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, ~~2021~~2022

☐ 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,

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 class	Trading Symbol(s)	Name of each exchange on which registered
Common Stock, par value $0.01 per share	FOLD	NASDAQ 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 27, ~~2021~~2022 was ~~266,555,274~~280,498,461 shares.

AMICUS THERAPEUTICS, INC.

Form 10-Q for the Quarterly Period Ended June 30, ~~2021~~2022

Page

PART I. FINANCIAL INFORMATION

Item 1.

Consolidated Financial Statements and Notes (unaudited)

Consolidated Balance Sheets as of June 30 3 0 , ~~2021~~2022 and December 31, ~~2020~~2021

Consolidated Statements of Operations for the ~~Three~~Three and Six Months Ended June 3 300 , ~~2021~~2022 and ~~2020~~2021

Consolidated Statements of Comprehensive Loss for the Three and ~~and~~ Six Months Ended June 3 0 , ~~2021~~2022 and ~~2020~~2021

Consolidated Statements of Changes in Stockholders' Equity for the ~~Three~~ Three and ~~Six~~ Six Months Ended June 3 0 30 , ~~2021~~2022 and ~~2020~~2021

Consolidated Statements of Cash Flows for the Six Months Ended June 30 3 0 , ~~2021~~2022 and ~~2020~~2021

Notes to Consolidated Financial Statements

Item 2.

Management’s Discussion and Analysis of Financial Condition and Results of Operations

Item 3.

Quantitative and Qualitative Disclosures about Market Risk

3029

Item 4.

Controls and Procedures

3029

PART II. OTHER INFORMATION

Item 1.

Item 1A.

Item 2.

Unregistered Sales of Equity Securities and Use of Proceeds

3129

Item 3.

Defaults Upon Senior Securities

3130

Item 4.

Mine Safety Disclosures

Item 5.

Item 6.

We have filed applications to register certain trademarks in the United States and abroad, including AMICUS THERAPEUTICS and design, AMICUS ASSIST and design, CHART and design, AT THE FOREFRONT OF THERAPIES FOR RARE AND ORPHAN DISEASES, HEALING BEYOND DISEASE, OUR GOOD STUFF, and Galafold® and design.

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," "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 our clinical trials of our drug ~~candidates and gene therapy candidates, including but not limited to AT-GAA, CLN6 and CLN3;~~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") ~~and gene therapies;~~;

•the future results of ~~on-going~~ preclinical research and subsequent clinical trials for cyclin-dependent kinase-like 5 ("CDKL5") deficiency disorder, Pompe gene therapy, Fabry gene therapy, Mucopolysaccharidosis Type IIIB ("MPS IIIB"), next generation Mucopolysaccharidosis Type IIIA ("MPS IIIA") and other 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;~~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;

•our ability to successfully commercialize Galafold® (also referred to as "migalastat HCl") and, if our regulatory ~~filings~~applications are ~~accepted and~~ approved, AT-GAA;

•our ability to manufacture or supply sufficient clinical or commercial products, including Galafold®~~, AT-GAA~~ and ~~our gene therapy candidates;~~AT-GAA;

•our ability to obtain reimbursement for Galafold® and, if our regulatory ~~filings~~applications are ~~accepted and~~ approved, AT-GAA;

•our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of Galafold®;,and, if approved and applicable, AT-GAA;

•our ability to obtain market acceptance of Galafold® and, if our regulatory ~~filings~~applications are ~~accepted and~~ approved, AT-GAA;

•the costs of preparing, filing, and prosecuting patent applications and maintaining, enforcing, and defending intellectual property-related ~~claims;~~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;

•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;

•~~our ability to adjust to changes in the European and United Kingdom markets in the wake of the United Kingdom leaving the European Union;~~

•the extent to which our business could be adversely impacted by the effects of the novel coronavirus ("COVID-19") outbreak, including due to actions by us, governments, our customers, orour 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, ~~2020,~~2021, 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 ~~therein~~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 ~~acquisitions, mergers, dispositions, joint ventures,~~ 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, ~~2020~~2021 (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.

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,
2021 December 31, 2020
Assets
Current assets:
Cash and cash equivalents $ 176,538 $ 163,240
Investments in marketable securities 206,530 320,029
Accounts receivable 49,172 46,923
Inventories 24,086 19,556
Prepaid expenses and other current assets 24,176 29,721
Total current assets 480,502 579,469
Operating lease right-of-use assets, less accumulated amortization of $8,150 and $7,574 at June 30, 2021 and December 31, 2020, respectively 22,028 23,296
Property and equipment, less accumulated depreciation of $17,410 and $14,487 at June 30, 2021 and December 31, 2020, respectively 42,365 43,863
In-process research & development 23,000 23,000
Goodwill 197,797 197,797
Other non-current assets 21,200 19,095
Total Assets $ 786,892 $ 886,520
Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable $ 13,762 $ 17,063
Accrued expenses and other current liabilities 71,325 96,841
Contingent consideration payable 19,800 8,900
Operating lease liabilities 7,106 6,872
Total current liabilities 111,993 129,676
Deferred reimbursements 7,406 7,406
Long-term debt 390,434 389,254
Contingent consideration payable 7,517 16,925
Deferred income taxes 4,896 4,896
Operating lease liabilities 44,201 45,604
Other non-current liabilities 6,535 6,379
Total liabilities 572,982 600,140
Commitments and contingencies 0 0
Stockholders’ equity:
Common stock, $0.01 par value, 500,000,000 shares authorized, 266,532,536 and 262,063,461 shares issued and outstanding at June 30, 2021 and December 31, 2020, respectively 2,685 2,650
Additional paid-in capital 2,364,494 2,308,578
Accumulated other comprehensive income (loss):
Foreign currency translation adjustment 9,255 8,412
Unrealized loss on available-for-sale securities (173) (185)
Warrants 0 12,387
Accumulated deficit (2,162,351) (2,045,462)
Total stockholders’ equity 213,910 286,380
Total Liabilities and Stockholders’ Equity $ 786,892 $ 886,520


	June 30, 2022		December 31, 2021
Assets
Current assets:
Cash and cash equivalents	$	235,639	$	245,197
Investments in marketable securities	151,202		237,299
Accounts receivable	52,556		52,672
Inventories	20,879		26,818
Prepaid expenses and other current assets	37,367		34,848
Total current assets	497,643		596,834
Operating lease right-of-use assets, net	30,447		20,586
Property and equipment, less accumulated depreciation of $22,188 and $19,882 at June 30, 2022 and December 31, 2021, respectively	33,657		42,496
In-process research & development	23,000		23,000
Goodwill	197,797		197,797
Other non-current assets	18,045		24,427
Total Assets	$	800,589	$	905,140
Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable	$	23,113	$	21,513
Accrued expenses and other current liabilities	114,703		98,153
Contingent consideration payable	19,266		18,900
Operating lease liabilities	7,543		7,409
Total current liabilities	164,625		145,975
Long-term debt	390,652		389,357
Operating lease liabilities	52,844		43,363
Deferred reimbursements	5,906		5,906
Deferred income taxes	4,930		4,930
Other non-current liabilities	8,207		8,240
Total liabilities	627,164		597,771
Commitments and contingencies	0		0
Stockholders’ equity:
Common stock, $0.01 par value, 500,000,000 shares authorized, 280,456,667 and 278,912,800 shares issued and outstanding at June 30, 2022 and December 31, 2021, respectively	2,811		2,808
Additional paid-in capital	2,631,110		2,595,419
Accumulated other comprehensive (loss) gain:
Foreign currency translation adjustment	(16,603)		5,251
Unrealized loss on available-for-sale securities	(637)		(270)
Warrants	83		83
Accumulated deficit	(2,443,339)		(2,295,922)
Total stockholders’ equity	173,425		307,369
Total Liabilities and Stockholders’ Equity	$	800,589	$	905,140

See accompanying Notes to Consolidated Financial Statements

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,
	2022		2021		2022		2021
Net product sales	$	80,731	$	77,413	$	159,446	$	143,815
Cost of goods sold	8,197		8,380		15,779		14,919
Gross profit	72,534		69,033		143,667		128,896
Operating expenses:
Research and development	78,319		63,003		159,836		127,120
Selling, general, and administrative	53,379		42,276		111,495		89,002
Changes in fair value of contingent consideration payable	115		1,021		(1,073)		1,492
Loss on impairment of assets	—		—		6,616		—
Depreciation and amortization	1,334		1,567		2,745		3,171
Total operating expenses	133,147		107,867		279,619		220,785
Loss from operations	(60,613)		(38,834)		(135,952)		(91,889)
Other (expense) income:
Interest income	356		50		489		215
Interest expense	(8,257)		(8,150)		(16,404)		(16,142)

Other income	7,268		234		9,170		(2,966)
Loss before income tax	(61,246)		(46,700)		(142,697)		(110,782)
Income tax expense	(911)		(4,525)		(4,720)		(6,107)
Net loss attributable to common stockholders	$	(62,157)	$	(51,225)	$	(147,417)	$	(116,889)
Net loss attributable to common stockholders per common share — basic and diluted	$	(0.21)	$	(0.19)	$	(0.51)	$	(0.44)
Weighted-average common shares outstanding — basic and diluted	291,970,562		266,398,516		288,646,587		265,384,865

Three Months Ended June 30, Six Months Ended June 30,
2021 2020 2021 2020
Net product sales $ 77,413 $ 62,353 $ 143,815 $ 122,878
Cost of goods sold 8,380 6,676 14,919 13,228
Gross profit 69,033 55,677 128,896 109,650
Operating expenses:
Research and development 63,003 69,611 127,120 158,731
Selling, general, and administrative 42,276 34,657 89,002 74,872
Changes in fair value of contingent consideration payable 1,021 715 1,492 1,646
Depreciation and amortization 1,567 2,039 3,171 3,803
Total operating expenses 107,867 107,022 220,785 239,052
Loss from operations (38,834) (51,345) (91,889) (129,402)
Other income (expense):
Interest income 50 865 215 2,380
Interest expense (8,150) (3,635) (16,142) (7,364)
Other expense 234 5,326 (2,966) (2,990)
Loss before income tax (46,700) (48,789) (110,782) (137,376)
Income tax expense (4,525) (3,703) (6,107) (4,064)
Net loss attributable to common stockholders $ (51,225) $ (52,492) $ (116,889) $ (141,440)
Net loss attributable to common stockholders per common share — basic and diluted $ (0.19) $ (0.20) $ (0.44) $ (0.55)
Weighted-average common shares outstanding — basic and diluted 266,398,516 257,973,329 265,384,865 257,548,623

See accompanying Notes to Consolidated Financial Statements

Amicus Therapeutics, Inc.

Consolidated Statements of Comprehensive Loss

(Unaudited)

(in thousands)
Three Months Ended June 30, Six Months Ended June 30,
2021 2020 2021 2020
Net loss $ (51,225) $ (52,492) $ (116,889) $ (141,440)
Other comprehensive gain (loss):
Foreign currency translation adjustment gain (loss), net of tax impact of $52, $(2,090), $184, and $(554), respectively 235 (2,116) 843 2,080
Unrealized gain on available-for-sale securities, net of tax impact of $3, $122, $3, and $66, respectively 12 457 12 248
Other comprehensive income $ 247 $ (1,659) $ 855 $ 2,328
Comprehensive loss $ (50,978) $ (54,151) $ (116,034) $ (139,112)


	Three Months Ended June 30,				Six Months Ended June 30,
	2022		2021		2022		2021
Net loss	$	(62,157)	$	(51,225)	$	(147,417)	$	(116,889)
Other comprehensive (loss) gain:
Foreign currency translation adjustment (loss) gain	$	(16,183)	$	235	$	(21,854)	$	843
Unrealized (loss) gain on available-for-sale securities	$	(29)	$	12	$	(367)	$	12
Other comprehensive (loss) gain	$	(16,212)	$	247	$	(22,221)	$	855
Comprehensive loss	$	(78,369)	$	(50,978)	$	(169,638)	$	(116,034)

See accompanying Notes to Consolidated Financial Statements

Amicus Therapeutics, Inc.

Consolidated Statements of Changes in Stockholders' Equity

(Unaudited)

(in thousands, except share amounts)
Three Months Ended June 30, 2021
Common Stock Additional
Paid-In
Capital Other
Comprehensive
Gain (Loss) Accumulated
Deficit Total
Stockholders'
Equity
Shares Amount
Balance at March 31, 2021 266,007,718 $ 2,680 $ 2,350,507 $ 8,835 $ (2,111,126) $ 250,896
Stock options exercised, net 434,551 5 2,495 — — 2,500
Employee withholding taxes related to restricted stock unit vesting 90,267 — (244) — — (244)
Stock-based compensation — — 11,736 — — 11,736
Unrealized holding gain on available-for-sale securities — — — 12 — 12
Foreign currency translation adjustment — — — 235 — 235
Net loss — — — — (51,225) (51,225)
Balance at June 30, 2021 266,532,536 $ 2,685 $ 2,364,494 $ 9,082 $ (2,162,351) $ 213,910


Three Months Ended June 30, 2022

	Common Stock			Additional Paid-In Capital		Warrants		Other Comprehensive Gain (Loss)		Accumulated Deficit		Total Stockholders' Equity
	Shares	Amount
Balance at March 31, 2022	280,133,856	$	2,809	$	2,617,935	$	83	$	(1,028)	$	(2,381,182)	$	238,617
Stock options exercised, net	189,256	2		997		—		—		—		999
Vesting of restricted stock units, net of taxes	133,555	—		(285)		—		—		—		(285)
Stock-based compensation	—	—		12,463		—		—		—		12,463
Unrealized holding 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, 2022	280,456,667	$	2,811	$	2,631,110	$	83	$	(17,240)	$	(2,443,339)	$	173,425

Six Months Ended June 30, 2021
Common Stock Additional
Paid-In
Capital Warrants Other
Comprehensive
Gain (Loss) Accumulated
Deficit Total
Stockholders'
Equity
Shares Amount
Balance at December 31, 2020 262,063,461 $ 2,650 $ 2,308,578 $ 12,387 $ 8,227 $ (2,045,462) $ 286,380
Stock options exercised, net 922,662 9 6,652 — — — 6,661
Employee withholding taxes related to restricted stock unit vesting 987,330 — (14,438) — — — (14,438)
Stock-based compensation — — 32,090 — — — 32,090
Warrants exercised 2,554,999 26 31,591 (12,387) — — 19,230
Equity component of the convertible notes 4,084 — 21 — — — 21
Unrealized holding gain on available-for-sale securities — — — — 12 — 12
Foreign currency translation adjustment — — — — 843 — 843
Net loss — — — — — (116,889) (116,889)
Balance at June 30, 2021 266,532,536 $ 2,685 $ 2,364,494 $ 0 $ 9,082 $ (2,162,351) $ 213,910


Six Months Ended June 30, 2022

	Common Stock			Additional Paid-In Capital		Warrants		Other Comprehensive Gain (Loss)		Accumulated Deficit		Total Stockholders' Equity
	Shares	Amount
Balance at December 31, 2021	278,912,800	$	2,808	$	2,595,419	$	83	$	4,981	$	(2,295,922)	$	307,369
Stock options exercised, net	334,705	3		1,855		—		—		—		1,858
Vesting of restricted stock units, net of taxes	1,209,162	—		(9,278)		—		—		—		(9,278)
Stock-based compensation	—	—		43,114		—		—		—		43,114
Unrealized holding 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, 2022	280,456,667	$	2,811	$	2,631,110	$	83	$	(17,240)	$	(2,443,339)	$	173,425

~~Amicus Therapeutics, Inc.~~

~~Consolidated Statements of Changes in Stockholders' Equity~~

~~(Unaudited)~~

~~(in thousands, except share amounts)~~


Three Months Ended June 30, 2020
Three Months Ended June 30, 2021															Three Months Ended June 30, 2021

		Common Stock			Additional Paid-In Capital		Warrants		Other Comprehensive Gain (Loss)		Accumulated Deficit		Total Stockholders' Equity			Common Stock			Additional Paid-In Capital		Other Comprehensive Gain (Loss)		Accumulated Deficit		Total Stockholders' Equity
		Shares	Amount													Shares	Amount
Balance at March 31, 2020		257,449,955	$	2,607	$	2,238,346	$	12,387	$	6,812	$	(1,857,558)	$	402,594
Balance at March 31, 2021															Balance at March 31, 2021	266,007,718	$	2,680	$	2,350,507	$	8,835	$	(2,111,126)	$	250,896
Stock options exercised, net	Stock options exercised, net	650,288	7		4,649		—		—		—		4,656		Stock options exercised, net	434,551	5		2,495		—		—		2,500
Employee withholding taxes related to restricted stock unit vesting		123,599	—		(554)		—		—		—		(554)
Vesting of restricted stock units, net of taxes															Vesting of restricted stock units, net of taxes	90,267	—		(244)		—		—		(244)
Stock-based compensation	Stock-based compensation	—	—		8,408		—		—		—		8,408		Stock-based compensation	—	—		11,736		—		—		11,736
Unrealized holding gain on available-for-sale securities	Unrealized holding gain on available-for-sale securities	—	—		—		—		457		—		457		Unrealized holding gain on available-for-sale securities	—	—		—		12		—		12
Foreign currency translation adjustment	Foreign currency translation adjustment	—	—		—		—		(2,116)		—		(2,116)		Foreign currency translation adjustment	—	—		—		235		—		235
Net loss	Net loss	—	—		—		—		—		(52,492)		(52,492)		Net loss	—	—		—		—		(51,225)		(51,225)
Balance at June 30, 2020		258,223,842	$	2,614	$	2,250,849	$	12,387	$	5,153	$	(1,910,050)	$	360,953
Balance at June 30, 2021															Balance at June 30, 2021	266,532,536	$	2,685	$	2,364,494	$	9,082	$	(2,162,351)	$	213,910


Six Months Ended June 30, 2020
Six Months Ended June 30, 2021															Six Months Ended June 30, 2021

		Common Stock			Additional Paid-In Capital		Warrants		Other Comprehensive Gain (Loss)		Accumulated Deficit		Total Stockholders' Equity			Common Stock			Additional Paid-In Capital		Warrants		Other Comprehensive Gain (Loss)		Accumulated Deficit		Total Stockholders' Equity
		Shares	Amount													Shares	Amount
Balance at December 31, 2019		255,417,869	$	2,598	$	2,227,225	$	12,387	$	2,825	$	(1,768,610)	$	476,425
Balance at December 31, 2020															Balance at December 31, 2020	262,063,461	$	2,650	$	2,308,578	$	12,387	$	8,227	$	(2,045,462)	$	286,380
Stock options exercised, net	Stock options exercised, net	1,609,235	16		10,717		—		—		—		10,733		Stock options exercised, net	922,662	9		6,652		—		—		—		6,661
Employee withholding taxes related to restricted stock unit vesting		1,196,738	—		(8,097)		—		—		—		(8,097)
Vesting of restricted stock units, net of taxes															Vesting of restricted stock units, net of taxes	987,330	—		(14,438)		—		—		—		(14,438)
Stock-based compensation	Stock-based compensation	—	—		21,004		—		—		—		21,004		Stock-based compensation	—	—		32,090		—		—		—		32,090
Warrants exercised															Warrants exercised	2,554,999	26		31,591		(12,387)		—		—		19,230
Equity component of the convertible notes															Equity component of the convertible notes	4,084	—		21		—		—		—		21
Unrealized holding gain on available-for-sale securities	Unrealized holding gain on available-for-sale securities	—	—		—		—		248		—		248		Unrealized holding gain on available-for-sale securities	—	—		—		—		12		—		12
Foreign currency translation adjustment	Foreign currency translation adjustment	—	—		—		—		2,080		—		2,080		Foreign currency translation adjustment	—	—		—		—		843		—		843
Net loss	Net loss	—	—		—		—		—		(141,440)		(141,440)		Net loss	—	—		—		—		—		(116,889)		(116,889)
Balance at June 30, 2020		258,223,842	$	2,614	$	2,250,849	$	12,387	$	5,153	$	(1,910,050)	$	360,953
Balance at June 30, 2021															Balance at June 30, 2021	266,532,536	$	2,685	$	2,364,494	$	—	$	9,082	$	(2,162,351)	$	213,910

See accompanying Notes to Consolidated Financial Statements

Amicus Therapeutics, Inc.

Consolidated Statements of Cash Flows

(Unaudited)

(in thousands)


		Six Months Ended June 30,					Six Months Ended June 30,
		2021		2020			2022		2021
Operating activities	Operating activities					Operating activities
Net loss	Net loss	$	(116,889)	$	(141,440)	Net loss	$	(147,417)	$	(116,889)
Adjustments to reconcile net loss to net cash used in operating activities:	Adjustments to reconcile net loss to net cash used in operating activities:					Adjustments to reconcile net loss to net cash used in operating activities:
Amortization of debt discount and deferred financing	Amortization of debt discount and deferred financing	1,200		532		Amortization of debt discount and deferred financing	1,295		1,200
Depreciation and amortization	Depreciation and amortization	3,171		3,803		Depreciation and amortization	2,745		3,171
Stock-based compensation	Stock-based compensation	32,090		21,004		Stock-based compensation	43,114		32,090

Non-cash changes in the fair value of contingent consideration payable	Non-cash changes in the fair value of contingent consideration payable	1,492		1,646		Non-cash changes in the fair value of contingent consideration payable	(1,073)		1,492
Foreign currency remeasurement loss	Foreign currency remeasurement loss	3,235		5,604		Foreign currency remeasurement loss	353		3,235
Asset impairment charges and other asset write-offs						Asset impairment charges and other asset write-offs	12,265		—
Changes in operating assets and liabilities:	Changes in operating assets and liabilities:					Changes in operating assets and liabilities:
Accounts receivable	Accounts receivable	(3,127)		(10,388)		Accounts receivable	(4,424)		(3,127)
Inventories	Inventories	(4,445)		1,351		Inventories	3,537		(4,445)
Prepaid expenses and other current assets	Prepaid expenses and other current assets	5,265		1,375		Prepaid expenses and other current assets	(4,481)		5,265
Accounts payable and accrued expenses		(28,485)		(23,916)
Accounts payable, accrued expenses, and other current liabilities						Accounts payable, accrued expenses, and other current liabilities	22,671		(28,485)
Other non-current assets and liabilities	Other non-current assets and liabilities	(1,784)		(1,031)		Other non-current assets and liabilities	(2,762)		(1,784)
Net cash used in operating activities	Net cash used in operating activities	$	(108,277)	$	(141,460)	Net cash used in operating activities	$	(74,177)	$	(108,277)
Investing activities	Investing activities					Investing activities
Sale and redemption of marketable securities	Sale and redemption of marketable securities	258,767		210,139		Sale and redemption of marketable securities	184,061		258,767
Purchases of marketable securities	Purchases of marketable securities	(145,255)		(45,005)		Purchases of marketable securities	(98,330)		(145,255)
Capital expenditures	Capital expenditures	(1,234)		(1,876)		Capital expenditures	(1,226)		(1,234)
Net cash provided by investing activities	Net cash provided by investing activities	$	112,278	$	163,258	Net cash provided by investing activities	$	84,505	$	112,278
Financing activities	Financing activities					Financing activities
Payment of finance leases	Payment of finance leases	(389)		(38)		Payment of finance leases	(41)		(389)
Payments of employee withholding taxes related to restricted stock unit vesting		(14,438)		(8,097)
Proceeds from warrants exercised						Proceeds from warrants exercised	—		19,230
Purchase of vested restricted stock units, net of taxes						Purchase of vested restricted stock units, net of taxes	(9,278)		(14,438)
Proceeds from stock options exercised, net	Proceeds from stock options exercised, net	6,661		10,734		Proceeds from stock options exercised, net	1,858		6,661
Proceeds from warrants exercised		19,230		0
Net cash provided by financing activities		$	11,064	$	2,599
Net cash (used in) provided by financing activities						Net cash (used in) provided by financing activities	$	(7,461)	$	11,064
Effect of exchange rate changes on cash, cash equivalents, and restricted cash	Effect of exchange rate changes on cash, cash equivalents, and restricted cash	$	(1,439)	$	(3,476)	Effect of exchange rate changes on cash, cash equivalents, and restricted cash	$	(12,462)	$	(1,439)
Net increase in cash, cash equivalents, and restricted cash at the end of the period		13,626		20,921
Cash, cash equivalents, and restricted cash at beginning of period		166,162		146,341
Net (decrease) increase in cash, cash equivalents, and restricted cash at the end of the period						Net (decrease) increase in cash, cash equivalents, and restricted cash at the end of the period	(9,595)		13,626
Cash, cash equivalents, and restricted cash at the beginning of period						Cash, cash equivalents, and restricted cash at the beginning of period	249,456		166,162
Cash, cash equivalents, and restricted cash at the end of period	Cash, cash equivalents, and restricted cash at the end of period	$	179,788	$	167,262	Cash, cash equivalents, and restricted cash at the end of period	$	239,861	$	179,788
Supplemental disclosures of cash flow information	Supplemental disclosures of cash flow information					Supplemental disclosures of cash flow information
Cash paid during the period for interest						Cash paid during the period for interest	$	15,108	$	15,109
Cash paid for taxes						Cash paid for taxes	$	710	$	4,526
Capital expenditures unpaid at the end of period						Capital expenditures unpaid at the end of period	$	53	$	191
Tenant improvements paid through lease incentives	Tenant improvements paid through lease incentives	$	67	$	455	Tenant improvements paid through lease incentives	$	—	$	67
Cash paid during the period for interest		$	15,109	$	10,709
Capital expenditures unpaid at the end of period		$	191	$	502
Cash paid for taxes		$	4,526	$	1,956

See accompanying Notes to Consolidated Financial Statements

Amicus Therapeutics, Inc.

Notes to the Consolidated Financial Statements

(Unaudited)

~~Note~~ 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 a portfolio of product opportunities ~~led by~~including the first, oral monotherapy for Fabry disease that has achieved widespread global approval and a differentiated biologic for Pompe disease, that is under review with the U.S. Food and ~~an industry leading rare disease gene therapy portfolio.~~Drug Administration ("FDA") as well as the European Medicines Agency ("EMA"). 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 United States ("U.S."), European Union ("E.U."), United Kingdom ("U.K."), and Japan, with multiple additional approvals granted and applications pending in several ~~additional~~ geographies around the world.

The lead biologics program of the Company's pipeline is Amicus Therapeutics GAA ("AT-GAA", also known as ATB200/AT2221, or cipaglucosidase alfa/miglustat), a novel, ~~clinical-stage,~~two-component, potential best-in-class treatment ~~paradigm~~ for Pompe disease. In February 2019, the ~~U.S. Food and Drug Administration ("FDA")~~FDA granted Breakthrough Therapy designation ("BTD") to AT-GAA for the treatment of ~~late-onset~~late onset Pompe disease.

In September 2021, the FDA set the Prescription Drug User Fee Act ("PDUFA") target action date of May 29, 2022 for the New Drug Application ("NDA") for miglustat and July 29, 2022 for the Biologics License Application ("BLA") for cipaglucosidase alfa. The Company has established an industry leading gene therapy portfolio of potential therapies for people living with rare metabolic diseases, through a license with Nationwide Children's Hospital ("Nationwide Children's"EMA validated the Marketing Authorization Application (“MAA”) ~~and a research collaboration with~~in the University of Pennsylvania ("Penn"). The Company's pipeline includes gene therapy programs in rare, neurologic lysosomal disorders ("LDs"), specifically: CLN6, CLN3, and CLN1 Batten disease, Pompe disease, Fabry disease, CDKL5 deficiency disorder ("CDD"), Mucopolysaccharidosis Type IIIB ("MPS IIIB"), as well as a next generation program in Mucopolysaccharidosis Type IIIA ("MPS IIIA"). This research collaboration with Penn also provides the Company with exclusive disease-specific access and option rights to develop potentially disruptive new gene therapy platform technologies and programs for most LDs and a broader portfolio of more prevalent rare diseases, including Rett Syndrome, Angelman Syndrome, Myotonic Dystrophy, and select other muscular dystrophies. In the firstfourth quarter of ~~2020,~~2021. On May 9, 2022, the FDA ~~granted Fast Track designation to~~extended the ~~CLN3 Batten disease gene therapy, AT-GTX-502,~~review period for the ~~treatment of pediatric patients less than 18 years of age. In September 2020 and February 2021, the European Medicines Agency granted Priority Medicines designation~~NDA for miglustat and the ~~FDA granted Fast Track Designation, respectively, to the CLN6 Batten disease gene therapy, AT-GTX-501,~~BLA for ~~the treatment~~cipaglucosidase alfa resulting in revised PDUFA action dates of ~~patients with variant late infantile neuronal ceroid lipofuscinosis 6 ("vLINCL6").~~August 29, 2022 and October 29, 2022, respectively.

The Company's operations have not been significantly impacted ~~from~~by the novel coronavirus (“COVID-19”) pandemic ~~thus far. However, the~~to date. The Company continued to observe ~~periodic increase in~~increased lag ~~times~~time between patient identification and Galafold® initiation due to the ~~resurgence~~continued prevalence of COVID-19 ~~into 2021.~~and its ongoing impact on access to treatment for people living with Fabry disease in certain markets. The Company has maintained operations in all geographies, secured its global supply chain for its commercial and clinical products, ~~and~~as well as maintained the operational integrity of its clinical trials, with minimal ~~disruption. The~~disruptions. Whether the Company ~~believes its ability to~~will continue to operate without any significant disruptions will depend on the continued health of its employees, the ongoing demand for Galafold® and the continued operation of its global supply chain. The Company has continued to provide uninterrupted access to medicines for those in need of treatment, while prioritizing the health and safety of its global workforce. However, the Company's results of operations in future periods may be negatively impacted by unknown future impacts from the COVID-19 pandemic.

The Company had an accumulated deficit of ~~$2.2~~$2.4 billion as of June 30, ~~2021~~2022 and anticipates incurring losses through the fiscal year ending December 31, ~~2021~~2022 and beyond. The Company has historically funded its operations through stock offerings, ~~debt issuances,~~ Galafold® revenues, debt issuances, collaborations, and other financing arrangements.

Based on its current operating ~~models,~~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 to achieve self-sustainability. 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.

~~Note~~ 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 ~~financial statements~~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, ~~2020.~~2021. 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, ~~2020.~~2021.

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 COVID-19 pandemic has had on its operations and financial results as of June 30, ~~2021~~2022 and through the issuance of ~~this report.~~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 ~~income (loss)~~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 current assets and 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, 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 related to its product sales of Galafold®. The Company's accounts receivable at June 30, ~~2021~~2022 have arisen from product sales primarily in Europe and the U.S. 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, ~~2021,~~2022, the Company recorded an allowance for doubtful accounts of $0.1 million.

Property and Equipment

Property and equipment are stated at cost, less accumulated depreciation. Depreciation is calculated over the estimated useful lives of the respective assets, which range from three to five years, or the lesser of the related initial term of the lease or useful life for leasehold improvements.

The initial cost of property and equipment consists of its purchase price and any directly attributable costs of bringing the asset to its working condition and location for its intended use. Expenditures incurred after the fixed assets have been put into operation, such as repairs and maintenance, are charged to income in the period in which the costs are incurred. Major replacements, improvements, and additions are capitalized in accordance with Company policy.

The Company evaluates long-lived assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset group may not be recoverable. If indications of impairment exist, projected future undiscounted cash flows associated with the asset or asset group are compared to the carrying value of the asset to determine whether the asset or asset group's value is 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 long-lived asset over its estimated fair value in the period at which such a determination is made.

During the six months ended June 30, 2022, in connection with the strategic prioritization of its gene therapy portfolio, the Company performed an assessment of its fixed assets. As a result, the Company recognized an impairment charge of $6.6 million.

Revenue Recognition

The Company's net product sales consist of sales of Galafold® for the treatment of Fabry disease. The Company has recorded revenue on sales where Galafold® is available either on a commercial basis or through a reimbursed early access ~~program ("EAP").~~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 from Galafold® disaggregated by geographic area:


		Three Months Ended June 30,				Six Months Ended June 30,					Three Months Ended June 30,				Six Months Ended June 30,
(in thousands)	(in thousands)	2021		2020		2021		2020		(in thousands)	2022		2021		2022		2021
U.S.	U.S.	$	23,678	$	20,807	$	44,531	$	38,579	U.S.	$	27,540	$	23,678	$	51,718	$	44,531
Ex-U.S.	Ex-U.S.	53,735		41,546		99,284		84,299		Ex-U.S.	53,191		53,735		107,728		99,284
Total net product sales	Total net product sales	$	77,413	$	62,353	$	143,815	$	122,878	Total net product sales	$	80,731	$	77,413	$	159,446	$	143,815

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 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.

~~Leases~~Research and Development Costs

~~The~~Research and development costs are expensed as incurred. Research and development expense consist primarily of costs related to personnel, including salaries and other personnel related expenses, consulting fees, and the cost of facilities and support services used in drug development. Assets acquired that are used for research and development and have no future alternative use are expensed as in-process research and development.

In the second quarter of 2022, as part of the Company’s strategic prioritization of its gene therapy portfolio, the Company ~~primarily enters into lease agreements~~recorded a non-recurring $20.0 million liability associated with the expense of contractual obligations from which the Company will no longer receive further economic benefit. A liability for ~~office space, equipment,~~costs that will continue to be incurred under a contract for its remaining term without economic benefit to the entity is recognized at the cease-use date. This liability is presented as a component of accrued expenses and vehicles. The leases have varying terms, some of which could include options to renew, extend, and early terminate. The Company determines if an arrangement is a lease at contract inception. Operating leases are included in right-of-use ("ROU") assets and leaseother current liabilities onwithin the Company's Consolidated Balance Sheets.

ROU assets represent the Company's right to control the use of an explicitly or implicitly identified fixed asset for a period of time and lease liabilities represent the Company's obligation to make lease payments arising from the lease. Control of an underlying asset is conveyed to the Company if the Company obtains the rights to direct the use of and to obtain substantially all of the economic benefits from using the underlying asset. ROU assets and liabilities are recognized at commencement date based on the present value of lease payments over the lease term. The Company uses its incremental borrowing rate based on the information available at commencement date in determining the present value of lease payments.

Lease payments included in the measurement of the lease liability are comprised of fixed payments. Variable lease payments are excluded from the ROU asset and lease liability and are recognized in the period in which the obligation for those payments is incurred. Variable lease payments are presented in the Consolidated Statements of Operations in the same line item as expenses arising from fixed lease payments for operating leases. The Company has lease agreements that include lease and non-lease components, which the Company accounts for as a single lease component for all underlying asset categories.

The lease term for all of the Company's leases includes the non-cancellable period of the lease plus any additional periods covered by either a Company option to extend (or not to terminate) the lease that the Company is reasonably certain to exercise, or an option to extend (or not to terminate) the lease controlled by the lessor.

Leases with an initial term of 12 months or less are not recorded on the Consolidated Balance Sheets. The Company recognizes lease expense for these leases on a straight-line basis over the lease term. The Company applies this policy to all underlying asset categories.

Recent Accounting Developments ~~- Guidance Adopted in 2021~~

ASU 2019-12 - In December 2019, the Financial Accounting Standard Board issued Accounting Standard Update ("ASU") 2019-12, Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes ("ASU 2019-12"). This new guidance removes specific exceptions to the general principles in Topic 740. It eliminates the need for an organization to analyze whether the following applies in a given period: (i) exception to the incremental approach for intraperiod tax allocation; (ii) exceptions to accounting for basis differences when there are ownership changes in foreign investments; and (iii) exception in interim period income tax accounting for year-to-date losses that exceed anticipated losses. ASU 2019-12 also improves financial statement preparers’ application of income tax-related guidance and simplifies the following: (i) franchise taxes that are partially based on income; (ii) transactions with a government that result in a step up in the tax basis of goodwill; (iii) separate financial statements of legal entities that are not subject to tax; and (iv) enacted changes in tax laws in interim periods. ASU 2019-12 is effective for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2020. The Company adopted this guidance prospectively on January 1, 2021. The adoption did not have a material impact on the Company's Consolidated Financial Statements or related disclosures.

~~Recent Accounting Developments - Guidance Not Yet Adopted~~

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.

~~Note~~

3. Cash, Cash Equivalents, Marketable Securities, and Restricted Cash

As of June 30, ~~2021,~~2022, the Company held ~~$176.5~~$235.6 million in cash and cash equivalents and ~~$206.5~~$151.2 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 ~~accumulated~~ other comprehensive 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, 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 many of these securities are either government backed or of the highest 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 June 30, 2021									As of June 30, 2022
(in thousands)	(in thousands)	Cost		Gross Unrealized Gain		Gross Unrealized Loss		Fair Value		(in thousands)	Cost		Gross Unrealized Gain		Gross Unrealized Loss		Fair Value
Cash and cash equivalents	Cash and cash equivalents	$	176,538	$	—	$	—	$	176,538	Cash and cash equivalents	$	235,639	$	—	$	—	$	235,639
Corporate debt securities		12,039		0		0		12,039
Commercial paper	Commercial paper	147,317		22		0		147,339		Commercial paper	103,084		1		(239)		102,846
U.S. government agency bonds										U.S. government agency bonds	45,157		—		(193)		44,964
Asset-backed securities	Asset-backed securities	20,681		3		(4)		20,680		Asset-backed securities	3,000		—		(9)		2,991
U.S. government agency bonds		26,067		4		0		26,071
Money market	Money market	350		0		0		350		Money market	350		—		—		350
Certificates of deposit	Certificates of deposit	51		0		0		51		Certificates of deposit	51		—		—		51

		$	383,043	$	29	$	(4)	$	383,068		$	387,281	$	1	$	(441)	$	386,841
Included in cash and cash equivalents	Included in cash and cash equivalents	$	176,538	$	—	$	—	$	176,538	Included in cash and cash equivalents	$	235,639	$	—	$	—	$	235,639
Included in marketable securities	Included in marketable securities	206,505		29		(4)		206,530		Included in marketable securities	151,642		1		(441)		151,202
Total cash, cash equivalents, and marketable securities	Total cash, cash equivalents, and marketable securities	$	383,043	$	29	$	(4)	$	383,068	Total cash, cash equivalents, and marketable securities	$	387,281	$	1	$	(441)	$	386,841


		As of December 31, 2020									As of December 31, 2021
(in thousands)	(in thousands)	Cost		Gross Unrealized Gain		Gross Unrealized Loss		Fair Value		(in thousands)	Cost		Gross Unrealized Gain		Gross Unrealized Loss		Fair Value
Cash and cash equivalents	Cash and cash equivalents	$	163,240	$	—	$	—	$	163,240	Cash and cash equivalents	$	245,197	$	—	$	—	$	245,197
Commercial paper										Commercial paper	174,578		7		(54)		174,531
Corporate debt securities	Corporate debt securities	39,525		4		(16)		39,513		Corporate debt securities	32,322		—		(11)		32,311
Commercial paper		217,087		14		(6)		217,095
Asset-backed securities	Asset-backed securities	9,420		18		0		9,438		Asset-backed securities	30,070		—		(14)		30,056
U.S. government agency bonds		53,583		3		(4)		53,582
Money market	Money market	350		0		0		350		Money market	350		—		—		350
Certificates of deposit		51		0		0		51
Certificate of deposit										Certificate of deposit	51		—		—		51
		$	483,256	$	39	$	(26)	$	483,269		$	482,568	$	7	$	(79)	$	482,496
Included in cash and cash equivalents	Included in cash and cash equivalents	$	163,240	$	—	$	—	$	163,240	Included in cash and cash equivalents	$	245,197	$	—	$	—	$	245,197
Included in marketable securities(1)		320,016		39		(26)		320,029
Included in marketable securities										Included in marketable securities	237,371		7		(79)		237,299
Total cash, cash equivalents, and marketable securities	Total cash, cash equivalents, and marketable securities	$	483,256	$	39	$	(26)	$	483,269	Total cash, cash equivalents, and marketable securities	$	482,568	$	7	$	(79)	$	482,496

For both the six months ended June 30, ~~2021 there were 0 realized gains or losses. For~~2022 and the fiscal year ended December 31, ~~2020,~~2021, there were ~~0minal~~no realized ~~gains.~~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, ~~2021~~2022 and December 31, ~~2020~~2021 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). gain. The fair value of these marketable securities in unrealized loss positions was ~~$17.2~~$140.5 million and ~~$124.9~~$173.4 million as of June 30, ~~2021~~2022 and December 31, ~~2020,~~2021, 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 of the same such amounts shown in the Consolidated Statements of Cash Flows.
(in thousands) June 30, 2021 June 30, 2020
Cash and cash equivalents $ 176,538 $ 164,573
Restricted cash 3,250 2,689
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows $ 179,788 $ 167,262


	As of June 30,
(in thousands)	2022		2021
Cash and cash equivalents	$	235,639	$	176,538
Restricted cash	4,222		3,250
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows	$	239,861	$	179,788

~~Note~~ 4. Inventories

Inventories consist of raw materials, work-in-process, and finished goods related to the manufacture of Galafold®. The following table summarizes the components of inventories:


(in thousands)	(in thousands)	June 30, 2021		December 31, 2020		(in thousands)	June 30, 2022		December 31, 2021
Raw materials	Raw materials	$	7,619	$	5,547	Raw materials	$	11,508	$	12,289
Work-in-process	Work-in-process	11,306		7,693		Work-in-process	5,943		10,699
Finished goods	Finished goods	5,161		6,316		Finished goods	3,428		3,830
Total inventories	Total inventories	$	24,086	$	19,556	Total inventories	$	20,879	$	26,818

The Company recorded a reserve for inventory of ~~$0.2 million and $0.1~~$1.1 million as of both June 30, ~~2021~~2022 and December 31, ~~2020, respectively.~~2021.

~~Note~~ 5. Debt

The Company's debt consists of the following:


(in thousands)	(in thousands)	June 30, 2021		December 31, 2020		(in thousands)	June 30, 2022		December 31, 2021
Senior Secured Term Loan due 2026:	Senior Secured Term Loan due 2026:					Senior Secured Term Loan due 2026:
Principal	Principal	$	400,000	$	400,000	Principal	$	400,000	$	400,000
Less: debt discount (1)	Less: debt discount (1)	(6,794)		(7,434)		Less: debt discount (1)	(5,334)		(6,074)
Less: deferred financing (1)	Less: deferred financing (1)	(5,111)		(5,592)		Less: deferred financing (1)	(4,014)		(4,569)
Net carrying value of the Senior Secured Term Loan		$	388,095	$	386,974

Convertible Notes (2):
Principal (3)		$	2,800	$	2,825
Less: debt discount (1)		(438)		(518)
Less: deferred financing (1)		(23)		(27)
Net carrying value of the Convertible Notes		$	2,339	$	2,280

Net carrying value of Long-term debt	Net carrying value of Long-term debt	$	390,434	$	389,254	Net carrying value of Long-term debt	$	390,652	$	389,357

(1) Included in the Consolidated Balance Sheets within long-term debt and amortized to interest expense over the remaining life of the ~~Convertible Notes and~~ Senior Secured Term Loan due 2026 using the effective interest rate method.

~~(2)~~ The Convertible Notes are currently convertible as the last reported sale price of the Company's common stock was equal to or more than 130% of the conversion price for at least 20 trading days of the 30 consecutive trading days ending on the last day of the quarter.

~~(3)~~ In the first quarter of 2021, the Company exchanged an aggregate principal amount of $25 thousand of Convertible Notes in exchange for an aggregate of approximately 4.1 thousand shares of the Company common stock, par value $0.01 per share.

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, ~~2021~~2022 and ~~2020,~~2021, respectively:


		Three Months Ended June 30,				Six Months Ended June 30,					Three Months Ended June 30,				Six Months Ended June 30,
(in thousands)	(in thousands)	2021		2020		2021		2020		(in thousands)	2022		2021		2022		2021
Contractual interest expense	Contractual interest expense	$	7,604	$	3,410	$	15,125	$	6,995	Contractual interest expense	$	7,589	$	7,604	$	15,089	$	15,125
Amortization of debt discount	Amortization of debt discount	$	383	$	268	$	720	$	474	Amortization of debt discount	$	375	$	383	$	739	$	720
Amortization of deferred financing	Amortization of deferred financing	$	262	$	33	$	485	$	58	Amortization of deferred financing	$	283	$	262	$	556	$	485

~~Note~~ 6. ~~Stockholders' Equity~~

~~During the first quarter of 2021, 1,260,000 and 1,294,999 warrants were exercised at $7.06 and $7.98 per share of common stock, respectively, resulting in gross cash proceeds of $19.2 million.~~

~~Note 7.~~ Share-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,							Three Months Ended June 30,						Six Months Ended June 30,
		2021			2020			2021			2020				2022			2021			2022			2021
Expected stock price volatility	Expected stock price volatility	65.5		%	74.9		%	66.4		%	75.2		%	Expected stock price volatility	61.3		%	65.5		%	62.2		%	66.4		%
Risk free interest rate	Risk free interest rate	0.7		%	0.4		%	0.5		%	1.6		%	Risk free interest rate	3.0		%	0.7		%	1.6		%	0.5		%
Expected life of options (years)	Expected life of options (years)	5.4			5.7			5.4			5.7			Expected life of options (years)	5.3			5.4			5.3			5.4
Expected annual dividend per share	Expected annual dividend per share	$	0		$	0		$	0		$	0		Expected annual dividend per share	$	—		$	—		$	—		$	—

A summary of the Company's stock options for the six months ended June 30, ~~2021~~2022 were as follows:


		Number of Shares	Weighted Average Exercise Price		Weighted Average Remaining Years	Aggregate Intrinsic Value			Number of Shares	Weighted Average Exercise Price		Weighted Average Remaining Years	Aggregate Intrinsic Value
		(in thousands)				(in millions)			(in thousands)				(in millions)
Options outstanding, December 31, 2020		14,032	$	9.54
Options outstanding, December 31, 2021								Options outstanding, December 31, 2021	14,731	$	11.08
Granted	Granted	2,453	$	18.54				Granted	5,402	$	11.55
Exercised	Exercised	(945)	$	7.29				Exercised	(339)	$	5.60
Forfeited	Forfeited	(524)	$	13.04				Forfeited	(333)	$	12.36
Expired	Expired	(54)	$	13.21				Expired	(178)	$	11.98
Options outstanding, June 30, 2021		14,962	$	11.02	6.6	$	15.1
Vested and unvested expected to vest, June 30, 2021		13,586	$	10.80	6.4	$	15.0
Exercisable at June 30, 2021		9,074	$	9.34	5.3	$	14.8
Options outstanding, June 30, 2022								Options outstanding, June 30, 2022	19,283	$	11.28	7.0	$	22.2
Vested and unvested expected to vest, June 30, 2022								Vested and unvested expected to vest, June 30, 2022	17,589	$	11.18	6.8	$	21.5
Exercisable at June 30, 2022								Exercisable at June 30, 2022	10,377	$	10.38	5.2	$	18.3

As of June 30, ~~2021,~~2022, the total unrecognized compensation cost related to non-vested stock options granted was ~~$36.0~~$39.5 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 six months ended June 30, ~~2021~~2022 is as follows:


		Number of Shares	Weighted Average Grant Date Fair Value		Weighted Average Remaining Years	Aggregate Intrinsic Value			Number of Shares	Weighted Average Grant Date Fair Value		Weighted Average Remaining Years	Aggregate Intrinsic Value
		(in thousands)				(in millions)			(in thousands)				(in millions)
Non-vested units as of December 31, 2020		7,080	$	11.35
Non-vested units as of December 31, 2021								Non-vested units as of December 31, 2021	7,341	$	13.90
Granted	Granted	2,451	$	18.89				Granted	4,815	$	11.97
Vested	Vested	(1,513)	$	16.81				Vested	(1,738)	$	12.40
Forfeited	Forfeited	(606)	$	13.77				Forfeited	(252)	$	12.10
Non-vested units as of June 30, 2021		7,412	$	13.96	2.5	$	71.5
Non-vested units as of June 30, 2022								Non-vested units as of June 30, 2022	10,166	$	13.13	2.4	$	109.2

All non-vested units are expected to vest over their normal term. As of June 30, ~~2021,~~2022, there was ~~$57.9~~$65.8 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 Consolidated Statements of Operations related to the equity awards:


		Three Months Ended June 30,				Six Months Ended June 30,					Three Months Ended June 30,				Six Months Ended June 30,
(in thousands)	(in thousands)	2021		2020		2021		2020		(in thousands)	2022		2021		2022		2021
Equity compensation expense recognized in:
Research and development expense	Research and development expense	$	3,152	$	3,362	$	9,457	$	8,615	Research and development expense	$	4,379	$	3,152	$	13,744	$	9,457
Selling, general, and administrative expense	Selling, general, and administrative expense	8,584		5,046		22,633		12,389		Selling, general, and administrative expense	8,084		8,584		29,370		22,633
Total equity compensation expense	Total equity compensation expense	$	11,736	$	8,408	$	32,090	$	21,004	Total equity compensation expense	$	12,463	$	11,736	$	43,114	$	32,090

~~Note 8.~~7. 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 June 30, ~~2021~~2022 are identified in the following tables:


(in thousands)	(in thousands)	Level 2		Total		(in thousands)	Level 2		Total
Assets:	Assets:					Assets:
Commercial paper	Commercial paper	$	147,339	$	147,339	Commercial paper	$	102,846	$	102,846
U.S. government agency bonds						U.S. government agency bonds	44,964		44,964
Asset-backed securities	Asset-backed securities	20,680		20,680		Asset-backed securities	2,991		2,991
Corporate debt securities		12,039		12,039
U.S. government agency bonds		26,071		26,071
Money market funds		4,582		4,582
Money market						Money market	5,362		5,362
		$	210,711	$	210,711		$	156,163	$	156,163


(in thousands)	(in thousands)	Level 2		Level 3		Total		(in thousands)	Level 2		Level 3		Total
Liabilities:	Liabilities:							Liabilities:
Contingent consideration payable	Contingent consideration payable	$	0	$	27,317	$	27,317	Contingent consideration payable	$	—	$	19,266	$	19,266
Deferred compensation plan liability	Deferred compensation plan liability	4,233		0		4,233		Deferred compensation plan liability	4,977		—		4,977
		$	4,233	$	27,317	$	31,550		$	4,977	$	19,266	$	24,243

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, ~~2020~~2021 are identified in the following tables:


(in thousands)	(in thousands)	Level 2		Total		(in thousands)	Level 2		Total
Assets:	Assets:					Assets:
Commercial paper	Commercial paper	$	217,095	$	217,095	Commercial paper	$	174,531	$	174,531
Corporate debt securities						Corporate debt securities	32,311		32,311
Asset-backed securities	Asset-backed securities	9,438		9,438		Asset-backed securities	30,056		30,056
Corporate debt securities		39,513		39,513
U.S. government agency bonds		53,582		53,582
Money market funds		4,427		4,427
Money market						Money market	5,150		5,150
		$	324,055	$	324,055		$	242,048	$	242,048


(in thousands)	(in thousands)	Level 2		Level 3		Total		(in thousands)	Level 2		Level 3		Total
Liabilities:	Liabilities:							Liabilities:
Contingent consideration payable	Contingent consideration payable	$	0	$	25,825	$	25,825	Contingent consideration payable	$	—	$	20,339	$	20,339
Deferred compensation plan liability	Deferred compensation plan liability	4,078		0		4,078		Deferred compensation plan liability	4,800		—		4,800
		$	4,078	$	25,825	$	29,903		$	4,800	$	20,339	$	25,139

The Company's Convertible Notes fall into the Level 2 category within the fair value level hierarchy. The fair value was determined using broker quotes in a non-active market for valuation. The fair value of the Convertible Notes at June 30, 2021 was $4.4 million.

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, ~~2021~~2022 or December 31, ~~2020.~~2021.

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 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

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 ~~primarily~~ for the ~~ATB-200~~ATB200 Pompe disease program:

Contingent Consideration

Liability

Fair Value as of June 30, ~~2021~~2022

Valuation Technique

Unobservable Input

Range

(in thousands)

Discount rate

7.5%

Clinical and regulatory milestones

~~25,914~~19,266

Probability weighted discounted cash flow

Probability of achievement of milestones

75% - ~~78%~~88%

Projected year of payments

~~2021~~2022 - ~~2022~~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 following table shows the change in the balance of contingent consideration payable for the three and six months ended June 30, ~~2021~~2022 and ~~2020,~~2021, respectively:


		Three Months Ended June 30,				Six Months Ended June 30,					Three Months Ended June 30,				Six Months Ended June 30,
(in thousands)	(in thousands)	2021		2020		2021		2020		(in thousands)	2022		2021		2022		2021
Balance, beginning of the period	Balance, beginning of the period	$	26,296	$	23,612	$	25,825	$	22,681	Balance, beginning of the period	$	19,151	$	26,296	$	20,339	$	25,825
Changes in fair value during the period, included in the Consolidated Statements of Operations	Changes in fair value during the period, included in the Consolidated Statements of Operations	1,021		715		1,492		1,646		Changes in fair value during the period, included in the Consolidated Statements of Operations	115		1,021		(1,073)		1,492
Balance, end of the period (1)	Balance, end of the period (1)	$	27,317	$	24,327	$	27,317	$	24,327	Balance, end of the period (1)	$	19,266	$	27,317	$	19,266	$	27,317

(1) As ~~of June 30, 2021, based on~~ certain milestones ~~that~~ are expected to be reached within the next twelve months, ~~$19.8 million~~the June 30, 2022 balance was recorded as a current liability in the Consolidated Balance Sheets.

~~Note 9.~~

8. 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,					Three Months Ended June 30,				Six Months Ended June 30,
(in thousands, except per share amounts)	(in thousands, except per share amounts)	2021		2020		2021		2020		(in thousands, except per share amounts)	2022		2021		2022		2021
Numerator:	Numerator:									Numerator:
Net loss attributable to common stockholders	Net loss attributable to common stockholders	$	(51,225)	$	(52,492)	$	(116,889)	$	(141,440)	Net loss attributable to common stockholders	$	(62,157)	$	(51,225)	$	(147,417)	$	(116,889)
Denominator:	Denominator:									Denominator:
Weighted average common shares outstanding — basic and diluted	Weighted average common shares outstanding — basic and diluted	266,398,516		257,973,329		265,384,865		257,548,623		Weighted average common shares outstanding — basic and diluted	291,970,562		266,398,516		288,646,587		265,384,865

Dilutive common stock equivalents would include the dilutive effect of common stock options, convertible debt units, RSUs, and warrants for common stock equivalents. 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,			As of June 30,
(in thousands)	(in thousands)	2021	2020	(in thousands)	2022	2021
Options to purchase common stock	Options to purchase common stock	14,962	18,414	Options to purchase common stock	19,283	14,962
Unvested restricted stock units				Unvested restricted stock units	10,166	7,412
Convertible notes	Convertible notes	458	462	Convertible notes	—	458
Outstanding warrants, convertible to common stock		0	2,555
Unvested restricted stock units		7,412	7,484
Total number of potentially issuable shares	Total number of potentially issuable shares	22,832	28,915	Total number of potentially issuable shares	29,449	22,832

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, 2021. 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, 2021 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 a portfolio of product opportunities ~~led by~~including the first, oral monotherapy for Fabry disease that has achieved widespread global approval and a differentiated biologic for Pompe disease that is under review with the U.S. Food and ~~an industry leading rare disease gene therapy portfolio.~~Drug Administration ("FDA") as well as the European Medicines Agency ("EMA"). 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 United States ("U.S."), European Union ("E.U."), United Kingdom ("U.K."), and Japan, with multiple additional approvals granted and applications pending in several geographies around the world.

The lead biologics program of our pipeline is Amicus Therapeutics GAA ("AT-GAA", also known as ATB200/AT2221, or cipaglucosidase alfa/miglustat), a novel, ~~clinical-stage,~~two-component, potential best-in-class treatment ~~paradigm~~ for Pompe disease. In February 2019, the ~~U.S. Food and Drug Administration ("FDA")~~FDA granted Breakthrough Therapy designation ("BTD") to AT-GAA for the treatment of ~~late-onset~~late onset Pompe disease.

~~We have established an industry leading gene therapy portfolio~~ In September 2021, the FDA set the Prescription Drug User Fee Act ("PDUFA") target action date of ~~potential therapies~~May 29, 2022 for ~~people living with rare metabolic diseases, through a license with Nationwide Children's Hospital~~the New Drug Application ("~~Nationwide Children’s"~~NDA") for miglustat and ~~a research collaboration with~~July 29, 2022 for the ~~University~~Biologics License Application ("BLA") for cipaglucosidase alfa. The EMA validated the Marketing Authorization Application (“MAA”) in the fourth quarter of Pennsylvania ("Penn"). Our pipeline includes gene therapy programs in rare, neurologic lysosomal disorders ("LDs"), specifically: CLN6, CLN3, and CLN1 Batten disease, Pompe disease, Fabry disease, CDKL5 deficiency disorder ("CDD"), Mucopolysaccharidosis Type IIIB ("MPS IIIB"), as well as a next generation program in Mucopolysaccharidosis Type IIIA ("MPS IIIA"). Our research collaboration with Penn also provides us with exclusive disease-specific access2021. On May 9, 2022, the FDA extended the review period for the NDA for miglustat and the ~~option rights to develop potentially disruptive new gene therapy platform technologies~~BLA for cipaglucosidase alfa resulting in revised PDUFA action dates of August 29, 2022 and programs for most LDs and a broader portfolio of more prevalent rare diseases, including Rett Syndrome, Angelman Syndrome, Myotonic Dystrophy, and select other muscular dystrophies. In the first quarter of 2020, the FDA granted Fast Track designation to the CLN3 Batten disease gene therapy, AT-GTX-502, for the treatment of pediatric patients less than 18 years of age. In September 2020 and February 2021, the European Medicines Agency ("EMA") granted Priority Medicines ("PRIME") designation and the FDA granted Fast Track designation, respectively, to the CLN6 Batten disease gene therapy, AT-GTX-501, for the treatment of patients with variant late infantile neuronal ceroid lipofuscinosis 6 ("vLINCL6").October 29, 2022, respectively.

Our Strategy

Our strategy is to create, manufacture, test, and deliver the highest quality medicines for people living with rare ~~metabolic~~ 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. ~~In addition to our programs in Fabry and Pompe diseases, we~~We are leveraging our global capabilities to develop and ~~expand~~broaden our ~~robust pipeline~~lead franchises in ~~genomic medicine. We have made significant progress toward fulfilling our vision of building a leading global biotechnology company~~Fabry and Pompe disease, with focused discovery work on ~~rare metabolic diseases.~~next generation therapies and novel platform technologies.

Our operations have not been significantly impacted ~~from~~by the novel coronavirus (“COVID-19”) pandemic thus far. ~~However, we~~The Company continued to observe ~~periodic increase in~~increased lag ~~times~~time between patient identification and Galafold®initiation due to the ~~resurgence~~continued prevalence of COVID-19 ~~into 2021.~~and its ongoing impact on access to treatment for people living with Fabry disease in certain markets. We have maintained operations in all geographies, secured our global supply chain for our commercial and clinical products, as well as maintained the operational integrity of our clinical trials, with minimum disruptions. Our ability to continue to operate without any significant disruptions will depend on the continued health of our employees, the ongoing demand for Galafold®and the continued operation of our global supply chain. We have continued to provide uninterrupted access to medicines for those in need of treatment, while prioritizing the health and safety of our global workforce. However, our results of operations in future periods may be negatively impacted by unknown future impacts from the COVID-19 pandemic.

Highlights of our progress include:

•Commercial and regulatory success in Fabry disease. For the six months ended June 30, ~~2021,~~2022, Galafold® revenue totaled ~~$77.4~~$159.4 million, an increase of ~~$15.1~~$15.6 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®. In the U.S., we

continue to see a significant 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 clinical program milestones. In ~~December 2020,~~February 2021, we ~~completed last patient, last visit in our global~~reported topline results from the Phase 3 ~~pivotal~~ study of AT-GAA (ATB200-03, also known as "PROPEL") with 123 patients at 62 sites in 24 countries. In February 2021, we subsequently reported topline results for the PROPEL study. Additionally, in 2020, orphan drug designation was received in Japan and the British Medicines and Healthcare Products Regulatory Agency ("MHRA") issued a Promising Innovative Medicine ("PIM") designation for AT-GAA for the treatment of late-onset Pompe disease.. In June 2021, the MHRA granted AT-GAA a positive scientific opinion through the Early Access to Medicines Scheme ("EAMS") which permits eligible adults living with late-onset Pompe disease ("LOPD") who have received alglucosidase alfa for at least 2 years to switch to AT-GAA prior to marketing authorization in the U.K. We ~~have also~~ completed the submission of the rolling BLA ~~for cipaglucosidase alfa~~ and ~~the New Drug Application ("NDA") for miglustat~~NDA to the ~~FDA.~~FDA, which was accepted for review in September 2021, and in the fourth quarter of 2021, the MAA was submitted and validated by the EMA. In March 2022, we announced positive long-term data from our ongoing phase 1/2 clinical study. Study participants treated with AT-GAA for up to 36 months 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. In June 2022, the French National Agency for the Medicines and Health Products Safety granted the first reimbursed access to AT-GAA under their compassionate access program.

•Pipeline advancement and growth. We ~~have established an industry leading gene therapy portfolio of medicines for people living~~are leveraging our global capabilities to develop and broaden our lead franchises in Fabry and Pompe disease, with ~~rare metabolic diseases through a license with Nationwide Children’s~~focused discovery work on next generation therapies and a research collaboration with Penn. Some recent advances include, in February 2021, we presented initial clinical data a from the Phase 1/2 CLN3 gene therapy study that suggests early signs of disease stabilization and the potential to slow the neurological disease progression in children living with CLN3 Batten disease. Additionally, in February 2021, we presented preclinical data from our Fabry disease gene therapy clinical candidate, AT-GTX-701, with an engineered GLA transgene improved for stability demonstrated greater substrate reduction than wild type constructs across all tissues and doses.novel platform technologies.

•Manufacturing. We ~~continued to manufacture our Pompe biologic at commercial scale (1,000L) for~~have managed our clinical ~~studies~~ and ~~early~~ commercial inventory. Our supply agreement with WuXi Biologics and current capacity are expected to produce sufficient quantities to support commercial needs after receipt of applicable regulatory approvals if obtained. For gene therapy, we are working with our strategic partners to support our clinical manufacturing capabilities. Thus far, our global supply chains during the COVID-19 pandemic such that as of the date hereof we have not experienced supply impacts. We have been ~~interrupted and we have maintained our ability~~able to ~~manufacture~~continue to meet required commercial demand for Galafold® as well as supply our ongoing Pompe disease clinical studies and access programs including EAMS without interruption. We have secured supply ~~during~~for our continued needs for the ~~COVID-19 pandemic.~~Pompe disease program through a long-term supply agreement with Wuxi Biologics. The agreement allows for the continuous manufacture of our biologic to support future clinical needs and our anticipated commercial requirements should we garner regulatory approvals as planned. We have contracts in place to supply miglustat, our small molecule component of AT-GAA, to support both clinical and future commercial requirements.

•Financial strength. Total cash, cash equivalents, and marketable securities as of June 30, ~~2021~~2022 was ~~$383.1~~$386.8 million. Based on the current operating ~~models,~~model, we believe that the current cash position, which includes expected revenues, is sufficient to fund our operations and ongoing research programs to achieve self-sustainability. 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~~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 ~~348~~350 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. In countries where mutations are provided only on the amenability website, these 1,384 amenable mutations are now available. Marketing authorization approvals have been granted in over 40 countries around the world, including the U.S., E.U., U.K., Japan, and others. In July 2021, Galafold® was approved in the E.U. for adolescents aged 12 years and older weighing 45 kg or more. We plan to continue to launch Galafold® in additional countries during ~~2021.~~2022, 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 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 44 orange book listed patents, including 4 U.S. Composition of Matter patents, of which 27 provide protection through at least 2038.

~~Gene Therapy~~Next Generation for Fabry Disease

We are committed to continued innovation for all people living with Fabry disease. ~~For people living with Fabry disease who have non-amenable variants, which are not suitable for Galafold~~® ~~as a monotherapy, our strategy is to develop~~Our pipeline includes a Fabry gene ~~therapy. In October 2018, we expanded our gene~~ therapy ~~portfolio through a~~and an academic research collaboration agreement with ~~Penn~~the University of Seville to ~~pursue research and development of novel gene therapies, including~~explore next generation pharmacological chaperones for Fabry disease, and other indications. In October 2019, we disclosed preliminary data from a Fabry adeno-associate viral ("AAV") gene therapy using an Amicus-engineered transgene that demonstrated high levels of GLA activity and robust GL-3 reduction in a mouse model of Fabry disease. In February 2021, we presented initial preclinical data from our investigational AAV gene therapy program. This initial preclinical study assessed a range of single doses of AAV in Gla knockout mice with either wild-type hGLA (“unmodified hGLA”) or an Amicus/Penn engineered hGLA transgenes (“engineered hGLA” or “AT-GTX- 701”). The engineered hGLA AAV gene therapy demonstrated stable homodimer formation, enhanced temperature, plasma and neutral ph stability compared to the unmodified hGLA AAV gene therapy. In the lowest tested dose of AT-GTX-701, Gla knockout mice showed partial substrate reduction, while highest tested dose resulted in near complete substrate reduction. Additionally, AT-GTX-701 demonstrated significantly greater lyso-Gb3/GL-3 substrate reduction across all Fabry disease relevant tissues including the dorsal root ganglia (“DRG”), kidney, and heart, with reductions at low dose being equal to or greater than the reductions observed at higher doses with wildtype transgene and provided the first evidence for DRG storage reduction in a Fabry mouse model treated with an AAV gene therapy.

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 ~~or cipaglucosidase alfa,~~ preventing ~~inactiviation~~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.~~("glycogen"). Miglustat is not an active ingredient that contributes directly to ~~substrate reduction ("glycogen").~~glycogen reduction.

We initiated ATB200-03 (or "PROPEL"), a global Phase 3 clinical study of AT-GAA in adult patients with late-onset Pompe disease in December 2018 and completed last patient, last visit in December 2020. In February 2021, we reported topline results from the Phase 3 PROPEL study. ~~Patients in PROPEL were randomized 2:1 so that for every two patients randomized to be treated with AT-GAA, one was randomized to be treated with alglucosidase alfa.~~ 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”). ~~117~~Nearly all patients ~~completed~~from the PROPEL study and all 117 voluntarily enrolled in the long-term extension study. The primary endpoint of the study was the mean change in 6-minute walk distance as compared with baseline measurements at 52 weeks across the combined ERT Switch and ERT Naïve patient populations. In this combined population patients taking AT-GAA (n=85) walked on average 21 meters farther at 52 weeks comparedcontinue to 7 meters with those treated with alglucosidase alfa (n=37). This primary endpoint in the combined population was assessed for superiority and while numerically greater, statistical significance for superiority on this combined population was not achieved for the AT-GAA arm as compared to the alglucosidase alfa arm (p=0.072).

Per the hierarchy of the statistical analysis plan, the first key secondary endpoint of the study was the mean change in percent-predicted Forced Vital Capacity (“FVC”) at 52 weeks across the combined population. In this combined population patients taking AT-GAA demonstrated a nominally statistically significant and clinically meaningful difference for superiority over those treated with alglucosidase alfa. AT-GAA significantly slowed the rate of respiratory decline in patients after 52 weeks. Patientsbe treated with AT-GAA ~~showed a 0.9% absolute decline in percent-predicted FVC, compared to a 4.0% absolute decline~~ in the alglucosidase alfa arm (p=0.023). Patients within the combined study population demonstrated statistically significant improvements on the GSGC (“Gait, Stairs, Gower’s Chair”) key secondary endpoint, which captures strength, coordination and mobility, compared to a worsening for alglucosidase alfa treated patients in the overall population (p<0.05). Additionally, lower MMT (Manual Muscle Testing), Patient-Reported Outcomes Measurement Information System (“PROMIS”) physical function and PROMIS fatigue secondary endpoints favored AT-GAA treated patients over alglucosidase alfa treated patients. Results also showed improvements in the two important biomarker endpoints of Pompe disease (Hex-4 and CK), which significantly favored AT-GAA compared to alglucosidase alfa (p<0.001). AT-GAA demonstrated a similar safety profile to alglucosidase alfa.

extension clinical study. The PROPEL Switch patients entered the study having been treated with alglucosidase alfa for a minimum of two years. More than two thirds (67%+) of those patients had been on ERT treatment for more than five years prior to enteringclinical data from the PROPEL study, ~~(mean of 7.4 years). A pre-specified analysis of~~ the ~~patients switching~~extension study as well as the Phase 1/2 study were included in the AT-GAA submissions to the FDA and the EMA.

In March 2022, we announced positive long-term data from ~~alglucosidase alfa on 6-minute walk distance showed that after 52 weeks from switching, AT-GAA treated patients (n=65) walked 16.9 meters farther than their~~

baseline, compared to 0.0 meters for those patients who were randomized to remain on alglucosidase alfa (n=30) (p=0.046). A pre-specified analysis of the patients switching from alglucosidase alfa on percent-predicted FVC showed that AT-GAA treated patients stabilized and slightly improved their respiratory function on this important measure while those patients remaining on alglucosidase alfa continued to significantly decline in respiratory muscle function. AT-GAA patients showed a 0.1% absolute increase in percent-predicted FVC while the alglucosidase alfa patients showed a 4.0% absolute decline over the course of the year (p=0.006).

~~The PROPEL Naïve patients~~our ongoing phase 1/2 clinical study. Study participants treated with AT-GAA for ~~52 weeks (n=20) walked 33 meters farther than their baseline,~~up to 36 months demonstrated persistent and durable effects on ~~the 6-minute~~six-minute walk test distance ~~endpoint. The Naïve~~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.

In addition, we are conducting ongoing clinical studies in pediatric patients treated with alglucosidase alfa (n=7) walked 38 meters further than their baseline. The difference between the two groups was not statistically significant (p=0.60). Additionally, patients never previously treated with any ERT showed similar declines in percent-predicted FVC at 52 weeks of –4.1% for ~~AT-GAA treated patients~~both LOPD and ~~–3.6% for alglucosidase alpha treated patients. The difference between the two groups was not statistically significant (p=0.57).~~infantile-onset Pompe disease ("IOPD") populations.

~~Gene Therapy~~Next Generation for Pompe Disease

As part of our long-term commitment to provide multiple solutions to address the significant unmet needs of the Pompe disease community, we are also ~~advancing a~~continuing discovery for next-generation ~~gene therapy treatment~~genetic medicines for Pompe disease. ~~In October 2018, we expanded our gene therapy portfolio through a collaboration agreement with Penn to pursue research and development of novel gene therapies for Pompe disease and other indications.~~

In April 2019, we presented initial preclinical data from our investigational AAV gene therapy program for Pompe disease. This initial preclinical study in Pompe knockout mice administered a single high dose of AAV gene therapy with either unmodified wild-type hGAA ("unmodified hGAA") or an Amicus/Penn engineered hGAA transgene with a Lysosomal-Targeting Cell receptor binding motif ("engineered hGAA"). The engineered hGAA AAV gene therapy demonstrated more robust and consistent glycogen reduction compared to unmodified hGAA AAV gene therapy, in all key tissues assessed in a Pompe mouse model. In the central nervous system, the engineered hGAA AAV gene therapy also showed robust glycogen reduction in neuronal cells, suggesting this may be an effective way to address neuronal aspects of Pompe disease. Unmodified hGAA AAV gene therapy showed minimal glycogen reduction in neuronal cells. This preclinical study provided initial validation for combining Amicus-engineered transgenes with Penn's AAV gene therapy technologies.

In May 2020, we presented preclinical data with the engineered hGAA AAV in single and combined central nervous system ("CNS") and systemic directed gene therapy in a mouse model of Pompe disease with advanced disease at treatment. The engineered hGAA AAV showed better targeting and clearance of glycogen storage at low doses in Pompe mice compared to unmodified hGAA AAV. High dose IV therapy showed strength rescue and the addition of high dose intracerabroventricular ("ICV") therapy to high dose IV provided incremental benefit.

~~Gene Therapy for Various Types of Batten Disease~~

Through our license with Nationwide Children’s and research collaboration with Penn, we are researching potential first-in-class gene therapies for multiple forms of Batten disease. Batten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system, also known as neuronal ceroid lipofuscinoses ("NCLs"). In these diseases, a defect in a specific gene triggers a cascade of problems that interferes with a cell's ability to recycle certain molecules. Each gene is called ceroid lipofuscinosis, neuronal ("CLN") and given a different number designation as its subtype. There are 13 known forms of Batten disease often referred to as CLN1-8; 10-14. The various types of Batten disease have similar features and symptoms but vary in severity and age of onset.

~~We have two clinical programs in CLN6 and CLN3 Batten disease, and several preclinical programs including CLN1 and other types of Batten disease.~~

~~Our Phase 1/2 study in CLN6 Batten disease enrolled thirteen patients who received a single one-time intrathecal~~administration of AAV-CLN6 gene therapy. As of June 2021, twelve of the thirteen patients in the Phase 1/2 study have completed and four have entered into the long term follow up study ("LTFU"), with the remaining patients expected to enter the LTFU where long-term data for safety and efficacy will continue to be generated. In April 2021, the first site opened for the Batten disease Natural History study, BAT-001, which will help further our understanding of disease progression. In October 2020, we reported additional positive interim results from our CLN6 Batten disease AAV-CLN6 gene therapy program, AT-GTX-501. Interim safety data are available for 13 patients with CLN6 Batten disease. Interim safety data demonstrated the treatment with AT-GTX-501 was generally well tolerated. The majority of adverse events ("AEs") were mild and unrelated to treatment. No pattern of adverse events related to AAV or CLN6 immunogenicity was observed. Interim efficacy data are available within the Hamburg Motor and Language scores and showed a meaningful effect in slowing disease progression for twelve patients reaching the 12-month timepoint and for eight patients up to 24 months, post-administration of the AAV-CLN6 gene therapy. Additionally, in October 2019, we reported interim clinical data that suggested stabilization of various components of Hamburg Seizure and Vision scores in most patients from baseline to month 12 or 24, in particular those patients treated at a younger age, compared to the progression expected in matched untreated patients.

In the fourth quarter of 2018, we announced the initiation of a Phase 1/2 study to evaluate the safety and efficacy of a single intrathecal administration of an AAV serotype AT-GTX-502 gene therapy in patients with CLN3 Batten disease. In the Phase 1/2 study, a total of three patients were dosed in the low-dose group, and based on the safety profile to date, the data safety monitoring board cleared us to begin enrollment in the high-dose cohort. One patient is currently dosed in the high-dose cohort. In February 2021, we announced initial safety for the first four patients up to 15 months post-administration of AT-GTX-502 and preliminary efficacy data for the first three patients in the low-dose cohort for up 15 months post-administration of AT-GTX-502, as well as one patient in the high-dose cohort for up to 3 months post-administration of AT-GTX-502. Initial results of the study suggest that AT-GTX-502 was well tolerated and demonstrated potential early signs of disease stabilization compared to a natural history dataset.

CDKL5 Deficiency Disorder

We are researching a potential first-in-class genetic medicine for ~~CDD~~CDKL5 deficiency disorder consisting of a CDKL5 protein engineered for cross correction, delivered as either a protein replacement or as a gene therapy through our collaboration with Penn. ~~CDKL5 is a gene on~~We are collaborating with the ~~X-chromosome encoding~~LouLou Foundation to assess the ~~CDKL5 protein that regulates~~natural history of the ~~expression of several essential proteins~~disease to identify endpoints for ~~normal brain development. Genetic mutations~~potential use in the CDKL5 gene result in CDKL5 protein deficiency and CDD. This disorder manifests clinically as persistent seizures starting in infancy, followed by severe impairment in neurological development. Most children affected by CDD cannot walk or care for themselves and may also suffer from scoliosis, visual impairment, sensory issues, and gastrointestinal complications.future studies.

~~Other Preclinical Gene Therapies~~Additional Next Generation Programs

We have a number of additional gene therapies in ~~active~~clinical and preclinical development, including potential gene therapies ~~for MPS IIIB as well as a next generation program~~ in ~~MPS IIIA. Our strategy is to develop first or best in class AAV gene therapies for these rare devastating pediatric neurological lysosomal storage diseases.~~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 ~~acquisitions~~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.

Consolidated Results of Operations

Three Months Ended June 30, ~~2021~~2022 compared to June 30, ~~2020~~2021

The following table provides selected financial information for the Company:


		Three Months Ended June 30,										Three Months Ended June 30,
(in thousands)	(in thousands)	2021			2020			Change			(in thousands)	2022			2021			Change
Net product sales	Net product sales	$	77,413		$	62,353		$	15,060		Net product sales	$	80,731		$	77,413		$	3,318
Cost of goods sold	Cost of goods sold	8,380			6,676			1,704			Cost of goods sold	8,197			8,380			(183)
Cost of goods sold as a percentage of net product sales	Cost of goods sold as a percentage of net product sales	10.8		%	10.7		%	0.1		%	Cost of goods sold as a percentage of net product sales	10.2		%	10.8		%	(0.6)		%
Operating expenses:	Operating expenses:										Operating expenses:
Research and development	Research and development	63,003			69,611			(6,608)			Research and development	78,319			63,003			15,316
Selling, general, and administrative	Selling, general, and administrative	42,276			34,657			7,619			Selling, general, and administrative	53,379			42,276			11,103
Changes in fair value of contingent consideration payable	Changes in fair value of contingent consideration payable	1,021			715			306			Changes in fair value of contingent consideration payable	115			1,021			(906)
Depreciation and amortization	Depreciation and amortization	1,567			2,039			(472)			Depreciation and amortization	1,334			1,567			(233)
Other income (expense):
Other (expense) income:											Other (expense) income:
Interest income	Interest income	50			865			(815)			Interest income	356			50			306
Interest expense	Interest expense	(8,150)			(3,635)			(4,515)			Interest expense	(8,257)			(8,150)			(107)

Other expense		234			5,326			(5,092)
Other income											Other income	7,268			234			7,034
Income tax expense	Income tax expense	(4,525)			(3,703)			(822)			Income tax expense	(911)			(4,525)			3,614
Net loss attributable to common stockholders	Net loss attributable to common stockholders	$	(51,225)		$	(52,492)		$	1,267		Net loss attributable to common stockholders	$	(62,157)		$	(51,225)		$	(10,932)

Net Product Sales. Net product sales increased ~~$15.1~~$3.3 million during the three months ended June 30, ~~2021~~2022 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.~~markets, partially offset by the $6.7 million unfavorable impact of foreign currency exchange.

Research and Development Expense. The following table summarizes our principal ~~product~~ development programs ~~for each product candidate in development~~ and the out-of-pocket, ~~third party~~third-party expenses ~~incurred with respect to each product candidate:~~incurred:
(in thousands) Three Months Ended June 30,
Projects 2021 2020
Third party direct project expenses
Galafold® (Fabry Disease)
$ 1,375 $ 2,759
AT-GAA (Pompe Disease) 23,840 27,360
Gene therapy programs 14,648 14,010
Pre-clinical and other programs 552 120
Total third-party direct project expenses 40,415 44,249
Other project costs
Personnel costs 15,986 18,981
Other costs 6,602 6,381
Total other project costs 22,588 25,362
Total research and development costs $ 63,003 $ 69,611


(in thousands)	Three Months Ended June 30,
Projects	2022		2021
Third party direct project expenses
Galafold® (Fabry Disease)	$	3,419	$	1,375
AT-GAA (Pompe Disease)	21,585		23,840
Gene therapy programs	27,225		14,648
Pre-clinical and other programs	—		552
Total third-party direct project expenses	52,229		40,415
Other project costs
Personnel costs	18,152		15,986
Other costs	7,938		6,602
Total other project costs	26,090		22,588
Total research and development costs	$	78,319	$	63,003

The ~~$6.6~~$15.3 million ~~decrease~~increase in research and development costs was primarily ~~due to~~driven by the ~~timing~~strategic prioritization of ~~clinical research and manufacturing costs associated with the advancement of clinical studies~~our gene therapy portfolio resulting in the ~~Pompe program and~~non-recurring expense of a ~~decrease in~~contractual obligation from which we will no longer receive further economic benefit. Additionally, personnel costs increased primarily due to ~~realignment with strategic priorities.~~share-based compensation.

Selling, General, and Administrative Expense. Selling, general, and administrative expense increased ~~$7.6~~$11.1 million, primarily driven by ~~increased personnel costs.~~

~~Interest Expense. Interest expense increased $4.5 million during~~ the ~~three months ended June 30, 2021 compared to the same period~~strategic prioritization of our gene therapy portfolio that resulted in the ~~prior year. The increase was driven by the $400 million Senior Secured Loan due 2026 entered in July 2020.~~write-off of cloud computing costs and software licensing fees.

Other ~~Expense.~~Income. The ~~$5.1~~$7.0 million variance was primarily ~~driven by~~related to foreign exchange gains ~~in the~~caused by local currency remeasurement of ~~our intercompany transactions.~~U.S. dollar balances.

Income Tax Expense. The income tax expense for the three months ended June 30, ~~2021~~2022 was ~~$4.5~~$0.9 million. We are subject to income taxes in various jurisdictions. Our tax liabilities are largely dependent on the distribution of pre-tax earnings among the many jurisdictions in which we operate.

Six Months Ended June 30, ~~2021~~2022 compared to June 30, ~~2020~~2021

The following table provides selected financial information for the Company:


		Six Months Ended June 30,										Six Months Ended June 30, 2022
(in thousands)	(in thousands)	2021			2020			Change			(in thousands)	2022			2021			Change
Net product sales	Net product sales	$	143,815		$	122,878		$	20,937		Net product sales	$	159,446		$	143,815		$	15,631
Cost of goods sold	Cost of goods sold	14,919			13,228			1,691			Cost of goods sold	15,779			14,919			860
Cost of goods sold as a percentage of net product sales	Cost of goods sold as a percentage of net product sales	10.4		%	10.8		%	(0.4)		%	Cost of goods sold as a percentage of net product sales	9.9		%	10.4		%	(0.5)		%
Operating expenses:	Operating expenses:										Operating expenses:
Research and development	Research and development	127,120			158,731			(31,611)			Research and development	159,836			127,120			32,716
Selling, general, and administrative	Selling, general, and administrative	89,002			74,872			14,130			Selling, general, and administrative	111,495			89,002			22,493
Changes in fair value of contingent consideration payable	Changes in fair value of contingent consideration payable	1,492			1,646			(154)			Changes in fair value of contingent consideration payable	(1,073)			1,492			(2,565)
Loss on impairment of assets											Loss on impairment of assets	6,616			—			6,616
Depreciation and amortization	Depreciation and amortization	3,171			3,803			(632)			Depreciation and amortization	2,745			3,171			(426)
Other income (expense):
Other (expense) income:											Other (expense) income:
Interest income	Interest income	215			2,380			(2,165)			Interest income	489			215			274
Interest expense	Interest expense	(16,142)			(7,364)			(8,778)			Interest expense	(16,404)			(16,142)			(262)

Other expense		(2,966)			(2,990)			24
Other income (expense)											Other income (expense)	9,170			(2,966)			12,136
Income tax expense	Income tax expense	(6,107)			(4,064)			(2,043)			Income tax expense	(4,720)			(6,107)			1,387
Net loss attributable to common stockholders	Net loss attributable to common stockholders	$	(116,889)		$	(141,440)		$	24,551		Net loss attributable to common stockholders	$	(147,417)		$	(116,889)		$	(30,528)

Net Product Sales. Net product sales increased ~~$20.9~~$15.6 million during the six months ended June 30, ~~2021~~2022 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.~~markets, partially offset by the $9.9 million unfavorable impact of foreign currency exchange.

Research and Development Expense. The following table summarizes our principal ~~product~~ development programs ~~for each product candidate in development~~ and the out-of-pocket, ~~third party~~third-party expenses ~~incurred with respect to each product candidate:~~incurred:
(in thousands) Six Months Ended June 30,
Projects 2021 2020
Third party direct project expenses
Galafold® (Fabry Disease)
$ 3,602 $ 4,520
AT-GAA (Pompe Disease) 44,954 62,267
Gene therapy programs 28,464 35,497
Pre-clinical and other programs 620 833
Total third-party direct project expenses 77,640 103,117
Other project costs
Personnel costs 36,165 41,699
Other costs 13,315 13,915
Total other project costs 49,480 55,614
Total research and development costs $ 127,120 $ 158,731


(in thousands)	Six Months Ended June 30,
Projects	2022		2021
Third party direct project expenses
Galafold® (Fabry Disease)	$	7,039	$	3,602
AT-GAA (Pompe Disease)	48,563		44,954
Gene therapy programs	44,917		28,464
Pre-clinical and other programs	93		620
Total third-party direct project expenses	100,612		77,640
Other project costs
Personnel costs	43,827		36,165
Other costs	15,397		13,315
Total other project costs	59,224		49,480
Total research and development costs	$	159,836	$	127,120

The ~~$31.6~~$32.7 million ~~decrease~~increase in research and development costs was primarily due to the ~~timing~~strategic prioritization of ~~clinical research and manufacturing costs associated with the advancement of clinical studies~~our gene therapy portfolio resulting in the ~~Pompe program, decrease in gene therapy programs driven by the pipeline growth as well as the timing~~non-recurring expense of ~~spend of the pipeline initiatives and a decrease~~contractual obligations from which we will no longer receive further economic benefit, an increase in personnel costs primarily due to ~~realignment~~share-based compensation, an increase in the Pompe disease program associated with ~~strategic priorities.~~

the timing of manufacturing costs, and an increase in clinical research costs in the Fabry disease program.

Selling, General, and Administrative Expense. Selling, general, and administrative expense increased ~~$14.1~~$22.5 million, ~~mainly driven by increased personnel costs.~~

~~Interest Expense. Interest expense increased $8.8 million during the six months ended June 30, 2021 compared to the same period in the prior year. The increase was~~primarily driven by the ~~$400~~strategic prioritization of our gene therapy portfolio that resulted in the write-off of cloud computing costs and software licensing fees and an increase in share-based compensation and marketing expenses.

Loss on Impairment of Assets. In connection with the strategic prioritization of our gene therapy portfolio, the Company performed an assessment of its assets and recognized a$6.6 million ~~Senior Secured Loan due 2026 entered in July 2020.~~loss on impairment of assets.

Other Income (Expense). The $12.1 million variance was primarily related to foreign exchange gains caused by local currency remeasurement of U.S. dollar balances.

Income Tax Expense. The income tax expense for the six months ended June 30, ~~2021~~2022 was ~~$6.1~~$4.7 million. We are subject to income taxes in various jurisdictions. Our tax liabilities are largely dependent on the distribution of pre-tax earnings among the many jurisdictions in which we operate.

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, ~~debt issuances,~~ Galafold® revenues, debt issuance, collaborations, and other financing arrangements.

Cash Flow Discussion

As of June 30, ~~2021,~~2022, we had cash, cash equivalents, and marketable securities of ~~$383.1~~$386.8 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 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 bonus. 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 used in operations for the six months ended June 30, 2021 was $108.3 million. The components of net cash used in operations included the net loss for the six months ended June 30, 2021 of $116.9 million and an overall decrease in cash from changes ~~from~~in operating assets and liabilities of $32.6 million, primarily related to the timing of contract manufacturing and research payments. This was partially offset by $32.1 million of stock compensation and $9.1 million of other non-cash adjustments.

Net Cash Provided by Investing Activities

Net cash ~~used in operations~~provided by investing activities for the six months ended June 30, ~~2020~~2022 was ~~$141.5~~$84.5 million. ~~The components~~Our investing activities have consisted primarily of ~~net~~purchases, sales, and maturities of investments and capital expenditures. Net cash ~~used in operations included the net loss~~provided by investing activities reflects $184.1 million for the ~~six months ended June 30, 2020~~sale and redemption of ~~$141.4~~marketable securities, partially offset by$98.3 millionfor the purchase of marketable securities and ~~the net change in operating assets and liabilities of $32.6 million. The change in operating assets was primarily due to an increase in accounts receivable by $10.4~~$1.2 million ~~due to increased commercial sales of Galafold® and a decrease in prepaid and other current assets of $1.4 million to support the commercial activities~~ for Galafold®. The net cash used in operations was also impacted by a decrease in accounts payable and accrued expenses of $23.9 million, mainly related to the payment of contract manufacturing and research costs, program expenses and personnel costs.

~~Net Cash Provided by Investing Activities~~capital expenditures.

Net cash provided by investing activities for the six months ended June 30, 2021 was $112.3 million. Our investing activities have consisted primarily of purchases and sales and maturities of investments and capital expenditures. Net cash provided by investing activities reflects $258.8 million for the sale and redemption of marketable securities, partially offset by $145.3 million for the purchase of marketable securities and $1.2 million for capital expenditures.

Net Cash (Used in) Provided by Financing Activities

Net cash ~~provided by investing~~used in financing activities for the six months ended June 30, ~~2020~~2022 was ~~$163.3~~$7.5 million. ~~Our investing activities have consisted primarily of purchases and sales and maturities of investments and capital expenditures.~~ Net cash ~~provided by investing~~used in financing activities primarily reflects ~~$210.1~~the purchase of vested restricted stock units of $9.3 million, ~~for the sale and redemption of marketable securities,~~ partially offset by ~~$45.0 million for the purchase of marketable securities and~~ $1.9 million ~~for~~of proceeds from the ~~acquisition~~exercise of ~~property and equipment.~~

~~Net Cash Provided by Financing Activities~~stock options.

Net cash provided by financing activities for the six months ended June 30, 2021 was $11.1 million. Net cash provided by financing activities primarily reflects $19.2 million from the exercise of the remaining outstanding warrants and $6.7 million from the exercise of stock options, partially offset by $14.4 million from ~~payments~~the purchase of ~~employee withholding taxes related to~~vested restricted stock ~~unit vesting.~~

Net cash provided in financing activities for the six months ended June 30, 2020 was $2.6 million. Net cash provided by financing activities primarily reflects $10.7 million from the exercise of stock options, partially offset by $8.1 million from payments of employee withholding taxes related to restricted stock unit vesting.units.

Funding Requirements

We expect to incur losses from operations for 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 our clinical trials of our drug ~~candidates and gene therapy candidates, including but not limited to AT-GAA, CLN6 and CLN3;~~candidates;

•the future results of ~~on-going~~ preclinical research and subsequent clinical trials for ~~CDD, Pompe gene therapy, Fabry gene therapy, MPS IIIB, next generation MPS IIIA, and other~~ 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;~~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;

•our ability to successfully commercialize Galafold® ~~("migalastat~~(also referred to as "migalastat HCl") and, if our regulatory ~~filings~~applications are ~~accepted and~~ approved, AT-GAA;

•our ability to manufacture or supply sufficient clinical or commercial products, including Galafold®~~, AT-GAA~~ and ~~our gene therapy candidates;~~AT-GAA;

•our ability to obtain reimbursement for Galafold® and, if our regulatory ~~filings~~applications are ~~accepted and~~ approved, AT-GAA;

•our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of Galafold®;,and, if approved and applicable, AT-GAA;

•our ability to obtain market acceptance of Galafold®and, if our regulatory ~~filings~~applications are ~~accepted and~~ approved, AT-GAA;

•the costs of preparing, filing, and prosecuting patent applications and maintaining, enforcing, and defending intellectual property-related ~~claims;~~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;

•the extent to which we acquire or invest in businesses, products, and technologies;

•our ability to establish licensing agreements, collaborations, partnerships or other similar arrangements and to obtain milestone, royalty, or other payments from any such collaborators;

•~~our ability to adjust to changes in the European and U.K. markets in the wake of the U.K. leaving the E.U.;~~

•the extent to which our business could be adversely impacted by the effects of ~~COVID-19~~the novel coronavirus ("COVID-19") outbreak, including due to actions by us, governments, our customers, orour 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.

While we continue to generate revenue from product sales, in the absence of additional funding, we expect our continuing operating losses to result in increases in our net cash used ~~in operations~~ over the next several quarters and years. We may seek additional funding through public or private financings of debt or equity. Based on our current operating ~~models,~~model, we believe that the current cash position, which includes expected revenues, is sufficient to fund our operations and ongoing research programs to achieve self-sustainability. Potential impacts of the COVID-19 pandemic, business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact our future capital requirements.

Financial Uncertainties Related to Potential Future Payments

Milestone Payments / Royalties

~~Celenex~~Callidus - In connection with our acquisition of Callidus in 2013, we agreed to pay up to an additional $35 million in connection with the achievement of certain clinical milestones and up to $80 million in connection with the achievement of certain regulatory approval milestones. As of June 30, 2022, $20 million and $68 million remain outstanding, respectively. Refer to “— Note 7. Assets and Liabilities Measured at Fair Value,” to the Consolidated Financial Statements.

Celenex - In connection with our acquisition of Celenex in 2018, we agreed to pay up to an additional $10 million in connection with the achievement of certain development milestones, $220 million in connection with the achievement of certain regulatory approval milestones across multiple programs and up to $75 million in tiered sales milestone payments.

Nationwide Children’s Hospital - - Celenex has an exclusive license agreement with Nationwide ~~Children’s.~~Children's Hospital ("Nationwide Children’s"). Under this license agreement, Nationwide Children’s is eligible to receive development and sales-based milestones of up to $7.8 million from us for each product.

~~Penn~~University of Pennsylvania - - Under our ~~research~~ collaboration agreement with ~~Penn,~~the University of Pennsylvania ("Penn"), Penn is eligible to receive certain milestone, royalty, and discovery research payments with respect to licensed products for each indication. Milestone payments are payable following the achievement of certain development and commercial milestone events in each indication, up to an aggregate of $88.0 million per indication. Royalty payments are based on net sales of licensed products on a licensed product-by-licensed product and country-by-country basis. We ~~will~~ provide $10.0 million each year during the five-year agreement to fund the discovery research program.

GlaxoSmithKline - - In July 2012, as amended in November 2013, we entered into an agreement with GlaxoSmithKline ("GSK"), pursuant to which ~~Amicus~~we obtained global rights to develop and commercialize Galafold® as a monotherapy and in combination with ERT for Fabry disease (“Collaboration Agreement”). Under the terms of the Collaboration Agreement, GSK is eligible to receive post-approval and sales-based milestones up to $40 million, as well as tiered royalties in the mid-teens in eight major markets outside the U.S.

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, including those described in greater detail below. 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 six months ended June 30, ~~2021~~2022 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, ~~2020.~~2021.

Recent Accounting Pronouncements

Please refer to "—Note 2. Summary of Significant Accounting Policies" in our Notes to Consolidated Financial Statements.

ITEM 3. QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK

~~Market risk is the risk of change in fair value of a financial instrument due to changes in interest rates, equity prices, creditworthiness, financing, exchange rates, or other factors.~~ Our primary market risk exposure relates to changes in interest rates in our cash, cash equivalents, and marketable securities. We place our investments in high-quality financial instruments, primarily money market funds, corporate debt securities, asset backed securities, and U.S. government agency notes with maturities of less than one year, which we believe are subject to limited interest rate and credit risk. The securities in our investment portfolio are not leveraged, are classified as available-for-sale and, due to the short-term nature, are subject to minimal interest rate risk. We believe that a 1% (100 basis points) change in average interest rates would either increase or decrease the market value of our investment portfolio by $0.7 million as of June 30, 2021. We currently do not hedge interest rate exposure and consistent with our investment policy, we do not use derivative financial instruments in our investment portfolio.

We are exposed to interest rate risk with respect to variable rate debt. At June 30, 2021, we had a $400 million Senior Secured Term Loan due 2026 that bears interest at a rate equal to the 3-month LIBOR, subject to a 1% floor, plus 6.5% per year. We do not currently hedge our variable interest rate debt. The annual average variable interest rate for our variable rate debt as of June 30, 2021 was 7.5%. A hypothetical 100 basis point increase or decrease in the average interest rate on our variable rate debt would result in a $1.0 million change in the interest expense as of June 30, 2021.

The Financial Conduct Authority has announced the intent to phase out the use of LIBOR by the end of 2021. If LIBOR is discontinued, we may need to renegotiate the terms of the Senior Secured Term Loan due 2026 in order to replace LIBOR with an alternative standard. As a result, we may incur incremental costs in transitioning to a new standard, and interest rates on our current or future indebtedness may be adversely affected by the new standard. The potential effect of any such event on our cost of capital cannot yet be determined, but we do not expect it to have a material impact on our consolidated financial condition, results of operations, or cash flows.

We face foreign exchange risk as a result of entering into transactions denominated in currencies other than U.S. dollars. We are not currently engaged in any foreign currency hedging activities. The current exposures arise primarily from cash, accounts receivable, intercompany receivables and payables, and net product sales denominated in foreign currencies. Both positive and negative impacts to our international product sales from movements in foreign currency exchange rates may be partially mitigated by the natural, opposite impact that foreign currency exchange rates have on our international operating expenses. A hypothetical 10% change in foreign exchange rates during any of the periods presented would not have had a material impact on our Consolidated Financial Statements.

~~For information regarding our exposure to certain~~ market risks, ~~see~~and the way we manage them, are summarized in Part II, Item 7A, ~~Quantitative~~"Quantitative and Qualitative Disclosures About Market ~~Risk, in~~Risk" of our Annual Report on Form 10-K for the fiscal year ended December 31, ~~2020. There~~2021. As of June 30, 2022, there have been no material changes into our ~~financial instrument portfolio~~market risks or ~~market risk exposures~~to our management of such risks since ~~our fiscal year ended~~ December 31, ~~2020.~~2021.

ITEM 4. 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

~~On July 13, 2021, Teva Pharmaceuticals Development, Inc. (“Teva”) filed~~We are not currently a ~~complaint against the Company in the United States District Court for the Eastern District of Pennsylvania alleging violations of the Creating and Restoring Equal Access~~party to Equivalent Samples Act (also known as the “CREATES ACT”). Teva is seeking monetary damages, fees and other relief in connection with the lawsuit. The Company believes the lawsuit is without merit and will vigorously defend itself.

For more information concerning the risks associated with litigation involving the Company generally, see our Risk Factor on our Annual Report on Form 10-K titled, “Litigation may adversely affect our business, financial condition, results of operations or liquidity.”any material legal proceedings.

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, ~~2020.~~2021.

ITEM 2. UNREGISTERED SALES OF EQUITY SECURITIES AND USE OF PROCEEDS

Recent Sales of Unregistered Securities

None.

Issuer Purchases of Equity Securities

The following table provides certain information with respect to purchase of our common stock during the three months ended June 30, ~~2021:~~2022:

Period
Total Number of Shares Purchased (1)
Average Price Paid per Share Total Number of Shares Purchased as Part of Publicly Announced Plans or Programs Maximum Number (or Approximate Dollar Value) of Shares That May Yet Be Purchased Under the Plans or Programs
April 1, 2021 through April 30, 2021 5,406 $ 10.02 — —
May 1, 2021 through May 31, 2021 13,013 $ 9.40 — —
June 1, 2021 through June 30, 2021 37,411 $ 10.16 — —
Total 55,830 $ 9.84 — —


Period	Total Number of Shares Purchased (1)	Average Price Paid per Share		Total Number of Shares Purchased as Part of Publicly Announced Plans or Programs	Maximum Number (or Approximate Dollar Value) of Shares That May Yet Be Purchased Under the Plans or Programs
April 1, 2022 through April 30, 2022	6,588	$	8.31	—	—
May 1, 2022 through May 31, 2022	23,164	$	7.64	—	—
June 1, 2022 through June 30, 2022	11,060	$	9.13	—	—
Total	40,812	$	8.15	—	—

______________________________

(1) Represents shares of common stock withheld to satisfy taxes associated with the vesting of restricted stock ~~awards~~units

ITEM 3. DEFAULTS UPON SENIOR SECURITIES

None.

ITEM 4. MINE SAFETY DISCLOSURES

None.

ITEM 5. OTHER INFORMATION

~~None~~None.

ITEM 6. EXHIBITS


Exhibit Number						Description


31.1						Certification of Principal Executive Officer pursuant to Rules 13a-14 and 15d-14 promulgated pursuant to the Securities Exchange Act of 1934, as amended

31.2						Certification of Principal Financial Officer pursuant to Rules 13a-14 and 15d-14 promulgated pursuant to the Securities Exchange Act of 1934, as amended

32.1						Certification of Principal Executive Officer and Principal Financial Officer pursuant to Section 906 of the Sarbanes-Oxley Act of 2002

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
104						Cover Page Interactive Data File (formatted in Inline XBRL and included in Exhibit 101)

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:	August ~~5, 2021~~4, 2022	By:	/s/ ~~John F. Crowley~~Bradley L. Campbell
			~~John F. Crowley~~Bradley L. Campbell
			~~Chairman~~President and Chief Executive Officer
			(Principal Executive Officer)

Date:	August ~~5, 2021~~4, 2022	By:	/s/ Daphne Quimi
			Daphne Quimi
			Chief Financial Officer
			(Principal Financial Officer)
			(Principal Accounting Officer)


	June 30, 2021		December 31, 2020
Assets
Current assets:
Cash and cash equivalents	$	176,538	$	163,240
Investments in marketable securities	206,530		320,029
Accounts receivable	49,172		46,923
Inventories	24,086		19,556
Prepaid expenses and other current assets	24,176		29,721
Total current assets	480,502		579,469
Operating lease right-of-use assets, less accumulated amortization of $8,150 and $7,574 at June 30, 2021 and December 31, 2020, respectively	22,028		23,296
Property and equipment, less accumulated depreciation of $17,410 and $14,487 at June 30, 2021 and December 31, 2020, respectively	42,365		43,863
In-process research & development	23,000		23,000
Goodwill	197,797		197,797
Other non-current assets	21,200		19,095
Total Assets	$	786,892	$	886,520
Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable	$	13,762	$	17,063
Accrued expenses and other current liabilities	71,325		96,841
Contingent consideration payable	19,800		8,900
Operating lease liabilities	7,106		6,872
Total current liabilities	111,993		129,676
Deferred reimbursements	7,406		7,406
Long-term debt	390,434		389,254
Contingent consideration payable	7,517		16,925
Deferred income taxes	4,896		4,896
Operating lease liabilities	44,201		45,604
Other non-current liabilities	6,535		6,379
Total liabilities	572,982		600,140
Commitments and contingencies	0		0
Stockholders’ equity:
Common stock, $0.01 par value, 500,000,000 shares authorized, 266,532,536 and 262,063,461 shares issued and outstanding at June 30, 2021 and December 31, 2020, respectively	2,685		2,650
Additional paid-in capital	2,364,494		2,308,578
Accumulated other comprehensive income (loss):
Foreign currency translation adjustment	9,255		8,412
Unrealized loss on available-for-sale securities	(173)		(185)
Warrants	0		12,387
Accumulated deficit	(2,162,351)		(2,045,462)
Total stockholders’ equity	213,910		286,380
Total Liabilities and Stockholders’ Equity	$	786,892	$	886,520


(in thousands)	June 30, 2021		June 30, 2020
Cash and cash equivalents	$	176,538	$	164,573
Restricted cash	3,250		2,689
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows	$	179,788	$	167,262