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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
 
FORM 10-Q
(Mark One)
 
☒       QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
For the quarterly period ended September 30, 2024
OR
☐       TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
For the transition period from             to
Commission file number 001-33497
Amicus Therapeutics, Inc.
(Exact Name of Registrant as Specified in Its Charter)
Delaware 71-0869350
(State or Other Jurisdiction of (I.R.S. Employer
Incorporation or Organization) Identification Number)
47 Hulfish Street, Princeton, NJ
08542
(Address of Principal Executive Offices)(Zip Code)
(609)
662-2000
(Registrant's Telephone Number, Including Area Code)
Securities registered pursuant to Section 12(b) of the Act:
Title of each classTrading Symbol(s)Name of each exchange on which registered
Common Stock, par value $0.01 per share
FOLDNASDAQ Global Market
Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days.  Yes ☒ No ☐
Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files).  Yes ☒ No ☐
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of "large accelerated filer," "accelerated filer," "smaller reporting company," and "emerging growth company" in Rule 12b-2 of the Exchange Act.
Large accelerated filer ☒Accelerated filer☐
Non-accelerated filer ☐Smaller reporting company☐
Emerging growth company☐
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No ☒
The number of shares outstanding of the registrant's common stock, $0.01 par value per share, as of October 25, 2024 was 298,805,773 shares.


AMICUS THERAPEUTICS, INC.
 
Form 10-Q for the Quarterly Period Ended September 30, 2024
 
 Page
 
Item 1.
Item 2.
Item 3.
Item 4.
  
 Item 1.
    
 Item 1A.
    
 Item 2.
    
 Item 3.
    
 Item 4.
    
 Item 5.
    
 Item 6.
  
  

i

SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
This Quarterly Report on Form 10-Q contains forward-looking statements that involve risks, uncertainties, and assumptions. Forward-looking statements are all statements, other than statements of historical facts, that discuss our current expectation and projections relating to our strategy, future operations, future financial position, future revenues, projected costs, prospects, plans, and objectives of management. These statements may be preceded by, followed by or include the words "aim," "anticipate," "believe," "can," "could," "estimate," "expect," "forecast," "intend," "likely," "may," "might," "outlook," "plan," "potential," "predict," "project," "seek," "should," "will," "would," the negatives or plurals thereof, and other words and terms of similar meaning, although not all forward-looking statements contain these identifying words.
We have based these forward-looking statements on our current expectations and projections about future events. Although we believe that our assumptions made in connection with the forward-looking statements are reasonable, we cannot assure you that the assumptions and expectations will prove to be correct. You should understand that the following important factors could affect our future results and could cause those results or other outcomes to differ materially from those expressed or implied in our forward-looking statements:
•the scope, progress, results and costs of clinical trials for our drug candidates;
•the cost of manufacturing drug supply for our commercial, clinical and preclinical studies, including the cost of manufacturing Pombiliti® (also referred to as "ATB200" or "cipaglucosidase alfa");
•the future results of preclinical research and subsequent clinical trials for pipeline candidates we may identify from time to time, including our ability to obtain regulatory approvals and commercialize such therapies;
•the costs, timing, and outcome of regulatory review of our product candidates;
•any changes in regulatory standards relating to the review of our product candidates;
•any changes in laws, rules or regulations affecting our ability to manufacture, transport, test, develop, or commercialize our products, including Galafold®, Pombiliti® + Opfolda®, or our product candidates;
•the costs of commercialization activities, including product marketing, sales, and distribution;
•the emergence of competing technologies and other adverse market developments;
•the estimates regarding the potential market opportunity for our products and product candidates;
•our ability to successfully commercialize Galafold® (also referred to as "migalastat HCl");
•our ability to successfully commercialize Pombiliti® + Opfolda® (together, also referred to as "AT-GAA") in the E.U., U.K., U.S., Switzerland and elsewhere, if regulatory applications are approved;
•our ability to manufacture or supply sufficient clinical or commercial products, including Galafold® and Pombiliti® + Opfolda®;
•our ability to obtain reimbursement for Galafold® and Pombiliti® + Opfolda®;
•our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of Galafold® and Pombiliti® + Opfolda®;
•our ability to obtain market acceptance of Galafold® and Pombiliti® + Opfolda®, or any other product developed or acquired that has received regulatory approval;
•the costs of preparing, filing, and prosecuting patent applications and maintaining, enforcing, and defending intellectual property-related claims, including Hatch-Waxman litigation;
•the impact of litigation that has been or may be brought against us or of litigation that we are pursuing or may pursue against others, including Hatch-Waxman litigation;
•the extent to which we acquire or invest in businesses, products, and technologies;
•our ability to successfully integrate acquired products and technologies into our business, or successfully divest or license existing products and technologies from our business, including the possibility that the expected benefits of the transactions will not be fully realized by us or may take longer to realize than expected;
•our ability to establish licensing agreements, collaborations, partnerships or other similar arrangements and to obtain milestone, royalty, or other economic benefits from any such collaborators;
1

•the costs associated with, and our ability to comply with, emerging environmental, social and governance standards, including climate reporting requirements at the local, state and national levels;
•our ability to successfully protect our information technology systems and maintain our global operations and supply chain without interruption;
•our ability to accurately forecast revenue, operating expenditures, or other metrics impacting profitability;
•fluctuations in foreign currency exchange rates; and
•changes in accounting standards.
In light of these risks and uncertainties, we may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions, and expectations disclosed in the forward-looking statements we make. We have included important factors in the cautionary statements included in Part I Item 1A — Risk Factors of the Annual Report on Form 10-K for the fiscal year ended December 31, 2023, that we believe could cause actual results or events to differ materially from the forward-looking statements that we make. Those factors and the other risk factors described herein are not necessarily all of the important factors that could cause actual results or developments to differ materially from those expressed in any of our forward-looking statements. Other unknown or unpredictable factors also could harm our results. Our forward-looking statements do not reflect the potential impact of any future collaborations, alliances, business combinations, partnerships, strategic out-licensing of certain assets, the acquisition of preclinical-stage, clinical-stage, marketed products or platform technologies or other investments we may make. Consequently, there can be no assurance that actual results or developments anticipated by us will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, us. Given these uncertainties, investors are cautioned not to place undue reliance on such forward-looking statements.
You should read this Quarterly Report on Form 10-Q in conjunction with our Annual Report on Form 10-K for the fiscal year ended December 31, 2023 (including the documents incorporated by reference therein) completely and with the understanding that our actual future results may be materially different from what we expect. These forward-looking statements speak only as of the date of this report. We undertake no obligation, and specifically decline any obligation, to publicly update or revise any forward-looking statements, even if experience or future developments make it clear that projected results expressed or implied in such statements will not be realized, except as may be required by law. 
2

PART I.    FINANCIAL INFORMATION
ITEM 1.    CONSOLIDATED FINANCIAL STATEMENTS AND NOTES (UNAUDITED)
Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(Unaudited)
(in thousands, except share and per share amounts)
September 30, 2024December 31, 2023
Assets
Current assets:
Cash and cash equivalents$233,647 $246,994 
Investments in marketable securities16,110 39,206 
Accounts receivable98,073 87,632 
Inventories115,338 59,696 
Prepaid expenses and other current assets35,306 49,533 
Total current assets498,474 483,061 
Operating lease right-of-use assets, net23,144 26,312 
Property and equipment, less accumulated depreciation of $29,324 and $25,429 at September 30, 2024 and December 31, 2023, respectively
30,438 31,667 
Intangible assets, less accumulated amortization of $4,974 and $2,510 at September 30, 2024 and December 31, 2023, respectively
18,026 20,490 
Goodwill197,797 197,797 
Other non-current assets18,678 18,553 
Total Assets$786,557 $777,880 
Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable$13,481 $15,120 
Accrued expenses and other current liabilities136,116 144,245 
Operating lease liabilities8,541 8,324 
Total current liabilities158,138 167,689 
Long-term debt389,494 387,858 
Operating lease liabilities46,623 48,877 
Other non-current liabilities13,477 13,282 
Total liabilities607,732 617,706 
Commitments and contingencies
Stockholders’ equity:
Common stock, $0.01 par value, 500,000,000 shares authorized, 298,691,094 and 293,594,209 shares issued and outstanding at September 30, 2024 and December 31, 2023, respectively
2,942 2,918 
Additional paid-in capital2,905,760 2,836,018 
Accumulated other comprehensive income (loss):
Foreign currency translation adjustment25,159 5,429 
Unrealized loss on available-for-sale securities(188)(188)
Warrants71 71 
Accumulated deficit(2,754,919)(2,684,074)
Total stockholders’ equity178,825 160,174 
Total Liabilities and Stockholders’ Equity$786,557 $777,880 

See accompanying Notes to Consolidated Financial Statements
3

Amicus Therapeutics, Inc.
Consolidated Statements of Operations
(Unaudited)
(in thousands, except share and per share amounts)
Three Months Ended September 30,Nine Months Ended September 30,
2024202320242023
Net product sales$141,517 $103,501 $378,589 $284,274 
Cost of goods sold13,279 9,946 38,107 26,002 
Gross profit128,238 93,555 340,482 258,272 
Operating expenses:
Research and development26,160 40,704 79,172 117,352 
Selling, general, and administrative75,106 65,651 236,711 205,031 
Changes in fair value of contingent consideration payable— 1,995 — 2,583 
Restructuring charges3,143 — 9,188 — 
Loss on impairment of assets— — — 1,134 
Depreciation and amortization2,170 2,228 6,506 5,691 
Total operating expenses106,579 110,578 331,577 331,791 
Income (loss) from operations21,659 (17,023)8,905 (73,519)
Other expense:
Interest income1,081 1,471 3,991 5,407 
Interest expense(12,692)(12,986)(37,640)(37,322)
Other (expense) income (3,263)3,833 (11,946)(13,007)
Income (loss) before income tax 6,785 (24,705)(36,690)(118,441)
Income tax (expense) benefit(13,514)3,128 (34,155)700 
Net loss attributable to common stockholders$(6,729)$(21,577)$(70,845)$(117,741)
Net loss attributable to common stockholders per common share — basic and diluted$(0.02)$(0.07)$(0.23)$(0.40)
Weighted-average common shares outstanding — basic and diluted304,690,596295,759,435303,792,479293,314,167
                                                                                    
See accompanying Notes to Consolidated Financial Statements
4

Amicus Therapeutics, Inc.
Consolidated Statements of Comprehensive Income (Loss)
(Unaudited)
(in thousands)
 Three Months Ended September 30,Nine Months Ended September 30,
 2024202320242023
Net loss$(6,729)$(21,577)$(70,845)$(117,741)
Other comprehensive income (loss), net of tax:
Foreign currency translation adjustment16,168 (10,910)19,730 5,416 
Unrealized gain (loss) on available-for-sale securities9 (18)— (79)
Other comprehensive income (loss)16,177 (10,928)19,730 5,337 
Comprehensive income (loss)$9,448 $(32,505)$(51,115)$(112,404)

See accompanying Notes to Consolidated Financial Statements
5

Amicus Therapeutics, Inc.
Consolidated Statements of Changes in Stockholders' Equity
(Unaudited)
(in thousands, except share amounts)
Three Months Ended September 30, 2024
Common StockAdditional
Paid-In
Capital		WarrantsOther
Comprehensive
Income		Accumulated
Deficit		Total
Stockholders'
Equity
SharesAmount
Balance at June 30, 2024296,428,877 $2,923 $2,868,925 $71 $8,794 $(2,748,190)$132,523 
Stock options exercised, net215,653 2 1,803 — — — 1,805 
Vesting of restricted stock units, net of taxes354,273 — (2,792)— — — (2,792)
Stock-based compensation— — 18,688 — — — 18,688 
Issuance of shares in connection with at-the-market offering, net of issuance costs1,692,291 17 19,136 — — — 19,153 
Unrealized gain on available-for-sale securities— — — — 9 — 9 
Foreign currency translation adjustment— — — — 16,168 — 16,168 
Net loss— — — — — (6,729)(6,729)
Balance at September 30, 2024298,691,094 $2,942 $2,905,760 $71 $24,971 $(2,754,919)$178,825 

Nine Months Ended September 30, 2024
Common StockAdditional
Paid-In
Capital		WarrantsOther
Comprehensive
Income		Accumulated
Deficit		Total
Stockholders'
Equity
SharesAmount
Balance at December 31, 2023293,594,209 $2,918 $2,836,018 $71 $5,241 $(2,684,074)$160,174 
Stock options exercised, net796,202 7 6,115 — — — 6,122 
Vesting of restricted stock units, net of taxes2,608,392 — (21,197)— — — (21,197)
Stock-based compensation— — 65,688 — — — 65,688 
Issuance of shares in connection with at-the-market offering, net of issuance costs1,692,291 17 19,136 — — — 19,153 
Foreign currency translation adjustment— — — — 19,730 — 19,730 
Net loss— — — — — (70,845)(70,845)
Balance at September 30, 2024298,691,094 $2,942 $2,905,760 $71 $24,971 $(2,754,919)$178,825 
6

Three Months Ended September 30, 2023
Common StockAdditional
Paid-In
Capital		WarrantsOther
Comprehensive
Loss		Accumulated
Deficit		Total
Stockholders'
Equity
SharesAmount
Balance at June 30, 2023286,992,923 $2,856 $2,733,148 $71 $4,160 $(2,628,654)$111,581 
Stock options exercised, net372,467 4 3,438 — — — 3,442 
Vesting of restricted stock units, net of taxes299,297 — (2,347)— — — (2,347)
Stock-based compensation— — 16,511 — — — 16,511 
Issuance of shares in connection with at-the-market offering, net of issuance costs3,002,354 30 36,525 — — — 36,555 
Unrealized gain on available-for-sale securities— — — — (18)— (18)
Foreign currency translation adjustment— — — — (10,910)— (10,910)
Net loss— — — — — (21,577)(21,577)
Balance at September 30, 2023290,667,041 $2,890 $2,787,275 $71 $(6,768)$(2,650,231)$133,237 

Nine Months Ended September 30, 2023
Common StockAdditional
Paid-In
Capital		WarrantsOther
Comprehensive
Income (Loss)		Accumulated
Deficit		Total
Stockholders'
Equity
SharesAmount
Balance at December 31, 2022281,108,273 $2,815 $2,664,744 $83 $(12,105)$(2,532,490)$123,047 
Stock options exercised, net1,025,684 11 7,836 — — — 7,847 
Vesting of restricted stock units, net of taxes2,068,048 (16,355)— — — (16,355)
Stock-based compensation— — 67,982 — — — 67,982 
Warrants exercised1,220,100 12 12 (12)— — 12 
Issuance of shares in connection with at-the-market offering, net of issuance costs5,244,936 52 63,056 — — — 63,108 
Unrealized loss on available-for-sale securities— — — — (79)— (79)
Foreign currency translation adjustment— — — — 5,416 — 5,416 
Net loss— — — — — (117,741)(117,741)
Balance at September 30, 2023290,667,041 $2,890 $2,787,275 $71 $(6,768)$(2,650,231)$133,237 


See accompanying Notes to Consolidated Financial Statements
7

Amicus Therapeutics, Inc.
Consolidated Statements of Cash Flows
(Unaudited)
(in thousands)
Nine Months Ended September 30,
20242023
Operating activities
Net loss$(70,845)$(117,741)
Adjustments to reconcile net loss to net cash used in operating activities:
Amortization of debt discount and deferred financing1,665 2,080 
Depreciation and amortization6,506 5,691 
Stock-based compensation65,688 67,982 
Non-cash changes in the fair value of contingent consideration payable— 2,583 
Foreign currency remeasurement loss15,167 18,121 
Deferred taxes— (4,939)
Other7,048 2,360 
Changes in operating assets and liabilities:
Accounts receivable(9,090)(8,614)
Inventories(61,632)(42,233)
Prepaid expenses and other current assets28,216 (26,010)
Accounts payable, accrued expenses, and other current liabilities(11,483)41,101 
Other non-current assets and liabilities(1,215)(4,993)
Payment of contingent consideration— (7,937)
Net cash used in operating activities$(29,975)$(72,549)
Investing activities
Sale and redemption of marketable securities101,875 180,828 
Purchases of marketable securities(78,778)(53,098)
Capital expenditures(3,265)(5,709)
Net cash provided by investing activities$19,832 $122,021 
Financing activities
Payment of finance leases(54)(82)
Withholding taxes paid on vested restricted stock units(21,197)(16,355)
Proceeds from stock options exercised, net6,122 7,847 
Proceeds from warrants exercised, net— 12 
Proceeds from the issuance of shares in connection with at-the-market offering, net of issuance costs19,153 63,108 
Payment of contingent consideration— (1,063)
Net cash provided by financing activities$4,024 $53,467 
Effect of exchange rate changes on cash, cash equivalents, and restricted cash$(7,138)$10,218 
Net (decrease) increase in cash, cash equivalents, and restricted cash at the end of the period(13,257)113,157 
Cash, cash equivalents, and restricted cash at the beginning of period250,077 153,115 
Cash, cash equivalents, and restricted cash at the end of period$236,820 $266,272 
Supplemental disclosures of cash flow information
Cash paid during the period for interest $47,587 $35,448 
Cash paid for taxes$2,729 $6,473 
Tenant improvements paid through lease incentives$217 $— 
Capital expenditures unpaid at the end of period$168 $877 

See accompanying Notes to Consolidated Financial Statements
8

Amicus Therapeutics, Inc.
Notes to the Consolidated Financial Statements
(Unaudited)
1. Description of Business
Amicus Therapeutics, Inc. (the "Company") is a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases. The Company seeks to deliver the highest quality therapies that have the potential to obsolete current treatments, provide significant benefits to patients, and be first- or best-in-class. The Company's two marketed therapies are Galafold®, the first oral monotherapy for people living with Fabry disease who have amenable genetic variants, and Pombiliti® + Opfolda®, a novel two-component treatment designed to improve uptake of active enzyme into key disease relevant tissues for adults living with late-onset Pompe disease.
Galafold® (also referred to as "migalastat"), is approved in over 40 countries around the world, including the United States ("U.S."), European Union ("E.U."), United Kingdom ("U.K."), and Japan. Additionally, Galafold® has been granted orphan drug status in the U.S., E.U., U.K., Japan and several other countries.
Pombiliti® + Opfolda® (also referred to as "cipaglucosidase alfa-atga/miglustat"), is approved in the U.S., the E.U., the U.K., and Switzerland. Multiple regulatory submissions and reimbursement processes with global health authorities are currently underway. Additionally, Pombiliti® + Opfolda® has been granted orphan drug designation or status in the U.S., U.K., Switzerland, and Japan and data exclusivity in the E.U.
The Company had an accumulated deficit of $2.8 billion as of September 30, 2024 and anticipates incurring losses through the fiscal year ending December 31, 2024. The Company has historically funded its operations through stock offerings, product revenues, debt issuances, collaborations, and other financing arrangements.
Based on its current operating model, which includes expected revenues, the Company believes the current cash position is sufficient to fund the Company's operations and ongoing research programs for at least the next 12 months. Potential business development opportunities, pipeline expansion, and investment in manufacturing capabilities could impact the Company's long-term capital requirements.
2. Summary of Significant Accounting Policies
Basis of Presentation
The accompanying unaudited Consolidated Financial Statements have been prepared in accordance with the U.S. generally accepted accounting principles (“U.S. GAAP”) for interim financial information and with the instructions to Form 10-Q and Article 10-01 of Regulation S-X. Accordingly, certain financial information that is normally included in annual financial statements prepared in accordance with U.S. GAAP, but that is not required for interim reporting purposes, has been omitted. In the opinion of management, the accompanying unaudited Consolidated Financial Statements reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company's interim financial information. Management has determined that the Company operates in one segment focused on the discovery, development, and commercialization of advanced therapies to treat a range of devastating rare and orphan diseases.
The accompanying unaudited Consolidated Financial Statements and related notes should be read in conjunction with the Company's financial statements and related notes as contained in the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2023. For a complete description of the Company's accounting policies, please refer to the Annual Report on Form 10-K for the fiscal year ended December 31, 2023.
Consolidation
The Consolidated Financial Statements include the accounts of the Company and its subsidiaries. Intercompany accounts and transactions are eliminated in consolidation.
Foreign Currency Transactions
The functional currency for most of the Company's foreign subsidiaries is their local currency. For non-U.S. subsidiaries that transact in a functional currency other than the U.S. dollar, assets and liabilities are translated at current rates of exchange at the balance sheet date. Income and expense items are translated at the weighted average foreign exchange rates for the
9

period. Adjustments resulting from the translation of the financial statements of the Company's foreign operations into U.S. dollars are excluded from the determination of net income and are recorded in accumulated other comprehensive income, a separate component of stockholders' equity. Transactions which are not in the functional currency of the entity are remeasured into the functional currency with gains or losses resulting from the remeasurement recorded in other expense.
Use of Estimates
The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates.
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash
The Company considers all highly liquid investments purchased with a maturity of three months or less at the date of acquisition to be cash equivalents. Marketable securities consist of fixed income investments with a maturity of greater than three months and other highly liquid investments that can be readily purchased or sold using established markets. These investments are classified as available-for-sale and are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are reported within other comprehensive income (loss) in the Company's Consolidated Statements of Comprehensive Income (Loss). Fair value is based on available market information including quoted market prices, broker or dealer quotations, or other observable inputs.
Restricted cash consists primarily of funds held to satisfy the requirements of certain agreements that are restricted in their use and is included as a component of other non-current assets on the Company's Consolidated Balance Sheets.
Concentration of Credit Risk
The Company's financial instruments that are exposed to concentration of credit risk consist primarily of cash, cash equivalents, 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 invests its marketable securities in high-quality commercial financial instruments. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on its cash, cash equivalents, or marketable securities.
The Company's accounts receivable at September 30, 2024 have primarily arisen from Galafold® sales in Europe, the U.S., and Japan. The Company periodically assesses the financial strength of its customers to establish allowances for anticipated losses, if any. For accounts receivable that have arisen from named patient sales, the payment terms are predetermined, and the Company evaluates the creditworthiness of each customer on a regular basis. As of September 30, 2024, the Company's allowance for doubtful accounts was $0.5 million.
Revenue Recognition
The Company has recorded revenue on sales where its products are available either on a commercial basis or through a reimbursed early access program. Product orders are generally received from distributors and pharmacies.
The Company recognizes revenue when its performance obligation to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of the products. The transaction price is determined based on fixed consideration in the Company's customer contracts and is recorded net of estimates for variable consideration, which primarily consist of third-party discounts and rebates. The identified variable consideration is recorded as a reduction of revenue at the time revenue from the sale is recognized. The Company recognizes revenue to the extent that it is probable that a significant revenue reversal will not occur in a future period. These estimates may differ from actual consideration received. The Company evaluates these estimates each reporting period to reflect known changes.
10

The following table summarizes the Company's net product sales disaggregated by product:
Three Months Ended September 30,Nine Months Ended September 30,
(in thousands)2024202320242023
Galafold®
$120,381 $100,733 $330,557 $281,177 
Pombiliti® + Opfolda®
21,136 2,768 48,032 3,097 
Total net product sales$141,517 $103,501 $378,589 $284,274 
The following table summarizes the Company's net product sales disaggregated by geographic area:
Three Months Ended September 30,Nine Months Ended September 30,
(in thousands)2024202320242023
U.S.$56,192 $37,801 $143,705 $103,760 
Ex-U.S.85,325 65,700 234,884 180,514 
Total net product sales$141,517 $103,501 $378,589 $284,274 
Inventories and Cost of Goods Sold
Inventories are stated at the lower of cost and net realizable value, determined by the first-in, first-out method. Inventories are reviewed periodically to identify slow-moving or obsolete inventory based on projected sales activity as well as product shelf-life. In evaluating the recoverability of inventories produced, the probability that revenue will be obtained from the future sale of the related inventory is considered and inventory value is written down for inventory quantities in excess of expected requirements. Expired inventory is disposed of and the related costs are recognized as cost of goods sold in the Company's Consolidated Statements of Operations.
Cost of goods sold includes the cost of inventory sold, manufacturing and supply chain costs, product shipping and handling costs, provisions for excess and obsolete inventory, as well as royalties payable. A portion of Pombiliti® + Opfolda® inventory was expensed as research and development costs prior to regulatory approval and as such, the cost of goods sold and related gross margins are not necessarily indicative of future costs of goods sold and gross margin.
Recent Accounting Developments - Guidance Adopted in 2024
In November 2023, the Financial Accounting Standards Board ("FASB") issued the Accounting Standards Update ("ASU") No. 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures. The amendments expand reportable segment disclosure requirements, primarily through enhanced disclosures about significant segment expenses. The amendments require, among other things, disclosure of the title and position of the chief operating decision maker and require that public entities with a single reportable segment provide all disclosures required by this update and existing segment disclosures in Topic 280. Annual disclosures are required for fiscal years beginning after December 15, 2023 and interim disclosures are required for periods within fiscal years beginning after December 15, 2024. Retrospective application is required unless it is impracticable, and early adoption is permitted. The Company adopted this guidance on January 1, 2024. This ASU applies to disclosure requirements only, and the Company will provide required annual disclosures as part of the 2024 Annual Report on Form 10-K and required interim disclosures as part of 2025 Quarterly Reports on Form 10-Q.
Recent Accounting Developments - Guidance Not Yet Adopted
11

In December 2023, the FASB issued ASU No. 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures. The ASU requires disclosure of disaggregated income taxes paid, prescribes standard categories for the components of the effective tax rate reconciliation, and modifies other income tax-related disclosures. ASU 2023-09 is effective for fiscal years beginning after December 15, 2024, must be applied prospectively with an option to apply retrospectively, and early adoption is permitted. The adoption of this guidance is not expected to have a significant impact on the Company's consolidated financial statements.
In November 2024, the FASB issued ASU 2024-03, "Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses." The standard is intended to require more detailed disclosures about specified categories of expenses (including employee compensation, depreciation, and amortization) included in certain expense captions presented on the face of the income statement. This ASU is effective for fiscal years beginning after December 15, 2026, and for interim periods within fiscal years beginning after December 15, 2027. Early adoption is permitted. The amendments may be applied either prospectively to financial statements issued for reporting periods after the effective date of this ASU or retrospectively to all prior periods presented in the financial statements. The Company is currently assessing the impact this standard will have on its consolidated financial statements.
3. Cash, Cash Equivalents, Marketable Securities, and Restricted Cash
As of September 30, 2024, the Company held $233.6 million in cash and cash equivalents and $16.1 million of marketable securities which are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are generally reported within other comprehensive income (loss) in the Consolidated Statements of Comprehensive Income (Loss). If a decline in the fair value of a marketable security below the Company's cost basis is determined to be other-than-temporary or if an available-for-sale debt security’s fair value is determined to be less than the amortized cost and the Company intends or is more than likely to sell the security before recovery and it is not considered a credit loss, such security is written down to its estimated fair value as a new cost basis and the amount of the write-down is included in the Consolidated Statements of Operations as an impairment charge. If the unrealized loss of an available-for-sale debt security is determined to be a result of credit loss, the Company would recognize an allowance and the corresponding credit loss would be included in the Consolidated Statements of Operations.
The Company regularly invests excess operating cash in deposits with major financial institutions, money market funds, notes issued by the U.S. government, as well as fixed income investments and U.S. bond funds, both of which can be readily purchased and sold using established markets. The Company believes that the market risk arising from its holdings of these financial instruments is mitigated as, in accordance with Company policy, such securities are of high credit rating. Investments that have original maturities greater than three months but less than one year are classified as current.
12

Cash, cash equivalents and marketable securities are classified as current unless mentioned otherwise below and consisted of the following:
 As of September 30, 2024
(in thousands)CostGross
Unrealized
Gain		Gross
Unrealized
Loss		Fair
Value
Cash and cash equivalents$233,647 $— $— $233,647 
Commercial paper5,991 — — 5,991 
Treasury bill9,958 10 — 9,968 
U.S. government agency bonds— — — — 
Money market100 — — 100 
Certificates of deposit51 — — 51 
$249,747 $10 $— $249,757 
Included in cash and cash equivalents$233,647 $— $— $233,647 
Included in marketable securities16,100 10 — 16,110 
Total cash, cash equivalents, and marketable securities$249,747 $10 $— $249,757 

 As of December 31, 2023
(in thousands)CostGross
Unrealized
Gain		Gross
Unrealized
Loss		Fair
Value
Cash and cash equivalents$246,994 $— $— $246,994 
Commercial paper14,651 12 — 14,663 
Treasury bill12,944 2 — 12,946 
U.S. government agency bonds11,450 — (4)11,446 
Money market100 — — 100 
Certificate of deposit51 — — 51 
$286,190 $14 $(4)$286,200 
Included in cash and cash equivalents$246,994 $— $— $246,994 
Included in marketable securities39,196 14 (4)39,206
Total cash, cash equivalents, and marketable securities$286,190 $14 $(4)$286,200 
For both the nine months ended September 30, 2024 and September 30, 2023, there were no realized gains or losses. The cost of securities sold is based on the specific identification method.
There were no unrealized loss positions as of September 30, 2024. Unrealized loss positions in the marketable securities as of December 31, 2023 reflect temporary impairments and are not a result of credit loss. Additionally, as these positions have been in a loss position for less than twelve months and the Company does not intend to sell these securities before recovery, the losses are recognized as a component of other comprehensive income (loss). The fair value of these marketable securities in unrealized loss positions are $0.0 million and $11.4 million as of September 30, 2024 and December 31, 2023, respectively.
The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Consolidated Balance Sheets that sum to the total shown in the Consolidated Statements of Cash Flows.
As of September 30,
(in thousands)20242023
Cash and cash equivalents$233,647 $263,320 
Restricted cash3,173 2,952 
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows$236,820 $266,272 
13

4. Inventories
The following table summarizes the components of the Company’s inventories for each of the periods indicated:
(in thousands)September 30, 2024December 31, 2023
Raw materials$76,203 $30,230 
Work-in-process31,485 22,597 
Finished goods7,650 6,869 
Total inventories$115,338 $59,696 
The Company's reserve for inventory was $3.5 million and $0.5 million as of September 30, 2024 and December 31, 2023, respectively.
5. Debt
The following table summarizes the Company's debt for each of the periods indicated:
(in thousands)September 30, 2024December 31, 2023
Senior Secured Term Loan due 2029:
Principal$400,000 $400,000 
Less: debt discount (1)
(8,339)(9,652)
Less: deferred financing (1)
(2,167)(2,490)
Net carrying value of long-term debt$389,494 $387,858 
______________________________
(1) Included in the Company's Consolidated Balance Sheets within long-term debt and amortized to interest expense over the remaining life of the Senior Secured Term Loan due 2029 using the effective interest rate method.
Interest Expense
The following table sets forth interest expense recognized related to the Company's debt for the three and nine months ended September 30, 2024 and 2023, respectively:
Three Months Ended September 30,Nine Months Ended September 30,
(in thousands)2024202320242023
Contractual interest expense$12,106 $12,270 $36,021 $35,289 
Amortization of debt discount$455 $411 $1,313 $1,187 
Amortization of deferred financing$119 $310 $352 $893 
6. Restructuring
The restructuring liabilities are recorded in accrued expenses and other current liabilities in the Company’s Consolidated Balance Sheets. Total charges incurred are summarized as follows:
(in thousands)Facility and Non-Lease CostsOther CostsTotal
Balance as of December 31, 2023
$— $— $— 
Restructuring charges6,045 3,143 9,188 
Non-cash items(2,844)— (2,844)
Cash settled(981)(2,136)(3,117)
Balance as of September 30, 2024
$2,220 $1,007 $3,227 
7. Stockholders' Equity
14

During the three and nine months ended September 30, 2024, the Company issued and sold an aggregate of 1,692,291 shares through its at-the-market equity program ("ATM program") at weighted-average public offering price of $11.70 per share, resulting in net proceeds of $19.2 million. As of September 30, 2024, an aggregate of $164.7 million worth of shares remain available to be issued and sold under the ATM program.
8. Stock-Based Compensation
The Amended and Restated 2007 Equity Incentive Plan (the "Plan") provides for the granting of restricted stock units and options to purchase common stock in the Company to employees, directors, advisors, and consultants at a price to be determined by the Board of Directors. The Plan is intended to encourage ownership of stock by employees and consultants of the Company and to provide additional incentives for them to promote the success of the business. The Board of Directors, or its committee, is responsible for determining the individuals to be granted options, the number of options each individual will receive, the option price per share, and the exercise period of each option.
Stock Option Grants
The fair value of the stock options granted were estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions:
 Three Months Ended September 30,Nine Months Ended September 30,
 2024202320242023
Expected stock price volatility55.5 %58.4 %57.1 %59.2 %
Risk free interest rate3.9 %4.3 %4.0 %3.9 %
Expected life of options (years)5.65.55.65.5
Expected annual dividend per share$— $— $— $— 
 A summary of the Company's stock options for the nine months ended September 30, 2024 were as follows:
Number of
Shares		Weighted Average Exercise 
Price		Weighted Average Remaining
Years		Aggregate
Intrinsic
Value
 (in thousands)  (in millions)
Options outstanding, December 31, 202323,002 $11.69   
Granted4,693 $13.69   
Exercised(791)$7.74   
Forfeited(548)$12.74   
Expired(217)$14.58 
Options outstanding, September 30, 202426,139 $12.12 6.0$13.6 
Vested and non-vested expected to vest, September 30, 202424,610 $12.09 5.9$13.5 
Exercisable at September 30, 202417,092 $11.75 4.7$13.0 
As of September 30, 2024, the total unrecognized compensation cost related to non-vested stock options granted was $43.4 million and is expected to be recognized over a weighted average period of three years.
15

Restricted Stock Units and Performance-Based Restricted Stock Units (collectively "RSUs")
RSUs awarded under the Plan are generally subject to graded vesting and are contingent on an employee's continued service. RSUs are generally subject to forfeiture if employment terminates prior to the release of vesting restrictions. The Company expenses the cost of the RSUs, which is determined to be the fair market value of the shares of common stock underlying the RSUs at the date of grant, ratably over the period during which the vesting restrictions lapse. A summary of non-vested RSU activity under the Plan for the nine months ended September 30, 2024 is as follows:
Number of
Shares		Weighted
Average Grant
Date Fair
Value		Weighted 
Average
Remaining 
Years		Aggregate
Intrinsic
Value
(in thousands)(in millions)
Non-vested units as of December 31, 202310,033 $13.37   
Granted3,893 $14.63   
Vested(3,860)$13.17   
Forfeited(613)$14.28   
Non-vested units as of September 30, 20249,453 $13.82 2.2$101.0 
As of September 30, 2024, there was $62.8 million of total unrecognized compensation cost related to non-vested RSUs with service-based vesting conditions. These costs are expected to be recognized over a weighted average period of two years.
Compensation Expense Related to Equity Awards
The following table summarizes information related to compensation expense recognized in the Company's Consolidated Statements of Operations related to the equity awards:
 Three Months Ended September 30,Nine Months Ended September 30,
(in thousands)2024202320242023
Research and development expense$4,397 $4,380 $12,329 $16,987 
Selling, general, and administrative expense14,291 12,131 53,359 50,995 
Total equity compensation expense$18,688 $16,511 $65,688 $67,982 
9. Assets and Liabilities Measured at Fair Value
The Company's financial assets and liabilities are measured at fair value and classified within the fair value hierarchy, which is defined as follows:
Level 1 — Quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date.
Level 2 — Inputs other than quoted prices in active markets that are observable for the asset or liability, either directly or indirectly.
Level 3 — Inputs that are unobservable for the asset or liability.
16

A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of September 30, 2024 are identified in the following tables:
(in thousands) Level 1Level 2Total
Assets:  
Commercial paper$— $5,991 $5,991 
Treasury bill— 9,968 9,968 
Money market7,922 — 7,922 
 $7,922 $15,959 $23,881 
(in thousands) Level 1Level 2Total
Liabilities:  
Deferred compensation plan liability$7,822 $— $7,822 
 $7,822 $— $7,822 
A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2023 are identified in the following tables:
(in thousands)Level 1Level 2Total
Assets:
Commercial paper$— $14,663 $14,663 
Treasury bill— 12,946 12,946 
U.S. government agency bonds— 11,446 11,446 
Money market7,631 — 7,631 
 $7,631 $39,055 $46,686 
(in thousands)Level 1Level 2Total
Liabilities:   
Deferred compensation plan liability7,531 — 7,531 
 $7,531 $— $7,531 
Deferred compensation plan liability is recorded as a component of other non-current liabilities on the Company's Consolidated Balance Sheets. The Company did not have any Level 3 assets or liabilities as of September 30, 2024 or December 31, 2023.
Cash, Money Market Funds, and Marketable Securities
The Company classifies its cash and money market funds within the fair value hierarchy as Level 1 as these assets are valued using quoted prices in an active market for identical assets at the measurement date. The Company considers its investments in marketable securities as available-for-sale and classifies these assets within the fair value hierarchy as Level 2 primarily utilizing broker quotes in a non-active market for valuation of these securities.
17

10. Basic and Diluted Net Loss per Common Share
The following table provides a reconciliation of the numerator and denominator used in computing basic and diluted net loss attributable to common stockholders per common share:
 Three Months Ended September 30,Nine Months Ended September 30,
(in thousands, except per share amounts) 2024202320242023
Numerator:  
Net loss attributable to common stockholders$(6,729)$(21,577)$(70,845)$(117,741)
Denominator:
Weighted average common shares outstanding — basic and diluted304,690,596 295,759,435 303,792,479 293,314,167 
Dilutive common stock equivalents would include the dilutive effect of outstanding common stock options and non-vested RSUs. Potentially dilutive common stock equivalents were excluded from the diluted earnings per share denominator for all periods because of their anti-dilutive effect. Weighted average common shares outstanding includes outstanding pre-funded warrants with an exercise price of $0.01.
The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method:
 As of September 30,
(in thousands) 20242023
Options to purchase common stock26,139 23,256 
Non-vested restricted stock units9,453 10,331 
Total number of potentially issuable shares35,592 33,587 

18

ITEM 2. MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS
The following discussion should be read in conjunction with the unaudited Consolidated Financial Statements and the notes thereto included in this Quarterly Report on Form 10-Q and the audited Consolidated Financial Statements and the notes thereto included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2023. Some of the statements we make in this section are forward-looking statements within the meaning of the federal securities laws. For a complete discussion of forward-looking statements, see the section in this Quarterly Report on Form 10-Q entitled “Special Note Regarding Forward-Looking Statements”. Certain risk factors may cause actual results, performance or achievements to differ materially from those expressed or implied by the following discussion. For a discussion of such risk factors, see the section in our Annual Report on Form 10-K for the fiscal year ended December 31, 2023 entitled “Risk Factors”.
Overview
We are a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases. We seek to deliver the highest quality therapies that have the potential to obsolete current treatments, provide significant benefits to patients, and be first- or best-in-class. Our two marketed therapies are Galafold®, the first oral monotherapy for people living with Fabry disease who have amenable genetic variants, and Pombiliti® + Opfolda®, a novel two-component treatment designed to improve uptake of active enzyme into key disease relevant tissues for adults living with late-onset Pompe disease.
Galafold® (also referred to as "migalastat") is approved in over 40 countries around the world, including the United States ("U.S."), European Union ("E.U."), United Kingdom ("U.K."), and Japan. Additionally, Galafold® has been granted orphan drug status in the U.S., E.U., U.K., Japan and several other countries.
Pombiliti® + Opfolda® (also referred to as "cipaglucosidase alfa-atga/miglustat") is approved in the U.S., the E.U., the U.K., and Switzerland. Multiple regulatory submissions and reimbursement processes with global health authorities are currently underway. Additionally, Pombiliti® + Opfolda® has been granted orphan drug designation or status in the U.S., U.K., Switzerland and Japan and data exclusivity in the E.U.
Our Strategy
Our strategy is to create, manufacture, test, and deliver the highest quality medicines for people living with rare diseases through internally developed, jointly developed, acquired, or in-licensed products and product candidates. We are leveraging our global capabilities to develop and broaden our franchises in Fabry and Pompe disease, with focused discovery work on next generation therapies and novel technologies.
Highlights of our progress include:
•Commercial success in Fabry disease. For the nine months ended September 30, 2024, Galafold® revenue was $330.6 million of consolidated revenue, which represented an increase of $49.4 million compared the same period in the prior year. We continue to see strong commercial momentum and expansion into additional geographies.
•Commercial and regulatory success in Pompe disease. For the nine months ended September 30, 2024, Pombiliti® + Opfolda® revenue was $48.0 million of consolidated revenue. Pombiliti® + Opfolda® was approved by the European Commission ("EC") in June 2023, the Medicines and Healthcare products Regulatory Agency ("MHRA)" of the United Kingdom in August 2023, the U.S. Food and Drug Administration ("FDA") in September 2023, and the Swissmedic of Switzerland in July 2024.
•Pipeline advancement and growth. We are leveraging our global capabilities to develop and broaden our franchises in Fabry and Pompe disease, with focused discovery work on next generation therapies and novel technologies.
•Financial strength. Total cash, cash equivalents, and marketable securities as of September 30, 2024 was $249.8 million.
19

 Our Commercial Products and Product Candidates
Galafold® (migalastat HCl) for Fabry Disease
Our oral precision medicine, Galafold®, was granted accelerated approval by the FDA in August 2018 for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene ("GLA") variant based on in vitro assay data. Galafold® was approved in the E.U. and U.K. in May 2016 as a first-line therapy for long-term treatment of adults and adolescents, aged 16 years and older, with a confirmed diagnosis of Fabry disease and who have an amenable variant. Marketing authorization approvals as well as approvals for adolescents aged 12 years and older weighing 45 kg or more have been granted in over 40 countries around the world. We plan to continue to launch Galafold® in additional countries upon receipt of marketing authorization.
As an orally administered monotherapy, Galafold® is designed to bind to and stabilize an endogenous alpha-galactosidase A ("alpha-Gal A") enzyme in those patients with genetic variants identified as amenable in a Good Laboratory Practice ("GLP") cell-based amenability assay.
Pombiliti® (cipaglucosidase alfa-atga) + Opfolda® (miglustat) for Pompe Disease
We have leveraged our biologics capabilities to develop Pombiliti® + Opfolda®, a novel treatment paradigm for Pompe disease. Pombiliti® + Opfolda® was approved by the EC in June 2023, the MHRA in August 2023, the FDA in September 2023, and the Swissmedic in July 2024 for adult late-onset Pompe disease ("LOPD") patients. Additional regulatory submissions and reimbursement processes with global health authorities are currently underway.
Pombiliti® + Opfolda® consists of a uniquely engineered rhGAA enzyme, cipaglucosidase alfa-atga, with an optimized carbohydrate structure to enhance cellular uptake, administered intravenously in combination with orally administered miglustat. Miglustat binds to and stabilizes the cipaglucosidase alfa-atga in circulation reducing inactivation of rhGAA to improve the uptake of active enzyme into key disease relevant tissues. Miglustat is not an active ingredient that contributes directly to glycogen reduction.
In addition, clinical studies are ongoing in pediatric patients for both the LOPD and infantile-onset Pompe disease ("IOPD") populations.
Next Generation Therapies
We are committed to continued innovation for all people living with Fabry or Pompe disease. As part of our long-term commitment, we are continuing discovery efforts for next-generation genetic medicines for Fabry and Pompe disease.
Strategic Alliances and Arrangements
We will continue to evaluate business development opportunities to build stockholder value and provide us with access to the financial, technical, clinical, commercial resources, and intellectual property necessary to develop and market technologies or products in rare and orphan diseases. We are exploring potential collaborations, alliances, and various other business development opportunities on a regular basis. These opportunities may include business combinations, partnerships, the strategic out-licensing of certain assets, or the acquisition of preclinical-stage, clinical-stage, or marketed products or novel technologies consistent with our corporate strategy to develop and provide therapies to patients living with rare and orphan diseases.
20

Consolidated Results of Operations
Three Months Ended September 30, 2024 compared to September 30, 2023
The following table provides selected financial information for the Company:
Three Months Ended September 30,
(in thousands)20242023Change
Net product sales$141,517 $103,501 $38,016 
Cost of goods sold13,279 9,946 3,333 
Cost of goods sold as a percentage of net product sales9.4 %9.6 %(0.2)%
Operating expenses:
Research and development26,160 40,704 (14,544)
Selling, general, and administrative75,106 65,651 9,455 
Changes in fair value of contingent consideration payable— 1,995 (1,995)
Restructuring charges3,143 — 3,143 
Depreciation and amortization2,170 2,228 (58)
Other expense:
Interest income1,081 1,471 (390)
Interest expense(12,692)(12,986)294 
Other (expense) income(3,263)3,833 (7,096)
Income tax (expense) benefit(13,514)3,128 (16,642)
Net loss attributable to common stockholders$(6,729)$(21,577)$14,848 
Net Product Sales. Net product sales increased $38.0 million during the three months ended September 30, 2024 compared to the same period in the prior year. The increase was primarily due to both the continued growth of Galafold® in Europe and the U.S. as well as the launch of Pombiliti® + Opfolda® in Europe and the U.S., and a $0.8 million favorable impact of foreign currency exchange.
Research and Development Expense. The following table summarizes our principal development programs and the out-of-pocket, third-party expenses incurred:
(in thousands)Three Months Ended September 30,
Projects20242023
Third party direct project expenses  
Galafold® (Fabry Disease)
$2,380 $4,505 
Pombiliti® + Opfolda® (Pompe Disease)
10,590 15,233 
Pre-clinical and other programs646 1,753 
Total third-party direct project expenses13,616 21,491 
Other project costs  
Personnel costs9,707 15,194 
Other costs2,837 4,019 
Total other project costs12,544 19,213 
Total research and development costs$26,160 $40,704 
The $14.5 million decrease in research and development costs was primarily driven by our Pombiliti® + Opfolda® commercial launch, decreasing both clinical spend and the number of employees supporting research and development efforts.
Selling, General, and Administrative Expense. Selling, general, and administrative expense increased $9.5 million, primarily driven by personnel costs resulting from an increase in the number of employees to support our commercial launch activities and third party professional fees.
21

Other (Expense) Income. The decrease of $7.1 million was primarily related to movement in foreign exchange rates caused by remeasurement of foreign-denominated balances.
Income Tax Expense. We are subject to income taxes in various jurisdictions. Our tax liabilities are largely dependent on the mix of pre-tax earnings among the many jurisdictions in which we operate and differences in the timing of the recognition of such earnings under the relevant accounting standards and tax rules.
Consolidated Results of Operations
Nine Months Ended September 30, 2024 compared to September 30, 2023
The following table provides selected financial information for the Company:
Nine Months Ended September 30,
(in thousands)20242023Change
Net product sales$378,589 $284,274 $94,315 
Cost of goods sold38,107 26,002 12,105 
Cost of goods sold as a percentage of net product sales10.1 %9.1 %1.0 %
Operating expenses:
Research and development79,172 117,352 (38,180)
Selling, general, and administrative236,711 205,031 31,680 
Changes in fair value of contingent consideration payable— 2,583 (2,583)
Restructuring charges9,188 — 9,188 
Loss on impairment of assets— 1,134 (1,134)
Depreciation and amortization6,506 5,691 815 
Other expense:
Interest income3,991 5,407 (1,416)
Interest expense(37,640)(37,322)(318)
Other expense(11,946)(13,007)1,061 
Income tax (expense) benefit(34,155)700 (34,855)
Net loss attributable to common stockholders$(70,845)$(117,741)$46,896 
Net Product Sales. Net product sales increased $94.3 million during the nine months ended September 30, 2024 compared to the same period in the prior year. The increase was primarily due to both the continued growth of Galafold® in Europe and the U.S. as well as the launch of Pombiliti® + Opfolda® in Europe and the U.S., partially offset by the $0.9 million unfavorable impact of foreign currency exchange.
22

Cost of goods sold. Cost of goods sold includes manufacturing costs as well as royalties associated with net product sales. Cost of goods sold as a percentage of net product sales increased 1.0% primarily due to inventory write-offs associated with validation efforts in the current period.
Research and Development Expense. The following table summarizes our principal development programs and the out-of-pocket, third-party expenses incurred:
(in thousands)Nine Months Ended September 30,
Projects20242023
Third party direct project expenses  
Galafold® (Fabry Disease)
$6,420 $10,971 
Pombiliti® + Opfolda® (Pompe Disease)
31,003 44,126 
Pre-clinical and other programs2,095 3,218 
Total third-party direct project expenses39,518 58,315 
Other project costs  
Personnel costs31,287 47,108 
Other costs8,367 11,929 
Total other project costs39,654 59,037 
Total research and development costs$79,172 $117,352 
The $38.2 million decrease in research and development costs was primarily driven by our Pombiliti® + Opfolda® commercial launch, decreasing both clinical spend and the number of employees supporting research and development efforts.
Selling, General, and Administrative Expense. Selling, general, and administrative expense increased $31.7 million, primarily driven by personnel costs resulting from an increase in the number of employees to support our commercial launch activities, external costs required to support the manufacture and sale of our commercial products, and third-party professional fees.
Restructuring Charges. Restructuring charges were primarily related to an initiative to reduce operating costs by abandoning a lease that we no longer believe is useful in our operations.
Income Tax Expense. We are subject to income taxes in various jurisdictions. Our tax liabilities are largely dependent on the mix of pre-tax earnings among the many jurisdictions in which we operate and differences in the timing of the recognition of such earnings under the relevant accounting standards and tax rules.
Liquidity and Capital Resources
As a result of our significant research and development expenditures, as well as expenditures to build a commercial organization to support the launch of Galafold® and Pombiliti® + Opfolda®, we have not been profitable and have generated operating losses since we were incorporated in 2002. We have historically funded our operations through stock offerings, product revenues, debt issuance, collaborations, and other financing arrangements.
Sources of Liquidity
In November 2022, we entered into a Sales Agreement with Goldman Sachs & Co. LLC to create an at-the-market equity program ("ATM program"), pursuant to which we may offer to sell shares of our common stock having an aggregate offering gross proceeds of up to $250.0 million. As of September 30, 2024, an aggregate of $164.7 million worth of shares remain available to be issued and sold under the ATM program.
23

Cash Flow Discussion
As of September 30, 2024, we had cash, cash equivalents, and marketable securities of $249.8 million. We invest cash in excess of our immediate requirements in regard to liquidity and capital preservation in a variety of interest-bearing instruments, including obligations of U.S. government agencies and money market accounts. Wherever possible, we seek to minimize the potential effects of concentration and degrees of risk. Although we maintain cash balances with financial institutions in excess of insured limits, we do not anticipate any losses with respect to such cash balances. For more details on the cash, cash equivalents, and marketable securities, refer to "— Note 3. Cash, Cash Equivalents, Marketable Securities, and Restricted Cash," in our Notes to Consolidated Financial Statements.
Net Cash Used in Operating Activities
Net cash used in operations for the nine months ended September 30, 2024 was $30.0 million. The components of net cash used in operations included the net loss for the nine months ended September 30, 2024 of $70.8 million and a net decrease in changes in operating assets and liabilities of $55.2 million offset by $65.7 million of stock compensation and $30.4 million of other non-cash adjustments. The changes in operating assets and liabilities were primarily due to an increase in inventory of $61.6 million to support our continued commercial growth and an increase in accounts receivable of $9.1 million, partially offset by a decrease in prepaid expenses and other current assets of $28.2 million and a decrease in accounts payable and accrued expenses of $11.5 million associated with timing of payments.
Net cash used in operations for the nine months ended September 30, 2023 was $72.5 million. The components of net cash used in operations included the net loss for the nine months ended September 30, 2023 of $117.7 million offset by $68.0 million of stock compensation, $25.9 million of other non-cash adjustments, and a net increase in changes in operating assets and liabilities of $48.7 million. The changes in operating assets and liabilities were primarily due to an increase in inventory of $42.2 million and an increase in prepaid expenses and other current assets of $26.0 million, partially offset by an increase in accounts payable and accrued expenses of $41.1 million associated with Pombiliti® + Opfolda® launch activities and increases in sales rebates associated with increased commercial sales of Galafold®.
Net Cash Provided by Investing Activities
Net cash provided by investing activities for the nine months ended September 30, 2024 was $19.8 million. Our investing activities have consisted primarily of purchases, sales, and maturities of investments and capital expenditures. Net cash provided by investing activities reflects $101.9 million from the sale and redemption of marketable securities, partially offset by $78.8 million for the purchase of marketable securities and $3.3 million for capital expenditures.
Net cash provided by investing activities for the nine months ended September 30, 2023 was $122.0 million. Our investing activities have consisted primarily of purchases, sales and maturities of investments and capital expenditures. Net cash provided by investing activities reflects $180.8 million from the sale and redemption of marketable securities, partially offset by $53.1 million for the purchase of marketable securities and $5.7 million for capital expenditures.
Net Cash Provided by Financing Activities
Net cash provided by financing activities for the nine months ended September 30, 2024 was $4.0 million. Net cash provided by financing activities primarily reflects $19.2 million of proceeds from the issuance of shares in connection with the ATM program offering, net of issuance costs, and $6.1 million of proceeds from the exercise of stock options, partially offset by the withholding taxes paid on vested restricted stock unites of $21.2 million.
Net cash provided by financing activities for the nine months ended September 30, 2023 was $53.5 million. Net cash provided by financing activities primarily reflects $63.1 million of proceeds from the issuance of shares in connection with the ATM program offering, net of issuance costs, and $7.8 million of proceeds from the exercise of stock options, partially offset by the withholding taxes paid on vested restricted stock units of $16.4 million.
24

Funding Requirements
We expect to continue to incur significant costs in the foreseeable future primarily due to research and development expenses, including expenses related to conducting clinical trials. Our future capital requirements will depend on a number of factors, including:
•the scope, progress, results and costs of clinical trials for our drug candidates;
•the cost of manufacturing drug supply for our commercial, clinical and preclinical studies, including the cost of manufacturing Pombiliti® (also referred to as "ATB200" or "cipaglucosidase alfa");
•the future results of preclinical research and subsequent clinical trials for pipeline candidates we may identify from time to time, including our ability to obtain regulatory approvals and commercialize such therapies;
•the costs, timing, and outcome of regulatory review of our product candidates;
•any changes in regulatory standards relating to the review of our product candidates;
•any changes in laws, rules or regulations affecting our ability to manufacture, transport, test, develop, or commercialize our products, including Galafold®, Pombiliti® + Opfolda®, or our product candidates;
•the costs of commercialization activities, including product marketing, sales, and distribution;
•the emergence of competing technologies and other adverse market developments;
•the estimates regarding the potential market opportunity for our products and product candidates;
•our ability to successfully commercialize Galafold® (also referred to as "migalastat HCl");
•our ability to successfully commercialize Pombiliti® + Opfolda® (together, also referred to as "AT-GAA") in the E.U., U.K., and U.S., Switzerland and elsewhere, if regulatory applications are approved;
•our ability to manufacture or supply sufficient clinical or commercial products, including Galafold® and Pombiliti® + Opfolda®;
•our ability to obtain reimbursement for Galafold® and Pombiliti® + Opfolda®;
•our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of Galafold® and Pombiliti® + Opfolda®;
•our ability to obtain market acceptance of Galafold® and Pombiliti® + Opfolda® or any other product developed or acquired that has received regulatory approval;
•the costs of preparing, filing, and prosecuting patent applications and maintaining, enforcing, and defending intellectual property-related claims, including Hatch-Waxman litigation;
•the impact of litigation that has been or may be brought against us or of litigation that we are pursuing or may pursue against others, including Hatch-Waxman litigation;
•the extent to which we acquire or invest in businesses, products, and technologies;
•our ability to successfully integrate our acquired products and technologies into our business, or successfully divest or license existing products and technologies from our business, including the possibility that the expected benefits of the transactions will not be fully realized by us or may take longer to realize than expected;
•our ability to establish licensing agreements, collaborations, partnerships or other similar arrangements and to obtain milestone, royalty, or other economic benefits from any such collaborators;
•the costs associated with, and our ability to comply with, emerging environmental, social and governance standards, including climate reporting requirements at the local, state and national levels;
•our ability to successfully protect our information technology systems and maintain our global operations and supply chain without interruption;
•our ability to accurately forecast revenue, operating expenditures, or other metrics impacting profitability;
•fluctuations in foreign currency exchange rates; and
•changes in accounting standards.
25

We may seek additional funding through public or private financings of debt or equity. Based on our current operating model, which includes expected revenues, we believe that the current cash position is sufficient to fund our operations and ongoing research programs for at least the next 12 months. Potential impacts of business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact our long-term capital requirements.
Critical Accounting Policies and Significant Judgments
The discussion and analysis of our financial condition and results of operations are based on our financial statements, which we have prepared in accordance with U.S. GAAP. The preparation of these financial statements requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements, as well as the reported revenues and expenses during the reporting periods. On an ongoing basis, we evaluate our estimates and judgments and make changes when necessary. We base our estimates on historical experience and on various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions.
There were no significant changes during the nine months ended September 30, 2024 to the items that we disclosed as our significant accounting policies and estimates described in "—Note 2. Summary of Significant Accounting Policies" to the Company's financial statements as contained in the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2023.
Recent Accounting Pronouncements
Please refer to "—Note 2. Summary of Significant Accounting Policies" in our Notes to Consolidated Financial Statements.
26

ITEM 3. QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK
Our market risks, and the way we manage them, are summarized in Part II, Item 7A, "Quantitative and Qualitative Disclosures About Market Risk" of our Annual Report on Form 10-K for the fiscal year ended December 31, 2023. As of September 30, 2024, there have been no material changes to our market risks or to our management of such risks since December 31, 2023.
ITEM 4. CONTROLS AND PROCEDURES
Evaluation of Disclosure Controls and Procedures
As of the end of the period covered by this Quarterly Report on Form 10-Q, an evaluation of the effectiveness of our disclosure controls and procedures (pursuant to Rule 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended (the "Exchange Act") was carried out under the supervision of our Principal Executive Officer and Principal Financial Officer, with the participation of our management. Based on that evaluation, the Principal Executive Officer and the Principal Financial Officer concluded that, as of the end of such period, our disclosure controls and procedures are effective in recording, processing, summarizing and reporting, on a timely basis, information required to be disclosed by us in the reports that we file or submit under the Exchange Act and are effective in ensuring that information required to be disclosed by us in the reports that we file or submit under the Exchange Act is accumulated and communicated to our management, including our Principal Executive Officer and Principal Financial Officer, as appropriate, to allow timely decisions regarding required disclosure.
During the fiscal quarter covered by this report, there has been no change in our internal control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act) that occurred during the last fiscal quarter that has materially affected, or is reasonably likely to materially affect, our internal control over financial reporting.
PART II. OTHER INFORMATION
ITEM 1. LEGAL PROCEEDINGS
In the fourth quarter of 2022, the Company received Paragraph IV Certification Notice Letters from Teva Pharmaceuticals USA, Inc. ("Teva"), Aurobindo Pharma Limited ("Aurobindo"), and Lupin Limited ("Lupin") in connection with Abbreviated New Drug Applications (“ANDA”) filed with the FDA requesting approval to market generic Galafold®. In November 2022, the Company filed four lawsuits against Teva, Lupin, and Aurobindo in the U.S. District Court for the District of Delaware (the "Court") for infringement of its Orange Book-listed patents. In the fourth quarter of 2023, a stipulation order to stay litigation with respect to Lupin was ordered. Additionally, in the first quarter of 2024, a stipulation was filed with the court and approved by the presiding judge, whereby the parties agreed to accept the Company’s definition of the terms that were in dispute. As such, the scheduled Markman hearing was deemed unneeded and cancelled.
In October 2024, the Company entered into a non-exclusive, non-transferable, royalty-free, fully paid-up license with Teva which will allow Teva to market its generic version of Galafold® in the United States beginning on January 30, 2037, or earlier in certain circumstances. In accordance with the license agreement, a consent judgment and permanent injunction was entered with the Court and all Hatch-Waxman litigation between Amicus and Teva has been terminated. As required by law, Amicus and Teva have submitted the confidential license agreement to the U.S. Federal Trade Commission and the U.S. Department of Justice for review.
The litigation will continue against Aurobindo as the remaining active party, and the litigation stay remains in place for Lupin. The Company has, and will continue to, vigorously enforce its Galafold® intellectual property rights.
ITEM 1A. RISK FACTORS
There have been no material changes to the risk factors previously disclosed in our Annual Report on Form 10-K for the fiscal year ended December 31, 2023.
ITEM 2. UNREGISTERED SALES OF EQUITY SECURITIES AND USE OF PROCEEDS
Recent Sales of Unregistered Securities
None.
27

Issuer Purchases of Equity Securities
The following table provides certain information with respect to purchase of our common stock during the three months ended September 30, 2024:
Period
Total Number of Shares Purchased (1)
Average Price Paid per ShareTotal Number of Shares Purchased as Part of Publicly Announced Plans or ProgramsMaximum Number (or Approximate Dollar Value) of Shares That May Yet Be Purchased Under the Plans or Programs
July 1, 2024 through July 31, 202492,738 $10.59 — — 
August 1, 2024 through August 31, 202423,287 $11.80 — — 
September 1, 2024 through September 30, 2024127,806 $11.60 — — 
Total243,831 $11.23 — — 
______________________________
(1) Represents shares of common stock withheld to satisfy taxes associated with the vesting of restricted stock units
ITEM 3. DEFAULTS UPON SENIOR SECURITIES
None.
ITEM 4. MINE SAFETY DISCLOSURES
None.
ITEM 5. OTHER INFORMATION
Rule 10b5-1 Trading Plans
The following table describes, for the quarterly period covered by this report, each director and officer (as defined in Rule 16a-1(f) under the Exchange Act who has adopted, modified, or terminated a trading plan intended to satisfy the affirmative defense of Rule 10b5-1(c) under the Exchange Act (each plan, a “Rule 10b5-1 Trading Plan”). Each Rule 10b5-1 Trading Plan described below was adopted during an open insider trading window and in accordance with the Company’s policies regarding both insider trading and transactions relating to Company securities.
Name
(Title)		Action Taken
(Date of Action)		Rule 10b5-1 Trading Plan Provides for Purchase/Sale
Duration of the Trading Plan(1)
Aggregate Number of Securities
Bradley Campbell
(President and Chief Executive Officer)
Adoption
(September 13, 2024)
SaleDecember 31, 2025
235,460 (2)
Jeffrey Castelli
(Chief Development Officer)
Adoption
(September 10, 2024)
SaleMay 31, 2025
132,954 (3)
David Clark
(Chief People Officer)
Adoption
(August 28, 2024)
SaleAugust 15, 2025
Indeterminable (4)
Samantha Prout
(Chief Accounting Officer)
Adoption
(September 12, 2024)
SaleJuly 1, 2025
Indeterminable (5)
Ellen Rosenberg
(Chief Legal Officer and Corporate Secretary)
Adoption
(September 10, 2024)
SaleDecember 31, 2025
95,621 (6)
(1) The dates in this column represent the scheduled expiration date of each director or officer’s Rule 10b5-1 Trading Plan. Each Rule 10b5-1 Trading Plan may terminate earlier than the date provided should all transactions contemplated thereunder occur prior to such date.
(2) Mr. Campbell’s Rule 10b5-1 Trading Plan provides for the (i) sale of 60,460 shares of common stock and (ii) exercise of up to 175,000 stock options and subsequent sale of up to 175,000 shares of common stock underlying such stock options.
(3) Mr. Castelli’s Rule 10b5-1 Trading Plan provides for the (i) sale of 82,954 shares of common stock and (ii) exercise of up to 50,000 stock options and subsequent sale of up to 50,000 shares of common stock underlying such stock options.
28

(4) Mr. Clark’s Rule 10b5-1 Trading Plan provides for the sale of common stock that will be obtained from the settlement of his 2022 PRSU awards and the respective vesting of his 2021, 2022, 2023 and 2024 annual restricted stock unit awards. The number of shares of common stock obtained and available for sale will be subject to the (i) level of achievement of each performance goal contained within the 2022 PRSU awards and (ii) shares of common stock withheld to satisfy applicable tax withholding obligations.
(5) Ms. Prout’s Rule 10b5-1 Trading Plan provides for the (i) exercise of up to 66,074 stock options and the sale of up to 66,074 underlying shares of common stock, (ii) sale of 7,243 shares of common stock and (iii) sale of an indeterminable number of shares of common stock to be obtained from the respective vesting of her 2021, 2022, 2023 and 2024 annual restricted stock unit awards. The number of shares of common stock obtained and available for sale in clause (iii) will be subject to the shares of common stock withheld to satisfy applicable tax withholding obligations.
(6) Ms. Rosenberg’s Rule 10b5-1 Trading Plan provides for the exercise of up to 95,621 stock options and the sale of up to 95,621 underlying shares of common stock.
29

ITEM 6. EXHIBITS
Exhibit
Number		 Description
31.1 
   
31.2 
   
32.1 
   
101.INS
Inline XBRL Instance Document - the instance document does not appear in the Interactive Data File because its XBRL tags are embedded within the Inline XBRL document
101.SCH Inline XBRL Taxonomy Extension Schema Document
101.CAL Inline XBRL Taxonomy Extension Calculation Linkbase Document
101.LAB Inline XBRL Taxonomy Extension Label Linkbase Document
101.PRE Inline XBRL Taxonomy Extension Presentation Linkbase Document
101.DEF Inline XBRL Taxonomy Extension Definition Linkbase Document
104Cover Page Interactive Data File (formatted in Inline XBRL and included in Exhibit 101)

____________________________________________________________________




30

SIGNATURES
Pursuant to the requirements of the Securities and Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
 AMICUS THERAPEUTICS, INC.
  
Date:November 6, 2024By:/s/ Bradley L. Campbell
  Bradley L. Campbell
  President and Chief Executive Officer
  (Principal Executive Officer)
  
Date:November 6, 2024By:/s/ Simon Harford
  Simon Harford
  Chief Financial Officer
  (Principal Financial Officer)

31

Exhibit 31.1
 
CERTIFICATIONS PURSUANT TO SECTION 302 OF
THE SARBANES-OXLEY ACT OF 2002
CERTIFICATION BY PRINCIPAL EXECUTIVE OFFICER
 
I, Bradley L. Campbell, certify that:
1.I have reviewed this quarterly report on Form 10-Q of Amicus Therapeutics, Inc.;
2.Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;
3.Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;
4.The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
(a)designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;
(b)designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and preparation of financial statements for external purposes in accordance with generally accepted accounting principles;
(c)evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and
(d)disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and
5.The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):
(a)all significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and
(b)any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.
 
Date: November 6, 2024/s/ Bradley L. Campbell
 Bradley L. Campbell
 President and Chief Executive Officer
 




Exhibit 31.2
 
CERTIFICATIONS PURSUANT TO SECTION 302 OF
THE SARBANES-OXLEY ACT OF 2002
CERTIFICATION BY PRINCIPAL FINANCIAL OFFICER
 
I, Simon Harford, certify that:
 
1.I have reviewed this quarterly report on Form 10-Q of Amicus Therapeutics, Inc.;
2.Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;
3.Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;
4.The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
(a)designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;
(b)designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and preparation of financial statements for external purposes in accordance with generally accepted accounting principles;
(c)evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and
(d)disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and
5.The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):
(a)all significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and
(b)any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.
 
Date: November 6, 2024/s/ Simon Harford
 Simon Harford
 Chief Financial Officer
 



Exhibit 32.1
 
CERTIFICATION OF PRINCIPAL EXECUTIVE OFFICER AND
PRINCIPAL FINANCIAL OFFICER PURSUANT TO
18 U.S.C. SECTION 1350, AS ADOPTED PURSUANT TO
SECTION 906 OF THE SARBANES-OXLEY ACT OF 2002
 
    Each of the undersigned hereby certifies, pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002, in his or her capacity as an officer of Amicus Therapeutics, Inc. (the “Company”), that, to his or her knowledge, the Quarterly Report of the Company on Form 10-Q for the period ended September 30, 2024, fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934 (15 U.S.C. 78m or 78o(d)) and that the information contained in such report fairly presents, in all material respects, the financial condition and results of operations of the Company. This written statement is being furnished to the Securities and Exchange Commission as an exhibit to such Form 10-Q. A signed original of this statement has been provided to the Company and will be retained by the Company and furnished to the Securities and Exchange Commission or its staff upon request.

 
Date: November 6, 2024By:/s/ Bradley L. Campbell
  Bradley L. Campbell
  President and Chief Executive Officer
   
Date: November 6, 2024By:/s/ Simon Harford
  Simon Harford
  Chief Financial Officer
 


v3.24.3
Cover - shares
 9 Months Ended
Sep. 30, 2024
Oct. 25, 2024
Cover [Abstract]    
Document Type 10-Q  
Document Quarterly Report true  
Document Period End Date Sep. 30, 2024  
Document Transition Report false  
Entity File Number 001-33497  
Entity Registrant Name Amicus Therapeutics, Inc.  
Entity Incorporation, State or Country Code DE  
Entity Tax Identification Number 71-0869350  
Entity Address, Address Line One 47 Hulfish Street  
Entity Address, City or Town Princeton  
Entity Address, State or Province NJ  
Entity Address, Postal Zip Code 08542  
City Area Code (609)  
Local Phone Number 662-2000  
Title of 12(b) Security Common Stock, par value $0.01 per share  
Trading Symbol FOLD  
Security Exchange Name NASDAQ  
Entity Current Reporting Status Yes  
Entity Interactive Data Current Yes  
Entity Filer Category Large Accelerated Filer  
Entity Small Business false  
Entity Emerging Growth Company false  
Entity Shell Company false  
Entity Common Stock, Shares Outstanding   298,805,773
Entity Central Index Key 0001178879  
Amendment Flag false  
Current Fiscal Year End Date --12-31  
Document Fiscal Year Focus 2024  
Document Fiscal Period Focus Q3  
v3.24.3
Consolidated Balance Sheets - USD ($)
$ in Thousands
Sep. 30, 2024
Dec. 31, 2023
Current assets:    
Cash and cash equivalents $ 233,647 $ 246,994
Investments in marketable securities 16,110 39,206
Accounts receivable 98,073 87,632
Inventories 115,338 59,696
Prepaid expenses and other current assets 35,306 49,533
Total current assets 498,474 483,061
Operating lease right-of-use assets, net 23,144 26,312
Property and equipment, less accumulated depreciation of $29,324 and $25,429 at September 30, 2024 and December 31, 2023, respectively 30,438 31,667
Intangible assets, less accumulated amortization of $4,974 and $2,510 at September 30, 2024 and December 31, 2023, respectively 18,026 20,490
Goodwill 197,797 197,797
Other non-current assets 18,678 18,553
Total Assets 786,557 777,880
Current liabilities:    
Accounts payable 13,481 15,120
Accrued expenses and other current liabilities 136,116 144,245
Operating lease liabilities 8,541 8,324
Total current liabilities 158,138 167,689
Long-term debt 389,494 387,858
Operating lease liabilities 46,623 48,877
Other non-current liabilities 13,477 13,282
Total liabilities 607,732 617,706
Commitments and contingencies
Stockholders’ equity:    
Common stock, $0.01 par value, 500,000,000 shares authorized, 298,691,094 and 293,594,209 shares issued and outstanding at September 30, 2024 and December 31, 2023, respectively 2,942 2,918
Additional paid-in capital 2,905,760 2,836,018
Accumulated other comprehensive income (loss):    
Foreign currency translation adjustment 25,159 5,429
Unrealized loss on available-for-sale securities (188) (188)
Warrants 71 71
Accumulated deficit (2,754,919) (2,684,074)
Total stockholders’ equity 178,825 160,174
Total Liabilities and Stockholders’ Equity $ 786,557 $ 777,880
v3.24.3
Consolidated Balance Sheets (Parenthetical) - USD ($)
$ in Thousands
Sep. 30, 2024
Dec. 31, 2023
Statement of Financial Position [Abstract]    
Accumulated depreciation of property and equipment $ 29,324 $ 25,429
Accumulated amortization of intangible assets $ 4,974 $ 2,510
Common stock, par value (in dollars per share) $ 0.01 $ 0.01
Common stock, shares authorized (in shares) 500,000,000 500,000,000
Common stock, shares issued (in shares) 298,691,094 293,594,209
Common stock, shares outstanding (in shares) 298,691,094 293,594,209
v3.24.3
Consolidated Statements of Operations - USD ($)
$ in Thousands
 3 Months Ended 9 Months Ended
Sep. 30, 2024
Sep. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Income Statement [Abstract]        
Net product sales $ 141,517 $ 103,501 $ 378,589 $ 284,274
Cost of goods sold 13,279 9,946 38,107 26,002
Gross profit 128,238 93,555 340,482 258,272
Operating expenses:        
Research and development 26,160 40,704 79,172 117,352
Selling, general, and administrative 75,106 65,651 236,711 205,031
Changes in fair value of contingent consideration payable 0 1,995 0 2,583
Restructuring charges 3,143 0 9,188 0
Loss on impairment of assets 0 0 0 1,134
Depreciation and amortization 2,170 2,228 6,506 5,691
Total operating expenses 106,579 110,578 331,577 331,791
Income (loss) from operations 21,659 (17,023) 8,905 (73,519)
Other expense:        
Interest income 1,081 1,471 3,991 5,407
Interest expense (12,692) (12,986) (37,640) (37,322)
Other (expense) income (3,263) 3,833 (11,946) (13,007)
Income (loss) before income tax 6,785 (24,705) (36,690) (118,441)
Income tax (expense) benefit (13,514) 3,128 (34,155) 700
Net loss attributable to common stockholders $ (6,729) $ (21,577) $ (70,845) $ (117,741)
Net loss attributable to common stockholders per common share - basic (in dollars per share) $ (0.02) $ (0.07) $ (0.23) $ (0.40)
Net loss attributable to common stockholders per common share — diluted (in dollars per share) $ (0.02) $ (0.07) $ (0.23) $ (0.40)
Weighted average common shares outstanding - basic (in shares) 304,690,596 295,759,435 303,792,479 293,314,167
Weighted average common shares outstanding - diluted (in shares) 304,690,596 295,759,435 303,792,479 293,314,167
v3.24.3
Consolidated Statements of Comprehensive Income (Loss) - USD ($)
$ in Thousands
 3 Months Ended 9 Months Ended
Sep. 30, 2024
Sep. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Statement of Comprehensive Income [Abstract]        
Net loss $ (6,729) $ (21,577) $ (70,845) $ (117,741)
Other comprehensive income (loss), net of tax:        
Foreign currency translation adjustment 16,168 (10,910) 19,730 5,416
Unrealized gain (loss) on available-for-sale securities 9 (18) 0 (79)
Other comprehensive income (loss) 16,177 (10,928) 19,730 5,337
Comprehensive income (loss) $ 9,448 $ (32,505) $ (51,115) $ (112,404)
v3.24.3
Consolidated Statements of Changes in Stockholders' Equity - USD ($)
$ in Thousands
Total
Common Stock
Additional Paid-In Capital
Warrants
Other Comprehensive Income (Loss)
Accumulated Deficit
Beginning balance (in shares) at Dec. 31, 2022   281,108,273        
Beginning balance at Dec. 31, 2022 $ 123,047 $ 2,815 $ 2,664,744 $ 83 $ (12,105) $ (2,532,490)
Increase (Decrease) in Stockholders' Equity            
Stock options exercised, net (in shares)   1,025,684        
Stock options exercised, net 7,847 $ 11 7,836      
Vesting of restricted stock units, net of taxes (in shares)   2,068,048        
Vesting of restricted stock units, net of taxes (16,355)   (16,355)      
Stock-based compensation 67,982   67,982      
Issuance of shares in connection with at-the-market offering, net of issuance costs (in shares)   5,244,936        
Issuance of shares in connection with at-the-market offering, net of issuance costs 63,108 $ 52 63,056      
Warrants exercised (in shares)   1,220,100        
Warrants exercised 12 $ 12 12 (12)    
Unrealized gain (loss) on available-for-sale securities (79)       (79)  
Foreign currency translation adjustment 5,416       5,416  
Net loss (117,741)         (117,741)
Ending balance (in shares) at Sep. 30, 2023   290,667,041        
Ending balance at Sep. 30, 2023 133,237 $ 2,890 2,787,275 71 (6,768) (2,650,231)
Beginning balance (in shares) at Jun. 30, 2023   286,992,923        
Beginning balance at Jun. 30, 2023 111,581 $ 2,856 2,733,148 71 4,160 (2,628,654)
Increase (Decrease) in Stockholders' Equity            
Stock options exercised, net (in shares)   372,467        
Stock options exercised, net 3,442 $ 4 3,438      
Vesting of restricted stock units, net of taxes (in shares)   299,297        
Vesting of restricted stock units, net of taxes (2,347)   (2,347)      
Stock-based compensation 16,511   16,511      
Issuance of shares in connection with at-the-market offering, net of issuance costs (in shares)   3,002,354        
Issuance of shares in connection with at-the-market offering, net of issuance costs 36,555 $ 30 36,525      
Unrealized gain (loss) on available-for-sale securities (18)       (18)  
Foreign currency translation adjustment (10,910)       (10,910)  
Net loss (21,577)         (21,577)
Ending balance (in shares) at Sep. 30, 2023   290,667,041        
Ending balance at Sep. 30, 2023 $ 133,237 $ 2,890 2,787,275 71 (6,768) (2,650,231)
Beginning balance (in shares) at Dec. 31, 2023 293,594,209 293,594,209        
Beginning balance at Dec. 31, 2023 $ 160,174 $ 2,918 2,836,018 71 5,241 (2,684,074)
Increase (Decrease) in Stockholders' Equity            
Stock options exercised, net (in shares) 791,000 796,202        
Stock options exercised, net $ 6,122 $ 7 6,115      
Vesting of restricted stock units, net of taxes (in shares)   2,608,392        
Vesting of restricted stock units, net of taxes (21,197)   (21,197)      
Stock-based compensation 65,688   65,688      
Issuance of shares in connection with at-the-market offering, net of issuance costs (in shares)   1,692,291        
Issuance of shares in connection with at-the-market offering, net of issuance costs 19,153 $ 17 19,136      
Unrealized gain (loss) on available-for-sale securities 0          
Foreign currency translation adjustment 19,730       19,730  
Net loss $ (70,845)         (70,845)
Ending balance (in shares) at Sep. 30, 2024 298,691,094 298,691,094        
Ending balance at Sep. 30, 2024 $ 178,825 $ 2,942 2,905,760 71 24,971 (2,754,919)
Beginning balance (in shares) at Jun. 30, 2024   296,428,877        
Beginning balance at Jun. 30, 2024 132,523 $ 2,923 2,868,925 71 8,794 (2,748,190)
Increase (Decrease) in Stockholders' Equity            
Stock options exercised, net (in shares)   215,653        
Stock options exercised, net 1,805 $ 2 1,803      
Vesting of restricted stock units, net of taxes (in shares)   354,273        
Vesting of restricted stock units, net of taxes (2,792)   (2,792)      
Stock-based compensation 18,688   18,688      
Issuance of shares in connection with at-the-market offering, net of issuance costs (in shares)   1,692,291        
Issuance of shares in connection with at-the-market offering, net of issuance costs 19,153 $ 17 19,136      
Unrealized gain (loss) on available-for-sale securities 9       9  
Foreign currency translation adjustment 16,168       16,168  
Net loss $ (6,729)         (6,729)
Ending balance (in shares) at Sep. 30, 2024 298,691,094 298,691,094        
Ending balance at Sep. 30, 2024 $ 178,825 $ 2,942 $ 2,905,760 $ 71 $ 24,971 $ (2,754,919)
v3.24.3
Consolidated Statements of Cash Flows - USD ($)
$ in Thousands
 9 Months Ended
Sep. 30, 2024
Sep. 30, 2023
Operating activities    
Net loss $ (70,845) $ (117,741)
Adjustments to reconcile net loss to net cash used in operating activities:    
Amortization of debt discount and deferred financing 1,665 2,080
Depreciation and amortization 6,506 5,691
Stock-based compensation 65,688 67,982
Non-cash changes in the fair value of contingent consideration payable 0 2,583
Foreign currency remeasurement loss 15,167 18,121
Deferred taxes 0 (4,939)
Other 7,048 2,360
Changes in operating assets and liabilities:    
Accounts receivable (9,090) (8,614)
Inventories (61,632) (42,233)
Prepaid expenses and other current assets 28,216 (26,010)
Accounts payable, accrued expenses, and other current liabilities (11,483) 41,101
Other non-current assets and liabilities (1,215) (4,993)
Payment of contingent consideration 0 (7,937)
Net cash used in operating activities (29,975) (72,549)
Investing activities    
Sale and redemption of marketable securities 101,875 180,828
Purchases of marketable securities (78,778) (53,098)
Capital expenditures (3,265) (5,709)
Net cash provided by investing activities 19,832 122,021
Financing activities    
Payment of finance leases (54) (82)
Withholding taxes paid on vested restricted stock units (21,197) (16,355)
Proceeds from stock options exercised, net 6,122 7,847
Proceeds from warrants exercised, net 0 12
Proceeds from the issuance of shares in connection with at-the-market offering, net of issuance costs 19,153 63,108
Payment of contingent consideration 0 (1,063)
Net cash provided by financing activities 4,024 53,467
Effect of exchange rate changes on cash, cash equivalents, and restricted cash (7,138) 10,218
Net (decrease) increase in cash, cash equivalents, and restricted cash at the end of the period (13,257) 113,157
Cash, cash equivalents, and restricted cash at the beginning of period 250,077 153,115
Cash, cash equivalents, and restricted cash at the end of period 236,820 266,272
Supplemental disclosures of cash flow information    
Cash paid during the period for interest 47,587 35,448
Cash paid for taxes 2,729 6,473
Tenant improvements paid through lease incentives 217 0
Capital expenditures unpaid at the end of period $ 168 $ 877
v3.24.3
Description of Business
 9 Months Ended
Sep. 30, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]  
Description of Business Description of Business
Amicus Therapeutics, Inc. (the "Company") is a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases. The Company seeks to deliver the highest quality therapies that have the potential to obsolete current treatments, provide significant benefits to patients, and be first- or best-in-class. The Company's two marketed therapies are Galafold®, the first oral monotherapy for people living with Fabry disease who have amenable genetic variants, and Pombiliti® + Opfolda®, a novel two-component treatment designed to improve uptake of active enzyme into key disease relevant tissues for adults living with late-onset Pompe disease.
Galafold® (also referred to as "migalastat"), is approved in over 40 countries around the world, including the United States ("U.S."), European Union ("E.U."), United Kingdom ("U.K."), and Japan. Additionally, Galafold® has been granted orphan drug status in the U.S., E.U., U.K., Japan and several other countries.
Pombiliti® + Opfolda® (also referred to as "cipaglucosidase alfa-atga/miglustat"), is approved in the U.S., the E.U., the U.K., and Switzerland. Multiple regulatory submissions and reimbursement processes with global health authorities are currently underway. Additionally, Pombiliti® + Opfolda® has been granted orphan drug designation or status in the U.S., U.K., Switzerland, and Japan and data exclusivity in the E.U.
The Company had an accumulated deficit of $2.8 billion as of September 30, 2024 and anticipates incurring losses through the fiscal year ending December 31, 2024. The Company has historically funded its operations through stock offerings, product revenues, debt issuances, collaborations, and other financing arrangements.
Based on its current operating model, which includes expected revenues, the Company believes the current cash position is sufficient to fund the Company's operations and ongoing research programs for at least the next 12 months. Potential business development opportunities, pipeline expansion, and investment in manufacturing capabilities could impact the Company's long-term capital requirements.
v3.24.3
Summary of Significant Accounting Policies
 9 Months Ended
Sep. 30, 2024
Accounting Policies [Abstract]  
Summary of Significant Accounting Policies Summary of Significant Accounting Policies
Basis of Presentation
The accompanying unaudited Consolidated Financial Statements have been prepared in accordance with the U.S. generally accepted accounting principles (“U.S. GAAP”) for interim financial information and with the instructions to Form 10-Q and Article 10-01 of Regulation S-X. Accordingly, certain financial information that is normally included in annual financial statements prepared in accordance with U.S. GAAP, but that is not required for interim reporting purposes, has been omitted. In the opinion of management, the accompanying unaudited Consolidated Financial Statements reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company's interim financial information. Management has determined that the Company operates in one segment focused on the discovery, development, and commercialization of advanced therapies to treat a range of devastating rare and orphan diseases.
The accompanying unaudited Consolidated Financial Statements and related notes should be read in conjunction with the Company's financial statements and related notes as contained in the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2023. For a complete description of the Company's accounting policies, please refer to the Annual Report on Form 10-K for the fiscal year ended December 31, 2023.
Consolidation
The Consolidated Financial Statements include the accounts of the Company and its subsidiaries. Intercompany accounts and transactions are eliminated in consolidation.
Foreign Currency Transactions
The functional currency for most of the Company's foreign subsidiaries is their local currency. For non-U.S. subsidiaries that transact in a functional currency other than the U.S. dollar, assets and liabilities are translated at current rates of exchange at the balance sheet date. Income and expense items are translated at the weighted average foreign exchange rates for the
period. Adjustments resulting from the translation of the financial statements of the Company's foreign operations into U.S. dollars are excluded from the determination of net income and are recorded in accumulated other comprehensive income, a separate component of stockholders' equity. Transactions which are not in the functional currency of the entity are remeasured into the functional currency with gains or losses resulting from the remeasurement recorded in other expense.
Use of Estimates
The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates.
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash
The Company considers all highly liquid investments purchased with a maturity of three months or less at the date of acquisition to be cash equivalents. Marketable securities consist of fixed income investments with a maturity of greater than three months and other highly liquid investments that can be readily purchased or sold using established markets. These investments are classified as available-for-sale and are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are reported within other comprehensive income (loss) in the Company's Consolidated Statements of Comprehensive Income (Loss). Fair value is based on available market information including quoted market prices, broker or dealer quotations, or other observable inputs.
Restricted cash consists primarily of funds held to satisfy the requirements of certain agreements that are restricted in their use and is included as a component of other non-current assets on the Company's Consolidated Balance Sheets.
Concentration of Credit Risk
The Company's financial instruments that are exposed to concentration of credit risk consist primarily of cash, cash equivalents, 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 invests its marketable securities in high-quality commercial financial instruments. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on its cash, cash equivalents, or marketable securities.
The Company's accounts receivable at September 30, 2024 have primarily arisen from Galafold® sales in Europe, the U.S., and Japan. The Company periodically assesses the financial strength of its customers to establish allowances for anticipated losses, if any. For accounts receivable that have arisen from named patient sales, the payment terms are predetermined, and the Company evaluates the creditworthiness of each customer on a regular basis. As of September 30, 2024, the Company's allowance for doubtful accounts was $0.5 million.
Revenue Recognition
The Company has recorded revenue on sales where its products are available either on a commercial basis or through a reimbursed early access program. Product orders are generally received from distributors and pharmacies.
The Company recognizes revenue when its performance obligation to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of the products. The transaction price is determined based on fixed consideration in the Company's customer contracts and is recorded net of estimates for variable consideration, which primarily consist of third-party discounts and rebates. The identified variable consideration is recorded as a reduction of revenue at the time revenue from the sale is recognized. The Company recognizes revenue to the extent that it is probable that a significant revenue reversal will not occur in a future period. These estimates may differ from actual consideration received. The Company evaluates these estimates each reporting period to reflect known changes.
The following table summarizes the Company's net product sales disaggregated by product:
Three Months Ended September 30,Nine Months Ended September 30,
(in thousands)2024202320242023
Galafold®
$120,381 $100,733 $330,557 $281,177 
Pombiliti® + Opfolda®
21,136 2,768 48,032 3,097 
Total net product sales$141,517 $103,501 $378,589 $284,274 
The following table summarizes the Company's net product sales disaggregated by geographic area:
Three Months Ended September 30,Nine Months Ended September 30,
(in thousands)2024202320242023
U.S.$56,192 $37,801 $143,705 $103,760 
Ex-U.S.85,325 65,700 234,884 180,514 
Total net product sales$141,517 $103,501 $378,589 $284,274 
Inventories and Cost of Goods Sold
Inventories are stated at the lower of cost and net realizable value, determined by the first-in, first-out method. Inventories are reviewed periodically to identify slow-moving or obsolete inventory based on projected sales activity as well as product shelf-life. In evaluating the recoverability of inventories produced, the probability that revenue will be obtained from the future sale of the related inventory is considered and inventory value is written down for inventory quantities in excess of expected requirements. Expired inventory is disposed of and the related costs are recognized as cost of goods sold in the Company's Consolidated Statements of Operations.
Cost of goods sold includes the cost of inventory sold, manufacturing and supply chain costs, product shipping and handling costs, provisions for excess and obsolete inventory, as well as royalties payable. A portion of Pombiliti® + Opfolda® inventory was expensed as research and development costs prior to regulatory approval and as such, the cost of goods sold and related gross margins are not necessarily indicative of future costs of goods sold and gross margin.
Recent Accounting Developments - Guidance Adopted in 2024
In November 2023, the Financial Accounting Standards Board ("FASB") issued the Accounting Standards Update ("ASU") No. 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures. The amendments expand reportable segment disclosure requirements, primarily through enhanced disclosures about significant segment expenses. The amendments require, among other things, disclosure of the title and position of the chief operating decision maker and require that public entities with a single reportable segment provide all disclosures required by this update and existing segment disclosures in Topic 280. Annual disclosures are required for fiscal years beginning after December 15, 2023 and interim disclosures are required for periods within fiscal years beginning after December 15, 2024. Retrospective application is required unless it is impracticable, and early adoption is permitted. The Company adopted this guidance on January 1, 2024. This ASU applies to disclosure requirements only, and the Company will provide required annual disclosures as part of the 2024 Annual Report on Form 10-K and required interim disclosures as part of 2025 Quarterly Reports on Form 10-Q.
Recent Accounting Developments - Guidance Not Yet Adopted
In December 2023, the FASB issued ASU No. 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures. The ASU requires disclosure of disaggregated income taxes paid, prescribes standard categories for the components of the effective tax rate reconciliation, and modifies other income tax-related disclosures. ASU 2023-09 is effective for fiscal years beginning after December 15, 2024, must be applied prospectively with an option to apply retrospectively, and early adoption is permitted. The adoption of this guidance is not expected to have a significant impact on the Company's consolidated financial statements.
v3.24.3
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash
 9 Months Ended
Sep. 30, 2024
Cash, Cash Equivalents, and Short-Term Investments [Abstract]  
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash Cash, Cash Equivalents, Marketable Securities, and Restricted Cash
As of September 30, 2024, the Company held $233.6 million in cash and cash equivalents and $16.1 million of marketable securities which are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are generally reported within other comprehensive income (loss) in the Consolidated Statements of Comprehensive Income (Loss). If a decline in the fair value of a marketable security below the Company's cost basis is determined to be other-than-temporary or if an available-for-sale debt security’s fair value is determined to be less than the amortized cost and the Company intends or is more than likely to sell the security before recovery and it is not considered a credit loss, such security is written down to its estimated fair value as a new cost basis and the amount of the write-down is included in the Consolidated Statements of Operations as an impairment charge. If the unrealized loss of an available-for-sale debt security is determined to be a result of credit loss, the Company would recognize an allowance and the corresponding credit loss would be included in the Consolidated Statements of Operations.
The Company regularly invests excess operating cash in deposits with major financial institutions, money market funds, notes issued by the U.S. government, as well as fixed income investments and U.S. bond funds, both of which can be readily purchased and sold using established markets. The Company believes that the market risk arising from its holdings of these financial instruments is mitigated as, in accordance with Company policy, such securities are of high credit rating. Investments that have original maturities greater than three months but less than one year are classified as current.
Cash, cash equivalents and marketable securities are classified as current unless mentioned otherwise below and consisted of the following:
 As of September 30, 2024
(in thousands)CostGross
Unrealized
Gain		Gross
Unrealized
Loss		Fair
Value
Cash and cash equivalents$233,647 $— $— $233,647 
Commercial paper5,991 — — 5,991 
Treasury bill9,958 10 — 9,968 
U.S. government agency bonds— — — — 
Money market100 — — 100 
Certificates of deposit51 — — 51 
$249,747 $10 $— $249,757 
Included in cash and cash equivalents$233,647 $— $— $233,647 
Included in marketable securities16,100 10 — 16,110 
Total cash, cash equivalents, and marketable securities$249,747 $10 $— $249,757 

 As of December 31, 2023
(in thousands)CostGross
Unrealized
Gain		Gross
Unrealized
Loss		Fair
Value
Cash and cash equivalents$246,994 $— $— $246,994 
Commercial paper14,651 12 — 14,663 
Treasury bill12,944 2 — 12,946 
U.S. government agency bonds11,450 — (4)11,446 
Money market100 — — 100 
Certificate of deposit51 — — 51 
$286,190 $14 $(4)$286,200 
Included in cash and cash equivalents$246,994 $— $— $246,994 
Included in marketable securities39,196 14 (4)39,206
Total cash, cash equivalents, and marketable securities$286,190 $14 $(4)$286,200 
For both the nine months ended September 30, 2024 and September 30, 2023, there were no realized gains or losses. The cost of securities sold is based on the specific identification method.
There were no unrealized loss positions as of September 30, 2024. Unrealized loss positions in the marketable securities as of December 31, 2023 reflect temporary impairments and are not a result of credit loss. Additionally, as these positions have been in a loss position for less than twelve months and the Company does not intend to sell these securities before recovery, the losses are recognized as a component of other comprehensive income (loss). The fair value of these marketable securities in unrealized loss positions are $0.0 million and $11.4 million as of September 30, 2024 and December 31, 2023, respectively.
The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Consolidated Balance Sheets that sum to the total shown in the Consolidated Statements of Cash Flows.
As of September 30,
(in thousands)20242023
Cash and cash equivalents$233,647 $263,320 
Restricted cash3,173 2,952 
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows$236,820 $266,272 
v3.24.3
Inventories
 9 Months Ended
Sep. 30, 2024
Inventory Disclosure [Abstract]  
Inventories Inventories
The following table summarizes the components of the Company’s inventories for each of the periods indicated:
(in thousands)September 30, 2024December 31, 2023
Raw materials$76,203 $30,230 
Work-in-process31,485 22,597 
Finished goods7,650 6,869 
Total inventories$115,338 $59,696 
The Company's reserve for inventory was $3.5 million and $0.5 million as of September 30, 2024 and December 31, 2023, respectively.
v3.24.3
Debt
 9 Months Ended
Sep. 30, 2024
Debt Disclosure [Abstract]  
Debt Debt
The following table summarizes the Company's debt for each of the periods indicated:
(in thousands)September 30, 2024December 31, 2023
Senior Secured Term Loan due 2029:
Principal$400,000 $400,000 
Less: debt discount (1)
(8,339)(9,652)
Less: deferred financing (1)
(2,167)(2,490)
Net carrying value of long-term debt$389,494 $387,858 
______________________________
(1) Included in the Company's Consolidated Balance Sheets within long-term debt and amortized to interest expense over the remaining life of the Senior Secured Term Loan due 2029 using the effective interest rate method.
Interest Expense
The following table sets forth interest expense recognized related to the Company's debt for the three and nine months ended September 30, 2024 and 2023, respectively:
Three Months Ended September 30,Nine Months Ended September 30,
(in thousands)2024202320242023
Contractual interest expense$12,106 $12,270 $36,021 $35,289 
Amortization of debt discount$455 $411 $1,313 $1,187 
Amortization of deferred financing$119 $310 $352 $893 
v3.24.3
Restructuring
 9 Months Ended
Sep. 30, 2024
Restructuring and Related Activities [Abstract]  
Restructuring Restructuring
The restructuring liabilities are recorded in accrued expenses and other current liabilities in the Company’s Consolidated Balance Sheets. Total charges incurred are summarized as follows:
(in thousands)Facility and Non-Lease CostsOther CostsTotal
Balance as of December 31, 2023
$— $— $— 
Restructuring charges6,045 3,143 9,188 
Non-cash items(2,844)— (2,844)
Cash settled(981)(2,136)(3,117)
Balance as of September 30, 2024
$2,220 $1,007 $3,227 
v3.24.3
Stockholders' Equity
 9 Months Ended
Sep. 30, 2024
Equity [Abstract]  
Stockholders' Equity Stockholders' Equity
During the three and nine months ended September 30, 2024, the Company issued and sold an aggregate of 1,692,291 shares through its at-the-market equity program ("ATM program") at weighted-average public offering price of $11.70 per share, resulting in net proceeds of $19.2 million. As of September 30, 2024, an aggregate of $164.7 million worth of shares remain available to be issued and sold under the ATM program.
v3.24.3
Stock-Based Compensation
 9 Months Ended
Sep. 30, 2024
Share-Based Payment Arrangement [Abstract]  
Stock-Based Compensation Stock-Based Compensation
The Amended and Restated 2007 Equity Incentive Plan (the "Plan") provides for the granting of restricted stock units and options to purchase common stock in the Company to employees, directors, advisors, and consultants at a price to be determined by the Board of Directors. The Plan is intended to encourage ownership of stock by employees and consultants of the Company and to provide additional incentives for them to promote the success of the business. The Board of Directors, or its committee, is responsible for determining the individuals to be granted options, the number of options each individual will receive, the option price per share, and the exercise period of each option.
Stock Option Grants
The fair value of the stock options granted were estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions:
 Three Months Ended September 30,Nine Months Ended September 30,
 2024202320242023
Expected stock price volatility55.5 %58.4 %57.1 %59.2 %
Risk free interest rate3.9 %4.3 %4.0 %3.9 %
Expected life of options (years)5.65.55.65.5
Expected annual dividend per share$— $— $— $— 
 A summary of the Company's stock options for the nine months ended September 30, 2024 were as follows:
Number of
Shares		Weighted Average Exercise 
Price		Weighted Average Remaining
Years		Aggregate
Intrinsic
Value
 (in thousands)  (in millions)
Options outstanding, December 31, 202323,002 $11.69   
Granted4,693 $13.69   
Exercised(791)$7.74   
Forfeited(548)$12.74   
Expired(217)$14.58 
Options outstanding, September 30, 202426,139 $12.12 6.0$13.6 
Vested and non-vested expected to vest, September 30, 202424,610 $12.09 5.9$13.5 
Exercisable at September 30, 202417,092 $11.75 4.7$13.0 
As of September 30, 2024, the total unrecognized compensation cost related to non-vested stock options granted was $43.4 million and is expected to be recognized over a weighted average period of three years.
Restricted Stock Units and Performance-Based Restricted Stock Units (collectively "RSUs")
RSUs awarded under the Plan are generally subject to graded vesting and are contingent on an employee's continued service. RSUs are generally subject to forfeiture if employment terminates prior to the release of vesting restrictions. The Company expenses the cost of the RSUs, which is determined to be the fair market value of the shares of common stock underlying the RSUs at the date of grant, ratably over the period during which the vesting restrictions lapse. A summary of non-vested RSU activity under the Plan for the nine months ended September 30, 2024 is as follows:
Number of
Shares		Weighted
Average Grant
Date Fair
Value		Weighted 
Average
Remaining 
Years		Aggregate
Intrinsic
Value
(in thousands)(in millions)
Non-vested units as of December 31, 202310,033 $13.37   
Granted3,893 $14.63   
Vested(3,860)$13.17   
Forfeited(613)$14.28   
Non-vested units as of September 30, 20249,453 $13.82 2.2$101.0 
As of September 30, 2024, there was $62.8 million of total unrecognized compensation cost related to non-vested RSUs with service-based vesting conditions. These costs are expected to be recognized over a weighted average period of two years.
Compensation Expense Related to Equity Awards
The following table summarizes information related to compensation expense recognized in the Company's Consolidated Statements of Operations related to the equity awards:
 Three Months Ended September 30,Nine Months Ended September 30,
(in thousands)2024202320242023
Research and development expense$4,397 $4,380 $12,329 $16,987 
Selling, general, and administrative expense14,291 12,131 53,359 50,995 
Total equity compensation expense$18,688 $16,511 $65,688 $67,982 
v3.24.3
Assets and Liabilities Measured at Fair Value
 9 Months Ended
Sep. 30, 2024
Fair Value Disclosures [Abstract]  
Assets and Liabilities Measured at Fair Value Assets and Liabilities Measured at Fair Value
The Company's financial assets and liabilities are measured at fair value and classified within the fair value hierarchy, which is defined as follows:
Level 1 — Quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date.
Level 2 — Inputs other than quoted prices in active markets that are observable for the asset or liability, either directly or indirectly.
Level 3 — Inputs that are unobservable for the asset or liability.
A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of September 30, 2024 are identified in the following tables:
(in thousands) Level 1Level 2Total
Assets:  
Commercial paper$— $5,991 $5,991 
Treasury bill— 9,968 9,968 
Money market7,922 — 7,922 
 $7,922 $15,959 $23,881 
(in thousands) Level 1Level 2Total
Liabilities:  
Deferred compensation plan liability$7,822 $— $7,822 
 $7,822 $— $7,822 
A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2023 are identified in the following tables:
(in thousands)Level 1Level 2Total
Assets:
Commercial paper$— $14,663 $14,663 
Treasury bill— 12,946 12,946 
U.S. government agency bonds— 11,446 11,446 
Money market7,631 — 7,631 
 $7,631 $39,055 $46,686 
(in thousands)Level 1Level 2Total
Liabilities:   
Deferred compensation plan liability7,531 — 7,531 
 $7,531 $— $7,531 
Deferred compensation plan liability is recorded as a component of other non-current liabilities on the Company's Consolidated Balance Sheets. The Company did not have any Level 3 assets or liabilities as of September 30, 2024 or December 31, 2023.
Cash, Money Market Funds, and Marketable Securities
The Company classifies its cash and money market funds within the fair value hierarchy as Level 1 as these assets are valued using quoted prices in an active market for identical assets at the measurement date. The Company considers its investments in marketable securities as available-for-sale and classifies these assets within the fair value hierarchy as Level 2 primarily utilizing broker quotes in a non-active market for valuation of these securities.
v3.24.3
Basic and Diluted Net Loss per Common Share
 9 Months Ended
Sep. 30, 2024
Earnings Per Share [Abstract]  
Basic and Diluted Net Loss per Common Share Basic and Diluted Net Loss per Common Share
The following table provides a reconciliation of the numerator and denominator used in computing basic and diluted net loss attributable to common stockholders per common share:
 Three Months Ended September 30,Nine Months Ended September 30,
(in thousands, except per share amounts) 2024202320242023
Numerator:  
Net loss attributable to common stockholders$(6,729)$(21,577)$(70,845)$(117,741)
Denominator:
Weighted average common shares outstanding — basic and diluted304,690,596 295,759,435 303,792,479 293,314,167 
Dilutive common stock equivalents would include the dilutive effect of outstanding common stock options and non-vested RSUs. Potentially dilutive common stock equivalents were excluded from the diluted earnings per share denominator for all periods because of their anti-dilutive effect. Weighted average common shares outstanding includes outstanding pre-funded warrants with an exercise price of $0.01.
The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method:
 As of September 30,
(in thousands) 20242023
Options to purchase common stock26,139 23,256 
Non-vested restricted stock units9,453 10,331 
Total number of potentially issuable shares35,592 33,587 
v3.24.3
Pay vs Performance Disclosure - USD ($)
$ in Thousands
 3 Months Ended 9 Months Ended
Sep. 30, 2024
Sep. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Pay vs Performance Disclosure        
Net loss $ (6,729) $ (21,577) $ (70,845) $ (117,741)
v3.24.3
Insider Trading Arrangements
 3 Months Ended 9 Months Ended
Sep. 30, 2024
shares
Sep. 30, 2024
shares
Trading Arrangements, by Individual    
Material Terms of Trading Arrangement  
The following table describes, for the quarterly period covered by this report, each director and officer (as defined in Rule 16a-1(f) under the Exchange Act who has adopted, modified, or terminated a trading plan intended to satisfy the affirmative defense of Rule 10b5-1(c) under the Exchange Act (each plan, a “Rule 10b5-1 Trading Plan”). Each Rule 10b5-1 Trading Plan described below was adopted during an open insider trading window and in accordance with the Company’s policies regarding both insider trading and transactions relating to Company securities.
Name
(Title)		Action Taken
(Date of Action)		Rule 10b5-1 Trading Plan Provides for Purchase/Sale
Duration of the Trading Plan(1)
Aggregate Number of Securities
Bradley Campbell
(President and Chief Executive Officer)
Adoption
(September 13, 2024)
SaleDecember 31, 2025
235,460 (2)
Jeffrey Castelli
(Chief Development Officer)
Adoption
(September 10, 2024)
SaleMay 31, 2025
132,954 (3)
David Clark
(Chief People Officer)
Adoption
(August 28, 2024)
SaleAugust 15, 2025
Indeterminable (4)
Samantha Prout
(Chief Accounting Officer)
Adoption
(September 12, 2024)
SaleJuly 1, 2025
Indeterminable (5)
Ellen Rosenberg
(Chief Legal Officer and Corporate Secretary)
Adoption
(September 10, 2024)
SaleDecember 31, 2025
95,621 (6)
(1) The dates in this column represent the scheduled expiration date of each director or officer’s Rule 10b5-1 Trading Plan. Each Rule 10b5-1 Trading Plan may terminate earlier than the date provided should all transactions contemplated thereunder occur prior to such date.
(2) Mr. Campbell’s Rule 10b5-1 Trading Plan provides for the (i) sale of 60,460 shares of common stock and (ii) exercise of up to 175,000 stock options and subsequent sale of up to 175,000 shares of common stock underlying such stock options.
(3) Mr. Castelli’s Rule 10b5-1 Trading Plan provides for the (i) sale of 82,954 shares of common stock and (ii) exercise of up to 50,000 stock options and subsequent sale of up to 50,000 shares of common stock underlying such stock options.
(4) Mr. Clark’s Rule 10b5-1 Trading Plan provides for the sale of common stock that will be obtained from the settlement of his 2022 PRSU awards and the respective vesting of his 2021, 2022, 2023 and 2024 annual restricted stock unit awards. The number of shares of common stock obtained and available for sale will be subject to the (i) level of achievement of each performance goal contained within the 2022 PRSU awards and (ii) shares of common stock withheld to satisfy applicable tax withholding obligations.
(5) Ms. Prout’s Rule 10b5-1 Trading Plan provides for the (i) exercise of up to 66,074 stock options and the sale of up to 66,074 underlying shares of common stock, (ii) sale of 7,243 shares of common stock and (iii) sale of an indeterminable number of shares of common stock to be obtained from the respective vesting of her 2021, 2022, 2023 and 2024 annual restricted stock unit awards. The number of shares of common stock obtained and available for sale in clause (iii) will be subject to the shares of common stock withheld to satisfy applicable tax withholding obligations.
(6) Ms. Rosenberg’s Rule 10b5-1 Trading Plan provides for the exercise of up to 95,621 stock options and the sale of up to 95,621 underlying shares of common stock.
Non-Rule 10b5-1 Arrangement Adopted false  
Rule 10b5-1 Arrangement Terminated false  
Non-Rule 10b5-1 Arrangement Terminated false  
Bradley Campbell [Member]    
Trading Arrangements, by Individual    
Name Bradley Campbell  
Title President and Chief Executive Officer  
Rule 10b5-1 Arrangement Adopted true  
Adoption Date September 13, 2024  
Arrangement Duration 474 days  
Aggregate Available 235,460 235,460
Jeffrey Castelli [Member]    
Trading Arrangements, by Individual    
Name Jeffrey Castelli  
Title Chief Development Officer  
Rule 10b5-1 Arrangement Adopted true  
Adoption Date September 10, 2024  
Arrangement Duration 263 days  
Aggregate Available 132,954 132,954
David Clark [Member]    
Trading Arrangements, by Individual    
Name David Clark  
Title Chief People Officer  
Rule 10b5-1 Arrangement Adopted true  
Adoption Date August 28, 2024  
Arrangement Duration 352 days  
Samantha Prout [Member]    
Trading Arrangements, by Individual    
Name Samantha Prout  
Title Chief Accounting Officer  
Rule 10b5-1 Arrangement Adopted true  
Adoption Date September 12, 2024  
Arrangement Duration 292 days  
Ellen Rosenberg [Member]    
Trading Arrangements, by Individual    
Name Ellen Rosenberg  
Title Chief Legal Officer and Corporate Secretary  
Rule 10b5-1 Arrangement Adopted true  
Adoption Date September 10, 2024  
Arrangement Duration 477 days  
Aggregate Available 95,621 95,621
Bradley Campbell Trading Arrangement, Common Stock [Member] | Bradley Campbell [Member]    
Trading Arrangements, by Individual    
Aggregate Available 60,460 60,460
Bradley Campbell Trading Arrangement, Stock Options [Member] | Bradley Campbell [Member]    
Trading Arrangements, by Individual    
Aggregate Available 175,000 175,000
Bradley Campbell Trading Arrangement, Common Stock Underlying Stock Options [Member] | Bradley Campbell [Member]    
Trading Arrangements, by Individual    
Aggregate Available 175,000 175,000
Jeffrey Castelli Trading Arrangement, Common Stock [Member] | Jeffrey Castelli [Member]    
Trading Arrangements, by Individual    
Aggregate Available 82,954 82,954
Jeffrey Castelli Trading Arrangement, Stock Options [Member] | Jeffrey Castelli [Member]    
Trading Arrangements, by Individual    
Aggregate Available 50,000 50,000
Jeffrey Castelli Trading Arrangement, Common Stock Underlying Stock Options [Member] | Jeffrey Castelli [Member]    
Trading Arrangements, by Individual    
Aggregate Available 50,000 50,000
Samantha Prout Trading Arrangement, Stock Options [Member] | Samantha Prout [Member]    
Trading Arrangements, by Individual    
Aggregate Available 66,074 66,074
Samantha Prout Trading Arrangement, Common Stock Underlying Stock Options [Member] | Samantha Prout [Member]    
Trading Arrangements, by Individual    
Aggregate Available 66,074 66,074
Samantha Prout Trading Arrangement, Common Stock [Member] | Samantha Prout [Member]    
Trading Arrangements, by Individual    
Aggregate Available 7,243 7,243
Ellen Rosenberg Trading Arrangement, Stock Options [Member] | Ellen Rosenberg [Member]    
Trading Arrangements, by Individual    
Aggregate Available 95,621 95,621
Ellen Rosenberg Trading Arrangement, Common Stock Underlying Stock Options [Member] | Ellen Rosenberg [Member]    
Trading Arrangements, by Individual    
Aggregate Available 95,621 95,621
v3.24.3
Summary of Significant Accounting Policies (Policies)
 9 Months Ended
Sep. 30, 2024
Accounting Policies [Abstract]  
Basis of Presentation
Basis of Presentation
The accompanying unaudited Consolidated Financial Statements have been prepared in accordance with the U.S. generally accepted accounting principles (“U.S. GAAP”) for interim financial information and with the instructions to Form 10-Q and Article 10-01 of Regulation S-X. Accordingly, certain financial information that is normally included in annual financial statements prepared in accordance with U.S. GAAP, but that is not required for interim reporting purposes, has been omitted. In the opinion of management, the accompanying unaudited Consolidated Financial Statements reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company's interim financial information. Management has determined that the Company operates in one segment focused on the discovery, development, and commercialization of advanced therapies to treat a range of devastating rare and orphan diseases.
The accompanying unaudited Consolidated Financial Statements and related notes should be read in conjunction with the Company's financial statements and related notes as contained in the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2023. For a complete description of the Company's accounting policies, please refer to the Annual Report on Form 10-K for the fiscal year ended December 31, 2023.
Consolidation
Consolidation
The Consolidated Financial Statements include the accounts of the Company and its subsidiaries. Intercompany accounts and transactions are eliminated in consolidation.
Foreign Currency Transactions
Foreign Currency Transactions
The functional currency for most of the Company's foreign subsidiaries is their local currency. For non-U.S. subsidiaries that transact in a functional currency other than the U.S. dollar, assets and liabilities are translated at current rates of exchange at the balance sheet date. Income and expense items are translated at the weighted average foreign exchange rates for the
period. Adjustments resulting from the translation of the financial statements of the Company's foreign operations into U.S. dollars are excluded from the determination of net income and are recorded in accumulated other comprehensive income, a separate component of stockholders' equity. Transactions which are not in the functional currency of the entity are remeasured into the functional currency with gains or losses resulting from the remeasurement recorded in other expense.
Use of Estimates
Use of Estimates
The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates.
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash
The Company considers all highly liquid investments purchased with a maturity of three months or less at the date of acquisition to be cash equivalents. Marketable securities consist of fixed income investments with a maturity of greater than three months and other highly liquid investments that can be readily purchased or sold using established markets. These investments are classified as available-for-sale and are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are reported within other comprehensive income (loss) in the Company's Consolidated Statements of Comprehensive Income (Loss). Fair value is based on available market information including quoted market prices, broker or dealer quotations, or other observable inputs.
Restricted cash consists primarily of funds held to satisfy the requirements of certain agreements that are restricted in their use and is included as a component of other non-current assets on the Company's Consolidated Balance Sheets.
Concentration of Credit Risk
Concentration of Credit Risk
The Company's financial instruments that are exposed to concentration of credit risk consist primarily of cash, cash equivalents, 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 invests its marketable securities in high-quality commercial financial instruments. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on its cash, cash equivalents, or marketable securities.
The Company's accounts receivable at September 30, 2024 have primarily arisen from Galafold® sales in Europe, the U.S., and Japan. The Company periodically assesses the financial strength of its customers to establish allowances for anticipated losses, if any. For accounts receivable that have arisen from named patient sales, the payment terms are predetermined, and the Company evaluates the creditworthiness of each customer on a regular basis.
Revenue Recognition
Revenue Recognition
The Company has recorded revenue on sales where its products are available either on a commercial basis or through a reimbursed early access program. Product orders are generally received from distributors and pharmacies.
The Company recognizes revenue when its performance obligation to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of the products. The transaction price is determined based on fixed consideration in the Company's customer contracts and is recorded net of estimates for variable consideration, which primarily consist of third-party discounts and rebates. The identified variable consideration is recorded as a reduction of revenue at the time revenue from the sale is recognized. The Company recognizes revenue to the extent that it is probable that a significant revenue reversal will not occur in a future period. These estimates may differ from actual consideration received. The Company evaluates these estimates each reporting period to reflect known changes.
Inventories and Cost of Goods Sold
Inventories and Cost of Goods Sold
Inventories are stated at the lower of cost and net realizable value, determined by the first-in, first-out method. Inventories are reviewed periodically to identify slow-moving or obsolete inventory based on projected sales activity as well as product shelf-life. In evaluating the recoverability of inventories produced, the probability that revenue will be obtained from the future sale of the related inventory is considered and inventory value is written down for inventory quantities in excess of expected requirements. Expired inventory is disposed of and the related costs are recognized as cost of goods sold in the Company's Consolidated Statements of Operations.
Cost of goods sold includes the cost of inventory sold, manufacturing and supply chain costs, product shipping and handling costs, provisions for excess and obsolete inventory, as well as royalties payable. A portion of Pombiliti® + Opfolda® inventory was expensed as research and development costs prior to regulatory approval and as such, the cost of goods sold and related gross margins are not necessarily indicative of future costs of goods sold and gross margin.
Recent Accounting Developments
Recent Accounting Developments - Guidance Adopted in 2024
In November 2023, the Financial Accounting Standards Board ("FASB") issued the Accounting Standards Update ("ASU") No. 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures. The amendments expand reportable segment disclosure requirements, primarily through enhanced disclosures about significant segment expenses. The amendments require, among other things, disclosure of the title and position of the chief operating decision maker and require that public entities with a single reportable segment provide all disclosures required by this update and existing segment disclosures in Topic 280. Annual disclosures are required for fiscal years beginning after December 15, 2023 and interim disclosures are required for periods within fiscal years beginning after December 15, 2024. Retrospective application is required unless it is impracticable, and early adoption is permitted. The Company adopted this guidance on January 1, 2024. This ASU applies to disclosure requirements only, and the Company will provide required annual disclosures as part of the 2024 Annual Report on Form 10-K and required interim disclosures as part of 2025 Quarterly Reports on Form 10-Q.
Recent Accounting Developments - Guidance Not Yet Adopted
In December 2023, the FASB issued ASU No. 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures. The ASU requires disclosure of disaggregated income taxes paid, prescribes standard categories for the components of the effective tax rate reconciliation, and modifies other income tax-related disclosures. ASU 2023-09 is effective for fiscal years beginning after December 15, 2024, must be applied prospectively with an option to apply retrospectively, and early adoption is permitted. The adoption of this guidance is not expected to have a significant impact on the Company's consolidated financial statements.
v3.24.3
Summary of Significant Accounting Policies (Tables)
 9 Months Ended
Sep. 30, 2024
Accounting Policies [Abstract]  
Schedule of Revenue Disaggregated by Geographical Area
The following table summarizes the Company's net product sales disaggregated by product:
Three Months Ended September 30,Nine Months Ended September 30,
(in thousands)2024202320242023
Galafold®
$120,381 $100,733 $330,557 $281,177 
Pombiliti® + Opfolda®
21,136 2,768 48,032 3,097 
Total net product sales$141,517 $103,501 $378,589 $284,274 
The following table summarizes the Company's net product sales disaggregated by geographic area:
Three Months Ended September 30,Nine Months Ended September 30,
(in thousands)2024202320242023
U.S.$56,192 $37,801 $143,705 $103,760 
Ex-U.S.85,325 65,700 234,884 180,514 
Total net product sales$141,517 $103,501 $378,589 $284,274 
v3.24.3
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash (Tables)
 9 Months Ended
Sep. 30, 2024
Cash, Cash Equivalents, and Short-Term Investments [Abstract]  
Schedule of Cash, Cash Equivalents and Marketable Securities
Cash, cash equivalents and marketable securities are classified as current unless mentioned otherwise below and consisted of the following:
 As of September 30, 2024
(in thousands)CostGross
Unrealized
Gain		Gross
Unrealized
Loss		Fair
Value
Cash and cash equivalents$233,647 $— $— $233,647 
Commercial paper5,991 — — 5,991 
Treasury bill9,958 10 — 9,968 
U.S. government agency bonds— — — — 
Money market100 — — 100 
Certificates of deposit51 — — 51 
$249,747 $10 $— $249,757 
Included in cash and cash equivalents$233,647 $— $— $233,647 
Included in marketable securities16,100 10 — 16,110 
Total cash, cash equivalents, and marketable securities$249,747 $10 $— $249,757 

 As of December 31, 2023
(in thousands)CostGross
Unrealized
Gain		Gross
Unrealized
Loss		Fair
Value
Cash and cash equivalents$246,994 $— $— $246,994 
Commercial paper14,651 12 — 14,663 
Treasury bill12,944 2 — 12,946 
U.S. government agency bonds11,450 — (4)11,446 
Money market100 — — 100 
Certificate of deposit51 — — 51 
$286,190 $14 $(4)$286,200 
Included in cash and cash equivalents$246,994 $— $— $246,994 
Included in marketable securities39,196 14 (4)39,206
Total cash, cash equivalents, and marketable securities$286,190 $14 $(4)$286,200 
Schedule of Cash, Cash Equivalents, and Restricted Cash
The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Consolidated Balance Sheets that sum to the total shown in the Consolidated Statements of Cash Flows.
As of September 30,
(in thousands)20242023
Cash and cash equivalents$233,647 $263,320 
Restricted cash3,173 2,952 
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows$236,820 $266,272 
v3.24.3
Inventories (Tables)
 9 Months Ended
Sep. 30, 2024
Inventory Disclosure [Abstract]  
Schedule of Inventories for the Period
The following table summarizes the components of the Company’s inventories for each of the periods indicated:
(in thousands)September 30, 2024December 31, 2023
Raw materials$76,203 $30,230 
Work-in-process31,485 22,597 
Finished goods7,650 6,869 
Total inventories$115,338 $59,696 
v3.24.3
Debt (Tables)
 9 Months Ended
Sep. 30, 2024
Debt Disclosure [Abstract]  
Schedule of Liability Components of Long-Term Debt
The following table summarizes the Company's debt for each of the periods indicated:
(in thousands)September 30, 2024December 31, 2023
Senior Secured Term Loan due 2029:
Principal$400,000 $400,000 
Less: debt discount (1)
(8,339)(9,652)
Less: deferred financing (1)
(2,167)(2,490)
Net carrying value of long-term debt$389,494 $387,858 
______________________________
(1) Included in the Company's Consolidated Balance Sheets within long-term debt and amortized to interest expense over the remaining life of the Senior Secured Term Loan due 2029 using the effective interest rate method.
Schedule of Components of Total Interest Expense
The following table sets forth interest expense recognized related to the Company's debt for the three and nine months ended September 30, 2024 and 2023, respectively:
Three Months Ended September 30,Nine Months Ended September 30,
(in thousands)2024202320242023
Contractual interest expense$12,106 $12,270 $36,021 $35,289 
Amortization of debt discount$455 $411 $1,313 $1,187 
Amortization of deferred financing$119 $310 $352 $893 
v3.24.3
Restructuring (Tables)
 9 Months Ended
Sep. 30, 2024
Restructuring and Related Activities [Abstract]  
Schedule of Total Charges Incurred Total charges incurred are summarized as follows:
(in thousands)Facility and Non-Lease CostsOther CostsTotal
Balance as of December 31, 2023
$— $— $— 
Restructuring charges6,045 3,143 9,188 
Non-cash items(2,844)— (2,844)
Cash settled(981)(2,136)(3,117)
Balance as of September 30, 2024
$2,220 $1,007 $3,227 
v3.24.3
Stock-Based Compensation (Tables)
 9 Months Ended
Sep. 30, 2024
Share-Based Payment Arrangement [Abstract]  
Schedule of Fair Value of Options
The fair value of the stock options granted were estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions:
 Three Months Ended September 30,Nine Months Ended September 30,
 2024202320242023
Expected stock price volatility55.5 %58.4 %57.1 %59.2 %
Risk free interest rate3.9 %4.3 %4.0 %3.9 %
Expected life of options (years)5.65.55.65.5
Expected annual dividend per share$— $— $— $— 
Schedule of Stock Options Activity A summary of the Company's stock options for the nine months ended September 30, 2024 were as follows:
Number of
Shares		Weighted Average Exercise 
Price		Weighted Average Remaining
Years		Aggregate
Intrinsic
Value
 (in thousands)  (in millions)
Options outstanding, December 31, 202323,002 $11.69   
Granted4,693 $13.69   
Exercised(791)$7.74   
Forfeited(548)$12.74   
Expired(217)$14.58 
Options outstanding, September 30, 202426,139 $12.12 6.0$13.6 
Vested and non-vested expected to vest, September 30, 202424,610 $12.09 5.9$13.5 
Exercisable at September 30, 202417,092 $11.75 4.7$13.0 
Schedule of Non-Vested RSU Activity under the Plan A summary of non-vested RSU activity under the Plan for the nine months ended September 30, 2024 is as follows:
Number of
Shares		Weighted
Average Grant
Date Fair
Value		Weighted 
Average
Remaining 
Years		Aggregate
Intrinsic
Value
(in thousands)(in millions)
Non-vested units as of December 31, 202310,033 $13.37   
Granted3,893 $14.63   
Vested(3,860)$13.17   
Forfeited(613)$14.28   
Non-vested units as of September 30, 20249,453 $13.82 2.2$101.0 
Schedule of Equity Compensation Expenses
The following table summarizes information related to compensation expense recognized in the Company's Consolidated Statements of Operations related to the equity awards:
 Three Months Ended September 30,Nine Months Ended September 30,
(in thousands)2024202320242023
Research and development expense$4,397 $4,380 $12,329 $16,987 
Selling, general, and administrative expense14,291 12,131 53,359 50,995 
Total equity compensation expense$18,688 $16,511 $65,688 $67,982 
v3.24.3
Assets and Liabilities Measured at Fair Value (Tables)
 9 Months Ended
Sep. 30, 2024
Fair Value Disclosures [Abstract]  
Schedule of Assets and Liabilities Subject to Fair Value Measurements
A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of September 30, 2024 are identified in the following tables:
(in thousands) Level 1Level 2Total
Assets:  
Commercial paper$— $5,991 $5,991 
Treasury bill— 9,968 9,968 
Money market7,922 — 7,922 
 $7,922 $15,959 $23,881 
(in thousands) Level 1Level 2Total
Liabilities:  
Deferred compensation plan liability$7,822 $— $7,822 
 $7,822 $— $7,822 
A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2023 are identified in the following tables:
(in thousands)Level 1Level 2Total
Assets:
Commercial paper$— $14,663 $14,663 
Treasury bill— 12,946 12,946 
U.S. government agency bonds— 11,446 11,446 
Money market7,631 — 7,631 
 $7,631 $39,055 $46,686 
(in thousands)Level 1Level 2Total
Liabilities:   
Deferred compensation plan liability7,531 — 7,531 
 $7,531 $— $7,531 
v3.24.3
Basic and Diluted Net Loss per Common Share (Tables)
 9 Months Ended
Sep. 30, 2024
Earnings Per Share [Abstract]  
Schedule of Reconciliation of the Numerator and Denominator Used in Computing Basic and Diluted Net Loss per Common Share
The following table provides a reconciliation of the numerator and denominator used in computing basic and diluted net loss attributable to common stockholders per common share:
 Three Months Ended September 30,Nine Months Ended September 30,
(in thousands, except per share amounts) 2024202320242023
Numerator:  
Net loss attributable to common stockholders$(6,729)$(21,577)$(70,845)$(117,741)
Denominator:
Weighted average common shares outstanding — basic and diluted304,690,596 295,759,435 303,792,479 293,314,167 
Schedule of Potential Shares of Common Stock that were Excluded from the Computation as they were Anti-Dilutive Using the Treasury Stock Method
The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method:
 As of September 30,
(in thousands) 20242023
Options to purchase common stock26,139 23,256 
Non-vested restricted stock units9,453 10,331 
Total number of potentially issuable shares35,592 33,587 
v3.24.3
Description of Business (Details) - USD ($)
$ in Thousands
Sep. 30, 2024
Dec. 31, 2023
Organization, Consolidation and Presentation of Financial Statements [Abstract]    
Accumulated deficit $ 2,754,919 $ 2,684,074
v3.24.3
Summary of Significant Accounting Policies - Narrative (Details)
$ in Millions
 9 Months Ended
Sep. 30, 2024
USD ($)
segment
Accounting Policies [Abstract]  
Number of reportable segments 1
Number of operating segments 1
Allowance for doubtful accounts receivable | $ $ 0.5
v3.24.3
Summary of Significant Accounting Policies - Revenue by Product and Geographical Area (Details) - USD ($)
$ in Thousands
 3 Months Ended 9 Months Ended
Sep. 30, 2024
Sep. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Disaggregation of Revenue [Line Items]        
Net product sales $ 141,517 $ 103,501 $ 378,589 $ 284,274
U.S.        
Disaggregation of Revenue [Line Items]        
Net product sales 56,192 37,801 143,705 103,760
Ex-U.S.        
Disaggregation of Revenue [Line Items]        
Net product sales 85,325 65,700 234,884 180,514
Galafold        
Disaggregation of Revenue [Line Items]        
Net product sales 120,381 100,733 330,557 281,177
Pombiliti + Opfolda        
Disaggregation of Revenue [Line Items]        
Net product sales $ 21,136 $ 2,768 $ 48,032 $ 3,097
v3.24.3
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Narrative (Details) - USD ($)
 9 Months Ended
Sep. 30, 2024
Sep. 30, 2023
Dec. 31, 2023
Cash, Cash Equivalents, and Short-Term Investments [Abstract]      
Cash and cash equivalents $ 233,647,000 $ 263,320,000 $ 246,994,000
Available-for-sale debt securities 16,110,000   39,206,000
Debt securities, realized gain (loss) 0 $ 0  
Fair value of available-for-sale debt securities in unrealized loss positions $ 0.0   $ 11,400,000
v3.24.3
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Cash and Available for Sale Securities (Details) - USD ($)
$ in Thousands
Sep. 30, 2024
Dec. 31, 2023
Sep. 30, 2023
Cash, Money Market Funds, and Marketable Securities      
Cash and cash equivalents $ 233,647 $ 246,994 $ 263,320
Fair value, cash balances 233,647 246,994  
Cost, available-for-sale securities 16,100 39,196  
Gross unrealized gain, available-for-sale securities 10 14  
Gross unrealized loss, available-for-sale securities 0 (4)  
Fair value, available-for-sale debt securities 16,110 39,206  
Cost, cash balances and available-for-sale securities 249,747 286,190  
Gross unrealized gain, cash balances and available-for-sale securities 10 14  
Gross unrealized loss, cash balances and available-for-sale securities 0 (4)  
Fair value, cash balances and available-for-sale securities 249,757 286,200  
Commercial paper      
Cash, Money Market Funds, and Marketable Securities      
Cost, available-for-sale securities 5,991 14,651  
Gross unrealized gain, available-for-sale securities 0 12  
Gross unrealized loss, available-for-sale securities 0 0  
Fair value, available-for-sale debt securities 5,991 14,663  
Treasury bill      
Cash, Money Market Funds, and Marketable Securities      
Cost, available-for-sale securities 9,958 12,944  
Gross unrealized gain, available-for-sale securities 10 2  
Gross unrealized loss, available-for-sale securities 0 0  
Fair value, available-for-sale debt securities 9,968 12,946  
U.S. government agency bonds      
Cash, Money Market Funds, and Marketable Securities      
Cost, available-for-sale securities 0 11,450  
Gross unrealized gain, available-for-sale securities 0 0  
Gross unrealized loss, available-for-sale securities 0 (4)  
Fair value, available-for-sale debt securities 0 11,446  
Money market      
Cash, Money Market Funds, and Marketable Securities      
Cost, available-for-sale securities 100 100  
Gross unrealized gain, available-for-sale securities 0 0  
Fair value, available-for-sale debt securities 100 100  
Gross unrealized loss, cash balances and available-for-sale securities 0 0  
Certificates of deposit      
Cash, Money Market Funds, and Marketable Securities      
Cost, available-for-sale securities 51 51  
Gross unrealized gain, available-for-sale securities 0 0  
Fair value, available-for-sale debt securities 51 51  
Gross unrealized loss, cash balances and available-for-sale securities $ 0 $ 0  
v3.24.3
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Reconciliation of Cash and Cash Equivalents and Restricted Cash (Details) - USD ($)
$ in Thousands
Sep. 30, 2024
Dec. 31, 2023
Sep. 30, 2023
Dec. 31, 2022
Cash, Cash Equivalents, and Short-Term Investments [Abstract]        
Cash and cash equivalents $ 233,647 $ 246,994 $ 263,320  
Restricted cash 3,173   2,952  
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows $ 236,820 $ 250,077 $ 266,272 $ 153,115
v3.24.3
Inventories (Details) - USD ($)
$ in Thousands
Sep. 30, 2024
Dec. 31, 2023
Inventory Disclosure [Abstract]    
Raw materials $ 76,203 $ 30,230
Work-in-process 31,485 22,597
Finished goods 7,650 6,869
Total inventories $ 115,338 $ 59,696
v3.24.3
Inventories - Narrative (Details) - USD ($)
$ in Millions
Sep. 30, 2024
Dec. 31, 2023
Inventory Disclosure [Abstract]    
Reserve for inventory $ 3.5 $ 0.5
v3.24.3
Debt - Summary of Long Term Debt (Details) - USD ($)
$ in Thousands
Sep. 30, 2024
Dec. 31, 2023
Debt Instrument [Line Items]    
Net carrying value of long-term debt $ 389,494 $ 387,858
Senior Loans | Senior Secured Term Loan due 2029    
Debt Instrument [Line Items]    
Principal 400,000 400,000
Less: debt discount (8,339) (9,652)
Less: deferred financing $ (2,167) $ (2,490)
v3.24.3
Debt - Interest Expense (Details) - USD ($)
$ in Thousands
 3 Months Ended 9 Months Ended
Sep. 30, 2024
Sep. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Debt Disclosure [Abstract]        
Contractual interest expense $ 12,106 $ 12,270 $ 36,021 $ 35,289
Amortization of debt discount 455 411 1,313 1,187
Amortization of deferred financing $ 119 $ 310 $ 352 $ 893
v3.24.3
Restructuring (Details) - USD ($)
$ in Thousands
 3 Months Ended 9 Months Ended
Sep. 30, 2024
Sep. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Restructuring Reserve [Roll Forward]        
Balance     $ 0  
Restructuring charges $ 3,143 $ 0 9,188 $ 0
Non-cash items     (2,844)  
Cash settled     (3,117)  
Balance 3,227   3,227  
Facility and Non-Lease Costs        
Restructuring Reserve [Roll Forward]        
Balance     0  
Restructuring charges     6,045  
Non-cash items     (2,844)  
Cash settled     (981)  
Balance 2,220   2,220  
Other Costs        
Restructuring Reserve [Roll Forward]        
Balance     0  
Restructuring charges     3,143  
Non-cash items     0  
Cash settled     (2,136)  
Balance $ 1,007   $ 1,007  
v3.24.3
Stockholders' Equity (Details) - ATM
$ / shares in Units, $ in Millions
 3 Months Ended 9 Months Ended
Sep. 30, 2024
USD ($)
$ / shares
shares
Sep. 30, 2024
USD ($)
$ / shares
shares
Subsidiary, Sale of Stock [Line Items]    
Number of shares issued, private placement (in shares) 1,692,291 1,692,291
Offering price per share (in dollars per share) | $ / shares $ 11.70 $ 11.70
Aggregate net proceeds from stock offering | $ $ 19.2 $ 19.2
Common stock, capital share reserved for future issuance 164,700,000 164,700,000
v3.24.3
Stock-Based Compensation - Weighted-average Assumptions (Details) - Options to purchase common stock - $ / shares
 3 Months Ended 9 Months Ended
Sep. 30, 2024
Sep. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]        
Expected stock price volatility 55.50% 58.40% 57.10% 59.20%
Risk free interest rate 3.90% 4.30% 4.00% 3.90%
Expected life of options (years) 5 years 7 months 6 days 5 years 6 months 5 years 7 months 6 days 5 years 6 months
Expected annual dividend per share (in dollars per share) $ 0 $ 0 $ 0 $ 0
v3.24.3
Stock-Based Compensation - Stock Option Activity (Details) - USD ($)
$ / shares in Units, shares in Thousands, $ in Millions
 9 Months Ended
Sep. 30, 2024
Number of Shares  
Balance at the beginning of the period (in shares) 23,002
Options, Granted (in shares) 4,693
Options, Exercised (in shares) (791)
Options, Forfeited (in shares) (548)
Options, Expired (in shares) (217)
Balance at the end of the period (in shares) 26,139
Vested and non-vested expected to vest as of the end of the period (in shares) 24,610
Exercisable at the end of the period (in shares) 17,092
Weighted Average Exercise  Price  
Balance at the beginning of the period (in dollars per share) $ 11.69
Options, Granted (in dollars per share) 13.69
Options, Exercised (in dollars per share) 7.74
Options, Forfeited (in dollars per share) 12.74
Options, Expired (in dollars per share) 14.58
Balance at the end of the period (in dollars per share) 12.12
Vested and non-vested expected to vest as of the end of the period (in dollars per share) 12.09
Exercisable at the end of the period (in dollars per share) $ 11.75
Weighted Average Remaining Years and Aggregate Intrinsic Value  
Balance at the end of the period 6 years
Vested and non-vested expected to vest at the end of the period 5 years 10 months 24 days
Exercisable at the end of the period 4 years 8 months 12 days
Aggregate intrinsic value, options outstanding $ 13.6
Aggregate intrinsic value, Vested and non-vested expected to vest 13.5
Aggregate intrinsic value of options exercisable $ 13.0
v3.24.3
Stock-Based Compensation - Narrative (Details)
$ in Millions
 9 Months Ended
Sep. 30, 2024
USD ($)
Options to purchase common stock  
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]  
Total unrecognized compensation costs $ 43.4
Unrecognized compensation costs, period for recognition (in years) 3 years
Non-vested restricted stock units  
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]  
Total unrecognized compensation costs $ 62.8
Unrecognized compensation costs, period for recognition (in years) 2 years
v3.24.3
Stock-Based Compensation - RSUs and PBRSUs Summary (Details) - Non-vested restricted stock units
$ / shares in Units, shares in Thousands, $ in Millions
 9 Months Ended
Sep. 30, 2024
USD ($)
$ / shares
shares
Number of Shares  
Non-vested units as of the beginning of the period (in shares) | shares 10,033
Granted (in shares) | shares 3,893
Vested (in shares) | shares (3,860)
Forfeited (in shares) | shares (613)
Non-vested units as of the end of the period (in shares) | shares 9,453
Weighted Average Grant Date Fair Value  
Non-vested units as of the beginning of the period (in dollars per share) | $ / shares $ 13.37
Granted (in dollars per share) | $ / shares 14.63
Vested (in dollars per share) | $ / shares 13.17
Forfeited (in dollars per share) | $ / shares 14.28
Non-vested units as of the end of the period (in dollars per share) | $ / shares $ 13.82
Weighted Average Remaining Years and Aggregate Intrinsic Value  
Non-vested units, weighted average remaining years 2 years 2 months 12 days
Non-vested units, aggregate intrinsic value | $ $ 101.0
v3.24.3
Stock-Based Compensation - Expense Summary (Details) - USD ($)
$ in Thousands
 3 Months Ended 9 Months Ended
Sep. 30, 2024
Sep. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]        
Total equity compensation expense $ 18,688 $ 16,511 $ 65,688 $ 67,982
Research and development expense        
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]        
Total equity compensation expense 4,397 4,380 12,329 16,987
Selling, general, and administrative expense        
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]        
Total equity compensation expense $ 14,291 $ 12,131 $ 53,359 $ 50,995
v3.24.3
Assets and Liabilities Measured at Fair Value (Details) - USD ($)
$ in Thousands
Sep. 30, 2024
Dec. 31, 2023
Assets:    
Fair value of assets $ 23,881 $ 46,686
Liabilities:    
Deferred compensation plan liability 7,822 7,531
Fair value of liabilities 7,822 7,531
Commercial paper    
Assets:    
Fair value of assets 5,991 14,663
Treasury bill    
Assets:    
Fair value of assets 9,968 12,946
U.S. government agency bonds    
Assets:    
Fair value of assets   11,446
Money market    
Assets:    
Fair value of assets 7,922 7,631
Level 1    
Assets:    
Fair value of assets 7,922 7,631
Liabilities:    
Deferred compensation plan liability 7,822 7,531
Fair value of liabilities 7,822 7,531
Level 1 | Commercial paper    
Assets:    
Fair value of assets 0 0
Level 1 | Treasury bill    
Assets:    
Fair value of assets 0 0
Level 1 | U.S. government agency bonds    
Assets:    
Fair value of assets   0
Level 1 | Money market    
Assets:    
Fair value of assets 7,922 7,631
Level 2    
Assets:    
Fair value of assets 15,959 39,055
Liabilities:    
Deferred compensation plan liability 0 0
Fair value of liabilities 0 0
Level 2 | Commercial paper    
Assets:    
Fair value of assets 5,991 14,663
Level 2 | Treasury bill    
Assets:    
Fair value of assets 9,968 12,946
Level 2 | U.S. government agency bonds    
Assets:    
Fair value of assets   11,446
Level 2 | Money market    
Assets:    
Fair value of assets $ 0 $ 0
v3.24.3
Basic and Diluted Net Loss per Common Share (Details) - USD ($)
$ in Thousands
 3 Months Ended 9 Months Ended
Sep. 30, 2024
Sep. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Numerator:        
Net loss attributable to common stockholders $ (6,729) $ (21,577) $ (70,845) $ (117,741)
Denominator:        
Weighted average common shares outstanding - basic (in shares) 304,690,596 295,759,435 303,792,479 293,314,167
Weighted average common shares outstanding - diluted (in shares) 304,690,596 295,759,435 303,792,479 293,314,167
Antidilutive securities        
Antidilutive securities excluded from computation of diluted earnings per share (in shares)     35,592,000 33,587,000
Net loss $ (6,729) $ (21,577) $ (70,845) $ (117,741)
Options to purchase common stock        
Antidilutive securities        
Antidilutive securities excluded from computation of diluted earnings per share (in shares)     26,139,000 23,256,000
Non-vested restricted stock units        
Antidilutive securities        
Antidilutive securities excluded from computation of diluted earnings per share (in shares)     9,453,000 10,331,000
v3.24.3
Basic and Diluted Net Loss per Common Share - Narrative (Details)
Sep. 30, 2024
$ / shares
Private Placement  
Antidilutive securities  
Warrants exercise price (in dollars per share) $ 0.01
Amicus Therapeutics (NASDAQ:FOLD)
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