Amicus Therapeutics, Inc. (FOLD) reported fourth-quarter 2023 adjusted earnings of 1 cent per share against the Zacks Consensus Estimate of a loss of 6 cents. The company had reported a loss of 17 cents per share in the year-ago quarter.The year-over-year improvement can be attributed to higher revenues of Galafold (migalastat) and the successful ongoing commercial launch of the newly approved Pombiliti + Opfolda.Revenues in the fourth quarter totaled $115.1 million, up 31% year over year on a reported basi ...

Olivier Le Moal Shares of Amicus Therapeutics (NASDAQ:FOLD) are up modestly since my September 2023 update where I argued that the limited label of Pombiliti+Opfolda may not matter for the product combination's uptake because it would have been used like the label says anyway. And while there are no real pipeline updates to look forward to, the company may attract a different kind of investor base going forward - investors predominantly focused on revenue and earnings momentum. Amicus should start deliverin ...

This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, manufacturing and supply plans, financing plans, and the projected revenues and cash position for the Company ...

Amicus Therapeutics (FOLD) came out with a quarterly loss of $0.11 per share versus the Zacks Consensus Estimate of a loss of $0.06. This compares to loss of $0.19 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -83.33%. A quarter ago, it was expected that this pharmaceutical company would post a loss of $0.08 per share when it actually produced a loss of $0.07, delivering a surprise of 12.50%.Over the last four quarters, the ...

2023 Total Revenue of $399.4M, a 21% Increase Year-over-Year Strong Patient Demand Continues for Pombiliti™ + Opfolda™ in the U.S., U.K., and Germany Projecting 2024 Galafold® Revenue Growth of 11-16% at CER Anticipating Full-Year Non-GAAP Profitability in 2024 Conference Call and Webcast Today at 8:30 a.m. ET PRINCETON, N.J., Feb. 28, 2024 (GLOBE NEWSWIRE) -- Amicus Therapeutics (NASDAQ:FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rar ...

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, 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 Jurisdicti ...

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 ...