable accounting guidance as follows, in thousands: Performance Obligations Standalone Selling Price Transaction Price Allocated Accounting Guidance Research Services Obligation $ 130,700 $ 183,100 ASC 606 C5 License Obligation $ 97,600 92,500 ASC 606 C5 Co-Co Obligation $ 364,600 246,000 ASC 606 $ 521,600 Transaction Price Allocated Deferred Revenue Performance Obligations As of December 31, 2022 As of December 31, 2022 As of December 31, 2021 Accounting Guidance Research Services Obligation $ 215,680 $ 26,200 $ 42,300 ASC 606 C5 License Obligation 97,200 7,000 26,900 ASC 606 C5 Co-Co Obligation 246,000 193,600 212,500 ASC 606 Total $ 558,880 $ 226,800 $ 281,700 |
Schedule of Revenue Recognized Based on Accounting Guidance | Revenue Recognized During Performance Obligations Year Ended December 31, 2022 Year Ended December 31, 2021 Year Ended December 31, 2020 Accounting Guidance Research Services Obligation $ 28,600 $ 37,600 $ 44,800 ASC 606 C5 License Obligation 32,500 44,600 7,100 ASC 606 C5 Co-Co Obligation 20,080 18,900 11,700 ASC 606 Total $ 81,180 $ 101,100 $ 63,600 |
LIABILITY RELATED TO THE SALE_2
LIABILITY RELATED TO THE SALE OF FUTURE ROYALTIES (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Related Party Transactions [Abstract] | |
Schedule of Royalty Liability | The following table shows the activity with respect to the liability related to the sale of future royalties, in thousands: Carrying value as of December 31, 2020 $ 1,071,541 Interest expense recognized 116,940 Payments (378) Carrying value as of December 31, 2021 1,188,103 Interest expense recognized 107,601 Payments (3,400) Carrying value as of December 31, 2022 $ 1,292,304 |
FAIR VALUE MEASUREMENTS (Tables
FAIR VALUE MEASUREMENTS (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Fair Value Disclosures [Abstract] | |
Fair Value of Assets Measured on a Recurring Basis | The following tables present information about our financial assets and liabilities that are measured at fair value on a recurring basis and indicate the fair value hierarchy of the valuation techniques we utilized to determine such fair value: (In thousands) As of December 31, Quoted Prices in Active Markets Significant Observable Inputs Significant Unobservable Inputs Financial assets Cash equivalents: Money market funds $ 270,394 $ 270,394 $ — $ — U.S. treasury securities 44,817 — 44,817 — U.S. government-sponsored enterprise securities 41,763 — 41,763 — Commercial paper 22,350 — 22,350 — Certificates of deposit 3,289 — 3,289 — Corporate notes 1,024 — 1,024 — Marketable debt securities: U.S. treasury securities 820,913 — 820,913 — U.S. government-sponsored enterprise securities 230,770 — 230,770 — Corporate notes 208,284 — 208,284 — Commercial paper 36,793 — 36,793 — Certificates of deposit 1,130 — 1,130 — Marketable equity securities 28,122 28,122 — — Restricted cash (money market funds) 1,197 1,197 — — Total financial assets $ 1,710,846 $ 299,713 $ 1,411,133 $ — Financial liabilities Development derivative liability $ 209,277 $ — $ — $ 209,277 (In thousands) As of December 31, Quoted Prices in Active Markets Significant Observable Inputs Significant Unobservable Inputs Financial assets Cash equivalents: Money market funds $ 255,869 $ 255,869 $ — $ — U.S. treasury securities 54,998 — 54,998 — Marketable debt securities: U.S. treasury securities 1,030,578 — 1,030,578 — Corporate notes 253,239 — 253,239 — U.S. government-sponsored enterprise securities 177,741 — 177,741 — Commercial paper 78,543 — 78,543 — Certificates of deposit 7,501 — 7,501 — Municipal securities 1,015 — 1,015 — Marketable equity securities 66,972 66,972 — — Restricted cash (money market funds) 1,195 1,195 — — Total financial assets $ 1,927,651 $ 324,036 $ 1,603,615 $ — Financial liabilities Development derivative liability $ 83,618 $ — $ — $ 83,618 |
MARKETABLE DEBT SECURITIES (Tab
MARKETABLE DEBT SECURITIES (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Investments, Debt and Equity Securities [Abstract] | |
Summary of Company's Marketable Debt Securities | The following tables summarize our marketable debt securities: As of December 31, 2022 (In thousands) Amortized Gross Gross Fair Value U.S. treasury securities $ 870,033 $ 79 $ (4,382) $ 865,730 U.S. government-sponsored enterprise securities 275,610 24 (3,101) 272,533 Corporate notes 211,398 16 (2,106) 209,308 Commercial paper 59,143 — — 59,143 Certificates of deposit 4,419 — — 4,419 Total $ 1,420,603 $ 119 $ (9,589) $ 1,411,133 As of December 31, 2021 (In thousands) Amortized Gross Gross Fair Value U.S. treasury securities $ 1,086,232 $ 6 $ (662) $ 1,085,576 Corporate notes 253,926 1 (688) 253,239 U.S. government-sponsored enterprise securities 178,027 2 (288) 177,741 Commercial paper 78,543 — — 78,543 Certificates of deposit 7,501 — — 7,501 Municipal securities 1,016 — (1) 1,015 Total $ 1,605,245 $ 9 $ (1,639) $ 1,603,615 |
Summary of Fair Value of Marketable Debt Securities | The fair values of our marketable debt securities by classification in the consolidated balance sheets were as follows: (In thousands) December 31, 2022 December 31, 2021 Cash and cash equivalents $ 113,243 $ 54,998 Marketable debt securities 1,297,890 1,548,617 Total $ 1,411,133 $ 1,603,615 |
OTHER BALANCE SHEET DETAILS (Ta
OTHER BALANCE SHEET DETAILS (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Balance Sheet Related Disclosures [Abstract] | |
Schedule of Inventory | The components of inventory are summarized as follows: As of December 31, (In thousands) 2022 2021 Raw materials $ 22,315 $ 14,754 Work in process 113,783 100,942 Finished goods 25,606 7,005 Total inventory $ 161,704 $ 122,701 |
Property, Plant and Equipment, Net | Property, plant and equipment, net consist of the following: As of December 31, (In thousands) 2022 2021 Buildings $ 269,322 $ 262,637 Leasehold improvements 230,848 152,045 Construction in progress 14,595 80,753 Laboratory equipment 82,586 61,351 Manufacturing equipment 45,311 43,739 Computer equipment and software 33,370 21,885 Furniture and fixtures 11,832 10,883 Land 9,080 9,080 696,944 642,373 Less: accumulated depreciation (173,450) (140,415) Total $ 523,494 $ 501,958 |
Accrued Expenses | Accrued expenses consist of the following: As of December 31, (In thousands) 2022 2021 Product rebates and discounts $ 208,998 $ 131,279 Compensation and related 130,690 93,583 Pre-clinical, clinical trial and manufacturing 94,702 83,534 Licensing and collaboration agreements 31,680 22,843 Consulting and professional services 19,848 17,784 Other 59,542 46,151 Total $ 545,460 $ 395,174 |
Schedule of Reconciliation of Cash, Cash Equivalents and Restricted Cash | The following table provides a reconciliation of cash, cash equivalents and restricted cash reported within our consolidated balance sheets that sum to the total of these amounts shown in the consolidated statements of cash flows: As of December 31, (In thousands) 2022 2021 2020 Cash and cash equivalents $ 866,394 $ 819,975 $ 496,580 Total restricted cash included in other assets 2,162 2,178 2,466 Total cash, cash equivalents, and restricted cash shown in the consolidated statements of cash flows $ 868,556 $ 822,153 $ 499,046 |
Summary of Changes in Accumulated Other Comprehensive Income (Loss) | The following table summarizes the changes in accumulated other comprehensive (loss) income, by component: (In thousands) Loss on Investment in Joint Venture Defined Benefit Pension Unrealized Gains (Losses) from Debt Securities Foreign Currency Translation Total Accumulated Other Comprehensive (Loss) Income Balance as of December 31, 2020 $ (32,792) $ (3,754) $ 348 $ (7,424) $ (43,622) Other comprehensive loss before reclassifications — 899 — 11,398 12,297 Amounts reclassified from other comprehensive income — 44 (1,978) — (1,934) Net other comprehensive income (loss) — 943 (1,978) 11,398 10,363 Balance as of December 31, 2021 (32,792) (2,811) (1,630) 3,974 (33,259) Other comprehensive income before reclassifications — — 11 (5,274) (5,263) Amounts reclassified from other comprehensive income — 1,719 (7,851) — (6,132) Net other comprehensive income (loss) — 1,719 (7,840) (5,274) (11,395) Balance as of December 31, 2022 $ (32,792) $ (1,092) $ (9,470) $ (1,300) $ (44,654) |
DEVELOPMENT DERIVATIVE LIABIL_2
DEVELOPMENT DERIVATIVE LIABILITY (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Derivative Instruments and Hedging Activities Disclosure [Abstract] | |
Development Derivative Liability Activity | The following table presents the activity with respect to the development derivative liability, in thousands: Carrying value as of December 31, 2020 $ 25,585 Amount received under the Funding Agreement 19,600 Loss recorded from remeasurement 38,433 Carrying value as of December 31, 2021 83,618 Amount received under the Funding Agreement 31,000 Loss recorded from remeasurement 94,659 Carrying value as of December 31, 2022 $ 209,277 |
STOCK-BASED COMPENSATION (Table
STOCK-BASED COMPENSATION (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Share-Based Payment Arrangement [Abstract] | |
Stock-based Compensation Expenses Included in Operating Costs and Expenses | The following table summarizes stock-based compensation expenses included in operating costs and expenses: Year Ended December 31, (In thousands) 2022 2021 2020 Research and development $ 92,161 $ 68,415 $ 60,464 Selling, general and administrative 138,488 97,302 79,409 Total $ 230,649 $ 165,717 $ 139,873 |
Summary of Stock-Based Compensation Expense | The following table summarizes stock-based compensation expense: Year Ended December 31, (In thousands) 2022 2021 2020 Stock-based compensation expense by type of award: Time-based stock options $ 114,901 $ 118,635 $ 112,971 Time-based restricted stock units 12,791 4,231 6,909 Performance-based restricted stock units 102,925 39,943 11,162 Other equity programs 4,057 6,235 9,402 Less: Stock-based compensation expense capitalized to inventory (4,025) (3,327) (571) Total $ 230,649 $ 165,717 $ 139,873 |
Summary of Unrecognized Stock-Based Compensation Expense, Net of Estimated Forfeitures | The following table summarizes our unrecognized stock-based compensation expense, net of estimated forfeitures, by type of awards, and the weighted-average period over which that expense is expected to be recognized: As of December 31, 2022 Unrecognized Expense, Net of Estimated Forfeitures (in thousands) Weighted-average Recognition Period (in years) Type of award: Time-based stock options $ 187,534 2.47 Time-based restricted stock units $ 30,676 2.09 Performance-based restricted stock units * $ 7,695 0.58 Other equity programs $ 4,225 0.45 __________________________________________ |
Valuation Assumptions for Stock Options | The following table summarizes the Black-Scholes valuation assumption inputs for employee stock options granted: Year Ended December 31, 2022 2021 2020 Risk-free interest rate 1.3 - 4.2% 0.4 - 1.4% 0.3 - 1.7% Expected dividend yield — — — Expected option life 5.1 - 7.0 years 5.4 - 6.8 years 5.4 - 7.2 years Expected volatility 50 - 60% 58 - 63% 61 - 63% |
Stock Option Activity | The following table summarizes the activity of our stock option plans, excluding performance-based stock options: Number of Options (in thousands) Weighted-average Exercise Price (per share) Weighted-average Remaining Contractual Term (in years) Aggregate Intrinsic Value (in thousands) Outstanding as of December 31, 2021 8,840 $ 103.87 Granted 1,874 $ 153.18 Exercised (2,490) $ 84.48 Cancelled (356) $ 129.47 Outstanding as of December 31, 2022 7,868 $ 120.59 6.53 $ 921,016 Exercisable as of December 31, 2022 4,450 $ 102.17 5.22 $ 602,969 Vested or expected to vest as of December 31, 2022 7,537 $ 119.24 6.44 $ 892,463 The following table summarizes the activity of our performance-based stock options granted under our equity plans: Number of Options (in thousands) Weighted-average Exercise Price (per share) Weighted-average Remaining Contractual Term (in years) Aggregate Intrinsic Value (in thousands) Outstanding as of December 31, 2021 1,175 $ 92.31 Granted — $ — Exercised (619) $ 87.85 Cancelled — $ — Outstanding as of December 31, 2022 556 $ 97.28 3.81 $ 77,975 Exercisable as of December 31, 2022 556 $ 97.28 3.81 $ 77,975 |
Restricted Stock Units and Performance-Based Restricted Stock Units Activity | The following table summarizes the activity of our restricted stock units and awards granted under our equity plans, excluding performance-based restricted stock units: Number of Units (in thousands) Weighted-average Grant Date Fair Value (per share) Outstanding as of December 31, 2021 75 $ 158.87 Awarded 249 $ 165.32 Released (23) $ 152.98 Cancelled (9) $ 143.85 Outstanding as of December 31, 2022 292 $ 165.27 Performance-Based Restricted Stock Units The following table summarizes the activity of our performance-based restricted stock units granted under our equity plans: Number of Units (in thousands) Weighted-average Grant Date Fair Value (per share) Outstanding as of December 31, 2021 1,136 $ 143.13 Awarded 690 $ 147.47 Released (501) $ 142.72 Cancelled (130) $ 143.95 Outstanding as of December 31, 2022 1,195 $ 144.77 |
Stock Purchase Rights Granted Under the Employee Stock Purchase Plan | The following table summarizes the Black-Scholes valuation assumption inputs for stock purchase rights granted under the employee stock purchase plan: Year Ended December 31, 2022 2021 2020 Risk-free interest rate 1.4% - 4.5% 0.03% - 0.06% 0.1% - 0.1% Expected dividend yield — — — Expected option life 6 months 6 months 6 months Expected volatility 53% - 71% 41% - 46% 40% - 50% |
LEASES (Tables)
LEASES (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Leases [Abstract] | |
Summary of Costs Included in Operating Expenses Related to Leases | The following table summarizes our costs included in operating expenses related to right of use lease assets we have entered into through December 31, 2022: (In thousands) Year Ended December 31, 2022 Year Ended December 31, 2021 Operating lease cost $ 45,789 $ 45,359 Variable lease cost 17,614 18,271 Total $ 63,403 $ 63,630 |
Summary of Future Lease Payments for Non-cancellable Operating Leases and Reconciliation to Carrying Amount of Operating Lease Liability | Future lease payments for non-cancellable operating leases and a reconciliation to the carrying amount of the operating lease liability presented in the consolidated balance sheet as of December 31, 2022 were as follows, in thousands: Year Ending December 31 2023 $ 43,028 2024 46,486 2025 42,867 2026 40,376 2027 38,753 2028 and thereafter 248,622 Total undiscounted lease liability 460,132 Less imputed interest (156,826) Total discounted lease liability $ 303,306 Current operating lease liability $ 41,967 Non-current operating lease liability 261,339 Total $ 303,306 |
INCOME TAXES (Tables)
INCOME TAXES (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Income Tax Disclosure [Abstract] | |
Schedule of Domestic and Foreign Components of Loss before Income Taxes | The domestic and foreign components of loss before income taxes are as follows: (In thousands) 2022 2021 2020 Domestic $ (1,148,604) $ (794,729) $ (682,859) Foreign 21,611 (57,415) (172,741) Loss before income taxes $ (1,126,993) $ (852,144) $ (855,600) |
Schedule of Provision for Income Taxes | The provision for income taxes consisted of the following: Year Ended December 31, (In thousands) 2022 2021 2020 Current provision: Domestic $ — $ 293 $ 61 Foreign 5,596 3,154 5,837 Total current provision 5,596 3,447 5,898 Deferred benefit: Domestic — — 393 Foreign (1,433) (2,767) (3,610) Total deferred benefit (1,433) (2,767) (3,217) Total provision for income taxes $ 4,163 $ 680 $ 2,681 |
Schedule of Components of Net Deferred Tax (Liability) Asset | Components of the net deferred tax asset are as follows: As of December 31, (In thousands) 2022 2021 Deferred tax assets: Net operating loss carryforwards $ 803,251 $ 745,985 Research and development and other credit carryforwards 381,032 342,431 Sale of future royalties 326,183 302,217 Lease liability 67,242 71,859 Deferred revenue 59,437 76,612 Deferred compensation 52,989 59,349 Intangible assets 264,564 279,082 Capitalized research and development expenditures 206,727 23,039 Other 133,376 48,242 Total deferred tax assets 2,294,801 1,948,816 Deferred tax liabilities: Property, plant and equipment, net (12,786) (13,170) Unrealized gain on marketable securities (5,728) (16,693) Right of use assets (46,819) (50,562) Deferred revenue tax accounting method change (24,995) (50,380) Deferred tax asset valuation allowance (2,193,633) (1,808,992) Net deferred tax asset $ 10,840 $ 9,019 |
Schedule of Effective Income Tax Rate Differs from Statutory Federal Income Tax Rate | Our effective income tax rate differs from the statutory federal income tax rate, as follows: Year Ended December 31, (In thousands) 2022 2021 2020 At U.S. federal statutory rate 21.0 % 21.0 % 21.0 % State taxes, net of federal effect 6.0 5.2 4.5 Stock-based compensation 4.4 4.6 2.2 Tax credits 2.7 4.5 3.3 Other permanent items (1.5) (1.0) (1.5) Foreign rate differential (0.5) (1.7) (3.5) Internal reorganization of certain intellectual property rights — 20.1 12.3 Other (0.8) (0.1) (2.7) Revaluation of deferred due to rate change (0.4) 1.1 — Valuation allowance (31.3) (53.8) (35.9) Effective income tax rate (0.4) % (0.1) % (0.3) % |
NATURE OF BUSINESS (Details)
NATURE OF BUSINESS (Details) | Dec. 31, 2022 product |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Number of marketed products | 5 |
Number of partnered products | 1 |
Number of commercialized products | 4 |
SUMMARY OF SIGNIFICANT ACCOUN_4
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Customers that Represent Greater than Ten Percent of Gross Revenues (Detail) - Revenue from Rights Concentration Risk - Gross Revenues | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Distributor A | |||
Concentration Risk [Line Items] | |||
Concentration risk percentage | 33% | 27% | 31% |
Regeneron Pharmaceuticals | |||
Concentration Risk [Line Items] | |||
Concentration risk percentage | 11% | 12% |
SUMMARY OF SIGNIFICANT ACCOUN_5
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Customers that Represent Greater than Ten Percent of Gross Accounts Receivable (Detail) - Accounts Receivable - Credit Concentration Risk | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Novartis AG | ||
Concentration Risk [Line Items] | ||
Concentration risk percentage | 20% | |
Distributor A | ||
Concentration Risk [Line Items] | ||
Concentration risk percentage | 32% | 14% |
Regeneron Pharmaceuticals | ||
Concentration Risk [Line Items] | ||
Concentration risk percentage | 12% | |
Distributor B | ||
Concentration Risk [Line Items] | ||
Concentration risk percentage | 10% | 10% |
SUMMARY OF SIGNIFICANT ACCOUN_6
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Investments in Marketable Securities and Cash Equivalents - Additional Information (Detail) | 12 Months Ended |
Dec. 31, 2022 | |
Accounting Policies [Abstract] | |
Marketable securities classified as cash equivalents, maximum original maturity | 90 days |
Policy for marketable securities | 90 days |
SUMMARY OF SIGNIFICANT ACCOUN_7
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Accounts Receivable (Detail) | 12 Months Ended |
Dec. 31, 2022 | |
Minimum | |
Significant Accounting Policies [Line Items] | |
Accounts receivable payment term | 30 days |
Maximum | |
Significant Accounting Policies [Line Items] | |
Accounts receivable payment term | 90 days |
SUMMARY OF SIGNIFICANT ACCOUN_8
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Estimated Useful Lives of Property, Plant and Equipment (Detail) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Property, Plant and Equipment [Line Items] | |||
Depreciation expense | $ 39.1 | $ 36.8 | $ 30.2 |
Laboratory equipment | |||
Property, Plant and Equipment [Line Items] | |||
Property, plant and equipment, useful life | 5 years | ||
Computer equipment and software | Minimum | |||
Property, Plant and Equipment [Line Items] | |||
Property, plant and equipment, useful life | 3 years | ||
Computer equipment and software | Maximum | |||
Property, Plant and Equipment [Line Items] | |||
Property, plant and equipment, useful life | 10 years | ||
Furniture and fixtures | |||
Property, Plant and Equipment [Line Items] | |||
Property, plant and equipment, useful life | 5 years | ||
Leasehold improvements | |||
Property, Plant and Equipment [Line Items] | |||
Leasehold improvements | Shorter of asset life or lease term | ||
Manufacturing Equipment | Minimum | |||
Property, Plant and Equipment [Line Items] | |||
Property, plant and equipment, useful life | 7 years | ||
Manufacturing Equipment | Maximum | |||
Property, Plant and Equipment [Line Items] | |||
Property, plant and equipment, useful life | 15 years | ||
Buildings | |||
Property, Plant and Equipment [Line Items] | |||
Property, plant and equipment, useful life | 40 years |
SUMMARY OF SIGNIFICANT ACCOUN_9
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Revenue Recognition (Detail) - USD ($) | Dec. 31, 2022 | Dec. 31, 2021 |
Accounting Policies [Abstract] | ||
Capitalized cost | $ 0 | $ 0 |
SUMMARY OF SIGNIFICANT ACCOU_10
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Research and Development (Detail) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Research and development | |||
Significant Accounting Policies [Line Items] | |||
Research and development expense costs associated with license fees | $ 7.3 | $ 16.8 | $ 2.8 |
SUMMARY OF SIGNIFICANT ACCOU_11
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Potential Common Shares Excluded from Calculation of Net Loss Per Common Share (Detail) - shares shares in Thousands | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Anti-dilutive securities excluded from computation of earnings per share | 13,527 | 11,225 | 12,852 |
Options to purchase common stock, inclusive of performance-based stock options | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Anti-dilutive securities excluded from computation of earnings per share | 8,424 | 10,015 | 11,692 |
Unvested restricted common stock, inclusive of performance-based restricted common stock | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Anti-dilutive securities excluded from computation of earnings per share | 1,487 | 1,210 | 1,160 |
Convertible Debt | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Anti-dilutive securities excluded from computation of earnings per share | 3,616 | 0 | 0 |
SUMMARY OF SIGNIFICANT ACCOU_12
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Segment Information (Detail) | 12 Months Ended |
Dec. 31, 2022 segment | |
Accounting Policies [Abstract] | |
Number of reporting segments | 1 |
NET PRODUCT REVENUES - Summary
NET PRODUCT REVENUES - Summary of Net Product Revenues (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Net product revenues | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | $ 894,329 | $ 662,138 | $ 361,520 |
ONPATTRO | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 557,608 | 474,737 | 306,081 |
AMVUTTRA | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 93,795 | 0 | 0 |
GIVLAARI | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 173,144 | 127,815 | 55,106 |
OXLUMO | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 69,782 | 59,586 | 333 |
United States | ONPATTRO | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 246,748 | 213,210 | 151,574 |
United States | AMVUTTRA | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 82,521 | 0 | 0 |
United States | GIVLAARI | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 115,659 | 92,747 | 42,797 |
United States | OXLUMO | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 27,698 | 18,876 | 0 |
Europe | ONPATTRO | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 224,063 | 190,435 | 107,755 |
Europe | AMVUTTRA | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 4,214 | 0 | 0 |
Europe | GIVLAARI | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 48,670 | 30,895 | 12,000 |
Europe | OXLUMO | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 37,915 | 38,949 | 333 |
Rest of World | ONPATTRO | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 86,797 | 71,092 | 46,752 |
Rest of World | AMVUTTRA | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 7,060 | 0 | 0 |
Rest of World | GIVLAARI | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | 8,815 | 4,173 | 309 |
Rest of World | OXLUMO | |||
Disaggregation of Revenue [Line Items] | |||
Total revenues | $ 4,169 | $ 1,761 | $ 0 |
NET PRODUCT REVENUES - Addition
NET PRODUCT REVENUES - Additional Information (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Disaggregation of Revenue [Line Items] | ||
Accounts receivable, net | $ 237,963 | $ 198,571 |
Payments for previous acquisition | 44,300 | 56,400 |
Net product revenues | ||
Disaggregation of Revenue [Line Items] | ||
Accounts receivable, net | $ 203,800 | $ 124,900 |
NET PRODUCT REVENUES - Summar_2
NET PRODUCT REVENUES - Summary of Balances and Activity in Each Product Revenue Allowance and Reserve Category (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Movement in Valuation Allowances and Reserves [Roll Forward] | ||
Beginning balance | $ 131,316 | $ 95,107 |
Provision related to current period sales | 273,570 | 187,978 |
Credit or payments made during the period for current year sales | (128,345) | (87,928) |
Credit or payments made during the period for prior year sales | (67,543) | (63,841) |
Total | 208,998 | 131,316 |
Chargebacks and Rebates | ||
Movement in Valuation Allowances and Reserves [Roll Forward] | ||
Beginning balance | 120,682 | 90,705 |
Provision related to current period sales | 245,236 | 167,691 |
Credit or payments made during the period for current year sales | (108,185) | (75,073) |
Credit or payments made during the period for prior year sales | (65,961) | (62,641) |
Total | 191,772 | 120,682 |
Trade Discounts and Allowances | ||
Movement in Valuation Allowances and Reserves [Roll Forward] | ||
Beginning balance | 522 | 639 |
Provision related to current period sales | 13,085 | 8,064 |
Credit or payments made during the period for current year sales | (10,310) | (7,665) |
Credit or payments made during the period for prior year sales | (847) | (516) |
Total | 2,450 | 522 |
Returns Reserve and Other Incentives | ||
Movement in Valuation Allowances and Reserves [Roll Forward] | ||
Beginning balance | 10,112 | 3,763 |
Provision related to current period sales | 15,249 | 12,223 |
Credit or payments made during the period for current year sales | (9,850) | (5,190) |
Credit or payments made during the period for prior year sales | (735) | (684) |
Total | $ 14,776 | $ 10,112 |
NET REVENUES FROM COLLABORATI_3
NET REVENUES FROM COLLABORATIONS - Net Revenue from Collaborators (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Total revenues | $ 1,037,418 | $ 844,287 | $ 492,853 |
Net revenues from collaborations | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Total revenues | 134,912 | 180,953 | 131,333 |
Net revenues from collaborations | Regeneron Pharmaceuticals | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Total revenues | 87,844 | 113,226 | 74,072 |
Net revenues from collaborations | Novartis AG | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Total revenues | 43,159 | 49,120 | 22,208 |
Net revenues from collaborations | Vir Biotechnology | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Total revenues | 1,755 | 16,897 | 31,396 |
Net revenues from collaborations | Other | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Total revenues | $ 2,154 | $ 1,710 | $ 3,657 |
NET REVENUES FROM COLLABORATI_4
NET REVENUES FROM COLLABORATIONS - Balance of Receivables and Contract Liabilities Related to Collaboration Agreements (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | ||
Receivables included in "Accounts receivable, net" | $ 32,342 | $ 73,266 |
Contract liabilities included in "Deferred revenue" | 235,528 | 88,627 |
Revenue recognized on contract liability | $ 55,500 | $ 62,900 |
NET REVENUES FROM COLLABORATI_5
NET REVENUES FROM COLLABORATIONS - Schedule of Research and Development Expenses Incurred by Type that are Directly Attributable to Collaboration Agreements (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Total research and development expenses | $ 883,015 | $ 792,156 | $ 654,819 |
Reduction in research and development expense | 16,900 | 17,100 | 11,100 |
Clinical Trial and Manufacturing | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Total research and development expenses | 13,082 | 35,300 | 33,415 |
Clinical Trial and Manufacturing | Regeneron Pharmaceuticals | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Total research and development expenses | 12,926 | 24,989 | 13,302 |
Clinical Trial and Manufacturing | Other | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Total research and development expenses | 156 | 10,311 | 20,113 |
External Services | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Total research and development expenses | 2,820 | 1,615 | 936 |
External Services | Regeneron Pharmaceuticals | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Total research and development expenses | 2,141 | 840 | 171 |
External Services | Other | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Total research and development expenses | 679 | 775 | 765 |
Other | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Total research and development expenses | 28,272 | 52,071 | 58,782 |
Other | Regeneron Pharmaceuticals | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Total research and development expenses | 27,935 | 47,582 | 44,360 |
Other | Other | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Total research and development expenses | $ 337 | $ 4,489 | $ 14,422 |
NET REVENUES FROM COLLABORATI_6
NET REVENUES FROM COLLABORATIONS - Regeneron Pharmaceuticals (Detail) - Global Strategic Collaboration - Regeneron Pharmaceuticals $ in Thousands | 1 Months Ended | 12 Months Ended | |
Dec. 31, 2022 USD ($) | Apr. 30, 2019 USD ($) program | Dec. 31, 2022 USD ($) | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Discovery period of programs development | 5 years | ||
Extended additional discovery period of programs development | 5 years | ||
Maximum royalties and commercial milestone payments upon potential product sale | $ 325,000 | ||
Upfront fee received | 400,000 | ||
Maximum additional milestone payments to be receive upon achievement of certain criteria | $ 200,000 | ||
Number of targeted programs | program | 30 | ||
Collaborative agreement termination notice period | 90 days | ||
Percentage of maximum royalty payments | 20% | ||
Transaction Price Allocated | $ 558,900 | $ 521,600 | $ 558,880 |
Increase in transaction price | (20,500) | ||
Transactional price remaining performance obligation | $ 289,100 | $ 289,100 | |
Funding At Program Initiation | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Potential proceeds from collaboration arrangement | 2,500 | ||
Funding At Lead Candidate Identification | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Potential proceeds from collaboration arrangement | 2,500 | ||
Funding An Annual Discovery | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Potential proceeds from collaboration arrangement | 30,000 | ||
Maximum | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Research term extension fee | 400,000 | ||
Collaborative arrangement milestone payments | $ 150,000 | ||
Royalty rate | 20% |
NET REVENUES FROM COLLABORATI_7
NET REVENUES FROM COLLABORATIONS - Schedule of Transaction Price Allocated (Details) - Global Strategic Collaboration - Regeneron Pharmaceuticals - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | |
Dec. 31, 2022 | Apr. 30, 2019 | Dec. 31, 2022 | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Transaction Price Allocated | $ 558,900 | $ 521,600 | $ 558,880 |
Research Services Obligation | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Standalone Selling Price | 130,700 | 130,700 | |
Transaction Price Allocated | 183,100 | 215,680 | |
C5 License Obligation | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Standalone Selling Price | 97,600 | 97,600 | |
Transaction Price Allocated | 92,500 | 97,200 | |
C5 Co-Co Obligation | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Standalone Selling Price | $ 364,600 | 364,600 | |
Transaction Price Allocated | $ 246,000 | $ 246,000 |
NET REVENUES FROM COLLABORATI_8
NET REVENUES FROM COLLABORATIONS - Schedule of Deferred Revenue (Details) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | ||
Dec. 31, 2022 | Apr. 30, 2019 | Dec. 31, 2022 | Dec. 31, 2021 | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||
Deferred revenue | $ 42,105 | $ 42,105 | $ 149,483 | |
Global Strategic Collaboration | Regeneron Pharmaceuticals | ||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||
Transaction Price Allocated | 558,900 | $ 521,600 | 558,880 | |
Deferred revenue | 226,800 | 226,800 | 281,700 | |
Research Services Obligation | Global Strategic Collaboration | Regeneron Pharmaceuticals | ||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||
Transaction Price Allocated | 183,100 | 215,680 | ||
Deferred revenue | 26,200 | 26,200 | 42,300 | |
C5 License Obligation | Global Strategic Collaboration | Regeneron Pharmaceuticals | ||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||
Transaction Price Allocated | 92,500 | 97,200 | ||
Deferred revenue | 7,000 | 7,000 | 26,900 | |
C5 Co-Co Obligation | Global Strategic Collaboration | Regeneron Pharmaceuticals | ||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||
Transaction Price Allocated | $ 246,000 | 246,000 | ||
Deferred revenue | $ 193,600 | $ 193,600 | $ 212,500 |
NET REVENUES FROM COLLABORATI_9
NET REVENUES FROM COLLABORATIONS - Schedule of Revenue Recognized by Accounting Guidance (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Revenues | $ 1,037,418 | $ 844,287 | $ 492,853 |
Regeneron Pharmaceuticals | Global Strategic Collaboration | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Revenues | 81,180 | 101,100 | 63,600 |
Regeneron Pharmaceuticals | Research Services Obligation | Global Strategic Collaboration | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Revenues recognized | 28,600 | 37,600 | 44,800 |
Regeneron Pharmaceuticals | C5 License Obligation | Global Strategic Collaboration | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Revenues recognized | 32,500 | 44,600 | 7,100 |
Regeneron Pharmaceuticals | C5 Co-Co Obligation | Global Strategic Collaboration | |||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||
Revenue recognized under ASC 808 | $ 20,080 | $ 18,900 | $ 11,700 |
NET REVENUES FROM COLLABORAT_10
NET REVENUES FROM COLLABORATIONS - Additional Information (Detail) $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | 119 Months Ended | ||||||
Dec. 31, 2022 USD ($) | Jul. 31, 2021 USD ($) | Dec. 31, 2021 USD ($) | Apr. 30, 2019 USD ($) | Sep. 30, 2021 USD ($) | Dec. 31, 2022 USD ($) performanceObligation | Dec. 31, 2022 USD ($) | Dec. 31, 2022 USD ($) | Dec. 31, 2022 program | Dec. 31, 2022 candidate | |
Novartis AG | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Collaborative agreement termination notice period | 180 days | |||||||||
Performance obligations identified | performanceObligation | 1 | |||||||||
Vir Biotechnology Inc | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Performance obligations identified | performanceObligation | 1 | |||||||||
Novartis License Agreement | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Upfront fee received | $ 12,500 | $ 12,500 | ||||||||
Transaction Price Allocated | 16,000 | |||||||||
Transaction price allocated to unsatisfied performance obligation | $ 11,000 | |||||||||
Minimum | Novartis AG | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Collaborative agreement termination notice period | 3 months | |||||||||
Pepti Dream Inc | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Upfront payment | $ 10,000 | $ 10,000 | ||||||||
Commercial milestones | $ 247,000 | |||||||||
Regeneron Pharmaceuticals | Global Strategic Collaboration | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Transactional price remaining performance obligation | 289,100 | |||||||||
Upfront fee received | $ 400,000 | |||||||||
Collaborative agreement termination notice period | 90 days | |||||||||
Transaction Price Allocated | $ 558,900 | $ 521,600 | $ 558,880 | |||||||
Regeneron Pharmaceuticals | Global Strategic Collaboration | Maximum | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Royalty rate | 20% | |||||||||
Vir Biotechnology Inc | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Transactional price remaining performance obligation | $ 36,300 | |||||||||
Collaborative agreement termination notice period | 90 days | |||||||||
Transaction Price Allocated | $ 111,000 | |||||||||
Development candidates to be delivered | 4 | 4 | ||||||||
2013 The Medicines Company Collaboration | Product Alliances | Novartis AG | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Amount earned upon achievement of milestone | $ 80,000 | |||||||||
Maximum number of potential future milestones | 100,000 | |||||||||
Potential future payment for the achievement of specified commercialization milestones | 90,000 | |||||||||
Potential future payment for the achievement of other regulatory milestones | $ 10,000 | |||||||||
2013 The Medicines Company Collaboration | Product Alliances | Maximum | Novartis AG | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Royalty rate | 20% | 20% | 20% | |||||||
2013 The Medicines Company Collaboration | Product Alliances | Minimum | Novartis AG | ||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||||||||||
Royalty rate | 10% | 10% | 10% |
LIABILITY RELATED TO THE SALE_3
LIABILITY RELATED TO THE SALE OF FUTURE ROYALTIES - Narrative (Details) - USD ($) $ in Thousands | 12 Months Ended | ||||
Dec. 31, 2022 | Jan. 01, 2030 | Dec. 31, 2021 | Dec. 31, 2020 | Apr. 30, 2020 | |
Liability Related To The Sale Of Future Royalties Line Items [Line Items] | |||||
Liability related to the sale of future royalties, net of current portion | $ 1,252,015 | $ 1,151,024 | |||
Blackstone Group Inc. | Net revenues from collaborations | |||||
Liability Related To The Sale Of Future Royalties Line Items [Line Items] | |||||
Collaborative arrangement, royalties and commercial milestones acquired by collaborator, percent | 50% | ||||
Commercial milestones acquired by collaborator, percent | 75% | ||||
Expected royalty interest payments | 1,000,000 | ||||
Royalty interest payment | 1,000,000 | ||||
Liability related to the sale of future royalties, net of current portion | $ 1,292,304 | $ 1,188,103 | $ 1,071,541 | $ 1,000,000 | |
Interest rate | 8% | ||||
Closing costs | $ 10,800 | ||||
Forecast | Blackstone Group Inc. | Net revenues from collaborations | |||||
Liability Related To The Sale Of Future Royalties Line Items [Line Items] | |||||
Collaborative arrangement, royalties and commercial milestones acquired by collaborator, percent | 55% |
LIABILITY RELATED TO THE SALE_4
LIABILITY RELATED TO THE SALE OF FUTURE ROYALTIES - Future Royalties (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Liability Related To Sale Of Future Royalties [Roll Forward] | ||
Beginning balance | $ 1,151,024 | |
Ending balance | 1,252,015 | $ 1,151,024 |
Blackstone Group Inc. | Net revenues from collaborations | ||
Liability Related To Sale Of Future Royalties [Roll Forward] | ||
Beginning balance | 1,188,103 | 1,071,541 |
Interest expense recognized | 107,601 | 116,940 |
Payments | (3,400) | (378) |
Ending balance | $ 1,292,304 | $ 1,188,103 |
FAIR VALUE MEASUREMENTS - Fair
FAIR VALUE MEASUREMENTS - Fair Value of Assets Measured on a Recurring Basis (Detail) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Financial assets | ||
Cash equivalents | $ 113,243 | $ 54,998 |
Marketable debt securities | 1,411,133 | 1,603,615 |
Marketable equity securities | $ 28,122 | $ 66,972 |
Financial liabilities | ||
Derivative Liability, Statement of Financial Position [Extensible Enumeration] | Other liabilities | Other liabilities |
Recurring | ||
Financial assets | ||
Marketable equity securities | $ 28,122 | $ 66,972 |
Total financial assets | 1,710,846 | 1,927,651 |
Financial liabilities | ||
Development derivative liability | 209,277 | 83,618 |
Recurring | Money market funds | ||
Financial assets | ||
Cash equivalents | 270,394 | 255,869 |
Restricted cash (money market funds) | 1,197 | 1,195 |
Recurring | U.S. treasury securities | ||
Financial assets | ||
Cash equivalents | 44,817 | 54,998 |
Marketable debt securities | 820,913 | 1,030,578 |
Recurring | Corporate notes | ||
Financial assets | ||
Cash equivalents | 1,024 | |
Marketable debt securities | 208,284 | 253,239 |
Recurring | U.S. government-sponsored enterprise securities | ||
Financial assets | ||
Cash equivalents | 41,763 | |
Marketable debt securities | 230,770 | 177,741 |
Recurring | Commercial paper | ||
Financial assets | ||
Cash equivalents | 22,350 | |
Marketable debt securities | 36,793 | 78,543 |
Recurring | Certificates of deposit | ||
Financial assets | ||
Cash equivalents | 3,289 | |
Marketable debt securities | 1,130 | 7,501 |
Recurring | Municipal securities | ||
Financial assets | ||
Marketable debt securities | 1,015 | |
Recurring | Quoted Prices in Active Markets (Level 1) | ||
Financial assets | ||
Marketable equity securities | 28,122 | 66,972 |
Total financial assets | 299,713 | 324,036 |
Financial liabilities | ||
Development derivative liability | 0 | 0 |
Recurring | Quoted Prices in Active Markets (Level 1) | Money market funds | ||
Financial assets | ||
Cash equivalents | 270,394 | 255,869 |
Restricted cash (money market funds) | 1,197 | 1,195 |
Recurring | Quoted Prices in Active Markets (Level 1) | U.S. treasury securities | ||
Financial assets | ||
Cash equivalents | 0 | 0 |
Marketable debt securities | 0 | 0 |
Recurring | Quoted Prices in Active Markets (Level 1) | Corporate notes | ||
Financial assets | ||
Cash equivalents | 0 | |
Marketable debt securities | 0 | 0 |
Recurring | Quoted Prices in Active Markets (Level 1) | U.S. government-sponsored enterprise securities | ||
Financial assets | ||
Cash equivalents | 0 | |
Marketable debt securities | 0 | 0 |
Recurring | Quoted Prices in Active Markets (Level 1) | Commercial paper | ||
Financial assets | ||
Cash equivalents | 0 | |
Marketable debt securities | 0 | 0 |
Recurring | Quoted Prices in Active Markets (Level 1) | Certificates of deposit | ||
Financial assets | ||
Cash equivalents | 0 | |
Marketable debt securities | 0 | 0 |
Recurring | Quoted Prices in Active Markets (Level 1) | Municipal securities | ||
Financial assets | ||
Marketable debt securities | 0 | |
Recurring | Significant Observable Inputs (Level 2) | ||
Financial assets | ||
Marketable equity securities | 0 | 0 |
Total financial assets | 1,411,133 | 1,603,615 |
Financial liabilities | ||
Development derivative liability | 0 | 0 |
Recurring | Significant Observable Inputs (Level 2) | Money market funds | ||
Financial assets | ||
Cash equivalents | 0 | 0 |
Restricted cash (money market funds) | 0 | 0 |
Recurring | Significant Observable Inputs (Level 2) | U.S. treasury securities | ||
Financial assets | ||
Cash equivalents | 44,817 | 54,998 |
Marketable debt securities | 820,913 | 1,030,578 |
Recurring | Significant Observable Inputs (Level 2) | Corporate notes | ||
Financial assets | ||
Cash equivalents | 1,024 | |
Marketable debt securities | 208,284 | 253,239 |
Recurring | Significant Observable Inputs (Level 2) | U.S. government-sponsored enterprise securities | ||
Financial assets | ||
Cash equivalents | 41,763 | |
Marketable debt securities | 230,770 | 177,741 |
Recurring | Significant Observable Inputs (Level 2) | Commercial paper | ||
Financial assets | ||
Cash equivalents | 22,350 | |
Marketable debt securities | 36,793 | 78,543 |
Recurring | Significant Observable Inputs (Level 2) | Certificates of deposit | ||
Financial assets | ||
Cash equivalents | 3,289 | |
Marketable debt securities | 1,130 | 7,501 |
Recurring | Significant Observable Inputs (Level 2) | Municipal securities | ||
Financial assets | ||
Marketable debt securities | 1,015 | |
Recurring | Significant Unobservable Inputs (Level 3) | ||
Financial assets | ||
Marketable equity securities | 0 | 0 |
Total financial assets | 0 | 0 |
Financial liabilities | ||
Development derivative liability | 209,277 | 83,618 |
Recurring | Significant Unobservable Inputs (Level 3) | Money market funds | ||
Financial assets | ||
Cash equivalents | 0 | 0 |
Restricted cash (money market funds) | 0 | 0 |
Recurring | Significant Unobservable Inputs (Level 3) | U.S. treasury securities | ||
Financial assets | ||
Cash equivalents | 0 | 0 |
Marketable debt securities | 0 | 0 |
Recurring | Significant Unobservable Inputs (Level 3) | Corporate notes | ||
Financial assets | ||
Cash equivalents | 0 | |
Marketable debt securities | 0 | 0 |
Recurring | Significant Unobservable Inputs (Level 3) | U.S. government-sponsored enterprise securities | ||
Financial assets | ||
Cash equivalents | 0 | |
Marketable debt securities | 0 | 0 |
Recurring | Significant Unobservable Inputs (Level 3) | Commercial paper | ||
Financial assets | ||
Cash equivalents | 0 | |
Marketable debt securities | 0 | 0 |
Recurring | Significant Unobservable Inputs (Level 3) | Certificates of deposit | ||
Financial assets | ||
Cash equivalents | 0 | |
Marketable debt securities | $ 0 | 0 |
Recurring | Significant Unobservable Inputs (Level 3) | Municipal securities | ||
Financial assets | ||
Marketable debt securities | $ 0 |
FAIR VALUE MEASUREMENTS - Addit
FAIR VALUE MEASUREMENTS - Additional Information (Detail) - transfer | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Fair Value Disclosures [Abstract] | ||
Transfers from Level 2 to Level 1 financial assets | 0 | 0 |
MARKETABLE DEBT SECURITIES - Su
MARKETABLE DEBT SECURITIES - Summary of Company's Marketable Debt Securities (Detail) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Schedule of Available-for-sale Securities [Line Items] | ||
Amortized Cost | $ 1,420,603 | $ 1,605,245 |
Gross Unrealized Gains | 119 | 9 |
Gross Unrealized Losses | (9,589) | (1,639) |
Fair Value | 1,411,133 | 1,603,615 |
U.S. treasury securities | ||
Schedule of Available-for-sale Securities [Line Items] | ||
Amortized Cost | 870,033 | 1,086,232 |
Gross Unrealized Gains | 79 | 6 |
Gross Unrealized Losses | (4,382) | (662) |
Fair Value | 865,730 | 1,085,576 |
Corporate notes | ||
Schedule of Available-for-sale Securities [Line Items] | ||
Amortized Cost | 211,398 | 253,926 |
Gross Unrealized Gains | 16 | 1 |
Gross Unrealized Losses | (2,106) | (688) |
Fair Value | 209,308 | 253,239 |
U.S. government-sponsored enterprise securities | ||
Schedule of Available-for-sale Securities [Line Items] | ||
Amortized Cost | 275,610 | 178,027 |
Gross Unrealized Gains | 24 | 2 |
Gross Unrealized Losses | (3,101) | (288) |
Fair Value | 272,533 | 177,741 |
Commercial paper | ||
Schedule of Available-for-sale Securities [Line Items] | ||
Amortized Cost | 59,143 | 78,543 |
Gross Unrealized Gains | 0 | 0 |
Gross Unrealized Losses | 0 | 0 |
Fair Value | 59,143 | 78,543 |
Certificates of deposit | ||
Schedule of Available-for-sale Securities [Line Items] | ||
Amortized Cost | 4,419 | 7,501 |
Gross Unrealized Gains | 0 | 0 |
Gross Unrealized Losses | 0 | 0 |
Fair Value | $ 4,419 | 7,501 |
Municipal securities | ||
Schedule of Available-for-sale Securities [Line Items] | ||
Amortized Cost | 1,016 | |
Gross Unrealized Gains | 0 | |
Gross Unrealized Losses | (1) | |
Fair Value | $ 1,015 |
MARKETABLE DEBT SECURITIES - _2
MARKETABLE DEBT SECURITIES - Summary of Fair Value of Marketable Debt Securities (Detail) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Investments, Debt and Equity Securities [Abstract] | ||
Cash and cash equivalents | $ 113,243 | $ 54,998 |
Marketable debt securities | 1,297,890 | 1,548,617 |
Total | $ 1,411,133 | $ 1,603,615 |
OTHER BALANCE SHEET DETAILS - S
OTHER BALANCE SHEET DETAILS - Schedule of Inventory (Detail) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Inventory [Line Items] | ||
Raw materials | $ 22,315 | $ 14,754 |
Work in process | 113,783 | 100,942 |
Finished goods | 25,606 | 7,005 |
Total inventory | 161,704 | 122,701 |
Capitalized inventory | 0 | 7,100 |
Other Assets | ||
Inventory [Line Items] | ||
Long-term inventory | $ 32,700 | $ 36,300 |
OTHER BALANCE SHEET DETAILS -_2
OTHER BALANCE SHEET DETAILS - Schedule of Property, Plant, and Equipment (Detail) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Property, Plant and Equipment [Line Items] | ||
Property, plant and equipment, gross | $ 696,944 | $ 642,373 |
Less: accumulated depreciation | (173,450) | (140,415) |
Total | 523,494 | 501,958 |
Buildings | ||
Property, Plant and Equipment [Line Items] | ||
Property, plant and equipment, gross | 269,322 | 262,637 |
Leasehold improvements | ||
Property, Plant and Equipment [Line Items] | ||
Property, plant and equipment, gross | 230,848 | 152,045 |
Construction in progress | ||
Property, Plant and Equipment [Line Items] | ||
Property, plant and equipment, gross | 14,595 | 80,753 |
Laboratory equipment | ||
Property, Plant and Equipment [Line Items] | ||
Property, plant and equipment, gross | 82,586 | 61,351 |
Manufacturing equipment | ||
Property, Plant and Equipment [Line Items] | ||
Property, plant and equipment, gross | 45,311 | 43,739 |
Computer equipment and software | ||
Property, Plant and Equipment [Line Items] | ||
Property, plant and equipment, gross | 33,370 | 21,885 |
Furniture and fixtures | ||
Property, Plant and Equipment [Line Items] | ||
Property, plant and equipment, gross | 11,832 | 10,883 |
Land | ||
Property, Plant and Equipment [Line Items] | ||
Property, plant and equipment, gross | $ 9,080 | $ 9,080 |
OTHER BALANCE SHEET DETAILS -_3
OTHER BALANCE SHEET DETAILS - Schedule of Accrued Expenses (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Balance Sheet Related Disclosures [Abstract] | ||
Product rebates and discounts | $ 208,998 | $ 131,279 |
Compensation and related | 130,690 | 93,583 |
Pre-clinical, clinical trial and manufacturing | 94,702 | 83,534 |
Licensing and collaboration agreements | 31,680 | 22,843 |
Consulting and professional services | 19,848 | 17,784 |
Other | 59,542 | 46,151 |
Total | $ 545,460 | $ 395,174 |
OTHER BALANCE SHEET DETAILS -_4
OTHER BALANCE SHEET DETAILS - Schedule of Cash and Cash Equivalents (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 |
Balance Sheet Related Disclosures [Abstract] | ||||
Cash and cash equivalents | $ 866,394 | $ 819,975 | $ 496,580 | |
Total restricted cash included in other assets | 2,162 | 2,178 | 2,466 | |
Total cash, cash equivalents, and restricted cash shown in the consolidated statements of cash flows | $ 868,556 | $ 822,153 | $ 499,046 | $ 549,628 |
OTHER BALANCE SHEET DETAILS -_5
OTHER BALANCE SHEET DETAILS - Summary of Changes in Accumulated Other Comprehensive Income (Loss) (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
AOCI Attributable to Parent, Net of Tax [Roll Forward] | |||
Beginning balance | $ 588,203 | $ 1,016,247 | $ 1,438,692 |
Other comprehensive loss before reclassifications | (5,263) | 12,297 | |
Amounts reclassified from other comprehensive income | (6,132) | (1,934) | |
Total other comprehensive (loss) income | (11,395) | 10,363 | (7,104) |
Ending balance | (158,223) | 588,203 | 1,016,247 |
Loss on Investment in Joint Venture | |||
AOCI Attributable to Parent, Net of Tax [Roll Forward] | |||
Beginning balance | (32,792) | (32,792) | |
Other comprehensive loss before reclassifications | 0 | 0 | |
Amounts reclassified from other comprehensive income | 0 | 0 | |
Total other comprehensive (loss) income | 0 | 0 | |
Ending balance | (32,792) | (32,792) | (32,792) |
Defined Benefit Pension Plans, Net of Tax | |||
AOCI Attributable to Parent, Net of Tax [Roll Forward] | |||
Beginning balance | (2,811) | (3,754) | |
Other comprehensive loss before reclassifications | 0 | 899 | |
Amounts reclassified from other comprehensive income | 1,719 | 44 | |
Total other comprehensive (loss) income | 1,719 | 943 | |
Ending balance | (1,092) | (2,811) | (3,754) |
Unrealized Gains (Losses) from Debt Securities | |||
AOCI Attributable to Parent, Net of Tax [Roll Forward] | |||
Beginning balance | (1,630) | 348 | |
Other comprehensive loss before reclassifications | 11 | 0 | |
Amounts reclassified from other comprehensive income | (7,851) | (1,978) | |
Total other comprehensive (loss) income | (7,840) | (1,978) | |
Ending balance | (9,470) | (1,630) | 348 |
Foreign Currency Translation Adjustment | |||
AOCI Attributable to Parent, Net of Tax [Roll Forward] | |||
Beginning balance | 3,974 | (7,424) | |
Other comprehensive loss before reclassifications | (5,274) | 11,398 | |
Amounts reclassified from other comprehensive income | 0 | 0 | |
Total other comprehensive (loss) income | (5,274) | 11,398 | |
Ending balance | (1,300) | 3,974 | (7,424) |
Accumulated Other Comprehensive (Loss) Income | |||
AOCI Attributable to Parent, Net of Tax [Roll Forward] | |||
Beginning balance | (33,259) | (43,622) | (36,518) |
Total other comprehensive (loss) income | (11,395) | 10,363 | (7,104) |
Ending balance | $ (44,654) | $ (33,259) | $ (43,622) |
CREDIT AGREEMENT - Additional I
CREDIT AGREEMENT - Additional Information (Details) $ in Thousands | 1 Months Ended | 12 Months Ended | ||||
Sep. 30, 2022 USD ($) | Dec. 31, 2022 USD ($) | Dec. 31, 2021 USD ($) | Dec. 31, 2020 USD ($) | Jun. 30, 2022 | Apr. 30, 2020 USD ($) tranche | |
Financing Receivable, Impaired [Line Items] | ||||||
Loss on extinguishment of debt | $ 76,586 | $ 0 | $ 0 | |||
Line of Credit | Secured Debt | ||||||
Financing Receivable, Impaired [Line Items] | ||||||
Number of tranches | tranche | 3 | |||||
Loan facility | $ 700,000 | |||||
Aggregate principal amount | $ 700,000 | |||||
Payments for other fees | 62,100 | |||||
Loss on extinguishment of debt | $ 76,600 | |||||
Funding fee | $ 17,500 | |||||
Interest rate floor | 8% | 9% | ||||
Line of Credit | LIBOR | Secured Debt | ||||||
Financing Receivable, Impaired [Line Items] | ||||||
Debt instrument, basis spread on variable rate | 7% |
CONVERTIBLE DEBT (Details)
CONVERTIBLE DEBT (Details) $ / shares in Units, $ in Thousands | 12 Months Ended | |||||
Sep. 15, 2022 day | Sep. 12, 2022 USD ($) $ / shares | Dec. 31, 2022 USD ($) $ / shares $ / Unit | Dec. 31, 2021 USD ($) $ / shares | Dec. 31, 2020 USD ($) | Sep. 13, 2022 USD ($) | |
Line of Credit Facility [Line Items] | ||||||
Common stock, previously reported value (in usd per share) | $ / shares | $ 0.01 | $ 0.01 | ||||
Purchase of capped calls related to convertible debt | $ 118,611 | $ 0 | $ 0 | |||
Convertible Debt | Convertible Senior Notes Due 2027 | ||||||
Line of Credit Facility [Line Items] | ||||||
Aggregate principal amount | $ 1,040,000 | |||||
Interest rate | 1% | 1% | ||||
Debt instrument conversion ratio | 0.0034941 | |||||
Debt instrument, conversion price (in usd per share) | $ / shares | $ 286.20 | |||||
Premium from conversion | 0.35 | |||||
Common stock, previously reported value (in usd per share) | $ / shares | $ 212 | |||||
Debt issuance costs | $ 19,200 | |||||
Fair value of long-term debt | 1,120,000 | |||||
Interest expense | $ 3,000 | |||||
Convertible Debt | Convertible Senior Notes Due 2027 | Debt Conversion Terms One | ||||||
Line of Credit Facility [Line Items] | ||||||
Trading days threshold | day | 20 | |||||
Consecutive trading days threshold | day | 30 | |||||
Percentage of stock price trigger | 130% | |||||
Convertible Debt | Convertible Senior Notes Due 2027 | Debt Conversion Terms Two | ||||||
Line of Credit Facility [Line Items] | ||||||
Trading days threshold | day | 5 | |||||
Consecutive trading days threshold | day | 10 | |||||
Percentage of stock price trigger | 98% | |||||
Convertible Debt | Convertible Senior Notes Due 2027 | Debt Conversion Terms Three | ||||||
Line of Credit Facility [Line Items] | ||||||
Trading days threshold | day | 20 | |||||
Consecutive trading days threshold | day | 30 | |||||
Percentage of stock price trigger | 130% | |||||
Debt instrument, redemption price percentage | 100% | |||||
Convertible Debt | Convertible Senior Notes Due 2027, Initial Amount | ||||||
Line of Credit Facility [Line Items] | ||||||
Aggregate principal amount | $ 900,000 | |||||
Convertible Debt | Convertible Senior Notes Due 2027, Additional Amount | ||||||
Line of Credit Facility [Line Items] | ||||||
Aggregate principal amount | $ 135,000 | |||||
Convertible Debt | Capped Call Transactions | ||||||
Line of Credit Facility [Line Items] | ||||||
Common stock, previously reported value (in usd per share) | $ / shares | $ 212 | |||||
Cap price (in usd per share) | $ / Unit | 424 | |||||
Premium on capped call transactions | 1 | |||||
Purchase of capped calls related to convertible debt | $ 118,600 |
DEVELOPMENT DERIVATIVE LIABIL_3
DEVELOPMENT DERIVATIVE LIABILITY - Narrative (Details) - USD ($) $ in Millions | 1 Months Ended | |
Aug. 31, 2020 | Dec. 31, 2022 | |
Derivative [Line Items] | ||
Cost of borrowing | 12% | |
Blackstone Life Sciences | ||
Derivative [Line Items] | ||
Cost of borrowing | 4% | |
Net revenues from collaborations | Blackstone Life Sciences | Vutrisiran and ALN-AGT | ||
Derivative [Line Items] | ||
Maximum funding | $ 150 | |
Net revenues from collaborations | Blackstone Life Sciences | HELIOS-B Phase 3 Clinical Trial | ||
Derivative [Line Items] | ||
Maximum funding | 70 | |
Net revenues from collaborations | Blackstone Life Sciences | ALN-AGT Phase 2 Clinical Trial | ||
Derivative [Line Items] | ||
Maximum funding | $ 26 | |
Fixed payment multiplier | 3.25 | |
Fixed payment, term | 4 years | |
Net revenues from collaborations | Blackstone Life Sciences | ALN-AGT Phase 3 Clinical Trial | ||
Derivative [Line Items] | ||
Maximum funding | $ 54 | |
Fixed payment multiplier | 4.5 | |
Fixed payment, term | 4 years | |
Net revenues from collaborations | Blackstone Life Sciences | Vutrisiran | ||
Derivative [Line Items] | ||
Royalties payable, percent | 1,000% | |
Royalties payable, term | 10 years | |
Fixed payment multiplier | 2.5 | |
Fixed payment, term | 2 years |
DEVELOPMENT DERIVATIVE LIABIL_4
DEVELOPMENT DERIVATIVE LIABILITY - Development Derivative Liability Activity (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation, Calculation [Roll Forward] | ||
Fair Value, Liability, Recurring Basis, Unobservable Input Reconciliation, Gain (Loss), Statement of Income or Comprehensive Income [Extensible Enumeration] | Other (expense) income, net | Other (expense) income, net |
Derivative | ||
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation, Calculation [Roll Forward] | ||
Development derivative liability beginning balance | $ 83,618 | $ 25,585 |
Amount received under the Funding Agreement | 31,000 | 19,600 |
Loss recorded from remeasurement | 94,659 | 38,433 |
Development derivative liability as of ending balance | $ 209,277 | $ 83,618 |
STOCK-BASED COMPENSATION - Addi
STOCK-BASED COMPENSATION - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | 12 Months Ended | ||||
May 31, 2022 | May 31, 2017 | Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | May 31, 2020 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Shares of common stock reserved for future issuance under stock options (in shares) | 20,711,629 | |||||
Employee stock purchase plan, offering period | 6 months | |||||
Not Full Value Award | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Shares counted against share pool (in shares) | 1 | |||||
Full Value Award | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Shares counted against share pool (in shares) | 1.5 | |||||
Stock Options | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Shares of common stock reserved for future issuance under stock options (in shares) | 8,423,552 | |||||
Equity awards, term | 10 years | |||||
Restricted stock units | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Shares of common stock reserved for future issuance under stock options (in shares) | 1,487,394 | |||||
Additional Equity Awards Available for Future Grant | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Shares of common stock reserved for future issuance under stock options (in shares) | 10,083,911 | |||||
Employee Stock Purchase Plan | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Stock incentive plan, shares authorized (in shares) | 1,965,789 | |||||
Shares of common stock reserved for future issuance under stock options (in shares) | 716,772 | |||||
Employee stock purchase plan, purchase price as a percentage of common stock closing price | 85% | |||||
Employee stock purchase plan, shares issued (in shares) | 119,285 | 124,101 | ||||
Performance Based Stock Options | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Stock options vested (in shares) | 0 | 197,102 | 0 | |||
Aggregate intrinsic value of stock option exercised | $ 74.4 | $ 40.2 | $ 34.1 | |||
Vesting period | 1 year | |||||
Time-based stock options | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Option vesting percentage on the first anniversary of grant date | 25% | |||||
Option vesting percentage at the end of each successive three-month period | 6.25% | |||||
Weighted average fair value of stock options granted (in dollars per share) | $ 80.65 | $ 82.59 | $ 66.28 | |||
Aggregate intrinsic value of stock option exercised | $ 289.3 | $ 247.8 | $ 177.8 | |||
Stock Incentive Plan 2018 | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Stock incentive plan, shares authorized (in shares) | 7,000,000 | |||||
Stock incentive plan, additional shares authorized (in shares) | 6,000,000 |
STOCK-BASED COMPENSATION - Expe
STOCK-BASED COMPENSATION - Expenses included in operating costs and expenses (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Total stock-based compensation expense | $ 230,649 | $ 165,717 | $ 139,873 |
Research and development | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Total stock-based compensation expense | 92,161 | 68,415 | 60,464 |
Selling, general and administrative | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Total stock-based compensation expense | $ 138,488 | $ 97,302 | $ 79,409 |
STOCK-BASED COMPENSATION - Summ
STOCK-BASED COMPENSATION - Summary of Stock-Based Compensation Expense (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Less: Stock-based compensation expense capitalized to inventory | $ (4,025) | $ (3,327) | $ (571) |
Total stock-based compensation expense | 230,649 | 165,717 | 139,873 |
Time-based stock options | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation, expensed and capitalized | 114,901 | 118,635 | 112,971 |
Time-based restricted stock units | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation, expensed and capitalized | 12,791 | 4,231 | 6,909 |
Performance-based restricted stock units | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation, expensed and capitalized | 102,925 | 39,943 | 11,162 |
Other equity programs | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation, expensed and capitalized | $ 4,057 | $ 6,235 | $ 9,402 |
STOCK-BASED COMPENSATION - Su_2
STOCK-BASED COMPENSATION - Summary of Unrecognized Stock-Based Compensation Expense, Net of Estimated Forfeitures (Detail) $ in Thousands | 12 Months Ended |
Dec. 31, 2022 USD ($) | |
Time-based stock options | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Unrecognized Expense, Net of Estimated Forfeitures (in thousands) | $ 187,534 |
Weighted-average Recognition Period (in years) | 2 years 5 months 19 days |
Time-based restricted stock units | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Unrecognized Expense, Net of Estimated Forfeitures (in thousands) | $ 30,676 |
Weighted-average Recognition Period (in years) | 2 years 1 month 2 days |
Performance-based restricted stock units | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Unrecognized Expense, Net of Estimated Forfeitures (in thousands) | $ 7,695 |
Weighted-average Recognition Period (in years) | 6 months 29 days |
Other equity programs | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Unrecognized Expense, Net of Estimated Forfeitures (in thousands) | $ 4,225 |
Weighted-average Recognition Period (in years) | 5 months 12 days |
STOCK-BASED COMPENSATION - Valu
STOCK-BASED COMPENSATION - Valuation Assumptions for Stock Options (Detail) - Stock Options | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Risk-free interest rate, minimum | 1.30% | 0.40% | 0.30% |
Risk-free interest rate, maximum | 4.20% | 1.40% | 1.70% |
Expected dividend yield | 0% | 0% | 0% |
Expected volatility, minimum | 50% | 58% | 61% |
Expected volatility, maximum | 60% | 63% | 63% |
Minimum | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Expected option life | 5 years 1 month 6 days | 5 years 4 months 24 days | 5 years 4 months 24 days |
Maximum | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Expected option life | 7 years | 6 years 9 months 18 days | 7 years 2 months 12 days |
STOCK-BASED COMPENSATION - Stoc
STOCK-BASED COMPENSATION - Stock Option Activity (Detail) $ / shares in Units, shares in Thousands, $ in Thousands | 12 Months Ended |
Dec. 31, 2022 USD ($) $ / shares shares | |
Time-based stock options | |
Number of Options | |
Outstanding, beginning balance (in shares) | shares | 8,840 |
Granted (in shares) | shares | 1,874 |
Exercised (in shares) | shares | (2,490) |
Cancelled (in shares) | shares | (356) |
Outstanding, ending balance (in shares) | shares | 7,868 |
Exercisable at December 31, 2021 (in shares) | shares | 4,450 |
Vested or expected to vest at December 31, 2021 (in shares) | shares | 7,537 |
Weighted-average Exercise Price | |
Outstanding, beginning balance (in dollars per share) | $ / shares | $ 103.87 |
Granted (in dollars per share) | $ / shares | 153.18 |
Exercised (in dollars per share) | $ / shares | 84.48 |
Cancelled (in dollars per share) | $ / shares | 129.47 |
Outstanding, ending balance (in dollars per share) | $ / shares | 120.59 |
Exercisable at December 31, 2021 (in dollars per share) | $ / shares | 102.17 |
Vested or expected to vest at December 31, 2021 (in dollars per share) | $ / shares | $ 119.24 |
Weighted-average Remaining Contractual Term | |
Outstanding | 6 years 6 months 10 days |
Exercisable | 5 years 2 months 19 days |
Vested or expected to vest | 6 years 5 months 8 days |
Aggregate Intrinsic Value | |
Outstanding | $ | $ 921,016 |
Exercisable | $ | 602,969 |
Vested or expected to vest | $ | $ 892,463 |
Performance Based Stock Options | |
Number of Options | |
Outstanding, beginning balance (in shares) | shares | 1,175 |
Granted (in shares) | shares | 0 |
Exercised (in shares) | shares | (619) |
Cancelled (in shares) | shares | 0 |
Outstanding, ending balance (in shares) | shares | 556 |
Exercisable at December 31, 2021 (in shares) | shares | 556 |
Weighted-average Exercise Price | |
Outstanding, beginning balance (in dollars per share) | $ / shares | $ 92.31 |
Granted (in dollars per share) | $ / shares | 0 |
Exercised (in dollars per share) | $ / shares | 87.85 |
Cancelled (in dollars per share) | $ / shares | 0 |
Outstanding, ending balance (in dollars per share) | $ / shares | 97.28 |
Exercisable at December 31, 2021 (in dollars per share) | $ / shares | $ 97.28 |
Weighted-average Remaining Contractual Term | |
Outstanding | 3 years 9 months 21 days |
Exercisable | 3 years 9 months 21 days |
Aggregate Intrinsic Value | |
Outstanding | $ | $ 77,975 |
Exercisable | $ | $ 77,975 |
STOCK-BASED COMPENSATION - Acti
STOCK-BASED COMPENSATION - Activity of Restricted Stock Units and Performance-Based Restricted Stock Units (Details) shares in Thousands | 12 Months Ended |
Dec. 31, 2022 $ / shares shares | |
Restricted stock units | |
Number of Units | |
Outstanding, beginning balance (in shares) | shares | 75 |
Granted (in shares) | shares | 249 |
Vested (in shares) | shares | (23) |
Cancelled (in shares) | shares | (9) |
Outstanding, ending balance (in shares) | shares | 292 |
Weighted-average Grant Date Fair Value | |
Outstanding, beginning balance (in dollars per share) | $ / shares | $ 158.87 |
Granted (in dollars per share) | $ / shares | 165.32 |
Vested (in dollars per share) | $ / shares | 152.98 |
Cancelled (in dollars per share) | $ / shares | 143.85 |
Outstanding, ending balance (in dollars per share) | $ / shares | $ 165.27 |
Performance-based restricted stock units | |
Number of Units | |
Outstanding, beginning balance (in shares) | shares | 1,136 |
Granted (in shares) | shares | 690 |
Vested (in shares) | shares | (501) |
Cancelled (in shares) | shares | (130) |
Outstanding, ending balance (in shares) | shares | 1,195 |
Weighted-average Grant Date Fair Value | |
Outstanding, beginning balance (in dollars per share) | $ / shares | $ 143.13 |
Granted (in dollars per share) | $ / shares | 147.47 |
Vested (in dollars per share) | $ / shares | 142.72 |
Cancelled (in dollars per share) | $ / shares | 143.95 |
Outstanding, ending balance (in dollars per share) | $ / shares | $ 144.77 |
STOCK-BASED COMPENSATION - St_2
STOCK-BASED COMPENSATION - Stock Purchase Rights Granted Under ESPP (Details) - Employee Stock Purchase Plan | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Risk-free interest rate, minimum | 1.40% | 0.03% | 0.10% |
Risk-free interest rate, maximum | 4.50% | 0.06% | 0.10% |
Expected dividend yield | 0% | 0% | 0% |
Expected option life | 6 months | 6 months | 6 months |
Expected volatility, minimum | 53% | 41% | 40% |
Expected volatility, maximum | 71% | 46% | 50% |
STOCKHOLDERS' EQUITY - Addition
STOCKHOLDERS' EQUITY - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | ||
Apr. 30, 2020 | Dec. 31, 2022 | Dec. 31, 2021 | |
Equity [Line Items] | |||
Preferred stock, shares authorized (in shares) | 5,000,000 | 5,000,000 | |
Preferred stock, par value (in dollars per share) | $ 0.01 | $ 0.01 | |
Preferred stock, shares outstanding (in shares) | 0 | 0 | |
Blackstone Group Inc. | Common Stock | Private Placement | |||
Equity [Line Items] | |||
Number of shares issued in transaction (in shares) | 963,486 | ||
Consideration received on transaction | $ 100 | ||
Price per share (in dollars per share) | $ 103.79 |
LEASES - Additional Information
LEASES - Additional Information (Detail) $ in Millions | 12 Months Ended | |||
May 01, 2018 | Dec. 31, 2022 USD ($) option facility | Dec. 31, 2021 USD ($) | Dec. 31, 2020 USD ($) | |
Operating Leased Assets [Line Items] | ||||
Number of facilities leased | facility | 3 | |||
Lease expense | $ | $ 58.6 | $ 59.5 | $ 50.7 | |
Net cash paid included in operating activities in cash flow | $ | $ 43.1 | $ 41.9 | ||
Operating lease, weighted average remaining lease term | 10 years | 11 years | ||
Operating lease, weighted-average discount rate | 8% | 8% | ||
BMR-675 West Kendall Lease | ||||
Operating Leased Assets [Line Items] | ||||
Lease term | 15 years | |||
Number of lease extension options | 2 | |||
Operating lease renewal options period | 5 years | |||
Third Street Lease | ||||
Operating Leased Assets [Line Items] | ||||
Number of lease extension options | 2 | |||
Operating lease renewal options period | 5 years | |||
101 Main Street Leases | ||||
Operating Leased Assets [Line Items] | ||||
Number of lease extension options | 1 | |||
Operating lease renewal options period | 5 years |
LEASES - Summary of Costs Inclu
LEASES - Summary of Costs Included in Operating Expenses Related to Leases (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Leases [Abstract] | ||
Operating lease cost | $ 45,789 | $ 45,359 |
Variable lease cost | 17,614 | 18,271 |
Total | $ 63,403 | $ 63,630 |
LEASES - Summary of Future Leas
LEASES - Summary of Future Lease Payments for Non-cancellable Operating Leases and Reconciliation to Carrying Amount of Operating Lease Liability (Detail) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Leases [Abstract] | ||
2023 | $ 43,028 | |
2024 | 46,486 | |
2025 | 42,867 | |
2026 | 40,376 | |
2027 | 38,753 | |
2028 and thereafter | 248,622 | |
Total undiscounted lease liability | 460,132 | |
Less imputed interest | (156,826) | |
Total discounted lease liability | 303,306 | |
Current operating lease liability | 41,967 | $ 40,548 |
Non-current operating lease liability | $ 261,339 | $ 281,347 |
INCOME TAXES - Schedule of Dome
INCOME TAXES - Schedule of Domestic and Foreign Components of Loss before Income Taxes (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Income Tax Disclosure [Abstract] | |||
Domestic | $ (1,148,604) | $ (794,729) | $ (682,859) |
Foreign | 21,611 | (57,415) | (172,741) |
Loss before income taxes | $ (1,126,993) | $ (852,144) | $ (855,600) |
INCOME TAXES - Schedule of Prov
INCOME TAXES - Schedule of Provision for Income Taxes (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Current provision: | |||
Domestic | $ 0 | $ 293 | $ 61 |
Foreign | 5,596 | 3,154 | 5,837 |
Total current provision | 5,596 | 3,447 | 5,898 |
Deferred benefit: | |||
Domestic | 0 | 0 | 393 |
Foreign | (1,433) | (2,767) | (3,610) |
Total deferred benefit | (1,433) | (2,767) | (3,217) |
Total provision for income taxes | $ 4,163 | $ 680 | $ 2,681 |
INCOME TAXES - Schedule of Comp
INCOME TAXES - Schedule of Components of Net Deferred Tax (Liability) Asset (Detail) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Deferred tax assets: | ||
Net operating loss carryforwards | $ 803,251 | $ 745,985 |
Research and development and other credit carryforwards | 381,032 | 342,431 |
Sale of future royalties | 326,183 | 302,217 |
Lease liability | 67,242 | 71,859 |
Deferred revenue | 59,437 | 76,612 |
Deferred compensation | 52,989 | 59,349 |
Intangible assets | 264,564 | 279,082 |
Capitalized research and development expenditures | 206,727 | 23,039 |
Other | 133,376 | 48,242 |
Total deferred tax assets | 2,294,801 | 1,948,816 |
Deferred tax liabilities: | ||
Property, plant and equipment, net | (12,786) | (13,170) |
Unrealized gain on marketable securities | (5,728) | (16,693) |
Right of use assets | (46,819) | (50,562) |
Deferred revenue tax accounting method change | (24,995) | (50,380) |
Deferred tax asset valuation allowance | (2,193,633) | (1,808,992) |
Net deferred tax asset | $ 10,840 | $ 9,019 |
INCOME TAXES - Schedule of Effe
INCOME TAXES - Schedule of Effective Income Tax Rate Differs from Statutory Federal Income Tax Rate (Detail) | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Effective Income Tax Rate Reconciliation, Percent | |||
At U.S. federal statutory rate | 21% | 21% | 21% |
State taxes, net of federal effect | 6% | 5.20% | 4.50% |
Stock-based compensation | 4.40% | 4.60% | 2.20% |
Tax credits | 2.70% | 4.50% | 3.30% |
Other permanent items | (1.50%) | (1.00%) | (1.50%) |
Foreign rate differential | (0.50%) | (1.70%) | (3.50%) |
Internal reorganization of certain intellectual property rights | 0% | 20.10% | 12.30% |
Other | (0.80%) | (0.10%) | (2.70%) |
Revaluation of deferred due to rate change | (0.40%) | 1.10% | 0% |
Valuation allowance | (31.30%) | (53.80%) | (35.90%) |
Effective income tax rate | (0.40%) | (0.10%) | (0.30%) |
INCOME TAXES - Additional Infor
INCOME TAXES - Additional Information (Detail) - USD ($) $ in Thousands | 12 Months Ended | 24 Months Ended | 36 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2022 | Dec. 31, 2020 | |
Income Taxes [Line Items] | |||||
Provision for income taxes | $ 4,163 | $ 680 | $ 2,681 | ||
Foreign | 5,596 | 3,154 | 5,837 | ||
Deferred tax benefit in foreign jurisdictions | (1,433) | (2,767) | (3,610) | ||
Increase (decrease) in valuation allowance | 384,600 | $ 459,300 | $ 303,700 | ||
Capitalized research and development expenditures | 870,600 | $ 870,600 | |||
Percentage of taxable income available to be offset | 80% | 100% | |||
Federal | |||||
Income Taxes [Line Items] | |||||
Net operating loss carryforward | 2,900,000 | $ 2,900,000 | |||
Net operating loss carryforwards, subject to expiration | 1,100,000 | 1,100,000 | |||
Net operating loss carryforwards, not subject to expiration | 1,800,000 | 1,800,000 | |||
State and local jurisdiction | |||||
Income Taxes [Line Items] | |||||
Net operating loss carryforward | 3,000,000 | 3,000,000 | |||
Foreign | |||||
Income Taxes [Line Items] | |||||
Net operating loss carryforward | 153,300 | 153,300 | |||
Research and Development, Including Orphan Drug, and State Investments Tax Credit | Federal | |||||
Income Taxes [Line Items] | |||||
Tax credit carryforwards | 343,000 | 343,000 | |||
Research and Development, Including Orphan Drug, and State Investments Tax Credit | State and local jurisdiction | |||||
Income Taxes [Line Items] | |||||
Tax credit carryforwards | $ 62,300 | $ 62,300 |
EMPLOYEE BENEFIT PLANS - Additi
EMPLOYEE BENEFIT PLANS - Additional Information (Details) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Defined Benefit Plan Disclosure [Line Items] | |||
Defined benefit and contribution plans, contributions and net periodic benefit costs | $ 16.5 | $ 13.1 | $ 12 |
401(k) Matching Plan 1 | |||
Defined Benefit Plan Disclosure [Line Items] | |||
Employer matching contribution, percent of match | 100% | ||
Employer matching contribution, percent of employees' gross pay | 2% | ||
401(k) Matching Plan 2 | |||
Defined Benefit Plan Disclosure [Line Items] | |||
Employer matching contribution, percent of match | 50% | ||
Employer matching contribution, percent of employees' gross pay | 4% |