Document and Entity Information
Document and Entity Information - shares | 3 Months Ended | |
Mar. 31, 2024 | Apr. 26, 2024 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Mar. 31, 2024 | |
Document Fiscal Year Focus | 2024 | |
Document Fiscal Period Focus | Q1 | |
Entity Registrant Name | SAREPTA THERAPEUTICS, INC. | |
Entity Central Index Key | 0000873303 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Shell Company | false | |
Entity File Number | 001-14895 | |
Entity Tax Identification Number | 93-0797222 | |
Entity Incorporation, State or Country Code | DE | |
Entity Address, Address Line One | 215 First Street | |
Entity Address, Address Line Two | Suite 415 | |
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 | 274-4000 | |
Title of 12(b) Security | Common Stock, $0.0001 par value per share | |
Trading Symbol | SRPT | |
Security Exchange Name | NASDAQ | |
Document Quarterly Report | true | |
Document Transition Report | false | |
Entity Common Stock, Shares Outstanding | 94,523,766 |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Current assets: | ||
Cash and cash equivalents | $ 427,290 | $ 428,430 |
Short-term investments | 963,453 | 1,247,820 |
Accounts receivable, net | 378,806 | 400,327 |
Inventory | 373,530 | 322,859 |
Manufacturing-related deposits and prepaids | 238,821 | 102,181 |
Other current assets | 82,965 | 77,714 |
Total current assets | 2,464,865 | 2,579,331 |
Property and equipment, net | 249,302 | 227,154 |
Right of use assets | 126,269 | 129,952 |
Non-current inventory | 207,542 | 191,368 |
Other non-current assets | 176,407 | 136,771 |
Total assets | 3,224,385 | 3,264,576 |
Current liabilities: | ||
Accounts payable | 91,536 | 164,918 |
Accrued expenses | 283,317 | 314,997 |
Deferred revenue, current portion | 112,000 | 50,416 |
Current portion of long-term debt | 105,586 | 105,483 |
Other current liabilities | 16,270 | 17,845 |
Total current liabilities | 608,709 | 653,659 |
Long-term debt | 1,133,660 | 1,132,515 |
Lease liabilities, net of current portion | 140,102 | 140,965 |
Deferred revenue, net of current portion | 325,000 | 437,000 |
Contingent consideration | 48,200 | 38,100 |
Other non-current liabilities | 7,522 | 3,000 |
Total liabilities | 2,263,193 | 2,405,239 |
Commitments and contingencies (Note 14) | ||
Stockholders’ equity: | ||
Preferred stock, $0.0001 par value, 3,333,333 shares authorized; none issued and outstanding | 0 | 0 |
Common stock, $0.0001 par value, 198,000,000 shares authorized; 94,490,157 and 93,731,831 issued and outstanding at March 31, 2024, and December 31, 2023, respectively | 9 | 9 |
Additional paid-in capital | 5,371,968 | 5,304,623 |
Accumulated other comprehensive (loss) income, net of tax | (691) | 918 |
Accumulated deficit | (4,410,094) | (4,446,213) |
Total stockholders’ equity | 961,192 | 859,337 |
Total liabilities and stockholders’ equity | $ 3,224,385 | $ 3,264,576 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Mar. 31, 2024 | Dec. 31, 2023 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 3,333,333 | 3,333,333 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 198,000,000 | 198,000,000 |
Common stock, issued | 94,490,157 | 93,731,831 |
Common stock, outstanding | 94,490,157 | 93,731,831 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Revenues: | ||
Products, net | $ 359,484 | $ 231,495 |
Type of Revenue [Extensible List] | us-gaap:ProductMember | us-gaap:ProductMember |
Collaboration and other | $ 53,980 | $ 22,005 |
Total revenues | 413,464 | 253,500 |
Cost and expenses: | ||
Cost of sales (excluding amortization of in-licensed rights) | 50,559 | 35,017 |
Research and development | 200,396 | 245,679 |
Selling, general and administrative | 127,003 | 110,714 |
Amortization of in-licensed rights | 601 | 178 |
Total cost and expenses | 378,559 | 391,588 |
Operating income (loss) | 34,905 | (138,088) |
Other income (loss), net: | ||
Loss on debt extinguishment | 0 | (387,329) |
Other income, net | 6,543 | 12,707 |
Total other income (loss), net | 6,543 | (374,622) |
Income (loss) before income tax expense | 41,448 | (512,710) |
Provision for Income tax | 5,329 | 4,045 |
Net income (loss) | 36,119 | (516,755) |
Other comprehensive income (loss): | ||
Unrealized gains (losses) on investments, net of tax | (1,609) | 1,245 |
Total other comprehensive (loss) income | (1,609) | 1,245 |
Comprehensive income (loss) | $ 34,510 | $ (515,510) |
Earnings (loss) per share: | ||
Earnings (loss) per common share, basic | $ 0.38 | $ (5.86) |
Earnings (loss) per common share, diluted | $ 0.37 | $ (5.86) |
Weighted average number of shares of common stock used in computing earnings (loss) per share | ||
Basic | 93,991,000 | 88,186,000 |
Diluted | 99,114,000 | 88,186,000 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Total | Common Stock [Member] | Additional Paid-In Capital [Member] | Accumulated Other Comprehensive (Loss) Income [Member] | Accumulated Deficit [Member] |
Balance at Dec. 31, 2022 | $ 384,950 | $ 9 | $ 4,296,841 | $ (1,664) | $ (3,910,236) |
Balance (in shares) at Dec. 31, 2022 | 87,950,000 | ||||
Exercise of options for common stock | 22,808 | 22,808 | |||
Exercise of options for common stock, shares | 267,000 | ||||
Vest of restricted stock units/awards | 390,000 | ||||
Issuance of common stock for exchange of 2024 Notes | 693,377 | 693,377 | |||
Issuance of common stock for exchange of 2024 Notes, shares | 4,456,000 | ||||
Partial settlement of capped call share options for 2024 Notes | 80,645 | 80,645 | |||
Issuance of common stock under employee stock purchase plan | 5,229 | 5,229 | |||
Issuance of common stock under employee stock purchase plan, shares | 77,000 | ||||
Stock-based compensation | 41,250 | 41,250 | |||
Unrealized gains from available-for-sale securities, net of tax | 1,245 | 1,245 | |||
Net Income (Loss) | (516,755) | (516,755) | |||
Balance at Mar. 31, 2023 | 712,749 | $ 9 | 5,140,150 | (419) | (4,426,991) |
Balance (in shares) at Mar. 31, 2023 | 93,140,000 | ||||
Balance at Dec. 31, 2023 | 859,337 | $ 9 | 5,304,623 | 918 | (4,446,213) |
Balance (in shares) at Dec. 31, 2023 | 93,732,000 | ||||
Exercise of options for common stock | 15,579 | 15,579 | |||
Exercise of options for common stock, shares | 207,000 | ||||
Vest of restricted stock units/awards | 461,000 | ||||
Partial settlement of capped call share options for 2024 Notes | 0 | ||||
Issuance of common stock under employee stock purchase plan | 6,561 | 6,561 | |||
Issuance of common stock under employee stock purchase plan, shares | 90,000 | ||||
Stock-based compensation | 45,205 | 45,205 | |||
Unrealized gains from available-for-sale securities, net of tax | (1,609) | (1,609) | |||
Net Income (Loss) | 36,119 | 36,119 | |||
Balance at Mar. 31, 2024 | $ 961,192 | $ 9 | $ 5,371,968 | $ (691) | $ (4,410,094) |
Balance (in shares) at Mar. 31, 2024 | 94,490,000 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | ||
Mar. 31, 2024 | Mar. 31, 2023 | ||
Cash flows from operating activities: | |||
Net income (loss) | $ 36,119 | $ (516,755) | |
Adjustments to reconcile net loss to cash flows from operating activities: | |||
Depreciation and amortization | 8,744 | 11,305 | |
Reduction in the carrying amounts of the right of use assets | 3,952 | 3,218 | |
Stock-based compensation | 40,692 | 41,250 | |
Accretion of investment discount, net | (12,401) | (8,651) | |
Loss on debt extinguishment | 0 | 387,329 | |
Non-cash change in the fair value of contingent consideration | 10,100 | 0 | |
Other | 329 | 1,964 | |
Changes in operating assets and liabilities, net: | |||
Net decrease (increase) in accounts receivable, net | 21,521 | (9,208) | |
Net increase in inventory | (60,512) | (306) | |
Net increase in manufacturing-related deposits and prepaids | (145,046) | (7,479) | |
Net increase in other assets | (2,315) | (22,517) | |
Net decrease in deferred revenue | (50,416) | (22,005) | |
Net decrease in accounts payable, accrued expenses, lease liabilities and other liabilities | (92,843) | (67,541) | |
Net cash used in operating activities | (242,076) | (209,396) | |
Cash flows from investing activities: | |||
Purchase of property and equipment | (32,444) | (9,485) | |
Purchase of available-for-sale securities | (216,450) | (426,763) | |
Maturity and sales of available-for-sale securities | 477,690 | 448,834 | |
Purchase of intangible assets | (10,000) | (94) | |
Net cash provided by investing activities | 218,796 | 12,492 | |
Cash flows from financing activities: | |||
Proceeds from exercise of stock options and purchase of stock under the Employee Stock Purchase Program | 22,140 | 28,037 | |
Partial settlement of capped call share options for 2024 Notes | 0 | 80,645 | |
Debt conversion costs for 2024 Notes | 0 | (6,887) | |
Net cash provided by financing activities | 22,140 | 101,795 | |
Decrease in cash, cash equivalents and restricted cash | (1,140) | (95,109) | |
Cash, cash equivalents and restricted cash: | |||
Beginning of period | 444,009 | 985,801 | |
End of period | 442,869 | 890,692 | |
Reconciliation of cash, cash equivalents and restricted cash: | |||
Total cash, cash equivalents and restricted cash | 442,869 | 890,692 | |
Cash and cash equivalents | 427,290 | 871,668 | |
Restricted cash in other assets | 15,579 | [1] | 19,024 |
Supplemental disclosure of cash flow information: | |||
Cash paid during the period for income taxes | 909 | 535 | |
Cash paid during the period for interest | 7,188 | 7,023 | |
Supplemental schedule of non-cash investing activities and financing activities: | |||
Intangible assets and property and equipment included in accounts payable and accrued expenses | 22,929 | 11,136 | |
Lease liabilities arising from obtaining right of use assets | 275 | 280 | |
Capitalized stock-based compensation and depreciation as inventory | 6,333 | ||
Common stock issued for exchange of 2024 Notes | $ 0 | $ 693,377 | |
[1] The Company had approximately $ 15.6 million in restricted cash included in other non-current assets on the Company ’s unaudited condensed consolidated balance sheets as of both March 31, 2024 and December 31, 2023. Restricted cash for both periods relates to (i) letters of credit established under the Company’s various property leases that serve as security for potential future default of lease payments, (ii) a letter of credit established under a certain commercial supply agreement and (iii) collateralized cash for the Company’ s credit cards. The restricted cash is unavailable for withdrawal or use for general obligations |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Pay vs Performance Disclosure | ||
Net Income (Loss) | $ 36,119 | $ (516,755) |
Insider Trading Arrangements
Insider Trading Arrangements | 3 Months Ended |
Mar. 31, 2024 shares | |
Trading Arrangements, by Individual | |
Material Terms of Trading Arrangement | Item 5. Other Information . During the three months ended March 31, 2024, two of our officers (as defined in Rule 16a-1(f) under the Exchange Act) entered into contracts, instructions or written plans for the purchase or sale of our securities that are intended to satisfy the conditions specified in Rule 10b5-1(c) under the Exchange Act for an affirmative defense against liability for trading in securities on the basis of material nonpublic information. We refer to these contracts, instructions, and written plans as " Rule 10b5-1 trading plans" and each one as a "Rule 10b5-1 trading plan." We describe the material terms of these trading plans below. Ryan Brown entered into and adopted a plan with an effective date of March 7, 2024 and end date of March 15, 2025 , covering 76,508 shares of common stock, 7,574 plus the net shares resulting upon the vesting of future restricted stock units, and 84,082 plus the net shares resulting upon the vesting of future restricted stock units. Bilal Arif entered into and adopted a plan with an effective date of March 11, 2024 and end date of March 15, 2025 , covering: 7,676 shares of common stock, 5,655 plus the net shares resulting upon the vesting of future restricted stock units, and 13,331 plus the net shares resulting upon the vesting of future restricted stock units. |
Rule 10b5-1 Arrangement Adopted | true |
Ryan Brown | |
Trading Arrangements, by Individual | |
Adoption Date | March 7, 2024 |
Termination Date | March 15, 2025 |
Aggregate Available | 84,082 |
Common Stock Covering Under Plan | 76,508 |
Common Stock Covering Resulting Upon The Vesting Of Future Restricted Stock | 7,574 |
Bilal Arif | |
Trading Arrangements, by Individual | |
Adoption Date | March 11, 2024 |
Termination Date | March 15, 2025 |
Aggregate Available | 13,331 |
Common Stock Covering Under Plan | 7,676 |
Common Stock Covering Resulting Upon The Vesting Of Future Restricted Stock | 5,655 |
ORGANIZATION AND NATURE OF BUSI
ORGANIZATION AND NATURE OF BUSINESS | 3 Months Ended |
Mar. 31, 2024 | |
Accounting Policies [Abstract] | |
ORGANIZATION AND NATURE OF BUSINESS | 1. ORGANIZATION AND NATURE OF BUSINESS Sarepta Therapeutics, Inc. (together with its wholly-owned subsidiaries, “Sarepta” or the “Company”) is a commercial-stage biopharmaceutical company focused on helping patients through the discovery and development of unique RNA-targeted therapeutics, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. Applying its proprietary, highly-differentiated and innovative technologies, and through collaborations with its strategic partners, the Company has developed multiple approved products for the treatment of Duchenne muscular dystrophy (“Duchenne”) and is developing potential therapeutic candidates for a broad range of diseases and disorders, including Duchenne, Limb-girdle muscular dystrophies (“LGMDs”) and other neuromuscular and central nervous system disorders. The Company's products in the U.S., EXONDYS 51 (eteplirsen) Injection (“EXONDYS 51”), VYONDYS 53 (golodirsen) Injection (“VYONDYS 53”), AMONDYS 45 (casimersen) Injection (“AMONDYS 45”) and ELEVIDYS, were granted accelerated approval by the U.S. Food and Drug Administration (the “FDA”) on September 19, 2016, December 12, 2019, February 25, 2021 and June 22, 2023, respectively. Indicated for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 51, exon 53 and exon 45 skipping, respectively, EXONDYS 51, VYONDYS 53 and AMONDYS 45 use the Company’s phosphorodiamidate morpholino oligomer (“PMO”) chemistry and exon-skipping technology to skip exon 51, exon 53 and exon 45 of the dystrophin gene. Exon skipping is intended to promote the production of an internally truncated but functional dystrophin protein. ELEVIDYS addresses the root genetic cause of Duchenne mutations in the dystrophin gene that result in the lack of dystrophin protein by delivering a gene that codes for a shortened form of dystrophin to muscle cells known as ELEVIDYS micro-dystrophin. ELEVIDYS is approved for the treatment of ambulatory pediatric patients aged four through five years with Duchenne. ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the Duchenne gene. As of March 31, 2024, the Company had approximately $ 1,440.3 million of cash, cash equivalents, restricted cash and investments, consisting of $ 427.3 million of cash and cash equivalents , $ 963.5 million of short-term investments, $ 33.9 million of non-current investments and $ 15.6 million of non-current restricted cash. The Company believes that its balance of cash, cash equivalents and investments as of the date of the issuance of this report is sufficient to fund its current operational plan for at least the next twelve months, though it may pursue additional cash resources through public or private debt and equity financings, seek funded research and development arrangements and additional government contracts and establish collaborations with or license its technology to other companies. |
SUMMARY OF SIGNIFICANT ACCOUNTI
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | 3 Months Ended |
Mar. 31, 2024 | |
Accounting Policies [Abstract] | |
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | 2. SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES Basis of Presentation The accompanying unaudited condensed consolidated financial statements, which have been prepared in accordance with accounting principles generally accepted in the United States (“U.S. GAAP”), reflect the accounts of Sarepta and its wholly-owned subsidiaries. All intercompany transactions between and among its consolidated subsidiaries have been eliminated. Management has determined that the Company operates in one segment: discovering, developing, manufacturing and delivering therapies to patients with rare diseases. In the opinion of the Company’s management, all adjustments of a normal recurring nature necessary for a fair presentation have been reflected. Certain financial information that is normally included in annual financial statements prepared in accordance with U.S. GAAP, but that is not required for interim reporting purposes, has been omitted. These unaudited condensed consolidated financial statements should be read in conjunction with the consolidated financial statements and related notes for the year ended December 31, 2023 which are contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, filed with the U.S. Securities and Exchange Commission on February 28, 2024. The results for the three months ended March 31, 2024 are not necessarily indicative of the results to be expected for the full year. Estimates and Uncertainties The preparation of the unaudited condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, equity, revenues, expenses and the disclosure of contingent assets and liabilities. Actual results could differ from those estimates. Concentration of Credit Risk Financial instruments that potentially subject the Company to concentrations of credit risk consist of cash held at financial institutions, cash equivalents, investments and accounts receivable, net, from customers. As of March 31, 2024, the Company’s cash was concentrated at three financial institutions, which potentially exposes the Company to credit risks. However, the Company does not believe that there is significant risk of non-performance by the financial institutions. The Company also purchases commercial paper, government and government agency bonds, corporate bonds and certificates of deposit issued by highly rated corporations, financial institutions and governments and limits the amount of credit exposure to any one issuer. These amounts may at times exceed federally insured limits. The Company has not experienced any credit losses related to these financial instruments and does not believe to be exposed to any significant credit risk related to these instruments. As of March 31, 2024, three entities accounted for 34 % , 22 % and 21 % of accounts receivable, net, respectively. As of December 31, 2023, four entities accounted for 40 % , 19 % , 19 % and 12 % of accounts receivable, net, respectively. As of March 31, 2024 , the Company believes its largest customers are of high credit quality and has not experienced any material credit losses related to such customers. Significant Accounting Policies For details about the Company's accounting policies, please read Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncements of the Annual Report on Form 10-K for the year ended December 31, 2023. There have not been any material changes to the Company's accounting policies or recent accounting pronouncements that could have a material impact through March 31, 2024 . |
LICENSE AND COLLABORATION AGREE
LICENSE AND COLLABORATION AGREEMENTS | 3 Months Ended |
Mar. 31, 2024 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
LICENSE AND COLLABORATION AGREEMENTS | 3. LICENSE AND COLLABORATION AGREEMENTS F. Hoffman-La Roche Ltd. For the three months ended March 31, 2024 and March 31, 2023, the Company recognized $ 48.0 million and $ 22.0 million, respectively, of collaboration revenue associated with the license, collaboration and option agreement (the “Roche Agreement”) with F. Hoffman-La Roche Ltd. (“Roche”). Under the Roche Agreement, the Company has granted Roche options to acquire ex-U.S. rights to certain future Duchenne development programs (the “Options”) in exchange for separate option exercise payments, milestone and royalty considerations, and cost-sharing provisions. These Options were accounted for as material rights related to individual programs and recorded in deferred revenue at the inception of the Roche Agreement. The value assigned to the material rights is included in deferred revenue until such rights expire or are exercised. On February 12, 2024, Roche declined to exercise its optional rights related to one external, early stage development program, which resulted in the immediate recognition of $ 48.0 million of collaboration revenue for the three months ended March 31, 2024. As of March 31, 2024 and December 31, 2023, the Company had total deferred revenue of $ 437.0 million and $ 485.0 million related to the separate material rights for the Options associated with the Roche Agreement, of which $ 112.0 million and $ 48.0 million are classified as current, respectively. The costs associated with co-development activities performed under the Roche Agreement are included in operating expenses, with any reimbursement of costs by Roche reflected as a reduction of such expenses when the related expense is incurred. For the three months ended March 31, 2024 and March 31, 2023, costs reimbursable by Roche and reflected as a reduction to operating expenses were $ 21.7 million and $ 20.3 million, respectively. As of March 31, 2024 and December 31, 2023, there was $ 30.1 million and $ 29.8 million of collaboration and other receivables included in other current assets on the unaudited condensed consolidated balance sheets. In accordance with the Roche Agreement, the Company agreed to enter into a supply agreement with Roche in order to supply them with clinical and commercial batches of ELEVIDYS (the “Supply Agreement”). While the Supply Agreement is in the process of being negotiated, the Company has delivered batches of commercial ELEVIDYS supply to Roche that were agreed upon on a purchase order-by-purchase order basis. For the three months ended March 31, 2024, the Company recognized $ 5.8 million of contract manufacturing revenue related to these shipments, with no similar activity for the three months ended March 31, 2023. This revenue is included in collaboration and other revenues in the unaudited condensed consolidated statements of comprehensive income (loss). For the three months ended March 31, 2024, the Company recognized $ 1.7 million of inventory costs related to these shipments, which is included in cost of sales (excluding amortization of in-licensed rights) in the unaudited condensed consolidated statements of comprehensive income (loss). Research and Option Agreements The Company has research and option agreements with third parties in order to develop various technologies and biologics that may be used in the administration of the Company’s genetic therapeutics. The agreements generally provide for research services related to pre-clinical development programs and options to license the technology for clinical development. Prior to the options under these agreements being executed, the Company may be required to make up to $ 34.8 million in research milestone payments. Under these agreements, there are $ 121.8 million in potential option payments to be made by the Company upon the determination to exercise the options. Additionally, if the options for each agreement are executed, the Company would incur additional contingent obligations and may be required to make development, regulatory, and sales milestone payments and royalty payments based on the net sales of the developed products upon commercialization. During the three months ended March 31, 2024 and March 31, 2023, the Company did not exercise any options nor have any additional research milestone payments become probable of occurring. Milestone Obligations The Company has license and collaboration agreements in place for which it could be obligated to pay, in addition to the payment of up-front fees upon execution of the agreements, certain milestone payments as a product candidate proceeds from the submission of an investigational new drug application through approval for commercial sale and beyond. As of March 31, 2024, the Company may be obligated to make up to $ 3.2 billion of future development, regulatory, commercial and up-front royalty payments associated with its collaboration and license agreements. These obligations exclude potential future option and milestone payments for options that have yet to be exercised within agreements entered into by the Company as of March 31, 2024, which are discussed above. For the three months ended March 31, 2023 , the Company recognized up-front, development milestone and other expenses of $ 0.5 million as research and development expense in the accompanying unaudited condensed consolidated statements of comprehensive income (loss), with no similar activity in the three months ended March 31, 2024 . |
FAIR VALUE MEASUREMENTS
FAIR VALUE MEASUREMENTS | 3 Months Ended |
Mar. 31, 2024 | |
Fair Value Disclosures [Abstract] | |
FAIR VALUE MEASUREMENTS | 4. FAIR VALUE MEASUREMENTS The Company has certain financial assets and liabilities that are recorded at fair value which have been classified as Level 1, 2 or 3 within the fair value hierarchy as described in the accounting standards for fair value measurements. • Level 1 — quoted prices for identical instruments in active markets; • Level 2 — quoted prices for similar instruments in active markets, quoted prices for identical or similar instruments in markets that are not active, and model-derived valuations in which all significant inputs and significant value drivers are observable in active markets; and • Level 3 — valuations derived from valuation techniques in which one or more significant value drivers are unobservable. During the three months ended March 31, 2024, there were no transfers into or out of Level 3. The tables below present information about the Company’s financial assets and liabilities that are measured and carried at fair value and indicate the level within the fair value hierarchy of the valuation techniques it utilizes to determine such fair value: Fair Value Measurement as of March 31, 2024 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 277,870 $ 277,870 $ — $ — Commercial paper 43,354 — 43,354 — Government and government agency bonds 780,997 — 780,997 — Corporate bonds 118,158 — 118,158 — Strategic investments 7,457 6,457 — 1,000 Certificates of deposit 54,863 — 54,863 — Total assets $ 1,282,699 $ 284,327 $ 997,372 $ 1,000 Liabilities Contingent consideration $ 48,200 $ — $ — $ 48,200 Total liabilities $ 48,200 $ — $ — $ 48,200 Fair Value Measurement as of December 31, 2023 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 63,919 $ 63,919 $ — $ — Commercial paper 113,362 — 113,362 — Government and government agency bonds 1,001,137 — 1,001,137 — Corporate bonds 130,380 — 130,380 — Strategic investments 6,527 5,527 — 1,000 Certificates of deposit 56,621 — 56,621 — Total assets $ 1,371,946 $ 69,446 $ 1,301,500 $ 1,000 Liabilities Contingent consideration $ 38,100 $ — $ — $ 38,100 Total liabilities $ 38,100 $ — $ — $ 38,100 The Company’s assets with a fair value categorized as Level 1 within the fair value hierarchy include money market funds and the Company's strategic investment in a biotechnology company listed on the Nasdaq Global Market. The Company's assets with a fair value categorized as Level 2 within the fair value hierarchy consist of commercial paper, government and government agency bonds, corporate bonds and certificates of deposit. These assets have been initially valued at the transaction price and subsequently valued at the end of each reporting period utilizing third-party pricing services. The Company uses observable market inputs to determine value, which primarily consist of reportable trades. The Company’s assets with a fair value categorized as Level 3 within the fair value hierarchy consist of a strategic investment in a private biotechnology company whose fair value measurement was based upon significant inputs not observable in the market and therefore represented a Level 3 measurement. At the end of each reporting period, the fair value of the Company's strategic investments, that are not listed securities are adjusted if the issuers were to issue similar or identical securities or when there is a triggering event for impairment. There were no valuation measurement events related to the fair value of the Company's Level 3 strategic investments during the three months ended March 31, 2024 or 2023, as no impairment indicators were identified nor were similar securities issued. The Company’s contingent consideration liability with a fair value categorized as Level 3 within the fair value hierarchy relates to the regulatory-related contingent payments to Myonexus Therapeutics, Inc. (“Myonexus”) selling shareholders as well as to two academic institutions under separate license agreements that meet the definition of a derivative. For more information related to Myonexus, please read Note 3, License and Collaboration Agreements to the financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 . The contingent consideration liability was estimated using an income approach based on the probability-weighted expected cash flows that incorporated industry-based probability adjusted assumptions relating to the achievement of the milestone and thus the likelihood of making the payments. This fair value measurement was based upon significant inputs not observable in the market and therefore represented a Level 3 measurement. Significant changes which increase or decrease the probabilities of achieving the milestone, or shorten or lengthen the time required to achieve the milestone, would result in a corresponding increase or decrease in the fair value of the liability. At the end of each reporting period, the fair value is adjusted to reflect the most current assumptions through earnings. The following table represents a roll-forward of the fair value of Level 3 financial liabilities for the period indicated: As of (in thousands) Fair value, as of December 31, 2023 $ 38,100 Change in estimated fair value 10,100 Fair value, as of March 31, 2024 $ 48,200 For the three months ended March 31, 2024, the Company recorded an increase of $ 10.1 millio n to account for the change in fair value of the Company's existing contingent consideration liabilities. These changes, which are recorded through earnings, were a result of updates made to certain inputs and assumptions impacting the probability-weighted expected cash flows, principally the probability of success of the underlying programs and the discount rate utilized to determine the present value of future payments to be made. As of March 31, 2024, the contingent consideration was recorded as a long-term liability on the Company's unaudited condensed consolidated balance sheets. For the three months ended March 31, 2023, there were no changes to the fair value of the contingent consideration liability. The fair value of the senior notes due on November 15, 2024 (the “2024 Notes”) and September 15, 2027 (the “2027 Notes”) is based on open market trades and is classified as Level 1 in the fair value hierarchy. The following table summarizes the carrying value and fair value of the Company's convertible notes for each of the period indicated: As of As of (in thousands) Net carrying value of the 2024 Notes $ 105,586 $ 105,483 Net carrying value of the 2027 Notes 1,133,660 1,132,515 Total carrying value of debt facilities $ 1,239,246 $ 1,237,998 Fair value of 2024 Notes $ 188,867 $ 144,833 Fair value of 2027 Notes 1,334,472 1,172,276 Total fair value of debt facilities $ 1,523,339 $ 1,317,109 The carrying amounts reported in the unaudited condensed consolidated balance sheets for cash and cash equivalents, accounts receivable, net and accounts payable approximated fair value because of the immediate or short-term maturity of these financial instruments |
CASH, CASH EQUIVALENTS AND MARK
CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES | 3 Months Ended |
Mar. 31, 2024 | |
Cash and Cash Equivalents [Abstract] | |
CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES | 5. CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES The following table summarizes the Company’s financial assets with maturities of less than 90 days from the date of purchase included in cash equivalents in the unaudited condensed consolidated balance sheets for each of the periods indicated: As of As of (in thousands) Money market funds $ 277,870 $ 63,919 Commercial paper — 53,680 Total $ 277,870 $ 117,599 It is the Company’s policy to mitigate credit risk in its financial assets by maintaining a well-diversified portfolio that limits the amount of exposure as to maturity and investment type. The weighted average maturity of the Company's available-for-sale securities as of March 31, 2024 and December 31, 2023 was approximately six and five months , respectively. The following tables summarize the Company ’s cash, cash equivalents, short-term investments and non-current investments for each of the periods indicated: As of March 31, 2024 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 427,290 $ — $ — $ 427,290 Commercial paper 43,354 — — 43,354 Government and government agency bonds 781,698 59 ( 760 ) 780,997 Corporate bonds 118,121 57 ( 20 ) 118,158 Certificates of deposit 54,863 — — 54,863 Total cash, cash equivalents and investments $ 1,425,326 $ 116 $ ( 780 ) $ 1,424,662 As reported: Cash and cash equivalents $ 427,290 $ — $ — $ 427,290 Short-term investments 963,987 116 ( 650 ) 963,453 Other non-current assets 34,049 — ( 130 ) 33,919 Total cash, cash equivalents and investments $ 1,425,326 $ 116 $ ( 780 ) $ 1,424,662 As of December 31, 2023 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 374,750 $ — $ — $ 374,750 Commercial paper 113,362 — — 113,362 Government and government agency bonds 1,000,302 1,006 ( 171 ) 1,001,137 Corporate bonds 130,270 118 ( 8 ) 130,380 Certificates of deposit 56,621 — — 56,621 Total cash, cash equivalents and investments $ 1,675,305 $ 1,124 $ ( 179 ) $ 1,676,250 As reported: Cash and cash equivalents $ 428,430 $ — $ — $ 428,430 Short-term investments 1,246,875 1,124 ( 179 ) 1,247,820 Total cash, cash equivalents and investments $ 1,675,305 $ 1,124 $ ( 179 ) $ 1,676,250 |
PRODUCT REVENUES, NET, ACCOUNTS
PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE, NET, AND RESERVES FOR PRODUCT REVENUES | 3 Months Ended |
Mar. 31, 2024 | |
Receivables, Net, Current [Abstract] | |
PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT REVENUES | 6. PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE, NET, AND RESERVES FOR PRODUCT REVENUES Net product revenues, which includes revenues associated with EXONDYS 51, AMONDYS 45 and VYONDYS 53 (collectively, the “PMO Products”) and ELEVIDYS consisted of the following: For the Three Months Ended 2024 2023 (in thousands) PMO Products United States $ 189,667 $ 200,451 Rest of World 35,881 31,044 Total PMO product revenues, net $ 225,548 $ 231,495 ELEVIDYS United States 133,936 — Total ELEVIDYS product revenue, net $ 133,936 $ — Total product revenues, net $ 359,484 $ 231,495 For the three months ended March 31, 2024 and 2023, no individual country outside the U.S. exceeded 10 % of total net product revenues. The following table summarizes the Company's net product revenues, by customer, for those customers that exceeded 10 % for the periods indicated: For the Three Months Ended 2024 2023 Product revenues, net Customer 1 36 % 49 % Customer 2 20 % 32 % As of March 31, 2024 and December 31, 2023, the Company's accounts receivable, net were $ 378.8 million and $ 400.3 million, respectiv ely, both of which were related to product sales, net of discounts and allowances. As of March 31, 2024 , the majority of the Company’s accounts receivable arose from product sales in the U.S. and all customers have standard payment terms that generally require payment within 65 to 90 days. Outside of the U.S., the majority of the Company’s customers have payment terms ranging between 90 and 150 days. Please refer to Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncements for discussion of the credit risk associated with accounts receivable, net. The following tables summarize an analysis of the change in reserves for discounts and allowances for each of the periods indicated: Chargebacks Rebates Prompt Pay Other Accruals Total (in thousands) Balance, as of December 31, 2023 $ 27,486 $ 98,194 $ 3,831 $ 35,261 $ 164,772 Provision 20,466 35,951 4,381 18,965 79,763 Adjustments relating to prior periods 783 ( 2,532 ) — — ( 1,749 ) Payments/credits ( 30,358 ) ( 36,623 ) ( 3,685 ) ( 16,220 ) ( 86,886 ) Balance, as of March 31, 2024 $ 18,377 $ 94,990 $ 4,527 $ 38,006 $ 155,900 Chargebacks Rebates Prompt Pay Other Accruals Total (in thousands) Balance, as of December 31, 2022 $ 417 $ 67,493 $ 3,343 $ 23,445 $ 94,698 Provision 2,827 30,488 3,572 14,567 51,454 Adjustments relating to prior periods — ( 2,162 ) — — ( 2,162 ) Payments/credits ( 3,244 ) ( 28,025 ) ( 3,389 ) ( 14,040 ) ( 48,698 ) Balance, as of March 31, 2023 $ — $ 67,794 $ 3,526 $ 23,972 $ 95,292 The following table summarizes the total reserves above included in the Company’s unaudited condensed consolidated balance sheets for each of the periods indicated: As of As of (in thousands) Reduction to accounts receivable $ 58,684 $ 64,697 Component of accrued expenses 97,216 100,075 Total reserves $ 155,900 $ 164,772 |
INVENTORY
INVENTORY | 3 Months Ended |
Mar. 31, 2024 | |
Inventory Disclosure [Abstract] | |
INVENTORY | 7. INVENTORY The following table summarizes the components of the Company’s inventory for each of the periods indicated: As of As of (in thousands) Raw materials $ 150,011 $ 133,963 Work in progress 391,558 318,458 Finished goods 39,503 61,806 Total inventory $ 581,072 $ 514,227 No material inventory reserves existed as of March 31, 2024 or December 31, 2023. The Company classifies inventory associated with its PMO Products as non-current inventory when consumption of the inventory is expected beyond the Company's normal PMO Product inventory operating cycle of two years. Non-current inventory consists of raw materials and work in progress associated with the PMO Products. The following table summarizes the balance sheet classification of the Company's inventory for each of the periods indicated: As of As of (in thousands) Balance sheet classification Inventory $ 373,530 $ 322,859 Non-current inventory 207,542 191,368 Total inventory $ 581,072 $ 514,227 |
OTHER ASSETS
OTHER ASSETS | 3 Months Ended |
Mar. 31, 2024 | |
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | |
OTHER ASSETS | 8. OTHER ASSETS The following table summarizes the Company’s other current assets for each of the periods indicated: As of As of (in thousands) Collaboration and other receivables $ 30,092 $ 29,786 Prepaid maintenance services 15,907 11,281 Prepaid clinical and pre-clinical expenses 11,552 10,280 Tax-related receivables and prepaids 7,274 6,862 Prepaid commercial expenses 3,231 2,729 Interest receivable 3,209 2,731 Prepaid insurance 1,952 3,352 Prepaid research expenses 1,672 2,512 Other 8,076 8,181 Total other current assets $ 82,965 $ 77,714 The following table summarizes the Company’s other non-current assets for each of the periods indicated: As of As of (in thousands) Manufacturing-related deposits and prepaids $ 82,610 $ 74,204 Non-current investments 33,919 — Intangible assets, net 28,930 29,620 Restricted cash* 15,579 15,579 Strategic investments 7,457 6,527 Prepaid maintenance services 4,569 5,466 Other 3,343 5,375 Total other non-current assets $ 176,407 $ 136,771 * The Company had approximately $ 15.6 million in restricted cash included in other non-current assets on the Company ’s unaudited condensed consolidated balance sheets as of both March 31, 2024 and December 31, 2023. Restricted cash for both periods relates to (i) letters of credit established under the Company’s various property leases that serve as security for potential future default of lease payments, (ii) a letter of credit established under a certain commercial supply agreement and (iii) collateralized cash for the Company’ s credit cards. The restricted cash is unavailable for withdrawal or use for general obligations . |
ACCRUED EXPENSES
ACCRUED EXPENSES | 3 Months Ended |
Mar. 31, 2024 | |
Payables and Accruals [Abstract] | |
ACCRUED EXPENSES | 9. ACCRUED EXPENSES The following table summarizes the Company’s accrued expenses for each of the periods indicated: As of As of (in thousands) Product revenue related reserves $ 97,216 $ 100,075 Accrued contract manufacturing costs 45,057 33,024 Accrued clinical and pre-clinical costs 36,078 34,669 Accrued employee compensation costs 32,112 78,732 Accrued professional fees 21,014 17,187 Accrued income taxes 18,292 13,766 Accrued fixed assets 11,587 3,231 Accrued royalties 11,206 12,070 Accrued research expenses 2,873 2,441 Accrued milestone and license expenses 2,000 11,375 Other 5,882 8,427 Total accrued expenses $ 283,317 $ 314,997 |
STOCK-BASED COMPENSATION
STOCK-BASED COMPENSATION | 3 Months Ended |
Mar. 31, 2024 | |
Share-Based Payment Arrangement [Abstract] | |
STOCK-BASED COMPENSATION | 10. STOCK-BASED COMPENSATION The following table summarizes the Company’s stock awards granted for each of the periods indicated: For the Three Months Ended March 31, 2024 2023 Grants Weighted Grants Weighted Stock options 316,378 $ 67.56 1,030,349 $ 72.80 Restricted stock units 1,161,985 (1) $ 128.58 1,075,911 (2) $ 154.91 (1) Included in restricted stock units (“RSUs”) for the three months ended March 31, 2024 are 88,600 shares with performance conditions, which are related to the achievement of certain financial performance goals, regulatory approval of certain of the Company's product candidates, and regulatory expansion of certain of the Company's commercial products. As of March 31, 2024, none of the performance conditions were probable of being achieved. If the performance milestones are achieved within the required time frame, the Company may recognize up t o $ 14.3 million of stock-based compensation related to these grants. Stock options and the remaining RSUs granted during the three months ended March 31, 2024 have only service-based criteria and vest over four years. (2) Included in the RSUs for the three months ended March 31, 2023 are 502,255 shares with performance conditions (the “March 2023 PSUs”) which are related to regulatory approval of certain of the Company's product candidates and achievement of a certain financial performance target. The Company recorded stock-based compensation expense of $ 8.9 million associated with the March 2023 PSUs during the three months ended March 31, 2024, with no similar expense recorded during the three months ended March 31, 2023 . Vesting of the March 2023 PSUs is contingent on the fulfillment of remaining service conditions. Stock-based Compensation Expense For the three months ended March 31, 2024 and 2023 , total stock-based compensation expense included in total expenses was $ 40.7 million and $ 41.3 million, respectively. The following table summarizes stock-based compensation expense by grant type and by function included within the unaudited condensed consolidated statements of comprehensive income (loss): For the Three Months Ended 2024 2023 (in thousands) Stock options $ 16,281 $ 23,830 Restricted stock units 27,527 16,140 Employee stock purchase plan 1,397 1,280 Subtotal $ 45,205 $ 41,250 Capitalized stock-based compensation costs* ( 4,513 ) — Total stock-based compensation expense included in expenses $ 40,692 $ 41,250 Research and development $ 16,273 $ 16,413 Selling, general and administrative 24,419 24,837 Total stock-based compensation expense included in expenses $ 40,692 $ 41,250 *Prior to the three months ended March 31, 2024 , capitalized stock-based compensation costs were not material. |
OTHER LOSS, NET
OTHER LOSS, NET | 3 Months Ended |
Mar. 31, 2024 | |
Other Income and Expenses [Abstract] | |
OTHER LOSS, NET | 11. OTHER INCOME (LOSS), NET The following table summarizes other income (loss), net f or each of the periods indicated: For the Three Months Ended 2024 2023 (in thousands) Accretion of investment discount, net $ 12,804 $ 10,039 Interest income 7,093 9,276 Interest expense ( 4,166 ) ( 6,323 ) Change in the fair value of contingent consideration ( 10,100 ) — Other, net 912 ( 285 ) Other income, net $ 6,543 $ 12,707 Loss on debt extinguishment — ( 387,329 ) Total other income (loss), net $ 6,543 $ ( 374,622 ) |
INCOME TAXES
INCOME TAXES | 3 Months Ended |
Mar. 31, 2024 | |
Income Tax Disclosure [Abstract] | |
INCOME TAXES | 12. INCOME TAXES During the three months ended March 31, 2024, the Company recorded an income tax provision of $ 5.3 million, representing an effective tax rate of 14.2 % . The Company continues to maintain a valuation allowance on its worldwide deferred tax assets with the exception of deferred tax assets in certain foreign jurisdictions, as it has concluded that it is more likely than not that these deferred tax assets will not be realized. The effective tax rate for the three months ended March 31, 2024 was less than the statutory tax rate of 21 % , principally due to the release of valuation allowance against certain tax attribute carryforwards utilized to offset current year income. During the three months ended March 31, 2023, the Company recorded an income tax provision of $ 4.0 million, representing an effective tax rate of ( 0.8 %) . For tax years beginning on or after January 1, 2022, the Tax Cuts and Jobs Act of 2017 eliminates the option to currently deduct research and development expenses and requires taxpayers to capitalize and amortize the costs over five years for research activities performed in the U.S. and 15 years for research activities performed outside the U.S. The requirement to capitalize research and development costs for tax purposes resulted in the Company having taxable profits and recording federal and state tax expense of $ 0.9 million and $ 3.1 million, respectively. The state tax expense of $ 3.1 million is primarily related to state taxable income, partially offset by net operating loss carryforwards. |
Earnings (Loss) Per Share
Earnings (Loss) Per Share | 3 Months Ended |
Mar. 31, 2024 | |
Earnings Per Share [Abstract] | |
Earnings (Loss) Per Share | 13. EARNINGS (LOSS) PER SHARE Basic earnings per share is computed by dividing net income by the weighted-average number of shares of common stock outstanding. Diluted earnings per share is computed by dividing net income by the weighted-average number of shares of common stock and dilutive common stock equivalents outstanding. The following table sets forth the computation of basic and diluted earnings (loss) per common share: For the Three Months Ended 2024 2023 (in thousands, except per share amounts) Numerator: Net income (loss) - basic $ 36,119 $ ( 516,755 ) Add: interest expense, net of tax, on the Company's convertible debt 429 — Net income (loss) - diluted $ 36,548 $ ( 516,755 ) Denominator: Weighted-average common shares outstanding, basic 93,991 88,186 Effect of dilutive securities: Common stock issuable under the Company's equity incentive plans 3,681 — Common stock issuable under the Company's convertible debt 1,442 — Weighted-average common shares outstanding, diluted 99,114 88,186 Earnings (loss) per common share, basic $ 0.38 $ ( 5.86 ) Earnings (loss) per common share, diluted $ 0.37 $ ( 5.86 ) The following table summarizes potential shares of common stock that were excluded from the computation of diluted earnings per share as they were anti-dilutive: For the Three Months Ended 2024 2023 (in thousands) Common stock issuable under the Company's equity incentive plans 4,270 12,553 Common stock issuable under the Company's convertible debt 8,100 9,542 Total number of potentially issuable common stock 12,370 22,095 |
COMMITMENTS AND CONTINGENCIES
COMMITMENTS AND CONTINGENCIES | 3 Months Ended |
Mar. 31, 2024 | |
Commitments and Contingencies Disclosure [Abstract] | |
COMMITMENTS AND CONTINGENCIES | 14. COMMITMENTS AND CONTINGENCIES Manufacturing Obligations The following table summarizes the aggregate non-cancelable contractual obligations arising from the Company’s manufacturing obligations: As of (in thousands) 2024 (April-December) $ 836,030 2025 165,293 2026 114,785 2027 77,645 2028 72,346 Total manufacturing commitments* $ 1,266,099 * Total manufacturing commitments include the Catalent, Inc. ( “Catalent”) manufacturing and supply agreement, for which the Company has ROU assets and lease liabilities recorded on the unaudited condensed consolidated balance sheets as of March 31, 2024. For more information, please read Note 21, Commitments and Contingencies to the financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 . Thermo Fisher Scientific, Inc. The Company entered into a development, commercial manufacturing, and supply agreement in June 2018 and, subsequently, entered into the first, second and third amendments in May 2019, July 2020 and October 2021, respectively, with Brammer Bio MA, LLC, an affiliate of Thermo Fisher Scientific, Inc. (“Thermo”) (collectively, the “Thermo Agreement”). In March 2023, the Company executed a fourth amendment (the “Amendment”) that modified the terms of the Thermo Agreement. The Amendment removed the previous minimum batch purchase commitment of $ 54.7 million per annum and associated fee for the remaining term of the Thermo Agreement. In connection with the elimination of such commitment and fee, the Amendment implemented a fee of up to $ 60.0 million, to be paid in three installments of $ 20.0 million each by March 1, 2024, December 31, 2024 and December 31, 2025, respectively, unless waived in part as described below. During the three months ended March 31, 2024 , the Company paid the first $ 20.0 million installment due March 1, 2024 . As the Company has yet to obtain regulatory approval to produce commercial supply of ELEVIDYS at Thermo manufacturing facilities as of March 31, 2024 , it recognized approximately $ 0.9 million as research and development expense during the three months ended March 31, 2024 related to this nonrefundable advance payment. As of March 31, 2024, the Company recorded the total remaining nonrefundable advance payment of $ 16.5 million in other current assets and other non-current assets on the unaudited condensed consolidated balance sheets. The second and third payment installments, which are associated with the years ending December 31, 2024 and 2025, will be waived if the Company meets certain minimum purchase thresholds under the Amendment. As of March 31, 2024, the Company believes it is probable that the minimum purchase thresholds will be met in the normal course of business throughout the term of the agreement and, therefore, no liabilities were recorded related to the second or third payment installments. For more information related to Thermo, please read Note 21, Commitments and Contingencies to the financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 . Litigation In the normal course of business, the Company from time to time is named as a party to various legal claims, actions and complaints, which have included and may include matters involving securities, employment, intellectual property, arising from the use of therapeutics utilizing its technology, or others. The Company records a loss contingency reserve for a legal proceeding when it considers the potential loss probable and it can reasonably estimate the amount of the loss or determine a probable range of loss. The Company provides disclosure when it considers a loss reasonably possible or when it determines that a loss in excess of a reserve is reasonably possible. The Company provides an estimate of such reasonably possible losses or an aggregate range of such reasonably possible losses, unless the Company believes that such an estimate cannot be made. The Company has not recorded any material accruals for loss contingencies, and in management's opinion, no material range of loss is estimable for the matters described below as of March 31, 2024. On September 15, 2020, REGENXBIO INC. (“Regenx”) and the Trustees of the University of Pennsylvania (“U-Penn”) filed a lawsuit against the Company and Sarepta Therapeutics Three, LLC, in the U.S. District Court for the District of Delaware. The plaintiffs assert patent infringement of U.S. Patent No. 10,526,617 (“the ‘617 Patent”) under 35 U.S.C.§§ 271(a)-(c) based on Sarepta’s alleged direct or indirect manufacture and use of the patented cultured host cell technology allegedly used to make adeno-associated virus (“AAV”) gene therapy products, including SRP-9001 (approved June 22, 2023 in the U.S. as ELEVIDYS®). Specifically, the Complaint essentially includes the allegation that Sarepta’s use, and the use by its contract manufacturers on its behalf, of a host cell containing a recombinant acid molecule that encodes a capsid protein having at least 95% amino acid identity to AAVrh10 infringes the ‘617 Patent asserted by Regenx. Plaintiffs seek injunctive relief, a judgment of infringement and willful infringement, damages that are no less than a reasonable royalty (treble damages), attorneys’ fees and costs, and such other relief as the court deems just and proper. On January 5, 2024, the Court granted Sarepta’s motion for summary judgment on the grounds that the asserted claims of Regenx’s ‘617 Patent are invalid because they cover patent-ineligible subject matter under 35 U.S.C. § 101. On January 12, 2024, the Court entered judgment and closed the case and Plaintiffs filed a notice of appeal to the U.S. Court of Appeals for the Federal Circuit. On June 20, 2023, Regenx and U-Penn commenced a second patent infringement lawsuit against Sarepta and its contract manufacturer, Catalent asserting patent alleged infringement of U. S. Patent No. 11,680,274 (“the ’274 Patent”). In the second lawsuit, Regenx and U-Penn allege that Sarepta and Catalent’s manufacture, use and commercial launch of ELEVIDYS® (SRP-9001) infringe the ’274 Patent. Sarepta answered the complaint on August 10, 2023, and a case schedule has been set with a trial commencing on November 17, 2025. On February 21, 2024, Sarepta submitted a petition for Inter Partes Review ("IPR") for filing with the Patent Trial and Appeal Board ("PTAB") at the USPTO. The petition seeks to invalidate the ‘274 Patent. On March 20, 2024, the district court stayed the litigation pending the PTAB’s determination on the IPR. On July 13, 2021, Nippon Shinyaku Co., Ltd. (“Nippon Shinyaku” or “NS”) filed a lawsuit against the Company in the U.S. District Court for the District of Delaware. NS asserts a claim for breach of contract arising from Sarepta filing seven petitions for Inter Partes Review (“IPR Petitions”) with the Patent Trial and Appeal Board at the USPTO in which Sarepta sought to invalidate certain NS patents concerning exon 53 skipping technology (U.S. Patent Nos. 9,708,361, 10,385,092, 10,407,461, 10,487,106, 10,647,741, 10,662,217, and 10,683,322, respectively, and collectively the “NS Patents”). In addition, NS asserts claims for patent infringement and willful infringement of each of the NS Patents allegedly arising from Sarepta’s activities, including the sale of, its exon 53 skipping product, VYONDYS 53 (golodirsen). NS further seeks a determination of non-infringement by NS alleged to arise from NS’s activities, including the sale of, its exon 53 skipping product, Viltepso (viltolarsen) and invalidity of certain patents licensed to the Company from the University of Western Australia ("UWA") (U.S. Patent Nos. 9,994,851, 10,227,590, and 10,266,827, collectively the “UWA Patents”). In its complaint, NS is seeking legal fees and costs, an unspecified amount of monetary relief (treble damages) attributed to Sarepta’s alleged infringement, and such other relief as the court deems just and proper. In January 2022, the PTAB granted institution of all claims of all NS Patents in response to Sarepta’s IPR Petitions and determined that Sarepta has demonstrated a reasonable likelihood of success in proving that the NS Patents are unpatentable. NS filed a motion for preliminary injunction solely seeking Sarepta’s withdrawal of the IPR Petitions, which was ultimately granted after the U.S. Court of Appeals for the Federal Circuit reversed and remanded to the district court on February 8, 2022. Sarepta subsequently withdrew the IPRs, which were terminated on June 14, 2022. On December 27, 2021, the district court partially granted and denied the motion to dismiss by Sarepta and ordered NS to file a Second Amended Complaint (“SAC”), which it did on January 14, 2022. In the SAC, NS maintains all claims of the original complaint of July 13, 2021, except a determination of non-infringement of the UWA Patents. On January 28, 2022, Sarepta filed its answer to the SAC, with defenses and counterclaims against NS and NS Pharma Inc. that include infringement of the UWA Patents allegedly arising from their activities concerning, including the sale of, its exon 53 skipping product, Viltepso (viltolarsen) and breach of contract. Sarepta is also seeking a determination of invalidity of the NS Patents. In its counterclaim complaint, Sarepta is seeking an award of relief in its defenses to NS’ allegations, a judgment of breach of contract, a determination of invalidity of the NS Patents, a judgment of infringement and willful infringement of the UWA Patents, legal fees and costs, an unspecified amount of monetary relief (treble damages) attributable to NS’ alleged infringement, and such other relief as the court deems just and proper. UWA has since been joined as a Plaintiff in Sarepta’s counterclaims against NS. On August 14, 2023, the Court granted cross motions to amend the pleadings, allowing Sarepta to add a counterclaim against NS for inequitable conduct, and NS to add counterclaims against Sarepta for inequitable conduct and Walker Process fraud. The parties have since stipulated to the dismissal of certain claims and defenses, including (1) NS’s claims of infringement of the ’361 Patent and claims 5 and 10 of the ’322 Patent; and (2) Sarepta’s non-infringement defenses to the remainder of the asserted NS Patents, except to the non-withdrawn claims of the ‘322 patent. Briefing on the parties’ summary judgment and Daubert motions was completed on January 26, 2024. On April 18, 2024, the Court bifurcated the Walker Process fraud claim. A trial is scheduled to commence on May 13, 2024. On or about June 5, 2023, Sarepta initiated a patent infringement lawsuit against Nippon Shinyaku in Japan, alleging that NS’s production, sales and offers to sell Viltepso infringe Sarepta’s Japanese Patent No. 6406782. NS filed its preliminary answer on July 13, 2023. A final hearing is set for July 25, 2024. |
SUMMARY OF SIGNIFICANT ACCOUN_2
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies) | 3 Months Ended |
Mar. 31, 2024 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying unaudited condensed consolidated financial statements, which have been prepared in accordance with accounting principles generally accepted in the United States (“U.S. GAAP”), reflect the accounts of Sarepta and its wholly-owned subsidiaries. All intercompany transactions between and among its consolidated subsidiaries have been eliminated. Management has determined that the Company operates in one segment: discovering, developing, manufacturing and delivering therapies to patients with rare diseases. In the opinion of the Company’s management, all adjustments of a normal recurring nature necessary for a fair presentation have been reflected. Certain financial information that is normally included in annual financial statements prepared in accordance with U.S. GAAP, but that is not required for interim reporting purposes, has been omitted. These unaudited condensed consolidated financial statements should be read in conjunction with the consolidated financial statements and related notes for the year ended December 31, 2023 which are contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, filed with the U.S. Securities and Exchange Commission on February 28, 2024. The results for the three months ended March 31, 2024 are not necessarily indicative of the results to be expected for the full year. |
Estimates and Uncertainties | Estimates and Uncertainties The preparation of the unaudited condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, equity, revenues, expenses and the disclosure of contingent assets and liabilities. Actual results could differ from those estimates. |
Concentration of Credit Risk | Concentration of Credit Risk Financial instruments that potentially subject the Company to concentrations of credit risk consist of cash held at financial institutions, cash equivalents, investments and accounts receivable, net, from customers. As of March 31, 2024, the Company’s cash was concentrated at three financial institutions, which potentially exposes the Company to credit risks. However, the Company does not believe that there is significant risk of non-performance by the financial institutions. The Company also purchases commercial paper, government and government agency bonds, corporate bonds and certificates of deposit issued by highly rated corporations, financial institutions and governments and limits the amount of credit exposure to any one issuer. These amounts may at times exceed federally insured limits. The Company has not experienced any credit losses related to these financial instruments and does not believe to be exposed to any significant credit risk related to these instruments. As of March 31, 2024, three entities accounted for 34 % , 22 % and 21 % of accounts receivable, net, respectively. As of December 31, 2023, four entities accounted for 40 % , 19 % , 19 % and 12 % of accounts receivable, net, respectively. As of March 31, 2024 , the Company believes its largest customers are of high credit quality and has not experienced any material credit losses related to such customers. |
Significant Accounting Policies | Significant Accounting Policies For details about the Company's accounting policies, please read Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncements of the Annual Report on Form 10-K for the year ended December 31, 2023. There have not been any material changes to the Company's accounting policies or recent accounting pronouncements that could have a material impact through March 31, 2024 . |
FAIR VALUE MEASUREMENTS (Tables
FAIR VALUE MEASUREMENTS (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Fair Value Disclosures [Abstract] | |
Assets and Liabilities Measured and Carried at Fair Value | The tables below present information about the Company’s financial assets and liabilities that are measured and carried at fair value and indicate the level within the fair value hierarchy of the valuation techniques it utilizes to determine such fair value: Fair Value Measurement as of March 31, 2024 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 277,870 $ 277,870 $ — $ — Commercial paper 43,354 — 43,354 — Government and government agency bonds 780,997 — 780,997 — Corporate bonds 118,158 — 118,158 — Strategic investments 7,457 6,457 — 1,000 Certificates of deposit 54,863 — 54,863 — Total assets $ 1,282,699 $ 284,327 $ 997,372 $ 1,000 Liabilities Contingent consideration $ 48,200 $ — $ — $ 48,200 Total liabilities $ 48,200 $ — $ — $ 48,200 Fair Value Measurement as of December 31, 2023 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 63,919 $ 63,919 $ — $ — Commercial paper 113,362 — 113,362 — Government and government agency bonds 1,001,137 — 1,001,137 — Corporate bonds 130,380 — 130,380 — Strategic investments 6,527 5,527 — 1,000 Certificates of deposit 56,621 — 56,621 — Total assets $ 1,371,946 $ 69,446 $ 1,301,500 $ 1,000 Liabilities Contingent consideration $ 38,100 $ — $ — $ 38,100 Total liabilities $ 38,100 $ — $ — $ 38,100 |
Summary of Fair Value of Level 3 Financial Liabilities | The following table represents a roll-forward of the fair value of Level 3 financial liabilities for the period indicated: As of (in thousands) Fair value, as of December 31, 2023 $ 38,100 Change in estimated fair value 10,100 Fair value, as of March 31, 2024 $ 48,200 |
Summary of Carrying Value and Fair Value of the Company's Convertible Notes | The following table summarizes the carrying value and fair value of the Company's convertible notes for each of the period indicated: As of As of (in thousands) Net carrying value of the 2024 Notes $ 105,586 $ 105,483 Net carrying value of the 2027 Notes 1,133,660 1,132,515 Total carrying value of debt facilities $ 1,239,246 $ 1,237,998 Fair value of 2024 Notes $ 188,867 $ 144,833 Fair value of 2027 Notes 1,334,472 1,172,276 Total fair value of debt facilities $ 1,523,339 $ 1,317,109 |
CASH, CASH EQUIVALENTS AND MA_2
CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Cash and Cash Equivalents [Abstract] | |
Summary of Company Financial Assets with Maturities of Less Than 90 Days Included in Cash Equivalents | The following table summarizes the Company’s financial assets with maturities of less than 90 days from the date of purchase included in cash equivalents in the unaudited condensed consolidated balance sheets for each of the periods indicated: As of As of (in thousands) Money market funds $ 277,870 $ 63,919 Commercial paper — 53,680 Total $ 277,870 $ 117,599 |
Summary of Company Cash, Cash Equivalents and Short-Term Investments | The following tables summarize the Company ’s cash, cash equivalents, short-term investments and non-current investments for each of the periods indicated: As of March 31, 2024 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 427,290 $ — $ — $ 427,290 Commercial paper 43,354 — — 43,354 Government and government agency bonds 781,698 59 ( 760 ) 780,997 Corporate bonds 118,121 57 ( 20 ) 118,158 Certificates of deposit 54,863 — — 54,863 Total cash, cash equivalents and investments $ 1,425,326 $ 116 $ ( 780 ) $ 1,424,662 As reported: Cash and cash equivalents $ 427,290 $ — $ — $ 427,290 Short-term investments 963,987 116 ( 650 ) 963,453 Other non-current assets 34,049 — ( 130 ) 33,919 Total cash, cash equivalents and investments $ 1,425,326 $ 116 $ ( 780 ) $ 1,424,662 As of December 31, 2023 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 374,750 $ — $ — $ 374,750 Commercial paper 113,362 — — 113,362 Government and government agency bonds 1,000,302 1,006 ( 171 ) 1,001,137 Corporate bonds 130,270 118 ( 8 ) 130,380 Certificates of deposit 56,621 — — 56,621 Total cash, cash equivalents and investments $ 1,675,305 $ 1,124 $ ( 179 ) $ 1,676,250 As reported: Cash and cash equivalents $ 428,430 $ — $ — $ 428,430 Short-term investments 1,246,875 1,124 ( 179 ) 1,247,820 Total cash, cash equivalents and investments $ 1,675,305 $ 1,124 $ ( 179 ) $ 1,676,250 |
PRODUCT REVENUES, NET, ACCOUN_2
PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE, NET, AND RESERVES FOR PRODUCT REVENUES (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Receivables, Net, Current [Abstract] | |
Summary of Product Revenues | Net product revenues, which includes revenues associated with EXONDYS 51, AMONDYS 45 and VYONDYS 53 (collectively, the “PMO Products”) and ELEVIDYS consisted of the following: For the Three Months Ended 2024 2023 (in thousands) PMO Products United States $ 189,667 $ 200,451 Rest of World 35,881 31,044 Total PMO product revenues, net $ 225,548 $ 231,495 ELEVIDYS United States 133,936 — Total ELEVIDYS product revenue, net $ 133,936 $ — Total product revenues, net $ 359,484 $ 231,495 |
Schedule of Product revenue Concentration risks | The following table summarizes the Company's net product revenues, by customer, for those customers that exceeded 10 % for the periods indicated: For the Three Months Ended 2024 2023 Product revenues, net Customer 1 36 % 49 % Customer 2 20 % 32 % |
Summary of Change in Reserves for Discounts and Allowances | The following tables summarize an analysis of the change in reserves for discounts and allowances for each of the periods indicated: Chargebacks Rebates Prompt Pay Other Accruals Total (in thousands) Balance, as of December 31, 2023 $ 27,486 $ 98,194 $ 3,831 $ 35,261 $ 164,772 Provision 20,466 35,951 4,381 18,965 79,763 Adjustments relating to prior periods 783 ( 2,532 ) — — ( 1,749 ) Payments/credits ( 30,358 ) ( 36,623 ) ( 3,685 ) ( 16,220 ) ( 86,886 ) Balance, as of March 31, 2024 $ 18,377 $ 94,990 $ 4,527 $ 38,006 $ 155,900 Chargebacks Rebates Prompt Pay Other Accruals Total (in thousands) Balance, as of December 31, 2022 $ 417 $ 67,493 $ 3,343 $ 23,445 $ 94,698 Provision 2,827 30,488 3,572 14,567 51,454 Adjustments relating to prior periods — ( 2,162 ) — — ( 2,162 ) Payments/credits ( 3,244 ) ( 28,025 ) ( 3,389 ) ( 14,040 ) ( 48,698 ) Balance, as of March 31, 2023 $ — $ 67,794 $ 3,526 $ 23,972 $ 95,292 |
Summary of Total Reserves Included in Consolidated Balance Sheets | The following table summarizes the total reserves above included in the Company’s unaudited condensed consolidated balance sheets for each of the periods indicated: As of As of (in thousands) Reduction to accounts receivable $ 58,684 $ 64,697 Component of accrued expenses 97,216 100,075 Total reserves $ 155,900 $ 164,772 |
INVENTORY (Tables)
INVENTORY (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Inventory Disclosure [Abstract] | |
Schedule of components of inventory | The following table summarizes the components of the Company’s inventory for each of the periods indicated: As of As of (in thousands) Raw materials $ 150,011 $ 133,963 Work in progress 391,558 318,458 Finished goods 39,503 61,806 Total inventory $ 581,072 $ 514,227 |
Summary Of Inventory Balance Sheet Classification | The following table summarizes the balance sheet classification of the Company's inventory for each of the periods indicated: As of As of (in thousands) Balance sheet classification Inventory $ 373,530 $ 322,859 Non-current inventory 207,542 191,368 Total inventory $ 581,072 $ 514,227 |
OTHER ASSETS (Tables)
OTHER ASSETS (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | |
Summary of Other Current Assets | The following table summarizes the Company’s other current assets for each of the periods indicated: As of As of (in thousands) Collaboration and other receivables $ 30,092 $ 29,786 Prepaid maintenance services 15,907 11,281 Prepaid clinical and pre-clinical expenses 11,552 10,280 Tax-related receivables and prepaids 7,274 6,862 Prepaid commercial expenses 3,231 2,729 Interest receivable 3,209 2,731 Prepaid insurance 1,952 3,352 Prepaid research expenses 1,672 2,512 Other 8,076 8,181 Total other current assets $ 82,965 $ 77,714 |
Summary of Other Non-current Assets | The following table summarizes the Company’s other non-current assets for each of the periods indicated: As of As of (in thousands) Manufacturing-related deposits and prepaids $ 82,610 $ 74,204 Non-current investments 33,919 — Intangible assets, net 28,930 29,620 Restricted cash* 15,579 15,579 Strategic investments 7,457 6,527 Prepaid maintenance services 4,569 5,466 Other 3,343 5,375 Total other non-current assets $ 176,407 $ 136,771 * The Company had approximately $ 15.6 million in restricted cash included in other non-current assets on the Company ’s unaudited condensed consolidated balance sheets as of both March 31, 2024 and December 31, 2023. Restricted cash for both periods relates to (i) letters of credit established under the Company’s various property leases that serve as security for potential future default of lease payments, (ii) a letter of credit established under a certain commercial supply agreement and (iii) collateralized cash for the Company’ s credit cards. The restricted cash is unavailable for withdrawal or use for general obligations . |
ACCRUED EXPENSES (Tables)
ACCRUED EXPENSES (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Payables and Accruals [Abstract] | |
Summary of Accrued Expenses | The following table summarizes the Company’s accrued expenses for each of the periods indicated: As of As of (in thousands) Product revenue related reserves $ 97,216 $ 100,075 Accrued contract manufacturing costs 45,057 33,024 Accrued clinical and pre-clinical costs 36,078 34,669 Accrued employee compensation costs 32,112 78,732 Accrued professional fees 21,014 17,187 Accrued income taxes 18,292 13,766 Accrued fixed assets 11,587 3,231 Accrued royalties 11,206 12,070 Accrued research expenses 2,873 2,441 Accrued milestone and license expenses 2,000 11,375 Other 5,882 8,427 Total accrued expenses $ 283,317 $ 314,997 |
STOCK-BASED COMPENSATION (Table
STOCK-BASED COMPENSATION (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Share-Based Payment Arrangement [Abstract] | |
Summary of Stock Awards Granted | The following table summarizes the Company’s stock awards granted for each of the periods indicated: For the Three Months Ended March 31, 2024 2023 Grants Weighted Grants Weighted Stock options 316,378 $ 67.56 1,030,349 $ 72.80 Restricted stock units 1,161,985 (1) $ 128.58 1,075,911 (2) $ 154.91 (1) Included in restricted stock units (“RSUs”) for the three months ended March 31, 2024 are 88,600 shares with performance conditions, which are related to the achievement of certain financial performance goals, regulatory approval of certain of the Company's product candidates, and regulatory expansion of certain of the Company's commercial products. As of March 31, 2024, none of the performance conditions were probable of being achieved. If the performance milestones are achieved within the required time frame, the Company may recognize up t o $ 14.3 million of stock-based compensation related to these grants. Stock options and the remaining RSUs granted during the three months ended March 31, 2024 have only service-based criteria and vest over four years. (2) Included in the RSUs for the three months ended March 31, 2023 are 502,255 shares with performance conditions (the “March 2023 PSUs”) which are related to regulatory approval of certain of the Company's product candidates and achievement of a certain financial performance target. The Company recorded stock-based compensation expense of $ 8.9 million associated with the March 2023 PSUs during the three months ended March 31, 2024, with no similar expense recorded during the three months ended March 31, 2023 . Vesting of the March 2023 PSUs is contingent on the fulfillment of remaining service conditions. |
Summary of Stock-Based Compensation Expense by Grant Type and by Function Included within Consolidated Statements of Comprehensive Income (Loss) | The following table summarizes stock-based compensation expense by grant type and by function included within the unaudited condensed consolidated statements of comprehensive income (loss): For the Three Months Ended 2024 2023 (in thousands) Stock options $ 16,281 $ 23,830 Restricted stock units 27,527 16,140 Employee stock purchase plan 1,397 1,280 Subtotal $ 45,205 $ 41,250 Capitalized stock-based compensation costs* ( 4,513 ) — Total stock-based compensation expense included in expenses $ 40,692 $ 41,250 Research and development $ 16,273 $ 16,413 Selling, general and administrative 24,419 24,837 Total stock-based compensation expense included in expenses $ 40,692 $ 41,250 *Prior to the three months ended March 31, 2024 , capitalized stock-based compensation costs were not material. |
OTHER (LOSS) INCOME, NET (Table
OTHER (LOSS) INCOME, NET (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Other Income and Expenses [Abstract] | |
Summary of Other Loss | The following table summarizes other income (loss), net f or each of the periods indicated: For the Three Months Ended 2024 2023 (in thousands) Accretion of investment discount, net $ 12,804 $ 10,039 Interest income 7,093 9,276 Interest expense ( 4,166 ) ( 6,323 ) Change in the fair value of contingent consideration ( 10,100 ) — Other, net 912 ( 285 ) Other income, net $ 6,543 $ 12,707 Loss on debt extinguishment — ( 387,329 ) Total other income (loss), net $ 6,543 $ ( 374,622 ) |
Earning (Loss) Per Share (Table
Earning (Loss) Per Share (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Earnings Per Share [Abstract] | |
Basic and Diluted Net Loss Per Share | The following table sets forth the computation of basic and diluted earnings (loss) per common share: For the Three Months Ended 2024 2023 (in thousands, except per share amounts) Numerator: Net income (loss) - basic $ 36,119 $ ( 516,755 ) Add: interest expense, net of tax, on the Company's convertible debt 429 — Net income (loss) - diluted $ 36,548 $ ( 516,755 ) Denominator: Weighted-average common shares outstanding, basic 93,991 88,186 Effect of dilutive securities: Common stock issuable under the Company's equity incentive plans 3,681 — Common stock issuable under the Company's convertible debt 1,442 — Weighted-average common shares outstanding, diluted 99,114 88,186 Earnings (loss) per common share, basic $ 0.38 $ ( 5.86 ) Earnings (loss) per common share, diluted $ 0.37 $ ( 5.86 ) |
Summary of Potential Shares of Common Stock that were Excluded from the Computation of Diluted Earnings per Share | The following table summarizes potential shares of common stock that were excluded from the computation of diluted earnings per share as they were anti-dilutive: For the Three Months Ended 2024 2023 (in thousands) Common stock issuable under the Company's equity incentive plans 4,270 12,553 Common stock issuable under the Company's convertible debt 8,100 9,542 Total number of potentially issuable common stock 12,370 22,095 |
COMMITMENTS AND CONTINGENCIES (
COMMITMENTS AND CONTINGENCIES (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Commitments and Contingencies Disclosure [Abstract] | |
Summary of Aggregate Non-Cancelable Contractual Obligations Arising from Manufacturing Obligations | The following table summarizes the aggregate non-cancelable contractual obligations arising from the Company’s manufacturing obligations: As of (in thousands) 2024 (April-December) $ 836,030 2025 165,293 2026 114,785 2027 77,645 2028 72,346 Total manufacturing commitments* $ 1,266,099 * Total manufacturing commitments include the Catalent, Inc. ( “Catalent”) manufacturing and supply agreement, for which the Company has ROU assets and lease liabilities recorded on the unaudited condensed consolidated balance sheets as of March 31, 2024. For more information, please read Note 21, Commitments and Contingencies to the financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 . |
Organization and Nature of Bu_2
Organization and Nature of Business - Additional Information (Detail) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 | Mar. 31, 2023 | ||
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |||||
Cash cash equivalents restricted cash and investments | $ 1,440,300 | ||||
Cash and cash equivalents | 427,290 | $ 428,430 | $ 871,668 | ||
Short-term investments | 963,453 | 1,247,820 | |||
Restricted cash non-current | 15,579 | [1] | $ 15,579 | [1] | $ 19,024 |
Non-current investments | $ 33,900 | ||||
[1] The Company had approximately $ 15.6 million in restricted cash included in other non-current assets on the Company ’s unaudited condensed consolidated balance sheets as of both March 31, 2024 and December 31, 2023. Restricted cash for both periods relates to (i) letters of credit established under the Company’s various property leases that serve as security for potential future default of lease payments, (ii) a letter of credit established under a certain commercial supply agreement and (iii) collateralized cash for the Company’ s credit cards. The restricted cash is unavailable for withdrawal or use for general obligations |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies - Additional Information (Detail) $ in Thousands | 3 Months Ended | 12 Months Ended | |
Mar. 31, 2024 USD ($) Segment | Mar. 31, 2023 USD ($) | Dec. 31, 2023 | |
Summary Of Significant Accounting Policies [Line Items] | |||
Number of operating segments | Segment | 1 | ||
Net Income (Loss) | $ | $ 36,119 | $ (516,755) | |
Customer One [Member] | Customer Concentration Risk [Member] | Accounts Receivable [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Concentration of credit risk percentage | 34% | 40% | |
Customer Two [Member] | Customer Concentration Risk [Member] | Accounts Receivable [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Concentration of credit risk percentage | 22% | 19% | |
Customer Three [Member] | Customer Concentration Risk [Member] | Accounts Receivable [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Concentration of credit risk percentage | 21% | 19% | |
Customer Four [Member] | Customer Concentration Risk [Member] | Accounts Receivable [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Concentration of credit risk percentage | 12% |
License and Collaboration Agr_2
License and Collaboration Agreements - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | |||
Feb. 12, 2024 | Mar. 31, 2024 | Mar. 31, 2023 | Dec. 31, 2023 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Product revenues, net | $ 359,484 | $ 231,495 | ||
Collaboration Receivable Current | 30,092 | $ 29,786 | ||
Development Milestone and Upfront Fee Recognized as Research and Development Expense | 0 | 500 | ||
ELEVIDYS [Member] | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Product revenues, net | 133,936 | 0 | ||
Collaborative Arrangement | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Payment for option exercise | 121,800 | |||
Collaborative Arrangement | Maximum [Member] | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Contingent research milestone payments | 34,800 | |||
Development Milestone and Upfront Fee Recognized as Research and Development Expense | 3,200,000 | |||
Collaborative Arrangement | Roche Holding A.G. [Member] | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Product revenues, net | $ 48,000 | 48,000 | 22,000 | |
Deferred Revenue | 437,000 | 485,000 | ||
Deferred Revenue, Current | 112,000 | 48,000 | ||
Research and development expense | 21,700 | 20,300 | ||
Collaboration Receivable | 30,100 | $ 29,800 | ||
Collaborative Arrangement | Roche Holding A.G. [Member] | ELEVIDYS [Member] | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Collaboration Receivable Current | 5,800 | $ 0 | ||
Inventory Cost | $ 1,700 |
Fair Value Measurements - Asset
Fair Value Measurements - Assets and Liabilities Measured and Carried at Fair Value (Detail) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | $ 1,282,699 | $ 1,371,946 |
Contingent consideration | 48,200 | 38,100 |
Total liabilities | 48,200 | 38,100 |
Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 277,870 | 63,919 |
Commercial Paper [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 43,354 | 113,362 |
Government and Government Agency Bonds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 780,997 | 1,001,137 |
Corporate Bonds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 118,158 | 130,380 |
Strategic Equity Investments [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 7,457 | 6,527 |
Certificates of Deposit [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 54,863 | 56,621 |
Level 1 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 284,327 | 69,446 |
Contingent consideration | 0 | 0 |
Total liabilities | 0 | 0 |
Level 1 [Member] | Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 277,870 | 63,919 |
Level 1 [Member] | Commercial Paper [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 0 | 0 |
Level 1 [Member] | Government and Government Agency Bonds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 0 | 0 |
Level 1 [Member] | Corporate Bonds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 0 | 0 |
Level 1 [Member] | Strategic Equity Investments [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 6,457 | 5,527 |
Level 1 [Member] | Certificates of Deposit [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 0 | 0 |
Level 2 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 997,372 | 1,301,500 |
Contingent consideration | 0 | 0 |
Total liabilities | 0 | 0 |
Level 2 [Member] | Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 0 | 0 |
Level 2 [Member] | Commercial Paper [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 43,354 | 113,362 |
Level 2 [Member] | Government and Government Agency Bonds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 780,997 | 1,001,137 |
Level 2 [Member] | Corporate Bonds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 118,158 | 130,380 |
Level 2 [Member] | Strategic Equity Investments [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 0 | 0 |
Level 2 [Member] | Certificates of Deposit [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 54,863 | 56,621 |
Level 3 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 1,000 | 1,000 |
Contingent consideration | 48,200 | 38,100 |
Total liabilities | 48,200 | 38,100 |
Level 3 [Member] | Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 0 | 0 |
Level 3 [Member] | Commercial Paper [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 0 | 0 |
Level 3 [Member] | Government and Government Agency Bonds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 0 | 0 |
Level 3 [Member] | Corporate Bonds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 0 | 0 |
Level 3 [Member] | Strategic Equity Investments [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 1,000 | 1,000 |
Level 3 [Member] | Certificates of Deposit [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | $ 0 | $ 0 |
Fair Value Measurements - Summa
Fair Value Measurements - Summary of Fair Value of Level 3 Financial Liabilities (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Fair value, as of December 31, 2023 | $ 38,100 | |
Change in estimated fair value | 10,100 | $ 0 |
Fair value, as of March 31, 2024 | 48,200 | |
Level 3 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Fair value, as of December 31, 2023 | 38,100 | |
Change in estimated fair value | 10,100 | |
Fair value, as of March 31, 2024 | $ 48,200 |
Fair Value Measurements - Sum_2
Fair Value Measurements - Summary of carrying value and fair value of the Company's convertible notes (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Debt Instrument [Line Items] | ||
Carrying value of debt facilities, current | $ 105,586 | $ 105,483 |
Carrying value of debt facilities | 1,239,246 | 1,237,998 |
Fair value of debt facilities | 1,523,339 | 1,317,109 |
2024 Notes | ||
Debt Instrument [Line Items] | ||
Carrying value of debt facilities, current | 105,586 | 105,483 |
Fair value of debt facilities | 188,867 | 144,833 |
2027 Notes | ||
Debt Instrument [Line Items] | ||
Carrying value of debt facilities | 1,133,660 | 1,132,515 |
Fair value of debt facilities | $ 1,334,472 | $ 1,172,276 |
Fair Value Measurements - Addit
Fair Value Measurements - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Change in estimated fair value | $ 10,100 | $ 0 |
Cash, Cash Equivalents and Ma_3
Cash, Cash Equivalents and Marketable Securities - Summary of Company Financial Assets with Maturities of Less Than 90 Days Included in Cash Equivalents (Detail) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | $ 277,870 | $ 117,599 |
Money Market Funds [Member] | ||
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | 277,870 | 63,919 |
Commercial Paper [Member] | ||
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | $ 0 | $ 53,680 |
Cash, Cash Equivalents and Ma_4
Cash, Cash Equivalents and Marketable Securities - Additional Information (Detail) | 3 Months Ended | 12 Months Ended |
Mar. 31, 2024 | Dec. 31, 2023 | |
Cash and Cash Equivalents [Line Items] | ||
Weighted average maturity period of available-for-sale securities | 6 months | 5 months |
Cash, Cash Equivalents and Ma_5
Cash, Cash Equivalents and Marketable Securities - Summary of Company Cash, Cash Equivalents and Short-term Investments (Detail) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 | Mar. 31, 2023 |
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Gross unrealized gains | $ 116 | $ 1,124 | |
Available for sale debt securities, Gross unrealized losses | (650) | (179) | |
Cash and cash equivalents, Amortized cost | 1,425,326 | 1,675,305 | |
Cash and cash equivalents | 427,290 | 428,430 | $ 871,668 |
Cash, cash equivalents and investments, Amortized cost | 427,290 | 428,430 | |
Cash, cash equivalents and investments, Gross unrealized gains | 116 | 1,124 | |
Cash, cash equivalents and investments, Gross unrealized losses | (780) | (179) | |
Cash, cash equivalents and investments, Fair value | 1,424,662 | 1,676,250 | |
Available for sale debt securities, Amortized cost | 963,987 | 1,246,875 | |
Available for sale debt securities current, Fair value | 963,453 | 1,247,820 | |
Cash and Money Market Funds [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Cash and cash equivalents, Amortized cost | 427,290 | 374,750 | |
Cash and cash equivalents | 427,290 | 374,750 | |
Commercial Paper [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Fair value | 43,354 | 113,362 | |
Cash and cash equivalents, Amortized cost | 43,354 | 113,362 | |
Government and Government Agency Bonds [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Gross unrealized gains | 59 | 1,006 | |
Available for sale debt securities, Gross unrealized losses | (760) | (171) | |
Available for sale debt securities, Fair value | 780,997 | 1,001,137 | |
Cash and cash equivalents, Amortized cost | 781,698 | 1,000,302 | |
Corporate Bond [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Gross unrealized gains | 57 | 118 | |
Available for sale debt securities, Gross unrealized losses | (20) | (8) | |
Available for sale debt securities, Fair value | 118,158 | 130,380 | |
Cash and cash equivalents, Amortized cost | 118,121 | 130,270 | |
Certificates of Deposit [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Fair value | 54,863 | 56,621 | |
Cash and cash equivalents, Amortized cost | 54,863 | $ 56,621 | |
Other Non-Current Assets [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Gross unrealized losses | (130) | ||
Available for sale debt securities, Fair value | 33,919 | ||
Other non-current assets Amortized cost | $ 34,049 |
Product Revenues, Net, Accoun_3
Product Revenues, Net, Accounts Receivable, Net and Reserves for Product Revenues (Additional Information) (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | Dec. 31, 2023 | |
Products, net | $ 359,484 | $ 231,495 | |
Revenues | 413,464 | $ 253,500 | |
Product sales receivable, net of discounts and allowances | $ 378,800 | $ 400,300 | |
Payment Terms | As of March 31, 2024, the majority of the Company’s accounts receivable arose from product sales in the U.S. and all customers have standard payment terms that generally require payment within 65 to 90 days. Outside of the U.S., the majority of the Company’s customers have payment terms ranging between 90 and 150 days. | ||
Sales Revenue Net [Member] | Customer Concentration Risk [Member] | Customer One [Member] | |||
Concentration of credit risk percentage | 36% | 49% | |
Sales Revenue Net [Member] | Customer Concentration Risk [Member] | Customer Two [Member] | |||
Concentration of credit risk percentage | 20% | 32% | |
Sales Revenue Net [Member] | Customer Concentration Risk [Member] | No Individual [Member] | |||
Concentration of credit risk percentage | 10% | 10% | |
Sales Revenue Net [Member] | Customer Concentration Risk [Member] | Customers Exceed [Member] | |||
Concentration of credit risk percentage | 10% | ||
Accounts Receivable [Member] | Customer Concentration Risk [Member] | Customer One [Member] | |||
Concentration of credit risk percentage | 34% | 40% | |
Accounts Receivable [Member] | Customer Concentration Risk [Member] | Customer Two [Member] | |||
Concentration of credit risk percentage | 22% | 19% | |
Accounts Receivable [Member] | Customer Concentration Risk [Member] | Customer Three [Member] | |||
Concentration of credit risk percentage | 21% | 19% |
Product Revenues, Net, Accoun_4
Product Revenues, Net, Accounts Receivable, Net and Reserves for Product Revenues - Summary of Product Revenues (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Product Information [Line Items] | ||
Products, net | $ 359,484 | $ 231,495 |
PMO Products [Member] | ||
Product Information [Line Items] | ||
Products, net | 225,548 | 231,495 |
PMO Products [Member] | United States [Member] | ||
Product Information [Line Items] | ||
Products, net | 189,667 | 200,451 |
PMO Products [Member] | Rest Of World [Member] | ||
Product Information [Line Items] | ||
Products, net | 35,881 | 31,044 |
ELEVIDYS [Member] | ||
Product Information [Line Items] | ||
Products, net | 133,936 | 0 |
ELEVIDYS [Member] | United States [Member] | ||
Product Information [Line Items] | ||
Products, net | $ 133,936 | $ 0 |
Product Revenues, Net, Accoun_5
Product Revenues, Net, Accounts Receivable, Net and Reserves for Product Revenues - Summarizes the concentration of the three largest customers (Details) - Customer Concentration Risk [Member] - Sales Revenue Net [Member] | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Customer One [Member] | ||
Principal Transaction Revenue [Line Items] | ||
Concentration of credit risk percentage | 36% | 49% |
Customer Two [Member] | ||
Principal Transaction Revenue [Line Items] | ||
Concentration of credit risk percentage | 20% | 32% |
Product Revenues, Net, Accoun_6
Product Revenues, Net, Accounts Receivable, Net and Reserves for Product RevenuesProduct Revenues, Net, Accounts Receivable, Net and Reserves for Product Revenues - Summary of Change in Reserves for Discounts and Allowances (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | $ 164,772 | $ 94,698 |
Provision | 79,763 | 51,454 |
Adjustments relating to prior periods | (1,749) | (2,162) |
Payments/credits | (86,886) | (48,698) |
Ending balance | 155,900 | 95,292 |
Chargebacks [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 27,486 | 417 |
Provision | 20,466 | 2,827 |
Adjustments relating to prior periods | 783 | 0 |
Payments/credits | (30,358) | (3,244) |
Ending balance | 18,377 | 0 |
Rebates [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 98,194 | 67,493 |
Provision | 35,951 | 30,488 |
Adjustments relating to prior periods | (2,532) | (2,162) |
Payments/credits | (36,623) | (28,025) |
Ending balance | 94,990 | 67,794 |
Prompt Pay [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 3,831 | 3,343 |
Provision | 4,381 | 3,572 |
Adjustments relating to prior periods | 0 | 0 |
Payments/credits | (3,685) | (3,389) |
Ending balance | 4,527 | 3,526 |
Other Accruals [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 35,261 | 23,445 |
Provision | 18,965 | 14,567 |
Adjustments relating to prior periods | 0 | 0 |
Payments/credits | (16,220) | (14,040) |
Ending balance | $ 38,006 | $ 23,972 |
Product Revenues, Net, Accoun_7
Product Revenues, Net, Accounts Receivable, Net and Reserves for Product Revenues - Summary of Total Reserves Included in Consolidated Balance Sheets (Detail) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 | Mar. 31, 2023 | Dec. 31, 2022 |
Receivables, Net, Current [Abstract] | ||||
Reduction to accounts receivable | $ 58,684 | $ 64,697 | ||
Component of accrued expenses | 97,216 | 100,075 | ||
Total reserves | $ 155,900 | $ 164,772 | $ 95,292 | $ 94,698 |
Inventory - Summary of Componen
Inventory - Summary of Components of Inventory (Detail) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 150,011 | $ 133,963 |
Work in progress | 391,558 | 318,458 |
Finished goods | 39,503 | 61,806 |
Total inventory | $ 581,072 | $ 514,227 |
Inventory - Summarizes The Bala
Inventory - Summarizes The Balance Sheet Classification (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Inventory Disclosure [Abstract] | ||
Inventory | $ 373,530 | $ 322,859 |
Non-current inventory | 207,542 | 191,368 |
Total inventory | $ 581,072 | $ 514,227 |
Other Assets - Summary of Other
Other Assets - Summary of Other Current Assets (Detail) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Prepaid Expense and Other Assets, Current [Abstract] | ||
Collaboration and other receivables | $ 30,092 | $ 29,786 |
Prepaid maintenance services | 15,907 | 11,281 |
Prepaid clinical and pre-clinical expenses | 11,552 | 10,280 |
Tax-related receivables and prepaids | 7,274 | 6,862 |
Prepaid research expenses | 1,672 | 2,512 |
Prepaid commercial expenses | 3,231 | 2,729 |
Interest receivable | 3,209 | 2,731 |
Prepaid insurance | 1,952 | 3,352 |
Other | 8,076 | 8,181 |
Total other current assets | $ 82,965 | $ 77,714 |
Other Assets - Summary of Oth_2
Other Assets - Summary of Other Non-current Assets (Detail) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 | Mar. 31, 2023 | ||
Prepaid Expense and Other Assets, Noncurrent [Abstract] | |||||
Manufacturing-related deposits and prepaids | $ 82,610 | $ 74,204 | |||
Non-current investments | 33,919 | 0 | |||
Intangible assets, net | 28,930 | 29,620 | |||
Restricted cash | 15,579 | [1] | 15,579 | [1] | $ 19,024 |
Strategic investments | 7,457 | 6,527 | |||
Prepaid maintenance services | 4,569 | 5,466 | |||
Other | 3,343 | 5,375 | |||
Total other non-current assets | $ 176,407 | $ 136,771 | |||
[1] The Company had approximately $ 15.6 million in restricted cash included in other non-current assets on the Company ’s unaudited condensed consolidated balance sheets as of both March 31, 2024 and December 31, 2023. Restricted cash for both periods relates to (i) letters of credit established under the Company’s various property leases that serve as security for potential future default of lease payments, (ii) a letter of credit established under a certain commercial supply agreement and (iii) collateralized cash for the Company’ s credit cards. The restricted cash is unavailable for withdrawal or use for general obligations |
Other Assets - Summary of Oth_3
Other Assets - Summary of Other Non-current Assets (Detail) (Parenthetical) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 | Mar. 31, 2023 | ||
Prepaid Expense and Other Assets, Current [Abstract] | |||||
Restricted cash non-current | $ 15,579 | [1] | $ 15,579 | [1] | $ 19,024 |
[1] The Company had approximately $ 15.6 million in restricted cash included in other non-current assets on the Company ’s unaudited condensed consolidated balance sheets as of both March 31, 2024 and December 31, 2023. Restricted cash for both periods relates to (i) letters of credit established under the Company’s various property leases that serve as security for potential future default of lease payments, (ii) a letter of credit established under a certain commercial supply agreement and (iii) collateralized cash for the Company’ s credit cards. The restricted cash is unavailable for withdrawal or use for general obligations |
Accrued Expenses - Summary of A
Accrued Expenses - Summary of Accrued Expenses (Detail) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Payables and Accruals [Abstract] | ||
Product revenue related reserves | $ 97,216 | $ 100,075 |
Accrued contract manufacturing costs | 45,057 | 33,024 |
Accrued clinical and pre-clinical costs | 36,078 | 34,669 |
Accrued employee compensation costs | 32,112 | 78,732 |
Accrued professional fees | 21,014 | 17,187 |
Accrued income taxes | 18,292 | 13,766 |
Accrued fixed assets | 11,587 | 3,231 |
Accrued royalties | 11,206 | 12,070 |
Accrued research expenses | 2,873 | 2,441 |
Accrued Milestone Expense Current | 2,000 | 11,375 |
Other | 5,882 | 8,427 |
Total accrued expenses | $ 283,317 | $ 314,997 |
Indebtedness - Additional Infor
Indebtedness - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | ||
Mar. 31, 2024 | Mar. 31, 2023 | Dec. 31, 2023 | |
Debt Instrument [Line Items] | |||
Partial settlement of capped call share options for 2024 Notes | $ 0 | $ 80,645 | |
Loss on debt extinguishment | 0 | (387,329) | |
Debt conversion costs for 2024 Notes | 0 | $ 6,887 | |
Long-term debt | 1,133,660 | $ 1,132,515 | |
Aggregate long-term debt | $ 1,239,246 | $ 1,237,998 |
Indebtedness - Summary of Debt
Indebtedness - Summary of Debt Facilities (Detail) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Debt Instrument [Line Items] | ||
Total carrying value of debt facilities | $ 1,239,246 | $ 1,237,998 |
Total carrying value of debt facilities | 1,133,660 | 1,132,515 |
Fair value of debt facilities | $ 1,523,339 | $ 1,317,109 |
Stock Based Compensation - Summ
Stock Based Compensation - Summary of Stock Awards Granted (Detail) - $ / shares | 3 Months Ended | |||
Mar. 31, 2024 | Mar. 31, 2023 | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Grants, Stock options | 316,378 | 1,030,349 | ||
Weighted Average Grant Date Fair Value, Stock options | $ 67.56 | $ 72.8 | ||
Grants, Restricted stock units | 1,161,985 | [1] | 1,075,911 | [2] |
Weighted Average Grant Date Fair Value, Restricted stock units | $ 128.58 | $ 154.91 | ||
Restricted Stock Units [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Grants, Stock options | 88,600 | 502,255 | ||
[1] (1) Included in restricted stock units (“RSUs”) for the three months ended March 31, 2024 are 88,600 shares with performance conditions, which are related to the achievement of certain financial performance goals, regulatory approval of certain of the Company's product candidates, and regulatory expansion of certain of the Company's commercial products. As of March 31, 2024, none of the performance conditions were probable of being achieved. If the performance milestones are achieved within the required time frame, the Company may recognize up t o $ 14.3 million of stock-based compensation related to these grants. Stock options and the remaining RSUs granted during the three months ended March 31, 2024 have only service-based criteria and vest over four years. (2) Included in the RSUs for the three months ended March 31, 2023 are 502,255 shares with performance conditions (the “March 2023 PSUs”) which are related to regulatory approval of certain of the Company's product candidates and achievement of a certain financial performance target. The Company recorded stock-based compensation expense of $ 8.9 million associated with the March 2023 PSUs during the three months ended March 31, 2024, with no similar expense recorded during the three months ended March 31, 2023 . Vesting of the March 2023 PSUs is contingent on the fulfillment of remaining service conditions. |
Summary of Stock Awards Granted
Summary of Stock Awards Granted (Parenthetical) (Details) - USD ($) $ in Millions | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Grants, Stock options | 316,378 | 1,030,349 |
Stock-based compensation expense | $ 40.7 | $ 41.3 |
Restricted Stock Units [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Grants, Stock options | 88,600 | 502,255 |
Stock-based compensation Grants | $ 14.3 | |
Stock-based compensation expense | $ 8.9 |
Stock Based Compensation - Addi
Stock Based Compensation - Additional Information (Detail) - USD ($) $ in Millions | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | ||
Stock-based compensation expense | $ 40.7 | $ 41.3 |
Grants, Stock options | 316,378 | 1,030,349 |
Summary of Stock-Based Compensa
Summary of Stock-Based Compensation Expense by Function Included within Condensed Consolidated Statements of Operations and Comprehensive Loss (Detail) - USD ($) $ in Millions | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||
Total stock-based compensation expense included in expenses | $ 40.7 | $ 41.3 |
Summary of Stock-Based Compen_2
Summary of Stock-Based Compensation Expense by Grant Type and by Function Included within Consolidated Statements of Comprehensive Income (Loss) (Detail) - USD ($) $ in Thousands | 3 Months Ended | ||
Mar. 31, 2024 | Mar. 31, 2023 | ||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Subtotal | $ 45,205 | $ 41,250 | |
Capitalized stock-based compensation costs | [1] | (4,513) | 0 |
Total stock-based compensation expense included in expenses | 40,692 | 41,250 | |
Research and Development Expense [Member] | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation expense included in expenses | 16,273 | 16,413 | |
Selling, General and Administrative [Member] | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation expense included in expenses | 24,419 | 24,837 | |
Employee Stock Option | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation expense included in expenses | 16,281 | 23,830 | |
Restricted Stock Awards/Units (RSAs/RSUs) [Member] | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation expense included in expenses | 27,527 | 16,140 | |
Employee Stock Purchase Plan [Member] | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation expense included in expenses | $ 1,397 | $ 1,280 | |
[1] *Prior to the three months ended March 31, 2024 , capitalized stock-based compensation costs were not material. |
Other Income (Loss), Net - Summ
Other Income (Loss), Net - Summary of Other Loss (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Nonoperating Income (Expense) [Abstract] | ||
Accretion of investment discount, net | $ 12,804 | $ 10,039 |
Interest income | 7,093 | 9,276 |
Interest expense | (4,166) | (6,323) |
Change in the fair value of contingent consideration | (10,100) | 0 |
Other, net | 912 | (285) |
Other income, net | 6,543 | 12,707 |
Loss on debt extinguishment | 0 | (387,329) |
Total other income (loss), net | $ 6,543 | $ (374,622) |
Income Taxes (Additional Inform
Income Taxes (Additional Information) (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Income Tax Disclosure [Abstract] | ||
Provision for Income tax | $ 5,329 | $ 4,045 |
Effective tax rate | 14.20% | 0.80% |
Statutory tax rate | 21% | |
Federal tax expense | $ 900 | |
State tax expense | $ 3,100 |
Earning (Loss) Per Share - Basi
Earning (Loss) Per Share - Basic and Diluted Net Loss Per Share (Detail) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Earnings Per Share [Abstract] | ||
Net Income (Loss) | $ 36,119 | $ (516,755) |
Add: interest expense, net of tax, on the Company's convertible debt | 429 | 0 |
Net income (loss) - diluted | $ 36,548 | $ (516,755) |
Weighted-average common shares outstanding - basic | 93,991,000 | 88,186,000 |
Common stock issuable under the Company's equity incentive plans | 3,681,000 | 0 |
Common stock issuable under the Company's convertible debt | 1,442,000 | 0 |
Weighted-average common shares outstanding - diluted | 99,114,000 | 88,186,000 |
Earnings (loss) per common share, basic | $ 0.38 | $ (5.86) |
Earnings (loss) per common share, diluted | $ 0.37 | $ (5.86) |
Earning (Loss) Per Share - Summ
Earning (Loss) Per Share - Summary of Potential Shares of Common Stock that were Excluded from the Computation of Diluted Earnings per Share (Details) - shares shares in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Total number of potentially issuable common stock | 12,370 | 22,095 |
Convertible Debt [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Total number of potentially issuable common stock | 8,100 | 9,542 |
Equity Incentive Plans [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Total number of potentially issuable common stock | 4,270 | 12,553 |
Commitments and Contingencies -
Commitments and Contingencies - Summary of Aggregate Non-Cancelable Contractual Obligations Arising from Manufacturing Obligations (Detail) $ in Thousands | Mar. 31, 2024 USD ($) | |
Commitments And Contingencies [Line Items] | ||
2024 (April-December) | $ 836,030 | |
2025 | 165,293 | |
2026 | 114,785 | |
2027 | 77,645 | |
Thereafter | 72,346 | |
Total manufacturing commitments | $ 1,266,099 | [1] |
[1] Total manufacturing commitments include the Catalent, Inc. ( “Catalent”) manufacturing and supply agreement, for which the Company has ROU assets and lease liabilities recorded on the unaudited condensed consolidated balance sheets as of March 31, 2024. For more information, please read Note 21, Commitments and Contingencies to the financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 . |
Commitments and Contingencies_2
Commitments and Contingencies (Additional Information) (Details) - USD ($) $ in Millions | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Commitments And Contingencies [Line Items] | ||
Purchase commitment | $ 54.7 | |
Amendment Implemented Fee | 60 | |
First Installments [Member] | ||
Commitments And Contingencies [Line Items] | ||
Amendment Implemented Fee | $ 20 | $ 20 |
Installments Date of implemented fee | Mar. 01, 2024 | |
Research and Development Expense [Member] | ||
Commitments And Contingencies [Line Items] | ||
Nonrefundable Advance Payment | $ 0.9 | |
Other Current Assets | ||
Commitments And Contingencies [Line Items] | ||
Nonrefundable Advance Payment | 16.5 | |
Other Non-Current Assets [Member] | ||
Commitments And Contingencies [Line Items] | ||
Nonrefundable Advance Payment | $ 16.5 |