Cover
Cover - USD ($) | 12 Months Ended | ||
Dec. 31, 2023 | Feb. 27, 2024 | Jun. 30, 2023 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Document Annual Report | true | ||
Document Period End Date | Dec. 31, 2023 | ||
Document Transition Report | false | ||
Entity File Number | 001-35969 | ||
Entity Registrant Name | PTC THERAPEUTICS, INC. | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Tax Identification Number | 04-3416587 | ||
Entity Address, Address Line One | 100 Corporate Court | ||
Entity Address, City or Town | South Plainfield, | ||
Entity Address, State or Province | NJ | ||
Entity Address, Postal Zip Code | 07080 | ||
City Area Code | 908 | ||
Local Phone Number | 222-7000 | ||
Title of 12(b) Security | Common Stock, $0.001 par value per share | ||
Trading Symbol | PTCT | ||
Security Exchange Name | NASDAQ | ||
Entity Well-known Seasoned Issuer | Yes | ||
Entity Voluntary Filers | No | ||
Entity Current Reporting Status | Yes | ||
Entity Interactive Data Current | Yes | ||
Entity Filer Category | Large Accelerated Filer | ||
Entity Small Business | false | ||
Entity Emerging Growth Company | false | ||
ICFR Auditor Attestation Flag | true | ||
Document Financial Statement Error Correction | false | ||
Entity Shell Company | false | ||
Entity Public Float | $ 2,330,616,515 | ||
Entity Common Stock, Shares Outstanding | 76,608,030 | ||
Documents Incorporated By Reference | DOCUMENTS INCORPORATED BY REFERENCE Part III of this Annual Report incorporates by reference information from the definitive Proxy Statement for the registrant’s 2024 Annual Meeting of Shareholders which is expected to be filed with the Securities and Exchange Commission not later than 120 days after the registrant’s fiscal year ended December 31, 2023. | ||
Entity Central Index Key | 0001070081 | ||
Current Fiscal Year End Date | --12-31 | ||
Document Fiscal Year Focus | 2023 | ||
Document Fiscal Period Focus | FY | ||
Amendment Flag | false | ||
Auditor Name | Ernst & Young LLP | ||
Auditor Firm ID | 42 | ||
Auditor Location | Iselin, New Jersey |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Current assets: | ||
Cash and cash equivalents | $ 594,001 | $ 279,834 |
Marketable securities | 282,738 | 130,871 |
Trade and royalty receivables, net | 160,822 | 155,614 |
Inventory, net | 30,577 | 21,808 |
Prepaid expenses and other current assets | 150,491 | 105,658 |
Total current assets | 1,218,629 | 693,785 |
Fixed assets, net | 87,089 | 72,590 |
Intangible assets, net | 379,497 | 705,891 |
Goodwill | 82,341 | 82,341 |
Operating lease ROU assets | 91,896 | 102,430 |
Deposits and other assets | 36,246 | 48,582 |
Total assets | 1,895,698 | 1,705,619 |
Current liabilities: | ||
Accounts payable and accrued expenses | 391,983 | 320,366 |
Deferred revenue | 801 | 1,351 |
Operating lease liabilities- current | 13,002 | 9,370 |
Finance lease liabilities- current | 3,000 | 3,000 |
Liability for sale of future royalties - current | 194,314 | 72,149 |
Total current liabilities | 603,100 | 406,236 |
Long-term debt | 284,213 | 571,722 |
Contingent consideration payable | 36,300 | 164,000 |
Deferred tax liability | 55,905 | 102,834 |
Operating lease liabilities- noncurrent | 97,627 | 100,860 |
Finance lease liabilities- noncurrent | 17,184 | 18,675 |
Liability for sale of future royalties- noncurrent | 1,619,783 | 685,737 |
Other long-term liabilities | 141 | 2,641 |
Total liabilities | 2,714,253 | 2,052,705 |
Stockholders' deficit: | ||
Common stock, $0.001 par value. Authorized 250,000,000 shares; issued and outstanding 75,708,889 shares at December 31, 2023. Authorized 250,000,000 shares; issued and outstanding 73,104,692 shares at December 31, 2022 | 75 | 72 |
Additional paid-in capital | 2,466,233 | 2,305,020 |
Accumulated other comprehensive (loss) income | (1,285) | 4,796 |
Accumulated deficit | (3,283,578) | (2,656,974) |
Total stockholders' deficit | (818,555) | (347,086) |
Total liabilities and stockholders' deficit | $ 1,895,698 | $ 1,705,619 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2023 | Dec. 31, 2022 |
Statement of Financial Position [Abstract] | ||
Common stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Common stock, authorized shares (in shares) | 250,000,000 | 250,000,000 |
Common stock, issued shares (in shares) | 75,708,889 | 73,104,692 |
Common stock, outstanding shares (in shares) | 75,708,889 | 73,104,692 |
Consolidated Statements of Oper
Consolidated Statements of Operations - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Revenues: | |||
Revenue | $ 937,822 | $ 698,801 | $ 538,593 |
Operating expenses: | |||
Cost of product sales, excluding amortization of acquired intangible assets | 65,486 | 44,678 | 32,328 |
Amortization of intangible assets | 222,635 | 116,554 | 54,751 |
Research and development | 666,563 | 651,496 | 540,684 |
Selling, general and administrative | 332,540 | 325,998 | 285,773 |
Change in the fair value of contingent consideration | (127,700) | (25,900) | (500) |
Intangible asset impairment | 217,800 | 33,384 | |
Total operating expenses | 1,377,324 | 1,146,210 | 913,036 |
Loss from operations | (439,502) | (447,409) | (374,443) |
Interest expense, net | (129,180) | (90,871) | (86,022) |
Other income (expense), net | 10,130 | (49,207) | (57,875) |
Loss on extinguishment of debt | (137,558) | ||
Loss before income tax benefit (expense) | (696,110) | (587,487) | (518,340) |
Income tax benefit (expense) | 69,506 | 28,470 | (5,561) |
Net loss attributable to common stockholders | $ (626,604) | $ (559,017) | $ (523,901) |
Weighted-average shares outstanding: | |||
Basic | 74,838,392 | 71,728,634 | 70,466,393 |
Diluted | 74,838,392 | 71,728,634 | 70,466,393 |
Net loss per share-basic and diluted (in dollars per share) | |||
Basic | $ (8.37) | $ (7.79) | $ (7.43) |
Diluted | $ (8.37) | $ (7.79) | $ (7.43) |
Net product revenue | |||
Revenues: | |||
Revenue | $ 661,249 | $ 535,228 | $ 428,904 |
Collaboration revenue | |||
Revenues: | |||
Revenue | 100,030 | 50,052 | 55,046 |
Royalty revenue | |||
Revenues: | |||
Revenue | 168,856 | 113,521 | 54,643 |
Manufacturing revenue | |||
Revenues: | |||
Revenue | $ 7,687 | $ 0 | $ 0 |
Consolidated Statements of Comp
Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Statement of Comprehensive Income [Abstract] | |||
Net Income (Loss) | $ (626,604) | $ (559,017) | $ (523,901) |
Other comprehensive income (loss): | |||
Unrealized gain (loss) on marketable securities, net of tax | 820 | 108 | (2,502) |
Foreign currency translation (loss) gain, net of tax | (6,901) | 28,970 | 39,177 |
Comprehensive loss | $ (632,685) | $ (529,939) | $ (487,226) |
Consolidated Statements of Stoc
Consolidated Statements of Stockholders' (Deficit) Equity - USD ($) $ in Thousands | Common stock | Additional paid-in capital Cumulative Effect Adjustment | Additional paid-in capital | Accumulated other comprehensive (loss) income | Accumulated deficit Cumulative Effect Adjustment | Accumulated deficit | Cumulative Effect Adjustment | Total |
Balance (in shares) at Dec. 31, 2020 | 69,718,096 | |||||||
Balance at the beginning of the period at Dec. 31, 2020 | $ 70 | $ (175,236) | $ 2,171,746 | $ (60,957) | $ 54,796 | $ (1,628,877) | $ (120,440) | $ 481,982 |
Increase (Decrease) in Stockholders' Equity | ||||||||
Exercise of options (in shares) | 635,871 | |||||||
Exercise of options | $ 1 | 17,308 | 17,309 | |||||
Restricted stock vesting and issuance (in shares) | 307,658 | |||||||
Issuance of common stock in connection with an employee stock purchase plan (in shares) | 166,601 | |||||||
Issuance of common stock in connection with an employee stock purchase plan | 5,792 | 5,792 | ||||||
Share-based compensation expense | 103,513 | 103,513 | ||||||
Receivable from investor | 483 | 483 | ||||||
Other | 25 | 25 | ||||||
Net Income (Loss) | (523,901) | (523,901) | ||||||
Comprehensive income | 36,675 | 36,675 | ||||||
Balance (in shares) at Dec. 31, 2021 | 70,828,226 | |||||||
Balance at the end of the period at Dec. 31, 2021 | $ 71 | 2,123,606 | (24,282) | (2,097,957) | 1,438 | |||
Increase (Decrease) in Stockholders' Equity | ||||||||
Issuance of common stock related to stock purchase agreement (in shares) | 1,095,290 | |||||||
Issuance of common stock related to stock purchase agreement | $ 1 | 49,999 | 50,000 | |||||
Exercise of options (in shares) | 496,863 | |||||||
Exercise of options | 14,632 | 14,632 | ||||||
Restricted stock vesting and issuance (in shares) | 490,008 | |||||||
Issuance of common stock in connection with an employee stock purchase plan (in shares) | 194,305 | |||||||
Issuance of common stock in connection with an employee stock purchase plan | 6,450 | 6,450 | ||||||
Share-based compensation expense | 110,333 | 110,333 | ||||||
Net Income (Loss) | (559,017) | (559,017) | ||||||
Comprehensive income | 29,078 | 29,078 | ||||||
Balance (in shares) at Dec. 31, 2022 | 73,104,692 | |||||||
Balance at the end of the period at Dec. 31, 2022 | $ 72 | 2,305,020 | 4,796 | (2,656,974) | (347,086) | |||
Increase (Decrease) in Stockholders' Equity | ||||||||
Exercise of options (in shares) | 822,482 | |||||||
Exercise of options | $ 1 | 24,039 | 24,040 | |||||
Restricted stock vesting and issuance (in shares) | 915,203 | |||||||
Restricted stock vesting and issuance, net | $ 1 | 1 | ||||||
Issuance of common stock in connection with an employee stock purchase plan (in shares) | 209,050 | |||||||
Issuance of common stock in connection with an employee stock purchase plan | 5,954 | 5,954 | ||||||
Issuance of common stock in connection with a milestone payable (in shares) | 657,462 | |||||||
Issuance of common stock in connection with a milestone payable | $ 1 | 29,569 | 29,570 | |||||
Share-based compensation expense | 101,636 | 101,636 | ||||||
Receivable from investor | 15 | 15 | ||||||
Net Income (Loss) | (626,604) | (626,604) | ||||||
Comprehensive income | (6,081) | (6,081) | ||||||
Balance (in shares) at Dec. 31, 2023 | 75,708,889 | |||||||
Balance at the end of the period at Dec. 31, 2023 | $ 75 | $ 2,466,233 | $ (1,285) | $ (3,283,578) | $ (818,555) |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Cash flows from operating activities | |||
Net Income (Loss) | $ (626,604) | $ (559,017) | $ (523,901) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Depreciation and amortization | 236,590 | 128,836 | 64,134 |
Non-cash operating lease expense | 9,827 | 9,884 | 7,386 |
Non-cash royalty revenue related to sale of future royalties | (93,460) | (48,738) | (23,460) |
Non-cash interest expense on liability related to sale of future royalties | 104,790 | 72,639 | 77,683 |
Intangible asset impairment | 217,800 | 33,384 | |
Change in valuation of contingent consideration | (127,700) | (25,900) | (500) |
Unrealized loss on ClearPoint Equity Investments | 1,515 | 3,560 | 6,078 |
Unrealized loss on ClearPoint convertible debt security | 2,678 | 5,740 | 8,281 |
Unrealized (gain) loss on marketable securities - equity investments | (2,517) | 7,992 | (1,673) |
Realized loss for the sale of Clearpoint Equity Investment | 782 | ||
Realized gain on redemption of marketable securities- equity investments | (4,383) | ||
Non-cash stock consideration, milestone payment | 29,570 | ||
Disposal of asset | 806 | 80 | |
Amortization of (discounts) premiums on investments, net | (2,200) | 1,713 | 5,299 |
Deferred income taxes | (46,930) | (34,276) | 377 |
Amortization of debt issuance costs | 1,873 | 1,901 | 1,848 |
Loss on extinguishment of debt | 55,625 | ||
Share-based compensation expense | 101,636 | 110,333 | 103,513 |
Unrealized foreign currency transaction (gains) losses, net | (14,113) | 13,263 | 47,391 |
Non cash foreign currency remeasurement loss on intercompany loan | 16,887 | ||
Changes in operating assets and liabilities: | |||
Inventory, net | (8,183) | (6,668) | 1,800 |
Prepaid expenses and other current assets | (44,992) | (51,621) | (15,310) |
Trade and royalty receivables, net | (1,539) | (48,468) | (44,991) |
Deposits and other assets | 5,222 | (2,913) | (232) |
Accounts payable and accrued expenses | 48,346 | 27,542 | 45,659 |
Other liabilities | (2,307) | (4,558) | (6,704) |
Deferred revenue | (550) | 1,351 | (4,010) |
Payments on contingent consideration | (9,600) | ||
Net cash used in operating activities | (158,418) | (356,654) | (251,332) |
Cash flows from investing activities | |||
Purchases of fixed assets | (28,438) | (32,016) | (28,213) |
Purchases of marketable securities - available for sale | (174,086) | (52,764) | (333,148) |
Purchases of marketable securities - equity investments | (38,398) | (22,787) | (210,018) |
Sale and redemption of marketable securities- available for sale | 21,544 | 405,234 | 843,498 |
Sale and redemption of marketable securities - equity investments | 132,228 | 112,958 | 4,281 |
Sale and redemption of ClearPoint Equity Investments | 2,594 | ||
Acquisition of product rights and licenses | (92,181) | (120,444) | (57,118) |
Purchase of equity investment in ClearPoint | (100) | ||
Net cash (used in) provided by investing activities | (176,737) | 290,181 | 219,182 |
Cash flows from financing activities | |||
Proceeds from exercise of options | 24,040 | 14,632 | 17,309 |
Repayment of senior secured term loan | (300,000) | ||
Debt extinguishment costs related to senior secured term loan | (81,933) | ||
Cash consideration received from Royalty Purchase Agreement | 1,000,000 | ||
Debt issuance costs related to secured term loan | (282) | (11,454) | |
Proceeds from issuance of senior secured term loan | 300,000 | ||
Repayment of Convertible Notes | (150,000) | ||
Payments on deferred and contingent consideration obligation | (40,400) | ||
Proceeds from employee stock purchase plan | 5,954 | 6,450 | 5,792 |
Payment of finance lease principal | (1,379) | (1,276) | (2,224) |
Proceeds from stock purchase agreement | 50,000 | ||
Net cash provided by financing activities | 646,400 | 167,952 | 20,877 |
Effect of exchange rate changes on cash | 3,114 | (2,772) | (7,821) |
Net increase (decrease) in cash and cash equivalents | 314,359 | 98,707 | (19,094) |
Cash and cash equivalents, and restricted cash beginning of period | 295,925 | 197,218 | 216,312 |
Cash and cash equivalents, and restricted cash end of period | 610,284 | 295,925 | 197,218 |
Supplemental disclosure of cash information | |||
Cash paid for interest | 36,131 | 18,463 | 9,588 |
Cash paid for income taxes | 14,155 | 4,922 | 7,708 |
Supplemental disclosure of non-cash investing and financing activity | |||
Unrealized gain (loss) on marketable securities, net of tax | 820 | 108 | (2,502) |
Right-of-use assets obtained in exchange for operating lease obligations | 35,817 | 645 | |
Acquisition of product rights and licenses | 54,618 | 33,239 | $ 22,294 |
Milestone payable | $ 2,500 | ||
Debt issuance costs related to senior secured term loan | 159 | ||
Capital expenditures unpaid at the end of period | $ 308 |
The Company
The Company | 12 Months Ended |
Dec. 31, 2023 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
The Company | 1. The Company PTC Therapeutics, Inc. (the “Company” or “PTC”) is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC’s ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC’s mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC’s strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes that this allows it to maximize value for all of its stakeholders. The Company has two products, Translarna™ (ataluren) and Emflaza ® The Company’s marketing authorization for Translarna in the EEA is subject to annual review and renewal by the EC following reassessment by the European Medicines Agency (“EMA”) of the benefit-risk balance of the authorization, which the Company refers to as the annual EMA reassessment. In September 2022, the Company submitted a Type II variation to the EMA to support conversion of the conditional marketing authorization for Translarna to a standard marketing authorization, which included a report on the placebo-controlled trial of Study 041 and data from the open-label extension. In February 2023, the Company also submitted an annual marketing authorization renewal request to the EMA. In September 2023, the Committee for Medicinal Products for Human Use (“CHMP’), gave a negative opinion on the conversion of the conditional marketing authorization to full marketing authorization of Translarna for the treatment of nmDMD and a negative opinion on the renewal of the existing conditional marketing authorization of Translarna for the treatment of nmDMD. On January 25, 2024, the CHMP issued a negative opinion for the renewal of the conditional marketing authorization following a re-examination procedure. In accordance with EMA regulations, the EC has 67 days to adopt the opinion. If the EC adopts the negative opinion, Translarna would no longer have marketing authorization in the member states of the EEA. Translarna is an investigational new drug in the United States. Following the Company’s announcement of top-line results from the placebo-controlled trial of Study 041 in June 2022, the Company submitted a meeting request to the U.S. Food and Drug Administration (“FDA”) to gain clarity on the regulatory pathway for a potential re-submission of a New Drug Application (“NDA”) for Translarna. The FDA provided initial written feedback that Study 041 does not provide substantial evidence of effectiveness to support NDA re-submission. The Company held a Type C meeting with the FDA in the fourth quarter of 2023 to discuss the totality of Translarna data. Based on this discussion, the FDA suggested that the Company request a pre-submission Type C meeting to discuss the specific contents of an NDA resubmission based on results from Study 041 and from the Company’s international drug registry study for nmDMD patients receiving Translarna. This meeting is scheduled for March 2024. The Company has developed Upstaza (eladocagene exuparvovec), a gene therapy used for the treatment of Aromatic L-Amino Acid Decarboxylase (“AADC”) deficiency (“AADC deficiency”), a rare central nervous system (“CNS”) disorder arising from reductions in the enzyme AADC that results from mutations in the dopa decarboxylase gene. In July 2022, the EC approved Upstaza for the treatment of AADC deficiency for patients 18 months and older within the EEA. In November 2022, the Medicines and Healthcare Products Regulatory Agency approved Upstaza for the treatment of AADC deficiency for patients 18 months and older within the United Kingdom. The Company is also preparing a biologics license application (“BLA”) for Upstaza for the treatment of AADC deficiency in the United States and anticipates submitting a BLA to the FDA in March 2024. The Company holds the rights for the commercialization of Tegsedi ® ® The Company also has a spinal muscular atrophy (“SMA”) collaboration with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc. (referred to collectively as “Roche”) and the Spinal Muscular Atrophy Foundation (“SMA Foundation”). The SMA program has one approved product, Evrysdi ® One of the Company’s most advanced clinical stage molecules is sepiapterin. Sepiapterin is a precursor to intracellular tetrahydrobiopterin, which is a critical enzymatic cofactor involved in metabolism and synthesis of numerous metabolic products, for orphan diseases. In May 2023, the Company announced that the primary endpoint was achieved in its registration-directed Phase 3 trial for sepiapterin for phenylketonuria (“PKU”) In addition to the Company’s SMA program, the Company’s splicing platform also includes PTC518, which is being developed for the treatment of Huntington’s disease (“HD”). The Company initiated a Phase 2 study of PTC518 for the treatment of HD in the first quarter of 2022, which consists of an initial 12-week placebo-controlled phase focused on safety, pharmacology and pharmacodynamic effects followed by a nine-month placebo-controlled phase focused on PTC518 biomarker effect. In June 2023, the Company announced interim data from the 12-week placebo-controlled phase. The Phase 2 study is actively ongoing outside the United States, while it has been paused within the United States as the FDA requested additional data to allow the Phase 2 study to proceed. The Company expects results from the 12-month interim data from the Phase 2 study of PTC518 for the treatment of HD in the second quarter of 2024. The Company expects to submit a safety data update to the FDA in the second quarter of 2024 to support lifting of the partial clinical hold on the program. The Company’s ferroptosis and inflammation platform consists of small molecule compounds that target oxidoreductase enzymes that regulate oxidative stress and inflammatory pathways central to the pathology of a number of CNS diseases. The two most advanced molecules in the Company’s ferroptosis and inflammation platform are vatiquinone and utreloxastat. The Company announced topline results from a registration-directed Phase 3 trial of vatiquinone in children and young adults with Friedreich ataxia, called MOVE-FA, in May 2023. While the study did not meet its primary endpoint, vatiquinone treatment did demonstrate significant benefit on key disease subscales, including the upright stability subscale, as well as on other disease relevant endpoints. In the first quarter of 2024, the Company met with the FDA, who expressed willingness to review an NDA for vatiquinone for the treatment of Friedreich ataxia based on the MOVE-FA trial as well as data from the ongoing open label extension study following the MOVE-FA trial, potentially allowing for the submission of an NDA in late 2024. In the first quarter of 2024, the Company also received scientific advice from the EMA on the MOVE-FA trial results, in which the EMA stated that the MOVE-FA data would likely not be sufficient for conditional authorization. The Company initiated a Phase 2 registration directed trial of utreloxastat for amyotrophic lateral sclerosis, or ALS, in the first quarter of 2022. The Company expect topline results from this trial in the fourth quarter of 2024. In addition, the Company has a pipeline of product candidates and discovery programs that are in early clinical, pre-clinical and research and development stages focused on the development of new treatments for multiple therapeutic areas for rare diseases. As of December 31, 2023, the Company had an accumulated deficit of approximately $3,283.6 million. The Company has financed its operations to date primarily through the private offerings of convertible senior notes (see Note 7), public and “at the market offerings” of common stock, proceeds from royalty purchase agreements (see Note 2), net proceeds from its borrowings under its credit agreement with Blackstone (see Note 7), private placements of its convertible preferred stock and common stock, collaborations, bank and institutional lender debt, other convertible debt, grant funding and clinical trial support from governmental and philanthropic organizations and patient advocacy groups in the disease area addressed by the Company’s product candidates. The Company has also relied on revenue generated from net sales of Translarna for the treatment of nmDMD in territories outside of the United States since 2014, Emflaza for the treatment of DMD in the United States since 2017 and Upstaza for the treatment of AADC deficiency in the EEA since May 2022. The Company has also relied on revenue associated with milestone and royalty payments from Roche pursuant to the License and Collaboration Agreement (the “SMA License Agreement”) dated as of November 23, 2011, by and among the Company, Roche and, for the limited purposes set forth therein, the SMA Foundation, under its SMA program. The Company expects that cash flows from the sales of its products, together with the Company’s cash, cash equivalents and marketable securities, will be sufficient to fund its operations for at least the next twelve months. |
Summary of significant accounti
Summary of significant accounting policies | 12 Months Ended |
Dec. 31, 2023 | |
Accounting Policies [Abstract] | |
Summary of significant accounting policies | 2. Summary of significant accounting policies Basis of presentation The accompanying consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) and include all adjustments necessary for the fair presentation of the Company’s financial position for the periods presented. Use of estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Significant estimates in these consolidated financial statements have been made in connection with the calculation of net product sales, royalty revenue, certain accruals related to the Company’s research and development expenses, valuation procedures for liability for sale of future royalties, indefinite lived intangible assets annual impairment assessment, fair value of the contingent consideration, and the provision for or benefit from income taxes. Actual results could differ from those estimates. Changes in estimates are reflected in reported results in the period in which they become known. Restricted Cash Restricted cash included in deposits and other assets on the consolidated balance sheet relates to an unconditional, irrevocable and transferable letter of credit that was entered into during the twelve-month period ended December 31, 2019 in connection with obligations under a facility lease for the Company’s leased biologics manufacturing facility in Hopewell Township, New Jersey. The amount of the letter of credit is $7.5 million, is to be maintained for a term of not less than five years and has the potential to be reduced to $3.8 million if after five years from the lease commencement the Company is not in default of its lease. Restricted cash also contains an unconditional, irrevocable and transferable letter of credit that was entered into during June 2022 in connection with obligations for the Company’s new facility lease in Warren, New Jersey. The amount of the letter of credit is $8.1 million and has the potential to be reduced to $4.1 million if after five years the Company is not in default of its lease. Both amounts are classified within deposits and other assets on the consolidated balance sheet due to the long-term nature of the letter of credit. Restricted cash also includes a bank guarantee of $0.6 million denominated in a foreign currency. The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the consolidated balance sheet that sum to the total of the same amounts shown in the statement of cash flows: End of Beginning of period- period- December 31, December 31, 2023 2022 Cash and cash equivalents $ 594,001 $ 279,834 Restricted cash included in deposits and other assets 16,283 16,091 Total Cash, cash equivalents and restricted cash per statement of cash flows $ 610,284 $ 295,925 Consolidation The consolidated financial statements include the accounts of PTC Therapeutics, Inc. and its wholly owned subsidiaries. All inter-company accounts, transactions, and profits have been eliminated in consolidation. Segment and geographic information Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision making group, in deciding how to allocate resources and in assessing performance. The Company views its operations and manages its business in one operating and reporting Cash equivalents The Company considers all highly liquid investments with a maturity of 90 days or less at the time of purchase to be cash equivalents. Cash equivalents are carried at cost which approximates fair value due to their short-term nature. Marketable securities The Company’s marketable securities consists of both debt securities and equity investments. The Company considers its investments in debt securities with original maturities of greater than 90 days to be available for sale securities. Securities under this classification are recorded at fair value and unrealized gains and losses within accumulated other comprehensive income. The estimated fair value of the available for sale securities is determined based on quoted market prices or rates for similar instruments. In addition, the cost of debt securities in this category is adjusted for amortization of premium and accretion of discount to maturity. For available for sale debt securities in an unrealized loss position, the Company assesses whether it intends to sell or if it is more likely than not that the Company will be required to sell the security before recovery of its amortized cost basis. If either of the criteria regarding intent or requirement to sell is met, the security’s amortized cost basis is written down to fair value. If the criteria are not met, the Company evaluates whether the decline in fair value has resulted from a credit loss or other factors. In making this assessment, management considers, among other factors, the extent to which fair value is less than amortized cost, any changes to the rating of the security by a rating agency, and adverse conditions specifically related to the security. If this assessment indicates that a credit loss exists, the present value of cash flows expected to be collected from the security are compared to the amortized cost basis of the security. If the present value of the cash flows expected to be collected is less than the amortized cost basis, a credit loss exists and an allowance for credit losses is recorded for the credit loss, limited by the amount that the fair value is less than the amortized costs basis. Any impairment that has not been recorded through an allowance for credit losses is recognized in other comprehensive income. For the years ended December 31, 2023 and 2022, no allowance was recorded for credit losses. Marketable securities that are equity investments are measured at fair value, as it is readily available, and as such are classified as Level 1 assets. Unrealized holding gains and losses for these equity investments are components of other income (expense), net within the consolidated statement of operations. Concentration of credit risk The Company’s financial instruments that are exposed to credit risks consist primarily of cash and cash equivalents, available-for-sale marketable securities and accounts receivable. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company has not experienced any credit losses in these accounts and does not believe it is exposed to any significant credit risk on these funds. The Company’s investment policy includes guidelines on the quality of the financial institutions and financial instruments the Company is allowed to invest in, which the Company believes minimizes the exposure to concentration of credit risk. The Company is subject to credit risk from its accounts receivable related to its product sales. The payment terms are predetermined and the Company evaluates the creditworthiness of each customer or distributor on a regular basis. The Company reserves all uninsured amounts billed directly to a patient until the time of cash receipt as collectability is not reasonably assured at the time the product is received. To date, the Company has not incurred any material credit losses. Fixed assets Fixed assets are stated at cost. Depreciation is computed starting when the asset is placed into service on a straight-line basis over the estimated useful life of the related asset as follows: Leasehold improvements Lesser of useful life or lease term Computer equipment and software 3 years Machinery and lab equipment 7 years Furniture and fixtures 7 years Inventory and cost of product sales Inventory Inventories are stated at the lower of cost and net realizable value with cost determined on a first-in, first-out basis by product. The Company capitalizes inventory costs associated with products following regulatory approval when future commercialization is considered probable and the future economic benefit is expected to be realized. Products which may be used in clinical development programs are included in inventory and charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes. Inventory used for marketing efforts are charged to selling, general and administrative expense. Amounts related to clinical development programs and marketing efforts are immaterial. The following table summarizes the components of the Company’s inventory for the periods indicated: December 31, 2023 December 31, 2022 Raw materials $ 952 $ 1,078 Work in progress 17,991 14,074 Finished goods 11,634 6,656 Total inventory $ 30,577 $ 21,808 The Company periodically reviews its inventories for excess amounts or obsolescence and writes down obsolete or otherwise unmarketable inventory to its estimated net realizable value. The Company recorded write downs of $12.5 million and $1.7 million for the years ended December 31, 2023 and 2022, respectively, primarily related to adjustments to inventory reserves and product approaching expiration. Additionally, though the Company’s product is subject to strict quality control and monitoring which it performs throughout the manufacturing processes, certain batches or units of product may not meet quality specifications resulting in a charge to cost of product sales. For the years ended December 31, 2023 and December 31, 2022, these amounts were immaterial. Cost of product sales Cost of product sales consists of the cost of inventory sold, manufacturing and supply chain costs, storage costs, amortization of the acquired intangible asset, royalty payments associated with net product sales, and royalty payments to collaborative partners associated with royalty revenues and collaboration revenue related to milestones. Production costs are expensed as cost of product sales when the related products are sold or royalty revenues and collaboration revenue milestones are earned. Accumulated other comprehensive income (loss) Accumulated other comprehensive income (loss) consists of unrealized gains or losses on marketable securities and foreign currency translation adjustments. Revenue recognition Net product revenue The Company’s net product revenue primarily consists of sales of Translarna in territories outside of the U.S. for the treatment of nmDMD and sales of Emflaza in the U.S. for the treatment of DMD. The Company recognizes revenue when its performance obligations with its customers have been satisfied. The Company’s performance obligations are to provide products based on customer orders from distributors, hospitals, specialty pharmacies or retail pharmacies. The performance obligations are satisfied at a point in time when the Company’s customer obtains control of the product, which is typically upon delivery. The Company invoices its customers after the products have been delivered and invoice payments are generally due within 30 to 90 days of the invoice date. The Company determines the transaction price based on fixed consideration in its contractual agreements. Contract liabilities arise in certain circumstances when consideration is due for goods the Company has yet to provide. As the Company has identified only one distinct performance obligation, the transaction price is allocated entirely to product sales. In determining the transaction price, a significant financing component does not exist since the timing from when the Company delivers product to when the customers pay for the product is typically less than one year. Customers in certain countries pay in advance of product delivery. In those instances, payment and delivery typically occur in the same month. The Company records product sales net of any variable consideration, which includes discounts, allowances, rebates related to Medicaid and other government pricing programs, and distribution fees. The Company uses the expected value or most likely amount method when estimating its variable consideration, unless discount or rebate terms are specified within contracts. The identified variable consideration is recorded as a reduction of revenue at the time revenues from product sales are recognized. These estimates for variable consideration are adjusted to reflect known changes in factors and may impact such estimates in the quarter those changes are known. Revenue recognized does not include amounts of variable consideration that are constrained. During the years ended December 31, 2023, 2022, and 2021, net product sales in the United States were $255.1 million, $218.3 million, and $187.3 million, respectively, consisting solely of sales of Emflaza, and net product sales outside of the United States were $406.1 million, $316.9 million, and $241.6 million respectively, consisting of sales of Translarna, Tegsedi, Waylivra, and Upstaza. Translarna net product revenues made up $355.8 million, $288.6 million, and $236.0 million of the net product sales outside of the United States for the years ended December 31, 2023, 2022, and 2021, respectively. During the years ended December 31, 2023 and 2022, two countries, the United States and Russia, accounted for at least 10% of the Company’s net product sales, representing $255.1 million and $86.0 million, and $218.3 million and $59.7 million of net product sales, respectively. During the year ended December 31, 2021, only the United States In relation to customer contracts, the Company incurs costs to fulfill a contract but does not incur costs to obtain a contract. These costs to fulfill a contract do not meet the criteria for capitalization and are expensed as incurred. The Company considers any shipping and handling costs that are incurred after the customer has obtained control of the product as a cost to fulfill a promise. Shipping and handling costs associated with finished goods delivered to customers are recorded as a selling expense. Collaboration and royalty revenue The terms of these agreements typically include payments to the Company of one or more of the following: nonrefundable, upfront license fees; milestone payments; research funding and royalties on future product sales. In addition, the Company generates service revenue through agreements that generally provide for fees for research and development services and may include additional payments upon achievement of specified events. At the inception of a collaboration arrangement, the Company needs to first evaluate if the arrangement meets the criteria in Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) Topic 808 “Collaborative Arrangements” to then determine if ASC Topic 606 is applicable by considering whether the collaborator meets the definition of a customer. If the criteria are met, the Company assesses the promises in the arrangement to identify distinct performance obligations. For licenses of intellectual property, the Company assesses, at contract inception, whether the intellectual property is distinct from other performance obligations identified in the arrangement. If the licensing of intellectual property is determined to be distinct, revenue is recognized for nonrefundable, upfront license fees when the license is transferred to the customer and the customer can use and benefit from the license. If the licensing of intellectual property is determined not to be distinct, then the license will be bundled with other promises in the arrangement into one distinct performance obligation. The Company needs to determine if the bundled performance obligation is satisfied over time or at a point in time. If the Company concludes that the nonrefundable, upfront license fees will be recognized over time, the Company will need to assess the appropriate method of measuring proportional performance. For milestone payments, the Company assesses, at contract inception, whether the development or sales-based milestones are considered probable of being achieved. If it is probable that a significant revenue reversal will occur, the Company will not record revenue until the uncertainty has been resolved. Milestone payments that are contingent upon regulatory approval are not considered probable of being achieved until the applicable regulatory approvals or other external conditions are obtained as such conditions are not within the Company’s control. If it is probable that a significant revenue reversal will not occur, the Company will estimate the milestone payments using the most likely amount method. The Company will re-assess the development and sales-based milestones each reporting period to determine the probability of achievement. The Company recognizes royalties from product sales at the later of when the related sales occur or when the performance obligation to which the royalty has been allocated has been satisfied. If it is probable that a significant revenue reversal will not occur, the Company will estimate the royalty payments using the most likely amount method. The Company recognizes revenue for reimbursements of research and development costs under collaboration agreements as the services are performed. The Company records these reimbursements as revenue and not as a reduction of research and development expenses as the Company has the risks and rewards as the principal in the research and development activities. For the years ended December 31, 2023, 2022, and 2021, the Company has recognized $100.0 million, $50.1 million, and $55.0 million of collaboration revenue, respectively, related to the SMA License Agreement with Roche. For the years ended December 31, 2023, 2022 and 2021, the Company has recognized $168.9 million, $113.5 million, and $54.6 million of royalty revenue, respectively, related to Evrysdi. Manufacturing Revenue The Company has manufacturing services related to the production of plasmid deoxyribonucleic acid (“DNA”) and adeno-associated virus (“AAV”) vectors for gene therapy applications for external customers. Performance obligations vary but may include manufacturing plasmid DNA and/or AAV vectors, material testing, stability studies, and other services related to material development. The transaction prices for these arrangements are fixed and include amounts stated in the contracts for each promised service. Typically, the performance obligations within a manufacturing contract are highly interdependent, in which case, the Company will combine them into a single performance obligation. The Company has determined that the assets created have no alternative use to the Company, and the Company has an enforceable right to payment for the performance completed to date, therefore revenue related to these services are recognized over time and is measured using an output method based on performance of manufacturing milestones completed to date. Manufacturing service contracts may also include performance obligations related to project management services or obtaining materials from third parties. The Company has determined that these are separate performance obligations for which revenue is recognized at the point in time the services are performed. For performance obligations related to obtaining third party materials, the Company has determined that it is the principal as the Company has control of the materials and has discretion in setting the price. Therefore, the Company recognizes revenue on a gross basis related to obtaining third party materials. Certain arrangements require a portion of the contract consideration to be received in advance at the commencement of the contract, and such advance payment is initially recorded as a contract liability. A contract asset may be recognized in the event the Company’s satisfaction of performance obligations outpaces customer billings. For the year ended December 31, 2023, the Company recognized $7.7 million of manufacturing revenue related to plasmid DNA and AAV vector production for external customers. No manufacturing revenue was recognized for the years ended December 31, 2022 and 2021. As of December 31, 2023, the Company has contract assets of $0.2 million and remaining performance obligations of $0.8 million related to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers. For the period ended December 31, 2022, the Company had remaining performance obligations of $1.4 million and no contracts assets related to plasmid DNA and AAV production for external customers. Allowance for doubtful accounts The Company maintains an allowance for estimated losses resulting from the inability of its customers to make required payments. The Company estimates uncollectible amounts based upon current customer receivable balances, the age of customer receivable balances, the customer’s financial condition and current economic trends. The Company also assesses whether an allowance for expected credit losses may be required which includes a review of the Company’s receivables portfolio, which are pooled on a customer basis or country basis. In making its assessment of whether an allowance for credit losses is required, the Company considers its historical experience with customers, current balances, levels of delinquency, regulatory and legal environments, and other relevant current and future forecasted economic conditions Liability for sale of future royalties On July 17, 2020, the Company, RPI Intermediate Finance Trust (“RPI”), and, for the limited purposes set forth in the agreement, Royalty Pharma PLC, entered into a royalty purchase agreement (the “Original Royalty Purchase Agreement”). Pursuant to the Original Royalty Purchase Agreement, the Company sold to RPI 42.933% (the “Original Assigned Royalty Rights”) of the Company’s right to receive sales-based royalty payments (the “Royalty”) on worldwide net sales of Evrysdi and any other product developed pursuant to the License and Collaboration Agreement (the “License Agreement”), dated as of November 23, 2011, by and among the Company, Roche and, for the limited purposes set forth therein, the SMA Foundation under the SMA program. In consideration for the sale of the Original Assigned Royalty Rights, RPI paid the Company $650.0 million in cash consideration. The Company has retained a 57.067% interest in the Royalty and all economic rights to receive the remaining potential regulatory and sales milestone payments under the License Agreement, which remaining milestone payments equal $150.0 million in the aggregate as of December 31, 2023. The Original Royalty Purchase Agreement was set to terminate 60 days following the earlier of the date on which Roche is no longer obligated to make any payments of the Royalty pursuant to the License Agreement and the date on which RPI has received $1.3 billion in respect of the Original Assigned Royalty Rights. Pursuant to the guidance in ASC 470-10-25-2, the Company determined that the $650.0 million cash consideration obtained pursuant to the Original Royalty Purchase Agreement should be classified as debt and recorded it as “liability for sale of future royalties-current” and “liability for sale of future royalties-noncurrent” on the Company’s consolidated balance sheet based on the timing of the expected payments to be made to RPI at the time of the transaction. The liability was subsequently amortized using the effective interest method over the life of the arrangement, in accordance with the respective guidance, utilizing the prospective method to account for subsequent changes in the estimated future payments to be made to RPI. On October 18, 2023, the Company, Royalty Pharma Investments 2019 ICAV (“Royalty Pharma”), and, for the limited purposes set forth in the agreement, Royalty Pharma plc, entered into an Amended and Restated Royalty Purchase Agreement (the “A&R Royalty Purchase Agreement”), which amends and restates in its entirety the Original Royalty Purchase Agreement. Pursuant to the A&R Royalty Purchase Agreement, the Company has sold or agreed to sell to Royalty Pharma certain portions of the Company’s remaining Royalty on worldwide net sales of Evrysdi and any other product (the “Products”) developed pursuant to the SMA License Agreement (all such retained Royalty rights of the Company, the “Retained Royalty Rights,” and all such Royalty rights that are sold to Royalty Pharma pursuant to the A&R Royalty Purchase Agreement, the “A&R Assigned Royalty Rights”). At closing, Royalty Pharma paid the Company $1.0 billion in cash consideration for 38.0447% of the Company’s Retained Royalty Rights (which is in addition to the 42.9330% assigned to Royalty Pharma in connection with the Original Royalty Purchase Agreement, for a total of 80.9777% of the total Royalty) until such time as Royalty Pharma has received payments in respect of the Original Assigned Royalty Rights equal to $1.3 billion in the aggregate, and thereafter 66.6667% of the total Royalty. In addition, the Company may sell to Royalty Pharma the remainder of the Company’s Retained Royalty Rights in exchange for an aggregate of $500.0 million in additional cash consideration after the closing of the A&R Royalty Purchase Agreement, less royalties received in respect of the Retained Royalty Rights put to Royalty Pharma, which will be payable by Royalty Pharma pursuant to five put options held by the Company that are exercisable at the Company’s option between January 1, 2024 and December 31, 2025. If the Company exercises two or fewer of the put options, Royalty Pharma may exercise a call option during the period from and after January 1, 2026 until and including March 31, 2026 for up to 50% of the remainder of the Company’s Retained Royalty Rights less amounts exercised by the Company via its put options at a purchase price that is proportional to the purchase price of the Company’s unexercised put options. Royalty Pharma’s exercise of the call option would result in Royalty Pharma owning 90.4888% of the total Royalty until such time as Royalty Pharma has received payments in respect of the Original Assigned Royalty Rights equal to $1.3 billion in the aggregate, and thereafter 83.3333% of the total Royalty. The A&R Royalty Purchase Agreement will terminate 60 days following the date on which Roche is no longer obligated to make any payments of the Royalty pursuant to the License Agreement. The change in rights and obligations from the A&R Royalty Purchase Agreement resulted in a change in the terms of the liability for sale of future royalties, which was evaluated by the Company in accordance with ASC 470-50, Debt — Modifications and Extinguishments. The Company determined that the present value of the cash flows under the A&R Royalty Purchase Agreement were substantially different from the present value of the cash flows under the Original Royalty Purchase Agreement. This resulted in the derecognition of the old liability for sale of future royalties and the new liability for sale of future royalties being recorded at fair value, which was determined to be $1,809.9 million as of the date of the A&R Royalty Purchase Agreement. This resulted in an extinguishment loss of $44.9 million, which was recorded within loss on extinguishment of debt, within the Company’s statement of operations. The fair value for the new liability for sale of future royalties on the date of the A&R Royalty Purchase Agreement was based on the Company’s estimates of future royalties expected to be paid to Royalty Pharma over the life of the arrangement, which was determined using forecasts from market data sources, which are considered Level 3 inputs. The liability is being amortized using the effective interest method over the life of the arrangement, in accordance with ASC 470 and ASC 835. The initial annual effective interest rate was determined to be 10.8%. The Company utilizes the prospective method to account for subsequent changes in the estimated future payments to be made to Royalty Pharma and updates the effective interest rate on a quarterly basis. Issuance costs related to the transaction were determined to be immaterial. Refer to Note 7 for further details. Leases The Company determines if an arrangement is a lease at inception. This determination generally depends on whether the arrangement conveys to the Company the right to control the use of an explicitly or implicitly identified fixed asset for a period of time in exchange for consideration. Control of an underlying asset is conveyed to the Company if the Company obtains the rights to direct the use of and to obtain substantially all of the economic benefits from using the underlying asset. The Company has lease agreements which include lease and non-lease components, which the Company accounts for as a single lease component for all leases. Operating and finance leases are classified as right of use ("ROU") assets, short term lease liabilities, and long term lease liabilities. Operating and finance lease ROU assets and lease liabilities are recognized at the commencement date based on the present value of lease payments over the lease term. ROU assets are Variable lease payments associated with the Company’s leases are recognized when the event, activity, or circumstance in the lease agreement on which those payments are assessed occurs. Variable lease payments are presented in the Company’s consolidated statements of operations in the same line item as expense arising from fixed lease payments for operating leases. Leases with an initial term of 12 months or less are not recorded on the consolidated balance sheet and the Company recognizes lease expense for these leases on a straight-line basis over the lease term. The Company applies this policy to all underlying asset categories. A lessee is required to discount its unpaid lease payments using the interest rate implicit in the lease or, if that rate cannot be readily determined, its incremental borrowing rate. As most of the Company’s leases do not provide an implicit rate, the Company uses its incremental borrowing rate based on the information available at the commencement date in determining the present value of lease payments. The Company gives consideration to its recent debt issuances as well as publicly available data for instruments with similar characteristics when calculating its incremental borrowing rates. The lease term for all of the Company’s leases includes the non-cancellable period of the lease plus any additional periods covered by either a Company option to extend (or not to terminate) the le certain to exercise, or an option to extend (or not to terminate) the lease controlled by the lessor. Leasehold improvements are capitalized and depreciated over the lesser of useful life or lease term. See Note 5 Leases for additional information. Research and development costs Research and development expenses include the clinical development costs associated with the Company’s product development programs and research and development costs associated with the Company’s discovery programs. These expenses include internal research and development costs and the costs of research and development conducted on behalf of the Company by third parties, including sponsored university-based research agreements and clinical study vendors. All research and development costs are expensed as incurred. Costs incurred in obtaining technology licenses are charged immediately to research and development expense if the technology licensed has not reached technological feasibility and has no alternative future uses. Advance payments made for goods and services that will be used in future research and development activities are deferred if the contracted party has not yet performed the related activities. The amount deferred is then expensed when the research and development activities are performed. As of December 31, 2023 and 2022, the short term deferred research and development advance payments were $2.6 million and $2.4 million, respectively, and are classified as prepaid expenses and other current assets on the consolidated balance sheet. As of December 31, 2023 and 2022, the long term deferred research and development advance payments were $4.7 million and $3.9 million, respectively, and are classified as deposits and other assets on the consolidated balance sheet. Fair value of financial instrume |
Fair value of financial instrum
Fair value of financial instruments and investments | 12 Months Ended |
Dec. 31, 2023 | |
Fair Value Disclosures [Abstract] | |
Fair value of financial instruments and investments | The Company follows the fair value measurement rules, which provide guidance on the use of fair value in accounting and disclosure for assets and liabilities when such accounting and disclosure is called for by other accounting literature. Company’s marketable securities are classified as Level 2 as they primarily utilize broker quotes in a nonactive market to value these securities. The Company owns common stock in ClearPoint Neuro, Inc. (“ClearPoint”) (formerly MRI Interventions, Inc.), a publicly traded medical device company. The ClearPoint equity investments (collectively, the “ClearPoint Equity Investments”) represent financial instruments, and therefore, are recorded at fair value, which is readily determinable. The ClearPoint Equity Investments are components of prepaids and other current assets as of December 31, 2023 and deposits and other assets as of December 31, 2022 on the consolidated balance sheet. The Company classifies its equity investments in ClearPoint as a Level 1 asset within the fair value hierarchy, as the value is based on a quoted market price in an active market, which is not adjusted. In January 2020, the Company purchased a $10.0 million convertible note from ClearPoint that the Company can convert into ClearPoint shares at a conversion rate of $6.00 per share at any point throughout the term of the loan, which matures five years from the purchase date. The Company determined that the convertible note represents an available for sale debt security and the Company has elected to record it at fair value under ASC 825. The Company classifies its ClearPoint convertible debt security as a Level 2 asset within the fair value hierarchy, as the value is based on inputs other than quoted prices that are observable. The fair value of the ClearPoint convertible debt security is determined at each reporting period by utilizing a Black-Scholes option pricing model, as well as a present value of expected cash flows from the debt security utilizing the risk free rate and the estimated credit spread as of the valuation date as the discount rate. The convertible debt security is considered to be long term and is included as a component of deposits and other assets on the consolidated balance sheet. Other than the ClearPoint Equity Investment and the ClearPoint convertible debt security, no other items included in deposits and other assets and prepaids and other current assets on the consolidated balance sheets are fair valued. The Company has investments in mutual funds, including one that is denominated in a foreign currency. All of these are equity investments and are classified as marketable securities on the Company’s consolidated balance sheets. These equity investments are reported at fair value, as they are readily available, and as such are classified as Level 1 assets. Unrealized holding gains and losses for these equity investments are included as components of other income (expense), net within the consolidated statement of operations. The table presented below is a summary of changes in the fair value for the Company’s marketable securities – equity investments, ClearPoint Equity Investments, and ClearPoint convertible debt security for the years ended December 31, 2023 and 2022: Ending Foreign Ending Balance at Currency Balance at December 31, Unrealized Realized Unrealized Investments Redemptions/ December 31, 2022 Gain/(Loss) Gain/(Loss) Gain Purchased Sale 2023 Marketable securities - equity investments $ 108,261 2,517 4,383 1,384 38,432 (132,343) $ 22,634 ClearPoint Equity Investments 10,965 (1,515) (782) — — (2,594) 6,074 ClearPoint Convertible Debt Security 15,231 (2,678) — — — — 12,553 Total Fair Value $ 134,457 $ (1,676) $ 3,601 $ 1,384 $ 38,432 $ (134,937) $ 41,261 Ending Foreign Ending Balance at Currency Balance at December 31, Unrealized Unrealized Investments December 31, 2021 Gain/(Loss) Gain/(Loss) Purchased Redemptions 2022 Marketable securities - equity investments $ 206,973 (7,992) (549) 22,787 (112,958) $ 108,261 ClearPoint Equity Investments 14,525 (3,560) — — — 10,965 ClearPoint Convertible Debt Security 20,971 (5,740) — — — 15,231 Total Fair Value $ 242,469 $ (17,292) $ (549) $ 22,787 $ (112,958) $ 134,457 Fair value of marketable securities that are classified as available for sale debt securities is based upon market prices using quoted prices in active markets for identical assets quoted on the last day of the period. In establishing the estimated fair value of the remaining available for sale debt securities, the Company used the fair value as determined by its investment advisors using observable inputs other than quoted prices. The following represents the fair value using the hierarchy described in Note 2 for the Company’s financial assets and liabilities that are required to be measured at fair value on a recurring basis as of December 31, 2023 and 2022: December 31, 2023 Quoted prices Significant in active other Significant markets for observable unobservable identical assets inputs inputs Total (level 1) (level 2) (level 3) Marketable securities - available for sale $ 260,104 $ — $ 260,104 $ — Marketable securities - equity investments $ 22,634 $ 22,634 $ — $ — ClearPoint Equity Investments $ 6,074 $ 6,074 $ — $ — ClearPoint convertible debt security $ 12,553 $ — $ 12,553 $ — Contingent consideration payable- development and regulatory milestones $ 26,600 $ — $ — $ 26,600 Contingent consideration payable- net sales milestones $ 9,700 $ — $ — $ 9,700 December 31, 2022 Quoted prices Significant in active other Significant markets for observable unobservable identical assets inputs inputs Total (level 1) (level 2) (level 3) Marketable securities - available for sale $ 22,610 $ — $ 22,610 $ — Marketable securities - equity investments $ 108,261 $ 108,261 $ — $ — ClearPoint Equity Investments $ 10,965 $ 10,965 $ — $ — ClearPoint convertible debt security $ 15,231 $ — $ 15,231 $ — Contingent consideration payable- development and regulatory milestones $ 82,500 $ — $ — $ 82,500 Contingent consideration payable- net sales milestones and royalties $ 81,500 $ — $ — $ 81,500 No transfers of assets between Level 1 and Level 2 of the fair value measurement hierarchy occurred during the years ended December 31, 2023 and 2022. The following is a summary of marketable securities accounted for as available for sale debt securities at December 31, 2023 and 2022: December 31, 2023 Amortized Gross Unrealized Cost Gains Losses Fair Value Commercial paper $ 117,044 $ 128 $ (12) $ 117,160 Corporate debt securities 1,650 — (2) 1,648 Government Obligations 141,084 212 — 141,296 Total $ 259,778 $ 340 $ (14) $ 260,104 December 31, 2022 Amortized Gross Unrealized Cost Gains Losses Fair Value Commercial paper $ 12,419 $ 5 $ — $ 12,424 Corporate debt securities 10,685 — (499) 10,186 Total $ 23,104 $ 5 $ (499) $ 22,610 For available for sale debt securities in an unrealized loss position, the Company assesses whether it intends to sell or if it is more likely than not that the Company will be required to sell the security before recovery of its amortized cost basis. If either of the criteria regarding intent or requirement to sell is met, the security’s amortized cost basis is written down to fair value. For the years ended December 31, 2023 and 2022, no write downs occurred. The Company does not intend to sell the investments and it is not more likely than not that the Company will be required to sell the investments before recovery of their amortized cost basis, which may be maturity. The Company also reviews its available for sale debt securities in an unrealized loss position and evaluates whether the decline in fair value has resulted from credit losses or other factors. This review is subjective, as it requires management to evaluate whether an event or change in circumstances has occurred in that period that may be related to credit issues. For the years ended December 31, 2023 and 2022, no allowance was recorded for credit losses. Unrealized gains and losses are reported as a component of accumulated other comprehensive (loss) income in stockholders’ deficit. For the year ended December 31, 2023, the Company had $0.3 million of realized losses from the sale of available for sale debt securities. For the year ended December 31, 2022, the Company had $4.0 million of realized gains from the sale of available for sale debt securities. Realized gains and losses are reported as a component of interest expense, net in the consolidated statement of operations. The unrealized losses and fair values of available for sale debt securities that have been in an unrealized loss position for a period of less than and greater than or equal to 12 months as of December 31, 2023 are as follows: December 31, 2023 Securities in an unrealized loss Securities in an unrealized loss position less than 12 months position greater than or equal to 12 months Total Unrealized losses Fair Value Unrealized losses Fair Value Unrealized losses Fair Value Commercial paper $ (12) 44,446 — — (12) 44,446 Corporate debt securities $ — — (2) 1,648 (2) $ 1,648 Total $ (12) $ 44,446 $ (2) $ 1,648 $ (14) $ 46,094 The unrealized losses and fair values of available for sale debt securities that have been in an unrealized loss position for a period of less than and greater than or equal to 12 months as of December 31, 2022 are as follows: December 31, 2022 Securities in an unrealized loss Securities in an unrealized loss position less than 12 months position greater than or equal to 12 months Total Unrealized losses Fair Value Unrealized losses Fair Value Unrealized losses Fair Value Corporate debt securities $ — — (499) 10,186 (499) $ 10,186 Total $ — $ — $ (499) $ 10,186 $ (499) $ 10,186 Available for sale debt securities on the balance sheet at December 31, 2023 and 2022 mature as follows: December 31, 2023 Less Than More Than 12 Months 12 Months Commercial paper $ 117,160 $ — Corporate debt securities 1,648 — Government obligations 141,296 — Total $ 260,104 $ — December 31, 2022 Less Than More Than 12 Months 12 Months Commercial paper $ 12,424 $ — Corporate debt securities — 10,186 Total $ 12,424 $ 10,186 The Company classifies all of its marketable securities as current as they are all either available for sale debt securities or equity investments and are available for current operations. Convertible senior notes In September 2019, the Company issued $287.5 million of 1.5% convertible senior notes due September 15, 2026 (the “2026 Convertible Notes). The fair value of the 2026 Convertible Notes, which differs from their carrying values, is influenced by interest rates, the Company’s stock price and stock price volatility and is determined by prices for the 2026 Convertible Notes observed in market trading which are Level 2 inputs. The estimated fair value of the 2026 Convertible Notes at December 31, 2023 and December 31, 2022 was $265.3 million and $281.7 million, respectively. Level 3 valuation The contingent consideration payable is fair valued each reporting period with the change in fair value recorded as a gain or loss within the change in the fair value of contingent consideration on the consolidated statements of operations. The fair value of the development and regulatory milestones are estimated utilizing a probability adjusted, discounted cash flow approach. The discount rates are estimated utilizing Corporate B rated bonds maturing in the years of expected payments based on the Company’s estimated development timelines for the acquired product candidate. The fair value of the net sales milestones is determined utilizing a valuation framework that estimates net sales volatility to simulate a range of possible payment scenarios. The average of the payments in these scenarios is then discounted to calculate present fair value. In May 2023, as part of the Company’s strategic portfolio prioritization, the Company decided to discontinue its preclinical and early research programs in its gene therapy platform, which included programs for Friedreich ataxia and Angelman syndrome. As a result, the Company fully impaired the Friedreich ataxia and Angelman syndrome intangible assets and determined that the fair value for all of the contingent consideration payable related to Friedreich ataxia and Angelman syndrome was $0. The change in fair value for the contingent consideration payable related to Friedreich ataxia and Angelman syndrome for the year ended December 31, 2023 was $128.4 million and is included in the change in fair value As of December 31, 2023, the weighted average discount rate for the Upstaza development and regulatory milestones was 5.9% and the weighted average probability of success was 90%. As of December 31, 2023, the weighted average discount rate for the Upstaza net sales milestones was 11.0% and the weighted average probability of success for the net sales milestones was 93%. The table presented below is a summary of changes in the fair value of the Company’s Level 3 valuation for the contingent consideration payables for the years ended December 31, 2023, and 2022: Contingent consideration payable- Contingent consideration payable- development and regulatory net sales milestones and royalties milestones - Agilis - Agilis Beginning balance as of December 31, 2021 $ 139,300 $ 100,600 Additions — — Change in fair value (6,800) (19,100) Payments (50,000) — Ending balance as of December 31, 2022 $ 82,500 $ 81,500 Additions — — Change in fair value (55,900) (71,800) Payments — — Ending balance as of December 31, 2023 $ 26,600 $ 9,700 In July 2022, the EC approved Upstaza for the treatment of AADC deficiency for patients 18 months and older within the EEA, which triggered a $50.0 million milestone payment to the former equityholders of Agilis in accordance with the terms of the Agilis Merger Agreement. In accordance with ASC 230, the portion of the $50.0 million milestone payment up to the acquisition date fair value of the contingent consideration liability is classified as a financing outflow and the amount paid in excess of the acquisition date fair value of that liability is classified as an operating outflow on the consolidated statements of cash flows for the year ended December 31, 2022. The following significant unobservable inputs were used in the valuation of the contingent consideration payables for the years ended December 31, 2023 and 2022: December 31, 2023 Fair Value Valuation Technique Unobservable Input Range Contingent consideration payable- $26,600 Probability-adjusted discounted cash flow Potential development and regulatory milestones $0 - $31 million Contingent considerable payable- net sales $9,700 Option-pricing model with Monte Carlo simulation Potential net sales milestones $0 - $50 million December 31, 2022 Fair Value Valuation Technique Unobservable Input Range Contingent consideration payable- $82,500 Probability-adjusted discounted cash flow Potential development and regulatory milestones $0 - $331 million Contingent considerable payable- net sales $81,500 Option-pricing model with Monte Carlo simulation Potential net sales milestones $0 - $150 million The contingent consideration payables are classified Level 3 liabilities as their valuation requires substantial judgment and estimation of factors that are not currently observable in the market. If different assumptions were used for the various inputs to the valuation approaches, including but not limited to, assumptions involving probability adjusted sales estimates for the gene therapy platform and estimated discount rates, the estimated fair value could be significantly higher or lower than the fair value determined. |
Fixed assets
Fixed assets | 12 Months Ended |
Dec. 31, 2023 | |
Property, Plant and Equipment [Abstract] | |
Fixed assets | 4. Fixed assets Fixed assets, net were as follows at December 31, 2023 and 2022: December 31, 2023 2022 Leasehold improvements $ 30,166 $ 28,969 Computer equipment and software 17,503 15,332 Machinery and lab equipment 62,837 47,496 Furniture and fixtures 3,849 3,812 Assets in process 24,008 14,349 138,363 109,958 Less accumulated depreciation (51,274) (37,368) Total $ 87,089 $ 72,590 Depreciation expense was approximately $13.9 million, $12.3 million, and $9.4 million for the years ended December 31, 2023, 2022, and 2021, respectively. |
Leases
Leases | 12 Months Ended |
Dec. 31, 2023 | |
Leases | |
Leases | 5. Leases The Company leases office space in South Plainfield, New Jersey for its principal office under two noncancelable operating leases through August 2024, in addition to office and laboratory space in Bridgewater, New Jersey and other locations throughout the United States, as well as office space in various countries for international employees primarily through workspace providers. The Company also leases approximately 220,500 square feet of office, manufacturing and laboratory space at a facility located in Hopewell Township, New Jersey (the “Campus”) pursuant to a Lease Agreement (the “Hopewell Lease”) with Hopewell Campus Owner LLC (the “Landlord”). The rental term of the Hopewell Lease commenced on July 1, 2020 and has an initial term of fifteen years (the “Hopewell Initial Term”), with two consecutive ten year renewal periods, each at the Company’s option. The aggregate rent for the Hopewell Initial Term will be approximately $111.5 million. The rental rate for the renewal periods will be 95% of the Prevailing Market Rate (as defined in the Hopewell Lease) and determined at the time of the exercise of the renewal. The Company is also responsible for maintaining certain insurance and the payment of proportional taxes, utilities and common area operating expenses. The Hopewell Lease contains customary events of default, representations, warranties and covenants. In May 2022, the Company entered into a Lease Agreement (the “Warren Lease”) with Warren CC Acquisitions, LLC (the “Warren Landlord”) relating to the lease of two entire buildings comprised of approximately 360,000 square feet of shell condition, modifiable space (the “Warren Premises”) at a facility located in Warren, New Jersey. The rental term of the Warren Lease commenced on June 1, 2022 (the “Commencement Date”), with an initial term of seventeen years (the “Warren Initial Term”), followed by three consecutive five-year renewal periods at the Company’s option. The aggregate base rent for the Warren Initial Term will be approximately $163.0 million; provided, however, that if the Company is not subject to an Event of Default (as defined in the Warren Lease), the Company will be entitled to a base rent abatement over the first three years of the Warren Initial Term of approximately $18.6 million, reducing the Company’s total base rent obligation to $144.4 million. The rental rate for the renewal periods will be at the Fair Market Rental Value (as defined in the Warren Lease) and determined at the time of the exercise of the renewal. Beginning in the second lease year, the Company is also responsible for the payment of all taxes and operating expenses for the Warren Premises. As a result, the Company recorded an operating lease ROU asset of $28.9 million and an operating lease ROU liability of $28.9 million as of the Commencement Date. The Company is developing the Warren Premises into office and laboratory space. The Company is entitled to an allowance of approximately $36.2 million to be provided by the Warren Landlord to be used towards such improvements. The Landlord is providing the allowance to cover those assets that are real property improvements, such as structural components, roofs, flooring, etc., whose useful lives are typically longer in nature. Upon the first issuance of a temporary certificate of occupancy for the Warren Premises, the Company will receive $5.0 million from the Landlord, which the Company has committed to fund into the construction account. The Company evaluated the leasehold improvements under ASC 842 and determined that the Company will be the owner of the improvements, and therefore the $36.2 million allowance and $5.0 million due from the Landlord were treated as lease incentives at the commencement of the lease and included in the calculation of the lease ROU asset and lease ROU liability, effectively reducing both at Commencement Date. The Company also has a finance lease related to its commercial manufacturing agreement with MassBiologics of the University of Massachusetts Medical School (“MassBio”). As of December 31, 2023, the balance of the finance lease liabilities-current and finance lease liabilities-non-current are $3.0 million and $17.2 million, respectively, and are directly related to the Company’s MassBio agreement. As of December 31, 2022, the balance of the finance lease liabilities-current and finance lease liabilities non-current were $3.0 million and $18.7 million, respectively. Additionally, during the years ended December 31, 2023 and December 31, 2022, the Company recorded finance lease costs of $1.5 million and $1.6 million, respectively, related to interest on the lease liability. The Company also leases certain vehicles, lab equipment, and office equipment under operating leases. The Company’s operating leases have remaining lease terms ranging from 0.2 years to 15.4 years and certain leases include renewal options to extend the lease for up to 15 years. Rent expense was $29.0 million, $25.2 million, and $21.4 million for the years ended December 31, 2023, 2022 and 2021. The components of lease expense were as follows: Year Ended December 31, 2023 Year Ended December 31, 2022 Year Ended December 31, 2021 Operating Lease Cost Fixed lease cost $ 21,952 $ 19,804 $ 16,411 Variable lease cost 5,846 4,557 4,361 Short-term lease cost 1,186 808 656 Total operating lease cost $ 28,984 $ 25,169 $ 21,428 Total operating lease cost is a component of operating expenses on the consolidated statements of operations. Supplemental lease term and discount rate information related to leases was as follows: December 31, 2023 December 31, 2022 Weighted-average remaining lease terms - operating leases (years) 11.55 11.61 Weighted-average discount rate - operating leases 8.69 % 8.61 % Weighted-average remaining lease terms - finance lease (years) 9.01 10.01 Weighted-average discount rate - finance lease 7.80 % 7.80 % Supplemental cash flow information related to leases was as follows: Year Ended December 31, 2023 2022 2021 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows from operating leases $ 15,338 $ 14,736 $ 13,683 Financing cash flows from finance lease 1,379 1,276 2,224 Operating cash flows from finance lease 1,621 1,724 776 Right-of-use assets obtained in exchange for lease obligations: Operating leases $ — $ 35,817 $ 645 Future minimum lease payments under non-cancelable leases as of December 31, 2023 were as follows: Operating Leases Finance Lease 2024 $ 18,352 $ 3,000 2025 20,450 3,000 2026 19,992 3,000 2027 17,945 3,000 2028 and thereafter 175,847 15,000 Total lease payments 252,586 27,000 Less: Imputed Interest expense 141,957 6,816 Total $ 110,629 $ 20,184 |
Accounts payable and accrued ex
Accounts payable and accrued expenses | 12 Months Ended |
Dec. 31, 2023 | |
Payables and Accruals [Abstract] | |
Accounts payable and accrued expenses | 6. Accounts payable and accrued expenses Accounts payable and accrued expenses at December 31, 2023 and 2022 consist of the following: December 31, 2023 2022 Employee compensation, benefits, and related accruals $ 62,643 $ 62,669 Income tax payable — 4,712 Consulting and contracted research 27,500 38,882 Professional fees 2,246 3,093 Sales allowance 77,176 63,787 Sales rebates 131,334 67,355 Royalties 74,111 40,546 Accounts payable 6,045 27,268 Other 10,928 12,054 Total $ 391,983 $ 320,366 During the year ended December 31, 2023, the Company incurred $32.8 million of restructuring costs from a reduction in workforce in connection with the Company’s strategic pipeline prioritization and discontinuation of its preclinical and early research programs in its gene therapy platform. The costs are included in research and development and selling, general and administrative expenses on the Company’s consolidated statement of operations. As of December 31, 2023, the remaining $9.0 million of accrued restructuring costs are included above within employee compensation, benefits, and related accruals. |
Debt
Debt | 12 Months Ended |
Dec. 31, 2023 | |
Debt Disclosure [Abstract] | |
Debt | 7. Debt Liability for sale of future royalties The following table shows the activity within the “liability for sale of future royalties- current” and “liability for sale of future royalties- noncurrent” accounts for the year ended December 31, 2023: Year Ended December 31, Liability for sale of future royalties- (current and noncurrent) 2023 Beginning balance as of December 31, 2022 $ 757,886 Less: Non-cash royalty revenue payable to RPI (93,460) Plus: Non-cash interest expense recognized 104,790 Plus: Cash Consideration 1,000,000 Plus: Loss on Debt Extinguishment 44,881 Ending balance $ 1,814,097 Effective interest rate as of December 31, 2023 10.8% Non-cash interest expense is recorded in the statement of operations within “Interest expense, net”. Senior Secured Term Loan On October 27, 2022 (the “Closing Date”), the Company entered into a credit agreement (the “Blackstone Credit Agreement”) for fundings of up to $950.0 million consisting of a committed loan facility of $450.0 million and further contemplating the potential for up to $500.0 million of additional financing, to the extent that the Company requested such additional financing and subject to the Lenders’ agreement to provide such additional financing and to mutual agreement on terms, among the Company, certain subsidiaries of the Company (together with the Company, the “Loan Parties”) and funds and other affiliated entities advised or managed by Blackstone Life Sciences and Blackstone Credit (collectively, “Blackstone”, and such lenders, together with their permitted assignees, the “Lenders” and each a “Lender”) and Wilmington Trust, National Association, as the administrative agent for the Lenders. The Blackstone Credit Agreement provided for a senior secured term loan facility funded on the Closing Date in the aggregate principal amount of $300.0 million (the “Initial Loans”) and a committed delayed draw term loan facility of up to $150.0 million (the “Delayed Draw Loans” and, together with the Initial Loans, the “Loans”) to be funded at the Company’s request within 18 months of the Closing Date subject to specified conditions. In addition, the Blackstone Credit Agreement contemplated the potential for further financings by Blackstone, by providing for incremental discretionary uncommitted further financings of up to $500.0 million. The Company capitalized approximately $11.6 million of debt issuance costs which are presented on the balance sheet as a direct deduction from the debt liability and are being amortized over the term of the senior secured term loan facility using the effective interest rate method. The Loans were to mature on the date that is seven years from the Closing Date. Borrowings under the Blackstone Credit Agreement bore interest at a variable rate equal to, at the Company’s option, either an adjusted Term SOFR rate plus seven and a quarter percent (7.25%) or the Base Rate plus six and a quarter percent (6.25%), subject to a floor of one percent (1%) and two percent (2%) with respect to Term SOFR rate and Base Rate (each as defined in the Blackstone Credit Agreement), respectively. Payment of the Loans were subject to certain premiums specified in the Blackstone Credit Agreement, in each case, from the date of the applicable Loan funded. All obligations under the Blackstone Credit Agreement were secured, subject to certain exceptions and specified inclusions, by security interests in certain assets of the Loan Parties, including (1) intellectual property and other assets related to Translarna, Emflaza, Upstaza, sepiapterin and, until certain release conditions were met, vatiquinone, in each case, together with any other forms, formulations, or methods of delivery of any such products, and regardless of trade or brand name, (2) future acquired intellectual property (but not internally developed intellectual property unrelated to other intellectual property collateral) and other related assets, and (3) the equity interests held by the Loan Parties in certain of their subsidiaries. The Blackstone Credit Agreement contained certain negative covenants with which the Company was to remain in compliance. The Blackstone Credit Agreement also required that the Company maintained consolidated liquidity of at least $100.0 million as of the last day of each fiscal quarter, which was to be increased to $200.0 million upon the Company consummating acquisitions meeting certain consolidated thresholds described therein. In addition, the Company was required under conditions specified in the Blackstone Credit Agreement to fund a reserve account up to certain amounts specified therein. The funds in the reserve account were available to prepay the Loans at any time at the Company’s option, and were, if funded, subject to release upon certain further conditions. Upon any such release, such funds were freely available for use by the Company subject to the generally applicable terms and conditions of the Blackstone Credit Agreement. The Blackstone Credit Agreement contained certain customary representations and warranties, affirmative covenants and provisions relating to events of default. On October 19, 2023, the Company terminated the Blackstone Credit Agreement. In connection with the termination of the Credit Agreement, the Company repaid outstanding principal of $300.0 million, accrued interest of $2.1 million, an additional $82.0 million in prepayment premiums, exit fees, and creditor expenses, and $0.2 million in legal fees. The Company recorded a loss on the extinguishment of debt of $92.7 million which is included on the statement of operations for the period ended December 31, 2023. The loss on extinguishment of debt consisted of $82.0 million in prepayment premiums, exit fees, and creditor expenses and debt issuance costs of $10.7 million. All liens and security interests securing the loans made pursuant to the Blackstone Credit Agreement were released upon termination. The Blackstone Credit Agreement consisted of the following: Year ended December 31, 2023 2022 Principal $ 300,000 $ 300,000 Less: Debt issuance costs — (11,322) Repayment of senior secured term loan (300,000) — Net carrying amount $ — $ 288,678 The following table sets forth total interest expense recognized related to the Blackstone Credit Agreement: Year Ended Year Ended December 31, December 31, 2023 2022 Contractual interest expense $ 30,198 $ 6,069 Amortization of debt issuance costs 702 290 Total $ 30,900 $ 6,359 Effective interest rate 13.1 % 12.2 % 2026 Convertible Notes In September 2019, the Company issued, at par value, $287.5 million aggregate principal amount of 1.50% convertible senior notes due 2026, which included an option to purchase up to an additional $37.5 million in aggregate principal amount of the 2026 Convertible Notes, which was exercised in full by the initial purchasers. The 2026 Convertible Notes bear cash interest at a rate of 1.50% per year, payable semi-annually on March 15 and September 15 of each year, beginning on March 15, 2020. The 2026 Convertible Notes will mature on September 15, 2026, unless earlier repurchased or converted. The net proceeds to the Company from the offering were $279.3 million after deducting the initial purchasers’ discounts and commissions and the offering expenses payable by the Company. The 2026 Convertible Notes are governed by an indenture (the “2026 Convertible Notes Indenture”) with U.S Bank National Association as trustee (the “2026 Convertible Notes Trustee”). Holders of the 2026 Convertible Notes may convert their 2026 Convertible Notes at their option at any time prior to the close of business on the business day immediately preceding March 15, 2026 only under the following circumstances: ● during any calendar quarter commencing on or after December 31, 2019 (and only during such calendar quarter), if the last reported sale price of the Company’s common stock for at least 20 trading days (whether or not consecutive) during a period of 30 consecutive trading days ending on the last trading day of the immediately preceding calendar quarter is greater than or equal to 130% of the conversion price on each applicable trading day; ● during the five business day period after any five consecutive trading day period (the “measurement period”) in which the trading price (as defined in the 2026 Convertible Notes Indenture) per $1,000 principal amount of 2026 Convertible Notes for each trading day of the measurement period was less than 98% of the product of the last reported sale price of the Company’s common stock and the conversion rate on each such trading day; ● during any period after the Company has issued notice of redemption until the close of business on the scheduled trading day immediately preceding the relevant redemption date; or ● upon the occurrence of specified corporate events. On or after March 15, 2026, until the close of business on the business day immediately preceding the maturity date, holders may convert their 2026 Convertible Notes at any time, regardless of the foregoing circumstances. Upon conversion, the Company will pay or deliver, as the case may be, cash, shares of the Company’s common stock or any combination thereof at the Company’s election. The conversion rate for the 2026 Convertible Notes was initially, and remains, 19.0404 shares of the Company’s common stock per $1,000 principal amount of the 2026 Convertible Notes, which is equivalent to an initial conversion price of approximately $52.52 per share of the Company’s common stock. The conversion rate may be subject to adjustment in some events but will not be adjusted for any accrued and unpaid interest. The Company was not permitted to redeem the 2026 Convertible Notes prior to September 20, 2023. The Company may redeem for cash all or any portion of the 2026 Convertible Notes, at its option, if the last reported sale price of its common stock has been at least 130% of the conversion price then in effect on the last trading day of, and for at least 19 other trading days (whether or not consecutive) during, any 30 consecutive trading day period ending on, and including, the trading day immediately preceding the date on which the Company provides notice of redemption, at a redemption price equal to 100% of the principal amount of the 2026 Convertible Notes to be redeemed, plus accrued and unpaid interest to, but excluding, the redemption date. No sinking fund is provided for the 2026 Convertible Notes, which means that the Company is not required to redeem or retire the 2026 Convertible Notes periodically. If the Company undergoes a “fundamental change” (as defined in the 2026 Convertible Notes Indenture), subject to certain conditions, holders of the 2026 Convertible Notes may require the Company to repurchase for cash all or part of their 2026 Convertible Notes at a repurchase price equal to 100% of the principal amount of the 2026 Convertible Notes to be repurchased, plus accrued and unpaid interest to, but excluding, the fundamental change repurchase date. The 2026 Convertible Notes represent senior unsecured obligations and will rank senior in right of payment to the Company’s future indebtedness that is expressly subordinated in right of payment to the notes, equal in right of payment to the Company’s existing and future unsecured indebtedness that is not so subordinated, effectively junior in right of payment to any of the Company’s secured indebtedness to the extent of the value of the assets securing such indebtedness, and structurally subordinated to all existing and future indebtedness and other liabilities (including trade payables) incurred by the Company’s subsidiaries. The 2026 Convertible Notes Indenture contains customary events of default with respect to the 2026 Convertible Notes, including that upon certain events of default (including the Company’s failure to make any payment of principal or interest on the 2026 Convertible Notes when due and payable) occurring and continuing, the 2026 Convertible Notes Trustee by notice to the Company, or the holders of at least 25% in principal amount of the outstanding 2026 Convertible Notes by notice to the Company and the Convertible Notes Trustee, may, and the 2026 Convertible Notes Trustee at the request of such holders (subject to the provisions of the 2026 Convertible Notes Indenture) will, declare 100% of the principal of and accrued and unpaid interest, if any, on all the 2026 Convertible Notes to be due and payable. In case of certain events of bankruptcy, insolvency or reorganization, involving the Company or a significant subsidiary, 100% of the principal of and accrued and unpaid interest on the 2026 Convertible Notes will automatically become due and payable. Upon such a declaration of acceleration, such principal and accrued and unpaid interest, if any, will be due and payable immediately. Prior to the adoption of ASU 2020-06, the Company accounted for the 2026 Convertible Notes as a liability and equity component where the carrying value of the liability component was valued based on a similar instrument. In accounting for the issuance of the 2026 Convertible Notes, the Company separated the 2026 Convertible Notes into liability and equity components. The carrying amount of the liability component was calculated by measuring the fair value of a similar liability that did not have an associated convertible feature. The carrying amount of the equity component representing the conversion option was determined by deducting the fair value of the liability component from the par value of the 2026 Convertible Notes as a whole. The excess of the principal amount of the liability component over its carrying amount, referred to as the debt discount, was amortized to interest expense over the seven-year term of the 2026 Convertible Notes. The equity component was not re-measured as long as it continued to meet the conditions for equity classification. The equity component recorded at issuance related to the 2026 Convertible Notes was $123.0 million and was recorded in additional paid-in capital. In accounting for the transaction costs related to the issuance of the 2026 Convertible Notes, the Company allocated the total costs incurred to the liability and equity components of the 2026 Convertible Notes based on their relative values. Transaction costs attributable to the liability component were amortized to interest expense over the seven-year term of the 2026 Convertible Notes, and transaction costs attributable to the equity component were netted with the equity components in stockholders’ equity. Additionally, the Company initially recorded a net deferred tax liability of $25.3 million in connection with the 2026 Convertible Notes. Effective January 1, 2021 the Company adopted ASU 2020-06. After adoption, the Company now accounts for the 2026 Convertible Notes as a single liability measured at amortized cost. As the equity component is no longer required to be split into a separate component, the Company recorded an adjustment for the initial $123.0 million that was allocated to additional paid in capital and $16.1 million of life to date interest expense recorded as amortization of debt discount. Additionally, the net deferred tax liability recorded for the 2026 Convertible Notes was reversed. The principal amount of the liability over its carrying amount is amortized to interest expense over the seven-year term of the 2026 Convertible Notes. Since the 2026 Convertible Notes are classified as a single liability, there is no debt discount required to be amortized. The 2026 Convertible Notes consist of the following: Year ended December 31, 2023 2022 Principal $ 287,500 $ 287,500 Less: Debt issuance costs (3,287) (4,456) Net carrying amount $ 284,213 $ 283,044 As of December 31, 2023, the remaining contractual life of the 2026 Convertible Notes is approximately 2.7 years. The following table sets forth total interest expense recognized related to the 2026 Convertible Notes: Year ended December 31, 2023 2022 Contractual interest expense $ 4,305 $ 4,313 Amortization of debt issuance costs 1,171 1,150 Total $ 5,476 $ 5,463 Effective interest rate 1.9 % 1.9 % In April 2022, under the terms of the 2026 Convertible Notes Indenture, the Company paid additional interest on the 2026 Convertible Notes at a rate equal to 0.5% per annum, for a total interest payment of approximately $2.1 million, for the period beginning September 25, 2020 and ending March 14, 2022. In September 2022, under the terms of the 2026 Convertible Notes Indenture, the Company paid additional interest on the 2026 Convertible Notes at a rate equal to 0.5% per annum, for a total interest payment of approximately $0.1 million, for the period beginning March 15, 2022 and ending April 8, 2022. These amounts are not included in the table above, but were recorded as interest expense, net within the statement of operations for the year ended December 31, 2022. 2022 Convertible Notes In August 2015, the Company issued, at par value, $150.0 million aggregate principal amount of 3.00% convertible senior notes due 2022 (the “2022 Convertible Notes”). On August 15, 2022, the Company repaid the outstanding principal amount and accrued interest, totaling $152.3 million, of the 2022 Convertible Notes that was due upon maturity in accordance with the terms of the notes. The following table sets forth total interest expense recognized related to the 2022 Convertible Notes: Year ended December 31, 2023 2022 Contractual interest expense $ — $ 2,800 Amortization of debt issuance costs — 460 Total $ — $ 3,260 Effective interest rate — % 3.5 % |
Capital structure
Capital structure | 12 Months Ended |
Dec. 31, 2023 | |
Stockholders' Equity Note [Abstract] | |
Capital structure | 8. Capital structure Common stock In August 2019, the Company entered into an At the Market Offering Sales Agreement (the “Sales Agreement”) with Cantor Fitzgerald and RBC Capital Markets, LLC (together, the “Sales Agents”), pursuant to which, the Company may offer and sell shares of its common stock, having an aggregate offering price of up to $125.0 million from time to time through the Sales Agents by any method that is deemed to be an “at the market offering” as defined in Rule 415(a)(4) promulgated under the Securities Act of 1933, as amended. No shares were sold pursuant to the Sales Agreement during the years ended December 31, 2023, 2022, and 2021. The remaining shares of the Company’s common stock available to be issued and sold, under the Sales Agreement, have an aggregate offering price of up to $93.0 million as of December 31, 2023. In June 2021, the Company filed a Certificate of Amendment to its Restated Certificate of Incorporation, which increased the number of authorized shares of the Company’s common stock from 125,000,000 to 250,000,000 shares. In connection with the execution of the Blackstone Credit Agreement, the Company and certain entities affiliated with the Lenders (the “Purchasers”) also entered into a stock purchase agreement (the “Stock Purchase Agreement”) on the Closing Date for the sale and issuance of 1,095,290 shares of common stock (the “Shares”) to the Purchasers at a price of $45.65 per share, for an aggregate purchase price of approximately $50.0 million. The per share price represents the closing price of the Company’s common stock on the Nasdaq Global Select Market on October 26, 2022. Under the Stock Purchase Agreement, the Company agreed to register the resale of the Shares on a registration statement to be filed with the Securities and Exchange Commission within 60 days of the Closing Date. The Company agreed to keep such registration statement effective for a period of six months following the Closing Date. In addition, subject to certain conditions, the Purchasers were entitled to participate in registered underwritten public offerings by the Company during such period. Pursuant to the terms of the Stock Purchase Agreement, the Purchasers and certain of their controlled affiliates agreed not to, without the prior written approval of the Company and subject to specified conditions, directly or indirectly acquire shares of the Company’s outstanding common stock in excess of specified thresholds, seek or propose any acquisition of all or substantially all of the assets of the Company, seek or propose a merger or other business combination involving the Company, solicit proxies or consents with respect to any securities of the Company, seek to influence the management, board of directors or policies of the Company, or undertake other specified actions related to the potential acquisition of additional equity interests in the Company, or to encourage others to do any of the above (the “Standstill Restrictions”). The Standstill Restrictions terminated upon the termination of the Blackstone Credit Agreement. The Purchasers also agreed not to sell or transfer the Shares without the prior written approval of the Company for a period of 90 days following the Closing Date, subject to certain exceptions. In February 2023, the Company completed enrollment of its Phase 3 placebo-controlled clinical trial for sepiapterin for PKU. In connection with this event and pursuant to the Censa Merger Agreement, the Company paid a $30.0 million development milestone to the former Censa securityholders during the year ended December 31, 2023. The Company elected to pay this milestone in the form of shares of its common stock, less certain cash payments in accordance with the Censa Merger Agreement. Pursuant to such election, the Company issued 657,462 shares of its common stock and paid $0.4 million to the former Censa securityholders. As of December 31, 2023, the Company’s number of authorized shares of common stock was 250,000,000. |
Net loss per share
Net loss per share | 12 Months Ended |
Dec. 31, 2023 | |
Earnings Per Share [Abstract] | |
Net loss per share | 9. Net loss per share Basic and diluted net loss per share is computed by dividing net loss available to common stockholders by the weighted-average number of common shares outstanding. Potentially dilutive securities were excluded from the diluted calculation because their effect would be anti-dilutive. The following table sets forth the computation of basic and diluted net loss per share for common stockholders: Year ended December 31, 2023 2022 2021 Numerator Net loss $ (626,604) $ (559,017) $ (523,901) Denominator Denominator for basic and diluted net loss per share 74,838,392 71,728,634 70,466,393 Net loss per share: Basic and diluted $ (8.37) * $ (7.79) * $ (7.43) * * For the years ended December 31, 2023, 2022, and 2021, the Company experienced a net loss and therefore did not report any dilutive share impact. The following table shows historical dilutive common share equivalents outstanding, which are not included in the above historical calculation, as the effect of their inclusion is anti-dilutive during each period. As of December 31, 2023 2022 2021 Stock Options 9,600,399 11,502,417 10,772,582 Unvested restricted stock awards and units 2,866,270 2,516,336 1,519,831 Total 12,466,669 14,018,753 12,292,413 |
Stock award plan
Stock award plan | 12 Months Ended |
Dec. 31, 2023 | |
Share-based Payment Arrangement [Abstract] | |
Stock award plan | 10. Stock award plan In May 2013, the Company’s Board of Directors and stockholders approved the 2013 Long Term Incentive Plan, which became effective upon the closing of the Company’s IPO. The 2013 Long Term Incentive Plan provides for the grant of incentive stock options, nonstatutory stock options, restricted stock awards and other stock-based awards. On June 8, 2022 (the “Restatement Effective Date”), the Company’s stockholders approved the Amended and Restated 2013 Long-Term Incentive Plan (the “Amended 2013 LTIP”). The Amended 2013 LTIP provides for the grant of incentive stock options, nonstatutory stock options, restricted stock units and other stock-based awards. The number of shares of common stock reserved for issuance under the Amended 2013 LTIP is the sum of (A) the number of shares of the Company’s common stock (up to 16,724,212 shares) that is equal to the sum of (1) the number of shares issued under the 2013 Long-Term Incentive Plan prior to the Restatement Effective Date, (2) the number of shares that remain available for issuance under the 2013 Long-Term Incentive Plan immediately prior to the Restatement Effective Date and (3) the number of shares subject to awards granted under the 2013 Long-Term Incentive Plan prior to the Restatement Effective Date that are outstanding as of the Restatement Effective Date, plus (B) from and after the Restatement Effective Date, an additional 8,475,000 shares of Common Stock. As of December 31, 2023, awards for 8,680,209 shares of common stock were available for issuance under the Amended 2013 LTIP. There are no additional shares of common stock available for issuance under the Company’s 1998 Employee, Director and Consultant Stock Option Plan, 2009 Equity and Long Term Incentive Plan or 2013 Stock Incentive Plan. In January 2020, the Company’s Board of Directors approved the 2020 Inducement Stock Incentive Plan. The 2020 Inducement Stock Incentive Plan provides for the grant of incentive stock options, nonstatutory stock options, restricted stock awards and other stock-based awards, initially up to an aggregate of 1,000,000 shares of common stock. Any grants made under the 2020 Inducement Stock Incentive Plan must be made pursuant to the Nasdaq Listing Rule 5635(c)(4) inducement grant exception as a material component of the Company’s new hires’ employment compensation. In December 2020, the Company’s Board of Directors approved an additional 1,000,000 shares of common stock that may be issued under the 2020 Inducement Stock Incentive Plan. In April 2022, the Company’s Board of Directors approved a reduction in the total number of shares of common stock that may be issued under the 2020 Inducement Stock Incentive Plan to 1,300,000 shares. In December 2022, the Company’s Board of Directors approved an additional 1,700,000 shares of common stock that may be issued under the 2020 Inducement Stock Inventive Plan. As of December 31, 2023, awards for 1,834,322 shares of common stock are available for issuance under the 2020 Inducement Stock Incentive Plan. The Board of Directors has the authority to select the individuals to whom options are granted and determine the terms of each option, including (i) the number of shares of common stock subject to the option; (ii) the date on which the option becomes exercisable; (iii) the option exercise price, which, in the case of incentive stock options, must be at least 100% (110% in the case of incentive stock options granted to a stockholder owning in excess of 10% of the Company’s stock) of the fair market value of the common stock as of the date of grant; and (iv) the duration of the option (which, in the case of incentive stock options, may not exceed ten years). Options typically vest over a four-year period. Inducement stock option awards Pursuant to the Nasdaq inducement grant exception, during the year ended December 31, 2023, the Company issued options to purchase an aggregate of 139,040 shares of common stock to certain new hire employees at a weighted-average exercise price of $41.25 per share under the 2020 Inducement Stock Incentive Plan. Additionally, during the year ended December 31, 2023, the Company issued 105,370 restricted stock units under the 2020 Inducement Stock Incentive Plan. An aggregate of 270,583 of options and 79,195 of restricted stock units previously granted as inducement awards were forfeited during the year ended December 31, 2023 in connection with employee separations from the Company. Stock option activity Weighted- Weighted- average Aggregate average remaining intrinsic Number of exercise contractual value(in options price term thousands) Outstanding at December 31, 2020 9,663,677 $ 38.72 Granted 2,487,234 $ 61.36 Exercised (635,871) $ 28.01 Forfeited (742,458) $ 52.04 Outstanding at December 31, 2021 10,772,582 $ 43.66 Granted 1,685,435 $ 38.55 Exercised (496,863) $ 29.45 Forfeited (458,737) $ 48.75 Outstanding at December 31, 2022 11,502,417 $ 43.33 Granted 1,117,284 $ 40.19 Exercised (822,482) $ 29.25 Forfeited/Cancelled (2,196,820) $ 45.85 Outstanding at December 31, 2023 9,600,399 $ 43.59 5.39 years $ 8,814 Vested or Expected to vest at December 31, 2023 1,796,687 $ 45.85 8.16 years $ 366 Exercisable at December 31, 2023 7,650,948 $ 43.08 4.68 years $ 8,387 The fair values of grants made in the years ended December 31, 2023, 2022 and 2021 were contemporaneously estimated on the date of grant using the following assumptions: 2023 2022 2021 Risk-free interest rate 3.54% - 4.69% 1.55% - 4.57% 0.51% - 1.24% Expected volatility 53% - 56% 54% - 74% 74% - 89% Expected term 5.5 years 5.5 years 5.5 years The Company assumed no expected dividends for all grants. The weighted average grant date fair value of options granted during the years ended December 31, 2023, 2022 and 2021 was $21.27, $23.54, and $43.05 per share, respectively. Restricted Stock Awards and Restricted Stock Units The following table summarizes information on the Company’s restricted stock awards and units: Restricted Stock Awards and Units Weighted Average Grant Number of Date Shares Fair Value Unvested at December 31, 2022 2,516,336 $ 45.67 Granted 2,104,128 38.75 Vested (920,488) 43.74 Forfeited (833,706) 42.31 Unvested at December 31, 2023 2,866,270 $ 41.82 Performance-based Restricted Stock Units Employee Stock Purchase Plan The Company recorded share-based compensation expense in the statement of operations related to incentive stock options, nonstatutory stock options, restricted stock awards, restricted stock units and the ESPP as follows: Year ended December 31, 2023 2022 2021 Research and development $ 52,941 $ 55,869 $ 53,632 Selling, general and administrative 48,695 54,464 49,881 Total $ 101,636 $ 110,333 $ 103,513 As of December 31, 2023, there was approximately $119.6 million of total unrecognized compensation cost related to unvested share-based compensation arrangements granted under the Company’s Plans. This cost is expected to be recognized as compensation expense over the weighted average remaining service period of approximately 2.1 years. |
Other comprehensive income (los
Other comprehensive income (loss) and accumulated other comprehensive items | 12 Months Ended |
Dec. 31, 2023 | |
Comprehensive Income (Loss), Net of Tax, Attributable to Parent [Abstract] | |
Other comprehensive income (loss) and accumulated other comprehensive items | 11. Other comprehensive (loss) income and accumulated other comprehensive items Other comprehensive (loss) income includes changes in equity that are excluded from net loss, such as unrealized gains and losses on marketable securities. The following table summarizes other comprehensive (loss) income and the changes in accumulated other comprehensive items, by component, for the years ended December 31, 2023, 2022, and 2021, respectively. Unrealized Total Gains (Losses) Accumulated On Foreign Other Marketable Currency Comprehensive Securities, net of tax Translation Items Balance at December 31, 2020 $ 1,900 $ (62,857) $ (60,957) Other comprehensive (loss) income before reclassifications (3,279) 39,177 35,898 Amounts reclassified from other comprehensive items 777 — 777 Other comprehensive (loss) income (2,502) 39,177 36,675 Balance at December 31, 2021 $ (602) $ (23,680) $ (24,282) Other comprehensive income before reclassifications 4,072 28,970 33,042 Amounts reclassified from other comprehensive items (3,964) — (3,964) Other comprehensive income 108 28,970 29,078 Balance at December 31, 2022 $ (494) $ 5,290 $ 4,796 Other comprehensive income (loss) before reclassifications 1,135 (6,901) (5,766) Amounts reclassified from other comprehensive items (315) — (315) Other comprehensive income (loss) 820 (6,901) (6,081) Balance at December 31, 2023 $ 326 $ (1,611) $ (1,285) Reclassified |
Revenue recognition
Revenue recognition | 12 Months Ended |
Dec. 31, 2023 | |
Revenue from Contract with Customer [Abstract] | |
Revenue recognition | 12. Revenue recognition Net product sales During the years ended December 31, 2023, 2022, and 2021, net product sales in the United States were $255.1 million, $218.3 million, and $187.3 million, respectively, consisting solely of sales of Emflaza, and net product sales outside of the United States were $406.1 million, $316.9 million, and $241.6 million respectively, consisting of sales of Translarna, Tegsedi, Waylivra, and Upstaza. Translarna net product revenues made up $355.8 million, $288.6 million, and $236.0 million of the net product sales outside the United States for the years ended December 31, 2023, 2022, and 2021, respectively. During the years ended December 31, 2023 and 2022, two countries, the United States and Russia, accounted for at least 10% of the Company’s net product sales, representing $255.1 million and $86.0 million, and $218.3 million and $59.7 million of the net product sales, respectively. During the year ended December 31, 2021, only the United States As of December 31, 2023 and 2022, the Company does not have a contract liabilities balance related to net product sales, and has not made significant changes to the judgments made in applying ASC Topic 606. Collaboration revenue and Royalty revenue In November 2011, the Company and the Spinal Muscular Atrophy Foundation (“SMA Foundation”) entered into a licensing and collaboration agreement with F. Hoffman-La Roche Ltd and Hoffman- La Roche Inc. (collectively, “Roche”). Under the terms of the SMA License Agreement, Roche acquired an exclusive worldwide license to the Company’s SMA program. Under the agreement, the Company is eligible to receive additional payments from Roche if specified events are achieved with respect to each licensed product, including up to $135.0 million in research and development event milestones, up to $325.0 million in sales milestones upon achievement of certain sales events, and up to double digit royalties on worldwide annual net sales of a commercial product. For the years ended December 31, 2023, 2022, and 2021, the Company recognized revenue related to the licensing and collaboration agreement with Roche of $100.0 million, $50.1 million, and $55.0 million, respectively. The below summarizes the milestone achievements associated with the Company’s SMA program during the years ended December 31, 2023, 2022, and 2021. The SMA program currently has one approved product, Evrysdi, which was approved in August 2020 by the FDA for the treatment of SMA in adults and children two months and older. The first commercial sale of Evrysdi in the EU was made in March 2021. This event triggered a $20.0 million milestone payment to the Company from Roche. The first commercial sale in Japan was made in August 2021, which was the final research and development milestone received by the Company. This event triggered a $10.0 million payment to the Company from Roche. In December 2021, the Company recorded its first sales milestone of $25.0 million for the achievement of $500.0 million in worldwide annual net sales from Evrysdi. The Company recorded these three milestone payments as collaboration revenue for the year ended December 31, 2021. of $100.0 million for the achievement of $1.5 billion in worldwide annual net sales from Evrysdi, which is recorded on the balance sheet within prepaid expenses and other current assets as of December 31, 2023. The remaining potential sales milestones as of December 31, 2023 is $150.0 million upon achievement of certain sales events. As of December 31, 2023, the Company does not have any remaining research and development milestones that can be received. In addition to research and development and sales milestones, the Company is eligible to receive up to double-digit royalties on worldwide annual net sales of a commercial product under the SMA License Agreement. For the years ended December 31, 2023, 2022, and 2021 the Company has recognized $168.9 million, $113.5 million, and $54.6 million of royalty revenue related to Evrysdi, respectively. Manufacturing Revenue For the year ended December 31, 2023, the Company recognized $7.7 million of manufacturing revenue related to the production of DNA and AAV vectors for gene therapy applications for external customers. No manufacturing revenue was recognized for the years ended December 31, 2022 and 2021. The Company has not made significant changes to the judgments made in applying ASC Topic 606 for the years ended 2023, 2022, and 2021. As of December 31, 2023 and 2022, the Company has a contract liabilities balance of $0.8 million and $1.4 million, respectively, relating to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers, which is recorded within deferred revenue on the consolidated balance sheet. For the year ended December 31, 2023, the Company recognized $1.4 million related to the amounts included in the contract liability balance at the beginning of the period. As of December 31, 2023, the Company has contract assets of $0.2 million related to plasmid DNA and AAV production for external customers, which is recorded within prepaid expenses and other current assets on the consolidated balance sheet. The Company did not have any contract assets for the period ending December 31, 2022. Remaining performance obligations As of December 31, 2023, and 2022, the Company has remaining performance obligations of $0.8 million and $1.4 million, respectively, related to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers. The Company expects to recognize revenue over the next one year, as the specific timing for satisfying the performance obligations is contingent upon a number of factors, including customers’ needs and schedules. |
Income taxes
Income taxes | 12 Months Ended |
Dec. 31, 2023 | |
Income Tax Disclosure [Abstract] | |
Income taxes | 13. Income taxes The loss from operations before tax benefit (expense) consisted of the following for the years ended December 31, 2023, 2022, and 2021: 2023 2022 2021 Domestic $ (784,744) $ (591,126) $ (487,726) Foreign 88,634 3,639 (30,614) Total $ (696,110) $ (587,487) $ (518,340) The Income Tax Provision consisted of the following for the years ended December 31, 2023, 2022 and 2021: 2023 2022 2021 Current: U.S. Federal $ — $ — $ — U.S. State and Local 27,226 (4,224) (3,844) Foreign (4,003) (1,582) (1,340) Deferred: U.S. Federal 36,408 23,689 — U.S. State and Local 10,521 10,587 (377) Foreign (646) — — Total tax benefit (expense) $ 69,506 $ 28,470 $ (5,561) A reconciliation of the U.S. statutory income tax rate to the Company’s effective tax rate is as follows: December 31, 2023 2022 2021 Federal income tax provision at statutory rate 21.00 % 21.00 % 21.00 % State income tax provision, net of federal benefit 0.32 3.07 (0.74) Permanent differences (1.43) (1.83) (4.06) Research and development 4.59 5.89 4.50 Change in valuation allowances (16.86) (23.36) (29.03) Change in deferred tax assets — (0.10) 12.05 Foreign tax rate differential 0.05 (0.17) 0.01 Tax rate change (1.26) 0.34 0.01 Release (Accrual) of uncertain tax positions 3.71 — (4.78) Other (0.12) — (0.03) Effective income tax rate 10.00 % 4.84 % (1.07) % Accounting for income taxes under U.S. GAAP requires that individual tax-paying entities of the company offset all deferred tax liabilities and assets within each particular tax jurisdiction and present them as a noncurrent deferred tax liability or asset. Amounts in different tax jurisdictions cannot be offset against each other. The noncurrent deferred income tax asset is recorded within deposits and other assets on the balance sheet. The amount of deferred income taxes are as follows: December 31, 2023 2022 Assets: Noncurrent deferred income taxes $ — $ — Liabilities: Noncurrent deferred income taxes (55,905) (102,834) Deferred income taxes - net $ (55,905) $ (102,834) The significant components of the Company’s deferred tax assets and liabilities at December 31, 2023 and 2022 are as follows: 2023 2022 Deferred tax assets: Accrued expense $ 25,400 $ 2,124 Amortization 137,808 52,532 Federal tax credits 205,485 174,802 State tax credits 9,817 9,787 Federal net operating losses 60,270 69,957 State net operating losses 18,680 10,316 Foreign net operating losses 4,052 4,837 Capitalized research and development costs 149,683 110,219 Share based compensation and other 30,757 27,054 Liability for sale of future royalties 190,659 185,589 Noncash interest expense 9,410 30,160 Other comprehensive loss (728) (719) Total gross deferred tax assets 841,293 676,658 Less valuation allowance (833,810) (672,172) Total deferred tax assets, net of valuation allowance $ 7,483 $ 4,486 Deferred tax liabilities: Depreciation $ (7,483) $ (4,486) Indefinite lived intangible (55,905) (102,834) Total gross deferred tax liabilities (63,388) (107,320) Net deferred tax assets (liabilities) $ (55,905) $ (102,834) For the year ended December 31, 2023, the Company generated taxable income in the U.S. of $102.5 million. The Company has not recorded any federal income tax provision after considering the federal NOL, section 250 deduction, available general business credits, and foreign tax credits. The Company recorded a state income tax benefit of $27.2 million which is primarily attributable to the receipt of an outstanding state tax refund received during the year ended December 31, 2023, and the subsequent release of the associated ASC 740 income tax reserve. At December 31, 2023 and 2022, the Company recorded a valuation allowance against its net deferred tax assets of $833.8 million and $672.2 million, respectively. The change in the valuation allowance during the years ended December 31, 2023 and 2022 was $161.6 million and $146.6 million, respectively. A valuation allowance has been recorded since, in the judgment of management, these assets are not more likely than not to be realized. The ultimate realization of deferred tax assets is dependent upon the generation of future taxable income during periods in which those temporary differences and carryforwards become deductible or are utilized. As of December 31, 2023, the Company had $287.0 million, $208.8 million, and $12.1 million of federal, state, and foreign net operating loss carryforwards, respectively. The Company recorded a deferred tax liability in conjunction with the Agilis Merger of $122.0 million in 2018, related to the tax basis difference in the IPRD indefinite-lived intangibles acquired. The Company’s policy is to record a deferred tax liability related to acquired IPR&D which may eventually be realized either upon amortization of the asset when the research is completed, and a product is successfully launched or the write-off of the asset if it is abandoned or unsuccessful. In July 2022, the Company received EMEA approval for a portion of the IPR&D assets, and thus, began the amortization of the intangible. In May 2023, as part of a strategic portfolio prioritization, the Company announced the discontinuation of its preclinical and early research programs in gene therapy, which included programs for Friedreich ataxia and Angelman syndrome. In conjunction with the announcement, the Company recorded an impairment to its indefinite-lived intangible for IP research and development relating to the Friedreich ataxia and Angelman syndrome gene therapy assets. As a result of the impairment, the Company recorded a deferred tax benefit during the 2023 tax year. As of December 31, 2023, research and development credit carryforward for federal purposes is $35.9 million. In addition, the Orphan Drug Credit Carryover available as of December 31, 2023 is $169.6 million. The Company had research and development carryforwards expiring of $0.6 million in 2023, with additional expiration in 2024 and onwards if not otherwise utilized as projected. The income tax benefit (expense) for the years ended December 31, 2023 and 2022 differed from the amounts computed by applying the U.S. federal income tax rate of 21% to loss before tax expense as a result of the IPR&D assets becoming partially amortizable in 2022, foreign taxes, the impact of temporary difference, including the updated section 174, the impact of permanent differences, including “global intangible low-taxed income” (“GILTI”), tax credits generated, true up of net operating loss carryforwards, and increase in the Company’s valuation allowance. Under the 2017 Tax Cuts and Jobs Act, the ability to currently deduct qualifying research and experimental costs under section 174, as well as software development costs, are eliminated for tax years beginning after December 31, 2021. Under the new rule, these costs must be capitalized and amortized over a five-year or fifteen-year period, depending on whether the research is conducted in the U.S. or abroad, respectively. The rule resulted in an increased current taxable income of the Company by $300.3 million for the tax year ended December 31, 2023. The Company applies the elements of FASB ASC 740-10 regarding accounting for uncertainty in income taxes. This clarifies the accounting for uncertainty in income taxes recognized in financial statements and required impact of a tax position to be recognized in the financial statements if that position is more likely than not of being sustained by the taxing authority. As of December 31, 2023, the Company recorded unrecognized tax benefits in the amount of $1.4 million including interest and penalties through 2023. The Company’s policy is to recognize interest and penalties related to tax matters within the income tax provision. Tax years beginning in 2014 are generally subject to examination by taxing authorities, although net operating losses from all years are subject to examinations and adjustments for at least three years following the year in which the attributes are used. The Company is currently under a wage tax audit in Germany for tax years 2018 through 2021. Although the outcome of tax audits is always uncertain, the company does not expect any adjustment to result for these years as of December 31, 2023. For all years through December 31, 2016, the Company generated research credits but has not conducted a study to document the qualified activities. This study may result in an adjustment to the Company’s research and development credit carryforwards; however, until a study is completed and any adjustment is known, no amounts are being presented as an uncertain tax position. A full valuation allowance has been provided against the Company’s research and development credits and, if an adjustment is required, this adjustment would be offset by an adjustment to the deferred tax asset established for the research and development credit carryforwards and the valuation allowance. As a result of U.S. tax reform legislation, distributions of profits from non-U.S. subsidiaries are not expected to cause a significant incremental U.S. tax impact in the future. However, distributions may be subject to non-U.S. withholding taxes if profits are distributed from certain jurisdictions. As of December 31, 2023, for purposes of ASC 740-10-25-3, the Company had $345.9 million of undistributed earnings from non-U.S. subsidiaries that it intends to reinvest permanently in its non-U.S. operations. As these ASC 740-10-25-3 earnings are considered permanently reinvested, no tax provision has been accrued. It is not feasible to estimate the amount of tax that might be payable on the eventual remittance of such earnings. Unrecognized Tax Benefits A reconciliation of the gross amount of unrecognized tax benefits, excluding accrued interest and penalties, is as follows: Unrecognized Tax Benefits Balance at December 31, 2022 27,217 Reductions based on settlements or expiration of statute of limitations (24,671) Interest received (1,186) Balance at December 31, 2023 $ 1,360 Uncertain tax positions, for which management’s assessment is that there is a more than 50% probability of sustaining the position upon challenge by a taxing authority based upon its technical merits, are subject to certain recognition and measurement criteria. The nature of the uncertain tax positions is often very complex and subject to change, and the amounts at issue can be substantial. The Company develops its cumulative probability assessment of the measurement of uncertain tax positions using internal experience, judgment, and assistance from professional advisors. The Company re-evaluates these uncertain tax positions on a quarterly basis based on a number of factors including, but not limited to, changes in facts or circumstances, changes in tax law, and effectively settled issues under audit and new audit activity. Any change in these factors could result in the recognition of a tax benefit or an additional charge to the tax provision. The Company records penalties and tax-related interest expense on unrecognized tax benefits as a component of the provision for income taxes in the accompanying consolidated statement of operations. The Company has not recorded any interest and penalties related to uncertain tax positions for the year ended December 31, 2023, in the accompanying consolidated balance sheet. Future changes in the Company’s unrecognized tax benefits will affect the Company’s annual effective tax rate. |
Commitments and contingencies
Commitments and contingencies | 12 Months Ended |
Dec. 31, 2023 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and contingencies | 14. Commitments and contingencies Under various agreements, the Company will be required to pay royalties and milestone payments upon the successful development and commercialization of products. The Company has entered into funding agreements with The Wellcome Trust Limited (“Wellcome Trust”) for the research and development of small molecule compounds in connection with its oncology and antibacterial programs. As the Company has discontinued development under its antibacterial program, it no longer expects that milestone and royalty payments from the Company to Wellcome Trust will apply under that agreement, resulting in a change to the total amount of development and regulatory milestone payments the Company may become obligated to pay for this program. Under the oncology platform funding agreement, to the extent that the Company develops and commercializes certain program intellectual property on a for-profit basis itself or in collaboration with a partner (provided the Company retains overall control of worldwide commercialization), the Company may become obligated to pay to Wellcome Trust development and regulatory milestone payments and single-digit royalties on sales of any research program product. The Company’s obligation to pay such royalties would continue on a country-by-country basis until the longer of the expiration of the last patent in the program intellectual property in such country covering the research program product and the expiration of market exclusivity of such product in such country. The Company made the first development milestone payment of $0.8 million to Wellcome Trust under the oncology platform funding agreement during the second quarter of 2016. During the year ended December 31, 2022, the Company incurred $2.5 million of development milestones in connection with the enrollment of patients in the registration-directed Phase 2/3 trial of unesbulin for the treatment of LMS, which is recorded in accounts payable and accrued expenses on the balance sheet and will be payable upon the earlier to occur of the first dose administered to the last patient enrolled in the study or the termination of dosing of all patients in the study. However, as part the Company's continuous platform review, the Company has decided to deprioritize its programs for unesbulin for the treatment of diffuse intrinsic pontine glioma and leiomyosarcoma. Accordingly, the Company no longer expects to pay additional milestones to Wellcome Trust under this agreement. The Company has also entered into a collaboration agreement with the SMA Foundation. The Company may become obligated to pay the SMA Foundation single-digit royalties on worldwide net product sales of any collaboration product that is successfully developed and subsequently commercialized or, with respect to collaboration products the Company outlicenses, including Evrysdi, a specified percentage of certain payments the Company receives from its licensee. The Company is not obligated to make such payments unless and until annual sales of a collaboration product exceed a designated threshold. Since inception, the SMA Foundation has earned $52.5 million, $35.3 million which was paid and $17.2 million which was accrued as of December 31, 2023. The Company has reached its obligations to make such payments to the SMA Foundation of an aggregate of $52.5 million as of December 31, 2023. Pursuant to the asset purchase agreement ("Asset Purchase Agreement") between the Company and Marathon Pharmaceuticals, LLC (now known as Complete Pharma Holdings, LLC) (“Marathon”), Marathon is entitled to receive contingent payments from the Company based on annual net sales of Emflaza up to a specified aggregate maximum amount over the expected commercial life of the asset. In addition, Marathon received a $50.0 million sales-based milestone during the year ended December 31, 2022. Pursuant to the Agilis Merger Agreement with Agilis, Agilis equityholders were previously entitled to receive contingent consideration payments from the Company based on (i) the achievement of certain development milestones up to an aggregate maximum amount of $60.0 million, (ii) the achievement of certain regulatory approval milestones together with a milestone payment following the receipt of a priority review voucher up to an aggregate maximum amount of $535.0 million, (iii) the achievement of certain net sales milestones up to an aggregate maximum amount of $150.0 million, and (iv) a percentage of annual net sales for Friedreich ataxia and Angelman syndrome during specified terms, ranging from 2%-6%. The Company was required to pay $40.0 million of the development milestone payments upon the passing of the second anniversary of the closing of the Agilis Merger, regardless of whether the applicable milestones have been achieved. Pursuant to the terms of the Rights Exchange Agreement, the Participating Rightholders canceled and forfeited their rights under the Agilis Merger Agreement to receive (i) $174.0 million, in the aggregate, of potential milestone payments based on the achievement of certain regulatory milestones and (ii) $37.6 million, in the aggregate, of $40.0 million in development milestone payments that would have been due upon the passing of the second anniversary of the closing of the Agilis Merger, regardless of whether the milestones are achieved. The Rights Exchange Agreement has no effect on the Agilis Merger Agreement other than to provide for the cancellation and forfeiture of the Participating Rightholders’ rights to receive $211.6 million, in the aggregate, of the milestone payments described above. As a result, all other rights and obligations under the Agilis Merger Agreement remain in effect pursuant to their terms, including the Company’s obligation to pay up to an aggregate maximum amount of $20.0 million upon the achievement of certain development milestones (representing the remaining portion of potential development milestone payments for which rights were not canceled and forfeited pursuant to the Rights Exchange Agreement while excluding the remaining $2.4 million milestone payment that was due and paid upon the passing of the second anniversary of the closing of the Agilis Merger), up to an aggregate maximum amount of $361.0 million upon the achievement of certain regulatory milestones (representing the remaining portion of potential regulatory milestone payments for which rights were not canceled and forfeited pursuant to the Rights Exchange Agreement), up to a maximum aggregate amount of $150.0 million upon the achievement of certain net sales milestones and a percentage of annual net sales for Friedreich ataxia and Angelman syndrome during specified terms, ranging from 2% to 6%, pursuant to the terms of the Agilis Merger Agreement. In July 2022, the EC approved Upstaza for the treatment of AADC deficiency for patients 18 months and older within the EEA. As a result of such approval, the Company paid the former equityholders of Agilis $50.0 million in accordance with the terms of the Agilis Merger Agreement in the year ended December 31, 2022. In May 2023, as part of the Company’s strategic portfolio prioritization, the Company decided to discontinue its preclinical and early research programs in its gene therapy platform, which included programs for Friedreich ataxia and Angelman syndrome. As a result, the Company does not expect the milestones related to Friedreich ataxia and Angelman syndrome to be achieved. In addition, the Company does not expect to pay the 2% to 6% royalties on annual net sales related to Friedreich ataxia and Angelman syndrome. As of December 31, 2023, the remaining potential development and regulatory milestones the Company expects to achieve is $31.1 million, and the remaining potential sales milestones the Company expects to achieve is $50.0 million, both of which relate solely to Upstaza. On October 25, 2019, the Company completed the acquisition of substantially all of the assets of BioElectron Technology Corporation (“BioElectron”), a Delaware corporation, including certain compounds that the Company has begun to develop as part of its Bio-e platform, pursuant to an asset purchase agreement by and between the Company and BioElectron, dated October 1, 2019 (the “BioElectron Asset Purchase Agreement”). BioElectron was a private company with a pipeline focused on inflammatory and central nervous system (CNS) disorders. The lead program, vatiquinone, is in late stage development for CNS disorders with substantial unmet need and significant commercial opportunity that are complementary to PTC’s existing pipeline. Subject to the terms and conditions of the BioElectron Asset Purchase Agreement, BioElectron may become entitled to receive contingent milestone payments of up to $200.0 million (in cash or in shares of the Company’s common stock, as determined by the Company) from the Company based on the achievement of certain regulatory and net sales milestones. Subject to the terms and conditions of the BioElectron Asset Purchase Agreement, BioElectron may also become entitled to receive contingent payments based on a percentage of net sales of certain products. Subject to the terms and conditions of the Agreement and Plan of Merger, dated as of May 5, 2020 (the “Censa Merger Agreement”) by and among the Company, Hydro Merger Sub, Inc., the Company’s wholly owned, indirect subsidiary, and, solely in its capacity as the representative, agent and attorney-in-fact of the securityholders of Censa, Shareholder Representative Services LLC (such merger pursuant thereto, the “Censa Merger”), former Censa securityholders may become entitled to receive contingent payments from the Company based on (i) the achievement of certain development and regulatory milestones up to an aggregate maximum amount of $217.5 million for sepiapterin’s two most advanced programs and receipt of a priority review voucher from the FDA as set forth in the Censa Merger Agreement, (ii) $109.0 million in development and regulatory milestones for each additional indication of sepiapterin, (iii) the achievement of certain net sales milestones up to an aggregate maximum amount of $160.0 million, (iv) a percentage of annual net sales during specified terms, ranging from single to low double digits of the applicable net sales threshold amount, and (v) any sublicense fees paid to the Company in consideration of any sublicense of Censa’s intellectual property to commercialize sepiapterin, on a country-by-country basis, which contingent payment shall equal to a mid-double digit percentage of any such sublicense fees. In February 2023, the Company completed enrollment of its Phase 3 placebo-controlled clinical trial for sepiapterin for PKU. In connection with this event and pursuant to the Censa Merger Agreement, the Company paid a $30.0 million development milestone to the former Censa securityholders during the year ended December 31, 2023. The Company elected to pay this milestone in the form of shares of its common stock, less certain cash payments in accordance with the Censa Merger Agreement. Pursuant to such election, the Company issued 657,462 shares of its common stock and paid $0.4 million to the former Censa securityholders. The Company expects to make payments to the former Censa securityholders of $65.0 million in the aggregate in cash upon the potential achievement in 2024 of regulatory milestones relating to sepiapterin pursuant to the Censa Merger Agreement. The Company also has the Tegsedi-Waylivra Agreement for the commercialization of Tegsedi and Waylivra, and products containing those compounds in countries in Latin America and the Caribbean. Akcea is entitled to receive royalty payments subject to certain terms set forth in the Tegsedi-Waylivra Agreement. The Company has employment agreements with certain employees which require the funding of a specific level of payments, if certain events, such as a change in control or termination without cause, occur. Additionally, the Company has royalty payments associated with Translarna, Emflaza, and Upstaza net product revenue, payable quarterly or annually in accordance with the terms of the related agreements. From time to time in the ordinary course of its business, the Company is subject to claims, legal proceedings and disputes. The Company is not currently aware of any material legal proceedings against it. |
Geographic information
Geographic information | 12 Months Ended |
Dec. 31, 2023 | |
Segments, Geographical Areas [Abstract] | |
Geographic information | 15. Geographic information The Company views its operations and manages its business in one operating segment. The following table presents financial information based on the geographic location of the facilities of the Company as of and for the years ended: Year Ended December 31, 2023 United States Non-US Total Total assets $ 1,582,962 $ 312,736 $ 1,895,698 Fixed assets, net $ 86,421 $ 668 $ 87,089 Revenue $ 531,661 $ 406,161 $ 937,822 Year Ended December 31, 2022 United States Non-US Total Total assets $ 1,473,770 $ 231,849 $ 1,705,619 Fixed assets, net $ 71,754 $ 836 $ 72,590 Revenue $ 381,862 $ 316,939 $ 698,801 |
401(k) plan
401(k) plan | 12 Months Ended |
Dec. 31, 2023 | |
Retirement Benefits [Abstract] | |
401(k) plan | 16. 401(k) plan The Company maintains a 401(k) plan for its employees. Employee contributions are voluntary. The Company may match employee contributions in amounts to be determined at the Company’s sole discretion. The Company provided an 100% matching contribution for up to the first 6% of each contributing employee’s base salary contributions for the years ended December 31, 2023, 2022 and 2021, respectively. The Company made matching contributions to the 401(k) plan and recorded expense of approximately $10.9 million, $8.4 million, and $6.6 million for the years ended December 31, 2023, 2022 and 2021, respectively. |
Intangible assets and goodwill
Intangible assets and goodwill | 12 Months Ended |
Dec. 31, 2023 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Intangible assets and goodwill | 17. Intangible assets and goodwill Definite-lived intangibles Definite lived intangible assets consisted of the following at December 31, 2023 and 2022: Ending Balance at Foreign Ending Balance at Definite-lived December 31, currency December 31, intangibles assets, gross 2022 Additions translation 2023 Emflaza $ 420,253 $ 107,164 $ — $ 527,417 Waylivra 9,316 533 369 10,218 Tegsedi 7,109 5,839 374 13,322 Upstaza 89,550 — — 89,550 Total definite-lived intangibles, gross $ 526,228 $ 113,536 $ 743 $ 640,507 Ending Balance at Foreign Ending Balance at Definite-lived December 31, currency December 31, intangibles assets, accumulated amortization 2022 Amortization translation 2023 Emflaza $ (266,023) $ (212,595) $ — $ (478,618) Waylivra (2,751) (1,080) (134) (3,965) Tegsedi (1,709) (1,498) (104) (3,311) Upstaza (3,420) (7,462) — (10,882) Total definite-lived intangibles, accumulated amortization $ (273,903) $ (222,635) $ (238) $ (496,776) Total definite-lived intangibles, net $ 143,731 Marathon is entitled to receive contingent payments from the Company based on annual net sales of Emflaza beginning in 2018, up to a specified aggregate maximum amount over the expected commercial life of the asset. In accordance with the guidance for an asset acquisition, the Company records the milestone payment when it becomes payable to Marathon and increase the cost basis for the Emflaza rights intangible asset. For the year ended December 31, 2023, milestone payments of $107.2 million were recorded. These payments are being amortized over the remaining useful life of the Emflaza rights asset on a straight line basis. As of December 31, 2023, a milestone payable to Marathon of $52.7 million was recorded on the balance sheet within accounts payable and accrued expenses. Akcea is also entitled to receive royalty payments subject to certain terms set forth in the Tegsedi-Waylivra Agreement related to sales of Waylivra and Tegsedi. In accordance with the guidance for an asset acquisition, the Company records royalty payments when they become payable to Akcea and increase the cost basis for the Waylivra and Tegsedi intangible assets, respectively. For the year ended December 31, 2023, royalty payments of $5.8 million and $0.5 million were recorded for Tegsedi and Waylivra, respectively. As of December 31, 2023, a royalty payable of $1.6 million and $0.4 million for Tegsedi and Waylivra, respectively, was recorded on the balance sheet within accounts payable and accrued expenses. For the years ended December 31, 2023, 2022, and 2021, the Company recognized amortization expense of $222.6 million, $116.6 million, and $54.8 million respectively, related to the Emflaza rights, Waylivra, Tegsedi, and Upstaza intangible assets. The estimated future amortization of the Emflaza rights, Waylivra, Tegsedi, and Upstaza intangible assets is expected to be as follows: As of December 31, 2023 2024 $ 59,312 2025 10,512 2026 10,512 2027 10,512 2028 and thereafter 52,883 Total $ 143,731 The weighted average remaining amortization period of the definite-lived intangibles as of December 31, 2023 is 6.4 years. Indefinite-lived intangibles Indefinite lived intangible assets consisted of the following at December 31, 2023 and 2022: Ending Balance at Reclass from Foreign Ending Balance at Indefinite-lived December 31, Indefinite Lived to currency December 31, intangibles assets 2022 Additions Definite Lived Impairment translation 2023 Upstaza $ 235,766 $ — $ — $ — $ — $ 235,766 PTC-FA 112,500 — — (112,500) — — PTC-AS 105,300 — — (105,300) — — Total indefinite-lived intangibles $ 453,566 $ — $ — $ (217,800) $ — $ 235,766 Total intangible assets, net $ 379,497 In connection with the acquisition of the Company’s gene therapy platform from Agilis, the Company acquired rights to Upstaza, for the treatment of AADC deficiency. AADC deficiency is a rare CNS disorder arising from reductions in the enzyme AADC that result from mutations in the dopa decarboxylase gene. The gene therapy platform also includes PTC-FA, an asset targeting Friedreich ataxia, a rare and life-shortening neurodegenerative disease caused by a single defect in the FXN gene which causes reduced production of the frataxin protein. Additionally, the gene therapy platform includes two other programs targeting CNS disorders, including PTC-AS for Angelman syndrome, a rare, genetic, neurological disorder characterized by severe developmental delays. In accordance with the acquisition method of accounting, the Company allocated the acquisition cost for the Agilis Merger to the underlying assets acquired and liabilities assumed, based upon the estimated fair values of those assets and liabilities at the date of acquisition. The Company classified the fair value of the acquired IPR&D as indefinite lived intangible assets until the successful completion or abandonment of the associated research and development efforts. As of December 31, 2022, the value allocated to the indefinite lived intangible assets was $453.6 million. In May 2023, as part of the Company’s strategic portfolio prioritization, the Company decided to discontinue its preclinical and early research programs in its gene therapy platform, which included PTC-FA and PTC-AS. As a result, the Company determined that the PTC-FA and PTC-AS indefinite-lived intangible assets were fully impaired and recorded impairment expense of $217.8 million during the second quarter of 2023, which is recorded as intangible asset impairment in the statement of operations. As of December 31, 2023, the remaining indefinite lived intangible asset balance is $235.8 million, consisting solely of Upstaza, which the Company plans to continue to develop and commercialize. The Company performed an annual test for its indefinite-lived intangible asset as of October 1, 2023 and concluded that no impairment exists as of December 31, 2023. Goodwill As a result of the Agilis Merger on August 23, 2018, the Company recorded $82.3 million of goodwill. There have been no changes to the balance of goodwill since the date of the Agilis Merger. Accordingly, the goodwill balance as of December 31, 2023 and 2022 was $82.3 million. The Company performed an annual impairment test for goodwill as of October 1, 2023. The Company’s single reporting unit had a negative carrying value and thus the Company determined there was no impairment of goodwill. |
Subsequent events
Subsequent events | 12 Months Ended |
Dec. 31, 2023 | |
Subsequent Events [Abstract] | |
Subsequent events | 18. Subsequent events The Company has evaluated all subsequent events and transactions through the filing date. There were no material events that impacted the audited consolidated financial statements or disclosures. |
Summary of significant accoun_2
Summary of significant accounting policies (Policies) | 12 Months Ended |
Dec. 31, 2023 | |
Accounting Policies [Abstract] | |
Basis of presentation | Basis of presentation The accompanying consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) and include all adjustments necessary for the fair presentation of the Company’s financial position for the periods presented. |
Use of estimates | Use of estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Significant estimates in these consolidated financial statements have been made in connection with the calculation of net product sales, royalty revenue, certain accruals related to the Company’s research and development expenses, valuation procedures for liability for sale of future royalties, indefinite lived intangible assets annual impairment assessment, fair value of the contingent consideration, and the provision for or benefit from income taxes. Actual results could differ from those estimates. Changes in estimates are reflected in reported results in the period in which they become known. |
Restricted Cash | Restricted Cash Restricted cash included in deposits and other assets on the consolidated balance sheet relates to an unconditional, irrevocable and transferable letter of credit that was entered into during the twelve-month period ended December 31, 2019 in connection with obligations under a facility lease for the Company’s leased biologics manufacturing facility in Hopewell Township, New Jersey. The amount of the letter of credit is $7.5 million, is to be maintained for a term of not less than five years and has the potential to be reduced to $3.8 million if after five years from the lease commencement the Company is not in default of its lease. Restricted cash also contains an unconditional, irrevocable and transferable letter of credit that was entered into during June 2022 in connection with obligations for the Company’s new facility lease in Warren, New Jersey. The amount of the letter of credit is $8.1 million and has the potential to be reduced to $4.1 million if after five years the Company is not in default of its lease. Both amounts are classified within deposits and other assets on the consolidated balance sheet due to the long-term nature of the letter of credit. Restricted cash also includes a bank guarantee of $0.6 million denominated in a foreign currency. The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the consolidated balance sheet that sum to the total of the same amounts shown in the statement of cash flows: End of Beginning of period- period- December 31, December 31, 2023 2022 Cash and cash equivalents $ 594,001 $ 279,834 Restricted cash included in deposits and other assets 16,283 16,091 Total Cash, cash equivalents and restricted cash per statement of cash flows $ 610,284 $ 295,925 |
Consolidation | Consolidation The consolidated financial statements include the accounts of PTC Therapeutics, Inc. and its wholly owned subsidiaries. All inter-company accounts, transactions, and profits have been eliminated in consolidation. |
Segment and geographic information | Segment and geographic information Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision making group, in deciding how to allocate resources and in assessing performance. The Company views its operations and manages its business in one operating and reporting |
Cash equivalents | Cash equivalents The Company considers all highly liquid investments with a maturity of 90 days or less at the time of purchase to be cash equivalents. Cash equivalents are carried at cost which approximates fair value due to their short-term nature. |
Marketable securities | Marketable securities The Company’s marketable securities consists of both debt securities and equity investments. The Company considers its investments in debt securities with original maturities of greater than 90 days to be available for sale securities. Securities under this classification are recorded at fair value and unrealized gains and losses within accumulated other comprehensive income. The estimated fair value of the available for sale securities is determined based on quoted market prices or rates for similar instruments. In addition, the cost of debt securities in this category is adjusted for amortization of premium and accretion of discount to maturity. For available for sale debt securities in an unrealized loss position, the Company assesses whether it intends to sell or if it is more likely than not that the Company will be required to sell the security before recovery of its amortized cost basis. If either of the criteria regarding intent or requirement to sell is met, the security’s amortized cost basis is written down to fair value. If the criteria are not met, the Company evaluates whether the decline in fair value has resulted from a credit loss or other factors. In making this assessment, management considers, among other factors, the extent to which fair value is less than amortized cost, any changes to the rating of the security by a rating agency, and adverse conditions specifically related to the security. If this assessment indicates that a credit loss exists, the present value of cash flows expected to be collected from the security are compared to the amortized cost basis of the security. If the present value of the cash flows expected to be collected is less than the amortized cost basis, a credit loss exists and an allowance for credit losses is recorded for the credit loss, limited by the amount that the fair value is less than the amortized costs basis. Any impairment that has not been recorded through an allowance for credit losses is recognized in other comprehensive income. For the years ended December 31, 2023 and 2022, no allowance was recorded for credit losses. Marketable securities that are equity investments are measured at fair value, as it is readily available, and as such are classified as Level 1 assets. Unrealized holding gains and losses for these equity investments are components of other income (expense), net within the consolidated statement of operations. |
Concentration of credit risk | Concentration of credit risk The Company’s financial instruments that are exposed to credit risks consist primarily of cash and cash equivalents, available-for-sale marketable securities and accounts receivable. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company has not experienced any credit losses in these accounts and does not believe it is exposed to any significant credit risk on these funds. The Company’s investment policy includes guidelines on the quality of the financial institutions and financial instruments the Company is allowed to invest in, which the Company believes minimizes the exposure to concentration of credit risk. The Company is subject to credit risk from its accounts receivable related to its product sales. The payment terms are predetermined and the Company evaluates the creditworthiness of each customer or distributor on a regular basis. The Company reserves all uninsured amounts billed directly to a patient until the time of cash receipt as collectability is not reasonably assured at the time the product is received. To date, the Company has not incurred any material credit losses. |
Fixed assets | Fixed assets Fixed assets are stated at cost. Depreciation is computed starting when the asset is placed into service on a straight-line basis over the estimated useful life of the related asset as follows: Leasehold improvements Lesser of useful life or lease term Computer equipment and software 3 years Machinery and lab equipment 7 years Furniture and fixtures 7 years |
Inventory and cost of product sales | Inventory and cost of product sales Inventory Inventories are stated at the lower of cost and net realizable value with cost determined on a first-in, first-out basis by product. The Company capitalizes inventory costs associated with products following regulatory approval when future commercialization is considered probable and the future economic benefit is expected to be realized. Products which may be used in clinical development programs are included in inventory and charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes. Inventory used for marketing efforts are charged to selling, general and administrative expense. Amounts related to clinical development programs and marketing efforts are immaterial. The following table summarizes the components of the Company’s inventory for the periods indicated: December 31, 2023 December 31, 2022 Raw materials $ 952 $ 1,078 Work in progress 17,991 14,074 Finished goods 11,634 6,656 Total inventory $ 30,577 $ 21,808 The Company periodically reviews its inventories for excess amounts or obsolescence and writes down obsolete or otherwise unmarketable inventory to its estimated net realizable value. The Company recorded write downs of $12.5 million and $1.7 million for the years ended December 31, 2023 and 2022, respectively, primarily related to adjustments to inventory reserves and product approaching expiration. Additionally, though the Company’s product is subject to strict quality control and monitoring which it performs throughout the manufacturing processes, certain batches or units of product may not meet quality specifications resulting in a charge to cost of product sales. For the years ended December 31, 2023 and December 31, 2022, these amounts were immaterial. Cost of product sales Cost of product sales consists of the cost of inventory sold, manufacturing and supply chain costs, storage costs, amortization of the acquired intangible asset, royalty payments associated with net product sales, and royalty payments to collaborative partners associated with royalty revenues and collaboration revenue related to milestones. Production costs are expensed as cost of product sales when the related products are sold or royalty revenues and collaboration revenue milestones are earned. |
Accumulated other comprehensive income (loss) | Accumulated other comprehensive income (loss) Accumulated other comprehensive income (loss) consists of unrealized gains or losses on marketable securities and foreign currency translation adjustments. |
Revenue recognition | Revenue recognition Net product revenue The Company’s net product revenue primarily consists of sales of Translarna in territories outside of the U.S. for the treatment of nmDMD and sales of Emflaza in the U.S. for the treatment of DMD. The Company recognizes revenue when its performance obligations with its customers have been satisfied. The Company’s performance obligations are to provide products based on customer orders from distributors, hospitals, specialty pharmacies or retail pharmacies. The performance obligations are satisfied at a point in time when the Company’s customer obtains control of the product, which is typically upon delivery. The Company invoices its customers after the products have been delivered and invoice payments are generally due within 30 to 90 days of the invoice date. The Company determines the transaction price based on fixed consideration in its contractual agreements. Contract liabilities arise in certain circumstances when consideration is due for goods the Company has yet to provide. As the Company has identified only one distinct performance obligation, the transaction price is allocated entirely to product sales. In determining the transaction price, a significant financing component does not exist since the timing from when the Company delivers product to when the customers pay for the product is typically less than one year. Customers in certain countries pay in advance of product delivery. In those instances, payment and delivery typically occur in the same month. The Company records product sales net of any variable consideration, which includes discounts, allowances, rebates related to Medicaid and other government pricing programs, and distribution fees. The Company uses the expected value or most likely amount method when estimating its variable consideration, unless discount or rebate terms are specified within contracts. The identified variable consideration is recorded as a reduction of revenue at the time revenues from product sales are recognized. These estimates for variable consideration are adjusted to reflect known changes in factors and may impact such estimates in the quarter those changes are known. Revenue recognized does not include amounts of variable consideration that are constrained. During the years ended December 31, 2023, 2022, and 2021, net product sales in the United States were $255.1 million, $218.3 million, and $187.3 million, respectively, consisting solely of sales of Emflaza, and net product sales outside of the United States were $406.1 million, $316.9 million, and $241.6 million respectively, consisting of sales of Translarna, Tegsedi, Waylivra, and Upstaza. Translarna net product revenues made up $355.8 million, $288.6 million, and $236.0 million of the net product sales outside of the United States for the years ended December 31, 2023, 2022, and 2021, respectively. During the years ended December 31, 2023 and 2022, two countries, the United States and Russia, accounted for at least 10% of the Company’s net product sales, representing $255.1 million and $86.0 million, and $218.3 million and $59.7 million of net product sales, respectively. During the year ended December 31, 2021, only the United States In relation to customer contracts, the Company incurs costs to fulfill a contract but does not incur costs to obtain a contract. These costs to fulfill a contract do not meet the criteria for capitalization and are expensed as incurred. The Company considers any shipping and handling costs that are incurred after the customer has obtained control of the product as a cost to fulfill a promise. Shipping and handling costs associated with finished goods delivered to customers are recorded as a selling expense. Collaboration and royalty revenue The terms of these agreements typically include payments to the Company of one or more of the following: nonrefundable, upfront license fees; milestone payments; research funding and royalties on future product sales. In addition, the Company generates service revenue through agreements that generally provide for fees for research and development services and may include additional payments upon achievement of specified events. At the inception of a collaboration arrangement, the Company needs to first evaluate if the arrangement meets the criteria in Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) Topic 808 “Collaborative Arrangements” to then determine if ASC Topic 606 is applicable by considering whether the collaborator meets the definition of a customer. If the criteria are met, the Company assesses the promises in the arrangement to identify distinct performance obligations. For licenses of intellectual property, the Company assesses, at contract inception, whether the intellectual property is distinct from other performance obligations identified in the arrangement. If the licensing of intellectual property is determined to be distinct, revenue is recognized for nonrefundable, upfront license fees when the license is transferred to the customer and the customer can use and benefit from the license. If the licensing of intellectual property is determined not to be distinct, then the license will be bundled with other promises in the arrangement into one distinct performance obligation. The Company needs to determine if the bundled performance obligation is satisfied over time or at a point in time. If the Company concludes that the nonrefundable, upfront license fees will be recognized over time, the Company will need to assess the appropriate method of measuring proportional performance. For milestone payments, the Company assesses, at contract inception, whether the development or sales-based milestones are considered probable of being achieved. If it is probable that a significant revenue reversal will occur, the Company will not record revenue until the uncertainty has been resolved. Milestone payments that are contingent upon regulatory approval are not considered probable of being achieved until the applicable regulatory approvals or other external conditions are obtained as such conditions are not within the Company’s control. If it is probable that a significant revenue reversal will not occur, the Company will estimate the milestone payments using the most likely amount method. The Company will re-assess the development and sales-based milestones each reporting period to determine the probability of achievement. The Company recognizes royalties from product sales at the later of when the related sales occur or when the performance obligation to which the royalty has been allocated has been satisfied. If it is probable that a significant revenue reversal will not occur, the Company will estimate the royalty payments using the most likely amount method. The Company recognizes revenue for reimbursements of research and development costs under collaboration agreements as the services are performed. The Company records these reimbursements as revenue and not as a reduction of research and development expenses as the Company has the risks and rewards as the principal in the research and development activities. For the years ended December 31, 2023, 2022, and 2021, the Company has recognized $100.0 million, $50.1 million, and $55.0 million of collaboration revenue, respectively, related to the SMA License Agreement with Roche. For the years ended December 31, 2023, 2022 and 2021, the Company has recognized $168.9 million, $113.5 million, and $54.6 million of royalty revenue, respectively, related to Evrysdi. Manufacturing Revenue The Company has manufacturing services related to the production of plasmid deoxyribonucleic acid (“DNA”) and adeno-associated virus (“AAV”) vectors for gene therapy applications for external customers. Performance obligations vary but may include manufacturing plasmid DNA and/or AAV vectors, material testing, stability studies, and other services related to material development. The transaction prices for these arrangements are fixed and include amounts stated in the contracts for each promised service. Typically, the performance obligations within a manufacturing contract are highly interdependent, in which case, the Company will combine them into a single performance obligation. The Company has determined that the assets created have no alternative use to the Company, and the Company has an enforceable right to payment for the performance completed to date, therefore revenue related to these services are recognized over time and is measured using an output method based on performance of manufacturing milestones completed to date. Manufacturing service contracts may also include performance obligations related to project management services or obtaining materials from third parties. The Company has determined that these are separate performance obligations for which revenue is recognized at the point in time the services are performed. For performance obligations related to obtaining third party materials, the Company has determined that it is the principal as the Company has control of the materials and has discretion in setting the price. Therefore, the Company recognizes revenue on a gross basis related to obtaining third party materials. Certain arrangements require a portion of the contract consideration to be received in advance at the commencement of the contract, and such advance payment is initially recorded as a contract liability. A contract asset may be recognized in the event the Company’s satisfaction of performance obligations outpaces customer billings. For the year ended December 31, 2023, the Company recognized $7.7 million of manufacturing revenue related to plasmid DNA and AAV vector production for external customers. No manufacturing revenue was recognized for the years ended December 31, 2022 and 2021. As of December 31, 2023, the Company has contract assets of $0.2 million and remaining performance obligations of $0.8 million related to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers. For the period ended December 31, 2022, the Company had remaining performance obligations of $1.4 million and no contracts assets related to plasmid DNA and AAV production for external customers. |
Allowance for doubtful accounts | Allowance for doubtful accounts The Company maintains an allowance for estimated losses resulting from the inability of its customers to make required payments. The Company estimates uncollectible amounts based upon current customer receivable balances, the age of customer receivable balances, the customer’s financial condition and current economic trends. The Company also assesses whether an allowance for expected credit losses may be required which includes a review of the Company’s receivables portfolio, which are pooled on a customer basis or country basis. In making its assessment of whether an allowance for credit losses is required, the Company considers its historical experience with customers, current balances, levels of delinquency, regulatory and legal environments, and other relevant current and future forecasted economic conditions |
Liability for sale of future royalties | Liability for sale of future royalties On July 17, 2020, the Company, RPI Intermediate Finance Trust (“RPI”), and, for the limited purposes set forth in the agreement, Royalty Pharma PLC, entered into a royalty purchase agreement (the “Original Royalty Purchase Agreement”). Pursuant to the Original Royalty Purchase Agreement, the Company sold to RPI 42.933% (the “Original Assigned Royalty Rights”) of the Company’s right to receive sales-based royalty payments (the “Royalty”) on worldwide net sales of Evrysdi and any other product developed pursuant to the License and Collaboration Agreement (the “License Agreement”), dated as of November 23, 2011, by and among the Company, Roche and, for the limited purposes set forth therein, the SMA Foundation under the SMA program. In consideration for the sale of the Original Assigned Royalty Rights, RPI paid the Company $650.0 million in cash consideration. The Company has retained a 57.067% interest in the Royalty and all economic rights to receive the remaining potential regulatory and sales milestone payments under the License Agreement, which remaining milestone payments equal $150.0 million in the aggregate as of December 31, 2023. The Original Royalty Purchase Agreement was set to terminate 60 days following the earlier of the date on which Roche is no longer obligated to make any payments of the Royalty pursuant to the License Agreement and the date on which RPI has received $1.3 billion in respect of the Original Assigned Royalty Rights. Pursuant to the guidance in ASC 470-10-25-2, the Company determined that the $650.0 million cash consideration obtained pursuant to the Original Royalty Purchase Agreement should be classified as debt and recorded it as “liability for sale of future royalties-current” and “liability for sale of future royalties-noncurrent” on the Company’s consolidated balance sheet based on the timing of the expected payments to be made to RPI at the time of the transaction. The liability was subsequently amortized using the effective interest method over the life of the arrangement, in accordance with the respective guidance, utilizing the prospective method to account for subsequent changes in the estimated future payments to be made to RPI. On October 18, 2023, the Company, Royalty Pharma Investments 2019 ICAV (“Royalty Pharma”), and, for the limited purposes set forth in the agreement, Royalty Pharma plc, entered into an Amended and Restated Royalty Purchase Agreement (the “A&R Royalty Purchase Agreement”), which amends and restates in its entirety the Original Royalty Purchase Agreement. Pursuant to the A&R Royalty Purchase Agreement, the Company has sold or agreed to sell to Royalty Pharma certain portions of the Company’s remaining Royalty on worldwide net sales of Evrysdi and any other product (the “Products”) developed pursuant to the SMA License Agreement (all such retained Royalty rights of the Company, the “Retained Royalty Rights,” and all such Royalty rights that are sold to Royalty Pharma pursuant to the A&R Royalty Purchase Agreement, the “A&R Assigned Royalty Rights”). At closing, Royalty Pharma paid the Company $1.0 billion in cash consideration for 38.0447% of the Company’s Retained Royalty Rights (which is in addition to the 42.9330% assigned to Royalty Pharma in connection with the Original Royalty Purchase Agreement, for a total of 80.9777% of the total Royalty) until such time as Royalty Pharma has received payments in respect of the Original Assigned Royalty Rights equal to $1.3 billion in the aggregate, and thereafter 66.6667% of the total Royalty. In addition, the Company may sell to Royalty Pharma the remainder of the Company’s Retained Royalty Rights in exchange for an aggregate of $500.0 million in additional cash consideration after the closing of the A&R Royalty Purchase Agreement, less royalties received in respect of the Retained Royalty Rights put to Royalty Pharma, which will be payable by Royalty Pharma pursuant to five put options held by the Company that are exercisable at the Company’s option between January 1, 2024 and December 31, 2025. If the Company exercises two or fewer of the put options, Royalty Pharma may exercise a call option during the period from and after January 1, 2026 until and including March 31, 2026 for up to 50% of the remainder of the Company’s Retained Royalty Rights less amounts exercised by the Company via its put options at a purchase price that is proportional to the purchase price of the Company’s unexercised put options. Royalty Pharma’s exercise of the call option would result in Royalty Pharma owning 90.4888% of the total Royalty until such time as Royalty Pharma has received payments in respect of the Original Assigned Royalty Rights equal to $1.3 billion in the aggregate, and thereafter 83.3333% of the total Royalty. The A&R Royalty Purchase Agreement will terminate 60 days following the date on which Roche is no longer obligated to make any payments of the Royalty pursuant to the License Agreement. The change in rights and obligations from the A&R Royalty Purchase Agreement resulted in a change in the terms of the liability for sale of future royalties, which was evaluated by the Company in accordance with ASC 470-50, Debt — Modifications and Extinguishments. The Company determined that the present value of the cash flows under the A&R Royalty Purchase Agreement were substantially different from the present value of the cash flows under the Original Royalty Purchase Agreement. This resulted in the derecognition of the old liability for sale of future royalties and the new liability for sale of future royalties being recorded at fair value, which was determined to be $1,809.9 million as of the date of the A&R Royalty Purchase Agreement. This resulted in an extinguishment loss of $44.9 million, which was recorded within loss on extinguishment of debt, within the Company’s statement of operations. The fair value for the new liability for sale of future royalties on the date of the A&R Royalty Purchase Agreement was based on the Company’s estimates of future royalties expected to be paid to Royalty Pharma over the life of the arrangement, which was determined using forecasts from market data sources, which are considered Level 3 inputs. The liability is being amortized using the effective interest method over the life of the arrangement, in accordance with ASC 470 and ASC 835. The initial annual effective interest rate was determined to be 10.8%. The Company utilizes the prospective method to account for subsequent changes in the estimated future payments to be made to Royalty Pharma and updates the effective interest rate on a quarterly basis. Issuance costs related to the transaction were determined to be immaterial. Refer to Note 7 for further details. |
Leases | Leases The Company determines if an arrangement is a lease at inception. This determination generally depends on whether the arrangement conveys to the Company the right to control the use of an explicitly or implicitly identified fixed asset for a period of time in exchange for consideration. Control of an underlying asset is conveyed to the Company if the Company obtains the rights to direct the use of and to obtain substantially all of the economic benefits from using the underlying asset. The Company has lease agreements which include lease and non-lease components, which the Company accounts for as a single lease component for all leases. Operating and finance leases are classified as right of use ("ROU") assets, short term lease liabilities, and long term lease liabilities. Operating and finance lease ROU assets and lease liabilities are recognized at the commencement date based on the present value of lease payments over the lease term. ROU assets are Variable lease payments associated with the Company’s leases are recognized when the event, activity, or circumstance in the lease agreement on which those payments are assessed occurs. Variable lease payments are presented in the Company’s consolidated statements of operations in the same line item as expense arising from fixed lease payments for operating leases. Leases with an initial term of 12 months or less are not recorded on the consolidated balance sheet and the Company recognizes lease expense for these leases on a straight-line basis over the lease term. The Company applies this policy to all underlying asset categories. A lessee is required to discount its unpaid lease payments using the interest rate implicit in the lease or, if that rate cannot be readily determined, its incremental borrowing rate. As most of the Company’s leases do not provide an implicit rate, the Company uses its incremental borrowing rate based on the information available at the commencement date in determining the present value of lease payments. The Company gives consideration to its recent debt issuances as well as publicly available data for instruments with similar characteristics when calculating its incremental borrowing rates. The lease term for all of the Company’s leases includes the non-cancellable period of the lease plus any additional periods covered by either a Company option to extend (or not to terminate) the le certain to exercise, or an option to extend (or not to terminate) the lease controlled by the lessor. Leasehold improvements are capitalized and depreciated over the lesser of useful life or lease term. See Note 5 Leases for additional information. |
Research and development costs | Research and development costs Research and development expenses include the clinical development costs associated with the Company’s product development programs and research and development costs associated with the Company’s discovery programs. These expenses include internal research and development costs and the costs of research and development conducted on behalf of the Company by third parties, including sponsored university-based research agreements and clinical study vendors. All research and development costs are expensed as incurred. Costs incurred in obtaining technology licenses are charged immediately to research and development expense if the technology licensed has not reached technological feasibility and has no alternative future uses. Advance payments made for goods and services that will be used in future research and development activities are deferred if the contracted party has not yet performed the related activities. The amount deferred is then expensed when the research and development activities are performed. As of December 31, 2023 and 2022, the short term deferred research and development advance payments were $2.6 million and $2.4 million, respectively, and are classified as prepaid expenses and other current assets on the consolidated balance sheet. As of December 31, 2023 and 2022, the long term deferred research and development advance payments were $4.7 million and $3.9 million, respectively, and are classified as deposits and other assets on the consolidated balance sheet. |
Fair value of financial instruments | Fair value of financial instruments The Company follows the fair value measurement rules, which provides guidance on the use of fair value in accounting and disclosure for assets and liabilities when such accounting and disclosure is called for by other accounting literature. These rules establish a fair value hierarchy for inputs to be used to measure fair value of financial assets and liabilities. This hierarchy prioritizes the inputs to valuation techniques used to measure fair value into three levels: Level 1 (highest priority), Level 2, and Level 3 (lowest priority). ● Level 1—Unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the balance sheet date. ● Level 2—Inputs other than quoted prices included within Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs include quoted prices for similar assets and liabilities in active markets, quoted prices for identical or similar assets or liabilities in markets that are not active, inputs other than quoted prices that are observable for the asset or liability (i.e., interest rates, yield curves, etc.), and inputs that are derived principally from or corroborated by observable market data by correlation or other means (market corroborated inputs). ● Level 3—Inputs are unobservable and reflect the Company’s assumptions as to what market participants would use in pricing the asset or liability. The Company develops these inputs based on the best information available. Cash equivalents, marketable securities, and equity investments are reflected in the accompanying financial statements at fair value. The carrying amount of receivables and accounts payable and accrued expenses approximates fair value due to the short-term nature of those instruments. |
Share-based compensation | Share-based compensation The Company measures the cost of employee services received in exchange for an award of equity instruments based on the grant date fair value of the award. Restricted stock awards are measured based on the fair market values of the underlying stock on the dates of grant. For service type awards, share-based compensation expense is recognized on a straight-line basis over the period during which the employee is required to provide service in exchange for the entire award. The fair value of options is calculated using the Black-Scholes option pricing model to determine the fair value of stock options on the date of grant based on key assumptions such as expected volatility and expected term. The Company estimates the expected volatility of options utilizing the Company’s historical stock volatility. The Company estimates the expected term of options utilizing the Company’s historical exercise data. The risk-free rate of the option is based on U.S. Government Securities Treasury Constant Maturities yields at the date of grant for a term similar to the expected term of the option. In connection with the adoption of FASB Accounting Standards Update (“ASU”) 2016-9, the Company made a policy election to continue its methodology for estimating its forfeiture rate. Stock-based compensation expense for performance stock units (“PSUs”) is determined using the grant date fair value, which is the quoted closing market price per share of the Company’s common stock on the Nasdaq Global Select Market on the grant date. Stock-based compensation expense for the PSUs will not be recognized until the achievement of the performance goal is deemed probable (the “Probable Date”), a determination that requires significant judgment by management, as the achievement of these goals have inherent risk and uncertainties. At the Probable Date, the Company records a cumulative catch-up expense for the portion of the grant date fair value attributable to the period from the grant date to the Probable Date. The remaining expense is recognized over the remaining service period on a straight-line basis. |
Income Taxes | Income taxes On December 15, 2022, the European Union (EU) member states formally adopted the EU’s Pillar Two Directive, which generally provides for a minimum effective tax rate of 15%, as established by the Organization for Economic Co-operation and Development (OECD) Pillar Two Framework that was supported by over 130 countries worldwide. The EU effective dates are January 1, 2024, and January 1, 2025, for different aspects of the directive. A significant number of other countries are also implementing similar legislation. As a result, the tax laws in the U.S. and other countries in which PTC and its affiliates do business could change on a prospective or retroactive basis and any such changes could materially adversely affect the Company’s business. The Company is continuing to evaluate the potential impact on future periods of the Pillar Two Framework, pending legislative adoption by additional individual countries, including those within the EU. On December 22, 2017, the U.S. government enacted the 2017 Tax Cuts and Jobs Act (“TCJA”), which significantly revised U.S. tax law by, among other provisions, lowering the U.S. federal statutory income tax rate to 21%, imposing a mandatory one-time transition tax on previously deferred foreign earnings, and eliminating or reducing certain income tax deductions. The Global Intangible Low-tax Income ("GILTI") provisions of the TCJA require the Company to include in its U.S. income tax return foreign subsidiary earnings in excess of an allowable return on the foreign subsidiary’s tangible assets. The Company has elected to account for GILTI tax in the period in which it is incurred, and therefore has not provided any deferred tax impacts of GILTI in its consolidated financial statements for the period ended December 31, 2023. Starting in 2022, TCJA amendments to IRC Section 174 no longer permits an immediate deduction for research and development (R&D) expenditures in the tax year that such costs are incurred. Instead, these IRC Section 174 development costs must now be capitalized and amortized over either a five- or 15-year period, depending on the location of the activities performed. The new amortization period begins with the midpoint of any taxable year that IRC Section 174 costs are first incurred, regardless of whether the expenditures were made prior to or after July 1 and runs until the midpoint of year five for activities conducted in the United States or year 15 in the case of development conducted on foreign soil. This tax law change resulted in an increased current taxable income of the Company by $300.3 million for the year ended December 31, 2023. Deferred tax assets and liabilities are recognized for the future tax consequences attributable to differences between the financial statement carrying amounts of existing assets and liabilities and their respective tax bases and net operating loss and credit carryforwards. Deferred tax assets and liabilities are measured at rates expected to apply to taxable income in the years in which those temporary differences and carryforwards are expected to be recovered or settled. The effect on deferred tax assets and liabilities of a change in tax rates is recognized in the statement of operations in the period that includes the enactment date. A valuation allowance is recorded when it is not more likely than not that all or a portion of the net deferred tax assets will be realized. On August 23, 2018, the Company completed its acquisition of Agilis Biotherapeutics, Inc. (“Agilis”), pursuant to an Agreement and Plan of Merger, dated as of July 19, 2018 (the “Agilis Merger Agreement”), by and among the Company, Agility Merger Sub, Inc., a Delaware corporation and the Company’s wholly owned, indirect subsidiary, Agilis and, solely in its capacity as the representative, agent and attorney-in-fact of the equityholders of Agilis, Shareholder Representative Services LLC, (the “Agilis Merger”). The Company recorded a deferred tax liability in conjunction with the Agilis Merger of $122.0 million in 2018, related to the tax basis difference in the In-Process Research and Development, or IPR&D, indefinite-lived intangibles acquired. The Company’s policy is to record a deferred tax liability related to acquired IPR&D which may eventually be realized either upon amortization of the asset when the research is completed and a product is successfully launched or the write-off of the asset if it is abandoned or unsuccessful. In July 2022, the Company received EMEA approval for a portion of the IPR&D assets, and thus, began the amortization of the intangible. In May 2023, as part of a strategic portfolio prioritization, the Company announced the discontinuation of its preclinical and early research programs for its gene therapy platform, which included programs for Friedreich ataxia and Angelman syndrome. In conjunction with the announcement, the Company recorded an impairment to its indefinite-lived intangible for IP research and development relating to the Friedreich ataxia and Angelman syndrome gene therapy assets. As a result of the impairment, the Company recorded a deferred tax benefit of $46.9 million during the 2023 tax year. |
Foreign currency | Foreign currency The functional currencies of the Company’s foreign subsidiaries primarily are the local currencies of the country in which the subsidiary operates. The Company had an intercompany loan which was recorded on a non-U.S. subsidiary and was formally denominated in U.S. dollars and was remeasured into local currency using the exchange rate as of the balance sheet date. During the year ended December 31, 2022, the loan agreement was amended to change the denomination from U.S. dollars to local currency, and the Company recorded a non-cash realized foreign exchange loss of $16.9 million, which was recorded within other income (expense), net on the consolidated statement of operations. The intercompany loan was settled during the year ended December 31, 2023. The Company’s asset and liability accounts, including the intercompany loan, are translated using the current exchange rate as of the balance sheet date. Stockholders’ equity accounts are translated using historical rates at the balance sheet date. Revenue and expense accounts are translated using a weighted average exchange rate over the period ended on the balance sheet date. Adjustments resulting from the translation of the financial statements of the Company’s foreign subsidiaries into U.S. dollars are accumulated as a separate component of stockholders’ equity within other comprehensive income. Gains or losses resulting from transactions denominated in foreign currencies are included in other income or expense, within the consolidated statements of income. |
Net (loss) income per share | Net (loss) income per share Basic net (loss) income per share is calculated by dividing the net (loss) income attributable to common stockholders by the weighted average number of common shares outstanding for the period, without consideration for common stock equivalents. Diluted net income per share is calculated by dividing the net income attributable to common stockholders by the weighted-average number of common share equivalents outstanding for the period determined using the treasury-stock method and the if-converted method. During periods in which the Company incurs net losses, both basic and diluted loss per share is calculated by dividing the net loss by the weighted average shares outstanding—potentially dilutive securities are excluded from the calculation because their effect would be anti-dilutive. Dilutive common stock equivalents are comprised of options and unvested restricted stock outstanding under the Company’s stock option plans. |
Business combinations and asset acquisitions | Business combinations and asset acquisitions The Company evaluates acquisitions of assets and other similar transactions to assess whether or not the transaction should be accounted for as a business combination or asset acquisition by first applying a screen to determine if substantially all of the fair value of the gross assets acquired is concentrated in a single identifiable asset or group of similar identifiable assets. If the screen is met, the transaction is accounted for as an asset acquisition. If the screen is not met, further determination is required as to whether or not the Company has acquired inputs and processes that have the ability to create outputs, which would meet the requirements of a business. If determined to be a business combination, the Company accounts for the transaction under the acquisition method of accounting as indicated in ASU 2017-01, “Business Combinations”, which requires the acquiring entity in a business combination to recognize the fair value of all assets acquired, liabilities assumed, and any non-controlling interest in the acquiree and establishes the acquisition date as the fair value measurement point. Accordingly, the Company recognizes assets acquired and liabilities assumed in business combinations, including contingent assets and liabilities, and non-controlling interest in the acquiree based on the fair value estimates as of the date of acquisition. In accordance with ASC 805, the Company recognizes and measures goodwill as of the acquisition date, as the excess of the fair value of the consideration paid over the fair value of the identified net assets acquired. The consideration for the Company’s business acquisitions may include future payments that are contingent upon the occurrence of a particular event or events. The obligations for such contingent consideration payments are recorded at fair value on the acquisition date. The contingent consideration obligations are then evaluated each reporting period. Changes in the fair value of contingent consideration, other than changes due to payments, are recognized as a gain or loss and recorded within the change in the fair value of deferred and contingent consideration in the consolidated statements of operations. If determined to be an asset acquisition, the Company accounts for the transaction under ASC 805-50, which requires the acquiring entity in an asset acquisition to recognize assets acquired and liabilities assumed based on the cost to the acquiring entity on a relative fair value basis, which includes transaction costs in addition to consideration given. No gain or loss is recognized as of the date of acquisition unless the fair value of noncash assets given as consideration differs from the assets’ carrying amounts on the acquiring entity’s books. Consideration transferred that is noncash will be measured based on either the cost (which will be measured based on the fair value of the consideration given) or the fair value of the assets acquired and liabilities assumed, whichever is more reliably measurable. Goodwill is not recognized in an asset acquisition and any excess consideration transferred over the fair value of the net assets acquired is allocated to the identifiable assets based on relative fair values. Contingent consideration payments in asset acquisitions are recognized when the contingency is resolved and the consideration is paid or becomes payable (unless the contingent consideration meets the definition of a derivative, in which case the amount becomes part of the basis in the asset acquired). Upon recognition of the contingent consideration payment, the amount is included in the cost of the acquired asset or group of assets. |
Finite-lived intangible assets | Finite-lived intangible assets The Company records the fair value of purchased intangible assets with finite useful lives as of the transaction date of a business combination or asset acquisition. Purchased intangible assets with finite useful lives are amortized to their estimated residual values over their estimated useful lives. |
Impairment of long-lived assets | Impairment of long-lived assets The Company monitors its long-lived assets and finite-lived intangibles for indicators of impairment. If such indicators are present, the Company assesses the recoverability of affected assets by determining whether the carrying value of such assets is less than the sum of the undiscounted future cash flows of the assets. If such assets are found not to be recoverable, the Company measures the amount of such impairment by comparing the carrying value of the assets to the fair value of the assets, with the fair value generally determined based on the present value of the expected future cash flows associated with the assets. The Company believes that no impairment of long-lived assets exists as of December 31, 2023. |
Indefinite-lived intangible assets | Indefinite-lived intangible assets Indefinite-lived intangible assets consist of IPR&D. IPR&D acquired directly in a transaction other than a business combination is capitalized if the projects will be further developed or have an alternative future use; otherwise they are expensed. The fair values of IPR&D projects and license agreement assets acquired in business combinations are capitalized. Several methods may be used to determine the estimated fair value of the IPR&D and license agreement asset acquired in a business combination. The Company utilizes the “income method” and uses estimated future net cash flows that are derived from projected sales revenues and estimated costs. These projections are based on factors such as relevant market size, patent protection, and expected pricing and industry trends. The estimated future net cash flows are then discounted to the present value using an appropriate discount rate. These assets are treated as indefinite-lived intangible assets until completion or abandonment of the projects, at which time the assets are amortized over the remaining useful life or written off, as appropriate. Intangible assets with indefinite lives, including IPR&D, are tested for impairment if impairment indicators arise and, at a minimum, annually. However, an entity is permitted to first assess qualitative factors to determine if a quantitative impairment test is necessary. Further testing is only required if the entity determines, based on the qualitative assessment, that it is more likely than not that an indefinite-lived intangible asset’s fair value is less than its carrying amount. Otherwise, no further impairment testing is required. The indefinite-lived intangible asset impairment test consists of a one-step analysis that compares the fair value of the intangible asset with its carrying amount. If the carrying amount of an intangible asset exceeds its fair value, an impairment loss is recognized in an amount equal to that excess. The Company considers many factors in evaluating whether the value of its intangible assets with indefinite lives may not be recoverable, including, but not limited to, expected growth rates, the cost of equity and debt capital, general economic conditions, the Company’s outlook and market performance of the Company’s industry and recent and forecasted financial performance. In May 2023, as part of the Company’s strategic portfolio prioritization, the Company decided to discontinue its preclinical and early research programs in its gene therapy platform, which included programs for Friedreich ataxia and Angelman syndrome. As a result, the Company determined that the Friedreich ataxia and Angelman syndrome indefinite lived intangible assets were fully impaired and recorded impairment expense of $217.8 million during the second quarter of 2023, which is recorded as intangible asset impairment in the statement of operations. Refer to Note 17 for further information regarding the Company’s intangible assets. The Company performed an annual test for its remaining indefinite-lived intangible asset as of October 1, 2023 and concluded that no impairment exists as of December 31, 2023. |
Goodwill | Goodwill Goodwill represents the amount of consideration paid in excess of the fair value of net assets acquired as a result of the Company’s business acquisitions accounted for using the acquisition method of accounting. Goodwill is not amortized and is subject to impairment testing at a reporting unit level on an annual basis or when a triggering event occurs that may indicate the carrying value of the goodwill is impaired. The Company reassesses its reporting units as part of its annual segment review. As of December 31, 2023, the Company concluded that it continues to operate as one reporting unit. An entity is permitted to first assess qualitative factors to determine if a quantitative impairment test is necessary. Further testing is only required if the entity determines, based on the qualitative assessment, that it is more likely than not that the fair value of the reporting unit is less than its carrying amount. The Company performed an annual test for goodwill as of October 1, 2023. The Company’s single reporting unit had a negative carrying value and thus the Company determined there was no impairment of goodwill. |
Recent accounting pronouncements | Recent accounting pronouncements In November 2023, the FASB issued ASU 2023-07, Segment Reporting (Topic 280) – Improvements to Reportable Segment Disclosures. This ASU requires that a public entity provide additional segment disclosures on an interim and annual basis. The amendments in this ASU should be applied retrospectively to all prior periods presented in the financial statements, unless impracticable. Upon transition, the segment expense categories and amounts disclosed in the prior periods should be based on the significant segment expense categories identified and disclosed in the period of adoption. The ASU is effective for fiscal years beginning after December 15, 2023 and interim periods within fiscal years beginning after December 15, 2024. Early adoption is permitted. The Company is currently planning to adopt this guidance when effective. The Company is assessing the impact of the adoption on the Company’s consolidated financial statements and accompanying footnotes. In December 2023, the FASB issued ASU 2023-09, Improvements to Income Tax Disclosures. ASU 2023-09 enhances the transparency about income tax information through improvements to income tax disclosures primarily related to the rate reconciliation and income taxes paid information. The guidance is effective for public business entities for annual periods beginning after December 15, 2024. For entities other than public business entities, the amendments are effective for annual periods beginning after December 15, 2025. Early adoption is permitted. The Company is currently planning to adopt this guidance when effective. The Company is assessing the impact of the adoption on the Company’s consolidated financial statements and accompanying footnotes. |
Summary of significant accoun_3
Summary of significant accounting policies (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Accounting Policies [Abstract] | |
Reconciliation of cash | The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the consolidated balance sheet that sum to the total of the same amounts shown in the statement of cash flows: End of Beginning of period- period- December 31, December 31, 2023 2022 Cash and cash equivalents $ 594,001 $ 279,834 Restricted cash included in deposits and other assets 16,283 16,091 Total Cash, cash equivalents and restricted cash per statement of cash flows $ 610,284 $ 295,925 |
Schedule of estimated useful life of related assets | Leasehold improvements Lesser of useful life or lease term Computer equipment and software 3 years Machinery and lab equipment 7 years Furniture and fixtures 7 years |
Schedule of Inventory | The following table summarizes the components of the Company’s inventory for the periods indicated: December 31, 2023 December 31, 2022 Raw materials $ 952 $ 1,078 Work in progress 17,991 14,074 Finished goods 11,634 6,656 Total inventory $ 30,577 $ 21,808 |
Fair value of financial instr_2
Fair value of financial instruments and investments - (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Fair Value Disclosures [Abstract] | |
Schedule of marketable securities | The table presented below is a summary of changes in the fair value for the Company’s marketable securities – equity investments, ClearPoint Equity Investments, and ClearPoint convertible debt security for the years ended December 31, 2023 and 2022: Ending Foreign Ending Balance at Currency Balance at December 31, Unrealized Realized Unrealized Investments Redemptions/ December 31, 2022 Gain/(Loss) Gain/(Loss) Gain Purchased Sale 2023 Marketable securities - equity investments $ 108,261 2,517 4,383 1,384 38,432 (132,343) $ 22,634 ClearPoint Equity Investments 10,965 (1,515) (782) — — (2,594) 6,074 ClearPoint Convertible Debt Security 15,231 (2,678) — — — — 12,553 Total Fair Value $ 134,457 $ (1,676) $ 3,601 $ 1,384 $ 38,432 $ (134,937) $ 41,261 Ending Foreign Ending Balance at Currency Balance at December 31, Unrealized Unrealized Investments December 31, 2021 Gain/(Loss) Gain/(Loss) Purchased Redemptions 2022 Marketable securities - equity investments $ 206,973 (7,992) (549) 22,787 (112,958) $ 108,261 ClearPoint Equity Investments 14,525 (3,560) — — — 10,965 ClearPoint Convertible Debt Security 20,971 (5,740) — — — 15,231 Total Fair Value $ 242,469 $ (17,292) $ (549) $ 22,787 $ (112,958) $ 134,457 |
Schedule of financial assets and liabilities that are required to be measured at fair value on a recurring basis | The following represents the fair value using the hierarchy described in Note 2 for the Company’s financial assets and liabilities that are required to be measured at fair value on a recurring basis as of December 31, 2023 and 2022: December 31, 2023 Quoted prices Significant in active other Significant markets for observable unobservable identical assets inputs inputs Total (level 1) (level 2) (level 3) Marketable securities - available for sale $ 260,104 $ — $ 260,104 $ — Marketable securities - equity investments $ 22,634 $ 22,634 $ — $ — ClearPoint Equity Investments $ 6,074 $ 6,074 $ — $ — ClearPoint convertible debt security $ 12,553 $ — $ 12,553 $ — Contingent consideration payable- development and regulatory milestones $ 26,600 $ — $ — $ 26,600 Contingent consideration payable- net sales milestones $ 9,700 $ — $ — $ 9,700 December 31, 2022 Quoted prices Significant in active other Significant markets for observable unobservable identical assets inputs inputs Total (level 1) (level 2) (level 3) Marketable securities - available for sale $ 22,610 $ — $ 22,610 $ — Marketable securities - equity investments $ 108,261 $ 108,261 $ — $ — ClearPoint Equity Investments $ 10,965 $ 10,965 $ — $ — ClearPoint convertible debt security $ 15,231 $ — $ 15,231 $ — Contingent consideration payable- development and regulatory milestones $ 82,500 $ — $ — $ 82,500 Contingent consideration payable- net sales milestones and royalties $ 81,500 $ — $ — $ 81,500 |
Summary of marketable securities accounted for as available-for-sale debt securities | The following is a summary of marketable securities accounted for as available for sale debt securities at December 31, 2023 and 2022: December 31, 2023 Amortized Gross Unrealized Cost Gains Losses Fair Value Commercial paper $ 117,044 $ 128 $ (12) $ 117,160 Corporate debt securities 1,650 — (2) 1,648 Government Obligations 141,084 212 — 141,296 Total $ 259,778 $ 340 $ (14) $ 260,104 December 31, 2022 Amortized Gross Unrealized Cost Gains Losses Fair Value Commercial paper $ 12,419 $ 5 $ — $ 12,424 Corporate debt securities 10,685 — (499) 10,186 Total $ 23,104 $ 5 $ (499) $ 22,610 |
Summary of unrealized losses and fair values of available-for-sale debt securities in a continuous unrealized loss position | The unrealized losses and fair values of available for sale debt securities that have been in an unrealized loss position for a period of less than and greater than or equal to 12 months as of December 31, 2023 are as follows: December 31, 2023 Securities in an unrealized loss Securities in an unrealized loss position less than 12 months position greater than or equal to 12 months Total Unrealized losses Fair Value Unrealized losses Fair Value Unrealized losses Fair Value Commercial paper $ (12) 44,446 — — (12) 44,446 Corporate debt securities $ — — (2) 1,648 (2) $ 1,648 Total $ (12) $ 44,446 $ (2) $ 1,648 $ (14) $ 46,094 The unrealized losses and fair values of available for sale debt securities that have been in an unrealized loss position for a period of less than and greater than or equal to 12 months as of December 31, 2022 are as follows: December 31, 2022 Securities in an unrealized loss Securities in an unrealized loss position less than 12 months position greater than or equal to 12 months Total Unrealized losses Fair Value Unrealized losses Fair Value Unrealized losses Fair Value Corporate debt securities $ — — (499) 10,186 (499) $ 10,186 Total $ — $ — $ (499) $ 10,186 $ (499) $ 10,186 |
Schedule of available-for-sale debt securities | Available for sale debt securities on the balance sheet at December 31, 2023 and 2022 mature as follows: December 31, 2023 Less Than More Than 12 Months 12 Months Commercial paper $ 117,160 $ — Corporate debt securities 1,648 — Government obligations 141,296 — Total $ 260,104 $ — December 31, 2022 Less Than More Than 12 Months 12 Months Commercial paper $ 12,424 $ — Corporate debt securities — 10,186 Total $ 12,424 $ 10,186 |
Summary of changes in the fair value of the Company's Level 3 valuation for contingent consideration payable | The table presented below is a summary of changes in the fair value of the Company’s Level 3 valuation for the contingent consideration payables for the years ended December 31, 2023, and 2022: Contingent consideration payable- Contingent consideration payable- development and regulatory net sales milestones and royalties milestones - Agilis - Agilis Beginning balance as of December 31, 2021 $ 139,300 $ 100,600 Additions — — Change in fair value (6,800) (19,100) Payments (50,000) — Ending balance as of December 31, 2022 $ 82,500 $ 81,500 Additions — — Change in fair value (55,900) (71,800) Payments — — Ending balance as of December 31, 2023 $ 26,600 $ 9,700 |
Fair Value Measurement Inputs and Valuation Techniques | The following significant unobservable inputs were used in the valuation of the contingent consideration payables for the years ended December 31, 2023 and 2022: December 31, 2023 Fair Value Valuation Technique Unobservable Input Range Contingent consideration payable- $26,600 Probability-adjusted discounted cash flow Potential development and regulatory milestones $0 - $31 million Contingent considerable payable- net sales $9,700 Option-pricing model with Monte Carlo simulation Potential net sales milestones $0 - $50 million December 31, 2022 Fair Value Valuation Technique Unobservable Input Range Contingent consideration payable- $82,500 Probability-adjusted discounted cash flow Potential development and regulatory milestones $0 - $331 million Contingent considerable payable- net sales $81,500 Option-pricing model with Monte Carlo simulation Potential net sales milestones $0 - $150 million |
Fixed assets (Tables)
Fixed assets (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Property, Plant and Equipment [Abstract] | |
Schedule of fixed assets, net | Fixed assets, net were as follows at December 31, 2023 and 2022: December 31, 2023 2022 Leasehold improvements $ 30,166 $ 28,969 Computer equipment and software 17,503 15,332 Machinery and lab equipment 62,837 47,496 Furniture and fixtures 3,849 3,812 Assets in process 24,008 14,349 138,363 109,958 Less accumulated depreciation (51,274) (37,368) Total $ 87,089 $ 72,590 |
Leases - (Tables)
Leases - (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Leases | |
Schedule of lease costs | The components of lease expense were as follows: Year Ended December 31, 2023 Year Ended December 31, 2022 Year Ended December 31, 2021 Operating Lease Cost Fixed lease cost $ 21,952 $ 19,804 $ 16,411 Variable lease cost 5,846 4,557 4,361 Short-term lease cost 1,186 808 656 Total operating lease cost $ 28,984 $ 25,169 $ 21,428 Supplemental lease term and discount rate information related to leases was as follows: December 31, 2023 December 31, 2022 Weighted-average remaining lease terms - operating leases (years) 11.55 11.61 Weighted-average discount rate - operating leases 8.69 % 8.61 % Weighted-average remaining lease terms - finance lease (years) 9.01 10.01 Weighted-average discount rate - finance lease 7.80 % 7.80 % |
Schedule of supplemental cash flow information related to leases | Supplemental cash flow information related to leases was as follows: Year Ended December 31, 2023 2022 2021 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows from operating leases $ 15,338 $ 14,736 $ 13,683 Financing cash flows from finance lease 1,379 1,276 2,224 Operating cash flows from finance lease 1,621 1,724 776 Right-of-use assets obtained in exchange for lease obligations: Operating leases $ — $ 35,817 $ 645 |
Schedule of future lease payments - Operating leases | Future minimum lease payments under non-cancelable leases as of December 31, 2023 were as follows: Operating Leases Finance Lease 2024 $ 18,352 $ 3,000 2025 20,450 3,000 2026 19,992 3,000 2027 17,945 3,000 2028 and thereafter 175,847 15,000 Total lease payments 252,586 27,000 Less: Imputed Interest expense 141,957 6,816 Total $ 110,629 $ 20,184 |
Accounts payable and accrued _2
Accounts payable and accrued expenses - (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Payables and Accruals [Abstract] | |
Schedule of components of accounts payable and accrued expenses | Accounts payable and accrued expenses at December 31, 2023 and 2022 consist of the following: December 31, 2023 2022 Employee compensation, benefits, and related accruals $ 62,643 $ 62,669 Income tax payable — 4,712 Consulting and contracted research 27,500 38,882 Professional fees 2,246 3,093 Sales allowance 77,176 63,787 Sales rebates 131,334 67,355 Royalties 74,111 40,546 Accounts payable 6,045 27,268 Other 10,928 12,054 Total $ 391,983 $ 320,366 |
Debt - (Tables)
Debt - (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Debt Instrument, Redemption [Line Items] | |
Summary of liability for sale of future royalties | The following table shows the activity within the “liability for sale of future royalties- current” and “liability for sale of future royalties- noncurrent” accounts for the year ended December 31, 2023: Year Ended December 31, Liability for sale of future royalties- (current and noncurrent) 2023 Beginning balance as of December 31, 2022 $ 757,886 Less: Non-cash royalty revenue payable to RPI (93,460) Plus: Non-cash interest expense recognized 104,790 Plus: Cash Consideration 1,000,000 Plus: Loss on Debt Extinguishment 44,881 Ending balance $ 1,814,097 Effective interest rate as of December 31, 2023 10.8% |
1.50% Convertible senior notes due 2026 | |
Debt Instrument, Redemption [Line Items] | |
Summary of convertible notes | The 2026 Convertible Notes consist of the following: Year ended December 31, 2023 2022 Principal $ 287,500 $ 287,500 Less: Debt issuance costs (3,287) (4,456) Net carrying amount $ 284,213 $ 283,044 |
Summary of interest expense recognized related to debt | The following table sets forth total interest expense recognized related to the 2026 Convertible Notes: Year ended December 31, 2023 2022 Contractual interest expense $ 4,305 $ 4,313 Amortization of debt issuance costs 1,171 1,150 Total $ 5,476 $ 5,463 Effective interest rate 1.9 % 1.9 % |
3.00% Convertible senior notes due 2022 | |
Debt Instrument, Redemption [Line Items] | |
Summary of interest expense recognized related to debt | The following table sets forth total interest expense recognized related to the 2022 Convertible Notes: Year ended December 31, 2023 2022 Contractual interest expense $ — $ 2,800 Amortization of debt issuance costs — 460 Total $ — $ 3,260 Effective interest rate — % 3.5 % |
Blackstone Credit Agreement | |
Debt Instrument, Redemption [Line Items] | |
Schedule of credit agreement | The Blackstone Credit Agreement consisted of the following: Year ended December 31, 2023 2022 Principal $ 300,000 $ 300,000 Less: Debt issuance costs — (11,322) Repayment of senior secured term loan (300,000) — Net carrying amount $ — $ 288,678 |
Summary of interest expense recognized related to debt | The following table sets forth total interest expense recognized related to the Blackstone Credit Agreement: Year Ended Year Ended December 31, December 31, 2023 2022 Contractual interest expense $ 30,198 $ 6,069 Amortization of debt issuance costs 702 290 Total $ 30,900 $ 6,359 Effective interest rate 13.1 % 12.2 % |
Net loss per share - (Tables)
Net loss per share - (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Earnings Per Share [Abstract] | |
Schedule of computation of basic and diluted net loss available to common stockholders | The following table sets forth the computation of basic and diluted net loss per share for common stockholders: Year ended December 31, 2023 2022 2021 Numerator Net loss $ (626,604) $ (559,017) $ (523,901) Denominator Denominator for basic and diluted net loss per share 74,838,392 71,728,634 70,466,393 Net loss per share: Basic and diluted $ (8.37) * $ (7.79) * $ (7.43) * * For the years ended December 31, 2023, 2022, and 2021, the Company experienced a net loss and therefore did not report any dilutive share impact. |
Schedule of historical dilutive common share equivalents outstanding | The following table shows historical dilutive common share equivalents outstanding, which are not included in the above historical calculation, as the effect of their inclusion is anti-dilutive during each period. As of December 31, 2023 2022 2021 Stock Options 9,600,399 11,502,417 10,772,582 Unvested restricted stock awards and units 2,866,270 2,516,336 1,519,831 Total 12,466,669 14,018,753 12,292,413 |
Stock award plan - (Tables)
Stock award plan - (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Share-based Payment Arrangement [Abstract] | |
Summary of stock option activity | Stock option activity Weighted- Weighted- average Aggregate average remaining intrinsic Number of exercise contractual value(in options price term thousands) Outstanding at December 31, 2020 9,663,677 $ 38.72 Granted 2,487,234 $ 61.36 Exercised (635,871) $ 28.01 Forfeited (742,458) $ 52.04 Outstanding at December 31, 2021 10,772,582 $ 43.66 Granted 1,685,435 $ 38.55 Exercised (496,863) $ 29.45 Forfeited (458,737) $ 48.75 Outstanding at December 31, 2022 11,502,417 $ 43.33 Granted 1,117,284 $ 40.19 Exercised (822,482) $ 29.25 Forfeited/Cancelled (2,196,820) $ 45.85 Outstanding at December 31, 2023 9,600,399 $ 43.59 5.39 years $ 8,814 Vested or Expected to vest at December 31, 2023 1,796,687 $ 45.85 8.16 years $ 366 Exercisable at December 31, 2023 7,650,948 $ 43.08 4.68 years $ 8,387 |
Schedule of assumptions used to estimate fair values of grants made on the date of grant | The fair values of grants made in the years ended December 31, 2023, 2022 and 2021 were contemporaneously estimated on the date of grant using the following assumptions: 2023 2022 2021 Risk-free interest rate 3.54% - 4.69% 1.55% - 4.57% 0.51% - 1.24% Expected volatility 53% - 56% 54% - 74% 74% - 89% Expected term 5.5 years 5.5 years 5.5 years |
Summary of information on the Company's restricted stock | The following table summarizes information on the Company’s restricted stock awards and units: Restricted Stock Awards and Units Weighted Average Grant Number of Date Shares Fair Value Unvested at December 31, 2022 2,516,336 $ 45.67 Granted 2,104,128 38.75 Vested (920,488) 43.74 Forfeited (833,706) 42.31 Unvested at December 31, 2023 2,866,270 $ 41.82 |
Schedule of share-based compensation expense recorded in the statement of operations | The Company recorded share-based compensation expense in the statement of operations related to incentive stock options, nonstatutory stock options, restricted stock awards, restricted stock units and the ESPP as follows: Year ended December 31, 2023 2022 2021 Research and development $ 52,941 $ 55,869 $ 53,632 Selling, general and administrative 48,695 54,464 49,881 Total $ 101,636 $ 110,333 $ 103,513 |
Other comprehensive income (l_2
Other comprehensive income (loss) and accumulated other comprehensive items - (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Comprehensive Income (Loss), Net of Tax, Attributable to Parent [Abstract] | |
Summary of other comprehensive income (loss) and the changes in accumulated other comprehensive items | The following table summarizes other comprehensive (loss) income and the changes in accumulated other comprehensive items, by component, for the years ended December 31, 2023, 2022, and 2021, respectively. Unrealized Total Gains (Losses) Accumulated On Foreign Other Marketable Currency Comprehensive Securities, net of tax Translation Items Balance at December 31, 2020 $ 1,900 $ (62,857) $ (60,957) Other comprehensive (loss) income before reclassifications (3,279) 39,177 35,898 Amounts reclassified from other comprehensive items 777 — 777 Other comprehensive (loss) income (2,502) 39,177 36,675 Balance at December 31, 2021 $ (602) $ (23,680) $ (24,282) Other comprehensive income before reclassifications 4,072 28,970 33,042 Amounts reclassified from other comprehensive items (3,964) — (3,964) Other comprehensive income 108 28,970 29,078 Balance at December 31, 2022 $ (494) $ 5,290 $ 4,796 Other comprehensive income (loss) before reclassifications 1,135 (6,901) (5,766) Amounts reclassified from other comprehensive items (315) — (315) Other comprehensive income (loss) 820 (6,901) (6,081) Balance at December 31, 2023 $ 326 $ (1,611) $ (1,285) |
Income taxes (Tables)
Income taxes (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Income Tax Disclosure [Abstract] | |
Schedule of Income before Income Tax, Domestic and Foreign | The loss from operations before tax benefit (expense) consisted of the following for the years ended December 31, 2023, 2022, and 2021: 2023 2022 2021 Domestic $ (784,744) $ (591,126) $ (487,726) Foreign 88,634 3,639 (30,614) Total $ (696,110) $ (587,487) $ (518,340) |
Components of Income Tax Provision | The Income Tax Provision consisted of the following for the years ended December 31, 2023, 2022 and 2021: 2023 2022 2021 Current: U.S. Federal $ — $ — $ — U.S. State and Local 27,226 (4,224) (3,844) Foreign (4,003) (1,582) (1,340) Deferred: U.S. Federal 36,408 23,689 — U.S. State and Local 10,521 10,587 (377) Foreign (646) — — Total tax benefit (expense) $ 69,506 $ 28,470 $ (5,561) |
Schedule of reconciliation of the U.S. statutory income tax rate to the Company's effective tax rate | A reconciliation of the U.S. statutory income tax rate to the Company’s effective tax rate is as follows: December 31, 2023 2022 2021 Federal income tax provision at statutory rate 21.00 % 21.00 % 21.00 % State income tax provision, net of federal benefit 0.32 3.07 (0.74) Permanent differences (1.43) (1.83) (4.06) Research and development 4.59 5.89 4.50 Change in valuation allowances (16.86) (23.36) (29.03) Change in deferred tax assets — (0.10) 12.05 Foreign tax rate differential 0.05 (0.17) 0.01 Tax rate change (1.26) 0.34 0.01 Release (Accrual) of uncertain tax positions 3.71 — (4.78) Other (0.12) — (0.03) Effective income tax rate 10.00 % 4.84 % (1.07) % |
Schedule of significant components of the Company's deferred tax assets and liabilities | December 31, 2023 2022 Assets: Noncurrent deferred income taxes $ — $ — Liabilities: Noncurrent deferred income taxes (55,905) (102,834) Deferred income taxes - net $ (55,905) $ (102,834) The significant components of the Company’s deferred tax assets and liabilities at December 31, 2023 and 2022 are as follows: 2023 2022 Deferred tax assets: Accrued expense $ 25,400 $ 2,124 Amortization 137,808 52,532 Federal tax credits 205,485 174,802 State tax credits 9,817 9,787 Federal net operating losses 60,270 69,957 State net operating losses 18,680 10,316 Foreign net operating losses 4,052 4,837 Capitalized research and development costs 149,683 110,219 Share based compensation and other 30,757 27,054 Liability for sale of future royalties 190,659 185,589 Noncash interest expense 9,410 30,160 Other comprehensive loss (728) (719) Total gross deferred tax assets 841,293 676,658 Less valuation allowance (833,810) (672,172) Total deferred tax assets, net of valuation allowance $ 7,483 $ 4,486 Deferred tax liabilities: Depreciation $ (7,483) $ (4,486) Indefinite lived intangible (55,905) (102,834) Total gross deferred tax liabilities (63,388) (107,320) Net deferred tax assets (liabilities) $ (55,905) $ (102,834) |
Schedule of unrecognized tax benefits | A reconciliation of the gross amount of unrecognized tax benefits, excluding accrued interest and penalties, is as follows: Unrecognized Tax Benefits Balance at December 31, 2022 27,217 Reductions based on settlements or expiration of statute of limitations (24,671) Interest received (1,186) Balance at December 31, 2023 $ 1,360 |
Geographic information (Tables)
Geographic information (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Segments, Geographical Areas [Abstract] | |
Summary of financial information based on geographical location | Year Ended December 31, 2023 United States Non-US Total Total assets $ 1,582,962 $ 312,736 $ 1,895,698 Fixed assets, net $ 86,421 $ 668 $ 87,089 Revenue $ 531,661 $ 406,161 $ 937,822 Year Ended December 31, 2022 United States Non-US Total Total assets $ 1,473,770 $ 231,849 $ 1,705,619 Fixed assets, net $ 71,754 $ 836 $ 72,590 Revenue $ 381,862 $ 316,939 $ 698,801 |
Intangible assets and goodwill
Intangible assets and goodwill - (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Schedule of definite lived intangible assets | Definite lived intangible assets consisted of the following at December 31, 2023 and 2022: Ending Balance at Foreign Ending Balance at Definite-lived December 31, currency December 31, intangibles assets, gross 2022 Additions translation 2023 Emflaza $ 420,253 $ 107,164 $ — $ 527,417 Waylivra 9,316 533 369 10,218 Tegsedi 7,109 5,839 374 13,322 Upstaza 89,550 — — 89,550 Total definite-lived intangibles, gross $ 526,228 $ 113,536 $ 743 $ 640,507 Ending Balance at Foreign Ending Balance at Definite-lived December 31, currency December 31, intangibles assets, accumulated amortization 2022 Amortization translation 2023 Emflaza $ (266,023) $ (212,595) $ — $ (478,618) Waylivra (2,751) (1,080) (134) (3,965) Tegsedi (1,709) (1,498) (104) (3,311) Upstaza (3,420) (7,462) — (10,882) Total definite-lived intangibles, accumulated amortization $ (273,903) $ (222,635) $ (238) $ (496,776) Total definite-lived intangibles, net $ 143,731 |
Schedule of estimated future amortization of intangible assets | The estimated future amortization of the Emflaza rights, Waylivra, Tegsedi, and Upstaza intangible assets is expected to be as follows: As of December 31, 2023 2024 $ 59,312 2025 10,512 2026 10,512 2027 10,512 2028 and thereafter 52,883 Total $ 143,731 |
Schedule of indefinite lived intangible assets | Indefinite lived intangible assets consisted of the following at December 31, 2023 and 2022: Ending Balance at Reclass from Foreign Ending Balance at Indefinite-lived December 31, Indefinite Lived to currency December 31, intangibles assets 2022 Additions Definite Lived Impairment translation 2023 Upstaza $ 235,766 $ — $ — $ — $ — $ 235,766 PTC-FA 112,500 — — (112,500) — — PTC-AS 105,300 — — (105,300) — — Total indefinite-lived intangibles $ 453,566 $ — $ — $ (217,800) $ — $ 235,766 Total intangible assets, net $ 379,497 |
The Company (Details)
The Company (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Long-term debt | ||
Accumulated deficit | $ (3,283,578) | $ (2,656,974) |
Summary of significant accoun_4
Summary of significant accounting policies - Narrative (Details) $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | ||||||||
Jun. 30, 2022 USD ($) | Jun. 30, 2023 USD ($) | Dec. 31, 2023 USD ($) segment | Dec. 31, 2023 USD ($) country | Dec. 31, 2023 USD ($) | Dec. 31, 2023 USD ($) | Dec. 31, 2023 USD ($) item | Dec. 31, 2022 USD ($) country segment | Dec. 31, 2021 USD ($) country | Dec. 31, 2019 USD ($) | Dec. 31, 2018 USD ($) | |
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Number of operating segments | segment | 1 | 1 | |||||||||
Number of reporting units | 1 | 1 | |||||||||
Allowance for credit loss | $ 0 | $ 0 | $ 0 | $ 0 | $ 0 | $ 0 | |||||
Increase in allowance for credit losses | 0 | 0 | |||||||||
Allowance for doubtful accounts receivable | 1,200 | 1,200 | 1,200 | $ 1,200 | 1,200 | 300 | |||||
Bad debt expense | 900 | 200 | $ 100 | ||||||||
Revenue | 937,822 | 698,801 | 538,593 | ||||||||
Cash consideration received from Royalty Purchase Agreement | 1,000,000 | ||||||||||
State income tax provision | (27,226) | $ 4,224 | $ 3,844 | ||||||||
Income taxes | |||||||||||
Federal income tax statutory rate | 21% | 21% | 21% | ||||||||
Deferred tax liability | 63,388 | 63,388 | 63,388 | $ 63,388 | 63,388 | $ 107,320 | |||||
Deferred tax benefit | 46,930 | 34,276 | $ (377) | ||||||||
Impairment of long-lived assets | 0 | ||||||||||
Non cash realized foreign exchange loss | 16,887 | ||||||||||
Increase in current taxable income resulting from the elimination of current deduction of qualifying R&D costs under Section 174. | 300,300 | ||||||||||
Impairment of indefinite-lived intangibles excluding goodwill | 217,800 | 33,384 | |||||||||
Impairment of goodwill | 0 | ||||||||||
Upstaza | |||||||||||
Income taxes | |||||||||||
Impairment of indefinite-lived intangibles excluding goodwill | 0 | ||||||||||
Indefinite-lived intangible assets | |||||||||||
Income taxes | |||||||||||
Impairment of indefinite-lived intangibles excluding goodwill | 217,800 | ||||||||||
Indefinite-lived intangible assets | PTC-FA and PTC-AS | |||||||||||
Income taxes | |||||||||||
Impairment of indefinite-lived intangibles excluding goodwill | $ 217,800 | 217,800 | |||||||||
Indefinite-lived intangible assets | Upstaza | |||||||||||
Income taxes | |||||||||||
Impairment of indefinite-lived intangibles excluding goodwill | 0 | ||||||||||
Agilis | |||||||||||
Income taxes | |||||||||||
Deferred tax liability | $ 122,000 | ||||||||||
Impairment of goodwill | 0 | ||||||||||
Prepaids and other current assets | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Deferred research and development advance payments, short-term | 2,600 | 2,600 | 2,600 | 2,600 | 2,600 | 2,400 | |||||
Deposits and other assets | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Bank guarantee denominated in foreign currency | 600 | 600 | 600 | 600 | 600 | ||||||
Deferred research and development advance payments, long-term | $ 4,700 | $ 4,700 | 4,700 | $ 4,700 | $ 4,700 | 3,900 | |||||
Hopewell Campus | Minimum | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Term for letter of credit (in years) | 5 years | ||||||||||
Hopewell Campus | Deposits and other assets | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Letters of credit | $ 7,500 | ||||||||||
Hopewell Campus | Fifth Anniversary | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Letters of credit | $ 3,800 | ||||||||||
Term for letter of credit (in years) | 5 years | ||||||||||
Warren Premises | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Period after which letter of credit may be reduced if lease is not in default | 5 years | ||||||||||
Warren Premises | Deposits and other assets | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Letters of credit | $ 8,100 | ||||||||||
Warren Premises | Fifth Anniversary | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Letters of credit | $ 4,100 | ||||||||||
Non-US | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Revenue | 406,161 | 316,939 | |||||||||
United States | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Revenue | 531,661 | $ 381,862 | |||||||||
Net product revenue | Geographic concentration risk | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Number of countries exceeding 10% of net product sales | country | 2 | 2 | 1 | ||||||||
Percentage of net product sales threshold | 10% | 10% | 10% | ||||||||
Net product revenue | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Revenue | 661,249 | $ 535,228 | $ 428,904 | ||||||||
Net product revenue | Non-US | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Revenue | 406,100 | 316,900 | 241,600 | ||||||||
Net product revenue | Net product revenue | Geographic concentration risk | Russia | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Revenue | 86,000 | 59,700 | |||||||||
Net product revenue | Net product revenue | Geographic concentration risk | United States | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Revenue | 255,100 | 218,300 | |||||||||
Emflaza | United States | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Revenue | 255,100 | 218,300 | 187,300 | ||||||||
Translarna | Non-US | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Revenue | $ 355,800 | $ 288,600 | $ 236,000 |
Summary of significant accoun_5
Summary of significant accounting policies - Reconciliation of cash (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 |
Accounting Policies [Abstract] | ||||
Cash and cash equivalents | $ 594,001 | $ 279,834 | ||
Restricted cash included in deposits and other assets | 16,283 | 16,091 | ||
Total Cash, cash equivalents and restricted cash per statement of cash flows | $ 610,284 | $ 295,925 | $ 197,218 | $ 216,312 |
Summary of significant accoun_6
Summary of significant accounting policies - Fixed Assets (Details) | Dec. 31, 2023 |
Computer equipment and software | |
Fixed assets | |
Estimated useful life | 3 years |
Machinery and lab equipment | |
Fixed assets | |
Estimated useful life | 7 years |
Furniture, fixtures, machinery and lab equipment | |
Fixed assets | |
Estimated useful life | 7 years |
Summary of significant accoun_7
Summary of significant accounting policies - Inventory (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Accounting Policies [Abstract] | ||
Raw materials | $ 952 | $ 1,078 |
Work in progress | 17,991 | 14,074 |
Finished goods | 11,634 | 6,656 |
Total inventory | 30,577 | 21,808 |
Inventory write-down | $ 12,500 | $ 1,700 |
Summary of significant accoun_8
Summary of significant accounting policies - Collaboration and Royalty Revenue (Details) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | |||
Sep. 30, 2022 | Dec. 31, 2021 | Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Collaboration and royalty revenue | |||||
Revenue | $ 937,822 | $ 698,801 | $ 538,593 | ||
Collaboration revenue | |||||
Collaboration and royalty revenue | |||||
Revenue | 100,030 | 50,052 | 55,046 | ||
Royalty revenue | |||||
Collaboration and royalty revenue | |||||
Revenue | 168,856 | 113,521 | 54,643 | ||
SMA License Agreement | Collaboration revenue | |||||
Collaboration and royalty revenue | |||||
Revenue | 100,000 | 50,100 | 55,000 | ||
SMA License Agreement | Royalty revenue | |||||
Collaboration and royalty revenue | |||||
Revenue | 168,900 | $ 113,500 | $ 54,600 | ||
SMA License Agreement | Sales Milestones | |||||
Collaboration and royalty revenue | |||||
Net sales milestone threshold achieved | $ 750,000 | $ 500,000 | 1,500,000 | ||
SMA License Agreement | Sales Milestones | Prepaids and other current assets | |||||
Collaboration and royalty revenue | |||||
Milestone payments due | $ 50,000 | $ 25,000 | $ 100,000 |
Summary of significant accoun_9
Summary of significant accounting policies - Manufacturing Revenue (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Revenue recognition | |||
Revenue | $ 937,822 | $ 698,801 | $ 538,593 |
Remaining performance obligations | 1,400 | ||
Manufacturing revenue | |||
Revenue recognition | |||
Revenue | 7,687 | 0 | $ 0 |
Contract with customer, asset | 200 | 0 | |
Remaining performance obligations | $ 800 | $ 1,400 |
Summary of significant accou_10
Summary of significant accounting policies - Liability for Sale of Future Royalties (Details) $ in Thousands | 12 Months Ended | ||
Oct. 18, 2023 USD ($) Option | Jul. 17, 2020 USD ($) | Dec. 31, 2023 USD ($) | |
Assigned royalty payments | |||
Cash consideration received from Royalty Purchase Agreement | $ 1,000,000 | ||
Assigned royalty payment, retained percentage | 57.067% | ||
Remaining potential milestones that can be achieved | $ 150,000 | ||
Effective interest rate of the liability component | 10.80% | ||
Loss on extinguishment of debt | $ 137,558 | ||
Aggregate Royalty Purchase Agreements | Royalty Pharma | |||
Assigned royalty payments | |||
Total percentage of royalty rights assigned | 80.9777% | ||
Original Royalty Purchase Agreement | |||
Assigned royalty payments | |||
Assigned royalty payment, percentage | 42.933% | ||
Cash consideration received from Royalty Purchase Agreement | $ 650,000 | ||
Royalty purchase agreement termination period once there are no further royalty payment obligations | 60 days | ||
Royalty purchase agreement, payment maximum | $ 1,300,000 | $ 1,300,000 | |
Original Royalty Purchase Agreement | Royalty Pharma | |||
Assigned royalty payments | |||
Royalty purchase agreement, payment maximum | 1,300,000 | ||
A&R Royalty Purchase Agreement | |||
Assigned royalty payments | |||
Cash consideration received from Royalty Purchase Agreement | $ 1,000,000 | ||
Royalty purchase agreement termination period once there are no further royalty payment obligations | 60 days | ||
Effective interest rate of the liability component | 10.80% | ||
Retained Royalty Rights percentage sold | 38.0447% | ||
Percentage of total royalty payable after threshold royalties received | 66.6667% | ||
Additional cash consideration | $ 500,000 | ||
Number of put options held by the company | Option | 5 | ||
Maximum number of put options which can be exercised before Royalty Pharma can exercise a call option | Option | 2 | ||
Maximum percentage of remaining retained royalty rights that are available upon exercise of call option | 50% | ||
Fair value of liability for sale of future royalties. | $ 1,809,900 | ||
Loss on extinguishment of debt | $ 44,900 | ||
A&R Royalty Purchase Agreement | Royalty Pharma | |||
Assigned royalty payments | |||
Percentage of maximum royalty payable until assigned royalty rights | 90.4888% | ||
Percentage of maximum royalty payable thereafter assigned royalty rights | 83.3333% |
Fair value of financial instr_3
Fair value of financial instruments and investments - Marketable Securities (Details) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | ||
Jan. 31, 2020 USD ($) $ / shares | Dec. 31, 2023 USD ($) fund | Dec. 31, 2022 USD ($) | Dec. 31, 2021 USD ($) | |
Changes in fair value | ||||
Unrealized Gain/(Loss) | $ 2,517 | $ (7,992) | $ 1,673 | |
Realized Gain/(Loss) | 4,383 | |||
Investments Purchased | 38,398 | 22,787 | 210,018 | |
Redemptions/Sale | (132,228) | (112,958) | (4,281) | |
Unrealized Gain/(Loss) | (1,515) | (3,560) | (6,078) | |
Realized Gain/(Loss) | (782) | |||
Redemptions/Sale | (2,594) | |||
Unrealized Gain/(Loss) | (2,678) | (5,740) | (8,281) | |
Beginning Balance - Total | 134,457 | 242,469 | ||
Unrealized Gain/(Loss) - Total | (1,676) | (17,292) | ||
Realized Gain/(Loss) - Total | 3,601 | |||
Foreign Currency Unrealized Gain/(Loss) - Total | 1,384 | (549) | ||
Investments Purchased - Total | 38,432 | 22,787 | ||
Redemptions/Sale - Total | (134,937) | (112,958) | ||
Ending Balance - Total | $ 41,261 | 134,457 | 242,469 | |
Equity Investments | ||||
Number of mutual funds denominated in a foreign currency | fund | 1 | |||
ClearPoint Convertible note | ||||
Purchase of convertible note | $ 174,086 | 52,764 | 333,148 | |
Marketable securities - equity investments | ||||
Changes in fair value | ||||
Beginning Balance | 108,261 | 206,973 | ||
Unrealized Gain/(Loss) | 2,517 | (7,992) | ||
Realized Gain/(Loss) | 4,383 | |||
Foreign Currency Unrealized Gain/(Loss) | 1,384 | (549) | ||
Investments Purchased | 38,432 | 22,787 | ||
Redemptions/Sale | (132,343) | (112,958) | ||
Ending Balance | 22,634 | 108,261 | 206,973 | |
ClearPoint Equity Investment | ||||
Changes in fair value | ||||
Beginning Balance | 10,965 | 14,525 | ||
Unrealized Gain/(Loss) | (1,515) | (3,560) | ||
Realized Gain/(Loss) | (782) | |||
Redemptions/Sale | (2,594) | |||
Ending Balance | 6,074 | 10,965 | 14,525 | |
ClearPoint convertible debt | ||||
Changes in fair value | ||||
Beginning Balance | 15,231 | 20,971 | ||
Unrealized Gain/(Loss) | (2,678) | (5,740) | ||
Ending Balance | $ 12,553 | $ 15,231 | $ 20,971 | |
ClearPoint Convertible note | ||||
Purchase of convertible note | $ 10,000 | |||
Conversion price - convertible note | $ / shares | $ 6 | |||
Term of convertible note | 5 years |
Fair value of financial instr_4
Fair value of financial instruments and investments - Hierarchy (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Financial assets and liabilities measured at fair value on recurring basis | ||
Marketable securities - available for sale | $ 260,104 | $ 22,610 |
Contingent consideration payable | 36,300 | 164,000 |
Transfers from Level 1 to Level 2 | 0 | 0 |
Transfers from Level 2 to Level 1 | 0 | 0 |
Recurring basis | ||
Financial assets and liabilities measured at fair value on recurring basis | ||
Marketable securities - available for sale | 260,104 | 22,610 |
Marketable securities - equity investments | 22,634 | 108,261 |
ClearPoint Equity Investments | 6,074 | 10,965 |
ClearPoint convertible debt security | 12,553 | 15,231 |
Recurring basis | Quoted prices in active markets for identical assets (Level 1) | ||
Financial assets and liabilities measured at fair value on recurring basis | ||
Marketable securities - equity investments | 22,634 | 108,261 |
ClearPoint Equity Investments | 6,074 | 10,965 |
Recurring basis | Significant other observable inputs (Level 2) | ||
Financial assets and liabilities measured at fair value on recurring basis | ||
Marketable securities - available for sale | 260,104 | 22,610 |
ClearPoint convertible debt security | 12,553 | 15,231 |
Recurring basis | Development and Regulatory Milestones | ||
Financial assets and liabilities measured at fair value on recurring basis | ||
Contingent consideration payable | 26,600 | 82,500 |
Recurring basis | Development and Regulatory Milestones | Significant unobservable inputs (Level 3) | ||
Financial assets and liabilities measured at fair value on recurring basis | ||
Contingent consideration payable | 26,600 | 82,500 |
Recurring basis | Net Sales Milestones and Royalties | ||
Financial assets and liabilities measured at fair value on recurring basis | ||
Contingent consideration payable | 9,700 | 81,500 |
Recurring basis | Net Sales Milestones and Royalties | Significant unobservable inputs (Level 3) | ||
Financial assets and liabilities measured at fair value on recurring basis | ||
Contingent consideration payable | $ 9,700 | $ 81,500 |
Fair value of financial instr_5
Fair value of financial instruments and investments - Marketable Securities, Unrealized Gains (Losses) (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | $ 259,778 | $ 23,104 |
Gross Unrealized, Gain | 340 | 5 |
Gross Unrealized, Loss | (14) | (499) |
Fair Value | 260,104 | 22,610 |
Write downs of available for sale debt securities | 0 | 0 |
Increase in allowance for credit losses | 0 | 0 |
Realized gain (loss) from sale of marketable securities | (300) | 4,000 |
Commercial paper | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 117,044 | 12,419 |
Gross Unrealized, Gain | 128 | 5 |
Gross Unrealized, Loss | (12) | 0 |
Fair Value | 117,160 | 12,424 |
Corporate debt securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 1,650 | 10,685 |
Gross Unrealized, Gain | 0 | 0 |
Gross Unrealized, Loss | (2) | (499) |
Fair Value | 1,648 | $ 10,186 |
Government obligations | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 141,084 | |
Gross Unrealized, Gain | 212 | |
Gross Unrealized, Loss | 0 | |
Fair Value | $ 141,296 |
Fair value of financial instr_6
Fair value of financial instruments and investments - Available-for-sale securities (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Debt Securities, Available-for-sale [Line Items] | ||
Securities in an unrealized loss position less than 12 months - Unrealized losses | $ (12) | |
Securities in an unrealized loss position less than 12 months - Fair Value | 44,446 | |
Securities in an unrealized loss position greater than 12 months - Unrealized losses | (2) | $ (499) |
Securities in an unrealized loss position greater than 12 months - Fair Value | 1,648 | 10,186 |
Total - Unrealized losses | (14) | (499) |
Total - Fair Value | 46,094 | 10,186 |
Commercial paper | ||
Debt Securities, Available-for-sale [Line Items] | ||
Securities in an unrealized loss position less than 12 months - Unrealized losses | (12) | |
Securities in an unrealized loss position less than 12 months - Fair Value | 44,446 | |
Total - Unrealized losses | (12) | |
Total - Fair Value | 44,446 | |
Corporate debt securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Securities in an unrealized loss position greater than 12 months - Unrealized losses | (2) | (499) |
Securities in an unrealized loss position greater than 12 months - Fair Value | 1,648 | 10,186 |
Total - Unrealized losses | (2) | (499) |
Total - Fair Value | $ 1,648 | $ 10,186 |
Fair value of financial instr_7
Fair value of financial instruments and investments - Marketable Securities, Balance Sheet Disclosures (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Debt Securities, Available-for-sale [Line Items] | ||
Marketable securities, Less Than 12 Months | $ 260,104 | $ 12,424 |
Marketable securities, More Than 12 Months | 10,186 | |
Commercial paper | ||
Debt Securities, Available-for-sale [Line Items] | ||
Marketable securities, Less Than 12 Months | 117,160 | 12,424 |
Corporate debt securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Marketable securities, Less Than 12 Months | 1,648 | |
Marketable securities, More Than 12 Months | $ 10,186 | |
Government obligations | ||
Debt Securities, Available-for-sale [Line Items] | ||
Marketable securities, Less Than 12 Months | $ 141,296 |
Fair value of financial instr_8
Fair value of financial instruments and investments - Convertible senior notes (Details) - Convertible debt - 1.50% Convertible senior notes due 2026 - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 | Sep. 30, 2019 |
Long-term debt | |||
Principal | $ 287,500 | $ 287,500 | $ 287,500 |
Interest rate ( as a percent ) | 1.50% | ||
Significant other observable inputs (level 2) | |||
Long-term debt | |||
Fair value of convertible notes | $ 265,300 | $ 281,700 |
Fair value of financial instr_9
Fair value of financial instruments and investments - Summary of changes in the fair value of the Company's Level 3 valuation (Details) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | |
Jul. 31, 2022 | Dec. 31, 2023 | Dec. 31, 2022 | |
Changes in the fair value of warrant liability and SARs liability | |||
Change in fair value - Income Statement location | Business Combination, Contingent Consideration Arrangements, Change in Amount of Contingent Consideration, Asset | ||
PTC-FA and PTC-AS | |||
Changes in the fair value of warrant liability and SARs liability | |||
Change in fair value | $ 128,400 | ||
Ending Balance | 0 | ||
Upstaza | |||
Changes in the fair value of warrant liability and SARs liability | |||
Ending Balance | 36,300 | ||
Development and Regulatory Milestones | Significant unobservable inputs (Level 3) | Agilis | |||
Changes in the fair value of warrant liability and SARs liability | |||
Ending Balance | 26,600 | ||
Development and Regulatory Milestones | Significant unobservable inputs (Level 3) | Agilis | Commitments | |||
Changes in the fair value of warrant liability and SARs liability | |||
Beginning Balance | 82,500 | $ 139,300 | |
Change in fair value | (55,900) | (6,800) | |
Payments | $ (50,000) | (50,000) | |
Ending Balance | 26,600 | 82,500 | |
Net Sales Milestones and Royalties | Significant unobservable inputs (Level 3) | Agilis | |||
Changes in the fair value of warrant liability and SARs liability | |||
Ending Balance | 9,700 | ||
Net Sales Milestones and Royalties | Significant unobservable inputs (Level 3) | Agilis | Commitments | |||
Changes in the fair value of warrant liability and SARs liability | |||
Beginning Balance | 81,500 | 100,600 | |
Change in fair value | (71,800) | (19,100) | |
Ending Balance | $ 9,700 | $ 81,500 |
Fair value of financial inst_10
Fair value of financial instruments and investments - Fair Value Liabilities Measured (Details) - Agilis | Dec. 31, 2023 USD ($) | Dec. 31, 2022 USD ($) | Dec. 31, 2021 USD ($) |
Development and Regulatory Milestones | Valuation Technique, Discounted Cash Flow | Probability of Success | Weighted Average | |||
Fair Value Valuation Inputs | |||
Rights Exchange Settlement measurement | 0.90 | ||
Development and Regulatory Milestones | Valuation Technique, Discounted Cash Flow | Measurement Input, Discount Rate | Weighted Average | |||
Fair Value Valuation Inputs | |||
Rights Exchange Settlement measurement | 0.059 | ||
Development and Regulatory Milestones | Commitments | Valuation Technique, Discounted Cash Flow | Development and Regulatory Milestone | Minimum | |||
Fair Value Valuation Inputs | |||
Potential milestones | $ 0 | $ 0 | |
Development and Regulatory Milestones | Commitments | Valuation Technique, Discounted Cash Flow | Development and Regulatory Milestone | Maximum | |||
Fair Value Valuation Inputs | |||
Potential milestones | $ 31,000,000 | $ 331,000,000 | |
Development and Regulatory Milestones | Commitments | Valuation Technique, Discounted Cash Flow | Probability of Success | Minimum | |||
Fair Value Valuation Inputs | |||
Rights Exchange Settlement measurement | 0.85 | 0.25 | |
Development and Regulatory Milestones | Commitments | Valuation Technique, Discounted Cash Flow | Probability of Success | Maximum | |||
Fair Value Valuation Inputs | |||
Rights Exchange Settlement measurement | 0.92 | 0.92 | |
Development and Regulatory Milestones | Commitments | Valuation Technique, Discounted Cash Flow | Measurement Input, Discount Rate | Minimum | |||
Fair Value Valuation Inputs | |||
Rights Exchange Settlement measurement | 0.058 | 0.062 | |
Development and Regulatory Milestones | Commitments | Valuation Technique, Discounted Cash Flow | Measurement Input, Discount Rate | Maximum | |||
Fair Value Valuation Inputs | |||
Rights Exchange Settlement measurement | 0.061 | 0.083 | |
Development and Regulatory Milestones | Significant unobservable inputs (Level 3) | |||
Fair Value Valuation Inputs | |||
Fair value | $ 26,600,000 | ||
Development and Regulatory Milestones | Significant unobservable inputs (Level 3) | Commitments | |||
Fair Value Valuation Inputs | |||
Fair value | $ 26,600,000 | $ 82,500,000 | $ 139,300,000 |
Net Sales Milestones and Royalties | Valuation Technique, Option Pricing Model | Probability of Success | Weighted Average | |||
Fair Value Valuation Inputs | |||
Rights Exchange Settlement measurement | 0.93 | ||
Net Sales Milestones and Royalties | Valuation Technique, Option Pricing Model | Measurement Input, Discount Rate | Weighted Average | |||
Fair Value Valuation Inputs | |||
Rights Exchange Settlement measurement | 0.110 | ||
Net Sales Milestones and Royalties | Commitments | Valuation Technique, Option Pricing Model | Sales Milestones | Minimum | |||
Fair Value Valuation Inputs | |||
Potential milestones | $ 0 | 0 | |
Net Sales Milestones and Royalties | Commitments | Valuation Technique, Option Pricing Model | Sales Milestones | Maximum | |||
Fair Value Valuation Inputs | |||
Potential milestones | $ 50,000,000 | $ 150,000,000 | |
Net Sales Milestones and Royalties | Commitments | Valuation Technique, Option Pricing Model | Probability of Success | Minimum | |||
Fair Value Valuation Inputs | |||
Rights Exchange Settlement measurement | 0.85 | 0.25 | |
Net Sales Milestones and Royalties | Commitments | Valuation Technique, Option Pricing Model | Probability of Success | Maximum | |||
Fair Value Valuation Inputs | |||
Rights Exchange Settlement measurement | 1 | 1 | |
Net Sales Milestones and Royalties | Commitments | Valuation Technique, Option Pricing Model | Percentage of Sales for Royalties | |||
Fair Value Valuation Inputs | |||
Rights Exchange Settlement measurement | 0 | ||
Net Sales Milestones and Royalties | Commitments | Valuation Technique, Option Pricing Model | Percentage of Sales for Royalties | Minimum | |||
Fair Value Valuation Inputs | |||
Rights Exchange Settlement measurement | 0.02 | ||
Net Sales Milestones and Royalties | Commitments | Valuation Technique, Option Pricing Model | Percentage of Sales for Royalties | Maximum | |||
Fair Value Valuation Inputs | |||
Rights Exchange Settlement measurement | 0.06 | ||
Net Sales Milestones and Royalties | Commitments | Valuation Technique, Option Pricing Model | Measurement Input, Discount Rate | |||
Fair Value Valuation Inputs | |||
Rights Exchange Settlement measurement | 0.11 | 0.115 | |
Net Sales Milestones and Royalties | Significant unobservable inputs (Level 3) | |||
Fair Value Valuation Inputs | |||
Fair value | $ 9,700,000 | ||
Net Sales Milestones and Royalties | Significant unobservable inputs (Level 3) | Commitments | |||
Fair Value Valuation Inputs | |||
Fair value | $ 9,700,000 | $ 81,500,000 | $ 100,600,000 |
Fixed assets (Details)
Fixed assets (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Fixed assets | |||
Gross fixed assets | $ 138,363 | $ 109,958 | |
Less accumulated depreciation and amortization | (51,274) | (37,368) | |
Property, Plant and Equipment, Net, Total | 87,089 | 72,590 | |
Depreciation and amortization | 13,900 | 12,300 | $ 9,400 |
Leasehold improvements | |||
Fixed assets | |||
Gross fixed assets | 30,166 | 28,969 | |
Computer equipment and software | |||
Fixed assets | |||
Gross fixed assets | 17,503 | 15,332 | |
Machinery and lab equipment | |||
Fixed assets | |||
Gross fixed assets | 62,837 | 47,496 | |
Furniture and fixtures | |||
Fixed assets | |||
Gross fixed assets | 3,849 | 3,812 | |
Assets in process | |||
Fixed assets | |||
Gross fixed assets | $ 24,008 | $ 14,349 |
Leases - Narrative (Details)
Leases - Narrative (Details) $ in Thousands | 1 Months Ended | 12 Months Ended | |||
Jul. 01, 2020 USD ($) ft² item | May 31, 2022 USD ($) ft² building period | Dec. 31, 2023 USD ($) lease | Dec. 31, 2022 USD ($) | Dec. 31, 2021 USD ($) | |
Leases | |||||
Operating lease, expense | $ 29,000 | $ 25,200 | $ 21,400 | ||
Finance Lease, Liability | 20,184 | ||||
Finance lease liabilities- current | 3,000 | 3,000 | |||
Finance lease liabilities- noncurrent | 17,184 | 18,675 | |||
Aggregate rent, Initial term | 252,586 | ||||
Operating lease ROU assets | 91,896 | 102,430 | |||
Operating Lease, Liability | 110,629 | ||||
MassBio | |||||
Leases | |||||
Finance lease cost | 1,500 | 1,600 | |||
Finance lease liabilities- current | 3,000 | 3,000 | |||
Finance lease liabilities- noncurrent | $ 17,200 | $ 18,700 | |||
Minimum | |||||
Leases | |||||
Operating lease, term of contract | 2 months 12 days | ||||
Maximum | |||||
Leases | |||||
Operating lease, term of contract | 15 years 4 months 24 days | ||||
Renewal term | 15 years | ||||
Principal Office - NJ | |||||
Leases | |||||
Number of operating leases | lease | 2 | ||||
Lessee, Operating Lease, Existence of Option to Terminate [true false] | false | ||||
Hopewell Campus | |||||
Leases | |||||
Operating lease, term of contract | 15 years | ||||
Area of real estate property | ft² | 220,500 | ||||
Renewal term | 10 years | ||||
Number of renewable terms | item | 2 | ||||
Lessee, Operating Lease, Existence of Option to Extend [true false] | true | ||||
Aggregate rent, Initial term | $ 111,500 | ||||
Percent of market rate | 95% | ||||
Warren Premises | |||||
Leases | |||||
Operating lease, term of contract | 17 years | ||||
Number of buildings leased | building | 2 | ||||
Area of real estate property | ft² | 360,000 | ||||
Renewal term | 5 years | ||||
Number of renewable terms | period | 3 | ||||
Lessee, Operating Lease, Existence of Option to Extend [true false] | true | ||||
Aggregate rent, Initial term | $ 163,000 | ||||
Operating lease ROU assets | 28,900 | ||||
Operating Lease, Liability | $ 28,900 | ||||
Base rent abatement period | 3 years | ||||
Base rent abatement | $ 18,600 | ||||
Aggregate rent net of base rate abatement, initial term | 144,400 | ||||
Allowance for lease improvements | 36,200 | ||||
Commitment to fund construction account | 3,600 | ||||
Payment due from lessor at issuance of temporary certificate of occupancy | $ 5,000 |
Leases - Lease costs (Details)
Leases - Lease costs (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Leases | |||
Fixed lease cost | $ 21,952 | $ 19,804 | $ 16,411 |
Variable lease cost | 5,846 | 4,557 | 4,361 |
Short-term lease cost | 1,186 | 808 | 656 |
Total operating lease cost | $ 28,984 | $ 25,169 | $ 21,428 |
Leases - Supplemental lease ter
Leases - Supplemental lease terms (Details) | Dec. 31, 2023 | Dec. 31, 2022 |
Leases | ||
Weighted-average remaining lease terms - operating leases (years) | 11 years 6 months 18 days | 11 years 7 months 9 days |
Weighted-average discount rate - operating leases | 8.69% | 8.61% |
Weighted-average remaining lease terms - finance lease (years) | 9 years 3 days | 10 years 3 days |
Weighted-average discount rate - finance lease | 7.80% | 7.80% |
Leases - Cash flow (Details)
Leases - Cash flow (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Leases | |||
Operating cash flows from operating leases | $ 15,338 | $ 14,736 | $ 13,683 |
Financing cash flows from finance lease | 1,379 | 1,276 | 2,224 |
Operating cash flows from finance leases | $ 1,621 | 1,724 | 776 |
Right-of-use assets obtained in exchange for operating lease obligations | $ 35,817 | $ 645 |
Leases - Lease payments (Detail
Leases - Lease payments (Details) $ in Thousands | Dec. 31, 2023 USD ($) |
Operating leases | |
2024 | $ 18,352 |
2025 | 20,450 |
2026 | 19,992 |
2027 | 17,945 |
2027 and thereafter | 175,847 |
Total lease payments | 252,586 |
Less: Imputed Interest expense | 141,957 |
Total | 110,629 |
Finance leases | |
2024 | 3,000 |
2025 | 3,000 |
2026 | 3,000 |
2025 | 3,000 |
2027 and thereafter | 15,000 |
Total lease payments | 27,000 |
Less: Imputed Interest expense | 6,816 |
Total | $ 20,184 |
Accounts payable and accrued _3
Accounts payable and accrued expenses - Components (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Payables and Accruals [Abstract] | ||
Employee compensation, benefits, and related accruals | $ 62,643 | $ 62,669 |
Income tax payable | 4,712 | |
Consulting and contracted research | 27,500 | 38,882 |
Professional fees | 2,246 | 3,093 |
Sales allowance | 77,176 | 63,787 |
Sales rebates | 131,334 | 67,355 |
Royalties | 74,111 | 40,546 |
Accounts payable | 6,045 | 27,268 |
Other | 10,928 | 12,054 |
Accounts payable and accrued expenses | $ 391,983 | $ 320,366 |
Accounts payable and accrued _4
Accounts payable and accrued expenses - Restructuring (Details) $ in Millions | 12 Months Ended |
Dec. 31, 2023 USD ($) | |
Restructuring costs | |
Accrued restructuring costs | $ 9 |
Selling, general and administrative | |
Restructuring costs | |
Restructuring costs | $ 32.8 |
Debt - Liability for Sale of Fu
Debt - Liability for Sale of Future Royalties (Details) $ in Thousands | 12 Months Ended |
Dec. 31, 2023 USD ($) | |
Debt Disclosure [Abstract] | |
Beginning balance | $ 757,886 |
Less: Non-cash royalty revenue payable to RPI | (93,460) |
Plus: non-cash interest expense recognized | 104,790 |
Cash consideration | 1,000,000 |
Loss on Debt Extinguishment | 44,881 |
Ending balance | $ 1,814,097 |
Effective interest rate of the liability component | 10.80% |
Debt - Narrative (Details)
Debt - Narrative (Details) | 1 Months Ended | 12 Months Ended | 18 Months Ended | ||||||
Aug. 15, 2022 USD ($) | Jan. 01, 2021 USD ($) | Apr. 08, 2022 USD ($) | Sep. 30, 2019 USD ($) D $ / shares | Dec. 31, 2023 USD ($) | Mar. 14, 2022 USD ($) | Dec. 31, 2022 USD ($) | Dec. 31, 2021 USD ($) | Aug. 31, 2015 USD ($) | |
Long-term debt | |||||||||
Long-term debt | $ 284,213,000 | $ 571,722,000 | |||||||
Additional paid-in capital | 2,466,233,000 | 2,305,020,000 | |||||||
1.50% Convertible senior notes due 2026 | |||||||||
Long-term debt | |||||||||
Net deferred tax liability in connection with convertible notes | $ 25,300,000 | ||||||||
Convertible debt | 1.50% Convertible senior notes due 2026 | |||||||||
Long-term debt | |||||||||
Net carrying amount | 284,213,000 | 283,044,000 | |||||||
Debt principal amount | $ 287,500,000 | $ 287,500,000 | $ 287,500,000 | ||||||
Debt instrument additional amount available for repurchase | $ 37,500,000 | ||||||||
Interest rate ( as a percent ) | 1.50% | ||||||||
Net proceeds from issuance of convertible notes | $ 279,300,000 | ||||||||
Trading days, number | D | 20 | ||||||||
Consecutive trading days, period | D | 30 | ||||||||
Stock price trigger | 130% | ||||||||
Business days, period | 5 days | ||||||||
Consecutive trading-day period | 5 days | ||||||||
Maximum product of the closing sale price of shares of the Company's common stock and the applicable conversion rate for such trading day | 98% | ||||||||
Conversion ratio | 19.0404 | ||||||||
Conversion price per share (in dollars per share) | $ / shares | $ 52.52 | ||||||||
Convertible instruments principal and unpaid interest payable upon events of default | 100% | ||||||||
Minimum percentage of principal held by convertible debt instrument holders required to issue notice for declaration of principal and unpaid interest payable upon events of default | 25% | ||||||||
Term of the convertible notes | 7 years | 7 years | 2 years 8 months 12 days | ||||||
Equity component of convertible notes, net | $ 123,000,000 | ||||||||
Debt discount to be amortized | $ 0 | ||||||||
Incremental interest rate per annum | 0.50% | 0.50% | |||||||
Additional interest paid | $ 100,000 | $ 2,100,000 | |||||||
Convertible debt | 1.50% Convertible senior notes due 2026 | Redemption on or after September 20, 2023 | |||||||||
Long-term debt | |||||||||
Trading days, number | D | 19 | ||||||||
Consecutive trading days, period | D | 30 | ||||||||
Stock price trigger | 130% | ||||||||
Redemption price as percent of principal amount | 100% | ||||||||
Sinking fund | $ 0 | ||||||||
Convertible debt | 3.00% Convertible senior notes due 2022 | |||||||||
Long-term debt | |||||||||
Debt principal amount | $ 150,000,000 | ||||||||
Interest rate ( as a percent ) | 3% | ||||||||
Repayment of convertible notes including accrued interest | $ 152,300,000 | ||||||||
Cumulative Effect Adjustment | 1.50% Convertible senior notes due 2026 | |||||||||
Long-term debt | |||||||||
Additional paid-in capital | (123,000,000) | ||||||||
Amortization of debt discount | $ 16,100,000 |
Debt - Senior Secured Term Loan
Debt - Senior Secured Term Loan (Details) - USD ($) $ in Thousands | 12 Months Ended | ||||
Oct. 19, 2023 | Oct. 27, 2022 | Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Debt Facility | |||||
Repayment of senior secured term loan | $ (300,000) | ||||
Prepayment premiums, expenses and other exit fees | 81,933 | ||||
Loss on extinguishment of debt | 137,558 | ||||
Interest expense | |||||
Amortization of debt issuance costs | $ 1,873 | $ 1,901 | $ 1,848 | ||
Effective interest rate | 10.80% | ||||
Blackstone Credit Agreement | |||||
Debt Facility | |||||
Maximum borrowing capacity | $ 950,000 | ||||
Minimum consolidated liquidity covenant | 100,000 | ||||
Increased minimum consolidated liquidity covenant after consummation of acquisitions meeting specified thresholds | $ 200,000 | ||||
Interest expense | |||||
Contractual interest expense | $ 30,198 | 6,069 | |||
Amortization of debt issuance costs | 702 | 290 | |||
Total | $ 30,900 | $ 6,359 | |||
Effective interest rate | 13.10% | 12.20% | |||
Blackstone Credit Agreement | Adjusted Term SOFR | |||||
Debt Facility | |||||
Basis spread on variable rate | 7.25% | ||||
Floor interest rate | 1% | ||||
Blackstone Credit Agreement | Base Rate | |||||
Debt Facility | |||||
Basis spread on variable rate | 6.25% | ||||
Floor interest rate | 2% | ||||
Committed Loan Facility | |||||
Debt Facility | |||||
Maximum borrowing capacity | $ 450,000 | ||||
Fund draw term | 18 months | ||||
Loan term | 7 years | ||||
Senior Secured Term Loan | |||||
Debt Facility | |||||
Debt issuance costs capitalized | $ 11,600 | ||||
Principal | $ 300,000 | 300,000 | $ 300,000 | ||
Less: Debt issuance costs | (11,322) | ||||
Repayment of senior secured term loan | $ (300,000) | (300,000) | |||
Net carrying amount | $ 288,678 | ||||
Payment of accrued interest | 2,100 | ||||
Prepayment premiums, expenses and other exit fees | 82,000 | 82,000 | |||
Legal fees | $ 200 | ||||
Loss on extinguishment of debt | 92,700 | ||||
Debt issuance costs | $ 10,700 | ||||
Committed Delayed Draw Term Loan Facility | |||||
Debt Facility | |||||
Maximum borrowing capacity | 150,000 | ||||
Uncommitted Incremental Facility | |||||
Debt Facility | |||||
Maximum borrowing capacity | $ 500,000 |
Debt - Convertible Notes (Detai
Debt - Convertible Notes (Details) - 1.50% Convertible senior notes due 2026 - Convertible debt - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 | Jan. 01, 2021 | Sep. 30, 2019 |
Long-term debt | ||||
Principal | $ 287,500 | $ 287,500 | $ 287,500 | |
Less: Debt issuance costs | (3,287) | (4,456) | ||
Less: Debt discount, net | $ 0 | |||
Net carrying amount | $ 284,213 | $ 283,044 |
Debt - Convertible Notes Intere
Debt - Convertible Notes Interest Expense (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Long-term debt | |||
Amortization of debt issuance costs | $ 1,873 | $ 1,901 | $ 1,848 |
Effective interest rate of the liability component | 10.80% | ||
Convertible debt | 3.00% Convertible senior notes due 2022 | |||
Long-term debt | |||
Contractual interest expense | 2,800 | ||
Amortization of debt issuance costs | 460 | ||
Total | $ 3,260 | ||
Effective interest rate of the liability component | 3.50% | ||
Convertible debt | 1.50% Convertible senior notes due 2026 | |||
Long-term debt | |||
Contractual interest expense | $ 4,305 | $ 4,313 | |
Amortization of debt issuance costs | 1,171 | 1,150 | |
Total | $ 5,476 | $ 5,463 | |
Effective interest rate of the liability component | 1.90% | 1.90% |
Capital structure - Narrative (
Capital structure - Narrative (Details) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | 12 Months Ended | |||||
Oct. 27, 2022 | Aug. 31, 2019 | Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | Jun. 30, 2021 | May 31, 2021 | |
Common stock | |||||||
Number of shares issued in transaction (in shares) | 0 | 0 | 0 | ||||
Common stock, authorized shares (in shares) | 250,000,000 | 250,000,000 | 250,000,000 | 125,000,000 | |||
Maximum | |||||||
Common stock | |||||||
Aggregate value of remaining shares to be issued and sold | $ 93 | ||||||
Stock Purchase Agreement | |||||||
Common stock | |||||||
Net consideration received on transaction | $ 50 | ||||||
Number of shares issued in transaction (in shares) | 1,095,290 | ||||||
Price per share (in USD per share) | $ 45.65 | ||||||
Number of days within which to register stock sale with the SEC per terms of the agreement | 60 days | ||||||
Period of time from the closing date that the entity agrees to keep the stock sale registration effective | 6 months | ||||||
Period of time from the closing date that the purchaser agrees not to resell or transfer entity's stock without written approval | 90 days | ||||||
Censa | |||||||
Common stock | |||||||
Development milestone obligation payments made | $ 30 | ||||||
Shares issued for milestone payment | 657,462 | ||||||
Development milestone obligation cash payments made | $ 0.4 | ||||||
Sales Agreement | Common stock | Maximum | |||||||
Common stock | |||||||
Net consideration received on transaction | $ 125 |
Net loss per share - Numerator
Net loss per share - Numerator and Denominator (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Numerator | |||
Net loss - Basic | $ (626,604) | $ (559,017) | $ (523,901) |
Net loss - Diluted | $ (626,604) | $ (559,017) | $ (523,901) |
Denominator | |||
Basic | 74,838,392 | 71,728,634 | 70,466,393 |
Diluted | 74,838,392 | 71,728,634 | 70,466,393 |
Net loss per share - Basic | $ (8.37) | $ (7.79) | $ (7.43) |
Net loss per share - Diluted | $ (8.37) | $ (7.79) | $ (7.43) |
Net loss per share - Antidiluti
Net loss per share - Antidilutive (Details) - shares | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Net loss per share | |||
Total shares excluded from calculation (in shares) | 12,466,669 | 14,018,753 | 12,292,413 |
Employee Stock Option | |||
Net loss per share | |||
Total shares excluded from calculation (in shares) | 9,600,399 | 11,502,417 | 10,772,582 |
Restricted stock awards and units | |||
Net loss per share | |||
Total shares excluded from calculation (in shares) | 2,866,270 | 2,516,336 | 1,519,831 |
Stock award plan - Narrative (D
Stock award plan - Narrative (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | ||||||||
Jun. 08, 2022 | Dec. 31, 2022 | Dec. 31, 2020 | Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | Apr. 30, 2022 | Jun. 30, 2021 | May 31, 2021 | Jan. 31, 2020 | |
Stock option plan | ||||||||||
Share-based compensation expense | $ 101,636 | $ 110,333 | $ 103,513 | |||||||
Unrecognized compensation cost | $ 119,600 | |||||||||
Weighted average remaining service period for recognition of unrecognized compensation cost | 2 years 1 month 6 days | |||||||||
Stock option | ||||||||||
Stock option plan | ||||||||||
Granted (in shares) | 1,117,284 | 1,685,435 | 2,487,234 | |||||||
Options forfeited (in shares) | 2,196,820 | 458,737 | 742,458 | |||||||
Expected dividend yield (as a percent) | 0% | 0% | 0% | |||||||
Weighted average grant date fair value (in dollars per share) | $ 21.27 | $ 23.54 | $ 43.05 | |||||||
Stock option | Maximum | ||||||||||
Stock option plan | ||||||||||
Vesting period | 4 years | |||||||||
Employee Stock Purchase Plan | ||||||||||
Stock option plan | ||||||||||
Number of shares authorized (in shares) | 2,000,000 | 1,000,000 | ||||||||
Award requisite service period | 6 months | |||||||||
Share-based compensation expense | $ 2,400 | |||||||||
Employee Stock Purchase Plan | Minimum | ||||||||||
Stock option plan | ||||||||||
Purchase price of common stock, percent | 85% | |||||||||
Employee stock purchase plan, voting percentage limit | 5% | |||||||||
1998 Employee, Director, and Consultant Stock Option Plan, 2009 Equity and Long-term Incentive Plan, and 2013 Stock Incentive Plan | ||||||||||
Stock option plan | ||||||||||
Number of shares available for issuance | 0 | |||||||||
Amended 2013 LTIP | ||||||||||
Stock option plan | ||||||||||
Number of additional shares authorized (in shares) | 8,475,000 | |||||||||
Amended 2013 LTIP | Maximum | ||||||||||
Stock option plan | ||||||||||
Number of shares authorized (in shares) | 16,724,212 | |||||||||
2013 Long Term Incentive Plan | ||||||||||
Stock option plan | ||||||||||
Number of shares available for issuance | 8,680,209 | |||||||||
2020 Inducement Stock Incentive Plan | ||||||||||
Stock option plan | ||||||||||
Number of shares available for issuance | 1,834,322 | |||||||||
Number of additional shares authorized (in shares) | 1,000,000 | |||||||||
2020 Inducement Stock Incentive Plan | Maximum | ||||||||||
Stock option plan | ||||||||||
Number of shares authorized (in shares) | 1,000,000 | |||||||||
2020 Inducement Stock Incentive Plan | Common stock | Maximum | ||||||||||
Stock option plan | ||||||||||
Number of shares authorized (in shares) | 1,300,000 | |||||||||
Number of additional shares authorized (in shares) | 1,700,000 | |||||||||
2020 Inducement Stock Incentive Plan | Stock option | ||||||||||
Stock option plan | ||||||||||
Granted (in shares) | 139,040 | |||||||||
Stockholder's specified ownership percentage | 10% | |||||||||
Expiration period | 10 years | |||||||||
Granted (in dollars per share) | $ 41.25 | |||||||||
Options forfeited (in shares) | 270,583 | |||||||||
2020 Inducement Stock Incentive Plan | Stock option | Minimum | ||||||||||
Stock option plan | ||||||||||
Stock options granted, exercise price as percentage of the fair market value of common stock at grant date | 100% | |||||||||
Stock options granted to stockholder with specified ownership percentage, exercise price as percentage of the fair market value of common stock at grant date | 110% | |||||||||
2020 Inducement Stock Incentive Plan | Restricted stock units | ||||||||||
Stock option plan | ||||||||||
Granted (in shares) | 105,370 | |||||||||
Units forfeited (in shares) | 79,195 |
Stock award plan - Stock Option
Stock award plan - Stock Option Activity (Details) - Stock option - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Number of options | |||
Outstanding at the beginning of the period (in shares) | 11,502,417 | 10,772,582 | 9,663,677 |
Granted (in shares) | 1,117,284 | 1,685,435 | 2,487,234 |
Exercised (in shares) | (822,482) | (496,863) | (635,871) |
Forfeited/Cancelled (in shares) | (2,196,820) | (458,737) | (742,458) |
Outstanding at the end of the period (in shares) | 9,600,399 | 11,502,417 | 10,772,582 |
Vested or Expected to vest at the end of the period (in shares) | 1,796,687 | ||
Exercisable at the end of the period (in shares) | 7,650,948 | ||
Weighted- average exercise price | |||
Outstanding at the beginning of the period (in dollars per share) | $ 43.33 | $ 43.66 | $ 38.72 |
Granted (in dollars per share) | 40.19 | 38.55 | 61.36 |
Exercised (in dollars per share) | 29.25 | 29.45 | 28.01 |
Forfeited/Cancelled (in dollars per share) | 45.85 | 48.75 | 52.04 |
Outstanding at the end of the period (in dollars per share) | 43.59 | $ 43.33 | $ 43.66 |
Vested or Expected to vest at the end of the period (in dollars per share) | 45.85 | ||
Exercisable at the end of the period (in dollars per share) | $ 43.08 | ||
Weighted- average remaining contractual term | |||
Outstanding at the end of the period | 5 years 4 months 20 days | ||
Vested or Expected to vest at the end of the period | 8 years 1 month 28 days | ||
Exercisable at the end of the period | 4 years 8 months 4 days | ||
Aggregate intrinsic value (in thousands) | |||
Outstanding at the end of the period (in dollars) | $ 8,814 | ||
Vested or Expected to vest at the end of the period (in dollars) | 366 | ||
Exercisable at the end of the period (in dollars) | $ 8,387 |
Stock award plan - Assumptions
Stock award plan - Assumptions Used (Details) - Stock option | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Valuation assumptions | |||
Expected term (in years) | 5 years 6 months | 5 years 6 months | 5 years 6 months |
Minimum | |||
Valuation assumptions | |||
Risk-free interest rate (as a percent) | 3.54% | 1.55% | 0.51% |
Expected volatility (as a percent) | 53% | 54% | 74% |
Maximum | |||
Valuation assumptions | |||
Risk-free interest rate (as a percent) | 4.69% | 4.57% | 1.24% |
Expected volatility (as a percent) | 56% | 74% | 89% |
Stock award plan - Restricted S
Stock award plan - Restricted Stock (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Number of Shares | ||||
Share-based compensation expense | $ 101,636 | $ 110,333 | $ 103,513 | |
Restricted stock awards and units | ||||
Number of Shares | ||||
Balance at the beginning of the period (in shares) | 2,516,336 | |||
Granted (in shares) | 2,104,128 | |||
Vested (in shares) | (920,488) | |||
Forfeited (in shares) | (833,706) | |||
Balance at the end of the period (in shares) | 2,866,270 | 2,866,270 | 2,516,336 | |
Weighted Average Grant Date Fair Value | ||||
Balance at the beginning of the period (in dollars per share) | $ 45.67 | |||
Granted (in dollars per share) | 38.75 | |||
Vested (in dollars per share) | 43.74 | |||
Forfeited (in dollars per share) | 42.31 | |||
Balance at the end of the period (in dollars per share) | $ 41.82 | $ 41.82 | $ 45.67 | |
Performance-based restricted stock units (PSUs) | ||||
Number of Shares | ||||
Granted (in shares) | 150,000 | |||
Share-based compensation expense | $ 0 | |||
Performance period | 2 years |
Stock award plan - Share-based
Stock award plan - Share-based compensation expense (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Stock option plan | |||
Share-based compensation expense | $ 101,636 | $ 110,333 | $ 103,513 |
Unrecognized compensation cost | $ 119,600 | ||
Weighted average remaining service period for recognition of unrecognized compensation cost | 2 years 1 month 6 days | ||
Research and development | |||
Stock option plan | |||
Share-based compensation expense | $ 52,941 | 55,869 | 53,632 |
Selling, general and administrative | |||
Stock option plan | |||
Share-based compensation expense | $ 48,695 | $ 54,464 | $ 49,881 |
Other comprehensive income (l_3
Other comprehensive income (loss) and accumulated other comprehensive items (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Other comprehensive income (loss) | |||
Balance at the beginning of the period | $ (347,086) | $ 1,438 | $ 481,982 |
Balance at the end of the period | (818,555) | (347,086) | 1,438 |
Unrealized Gains (Losses) On Marketable Securities, net of tax | |||
Other comprehensive income (loss) | |||
Balance at the beginning of the period | (494) | (602) | 1,900 |
Other comprehensive income (loss) before reclassifications | 1,135 | 4,072 | (3,279) |
Amounts reclassified from other comprehensive items | (315) | (3,964) | 777 |
Other comprehensive income (loss) | 820 | 108 | (2,502) |
Balance at the end of the period | 326 | (494) | (602) |
Foreign Currency Translation | |||
Other comprehensive income (loss) | |||
Balance at the beginning of the period | 5,290 | (23,680) | (62,857) |
Other comprehensive income (loss) before reclassifications | (6,901) | 28,970 | 39,177 |
Other comprehensive income (loss) | (6,901) | 28,970 | 39,177 |
Balance at the end of the period | (1,611) | 5,290 | (23,680) |
AOCI Attributable to Parent | |||
Other comprehensive income (loss) | |||
Balance at the beginning of the period | 4,796 | (24,282) | (60,957) |
Other comprehensive income (loss) before reclassifications | (5,766) | 33,042 | 35,898 |
Amounts reclassified from other comprehensive items | (315) | (3,964) | 777 |
Other comprehensive income (loss) | (6,081) | 29,078 | 36,675 |
Balance at the end of the period | $ (1,285) | $ 4,796 | $ (24,282) |
Revenue recognition - Net Produ
Revenue recognition - Net Product Sales (Details) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 USD ($) country Distributor | Dec. 31, 2022 USD ($) country Distributor | Dec. 31, 2021 USD ($) country Distributor | |
Revenue recognition | |||
Revenue | $ 937,822 | $ 698,801 | $ 538,593 |
United States | |||
Revenue recognition | |||
Revenue | 531,661 | 381,862 | |
Non-US | |||
Revenue recognition | |||
Revenue | 406,161 | 316,939 | |
Net product revenue | |||
Revenue recognition | |||
Contract with Customer, Liability | $ 0 | $ 0 | |
Net product revenue | Geographic concentration risk | |||
Revenue recognition | |||
Number of countries exceeding 10% of net product sales | country | 2 | 2 | 1 |
Percentage of net product sales threshold | 10% | 10% | 10% |
Net product revenue | Customer concentration risk | |||
Revenue recognition | |||
Number of distributors | Distributor | 2 | 2 | 2 |
Percentage of net product sales threshold | 10% | 10% | 10% |
Net product revenue | |||
Revenue recognition | |||
Revenue | $ 661,249 | $ 535,228 | $ 428,904 |
Net product revenue | Non-US | |||
Revenue recognition | |||
Revenue | 406,100 | 316,900 | 241,600 |
Net product revenue | Net product revenue | Geographic concentration risk | United States | |||
Revenue recognition | |||
Revenue | 255,100 | 218,300 | |
Net product revenue | Net product revenue | Geographic concentration risk | Russia | |||
Revenue recognition | |||
Revenue | 86,000 | 59,700 | |
Emflaza | United States | |||
Revenue recognition | |||
Revenue | 255,100 | 218,300 | 187,300 |
Translarna | Non-US | |||
Revenue recognition | |||
Revenue | $ 355,800 | $ 288,600 | $ 236,000 |
Revenue recognition - Collabora
Revenue recognition - Collaboration and Royalty Revenue (Details) $ in Thousands | 1 Months Ended | 12 Months Ended | ||||||
Sep. 30, 2022 USD ($) | Dec. 31, 2021 USD ($) | Dec. 31, 2023 USD ($) | Dec. 31, 2022 USD ($) | Dec. 31, 2021 USD ($) payment | Aug. 31, 2021 USD ($) | Mar. 31, 2021 USD ($) | Nov. 30, 2011 USD ($) | |
Revenue recognition | ||||||||
Remaining potential milestones that can be achieved | $ 150,000 | |||||||
Revenue | 937,822 | $ 698,801 | $ 538,593 | |||||
Collaboration revenue | ||||||||
Revenue recognition | ||||||||
Revenue | 100,030 | 50,052 | 55,046 | |||||
Royalty revenue | ||||||||
Revenue recognition | ||||||||
Revenue | 168,856 | 113,521 | 54,643 | |||||
SMA License Agreement | Collaboration revenue | ||||||||
Revenue recognition | ||||||||
Revenue | 100,000 | 50,100 | 55,000 | |||||
SMA License Agreement | Royalty revenue | ||||||||
Revenue recognition | ||||||||
Revenue | 168,900 | $ 113,500 | $ 54,600 | |||||
Research and Development and Sales Milestones | Collaboration revenue | ||||||||
Revenue recognition | ||||||||
Number of milestone payments. | payment | 3 | |||||||
Research And Development Event Milestones | SMA License Agreement | ||||||||
Revenue recognition | ||||||||
Remaining potential milestones that can be achieved | 0 | |||||||
Milestone payments received | $ 10,000 | $ 20,000 | ||||||
Sales Milestones | SMA License Agreement | ||||||||
Revenue recognition | ||||||||
Remaining potential milestones that can be achieved | 150,000 | |||||||
Net sales milestone threshold achieved | $ 750,000 | $ 500,000 | 1,500,000 | |||||
Sales Milestones | SMA License Agreement | Prepaids and other current assets | ||||||||
Revenue recognition | ||||||||
Milestone payments due | $ 50,000 | $ 25,000 | $ 100,000 | |||||
Maximum | Research And Development Event Milestones | SMA License Agreement | Roche | ||||||||
Revenue recognition | ||||||||
Remaining potential milestones that can be achieved | $ 135,000 | |||||||
Maximum | Sales Milestones | SMA License Agreement | Roche | ||||||||
Revenue recognition | ||||||||
Remaining potential milestones that can be achieved | $ 325,000 |
Revenue recognition - Manufactu
Revenue recognition - Manufacturing Revenue (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Revenue recognition | |||
Revenue | $ 937,822 | $ 698,801 | $ 538,593 |
Manufacturing revenue | |||
Revenue recognition | |||
Revenue | 7,687 | 0 | $ 0 |
Contract with customer, asset | 200 | 0 | |
Contract liability revenues recognized in period | 1,400 | ||
Manufacturing revenue | Deferred revenue | |||
Revenue recognition | |||
Contract with customer, liability | 800 | 1,400 | |
Manufacturing revenue | Prepaids and other current assets | |||
Revenue recognition | |||
Contract with customer, asset | $ 200 | $ 0 |
Revenue recognition - Performan
Revenue recognition - Performance Obligations (Details) - USD ($) $ in Millions | Dec. 31, 2023 | Dec. 31, 2022 |
Remaining Performance Obligation | ||
Remaining performance obligations | $ 1.4 | |
Revenue, Remaining Performance Obligation, Expected Timing of Satisfaction, Start Date [Axis]: 2024-01-01 | ||
Remaining Performance Obligation | ||
Remaining performance obligations | $ 0.8 | |
Revenue, performance obligation, period | 1 year |
Income taxes - Narrative (Detai
Income taxes - Narrative (Details) - USD ($) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2018 | |
Operating loss carryforwards | ||||
Taxable income before consideration of NOL | $ 102,500 | |||
State income tax benefit | 27,226 | $ (4,224) | $ (3,844) | |
Deferred tax valuation allowance | 833,810 | 672,172 | ||
Valuation allowance, deferred tax asset, increase (decrease) | 161,600 | 146,600 | ||
Deferred tax liability on indefinite-lived intangibles acquired | $ 55,905 | $ 102,834 | ||
Federal income tax provision at statutory rate | 21% | 21% | 21% | |
Uncertain tax position | $ 0 | |||
Increase in current taxable income resulting from the elimination of current deduction of qualifying R&D costs under Section 174. | 300,300 | |||
Undistributed earnings from non-US subsidiaries | 345,900 | |||
Undistributed earnings from non-US subsidiaries, tax | 0 | |||
Unrecognized tax benefits | 1,360 | $ 27,217 | ||
Agilis | ||||
Operating loss carryforwards | ||||
Deferred tax liability on indefinite-lived intangibles acquired | $ 122,000 | |||
Federal | ||||
Operating loss carryforwards | ||||
Net operating loss carryforwards | 287,000 | |||
Research and development credit carryforwards | 35,900 | |||
Research and development credit carryforwards expiring during the period | 600 | |||
Orphan Drug Credit carryover | 169,600 | |||
State | ||||
Operating loss carryforwards | ||||
Net operating loss carryforwards | 208,800 | |||
Foreign Tax Authority | ||||
Operating loss carryforwards | ||||
Net operating loss carryforwards | $ 12,100 |
Income taxes - Loss from operat
Income taxes - Loss from operations before tax (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | |||
Domestic | $ (784,744) | $ (591,126) | $ (487,726) |
Foreign | 88,634 | 3,639 | (30,614) |
Loss before income tax benefit (expense) | $ (696,110) | $ (587,487) | $ (518,340) |
Income taxes - Provision for Ta
Income taxes - Provision for Taxes (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Current: | |||
U.S. Federal | $ 0 | $ 0 | $ 0 |
U.S. State and Local | 27,226 | (4,224) | (3,844) |
Foreign | (4,003) | (1,582) | (1,340) |
Deferred: | |||
U.S. Federal | 36,408 | 23,689 | 0 |
U.S. State and Local | 10,521 | 10,587 | (377) |
Foreign | (646) | 0 | 0 |
Total tax benefit (expense) | $ 69,506 | $ 28,470 | $ (5,561) |
Income taxes - Tax Rate Reconci
Income taxes - Tax Rate Reconciliation (Details) | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Reconciliation of the U.S. statutory income tax rate to the entity's effective tax rate | |||
Federal income tax provision at statutory rate | 21% | 21% | 21% |
State income tax provision, net of federal benefit | 0.32% | 3.07% | (0.74%) |
Permanent differences | (1.43%) | (1.83%) | (4.06%) |
Research and development | 4.59% | 5.89% | 4.50% |
Change in valuation allowances | (16.86%) | (23.36%) | (29.03%) |
Change in deferred tax assets | (0.10%) | 12.05% | |
Foreign tax rate differential | 0.05% | (0.17%) | 0.01% |
Tax rate change | (1.26%) | 0.34% | 0.01% |
Release (accrual) of uncertain tax positions | 3.71% | (4.78%) | |
Other | (0.12%) | (0.03%) | |
Effective income tax rate | 10% | 4.84% | (1.07%) |
Income taxes - Deferred Tax Ass
Income taxes - Deferred Tax Assets and Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Deferred tax assets: | ||
Accrued expense | $ 25,400 | $ 2,124 |
Amortization | 137,808 | 52,532 |
Federal tax credits | 205,485 | 174,802 |
State tax credits | 9,817 | 9,787 |
Federal net operating losses | 60,270 | 69,957 |
State net operating losses | 18,680 | 10,316 |
Foreign net operating losses | 4,052 | 4,837 |
Capitalized research and development costs | 149,683 | 110,219 |
Share based compensation and other | 30,757 | 27,054 |
Liability for sale of future royalties | 190,659 | 185,589 |
Noncash interest expense | 9,410 | 30,160 |
Other comprehensive loss | (728) | (719) |
Total gross deferred tax assets | 841,293 | 676,658 |
Less valuation allowance | (833,810) | (672,172) |
Total deferred tax assets, net of valuation allowance | 7,483 | 4,486 |
Deferred tax liabilities: | ||
Depreciation | (7,483) | (4,486) |
Indefinite lived intangible | (55,905) | (102,834) |
Total gross deferred tax liabilities | (63,388) | (107,320) |
Net deferred tax assets (liabilities) | $ (55,905) | $ (102,834) |
Income taxes - Unrecognized Tax
Income taxes - Unrecognized Tax Benefits (Details) $ in Thousands | 12 Months Ended |
Dec. 31, 2023 USD ($) | |
Uncertain income tax benefits | |
Beginning Balance | $ 27,217 |
Reductions based on settlements or expiration of statute of limitations | (24,671) |
Interest received | (1,186) |
Ending Balance | $ 1,360 |
Commitments and contingencies -
Commitments and contingencies - Narrative (Details) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | ||||
Jul. 31, 2022 | Dec. 31, 2023 | Dec. 31, 2022 | May 29, 2020 | Apr. 29, 2020 | Jun. 30, 2016 | |
Other Commitments | ||||||
Milestone payable | $ 2,500 | |||||
Accrued royalties | 74,111 | $ 40,546 | ||||
Wellcome Trust | ||||||
Other Commitments | ||||||
Development milestone payment obligations | 2,500 | $ 800 | ||||
Censa | ||||||
Other Commitments | ||||||
Asset acquisition, development and regulatory milestones | $ 109,000 | |||||
Development milestone obligation payments made | 30,000 | |||||
Development milestone obligation cash payments made | $ 400 | |||||
Shares issued for milestone payment | 657,462 | |||||
Censa | Maximum | ||||||
Other Commitments | ||||||
Asset acquisition, milestone, amount | 217,500 | |||||
Asset acquisition, net sales milestone | $ 160,000 | |||||
Former Censa Securityholders | ||||||
Other Commitments | ||||||
Potential developmental and regulatory milestones | $ 65,000 | |||||
Development milestone obligation payments made | 30,000 | |||||
Development milestone obligation cash payments made | $ 400 | |||||
Shares issued for milestone payment | 657,462 | |||||
Agilis Merger Agreement | Marathon | ||||||
Other Commitments | ||||||
Development milestone payment obligations | $ 50,000 | |||||
SMA License Agreement | SMA Foundation | ||||||
Other Commitments | ||||||
Royalty expense | $ 52,500 | |||||
Royalty payments | 35,300 | |||||
Accrued royalties | 17,200 | |||||
SMA License Agreement | SMA Foundation | Maximum | ||||||
Other Commitments | ||||||
Potential royalty payments due on net product sales | 52,500 | |||||
Agilis | ||||||
Other Commitments | ||||||
Development milestone payments which the entity is obligated to pay | 40,000 | |||||
Potential developmental and regulatory milestones | 31,100 | |||||
Potential sales milestones | 50,000 | $ 150,000 | ||||
Milestone obligation payments made | $ 50,000 | |||||
Development milestone obligation payments made | $ 2,400 | |||||
Agilis | Minimum | ||||||
Other Commitments | ||||||
Product sales (as a percent) | 2% | 2% | ||||
Agilis | Maximum | ||||||
Other Commitments | ||||||
Development milestone payment obligations | $ 60,000 | |||||
Priority review voucher amount | 535,000 | |||||
Potential sales milestones | $ 150,000 | |||||
Product sales (as a percent) | 6% | 6% | ||||
Development milestone payment obligations, net of cancellation and forfeiture | $ 20,000 | |||||
Contingent liability, milestone, potential achievements, priority review voucher amount, net of cancellation and forfeiture | 361,000 | |||||
Agilis | Agilis Merger Agreement | ||||||
Other Commitments | ||||||
Development milestone payment obligations | 40,000 | |||||
Agilis | Rights Exchange Agreement | ||||||
Other Commitments | ||||||
Contingent liability cancellation and forfeiture of potential milestone payments | 174,000 | |||||
Development milestone payment obligations, cancellation and forfeiture | 37,600 | |||||
Contingent liability, cancellation and forfeiture | 211,600 | |||||
BioElectron | Maximum | ||||||
Other Commitments | ||||||
Development milestone payment obligations | $ 200,000 |
Geographic information (Details
Geographic information (Details) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 USD ($) segment | Dec. 31, 2022 USD ($) segment | Dec. 31, 2021 USD ($) | |
Segment Reporting Information [Line Items] | |||
Number of operating segments | segment | 1 | 1 | |
Total assets | $ 1,895,698 | $ 1,705,619 | |
Fixed assets, net | 87,089 | 72,590 | |
Revenue | 937,822 | 698,801 | $ 538,593 |
United States | |||
Segment Reporting Information [Line Items] | |||
Total assets | 1,582,962 | 1,473,770 | |
Fixed assets, net | 86,421 | 71,754 | |
Revenue | 531,661 | 381,862 | |
Non-US | |||
Segment Reporting Information [Line Items] | |||
Total assets | 312,736 | 231,849 | |
Fixed assets, net | 668 | 836 | |
Revenue | $ 406,161 | $ 316,939 |
401(k) plan (Details)
401(k) plan (Details) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Retirement Benefits [Abstract] | |||
Matching contribution (as a percent of total salary) | 100% | 100% | 100% |
Percentage of employee's base salary, matched by employer | 6% | 6% | 6% |
Expense recorded | $ 10.9 | $ 8.4 | $ 6.6 |
Intangible assets and goodwil_2
Intangible assets and goodwill - Definite-lived Intangibles (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Definite-lived intangible assets | |||
Milestone Payable | $ 2,500 | ||
Accrued royalties | 74,111 | $ 40,546 | |
Definite-lived intangibles | |||
Amortization | (222,635) | (116,554) | $ (54,751) |
Finite-Lived Intangible Assets, Net | $ 143,731 | ||
Weighted Average | |||
Definite-lived intangible assets | |||
Remaining amortization period | 6 years 4 months 24 days | ||
Emflaza asset acquisition | |||
Definite-lived intangible assets | |||
Milestone obligation payments recorded | $ 107,200 | ||
Marathon | Emflaza asset acquisition | Accounts payable and accrued expenses | |||
Definite-lived intangible assets | |||
Milestone Payable | 52,700 | ||
Akcea | Waylivra | |||
Definite-lived intangible assets | |||
Royalty payments | 500 | ||
Akcea | Waylivra | Accounts payable and accrued expenses | |||
Definite-lived intangible assets | |||
Accrued royalties | 400 | ||
Akcea | Tegsedi | |||
Definite-lived intangible assets | |||
Royalty payments | 5,800 | ||
Akcea | Tegsedi | Accounts payable and accrued expenses | |||
Definite-lived intangible assets | |||
Accrued royalties | 1,600 | ||
Definite-lived intangible assets | |||
Definite-lived intangibles | |||
Beginning balance - Definite-lived assets, Gross | 526,228 | ||
Additions | 113,536 | ||
Reclass from Indefinite Lived to Definite Lived | 743 | ||
Impairment | 640,507 | ||
Ending balance - Definite-lived assets, Gross | 526,228 | ||
Beginning balance - Accumulated amortization | (273,903) | ||
Amortization | (222,635) | ||
Foreign Currency Translation - Accumulated amortization | (238) | ||
Ending balance - Accumulated amortization | (496,776) | (273,903) | |
Finite-Lived Intangible Assets, Net | 143,731 | ||
Definite-lived intangible assets | Emflaza | |||
Definite-lived intangibles | |||
Beginning balance - Definite-lived assets, Gross | 420,253 | ||
Additions | 107,164 | ||
Impairment | 527,417 | ||
Ending balance - Definite-lived assets, Gross | 420,253 | ||
Beginning balance - Accumulated amortization | (266,023) | ||
Amortization | (212,595) | ||
Ending balance - Accumulated amortization | (478,618) | (266,023) | |
Definite-lived intangible assets | Waylivra | |||
Definite-lived intangibles | |||
Beginning balance - Definite-lived assets, Gross | 9,316 | ||
Additions | 533 | ||
Reclass from Indefinite Lived to Definite Lived | 369 | ||
Impairment | 10,218 | ||
Ending balance - Definite-lived assets, Gross | 9,316 | ||
Beginning balance - Accumulated amortization | (2,751) | ||
Amortization | (1,080) | ||
Foreign Currency Translation - Accumulated amortization | (134) | ||
Ending balance - Accumulated amortization | (3,965) | (2,751) | |
Definite-lived intangible assets | Tegsedi | |||
Definite-lived intangibles | |||
Beginning balance - Definite-lived assets, Gross | 7,109 | ||
Additions | 5,839 | ||
Reclass from Indefinite Lived to Definite Lived | 374 | ||
Impairment | 13,322 | ||
Ending balance - Definite-lived assets, Gross | 7,109 | ||
Beginning balance - Accumulated amortization | (1,709) | ||
Amortization | (1,498) | ||
Foreign Currency Translation - Accumulated amortization | (104) | ||
Ending balance - Accumulated amortization | (3,311) | (1,709) | |
Definite-lived intangible assets | Upstaza | |||
Definite-lived intangibles | |||
Beginning balance - Definite-lived assets, Gross | 89,550 | ||
Impairment | 89,550 | ||
Ending balance - Definite-lived assets, Gross | 89,550 | ||
Beginning balance - Accumulated amortization | (3,420) | ||
Amortization | (7,462) | ||
Ending balance - Accumulated amortization | $ (10,882) | $ (3,420) |
Intangible assets and goodwil_3
Intangible assets and goodwill - Future Amortization (Details) $ in Thousands | Dec. 31, 2023 USD ($) |
Goodwill and Intangible Assets Disclosure [Abstract] | |
2024 | $ 59,312 |
2025 | 10,512 |
2026 | 10,512 |
2027 | 10,512 |
2028 and thereafter | 52,883 |
Total | $ 143,731 |
Intangible assets and goodwil_4
Intangible assets and goodwill - Indefinite-lived Intangibles (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |
Jun. 30, 2023 | Dec. 31, 2023 | Dec. 31, 2022 | |
Indefinite-lived intangibles | |||
Impairment | $ (217,800) | $ (33,384) | |
Intangible assets, net | 379,497 | 705,891 | |
Upstaza | |||
Indefinite-lived intangibles | |||
Impairment | 0 | ||
Indefinite-lived intangible assets | |||
Indefinite-lived intangibles | |||
Beginning Balance | 453,566 | 453,600 | |
Impairment | (217,800) | ||
Ending Balance | 235,766 | 453,566 | |
Indefinite-lived intangible assets | Upstaza | |||
Indefinite-lived intangibles | |||
Beginning Balance | 235,766 | ||
Impairment | 0 | ||
Ending Balance | 235,766 | 235,766 | |
Indefinite-lived intangible assets | PTC-FA and PTC-AS | |||
Indefinite-lived intangibles | |||
Impairment | $ (217,800) | (217,800) | |
Indefinite-lived intangible assets | PTC-FA | |||
Indefinite-lived intangibles | |||
Beginning Balance | 112,500 | ||
Impairment | (112,500) | ||
Ending Balance | 112,500 | ||
Indefinite-lived intangible assets | PTC-AS | |||
Indefinite-lived intangibles | |||
Beginning Balance | 105,300 | ||
Impairment | $ (105,300) | ||
Ending Balance | $ 105,300 |
Intangible assets and goodwil_5
Intangible assets and goodwill - Goodwill (Details) - USD ($) $ in Thousands | 12 Months Ended | 64 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2023 | Dec. 31, 2022 | Aug. 23, 2018 | |
Goodwill | ||||
Goodwill | $ 82,341 | $ 82,341 | $ 82,341 | |
Impairment of goodwill | 0 | |||
Agilis | ||||
Goodwill | ||||
Goodwill | 82,300 | 82,300 | $ 82,300 | $ 82,300 |
Changes in goodwill | $ 0 | |||
Impairment of goodwill | $ 0 |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Pay vs Performance Disclosure | |||
Net Income (Loss) | $ (626,604) | $ (559,017) | $ (523,901) |
Insider Trading Arrangements
Insider Trading Arrangements | 3 Months Ended |
Dec. 31, 2023 shares | |
Trading Arrangements, by Individual | |
Material Terms of Trading Arrangement | Director and Officer Trading Arrangements A portion of the compensation of our directors and officers (as defined in Rule 16a-1(f) under the Securities Exchange Act of 1934, as amended, or the Exchange Act) is in the form of equity awards and, from time to time, directors and officers engage in open-market transactions with respect to the securities acquired pursuant to such equity awards or other Company securities, including to satisfy tax withholding obligations when equity awards vest or are exercised, and for diversification or other personal reasons. Transactions in Company securities by directors and officers are required to be made in accordance with our insider trading policy, which requires that the transactions be in accordance with applicable U.S. federal securities laws that prohibit trading while in possession of material nonpublic information. Rule 10b5-1 under the Exchange Act provides an affirmative defense that enables directors and officers to prearrange transactions in Company securities in a manner that avoids concerns about initiating transactions while in possession of material nonpublic information. The following table describes, for the quarterly period covered by this report, each trading arrangement for the sale or purchase of Company securities adopted or terminated by our directors and officers that is either (1) a contract, instruction or written plan intended to satisfy the affirmative defense conditions of Rule 10b5-1(c), or a “Rule 10b5-1 trading arrangement”, or (2) a “non-Rule 10b5-1 trading arrangement” (as defined in Item 408(c) of Regulation S-K): Name Action Taken Type of Trading Nature of Trading Duration of Trading Aggregate Number Mark Boulding (Chief Legal Officer) Adoption (October 31, 2023) Rule 10b5-1 trading arrangement Sale Until February 14, 2025 or such earlier date upon which all transactions are completed. Up to 215,899 shares. Matthew Klein Adoption Rule 10b5-1 trading arrangement for sell-to-cover transactions for RSUs granted on December28, 2023 Sale Until final settlement of RSUs on or around December 29, 2026 Indeterminable (1) (1) The number of shares subject to this RSU grant that will be sold to satisfy applicable tax withholding obligations upon vesting is unknown as the number will vary based on the extent to which vesting conditions are satisfied and the market price of the Company’s common stock at the time of settlement. This trading arrangement provides for the automatic sale of shares that would otherwise be issuable on each settlement date of the RSU in an amount sufficient to satisfy the applicable withholding obligation, with the proceeds of the sale delivered to the Company in satisfaction of the applicable withholding obligation. |
Non-Rule 10b5-1 Arrangement Adopted | false |
Common Stock Trading Arrangement | Mark Boulding | |
Trading Arrangements, by Individual | |
Name | Mark Boulding |
Title | Chief Legal Officer |
Rule 10b5-1 Arrangement Adopted | true |
Adoption Date | October 31, 2023 |
Termination Date | February 14, 2025 |
Aggregate Available | 215,899 |
Restricted Stock Unit Trading Arrangement | Matthew Klein | |
Trading Arrangements, by Individual | |
Name | Matthew Klein |
Title | Chief Executive Officer |
Rule 10b5-1 Arrangement Adopted | true |
Adoption Date | December 28, 2023 |
Termination Date | December 29, 2026 |