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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 June 30, 2023
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)
3675 Market Street,
Philadelphia,
PA
19104
(Address of Principal Executive Offices)(Zip Code)
(215)
921-7600
(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 July 26, 2023 was 287,120,937 shares.



AMICUS THERAPEUTICS, INC.
 
Form 10-Q for the Quarterly Period Ended June 30, 2023
 
 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 Pompe Enzyme Replacement Therapy ("ERT" or "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 these therapies and obtain market acceptance for such therapies;
the costs, timing, and outcome of regulatory review of our product candidates, including AT-GAA;
any changes in regulatory standards relating to the review of our product candidates, including AT-GAA;
the number and development requirements of other product candidates that we pursue;
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 product and product candidates, including AT-GAA;
our ability to successfully commercialize Galafold® (also referred to as "migalastat HCl");
our ability to successfully commercialize Pombiliti and Opfolda (also referred to as AT-GAA) in the E.U., and elsewhere, if our regulatory applications for AT-GAA are approved;
our ability to manufacture or supply sufficient clinical or commercial products, including Galafold®, Pombiliti and Opfolda;
our ability to obtain reimbursement for Galafold®;
our ability to obtain reimbursement for Pombiliti and Opfolda in the E.U., and elsewhere, if our regulatory applications for AT-GAA are approved;
our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of Galafold®;
our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of Pombiliti and Opfoldain the E.U., and elsewhere, if our regulatory applications for AT-GAA are approved;
our ability to obtain market acceptance of Galafold® and, if our regulatory applications are approved, AT-GAA;
our ability to obtain market acceptance of Pombiliti and Opfoldain the E.U., and elsewhere, if our regulatory applications for AT-GAA are approved;
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;
1


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 payments from any such collaborators;
the extent to which our business could be adversely impacted by the effects of the novel coronavirus ("COVID-19") outbreak, including actions by us, governments, our customers, our suppliers, or other third parties to control the spread of COVID-19, or by other health epidemics or pandemics;
the costs associated with, and our ability to comply with, emerging environmental, social and governance standards;
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, 2022, 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, 2022 (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)
June 30,
2023
December 31, 2022
Assets
Current assets:
Cash and cash equivalents$211,307 $148,813 
Investments in marketable securities54,319 144,782 
Accounts receivable63,716 66,196 
Inventories51,381 23,816 
Prepaid expenses and other current assets52,099 40,209 
Total current assets432,822 423,816 
Operating lease right-of-use assets, net28,042 29,534 
Property and equipment, less accumulated depreciation of $24,060 and $22,281 at June 30, 2023 and December 31, 2022, respectively
30,238 30,778 
Intangible assets, less accumulated amortization of $855 and $0 at June 30, 2023 and December 31, 2022, respectively
22,145 23,000 
Goodwill197,797 197,797 
Other non-current assets19,049 19,242 
Total Assets$730,093 $724,167 
Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable$13,522 $15,413 
Accrued expenses and other current liabilities124,868 93,636 
Contingent consideration payable13,005 21,417 
Operating lease liabilities7,840 8,552 
Total current liabilities159,235 139,018 
Long-term debt393,350 391,990 
Operating lease liabilities50,976 51,578 
Deferred reimbursements5,906 4,656 
Deferred income taxes 4,939 
Other non-current liabilities9,045 8,939 
Total liabilities618,512 601,120 
Commitments and contingencies
Stockholders’ equity:
Common stock, $0.01 par value, 500,000,000 shares authorized, 286,992,923 and 281,108,273 shares issued and outstanding at June 30, 2023 and December 31, 2022, respectively
2,856 2,815 
Additional paid-in capital2,733,148 2,664,744 
Accumulated other comprehensive gain (loss):
Foreign currency translation adjustment4,337 (11,989)
Unrealized loss on available-for-sale securities(177)(116)
Warrants71 83 
Accumulated deficit(2,628,654)(2,532,490)
Total stockholders’ equity111,581 123,047 
Total Liabilities and Stockholders’ Equity$730,093 $724,167 

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 June 30,Six Months Ended June 30,
2023202220232022
Net product sales$94,503 $80,731 $180,773 $159,446 
Cost of goods sold9,114 8,197 16,056 15,779 
Gross profit85,389 72,534 164,717 143,667 
Operating expenses:
Research and development35,149 78,319 76,648 159,836 
Selling, general, and administrative65,423 53,379 139,380 111,495 
Changes in fair value of contingent consideration payable337 115 588 (1,073)
Loss on impairment of assets1,134  1,134 6,616 
Depreciation and amortization2,206 1,334 3,463 2,745 
Total operating expenses104,249 133,147 221,213 279,619 
Loss from operations(18,860)(60,613)(56,496)(135,952)
Other (expense) income:
Interest income1,737 356 3,936 489 
Interest expense(12,492)(8,257)(24,336)(16,404)
Other (expense) income(10,902)7,268 (16,840)9,170 
Loss before income tax (40,517)(61,246)(93,736)(142,697)
Income tax expense(2,715)(911)(2,428)(4,720)
Net loss attributable to common stockholders$(43,232)$(62,157)$(96,164)$(147,417)
Net loss attributable to common stockholders per common share — basic and diluted$(0.15)$(0.21)$(0.33)$(0.51)
Weighted-average common shares outstanding — basic and diluted292,797,002291,970,562292,071,201288,646,587
                                                                                    
See accompanying Notes to Consolidated Financial Statements
4


Amicus Therapeutics, Inc.
Consolidated Statements of Comprehensive Loss
(Unaudited)
(in thousands)
 Three Months Ended June 30,Six Months Ended June 30,
 2023202220232022
Net loss$(43,232)$(62,157)$(96,164)$(147,417)
Other comprehensive gain (loss):
Foreign currency translation adjustment gain (loss)10,880 (16,183)16,326 (21,854)
Unrealized gain (loss) on available-for-sale securities24 (29)(61)(367)
Other comprehensive gain (loss) 10,904 (16,212)16,265 (22,221)
Comprehensive loss$(32,328)$(78,369)$(79,899)$(169,638)

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 June 30, 2023
Common StockAdditional
Paid-In
Capital
WarrantsOther
Comprehensive
Gain (Loss)
Accumulated
Deficit
Total
Stockholders'
Equity
SharesAmount
Balance at March 31, 2023283,300,585 $2,820 $2,691,836 $83 $(6,744)$(2,585,422)$102,573 
Stock options exercised, net269,109 4 1,746 — — — 1,750 
Vesting of restricted stock units, net of taxes155,776 — (1,202)— — — (1,202)
Stock-based compensation— — 16,577 — — — 16,577 
Issuance of shares in connection with at-the-market offering, net of issuance costs2,047,353 20 24,179 — — — 24,199 
Warrants exercised1,220,100 12 12 (12)— — 12 
Unrealized gain on available-for-sale securities— — — — 24 — 24 
Foreign currency translation adjustment— — — — 10,880 — 10,880 
Net loss— — — — — (43,232)(43,232)
Balance at June 30, 2023286,992,923 $2,856 $2,733,148 $71 $4,160 $(2,628,654)$111,581 

Six Months Ended June 30, 2023
Common StockAdditional
Paid-In
Capital
WarrantsOther
Comprehensive
Gain (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, net653,217 7 4,398 — — — 4,405 
Vesting of restricted stock units, net of taxes1,768,751 (14,008)— — — (14,008)
Stock-based compensation— — 51,471 — — — 51,471 
Warrants exercised1,220,100 12 12 (12)— — 12 
Issuance of shares in connection with at-the-market offering, net of issuance costs2,242,582 22 26,531 — — — 26,553 
Unrealized loss on available-for-sale securities— — — — (61)— (61)
Foreign currency translation adjustment— — — — 16,326 — 16,326 
Net loss— — — — — (96,164)(96,164)
Balance at June 30, 2023286,992,923 $2,856 $2,733,148 $71 $4,160 $(2,628,654)$111,581 
6


Three Months Ended June 30, 2022
Common StockAdditional
Paid-In
Capital
WarrantsOther
Comprehensive
Gain (Loss)
Accumulated
Deficit
Total
Stockholders'
Equity
SharesAmount
Balance at March 31, 2022280,133,856 $2,809 $2,617,935 $83 $(1,028)$(2,381,182)$238,617 
Stock options exercised, net189,256 2 997 — — — 999 
Vesting of restricted stock units, net of taxes133,555 — (285)— — — (285)
Stock-based compensation— — 12,463 — — — 12,463 
Unrealized loss on available-for-sale securities— — — — (29)— (29)
Foreign currency translation adjustment— — — — (16,183)— (16,183)
Net loss— — — — — (62,157)(62,157)
Balance at June 30, 2022280,456,667 $2,811 $2,631,110 $83 $(17,240)$(2,443,339)$173,425 

Six Months Ended June 30, 2022
Common StockAdditional
Paid-In
Capital
WarrantsOther
Comprehensive
Gain (Loss)
Accumulated
Deficit
Total
Stockholders'
Equity
SharesAmount
Balance at December 31, 2021278,912,800 $2,808 $2,595,419 $83 $4,981 $(2,295,922)$307,369 
Stock options exercised, net334,705 3 1,855 — — — 1,858 
Vesting of restricted stock units, net of taxes1,209,162 — (9,278)— — — (9,278)
Stock-based compensation— — 43,114 — — — 43,114 
Unrealized loss on available-for-sale securities— — — — (367)— (367)
Foreign currency translation adjustment— — — — (21,854)— (21,854)
Net loss— — — — — (147,417)(147,417)
Balance at June 30, 2022280,456,667 $2,811 $2,631,110 $83 $(17,240)$(2,443,339)$173,425 


See accompanying Notes to Consolidated Financial Statements
7


Amicus Therapeutics, Inc.
Consolidated Statements of Cash Flows
(Unaudited)
(in thousands)
Six Months Ended June 30,
20232022
Operating activities
Net loss$(96,164)$(147,417)
Adjustments to reconcile net loss to net cash used in operating activities:
Amortization of debt discount and deferred financing1,359 1,295 
Depreciation and amortization3,463 2,745 
Stock-based compensation51,471 43,114 
Non-cash changes in the fair value of contingent consideration payable588 (1,073)
Foreign currency remeasurement loss23,904 353 
Deferred taxes(4,939) 
Asset impairment charges and other asset write-offs2,060 12,265 
Changes in operating assets and liabilities:
Accounts receivable2,705 (4,424)
Inventories(27,483)3,537 
Prepaid expenses and other current assets(12,263)(4,481)
Accounts payable, accrued expenses, and other current liabilities31,620 22,671 
Other non-current assets and liabilities(2,586)(2,762)
Payment of contingent consideration(7,937) 
Net cash used in operating activities$(34,202)$(74,177)
Investing activities
Sale and redemption of marketable securities126,848 184,061 
Purchases of marketable securities(36,448)(98,330)
Capital expenditures(4,144)(1,226)
Net cash provided by investing activities$86,256 $84,505 
Financing activities
Payment of finance leases(51)(41)
Withholding taxes paid on vested restricted stock units(14,008)(9,278)
Proceeds from stock options exercised, net4,405 1,858 
Proceeds from warrants exercised, net12  
Proceeds from the issuance of shares in connection with at-the-market offering, net of issuance costs26,553  
Payment of contingent consideration(1,063) 
Net cash provided by (used in) financing activities$15,848 $(7,461)
Effect of exchange rate changes on cash, cash equivalents, and restricted cash$(6,684)$(12,462)
Net increase (decrease) in cash, cash equivalents, and restricted cash at the end of the period61,218 (9,595)
Cash, cash equivalents, and restricted cash at the beginning of period153,115 249,456 
Cash, cash equivalents, and restricted cash at the end of period$214,333 $239,861 
Supplemental disclosures of cash flow information
Cash paid during the period for interest $23,155 $15,108 
Cash paid for taxes$5,978 $710 
Capital expenditures unpaid at the end of period$297 $53 

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 has developed and commercialized the first oral monotherapy for Fabry disease that has achieved widespread global approval and the first two-component therapy for Pompe disease that has been approved in the European Union (“E.U.”) and the United Kingdom (“U.K.”), and is under regulatory review in the United States (“U.S.”). The Company is committed to discovering and developing next generation therapies in Fabry and Pompe diseases.
The cornerstone of the Company's portfolio is Galafold® (also referred to as "migalastat"), the first and only approved oral precision medicine for people living with Fabry disease who have amenable genetic variants. Migalastat is currently approved under the trade name Galafold® in the U.S., E.U., U.K., and Japan, with multiple additional approvals granted and applications pending in several geographies around the world.
Pombiliti and Opfolda (also referred to as AT-GAA, ATB200/AT2221, or cipaglucosidase alfa/miglustat), is a novel, two-component treatment for Pompe disease that was approved by the European Commission ("EC") in June 2023 and the U.K. Medicines and Healthcare products Regulatory Agency ("MHRA") in August 2023. The Company began launch activities for Pombiliti and Opfolda in Germany and will commence the reimbursement processes with healthcare authorities in other European countries. In October 2022, the U.S. Food and Drug Administration ("FDA") deferred action on the BLA for cipaglucosidase alfa, citing the inability to complete the manufacturing site inspection prior to the PDUFA action date. In the second quarter of 2023, the FDA completed the required pre-approval inspection of the manufacturing site and the review is ongoing.
The Company continues to monitor the novel coronavirus (“COVID-19”) pandemic. The Company's commercial operations have not been significantly impacted by the COVID-19 pandemic and the Company continues to see a gradual improvement in patient identification and Galafold® initiation. The Company has been able to continue to meet required commercial demand for Galafold® as well as supply Pombiliti and Opfolda commercial launch inventory and its ongoing clinical studies and access programs without interruption. In regard to the Company's regulatory operations, the FDA deferred action on the pending BLA for cipaglucosidase alfa, as a site inspection could not be completed by the PDUFA action date due to COVID-19 related travel restrictions in China. In the second quarter of 2023, the FDA completed the required pre-approval inspection of the manufacturing site. Per FDA guidance relating to pre-approval inspections during the COVID-19 pandemic, receipt of a deferral action indicates no deficiencies have been identified and the application otherwise satisfies the requirements for approval.
The Company had an accumulated deficit of $2.6 billion as of June 30, 2023 and anticipates incurring losses through the fiscal year ending December 31, 2023. The Company has historically funded its operations through stock offerings, Galafold® revenues, debt issuances, collaborations, and other financing arrangements.
Based on its current operating model, the Company believes that the current cash position, which includes expected revenues, is sufficient to fund the Company's operations and ongoing research programs for at least the next 12 months. Potential impacts of the COVID-19 pandemic, business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact the Company's future capital requirements.
9


2. Summary of Significant Accounting Policies
Basis of Presentation
The Company has prepared the accompanying unaudited Consolidated Financial Statements 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, they do not include all of the information and disclosures required by U.S. GAAP for complete financial statements. 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.
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, 2022. 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, 2022.
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 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.
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.
Additionally, the Company assessed the impact the COVID-19 pandemic had on its operations and financial results as of June 30, 2023 and through the issuance of these financial statements. The Company’s analysis was informed by the facts and circumstances as they were known to the Company. This assessment considered the impact COVID-19 may have on financial estimates and assumptions that affect the reported amounts of assets and liabilities and revenue and expenses.
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 loss in the Company's Consolidated Statements of Comprehensive 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 in other non-current assets on the Company's Consolidated Balance Sheets.
10


Concentration of Credit Risk
The Company's financial instruments that are exposed to concentration of credit risk consist primarily of cash, cash equivalents, and marketable securities. 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 is subject to credit risk from its accounts receivable primarily related to its product sales of Galafold®. The Company's accounts receivable at June 30, 2023 have arisen from product sales primarily in Europe, the U.S., and Japan. The Company will periodically assess 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 June 30, 2023, the Company's allowance for doubtful accounts was $0.1 million.
Revenue Recognition
The Company's net product sales consist primarily of sales of Galafold® for the treatment of Fabry disease. Galafold® sales for the three and six months ended June 30, 2023 were $94.3 million and $180.4 million, respectively, and $80.7 million and $159.4 million for the three and six months ended June 30, 2022, respectively. The Company has recorded revenue on sales where Galafold® is available either on a commercial basis or through a reimbursed early access program. Orders for Galafold® are generally received from distributors and pharmacies, with the ultimate payor often a government authority.
The Company recognizes revenue when its performance obligations to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of Galafold®. 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 are third party discounts and rebates. The identified variable consideration is recorded as a reduction of revenue at the time revenue from the sale of Galafold® 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 geographic area:
Three Months Ended June 30,Six Months Ended June 30,
(in thousands)2023202220232022
U.S.$37,128 $27,540 $65,959 $51,718 
Ex-U.S.57,375 53,191 114,814 107,728 
Total net product sales$94,503 $80,731 $180,773 $159,446 
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.
11


Intangible Assets and Goodwill
The Company records goodwill in a business combination when the total consideration exceeds the fair value of the net tangible and identifiable intangible assets acquired. Goodwill is assessed annually for impairment on October 1 and whenever events or circumstances indicate that the carrying amount of an asset may not be recoverable. The Company first assesses the qualitative factors to determine if a quantitative test is necessary. If required, or if the Company elects to bypass the qualitative assessment, a quantitative goodwill impairment test is conducted. If it is determined the Company's single reporting unit's carrying value, including goodwill, exceeds its fair value, an impairment loss is recorded for the difference.
Finite-lived intangible assets are recorded at cost, net of accumulated amortization, and, if applicable, impairment charges. Amortization of finite-lived intangible assets is recorded over the assets’ estimated useful lives on a straight-line basis or based on the pattern in which economic benefits are consumed, if reliably determinable. The Company reviews finite-lived intangible assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. If impairment is determined, the Company writes down the asset to its estimated fair value and records an impairment loss equal to the excess of the carrying value of the asset over its estimated fair value in the period at which such a determination is made.
No indicators of impairment were noted during the six months ended June 30, 2023.
Recent Accounting Developments
The Company has evaluated recent accounting pronouncements and believes that none of them will have a material effect on the Company's Consolidated Financial Statements or related disclosures.

3. Intangible Assets
As of June 30, 2023, the Company's intangible assets consisted of lead enzyme replacement therapy assets acquired with the Callidus Biopharma, Inc. acquisition in 2013, previously accounted for as in-process research and development. In March 2023, as a result of the EC's approval of Pombiliti, the Company began amortizing the assets over the initial regulatory exclusivity period of 7 years. The Company completed an impairment assessment before changing the classification to definite-lived intangible asset noting no impairment. Amortization expense for the three and six months ended June 30, 2023 was $0.8 million and $0.9 million, respectively. Total estimated amortization for the finite-lived intangible assets is estimated to be $2.5 million for the year ended December 31, 2023 and $3.3 million for the next four years thereafter.

4. Cash, Cash Equivalents, Marketable Securities, and Restricted Cash
As of June 30, 2023, the Company held $211.3 million in cash and cash equivalents and $54.3 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 gain (loss) in the Company's Consolidated Statements of Comprehensive 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 earnings 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 earnings.
The Company regularly invests excess operating cash in deposits with major financial institutions and money market funds, as well as fixed income investments 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, 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 June 30, 2023
(in thousands)CostGross
Unrealized
Gain
Gross
Unrealized
Loss
Fair
Value
Cash and cash equivalents$211,307 $— $— $211,307 
Commercial paper44,022 10 (1)44,031 
U.S. government agency bonds9,875 12  9,887 
Money market350   350 
Certificates of deposit51   51 
$265,605 $22 $(1)$265,626 
Included in cash and cash equivalents$211,307 $— $— $211,307 
Included in marketable securities54,298 22 (1)54,319 
Total cash, cash equivalents, and marketable securities$265,605 $22 $(1)$265,626 

 As of December 31, 2022
(in thousands)CostGross
Unrealized
Gain
Gross
Unrealized
Loss
Fair
Value
Cash and cash equivalents$148,813 $— $— $148,813 
Commercial paper144,299 82  144,381 
Money market350   350 
Certificate of deposit51   51 
$293,513 $82 $ $293,595 
Included in cash and cash equivalents$148,813 $— $— $148,813 
Included in marketable securities144,700 82  144,782
Total cash, cash equivalents, and marketable securities$293,513 $82 $ $293,595 
For both the six months ended June 30, 2023 and the fiscal year ended December 31, 2022, there were no realized gains or losses. The cost of securities sold is based on the specific identification method.
Unrealized loss positions in the marketable securities as of June 30, 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 in other comprehensive gain (loss). The fair value of these marketable securities in unrealized loss positions was $3.0 million as of June 30, 2023. The Company had no securities in an unrealized loss position as of December 31, 2022.
The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Company's Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Company's Consolidated Statements of Cash Flows.
As of June 30,
(in thousands)20232022
Cash and cash equivalents$211,307 $235,639 
Restricted cash3,026 4,222 
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows$214,333 $239,861 

13


5. Inventories
Inventories as of June 30, 2023 and December 31, 2022 consisted of the following:
(in thousands)June 30, 2023December 31, 2022
Raw materials$36,379 $10,054 
Work-in-process7,327 9,615 
Finished goods7,675 4,147 
Total inventories$51,381 $23,816 
The Company's reserve for inventory was $1.0 million and $0.4 million as of June 30, 2023 and December 31, 2022, respectively.

6. Debt
The Company's debt consists of the following:
(in thousands)June 30, 2023December 31, 2022
Senior Secured Term Loan due 2026:
Principal$400,000 $400,000 
Less: debt discount (1)
(3,795)(4,571)
Less: deferred financing (1)
(2,855)(3,439)
Net carrying value of Long-term debt$393,350 $391,990 
______________________________
(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 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 six months ended June 30, 2023 and 2022, respectively:
Three Months Ended June 30,Six Months Ended June 30,
(in thousands)2023202220232022
Contractual interest expense$11,789 $7,589 $23,019 $15,089 
Amortization of debt discount$395 $375 $776 $739 
Amortization of deferred financing$297 $283 $583 $556 

7. Stockholder's Equity
During the three and six months ended June 30, 2023, the Company issued and sold an aggregate of 2,047,353 and 2,242,582 shares, respectively, through its at-the-market equity program ("ATM program") at weighted-average public offering prices of $12.21 and $12.25 per share, resulting in net proceeds of $24.2 million and $26.6 million, respectively. As of June 30, 2023, an aggregate of $222.5 million worth of shares remain available to be issued and sold under the ATM program.

14


8. Stock-Based Compensation
The Company's 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 Company's 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 Company's 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 is estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions:
 Three Months Ended June 30,Six Months Ended June 30,
 2023202220232022
Expected stock price volatility59.2 %61.3 %59.3 %62.2 %
Risk free interest rate3.8 %3.0 %3.9 %1.6 %
Expected life of options (years)5.55.35.55.3
Expected annual dividend per share$ $ $ $ 
 A summary of the Company's stock options for the six months ended June 30, 2023 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, 202219,064 $11.31   
Granted5,194 $11.97   
Exercised(655)$6.73   
Forfeited(160)$11.36   
Expired(35)$13.99 
Options outstanding, June 30, 202323,408 $11.58 6.9$41.1 
Vested and unvested expected to vest, June 30, 202321,510 $11.52 6.7$39.3 
Exercisable at June 30, 202313,061 $11.02 5.3$32.5 
As of June 30, 2023, 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 six months ended June 30, 2023 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, 20229,717 $13.07   
Granted3,967 $13.06   
Vested(2,752)$12.12   
Forfeited(567)$9.86   
Non-vested units as of June 30, 202310,365 $13.53 2.3$130.2 
As of June 30, 2023, there was $68.5 million of total unrecognized compensation cost related to unvested 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 June 30,Six Months Ended June 30,
(in thousands)2023202220232022
Research and development expense$4,117 $4,379 $12,607 $13,744 
Selling, general, and administrative expense12,460 8,084 38,864 29,370 
Total equity compensation expense$16,577 $12,463 $51,471 $43,114 

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 June 30, 2023 are identified in the following tables:
(in thousands) Level 2Total
Assets:  
Commercial paper$44,031 $44,031 
U.S. government agency bonds9,887 9,887 
Money market7,133 7,133 
 $61,051 $61,051 
(in thousands) Level 2Level 3Total
Liabilities:   
Contingent consideration payable$ $13,005 $13,005 
Deferred compensation plan liability6,783  6,783 
 $6,783 $13,005 $19,788 
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, 2022 are identified in the following tables:
(in thousands)Level 2Total
Assets:
Commercial paper$144,381 $144,381 
Money market5,808 5,808 
 $150,189 $150,189 
(in thousands)Level 2Level 3Total
Liabilities:   
Contingent consideration payable$ $21,417 $21,417 
Deferred compensation plan liability5,458  5,458 
 $5,458 $21,417 $26,875 
The Company's Senior Secured Term Loan due 2026 falls into the Level 2 category within the fair value level hierarchy and the fair value was determined using quoted prices for similar liabilities in active markets, as well as inputs that are observable for the liability (other than quoted prices), such as interest rates that are observable at commonly quoted intervals. The carrying value of the Senior Secured Term Loan due 2026 approximates the fair value.
The Company did not have any Level 3 assets as of June 30, 2023 or December 31, 2022.
Cash, Money Market Funds, and Marketable Securities
The Company classifies its cash 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 and the money market funds within the fair value hierarchy as Level 2 primarily utilizing broker quotes in a non-active market for valuation of these securities.
Contingent Consideration Payable
The contingent consideration payable resulted from the acquisition of Callidus Biopharma, Inc. ("Callidus") in November 2013. The most recent valuation was determined using a probability weighted discounted cash flow valuation approach. Gains and losses are included in the Company's Consolidated Statements of Operations.
The contingent consideration payable for Callidus has been classified as a Level 3 recurring liability as its valuation requires substantial judgment and estimation of factors that are not currently observable in the market. If different assumptions
17


were used for the various inputs to the valuation approach, the estimated fair value could be significantly higher or lower than the fair value the Company determined.
The following significant unobservable inputs were used in the valuation of the contingent consideration payable of Callidus for the ATB200 Pompe disease program:
Contingent Consideration
Liability
Fair Value as of June 30, 2023Valuation TechniqueUnobservable InputRange
(in thousands)
     
  Discount rate11.7%
  
Clinical and regulatory milestones$13,005 Probability weighted discounted cash flowProbability of achievement of milestones
88%
  
  Projected year of payments
2023
Contingent consideration liabilities are remeasured to fair value each reporting period using discount rates, probabilities of payment, and projected payment dates. Projected contingent payment amounts related to clinical and regulatory based milestones are discounted back to the current period using a discounted cash flow model. Increases in discount rates and the time to payment may result in lower fair value measurements. Increases or decreases in any of those inputs together, or in isolation, may result in a significantly lower or higher fair value measurement. There is no assurance that any of the conditions for the milestone payments will be met.
The Company reached a regulatory milestone in March 2023 associated with the EC granting approval for Pombiliti, related to the contingent consideration of Callidus for the ATB200 Pompe disease program. The satisfaction of this milestone resulted in a milestone payment of $9.0 million.
The following table shows the change in the balance of contingent consideration payable for the three and six months ended June 30, 2023 and 2022, respectively:
Three Months Ended June 30,Six Months Ended June 30,
(in thousands)2023202220232022
Balance, beginning of the period$12,668 $19,151 $21,417 $20,339 
Changes in fair value during the period, included in the Consolidated Statements of Operations337 115 588 (1,073)
Milestone payment in cash  (9,000) 
Balance, end of the period (1)
$13,005 $19,266 $13,005 $19,266 
______________________________
(1) As certain milestones are expected to be reached within the next twelve months, the June 30, 2023 balance was recorded as a current liability in the Company's Consolidated Balance Sheets.
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10. Basic and Diluted Net Loss per Common Share
The following table provides a reconciliation of the numerator and denominator used in computing basic and diluted net loss attributable to common stockholders per common share:
 Three Months Ended June 30,Six Months Ended June 30,
(in thousands, except per share amounts) 2023202220232022
Numerator:  
Net loss attributable to common stockholders$(43,232)$(62,157)$(96,164)$(147,417)
Denominator:
Weighted average common shares outstanding — basic and diluted292,797,002 291,970,562 292,071,201 288,646,587 
Dilutive common stock equivalents would include the dilutive effect of outstanding common stock options and unvested 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 June 30,
(in thousands) 20232022
Options to purchase common stock23,408 19,283 
Unvested restricted stock units10,365 10,166 
Total number of potentially issuable shares33,773 29,449 

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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, 2022. 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, 2022 entitled “Risk Factors”.
Overview
We are a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases. We have developed and commercialized the first oral monotherapy for Fabry disease that has achieved widespread global approval and the first two-component therapy for Pompe disease that has been approved in the European Union (“E.U.”) and the United Kingdom (“U.K.”), and is under regulatory review in the United States (“U.S.”). We are committed to discovering and developing next generation therapies in Fabry and Pompe diseases.
The cornerstone of our portfolio is Galafold® (also referred to as "migalastat"), the first and only approved oral precision medicine for people living with Fabry disease who have amenable genetic variants. Migalastat is currently approved under the trade name Galafold® in the U.S., E.U., U.K., and Japan, with multiple additional approvals granted and applications pending in several geographies around the world.
Pombiliti and Opfolda (also referred to as AT-GAA, ATB200/AT2221, or cipaglucosidase alfa/miglustat), is a novel, two-component treatment for Pompe disease that was approved by the European Commission ("EC") in June 2023 and the U.K. Medicines and Healthcare products Regulatory Agency ("MHRA") in August 2023. We began launch activities for Pombiliti and Opfolda in Germany and will commence the reimbursement processes with healthcare authorities in other European countries. In October 2022, the U.S. Food and Drug Administration ("FDA") deferred action on the BLA for cipaglucosidase alfa, citing the inability to complete the manufacturing site inspection prior to the PDUFA action date. In the second quarter of 2023, the FDA completed the required pre-approval inspection of the manufacturing site and the review is ongoing.
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 that have the potential to obsolete current treatments, provide significant benefits to patients, and be first- or best-in-class. We are leveraging our global capabilities to develop and broaden our lead franchises in Fabry and Pompe disease, with focused discovery work on next generation therapies and novel platform technologies.
We continue to monitor the novel coronavirus ("COVID-19") pandemic. Our commercial operations have not been significantly impacted by the COVID-19 pandemic and we continue to see a gradual improvement in patient identification and Galafold® initiation. We have been able to continue to meet required commercial demand for Galafold® as well as supply Pombiliti and Opfolda commercial launch inventory and our ongoing clinical studies and access programs without interruption. In regard to our regulatory operations, the FDA deferred action on the pending BLA for cipaglucosidase alfa, as a site inspection could not be completed by the PDUFA action date due to COVID-19 related travel restrictions in China. In the second quarter of 2023, the FDA completed the required pre-approval inspection of the manufacturing site. Per FDA guidance relating to pre-approval inspections during the COVID-19 pandemic, receipt of a deferral action indicates no deficiencies have been identified and the application otherwise satisfies the requirements for approval.
Highlights of our progress include:
Commercial and regulatory success in Fabry disease. For the six months ended June 30, 2023, Galafold® revenue was $180.4 million of consolidated revenue, which represented an increase of $21.0 million compared to the same period in the prior year. We continue to see strong commercial momentum and expansion into additional geographies. In countries where we have been operating the longest, we see an increasing proportion of previously untreated patients come onto Galafold® as compared to treatment experienced patients. In the U.S., we continue to see a significant
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increase in patients from a growing and very wide prescriber base. Across all markets, we see a high rate of compliance and adherence to this oral treatment option.
Pompe disease program milestones. Pombiliti and Opfolda were approved by the EC in June 2023 and MHRA in August 2023. The regulatory reviews in the U.S. remain ongoing. Additionally, multiple expanded access mechanisms are in place around the globe, including in the U.S., U.K., Japan, and others.
Pipeline advancement and growth. We are leveraging our global capabilities to develop and broaden our lead franchises in Fabry and Pompe disease, with focused discovery work on next generation therapies and novel platform technologies.
Financial strength. Total cash, cash equivalents, and marketable securities as of June 30, 2023 was $265.6 million. Based on the current operating model, we believe that the current cash position, which includes expected revenues, is sufficient to fund our operations and ongoing research programs for at least the next 12 months. Potential impacts of the COVID-19 pandemic, business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact our future capital requirements.
 Our Commercial Product and Product Candidates
Galafold® (migalastat HCl) for Fabry Disease
Our oral precision medicine Galafold® was granted accelerated approval by the FDA in August 2018 under the brand name Galafold® 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. The FDA has approved Galafold® for 351 amenable GLA variants. 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 mutation (variant). The approved E.U. and U.K. labels include 1,384 mutations amenable to Galafold® treatment, which represent up to half of all patients with Fabry disease. 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, including for adolescents aged 12 years and older.
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. Galafold® is an oral precision medicine intended to treat Fabry disease in patients who have amenable genetic variants, and at this time, it is not intended for concomitant use with ERT.
The Galafold® U.S. patent portfolio encompasses 53 Orange Book listed patents, including 9 composition-of-matter patents, of which 37 provide protection through at least 2038.
Next Generation for Fabry Disease
We are committed to continued innovation for all people living with Fabry disease. As part of our long-term commitment, we have an academic research collaboration agreement to explore next generation pharmacological chaperones for Fabry disease.
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Novel ERT for Pompe Disease
We are leveraging our biologics capabilities to develop AT-GAA, a novel treatment paradigm for Pompe disease. AT-GAA consists of a uniquely engineered rhGAA enzyme, ATB200, or cipaglucosidase alfa, with an optimized carbohydrate structure to enhance lysosomal uptake, administered in combination with AT2221, or miglustat, that functions as an enzyme stabilizer. Miglustat binds to and stabilizes ATB200 preventing inactivation of rhGAA in circulation to improve the uptake of active enzyme in key disease-relevant tissues, resulting in increased clearance of accumulated substrate, ("glycogen"). Miglustat is not an active ingredient that contributes directly to glycogen reduction.
In February 2021, we reported topline results from the Phase 3 PROPEL study. Of the Pompe disease patients enrolled, 77% were being treated with alglucosidase alfa (n=95) immediately prior to enrollment (“Switch”) and 23% had never been treated with any ERT (n=28) (“Naïve”). Nearly all patients from the PROPEL study continue to be treated with AT-GAA in the extension clinical study. The clinical data from the PROPEL study, the extension study as well as the Phase 1/2 study were included in the AT-GAA submissions to the FDA and the EMA.
In October 2022 and February 2023, we reported positive long-term data from our ongoing phase 1/2 clinical study and Phase 3 open-label extension study, respectively. Phase 1/2 and 3 study participants treated with AT-GAA for up to 48 months and up to 2 years, respectively, demonstrated persistent and durable effects on six-minute walk test distance and measures of motor function and muscle strength, stability, or increase in forced vital capacity, and reductions in biomarkers of muscle damage and disease substrate.
Pombiliti and Opfolda were approved by the EC in June 2023 and MHRA in August 2023. We began launch activities for Pombiliti and Opfolda in Germany and will commence reimbursement processes with healthcare authorities in other European countries.
In addition, we are conducting ongoing clinical studies in pediatric patients for both LOPD and infantile-onset Pompe disease ("IOPD") populations.
Next Generation for Pompe Disease
We are committed to continued innovation for all people living with Pompe disease. As part of our long-term commitment, we are also continuing discovery for next-generation genetic medicines for Pompe disease.
Additional Development and Next Generation Programs
We are researching potential therapies for CDKL5 deficiency disorder ("CDD"). We are collaborating with the LouLou Foundation to assess the natural history of the disease to identify endpoints for potential use in future studies. We also have a number of additional gene therapies in clinical and preclinical development, including potential gene therapies in multiple forms of Batten disease.
Strategic Alliances and Arrangements
We will continue to evaluate business development opportunities as appropriate to build stockholder value and provide us with access to the financial, technical, clinical, and commercial resources necessary to develop and market technologies or products with a focus on rare and orphan diseases. We are exploring potential collaborations, alliances, and 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 platform technologies consistent with our strategic plan to develop and provide therapies to patients living with rare and orphan diseases.

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Consolidated Results of Operations
Three Months Ended June 30, 2023 compared to June 30, 2022
The following table provides selected financial information for the Company:
Three Months Ended June 30,
(in thousands)20232022Change
Net product sales$94,503 $80,731 $13,772 
Cost of goods sold9,114 8,197 917 
Cost of goods sold as a percentage of net product sales9.6 %10.2 %(0.6)%
Operating expenses:
Research and development35,149 78,319 (43,170)
Selling, general, and administrative65,423 53,379 12,044 
Changes in fair value of contingent consideration payable337 115 222 
Loss on impairment of assets1,134 — 1,134 
Depreciation and amortization2,206 1,334 872 
Other (expense) income:
Interest income1,737 356 1,381 
Interest expense(12,492)(8,257)(4,235)
Other (expense) income(10,902)7,268 (18,170)
Income tax expense(2,715)(911)(1,804)
Net loss attributable to common stockholders$(43,232)$(62,157)$18,925 
Net Product Sales. Net product sales increased $13.8 million during the three months ended June 30, 2023 compared to the same period in the prior year. The increase was primarily due to continued growth in the U.S., Europe and Japan markets.
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 June 30,
Projects20232022
Third party direct project expenses  
Galafold® (Fabry Disease)
$3,822 $3,419 
AT-GAA (Pompe Disease)13,117 21,585 
Gene therapy programs520 27,225 
Pre-clinical and other programs345 — 
Total third-party direct project expenses17,804 52,229 
Other project costs  
Personnel costs13,662 18,152 
Other costs3,683 7,938 
Total other project costs17,345 26,090 
Total research and development costs$35,149 $78,319 
The $43.2 million decrease in research and development costs was primarily driven by the strategic prioritization of our gene therapy portfolio, which resulted in the recognition of contract exit costs in the prior year. Additionally, Pompe disease program spend decreased due to reduced clinical manufacturing costs. Personnel costs decreased in connection with the reallocation of resources to support our anticipated AT-GAA commercial launch and continued growth of Galafold®.
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Selling, General, and Administrative Expense. Selling, general, and administrative expense increased $12.0 million, primarily driven by personnel costs in connection with the reallocation of resources to support AT-GAA commercial launch activities and third-party professional fees, partially offset by the write-off of cloud computing costs and software licensing fees in the prior period in connection with the strategic prioritization of our gene therapy portfolio.
Interest Expense. The $4.2 million variance was due to a higher variable interest rate on debt period over period.
Other (Expense) Income. The $18.2 million variance was primarily related to foreign exchange losses caused by remeasurement of foreign-denominated balances.
Consolidated Results of Operations
Six Months Ended June 30, 2023 compared to June 30, 2022
The following table provides selected financial information for the Company:
Six Months Ended June 30,
(in thousands)20232022Change
Net product sales$180,773 $159,446 $21,327 
Cost of goods sold16,056 15,779 277 
Cost of goods sold as a percentage of net product sales8.9 %9.9 %(1.0)%
Operating expenses:
Research and development76,648 159,836 (83,188)
Selling, general, and administrative139,380 111,495 27,885 
Changes in fair value of contingent consideration payable588 (1,073)1,661 
Loss on impairment of assets1,134 6,616 (5,482)
Depreciation and amortization3,463 2,745 718 
Other (expense) income:
Interest income3,936 489 3,447 
Interest expense(24,336)(16,404)(7,932)
Other (expense) income(16,840)9,170 (26,010)
Income tax expense(2,428)(4,720)2,292 
Net loss attributable to common stockholders$(96,164)$(147,417)$51,253 
Net Product Sales. Net product sales increased $21.3 million during the six months ended June 30, 2023 compared to the same period in the prior year. The increase was primarily due to continued growth in the U.S., Europe and Japan markets, partially offset by the $4.1 million unfavorable impact of foreign currency exchange.
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 decreased 1.0% primarily due to the increased proportion of sales in countries not subject to royalties.

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Research and Development Expense. The following table summarizes our principal development programs and the out-of-pocket, third-party expenses incurred:
(in thousands)Six Months Ended June 30,
Projects20232022
Third party direct project expenses  
Galafold® (Fabry Disease)
$6,466 $7,039 
AT-GAA (Pompe Disease)28,893 48,563 
Gene therapy programs755 44,917 
Pre-clinical and other programs710 93 
Total third-party direct project expenses36,824 100,612 
Other project costs  
Personnel costs31,914 43,827 
Other costs7,910 15,397 
Total other project costs39,824 59,224 
Total research and development costs$76,648 $159,836 
The $83.2 million decrease in research and development costs was primarily driven by the strategic prioritization of our gene therapy portfolio, which resulted in the recognition of contract exit costs in the prior year. Additionally, Pompe disease program spend decreased due to reduced clinical manufacturing costs. Personnel costs decreased in connection with the reallocation of resources to support our anticipated AT-GAA commercial launch and continued growth of Galafold®.
Selling, General, and Administrative Expense. Selling, general, and administrative expense increased $27.9 million, primarily driven by personnel costs in connection with the reallocation of resources to support our anticipated AT-GAA commercial launch and third-party professional fees, partially offset by the write-off of cloud computing costs and software licensing fees in the prior year in connection with the strategic prioritization of our gene therapy portfolio.
Loss on Impairment of Assets. Loss on impairment of assets decreased $5.5 million primarily in connection with the strategic prioritization of our gene therapy portfolio in the prior year, which resulted in us recognizing a loss on impairment of assets.
Interest Expense. The $7.9 million variance was due to a higher variable interest rate on debt period over period.
Other (Expense) Income. The $26.0 million variance was primarily related to foreign exchange losses caused by remeasurement of foreign-denominated balances.
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®, 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, Galafold® revenues, debt issuance, collaborations, and other financing arrangements.
Sources of Liquidity
In November 2022, we entered into a Sales Agreement with The 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. During the three and six months ended June 30, 2023, we issued and sold an aggregate of 2,047,353 and 2,242,582 shares through our ATM program at a weighted-average public offering price of $12.21 and $12.25 per share, resulting in net proceeds of $24.2 million and $26.6 million, respectively. As of June 30, 2023, an aggregate of $222.5 million worth of shares remain available to be issued and sold under the ATM program.
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Cash Flow Discussion
As of June 30, 2023, we had cash, cash equivalents, and marketable securities of $265.6 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 4. 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 six months ended June 30, 2023 was $34.2 million. The components of net cash used in operations included the net loss for the six months ended June 30, 2023 of $96.2 million offset by $51.5 million of stock compensation, $26.4 million of other non-cash adjustments, and a net increase in changes in operating assets and liabilities of $15.9 million. The changes in operating assets and liabilities were primarily due to an increase in inventory of $27.5 million and an increase in prepaid expenses and other current assets of $12.3 million, partially offset by an increase in accounts payable and accrued expenses of $31.6 million associated with Pombiliti and Opfolda launch activities and increases in sales rebates associated with increased commercial sales of Galafold®.
Net cash used in operations for the six months ended June 30, 2022 was $74.2 million. The components of net cash used in operations included the net loss for the six months ended June 30, 2022 of $147.4 million offset by $43.1 million of stock compensation, $15.6 million of other non-cash adjustments, and a net increase in changes in operating assets and liabilities of $14.5 million. The changes in operating assets and liabilities were primarily due to an increase in accounts payable and accrued expenses of $22.7 million associated with the strategic prioritization of our gene therapy portfolio resulting in the non-recurring expense of contractual obligations from which we will no longer receive further economic benefit and tax accruals, offset by payments of contract manufacturing, third party research and development services, and annual performance bonuses. The net cash used in operations was also impacted by an increase in accounts receivable of $4.4 million due to increased commercial sales of Galafold®.
Net Cash Provided by Investing Activities
Net cash provided by investing activities for the six months ended June 30, 2023 was $86.3 million. Our investing activities have consisted primarily of purchases, sales, and maturities of investments and capital expenditures. Net cash provided by investing activities reflects $126.8 million for the sale and redemption of marketable securities, partially offset by $36.4 million for the purchase of marketable securities and $4.1 million for capital expenditures.
Net cash provided by investing activities for the six months ended June 30, 2022 was $84.5 million. Our investing activities have consisted primarily of purchases, sales and maturities of investments and capital expenditures. Net cash provided by investing activities reflects $184.1 million for the sale and redemption of marketable securities, partially offset by $98.3 million for the purchase of marketable securities and $1.2 million for capital expenditures.
Net Cash Provided by (Used in) Financing Activities
Net cash provided by financing activities for the six months ended June 30, 2023 was $15.8 million. Net cash provided by financing activities primarily reflects $26.6 million of proceeds from the issuance of shares in connection with the ATM program offering, net of issuance costs, and $4.4 million of proceeds from the exercise of stock options, partially offset by the withholding taxes paid on vested restricted stock units of $14.0 million.
Net cash used in financing activities for the six months ended June 30, 2022 was $7.5 million. Net cash used in financing activities primarily reflects the withholding taxes paid on vested restricted stock units of $9.3 million, partially offset by $1.9 million of proceeds from the exercise of stock options.
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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 Pompe Enzyme Replacement Therapy ("ERT" or "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 these therapies and obtain market acceptance for such therapies;
the costs, timing, and outcome of regulatory review of our product candidates, including AT-GAA;
any changes in regulatory standards relating to the review of our product candidates, including AT-GAA;
the number and development requirements of other product candidates that we pursue;
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 product and product candidates, including AT-GAA;
our ability to successfully commercialize Galafold® (also referred to as "migalastat HCl");
our ability to successfully commercialize Pombiliti and Opfolda (also referred to as AT-GAA) in the E.U., and elsewhere, if our regulatory applications for AT-GAA are approved;
our ability to manufacture or supply sufficient clinical or commercial products, including Galafold®, Pombiliti and Opfolda;
our ability to obtain reimbursement for Galafold®;
our ability to obtain reimbursement for Pombiliti and Opfolda in the E.U., and elsewhere, if our regulatory applications for AT-GAA are approved;
our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of Galafold®;
our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of Pombiliti and Opfoldain the E.U., and elsewhere, if our regulatory applications for AT-GAA are approved;
our ability to obtain market acceptance of Galafold®;
our ability to obtain market acceptance of Pombiliti and Opfoldain the E.U., and elsewhere, if our regulatory applications for AT-GAA are approved;
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 payments from any such collaborators;
the extent to which our business could be adversely impacted by the effects of the novel coronavirus ("COVID-19") outbreak, including actions by us, governments, our customers, our suppliers, or other third parties to control the spread of COVID-19, or by other health epidemics or pandemics;