Financial Data and Key Metrics Changes - Total revenue for Q3 2023 reached $103.5 million, a 27% increase year-over-year, with a 22% growth at constant exchange rates [41][42] - Galafold achieved over $100 million in quarterly revenue for the first time, representing a 19% growth from Q3 2022 at constant currency [30][34] - The net loss for Q3 2023 was reduced to $22 million, or $0.07 per share, compared to a net loss of $33 million, or $0.12 per share in the prior year [42] Business Line Data and Key Metrics Changes - Galafold reported a revenue growth of 17% year-over-year at constant exchange rates, with a strong demand from both naive and treated patient populations [20][30] - Pombiliti and Opfolda are expected to generate around $10 million in revenue for the full year 2023, aligning with consensus expectations [24][31] - The company has transitioned all expanded access and clinical trial patients to commercial products in Germany, with positive feedback from healthcare providers [38][64] Market Data and Key Metrics Changes - The company holds approximately 60% of the global market share of treated admissible patients for Galafold, with strong uptake in naive populations [21][62] - The demand for Galafold continues to be strong globally, with significant growth expected in the family market, potentially reaching $1 billion in annual revenue in about five years [62][63] - The company is actively engaged in pricing and reimbursement negotiations in various countries, including Hong Kong, Turkey, and Singapore [22][64] Company Strategy and Development Direction - The company aims to sustain double-digit growth for Galafold and expand into new geographies while maintaining a strong financial position [19][33] - The focus is on transitioning clinical trial and expanded access patients to commercial supply while ensuring physicians have the necessary information about Pombiliti and Opfolda [64][66] - The company is investing in next-generation therapies for Fabry and Pompe diseases, including gene therapy and next-generation chaperones [40][94] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving non-GAAP profitability in Q4 2023, supported by strong revenue growth and careful expense management [18][42] - The company anticipates continued demand from newly diagnosed patients and aims to treat patients earlier in their disease progression [93] - Management highlighted the importance of generating new evidence and publications to support the launch of Pombiliti and Opfolda [6][9] Other Important Information - The company has submitted reimbursement dossiers in over a dozen countries and is focused on securing broad patient access throughout the EU [65] - The recent refinancing of debt under more favorable terms positions the company well for long-term growth [43] Q&A Session Questions and Answers Question: Can you provide more color on the cadence of the launch? - Management indicated that the goal is to convert existing patients first, with expectations for new patient starts to increase next year as the conversion process is completed [46][47] Question: What are the key regions for regulatory submissions in 2024? - Management noted that multiple regulatory submissions are planned for next year, with revenue contributions expected primarily in 2025 [50] Question: Can you clarify what PRFs are and the progress on commercial patients? - PRFs refer to patient referral forms, with 66% of clinical trial patients having submitted these forms for reimbursement [52][54] Question: How are physicians deciding between your product and Nixviazyme? - Approximately 75% of patients are reported by physicians as not improving on standard care, which drives the decision to consider switching to Pombiliti and Opfolda [87][88]

[Special Note Regarding Forward-Looking Statements](index=3&type=section&id=SPECIAL%20NOTE%20REGARDING%20FORWARD-LOOKING%20STATEMENTS) This section cautions that forward-looking statements, based on current expectations, involve risks and uncertainties, and actual results may differ materially, with no obligation for the company to update them [Forward-Looking Statements Disclosure](index=3&type=section&id=Forward-Looking%20Statements%20Disclosure) This section cautions that forward-looking statements, based on current expectations, involve risks and uncertainties, and actual results may differ materially, with no obligation for the company to update them - Forward-looking statements discuss current expectations and projections related to strategy, future operations, financial position, revenues, costs, prospects, plans, and management objectives[10](index=10&type=chunk) - Key factors that could cause actual results to differ materially include the scope, progress, results, and costs of clinical trials, manufacturing costs, regulatory review outcomes, commercialization activities, market opportunity estimates, and the ability to successfully commercialize Galafold, Pombiliti, and Opfolda[12](index=12&type=chunk)[13](index=13&type=chunk) - Investors are cautioned not to place undue reliance on forward-looking statements, and the company undertakes no obligation to publicly update or revise them, except as required by law[13](index=13&type=chunk)[14](index=14&type=chunk) [PART I. FINANCIAL INFORMATION](index=5&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Item 1. Consolidated Financial Statements and Notes (unaudited)](index=5&type=section&id=Item%201.%20Consolidated%20Financial%20Statements%20and%20Notes%20(unaudited)) This section presents Amicus Therapeutics' unaudited consolidated financial statements, including balance sheets, statements of operations, comprehensive loss, changes in equity, and cash flows, with notes on business and accounting policies [Consolidated Balance Sheets](index=5&type=section&id=Consolidated%20Balance%20Sheets) Consolidated Balance Sheet Highlights (in thousands) | Metric | Sep 30, 2023 | Dec 31, 2022 | Change | % Change | | :-------------------------------- | :----------- | :----------- | :------- | :------- | | Total Assets | $764,084 | $724,167 | $39,917 | 5.51% | | Cash and cash equivalents | $263,320 | $148,813 | $114,507 | 76.95% | | Investments in marketable securities | $16,980 | $144,782 | $(127,802) | -88.27% | | Total current assets | $463,256 | $423,816 | $39,440 | 9.31% | | Total Liabilities | $630,847 | $601,120 | $29,727 | 4.95% | | Total current liabilities | $169,454 | $139,018 | $30,436 | 21.90% | | Long-term debt | $394,071 | $391,990 | $2,081 | 0.53% | | Total Stockholders' Equity | $133,237 | $123,047 | $10,190 | 8.28% | [Consolidated Statements of Operations](index=6&type=section&id=Consolidated%20Statements%20of%20Operations) Consolidated Statements of Operations Highlights (in thousands, except per share) | Metric | 3 Months Ended Sep 30, 2023 | 3 Months Ended Sep 30, 2022 | YoY Change | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | YoY Change | | :----------------------------------- | :-------------------------- | :-------------------------- | :--------- | :-------------------------- | :-------------------------- | :--------- | | Net product sales | $103,501 | $81,691 | 26.71% | $284,274 | $241,137 | 17.89% | | Gross profit | $93,555 | $68,255 | 37.07% | $258,272 | $211,922 | 21.87% | | Research and development | $40,704 | $52,970 | -23.16% | $117,352 | $212,806 | -44.95% | | Selling, general, and administrative | $65,651 | $47,272 | 38.88% | $205,031 | $158,767 | 29.14% | | Loss from operations | $(17,023) | $(33,840) | 49.68% | $(73,519) | $(169,792) | 56.69% | | Net loss attributable to common stockholders | $(21,577) | $(33,286) | 35.20% | $(117,741) | $(180,703) | 34.86% | | Net loss per common share (basic and diluted) | $(0.07) | $(0.12) | 41.67% | $(0.40) | $(0.63) | 36.51% | [Consolidated Statements of Comprehensive Loss](index=7&type=section&id=Consolidated%20Statements%20of%20Comprehensive%20Loss) Consolidated Statements of Comprehensive Loss Highlights (in thousands) | Metric | 3 Months Ended Sep 30, 2023 | 3 Months Ended Sep 30, 2022 | YoY Change | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | YoY Change | | :----------------------------------- | :-------------------------- | :-------------------------- | :--------- | :-------------------------- | :-------------------------- | :--------- | | Net loss | $(21,577) | $(33,286) | 35.20% | $(117,741) | $(180,703) | 34.86% | | Other comprehensive (loss) gain | $(10,928) | $(21,838) | 49.96% | $5,337 | $(44,059) | 112.11% | | Comprehensive loss | $(32,505) | $(55,124) | 41.03% | $(112,404) | $(224,762) | 50.00% | [Consolidated Statements of Changes in Stockholders' Equity](index=8&type=section&id=Consolidated%20Statements%20of%20Changes%20in%20Stockholders'%20Equity) Changes in Stockholders' Equity (in thousands) | Metric | Balance at Dec 31, 2022 | 9 Months Ended Sep 30, 2023 Changes | Balance at Sep 30, 2023 | | :-------------------------- | :---------------------- | :------------------------------------ | :---------------------- | | Common Stock Amount | $2,815 | $75 | $2,890 | | Additional Paid-In Capital | $2,664,744 | $122,531 | $2,787,275 | | Accumulated Other Comprehensive Loss | $(12,105) | $5,337 | $(6,768) | | Accumulated Deficit | $(2,532,490) | $(117,741) | $(2,650,231) | | Total Stockholders' Equity | $123,047 | $10,190 | $133,237 | - The company issued **5,244,936 shares** through its at-the-market (ATM) offering, generating net proceeds of **$63.1 million** during the nine months ended September 30, 2023[26](index=26&type=chunk) [Consolidated Statements of Cash Flows](index=10&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) Consolidated Statements of Cash Flows Highlights (in thousands) | Metric | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | YoY Change | | :----------------------------------- | :-------------------------- | :-------------------------- | :--------- | | Net cash used in operating activities | $(72,549) | $(85,803) | 15.44% | | Net cash provided by investing activities | $122,021 | $159,020 | -23.27% | | Net cash provided by (used in) financing activities | $53,467 | $(8,020) | 766.05% | | Net increase in cash, cash equivalents, and restricted cash | $113,157 | $32,077 | 252.76% | | Cash, cash equivalents, and restricted cash at end of period | $266,272 | $281,533 | -5.42% | [Notes to Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) [1. Description of Business](index=11&type=section&id=1.%20Description%20of%20Business) - Amicus Therapeutics is a global biotechnology company focused on rare diseases, commercializing Galafold for Fabry disease and Pombiliti + Opfolda for late-onset Pompe disease[37](index=37&type=chunk)[38](index=38&type=chunk) - Galafold is the first oral monotherapy for Fabry disease, approved in the U.S., E.U., U.K., and Japan[38](index=38&type=chunk) - Pombiliti + Opfolda, a two-component treatment for late-onset Pompe disease, received approvals in the E.U. (June 2023), U.K. (August 2023), and U.S. (September 2023), with launch activities commenced[38](index=38&type=chunk) - The company had an accumulated deficit of **$2.7 billion** as of September 30, 2023, and expects to incur losses through fiscal year 2023[38](index=38&type=chunk) - In October 2023, the company secured a **$400 million** Senior Secured Term Loan due 2029 and a **$29.8 million** private placement, using proceeds to prepay existing debt and fund operations[38](index=38&type=chunk)[39](index=39&type=chunk) - Management believes current cash, including expected revenues, is sufficient to fund operations and research programs for at least the next 12 months[40](index=40&type=chunk) [2. Summary of Significant Accounting Policies](index=12&type=section&id=2.%20Summary%20of%20Significant%20Accounting%20Policies) - Unaudited Consolidated Financial Statements are prepared in accordance with U.S. GAAP for interim financial information[41](index=41&type=chunk) - The functional currency for most foreign subsidiaries is their local currency, with translation adjustments recorded in accumulated other comprehensive income[44](index=44&type=chunk) - Cash equivalents are highly liquid investments with maturities of three months or less; marketable securities are classified as available-for-sale and reported at fair value[46](index=46&type=chunk) - Revenue is recognized when performance obligations are satisfied, typically when pharmacies or distributors obtain control, net of estimated variable consideration[52](index=52&type=chunk) Net Product Sales by Product (in thousands) | Product | 3 Months Ended Sep 30, 2023 | 3 Months Ended Sep 30, 2022 | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | | :-------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Galafold® | $100,733 | $81,631 | $281,177 | $241,056 | | Pombiliti™ + Opfolda™ | $2,768 | $60 | $3,097 | $81 | | Total | $103,501 | $81,691 | $284,274 | $241,137 | Net Product Sales by Geographic Area (in thousands) | Geographic Area | 3 Months Ended Sep 30, 2023 | 3 Months Ended Sep 30, 2022 | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | | :---------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | U.S. | $37,801 | $30,222 | $103,760 | $81,940 | | Ex-U.S. | $65,700 | $51,469 | $180,514 | $159,197 | | Total | $103,501 | $81,691 | $284,274 | $241,137 | - Manufacturing costs for Pombiliti + Opfolda were expensed as R&D until regulatory approval, then capitalized[54](index=54&type=chunk) - Goodwill and finite-lived intangible assets are assessed annually for impairment; no impairment indicators were noted for the nine months ended September 30, 2023[57](index=57&type=chunk)[58](index=58&type=chunk)[59](index=59&type=chunk) [3. Intangible Assets](index=14&type=section&id=3.%20Intangible%20Assets) - Intangible assets primarily consist of lead enzyme replacement therapy assets from the 2013 Callidus Biopharma acquisition[61](index=61&type=chunk) - Following EC approval of Pombiliti in March 2023, these assets began to be amortized over an initial regulatory exclusivity period of **7 years**[61](index=61&type=chunk) Amortization Expense (in thousands) | Period | Amortization Expense | | :-------------------------- | :------------------- | | 3 Months Ended Sep 30, 2023 | $0.8 million | | 9 Months Ended Sep 30, 2023 | $1.7 million | | Estimated for Year Ending Dec 31, 2023 | $2.5 million | | Estimated for Next Four Years (per year) | $3.3 million | [4. Cash, Cash Equivalents, Marketable Securities, and Restricted Cash](index=15&type=section&id=4.%20Cash,%20Cash%20Equivalents,%20Marketable%20Securities,%20and%20Restricted%20Cash) Cash, Cash Equivalents, and Marketable Securities (in thousands) | Category | Sep 30, 2023 (Fair Value) | Dec 31, 2022 (Fair Value) | | :----------------------------------- | :-------------------------- | :-------------------------- | | Cash and cash equivalents | $263,320 | $148,813 | | Marketable securities | $16,980 | $144,782 | | Total | $280,300 | $293,595 | - The company invests excess operating cash in high-credit-rating deposits, money market funds, and fixed income investments[63](index=63&type=chunk) - Unrealized losses on marketable securities as of September 30, 2023, are considered temporary impairments, not credit losses, and are recognized in other comprehensive (loss) gain[65](index=65&type=chunk) Reconciliation of Cash, Cash Equivalents, and Restricted Cash (in thousands) | Category | Sep 30, 2023 | Sep 30, 2022 | | :---------------------------------------------------------------- | :----------- | :----------- | | Cash and cash equivalents | $263,320 | $277,592 | | Restricted cash | $2,952 | $3,941 | | Total cash, cash equivalents, and restricted cash (Cash Flow Statement) | $266,272 | $281,533 | [5. Inventories](index=16&type=section&id=5.%20Inventories) Inventories (in thousands) | Category | Sep 30, 2023 | Dec 31, 2022 | | :--------------- | :----------- | :----------- | | Raw materials | $36,062 | $10,054 | | Work-in-process | $14,316 | $9,615 | | Finished goods | $6,558 | $4,147 | | Total inventories | $56,936 | $23,816 | - The company's reserve for inventory was **$0.3 million** as of September 30, 2023, down from **$0.4 million** at December 31, 2022[68](index=68&type=chunk) [6. Debt](index=16&type=section&id=6.%20Debt) Long-term Debt (in thousands) | Metric | Sep 30, 2023 | Dec 31, 2022 | | :-------------------------------- | :----------- | :----------- | | Senior Secured Term Loan due 2026 (Principal) | $400,000 | $400,000 | | Less: debt discount | $(3,384) | $(4,579) | | Less: deferred financing | $(2,545) | $(3,431) | | Net carrying value of Long-term debt | $394,071 | $391,990 | - In October 2023, the company entered into a new **$400 million** Senior Secured Term Loan due 2029 with Blackstone, with net proceeds of **$387.4 million**[69](index=69&type=chunk) - Proceeds from the new loan and a private placement were used to prepay the Senior Secured Term Loan due 2026, expecting a **$13.9 million** loss from early extinguishment of debt in Q4 2023[70](index=70&type=chunk) Interest Expense (in thousands) | Metric | 3 Months Ended Sep 30, 2023 | 3 Months Ended Sep 30, 2022 | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Contractual interest expense | $12,270 | $8,945 | $35,289 | $24,030 | | Amortization of debt discount | $411 | $382 | $1,187 | $1,120 | | Amortization of deferred financing | $310 | $286 | $893 | $840 | [7. Stockholder's Equity](index=17&type=section&id=7.%20Stockholder's%20Equity) - During the nine months ended September 30, 2023, the company issued **5,244,936 shares** through its at-the-market (ATM) equity program, generating net proceeds of **$63.1 million**[73](index=73&type=chunk) - As of September 30, 2023, **$184.4 million** worth of shares remain available under the ATM program[73](index=73&type=chunk) - In October 2023, a private placement of **2,467,104 common shares** to Blackstone generated **$29.8 million** in net proceeds[74](index=74&type=chunk) [8. Stock-Based Compensation](index=17&type=section&id=8.%20Stock-Based%20Compensation) - The company's Amended and Restated 2007 Equity Incentive Plan allows for granting restricted stock units (RSUs) and stock options to employees, directors, advisors, and consultants[75](index=75&type=chunk) Stock Option Activity (9 Months Ended Sep 30, 2023) | Metric | Number of Shares (in thousands) | Weighted Average Exercise Price | | :------------------------------------ | :------------------------------ | :------------------------------ | | Options outstanding, Dec 31, 2022 | 19,064 | $11.31 | | Granted | 5,522 | $12.03 | | Exercised | (1,032) | $7.61 | | Forfeited | (248) | $11.51 | | Expired | (50) | $14.39 | | Options outstanding, Sep 30, 2023 | 23,256 | $11.64 | - As of September 30, 2023, total unrecognized compensation cost for non-vested stock options was **$39.8 million**, to be recognized over a weighted average period of **three years**[77](index=77&type=chunk) Non-Vested RSU Activity (9 Months Ended Sep 30, 2023) | Metric | Number of Shares (in thousands) | Weighted Average Grant Date Fair Value | | :------------------------------------ | :------------------------------ | :------------------------------------- | | Non-vested units as of Dec 31, 2022 | 9,717 | $13.07 | | Granted | 4,514 | $13.08 | | Vested | (3,252) | $12.25 | | Forfeited | (648) | $10.07 | | Non-vested units as of Sep 30, 2023 | 10,331 | $13.56 | - As of September 30, 2023, **$63.1 million** of unrecognized compensation cost related to unvested RSUs is expected to be recognized over a weighted average period of **two years**[78](index=78&type=chunk) Total Equity Compensation Expense (in thousands) | Expense Category | 3 Months Ended Sep 30, 2023 | 3 Months Ended Sep 30, 2022 | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | | :-------------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Research and development expense | $4,380 | $5,428 | $16,987 | $19,170 | | Selling, general, and administrative expense | $12,131 | $9,344 | $50,995 | $38,716 | | Total equity compensation expense | $16,511 | $14,772 | $67,982 | $57,886 | [9. Assets and Liabilities Measured at Fair Value](index=19&type=section&id=9.%20Assets%20and%20Liabilities%20Measured%20at%20Fair%20Value) - Financial assets and liabilities are classified within a fair value hierarchy: Level 1 (quoted prices in active markets), Level 2 (observable inputs other than quoted prices), and Level 3 (unobservable inputs)[80](index=80&type=chunk)[81](index=81&type=chunk) Fair Value of Recurring Assets (in thousands) | Asset Category | Sep 30, 2023 (Level 2) | Dec 31, 2022 (Level 2) | | :--------------- | :--------------------- | :--------------------- | | Commercial paper | $16,829 | $144,381 | | Money market | $6,818 | $5,808 | | Total | $23,647 | $150,189 | Fair Value of Recurring Liabilities (in thousands) | Liability Category | Sep 30, 2023 (Level 2) | Dec 31, 2022 (Level 2) | Dec 31, 2022 (Level 3) | | :-------------------------- | :--------------------- | :--------------------- | :--------------------- | | Deferred compensation plan liability | $6,718 | $5,458 | — | | Contingent consideration payable | — | — | $21,417 | | Total | $6,718 | $5,458 | $21,417 | - The company had no Level 3 assets as of September 30, 2023, or December 31, 2022[82](index=82&type=chunk) - Contingent consideration payable resulted from the Callidus Biopharma acquisition, with regulatory milestones of **$9.0 million** (EC approval) and **$15.0 million** (FDA approval) for Pombiliti reached in March and September 2023, respectively[84](index=84&type=chunk) Contingent Consideration Payable (in thousands) | Metric | 3 Months Ended Sep 30, 2023 | 3 Months Ended Sep 30, 2022 | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | | :---------------------------------------------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Balance, beginning of the period | $13,005 | $19,266 | $21,417 | $20,339 | | Changes in fair value during the period | $1,995 | $567 | $2,583 | $(506) | | Milestone paid or payable in cash | $(15,000) | — | $(24,000) | — | | Balance, end of the period | — | $19,833 | — | $19,833 | [10. Basic and Diluted Net Loss per Common Share](index=20&type=section&id=10.%20Basic%20and%20Diluted%20Net%20Loss%20per%20Common%20Share) Net Loss per Common Share (in thousands, except per share amounts) | Metric | 3 Months Ended Sep 30, 2023 | 3 Months Ended Sep 30, 2022 | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | | :---------------------------------------------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Net loss attributable to common stockholders | $(21,577) | $(33,286) | $(117,741) | $(180,703) | | Weighted average common shares outstanding — basic and diluted | 295,759,435 | 289,223,709 | 293,314,167 | 288,841,092 | | Net loss per common share — basic and diluted | $(0.07) | $(0.12) | $(0.40) | $(0.63) | - Potentially dilutive common stock equivalents (options and unvested RSUs) were excluded from diluted EPS calculations due to their anti-dilutive effect[85](index=85&type=chunk) Potential Shares Excluded from Diluted EPS (in thousands) | Category | As of Sep 30, 2023 | As of Sep 30, 2022 | | :-------------------------------- | :----------------- | :----------------- | | Options to purchase common stock | 23,256 | 19,230 | | Unvested restricted stock units | 10,331 | 9,830 | | Total number of potentially issuable shares | 33,587 | 29,070 | [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=21&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's financial condition and results of operations, covering business developments, product performance, strategic initiatives, and an analysis of financial results, liquidity, and critical accounting policies [Overview](index=21&type=section&id=Overview) - Amicus Therapeutics is a global biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases[88](index=88&type=chunk) - The company has commercialized Galafold for Fabry disease and Pombiliti + Opfolda for late-onset Pompe disease, with recent approvals in the E.U., U.K., and U.S. for the latter[88](index=88&type=chunk)[89](index=89&type=chunk) [Our Strategy](index=21&type=section&id=Our%20Strategy) - The company's strategy is to create and deliver high-quality medicines for rare diseases through internally developed, jointly developed, acquired, or in-licensed products[90](index=90&type=chunk) - Key progress highlights include strong commercial momentum for Galafold (**$281.2 million** revenue for nine months ended Sep 30, 2023), regulatory approvals for Pombiliti + Opfolda, pipeline advancement in Fabry and Pompe diseases, and a strong financial position with **$280.3 million** in cash, cash equivalents, and marketable securities as of September 30, 2023[91](index=91&type=chunk) - Current cash position is believed to be sufficient to fund operations and research programs for at least the next 12 months[91](index=91&type=chunk) [Our Commercial Products and Product Candidates](index=22&type=section&id=Our%20Commercial%20Products%20and%20Product%20Candidates) - Galafold (migalastat HCl) is an oral precision medicine for Fabry disease, approved for **351 amenable GLA variants** in the U.S. and **1,384 mutations** in the E.U. and U.K., covering up to half of all Fabry patients[93](index=93&type=chunk) - The Galafold U.S. patent portfolio includes **54 Orange Book listed patents**, with **38** providing protection through at least 2038[93](index=93&type=chunk) - Pombiliti + Opfolda (AT-GAA) is a novel two-component treatment for late-onset Pompe disease, approved by the EC (June 2023), MHRA (August 2023), and FDA (September 2023)[95](index=95&type=chunk) - The company is also pursuing next-generation genetic medicines for both Fabry and Pompe diseases and evaluating business development opportunities for rare and orphan diseases[94](index=94&type=chunk)[96](index=96&type=chunk)[97](index=97&type=chunk) [Consolidated Results of Operations - Three Months Ended September 30, 2023 compared to September 30, 2022](index=23&type=section&id=Consolidated%20Results%20of%20Operations%20-%20Three%20Months%20Ended%20September%2030,%202023%20compared%20to%20September%2030,%202022) Key Financial Changes (3 Months Ended Sep 30, 2023 vs 2022, in thousands) | Metric | 2023 | 2022 | Change | | :----------------------------------- | :----------- | :----------- | :------- | | Net product sales | $103,501 | $81,691 | $21,810 | | Cost of goods sold | $9,946 | $13,436 | $(3,490) | | Cost of goods sold as % of net product sales | 9.6% | 16.4% | -6.8% | | Research and development | $40,704 | $52,970 | $(12,266) | | Selling, general, and administrative | $65,651 | $47,272 | $18,379 | | Net loss attributable to common stockholders | $(21,577) | $(33,286) | $11,709 | - Net product sales increased by **$21.8 million**, driven by Galafold growth in the U.S., Europe, and Japan, Pombiliti + Opfolda launch in Europe, and a **$3.8 million** favorable foreign currency exchange impact[98](index=98&type=chunk) - Research and development costs decreased by **$12.3 million** primarily due to reduced clinical manufacturing costs for the Pompe disease program, with resources reallocated to commercial launch activities[98](index=98&type=chunk) - Selling, general, and administrative expense increased by **$18.4 million** due to personnel costs supporting Pombiliti + Opfolda commercial launch and third-party professional fees[99](index=99&type=chunk) [Consolidated Results of Operations - Nine Months Ended September 30, 2023 compared to September 30, 2022](index=24&type=section&id=Consolidated%20Results%20of%20Operations%20-%20Nine%20Months%20Ended%20September%2030,%202023%20compared%20to%20September%2030,%202022) Key Financial Changes (9 Months Ended Sep 30, 2023 vs 2022, in thousands) | Metric | 2023 | 2022 | Change | | :----------------------------------- | :----------- | :----------- | :------- | | Net product sales | $284,274 | $241,137 | $43,137 | | Cost of goods sold | $26,002 | $29,215 | $(3,213) | | Cost of goods sold as % of net product sales | 9.1% | 12.1% | -3.0% | | Research and development | $117,352 | $212,806 | $(95,454) | | Selling, general, and administrative | $205,031 | $158,767 | $46,264 | | Net loss attributable to common stockholders | $(117,741) | $(180,703) | $62,962 | - Net product sales increased by **$43.1 million**, driven by continued growth of Galafold and the launch of Pombiliti + Opfolda in Europe[101](index=101&type=chunk) - Research and development costs decreased by **$95.5 million**, primarily due to the strategic deprioritization of the gene therapy portfolio and reduced clinical manufacturing costs for the Pompe disease program[102](index=102&type=chunk) - Selling, general, and administrative expense increased by **$46.3 million**, mainly due to personnel costs for Pombiliti + Opfolda commercial launch and third-party professional fees[102](index=102&type=chunk) - The company recognized an income tax benefit primarily from a partial release of a valuation allowance on deferred tax assets due to reclassification of in-process R&D to a definite-lived intangible asset[104](index=104&type=chunk) [Liquidity and Capital Resources](index=25&type=section&id=Liquidity%20and%20Capital%20Resources) - The company has historically funded operations through stock offerings, product revenues, debt issuance, and collaborations, having generated operating losses since 2002[105](index=105&type=chunk) - As of September 30, 2023, cash, cash equivalents, and marketable securities totaled **$280.3 million**[107](index=107&type=chunk) - Net cash used in operating activities was **$72.5 million** for the nine months ended September 30, 2023, an improvement from **$85.8 million** in the prior year, driven by lower net loss and increased accounts payable[108](index=108&type=chunk) - Net cash provided by investing activities was **$122.0 million** for the nine months ended September 30, 2023, primarily from the sale and redemption of marketable securities[109](index=109&type=chunk) - Net cash provided by financing activities was **$53.5 million** for the nine months ended September 30, 2023, mainly from ATM program proceeds (**$63.1 million**) and stock option exercises, partially offset by withholding taxes on RSUs[111](index=111&type=chunk) - Future capital requirements depend on clinical trial costs, manufacturing, regulatory approvals, commercialization, and potential business development[113](index=113&type=chunk) - Management believes current cash and expected revenues are sufficient for at least the next 12 months, but may seek additional funding[114](index=114&type=chunk) [Critical Accounting Policies and Significant Judgments](index=28&type=section&id=Critical%20Accounting%20Policies%20and%20Significant%20Judgments) - The preparation of financial statements requires management to make estimates and assumptions affecting reported amounts of assets, liabilities, revenues, and expenses[115](index=115&type=chunk) - No significant changes occurred during the nine months ended September 30, 2023, to the previously disclosed significant accounting policies and estimates[116](index=116&type=chunk) [Recent Accounting Pronouncements](index=28&type=section&id=Recent%20Accounting%20Pronouncements) - The company has evaluated recent accounting pronouncements and believes none will have a material effect on its Consolidated Financial Statements or related disclosures[60](index=60&type=chunk)[117](index=117&type=chunk) [Item 3. Quantitative and Qualitative Disclosures about Market Risk](index=29&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) This section discusses the company's market risk exposure, primarily interest rate risk on variable-rate debt, noting the transition from LIBOR to Adjusted Term SOFR and estimating interest rate change impacts - Effective July 1, 2023, the reference rate for the Senior Secured Term Loan due 2026 transitioned from LIBOR to Adjusted Term SOFR, plus a credit spread adjustment of **0.26%**[119](index=119&type=chunk) - A hypothetical **100 basis point** increase or decrease in the interest rate on variable-rate debt would result in a **$1.0 million** change in quarterly interest expense as of September 30, 2023[119](index=119&type=chunk) - The Senior Secured Term Loan due 2026 was prepaid using proceeds from the new Senior Secured Term Loan due 2029 and a private placement[120](index=120&type=chunk) - No material changes to market risks or their management have occurred since December 31, 2022, except for the debt reference rate transition[121](index=121&type=chunk) [Item 4. Controls and Procedures](index=29&type=section&id=Item%204.%20Controls%20and%20Procedures) This section confirms the effectiveness of disclosure controls and procedures, reporting no material changes in internal control over financial reporting during the quarter - The Principal Executive Officer and Principal Financial Officer concluded that disclosure controls and procedures were effective as of September 30, 2023[122](index=122&type=chunk) - No material changes in internal control over financial reporting occurred during the fiscal quarter ended September 30, 2023[123](index=123&type=chunk) [PART II. OTHER INFORMATION](index=29&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=29&type=section&id=Item%201.%20Legal%20Proceedings) This section discloses legal proceedings, specifically patent infringement lawsuits filed by the company against generic drug manufacturers regarding Galafold - In Q4 2022, the company received Paragraph IV Certification Notice Letters from Teva, Aurobindo, and Lupin regarding Abbreviated New Drug Applications (ANDA) for generic Galafold[124](index=124&type=chunk) - The company filed four lawsuits against Teva, Lupin, and Aurobindo in November 2022 for infringement of its Orange Book-listed patents and intends to vigorously enforce its Galafold intellectual property rights[124](index=124&type=chunk) [Item 1A. Risk Factors](index=29&type=section&id=Item%201A.%20Risk%20Factors) This section states no material changes to risk factors previously disclosed in the Annual Report on Form 10-K for the fiscal year ended December 31, 2022 - No material changes to the risk factors previously disclosed in the Annual Report on Form 10-K for the fiscal year ended December 31, 2022, have occurred[125](index=125&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=29&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This section reports on the company's equity security purchases, specifically shares withheld to satisfy taxes on vested restricted stock units - No unregistered sales of equity securities were reported[126](index=126&type=chunk) Issuer Purchases of Equity Securities (3 Months Ended Sep 30, 2023) | Period | Total Number of Shares Purchased | Average Price Paid per Share | | :------------------------------------ | :----------------------------- | :--------------------------- | | July 1, 2023 through July 31, 2023 | 53,583 | $13.40 | | August 1, 2023 through August 31, 2023 | 123,214 | $12.79 | | September 1, 2023 through September 30, 2023 | 24,701 | $13.07 | | Total | 201,498 | $12.99 | - Shares purchased represent common stock withheld to satisfy taxes associated with the vesting of restricted stock units[127](index=127&type=chunk) [Item 3. Defaults Upon Senior Securities](index=30&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) This section reports no defaults upon senior securities during the reported period - No defaults upon senior securities were reported[128](index=128&type=chunk) [Item 4. Mine Safety Disclosures](index=30&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This section indicates no mine safety disclosures to report - No mine safety disclosures were reported[129](index=129&type=chunk) [Item 5. Other Information](index=31&type=section&id=Item%205.%20Other%20Information) This section details Rule 10b5-1 Trading Plans adopted by the company's directors and officers during the quarter, outlining their nature and duration - Several directors and officers adopted Rule 10b5-1 Trading Plans during the quarter ended September 30, 2023, for the sale of common stock[131](index=131&type=chunk)[133](index=133&type=chunk) - These plans were adopted during an open insider trading window and in accordance with company policies[131](index=131&type=chunk) - The plans involve the exercise of stock options and sale of underlying shares, or the sale of shares obtained from the settlement of restricted stock unit awards, with the number of shares often indeterminable due to performance goals and tax withholdings[132](index=132&type=chunk) [Item 6. Exhibits](index=32&type=section&id=Item%206.%20Exhibits) This section lists all exhibits filed with the Form 10-Q, including loan agreements, securities purchase agreements, employment agreements, certifications, and XBRL documents - Exhibits include the Loan Agreement and Securities Purchase Agreement dated October 2, 2023, related to the new Senior Secured Term Loan and private placement[134](index=134&type=chunk) - Certifications from the Principal Executive Officer and Principal Financial Officer pursuant to Rules 13a-14, 15d-14, and Section 906 of the Sarbanes-Oxley Act of 2002 are included[134](index=134&type=chunk) - Inline XBRL documents (Instance, Schema, Calculation, Label, Presentation, Definition Linkbase) are provided for interactive data filing[134](index=134&type=chunk)[135](index=135&type=chunk) [SIGNATURES](index=33&type=section&id=SIGNATURES) [Report Signatures](index=33&type=section&id=Report%20Signatures) This section contains the official signatures of the company's President and CEO, Bradley L. Campbell, and CFO, Simon Harford, certifying the report filing - The report is duly signed on November 8, 2023, by Bradley L. Campbell, President and Chief Executive Officer, and Simon Harford, Chief Financial Officer[139](index=139&type=chunk)

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-33497 Amicus Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 71-0869350 (State or Other Jurisd ...

For the quarterly period ended March 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to 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 Commission file number 001-33497 Amicus Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 71-0869350 (State or Other Juris ...

Galafold Performance and Outlook - Galafold achieved revenue of $329 million in 2022, with a 16% operational growth at Constant Exchange Rates (CER)[17, 37] - Q4 2022 Galafold revenue reached $88 million[14] - The company anticipates Galafold revenue growth of 12-17% at CER in 2023[3, 9, 25] - Galafold has treated over 2,000 individuals globally[18, 20] AT-GAA Development and Regulatory Status - AT-GAA is under global regulatory review for Pompe disease[3] - Regulatory approvals for AT-GAA in the three largest Pompe markets are anticipated in 2023[31] - The European Commission (EC) decision for Pombiliti™ (cipaglucosidase alfa) is expected in Q1 2023, with a CHMP opinion for Miglustat expected in Q2 2023 and an EC decision in Q3 2023[31] - FDA approval for AT-GAA is anticipated in Q3 2023[31] Financial Goals and Profitability - The company is focused on prudent expense management and aims to achieve non-GAAP profitability in the second half of 2023[3, 25, 39] - 2023 non-GAAP operating expense guidance is set at $340 million to $360 million[25] - Cash as of December 31, 2022, was $294 million[3] AT-GAA Clinical Data - Approximately 200 people living with Pompe disease are currently receiving AT-GAA through extension studies and expanded access programs[23]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-33497 Amicus Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 71-0869350 (State or Other Jurisdicti ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-33497 Amicus Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 71-0869350 (State or Other J ...

Galafold Performance and Outlook - Galafold achieved $80.7 million in revenue for 2Q22[10], contributing to $1594 million in 1H22 revenue, representing a 109% reported growth[11] and 178% operational growth at CER[11] - The company is reiterating FY22 Galafold revenue guidance of $350 million to $365 million at CER, indicating a 15% to 20% growth at CER[4, 8, 10, 12] - The company anticipates near-term growth to $500 million, driven by continued penetration into existing markets, expansion into new geographies, and broadening of labels[13] AT-GAA Development and Launch - AT-GAA is under global regulatory reviews, with a U S PDUFA date in 2H2022 and a CHMP opinion expected late 2022[4, 20, 21] - Over 175 people living with Pompe disease are currently receiving AT-GAA through clinical extension studies and expanded access programs[20] - Phase 3 PROPEL study results showed greater improvement with AT-GAA vs alglucosidase alfa in ERT-naïve and ERT-experienced patients[18] Financial Position and Strategy - The company reported $386.8 million in cash as of June 30, 2022[4] - The company is focused on prudent expense management to achieve profitability in 2023[4, 5, 39] - 2Q22 operating expenses increased, reflecting manufacturing costs for AT-GAA launch and non-recurring expenses related to gene therapy portfolio reprioritization[37] - The company's 2022 non-GAAP operating expense guidance is $470 million to $485 million[39]

PART I. FINANCIAL INFORMATION [Consolidated Financial Statements and Notes (unaudited)](index=6&type=section&id=Item%201.%20Consolidated%20Financial%20Statements%20and%20Notes%20(unaudited)) The company's Q2 2022 financial statements reflect increased revenue but a wider net loss, driven by higher operating expenses and a non-recurring charge [Consolidated Balance Sheets](index=6&type=section&id=Consolidated%20Balance%20Sheets) As of June 30, 2022, total assets decreased to **$800.6 million**, liabilities increased to **$627.2 million**, and stockholders' equity declined to **$173.4 million** Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2022 | December 31, 2021 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $235,639 | $245,197 | | Total current assets | $497,643 | $596,834 | | Total Assets | $800,589 | $905,140 | | **Liabilities & Equity** | | | | Total current liabilities | $164,625 | $145,975 | | Long-term debt | $390,652 | $389,357 | | Total liabilities | $627,164 | $597,771 | | Total stockholders' equity | $173,425 | $307,369 | [Consolidated Statements of Operations](index=7&type=section&id=Consolidated%20Statements%20of%20Operations) Q2 2022 net product sales increased to **$80.7 million**, but net loss widened to **$62.2 million** due to higher R&D and SG&A expenses Statement of Operations Summary (in thousands, except per share data) | Metric | Q2 2022 | Q2 2021 | H1 2022 | H1 2021 | | :--- | :--- | :--- | :--- | :--- | | Net product sales | $80,731 | $77,413 | $159,446 | $143,815 | | Gross profit | $72,534 | $69,033 | $143,667 | $128,896 | | Research and development | $78,319 | $63,003 | $159,836 | $127,120 | | Selling, general, and administrative | $53,379 | $42,276 | $111,495 | $89,002 | | Loss from operations | $(60,613) | $(38,834) | $(135,952) | $(91,889) | | Net loss | $(62,157) | $(51,225) | $(147,417) | $(116,889) | | Net loss per share | $(0.21) | $(0.19) | $(0.51) | $(0.44) | [Consolidated Statements of Cash Flows](index=11&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operating activities improved to **$74.2 million** in H1 2022, with overall cash decreasing by **$9.6 million** Cash Flow Summary for Six Months Ended June 30 (in thousands) | Activity | 2022 | 2021 | | :--- | :--- | :--- | | Net cash used in operating activities | $(74,177) | $(108,277) | | Net cash provided by investing activities | $84,505 | $112,278 | | Net cash (used in) provided by financing activities | $(7,461) | $11,064 | | **Net (decrease) increase in cash** | **$(9,595)** | **$13,626** | [Notes to Consolidated Financial Statements](index=12&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) Notes detail business focus, accounting policies, AT-GAA regulatory status, a **$20.0 million** non-recurring charge, **$6.6 million** asset impairment, **$400 million** term loan, and **$19.3 million** contingent consideration - The company's lead pipeline biologic, AT-GAA for Pompe disease, is under review by the FDA and EMA. The FDA extended the PDUFA action dates to August 29, 2022 (for miglustat) and October 29, 2022 (for cipaglucosidase alfa)[38](index=38&type=chunk) - In Q2 2022, due to a strategic prioritization of its gene therapy portfolio, the company recorded a non-recurring **$20.0 million** liability for contractual obligations and recognized a **$6.6 million** impairment charge on fixed assets[60](index=60&type=chunk)[54](index=54&type=chunk) - The company's debt consists of a **$400 million** Senior Secured Term Loan due 2026, with a net carrying value of **$390.7 million** as of June 30, 2022[69](index=69&type=chunk) - Contingent consideration payable for the ATB200 Pompe disease program (from the Callidus acquisition) was valued at **$19.3 million** as of June 30, 2022, classified as a Level 3 liability[80](index=80&type=chunk)[82](index=82&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=23&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses Galafold's success, AT-GAA's progress, and H1 2022 financial results showing revenue growth offset by wider losses due to increased expenses and one-time charges - The company's strategy is to develop first- or best-in-class medicines for rare diseases, leveraging its global capabilities to broaden its lead franchises in Fabry and Pompe disease[89](index=89&type=chunk) - Total cash, cash equivalents, and marketable securities were **$386.8 million** as of June 30, 2022. The company believes this position is sufficient to fund operations to achieve self-sustainability[92](index=92&type=chunk)[112](index=112&type=chunk) - The company faces significant future potential milestone payments related to its acquisitions and collaborations, including up to **$88 million** remaining for the Callidus acquisition (Pompe program) and up to **$88 million** per indication for its collaboration with the University of Pennsylvania[120](index=120&type=chunk)[123](index=123&type=chunk) [Results of Operations](index=26&type=section&id=Results%20of%20Operations) Net product sales increased year-over-year, but operating expenses rose significantly due to non-recurring charges and higher personnel costs, leading to wider net losses Comparison of Three Months Ended June 30, 2022 and 2021 (in thousands) | Metric | 2022 | 2021 | Change | | :--- | :--- | :--- | :--- | | Net product sales | $80,731 | $77,413 | $3,318 | | Research and development | $78,319 | $63,003 | $15,316 | | Selling, general, and administrative | $53,379 | $42,276 | $11,103 | | Net loss | $(62,157) | $(51,225) | $(10,932) | - The **$15.3 million** increase in Q2 2022 R&D costs was primarily driven by a non-recurring expense from a contractual obligation related to the strategic prioritization of the gene therapy portfolio[103](index=103&type=chunk) Comparison of Six Months Ended June 30, 2022 and 2021 (in thousands) | Metric | 2022 | 2021 | Change | | :--- | :--- | :--- | :--- | | Net product sales | $159,446 | $143,815 | $15,631 | | Research and development | $159,836 | $127,120 | $32,716 | | Selling, general, and administrative | $111,495 | $89,002 | $22,493 | | Loss on impairment of assets | $6,616 | $— | $6,616 | | Net loss | $(147,417) | $(116,889) | $(30,528) | - For H1 2022, net product sales growth of **$15.6 million** was partially offset by an unfavorable foreign currency impact of **$9.9 million**[106](index=106&type=chunk) [Liquidity and Capital Resources](index=28&type=section&id=Liquidity%20and%20Capital%20Resources) The company ended Q2 2022 with **$386.8 million** in cash and marketable securities, believing it sufficient to fund operations to self-sustainability - As of June 30, 2022, the company had cash, cash equivalents, and marketable securities of **$386.8 million**[112](index=112&type=chunk) - Net cash used in operating activities for H1 2022 was **$74.2 million**, an improvement from **$108.3 million** in H1 2021. The change was primarily due to non-cash adjustments and timing of payments for R&D and other expenses[113](index=113&type=chunk) - The company has significant potential future milestone and royalty payment obligations to partners including Callidus, Celenex, Nationwide Children's Hospital, University of Pennsylvania, and GSK[120](index=120&type=chunk)[121](index=121&type=chunk)[122](index=122&type=chunk)[123](index=123&type=chunk)[124](index=124&type=chunk) [Quantitative and Qualitative Disclosures about Market Risk](index=32&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) No material changes to the company's market risks or their management have occurred since December 31, 2021 - As of June 30, 2022, there have been no material changes to the company's market risks since the fiscal year ended December 31, 2021[129](index=129&type=chunk) [Controls and Procedures](index=32&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of June 30, 2022, with no material changes in internal control over financial reporting - An evaluation concluded that the company's disclosure controls and procedures are effective as of the end of the period covered by the report[130](index=130&type=chunk) - No change in internal control over financial reporting occurred during the last fiscal quarter that has materially affected, or is reasonably likely to materially affect, internal controls[131](index=131&type=chunk) PART II. OTHER INFORMATION [Legal Proceedings](index=32&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any material legal proceedings - The company is not currently a party to any material legal proceedings[132](index=132&type=chunk) [Risk Factors](index=32&type=section&id=Item%201A.%20Risk%20Factors) No material changes have occurred to the risk factors previously disclosed in the Annual Report on Form 10-K for FY2021 - No material changes have been made to the risk factors disclosed in the Annual Report on Form 10-K for the fiscal year ended December 31, 2021[133](index=133&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=32&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered sales of equity securities, but withheld **40,812** shares at **$8.15** per share for tax obligations - There were no recent sales of unregistered securities[134](index=134&type=chunk) Issuer Purchases of Equity Securities (Q2 2022) | Period | Total Shares Purchased | Average Price Paid per Share | | :--- | :--- | :--- | | April 2022 | 6,588 | $8.31 | | May 2022 | 23,164 | $7.64 | | June 2022 | 11,060 | $9.13 | | **Total** | **40,812** | **$8.15** | [Defaults Upon Senior Securities](index=33&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reported no defaults upon senior securities - None[136](index=136&type=chunk) [Exhibits](index=34&type=section&id=Item%206.%20Exhibits) The report lists various exhibits filed with the SEC, including CEO and CFO certifications and Inline XBRL documents - Exhibits filed include CEO and CFO certifications pursuant to Rules 13a-14 and 15d-14, and Section 906 of the Sarbanes-Oxley Act[139](index=139&type=chunk)

PART I. [FINANCIAL INFORMATION](index=5&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) This section presents the company's unaudited consolidated financial statements, including balance sheets, statements of operations, cash flows, and related notes, along with management's discussion and analysis [Item 1. Consolidated Financial Statements and Notes (unaudited)](index=5&type=section&id=Item%201.%20Consolidated%20Financial%20Statements%20and%20Notes%20(unaudited)) This section presents Amicus Therapeutics, Inc.'s unaudited consolidated financial statements, detailing financial position, operational results, and cash flows for the period ended March 31, 2022 [Consolidated Balance Sheets](index=5&type=section&id=Consolidated%20Balance%20Sheets) As of March 31, 2022, the company reported total assets of $826.9 million, a decrease from $905.1 million at year-end 2021, primarily due to a reduction in cash, cash equivalents, and marketable securities Consolidated Balance Sheet Highlights (in thousands) | Account | March 31, 2022 | December 31, 2021 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $233,317 | $245,197 | | Investments in marketable securities | $177,878 | $237,299 | | Total current assets | $518,900 | $596,834 | | Total Assets | $826,938 | $905,140 | | **Liabilities & Equity** | | | | Total current liabilities | $129,699 | $145,975 | | Long-term debt | $389,994 | $389,357 | | Total liabilities | $588,321 | $597,771 | | Total stockholders' equity | $238,617 | $307,369 | [Consolidated Statements of Operations](index=6&type=section&id=Consolidated%20Statements%20of%20Operations) For the three months ended March 31, 2022, net product sales grew to $78.7 million from $66.4 million in the prior-year period, while net loss widened to $85.3 million due to increased expenses and an impairment charge Consolidated Statements of Operations Highlights (in thousands, except per share data) | Metric | Q1 2022 | Q1 2021 | | :--- | :--- | :--- | | Net product sales | $78,715 | $66,402 | | Gross profit | $71,133 | $59,863 | | Research and development | $81,517 | $64,117 | | Selling, general, and administrative | $58,116 | $46,726 | | Loss from operations | ($75,339) | ($53,055) | | Net loss | ($85,260) | ($65,664) | | Net loss per share — basic and diluted | ($0.30) | ($0.25) | [Consolidated Statements of Cash Flows](index=9&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) For the first quarter of 2022, net cash used in operating activities was $58.3 million, offset by $58.2 million provided by investing activities, resulting in a net decrease in cash of $11.6 million Consolidated Cash Flow Summary (in thousands) | Activity | Q1 2022 | Q1 2021 | | :--- | :--- | :--- | | Net cash used in operating activities | ($58,315) | ($72,354) | | Net cash provided by investing activities | $58,213 | $86,565 | | Net cash (used in) provided by financing activities | ($8,154) | $8,829 | | Net (decrease) increase in cash | ($11,633) | $21,887 | | Cash at end of period | $237,823 | $188,049 | [Notes to Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) This section details the company's business, accounting policies, and financial statement components, including product pipeline updates and cash sufficiency outlook - The company's lead product is **Galafold®** for **Fabry disease**, and its lead pipeline candidate is **AT-GAA** for **Pompe disease**, which is under review by the **FDA** and **EMA**[29](index=29&type=chunk)[30](index=30&type=chunk) - The **FDA** extended the **PDUFA action dates** for **AT-GAA** components to **August 29, 2022**, and **October 29, 2022**[30](index=30&type=chunk) - The company believes its current cash position, including expected revenues, is **sufficient to fund operations to achieve self-sustainability**[31](index=31&type=chunk) - An **impairment charge** of **$6.6 million** was recognized in **Q1 2022** due to the strategic prioritization of its gene therapy portfolio[46](index=46&type=chunk) Net Product Sales of Galafold® by Geographic Area (in thousands) | Region | Q1 2022 | Q1 2021 | | :--- | :--- | :--- | | U.S. | $24,178 | $20,858 | | Ex-U.S. | $54,537 | $45,544 | | **Total** | **$78,715** | **$66,402** | [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=21&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's financial condition and operational results for Q1 2022, focusing on revenue growth, increased net loss, and liquidity position - **Galafold® revenue** for **Q1 2022** was **$78.7 million**, an **increase of $12.3 million (19%)** compared to **Q1 2021**, driven by growth in the U.S., Europe, and Japan[82](index=82&type=chunk)[94](index=94&type=chunk) - The company's lead pipeline biologic, **AT-GAA** for **Pompe disease**, is under review by the **FDA** and **EMA**, with **revised PDUFA dates** in **August and October 2022**[80](index=80&type=chunk) Comparison of Operating Results (in thousands) | Line Item | Q1 2022 | Q1 2021 | Change | | :--- | :--- | :--- | :--- | | Net product sales | $78,715 | $66,402 | $12,313 | | Research and development | $81,517 | $64,117 | $17,400 | | Selling, general, and administrative | $58,116 | $46,726 | $11,390 | | Loss on impairment of assets | $6,616 | $— | $6,616 | | Net loss | ($85,260) | ($65,664) | ($19,596) | - The **increase in R&D costs** was primarily due to **manufacturing costs for the Pompe program**, **contract exit costs for gene therapy programs**, and **higher personnel costs**[95](index=95&type=chunk) - Total cash, cash equivalents, and marketable securities as of **March 31, 2022**, was **$411.2 million**[85](index=85&type=chunk)[99](index=99&type=chunk) [Item 3. Quantitative and Qualitative Disclosures about Market Risk](index=28&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) The company states that as of March 31, 2022, there have been no material changes to its market risks or the management of such risks since the disclosures made in its Annual Report on Form 10-K for the fiscal year ended December 31, 2021 - There have been **no material changes** to the company's market risks since **December 31, 2021**[116](index=116&type=chunk) [Item 4. Controls and Procedures](index=28&type=section&id=Item%204.%20Controls%20and%20Procedures) Management, including the Principal Executive Officer and Principal Financial Officer, evaluated the effectiveness of the company's disclosure controls and procedures, concluding they were effective with no material changes in internal control over financial reporting - The Principal Executive Officer and Principal Financial Officer concluded that the company's disclosure controls and procedures were **effective** as of the end of the period[117](index=117&type=chunk) - **No material changes** were made to the company's internal control over financial reporting during the quarter[118](index=118&type=chunk) PART II. [OTHER INFORMATION](index=28&type=section&id=PART%20II.%20OTHER%20INFORMATION) This section provides additional disclosures, including legal proceedings, risk factors, equity security sales, and a list of exhibits filed with the report [Item 1. Legal Proceedings](index=28&type=section&id=Item%201.%20Legal%20Proceedings) The company reports that it is not currently a party to any material legal proceedings - The company is **not currently involved in any material legal proceedings**[119](index=119&type=chunk) [Item 1A. Risk Factors](index=28&type=section&id=Item%201A.%20Risk%20Factors) The company states there have been no material changes to the risk factors that were previously disclosed in its Annual Report on Form 10-K for the fiscal year ended December 31, 2021 - There have been **no material changes** to the risk factors disclosed in the company's **2021 Form 10-K**[120](index=120&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=28&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered sales of equity securities during the period and detailed issuer purchases of equity securities for tax obligations related to vested restricted stock units - There were **no sales of unregistered securities** in the quarter[121](index=121&type=chunk) - The company repurchased **797,911 shares** of common stock at an average price of **$11.38 per share** to satisfy taxes related to vested restricted stock units[122](index=122&type=chunk) [Item 3. Defaults Upon Senior Securities](index=29&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reported no defaults upon senior securities - None[123](index=123&type=chunk) [Item 4. Mine Safety Disclosures](index=29&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) The company reported no mine safety disclosures - None[124](index=124&type=chunk) [Item 5. Other Information](index=29&type=section&id=Item%205.%20Other%20Information) The company reported no other information - None[125](index=125&type=chunk) [Item 6. Exhibits](index=30&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the Form 10-Q, including a termination agreement, an employment agreement, officer certifications, and interactive data files (XBRL) - Exhibits filed include a **termination agreement**, an **employment agreement** for John F. Crowley, and **certifications** by the Principal Executive Officer and Principal Financial Officer[127](index=127&type=chunk) [Signatures](index=31&type=section&id=SIGNATURES) This section contains the required signatures, affirming the accuracy and completeness of the filed report