Amicus Therapeutics, Inc. (NASDAQ:FOLD) Q3 2021 Earnings Conference Call November 9, 2021 8:30 AM ET Company Participants Andrew Faughnan – Head-Investor Relations John Crowley – Chairman and Chief Executive Officer Bradley Campbell – President and Chief Operating Officer Jeff Castelli – Chief Development Officer Daphne Quimi – Chief Financial Officer Conference Call Participants Anvita Gupta – Cowen Anupam Rama – JPMorgan Joseph Schwartz – SVB Leerink Dae Gon Ha – Stifel Yun Zhong – BTIG Nishant Gandhi – N ...

PART I. FINANCIAL INFORMATION [Item 1. Consolidated Financial Statements and Notes (unaudited)](index=5&type=section&id=Item%201.%20Consolidated%20Financial%20Statements%20and%20Notes%20(unaudited)) Financial statements reflect Galafold-driven revenue growth, operating losses, and strengthened cash from recent private placement [Consolidated Balance Sheets](index=5&type=section&id=Consolidated%20Balance%20Sheets) Consolidated Balance Sheet Highlights (in thousands) | Account | Sep 30, 2021 | Dec 31, 2020 | | :--- | :--- | :--- | | Cash and cash equivalents | $385,903 | $163,240 | | Investments in marketable securities | $171,057 | $320,029 | | **Total Assets** | **$956,675** | **$886,520** | | Long-term debt | $388,719 | $389,254 | | **Total Liabilities** | **$580,046** | **$600,140** | | **Total Stockholders' Equity** | **$376,629** | **$286,380** | [Consolidated Statements of Operations](index=6&type=section&id=Consolidated%20Statements%20of%20Operations) Statement of Operations Summary (in thousands, except per share data) | Metric | Q3 2021 | Q3 2020 | YTD 2021 | YTD 2020 | | :--- | :--- | :--- | :--- | :--- | | Net product sales | $79,545 | $67,437 | $223,360 | $190,315 | | Gross profit | $67,849 | $59,038 | $196,745 | $168,688 | | Research and development | $59,333 | $70,419 | $186,453 | $229,150 | | Selling, general, and administrative | $46,107 | $37,850 | $135,109 | $112,722 | | Loss from operations | $(42,399) | $(52,761) | $(134,288) | $(182,163) | | **Net loss** | **$(50,294)** | **$(64,011)** | **$(167,183)** | **$(205,451)** | | Net loss per share | $(0.19) | $(0.25) | $(0.63) | $(0.80) | [Consolidated Statements of Cash Flows](index=10&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) Cash Flow Summary for Nine Months Ended Sep 30 (in thousands) | Activity | 2021 | 2020 | | :--- | :--- | :--- | | Net cash used in operating activities | $(131,977) | $(183,468) | | Net cash provided by investing activities | $146,850 | $9,197 | | Net cash provided by financing activities | $212,177 | $241,310 | | **Net increase in cash** | **$222,909** | **$67,084** | [Notes to Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) Notes detail business, lead products Galafold and AT-GAA, planned gene therapy spin-off, and financial policies - The company's lead product is **Galafold** for Fabry disease, and its lead biologic program is **AT-GAA** for Pompe disease, with PDUFA target action dates of **May 29, 2022 (NDA)** and **July 29, 2022 (BLA)**[35](index=35&type=chunk) - In September 2021, the company announced its intent to spin off its gene therapy business into a new company, **Caritas Therapeutics**, via a SPAC business combination, with Amicus becoming the largest stockholder with an approximate **36% ownership stake**[37](index=37&type=chunk)[42](index=42&type=chunk) - In September 2021, the company raised approximately **$199.8 million** in net proceeds from a private placement of common stock and pre-funded warrants[44](index=44&type=chunk)[78](index=78&type=chunk) - The remaining **$2.8 million** principal of Convertible Notes due 2023 were exchanged for approximately 472,356 shares of common stock during Q1 and Q3 2021, resulting in a net loss from extinguishment of debt of **$0.3 million**[73](index=73&type=chunk) - A regulatory milestone was reached in September 2021 related to the FDA's acceptance of the BLA submission for the ATB-200 Pompe disease program, triggering a **$6.0 million** milestone payment[93](index=93&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=24&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses Galafold performance, AT-GAA progress, gene therapy spin-off, and sufficient cash for self-sustainability [Overview and Strategy](index=24&type=section&id=Overview%20and%20Strategy) - The company's strategy focuses on developing first- or best-in-class medicines for rare metabolic diseases, particularly Fabry (Galafold) and Pompe (AT-GAA) diseases[108](index=108&type=chunk) - A key strategic move is the planned launch of **Caritas Therapeutics** through a SPAC transaction to house the Amicus gene therapy business, with Amicus retaining a **~36% stake**[101](index=101&type=chunk)[102](index=102&type=chunk)[106](index=106&type=chunk) - The company believes its cash position of **$557.0 million** as of September 30, 2021, is sufficient to fund operations to achieve self-sustainability[111](index=111&type=chunk) [Commercial Product and Product Candidates](index=26&type=section&id=Commercial%20Product%20and%20Product%20Candidates) - **Galafold (Fabry Disease):** This oral precision medicine is approved in over 40 countries, including E.U. approval for adolescents aged 12 and older in July 2021[111](index=111&type=chunk) - **AT-GAA (Pompe Disease):** The Phase 3 PROPEL study's primary endpoint (6-minute walk distance) was numerically greater for AT-GAA but did not achieve statistical superiority (p=0.072), though a key secondary endpoint, Forced Vital Capacity (FVC), showed a statistically significant and clinically meaningful benefit (p=0.023)[114](index=114&type=chunk)[115](index=115&type=chunk) - **Gene Therapy Pipeline:** The company is advancing multiple gene therapy programs for Fabry, Pompe, and various Batten disease types (CLN6, CLN3, CLN1), which are part of the planned spin-off into Caritas Therapeutics[112](index=112&type=chunk)[118](index=118&type=chunk)[122](index=122&type=chunk) [Consolidated Results of Operations](index=30&type=section&id=Consolidated%20Results%20of%20Operations) Comparison of Three Months Ended September 30 (in thousands) | Account | 2021 | 2020 | Change | | :--- | :--- | :--- | :--- | | Net product sales | $79,545 | $67,437 | $12,108 | | Research and development | $59,333 | $70,419 | $(11,086) | | Selling, general, and administrative | $46,107 | $37,850 | $8,257 | | **Net loss** | **$(50,294)** | **$(64,011)** | **$13,717** | Comparison of Nine Months Ended September 30 (in thousands) | Account | 2021 | 2020 | Change | | :--- | :--- | :--- | :--- | | Net product sales | $223,360 | $190,315 | $33,045 | | Research and development | $186,453 | $229,150 | $(42,697) | | Selling, general, and administrative | $135,109 | $112,722 | $22,387 | | **Net loss** | **$(167,183)** | **$(205,451)** | **$38,268** | - The decrease in R&D expense for the nine months ended Sep 30, 2021, was primarily due to the timing of clinical research and manufacturing costs for the Pompe and gene therapy programs, and decreased personnel costs from strategic realignment[137](index=137&type=chunk) [Liquidity and Capital Resources](index=32&type=section&id=Liquidity%20and%20Capital%20Resources) - As of September 30, 2021, the company had cash, cash equivalents, and marketable securities totaling **$557.0 million**[141](index=141&type=chunk) - Net cash provided by financing activities for the nine months ended Sep 30, 2021, was **$212.2 million**, primarily reflecting **$199.8 million** in net proceeds from the September 2021 private placement[146](index=146&type=chunk) - The company believes its current cash position, including expected revenues and proceeds from the recent private placement, is sufficient to fund operations and research programs to achieve self-sustainability[150](index=150&type=chunk) [Item 3. Quantitative and Qualitative Disclosures about Market Risk](index=36&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) The company's primary market risks relate to interest rates on investments and debt, and foreign currency exchange rates - The company is exposed to interest rate risk on its **$400 million** Senior Secured Term Loan, which bears interest at 3-month LIBOR (with a 1% floor) plus 6.5%, where a hypothetical 100 basis point change would result in a **$1.0 million** change in annual interest expense[159](index=159&type=chunk) - The company faces foreign exchange risk from international operations but believes a hypothetical 10% change in exchange rates would not materially impact its consolidated financial statements[161](index=161&type=chunk) [Item 4. Controls and Procedures](index=36&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded the company's disclosure controls and procedures were effective, with no material changes to internal control over financial reporting - Based on an evaluation as of September 30, 2021, the Principal Executive Officer and Principal Financial Officer concluded that the company's disclosure controls and procedures are effective[163](index=163&type=chunk) PART II. OTHER INFORMATION [Item 1. Legal Proceedings](index=37&type=section&id=Item%201.%20Legal%20Proceedings) A complaint filed by Teva Pharmaceuticals Development, Inc. against the company was dismissed with prejudice on September 2, 2021 - A complaint filed by Teva Pharmaceuticals Development, Inc. was dismissed with prejudice on September 2, 2021[165](index=165&type=chunk) [Item 1A. Risk Factors](index=37&type=page&id=Item%201A.%20Risk%20Factors) A new risk factor highlights that the proposed gene therapy spin-off may not be completed or achieve anticipated benefits - A new risk factor highlights that the proposed spin-off of the gene therapy business into **Caritas Therapeutics** may not be completed in the expected timeframe, or at all, and may not achieve the expected strategic or financial benefits[167](index=167&type=chunk)[169](index=169&type=chunk) - The spin-off is subject to customary conditions, including approval by ARYA's shareholders and the SEC declaring the Form S-4 registration statement effective[168](index=168&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=37&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered sales of equity securities but repurchased shares to satisfy tax obligations from restricted stock awards - There were no unregistered sales of equity securities in the period[171](index=171&type=chunk) Issuer Purchases of Equity Securities (Q3 2021) | Period | Total Shares Purchased | Average Price Paid per Share | | :--- | :--- | :--- | | July 2021 | 3,130 | $9.18 | | August 2021 | 5,826 | $10.58 | | September 2021 | 4,901 | $11.14 | | **Total** | **13,857** | **$10.46** | *Represents shares withheld to satisfy taxes associated with vesting of restricted stock awards* [Item 6. Exhibits](index=39&type=section&id=Item%206.%20Exhibits) This section lists key exhibits filed, including agreements for the Caritas Therapeutics transaction and recent private placement - Exhibits filed include the Business Combination Agreement with ARYA Sciences Acquisition Corp IV, and Securities Purchase Agreements with Redmile Group and Perceptive Life Sciences Master Fund, among others, for the recent private placement[177](index=177&type=chunk)

Amicus Therapeutics, Inc. (NASDAQ:FOLD) Formation of Caritas Therapeutics Conference Call September 29, 2021 8:00 AM ET Company Participants Andrew Faughnan - Executive Director, IR John F. Crowley - Chairman and CEO Bradley L. Campbell - President and COO Daphne Quimi - CFO Conference Call Participants Ritu Baral - Cowen Tazeen Ahmad - Bank of America Merrill Lynch Eliana Merle - UBS Investment Bank Anupam Rama - JP Morgan Elizabeth Webster - Goldman Sachs Joseph Schwartz - SVB Leerink Dae Gon Ha - Stifel, ...

Transaction Overview - Amicus is launching Caritas Therapeutics as a next-generation genetic medicine company through a business combination with ARYA IV, a special purpose acquisition company (SPAC)[9, 19] - Caritas will launch with approximately $400 million in capital through SPAC, PIPE (Private Investment in Public Equity), and Amicus funding[4, 9, 10, 19] - Amicus will retain an approximate 36% pro forma ownership in Caritas[9, 10, 19] Leadership and Strategy - John Crowley will lead Caritas as Chairman and CEO, while remaining with Amicus as Chairman Emeritus and Chief Strategic Advisor[4, 17] - Bradley Campbell will succeed John Crowley as Amicus Chief Executive Officer upon transaction closing[4, 18] - The transaction aims to enhance the ability of both Amicus and Caritas to address unmet needs of patients with rare diseases and accelerate the development of the gene therapy portfolio[11, 19] Financial Impact - Amicus expects to become profitable as of 2023[4, 13] - Amicus is further funded through a completed $200 million private investment[4] - Two lead programs have potential $500 million - $1 billion peak sales opportunity[5, 13] Product and Pipeline - Amicus' AT-GAA NDA/BLA accepted for review with PDUFA action dates of May 29, 2022, and July 29, 2022[4] - Amicus' FY21 Revenue Guidance is $300M-$315M[6]

Amicus Therapeutics, Inc. (NASDAQ:FOLD) Q2 2021 Earnings Conference Call August 5, 2021 8:00 AM ET Company Participants Andrew Faughnan - Head, Investor Relations John Crowley - Chairman and Chief Executive Officer Bradley Campbell - President and Chief Operating Officer Daphne Quimi - Chief Financial Officer Jeff Castelli - Chief Development Officer Mitchell Goldman - Chief Medical Officer Conference Call Participants Ritu Baral - Cowen Ellie Merle - UBS Kristen Kluska - Cantor Fitzgerald Anupam Rama - JPM ...

[PART I. FINANCIAL INFORMATION](index=3&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) This section provides the company's unaudited consolidated financial information for the specified periods [Item 1. Consolidated Financial Statements and Notes (unaudited)](index=3&type=section&id=Item%201.%20Consolidated%20Financial%20Statements%20and%20Notes%20(unaudited)) This section presents the unaudited consolidated financial statements of Amicus Therapeutics, Inc. for the periods ended June 30, 2021, and December 31, 2020, including balance sheets, statements of operations, comprehensive loss, changes in stockholders' equity, and cash flows, along with detailed notes explaining significant accounting policies and specific financial line items [Consolidated Balance Sheets](index=5&type=section&id=Consolidated%20Balance%20Sheets) This section presents the company's financial position, including assets, liabilities, and equity, at specific dates | Metric | Dec 31, 2020 (in thousands) | Jun 30, 2021 (in thousands) | Change (in thousands) | | :-------------------------------- | :-------------------------- | :-------------------------- | :-------------------- | | Total Assets | $886,520 | $786,892 | $(99,628) | | Cash and cash equivalents | $163,240 | $176,538 | $13,298 | | Investments in marketable securities | $320,029 | $206,530 | $(113,499) | | Total Liabilities | $600,140 | $572,982 | $(27,158) | | Total Stockholders' Equity | $286,380 | $213,910 | $(72,470) | [Consolidated Statements of Operations](index=6&type=section&id=Consolidated%20Statements%20of%20Operations) This section details the company's revenues, expenses, and net loss over specific reporting periods | Metric (in thousands) | Q2 2021 | Q2 2020 | YoY Change | YoY % Change | | :-------------------------------- | :------ | :------ | :--------- | :----------- | | Net product sales | $77,413 | $62,353 | $15,060 | 24.2% | | Gross profit | $69,033 | $55,677 | $13,356 | 24.0% | | Research and development | $63,003 | $69,611 | $(6,608) | -9.5% | | Selling, general, and administrative | $42,276 | $34,657 | $7,619 | 22.0% | | Loss from operations | $(38,834) | $(51,345) | $12,511 | -24.4% | | Net loss attributable to common stockholders | $(51,225) | $(52,492) | $1,267 | -2.4% | | Net loss per common share (basic and diluted) | $(0.19) | $(0.20) | $0.01 | -5.0% | | Metric (in thousands) | H1 2021 | H1 2020 | YoY Change | YoY % Change | | :-------------------------------- | :------ | :------ | :--------- | :----------- | | Net product sales | $143,815 | $122,878 | $20,937 | 17.0% | | Gross profit | $128,896 | $109,650 | $19,246 | 17.6% | | Research and development | $127,120 | $158,731 | $(31,611) | -19.9% | | Selling, general, and administrative | $89,002 | $74,872 | $14,130 | 18.9% | | Loss from operations | $(91,889) | $(129,402) | $37,513 | -29.0% | | Net loss attributable to common stockholders | $(116,889) | $(141,440) | $24,551 | -17.4% | | Net loss per common share (basic and diluted) | $(0.44) | $(0.55) | $0.11 | -20.0% | [Consolidated Statements of Comprehensive Loss](index=7&type=section&id=Consolidated%20Statements%20of%20Comprehensive%20Loss) This section reports the company's net loss and other comprehensive income (loss) components | Metric (in thousands) | Q2 2021 | Q2 2020 | YoY Change | | :-------------------------------- | :------ | :------ | :--------- | | Net loss | $(51,225) | $(52,492) | $1,267 | | Foreign currency translation adjustment gain (loss) | $235 | $(2,116) | $2,351 | | Unrealized gain on available-for-sale securities | $12 | $457 | $(445) | | Comprehensive loss | $(50,978) | $(54,151) | $3,173 | | Metric (in thousands) | H1 2021 | H1 2020 | YoY Change | | :-------------------------------- | :------ | :------ | :--------- | | Net loss | $(116,889) | $(141,440) | $24,551 | | Foreign currency translation adjustment gain (loss) | $843 | $2,080 | $(1,237) | | Unrealized gain on available-for-sale securities | $12 | $248 | $(236) | | Comprehensive loss | $(116,034) | $(139,112) | $23,078 | [Consolidated Statements of Changes in Stockholders' Equity](index=8&type=section&id=Consolidated%20Statements%20of%20Changes%20in%20Stockholders'%20Equity) This section outlines changes in the company's equity accounts, including capital and accumulated deficit | Metric (in thousands) | Dec 31, 2020 | Jun 30, 2021 | Change | | :-------------------------------- | :----------- | :----------- | :------- | | Total Stockholders' Equity | $286,380 | $213,910 | $(72,470) | | Additional Paid-In Capital | $2,308,578 | $2,364,494 | $55,916 | | Accumulated Deficit | $(2,045,462) | $(2,162,351) | $(116,889) | | Warrants | $12,387 | $0 | $(12,387) | - Warrants exercised generated **$19.23 million** in proceeds and eliminated the **$12.39 million** warrant liability[26](index=26&type=chunk)[69](index=69&type=chunk) [Consolidated Statements of Cash Flows](index=10&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) This section summarizes the company's cash inflows and outflows from operating, investing, and financing activities | Metric (in thousands) | H1 2021 | H1 2020 | YoY Change | | :-------------------------------- | :------ | :------ | :--------- | | Net cash used in operating activities | $(108,277) | $(141,460) | $33,183 | | Net cash provided by investing activities | $112,278 | $163,258 | $(50,980) | | Net cash provided by financing activities | $11,064 | $2,599 | $8,465 | | Net increase in cash, cash equivalents, and restricted cash | $13,626 | $20,921 | $(7,295) | | Cash, cash equivalents, and restricted cash at end of period | $179,788 | $167,262 | $12,526 | [Notes to Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) This section provides detailed explanations of the company's significant accounting policies and financial statement line items [Note 1. Description of Business](index=11&type=section&id=Note%201.%20Description%20of%20Business) Amicus Therapeutics is a global biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases, with a portfolio including Galafold for Fabry disease, AT-GAA for Pompe disease, and an industry-leading gene therapy pipeline. The company anticipates incurring losses but believes its current cash position and expected revenues are sufficient to achieve self-sustainability - Amicus Therapeutics is a global biotechnology company specializing in novel medicines for rare diseases, with a focus on Fabry disease (Galafold), Pompe disease (AT-GAA), and a growing gene therapy portfolio[35](index=35&type=chunk)[36](index=36&type=chunk)[37](index=37&type=chunk) - The company expects to incur losses through 2021 and beyond, with an accumulated deficit of **$2.2 billion** as of June 30, 2021, but believes current cash and expected revenues are sufficient for self-sustainability[38](index=38&type=chunk) [Note 2. Summary of Significant Accounting Policies](index=12&type=section&id=Note%202.%20Summary%20of%20Significant%20Accounting%20Policies) This note outlines the basis of presentation for the unaudited consolidated financial statements, prepared in accordance with U.S. GAAP for interim financial information. It details policies on consolidation, foreign currency transactions, use of estimates (including COVID-19 impact assessment), and the classification of cash, cash equivalents, marketable securities, and restricted cash. The company adopted ASU 2019-12 on January 1, 2021, with no material impact - Financial statements are prepared in accordance with U.S. GAAP for interim reporting, with all necessary recurring adjustments[39](index=39&type=chunk) - The company assesses COVID-19 impact on operations and financial results, noting no significant disruptions to date but acknowledging potential future negative impacts[38](index=38&type=chunk)[44](index=44&type=chunk) - Cash equivalents are highly liquid investments with maturities of three months or less; marketable securities are available-for-sale fixed income investments with maturities greater than three months, reported at fair value[45](index=45&type=chunk) [Note 3. Cash, Cash Equivalents, Marketable Securities, and Restricted Cash](index=14&type=section&id=Note%203.%20Cash,%20Cash%20Equivalents,%20Marketable%20Securities,%20and%20Restricted%20Cash) As of June 30, 2021, the company held **$176.5 million** in cash and cash equivalents and **$206.5 million** in marketable securities, totaling **$383.1 million**. These investments are primarily in high-quality commercial financial instruments, with unrealized losses considered temporary. Restricted cash amounted to **$3.25 million** | Asset (in thousands) | Jun 30, 2021 | Dec 31, 2020 | | :-------------------------------- | :----------- | :----------- | | Cash and cash equivalents | $176,538 | $163,240 | | Marketable securities | $206,530 | $320,029 | | Total cash, cash equivalents, and marketable securities | $383,068 | $483,269 | | Restricted cash | $3,250 | N/A (not explicitly stated for Dec 31, 2020 in this table) | - Unrealized loss positions in marketable securities (**$17.2 million** as of June 30, 2021) are considered temporary impairments, not credit losses, and are recognized in other comprehensive gain (loss)[62](index=62&type=chunk) [Note 4. Inventories](index=16&type=section&id=Note%204.%20Inventories) Inventories, consisting of raw materials, work-in-process, and finished goods for Galafold, increased to **$24.1 million** at June 30, 2021, from **$19.6 million** at December 31, 2020. The company maintains a reserve for inventory | Inventory Component (in thousands) | Jun 30, 2021 | Dec 31, 2020 | | :--------------------------------- | :----------- | :----------- | | Raw materials | $7,619 | $5,547 | | Work-in-process | $11,306 | $7,693 | | Finished goods | $5,161 | $6,316 | | Total inventories | $24,086 | $19,556 | [Note 5. Debt](index=16&type=section&id=Note%205.%20Debt) The company's long-term debt primarily consists of a **$400 million** Senior Secured Term Loan due 2026 and Convertible Notes. The net carrying value of long-term debt increased slightly to **$390.4 million** at June 30, 2021, from **$389.3 million** at December 31, 2020. Interest expense significantly increased due to the Senior Secured Term Loan | Debt Component (in thousands) | Jun 30, 2021 | Dec 31, 2020 | | :-------------------------------- | :----------- | :----------- | | Net carrying value of Senior Secured Term Loan | $388,095 | $386,970 | | Net carrying value of Convertible Notes | $2,339 | $2,284 | | Total Long-term debt | $390,434 | $389,254 | | Interest Expense (in thousands) | Q2 2021 | Q2 2020 | H1 2021 | H1 2020 | | :------------------------------ | :------ | :------ | :------ | :------ | | Contractual interest expense | $7,604 | $3,410 | $15,125 | $6,990 | | Amortization of debt discount | $383 | $268 | $720 | $470 | | Amortization of deferred financing | $262 | $33 | $485 | $50 | [Note 6. Stockholders' Equity](index=17&type=section&id=Note%206.%20Stockholders'%20Equity) During the first quarter of 2021, the company received **$19.2 million** in gross cash proceeds from the exercise of 2,554,999 warrants - Warrants exercised in Q1 2021 generated **$19.2 million** in gross cash proceeds[69](index=69&type=chunk) [Note 7. Share-Based Compensation](index=17&type=section&id=Note%207.%20Share-Based%20Compensation) The company's share-based compensation plan includes stock options and restricted stock units (RSUs). Total unrecognized compensation cost for non-vested stock options was **$36.0 million** (expected over 3 years), and for unvested RSUs was **$57.9 million** (expected over 2 years) as of June 30, 2021. Total equity compensation expense for the six months ended June 30, 2021, was **$32.09 million**, up from **$21.00 million** in the prior year | Metric (in thousands) | H1 2021 | H1 2020 | YoY Change | | :-------------------------------- | :------ | :------ | :--------- | | Total equity compensation expense | $32,090 | $21,004 | $11,086 | - Unrecognized compensation cost for non-vested stock options was **$36.0 million** (weighted average period of 3 years) and for unvested RSUs was **$57.9 million** (weighted average period of 2 years) as of June 30, 2021[73](index=73&type=chunk)[74](index=74&type=chunk) [Note 8. Assets and Liabilities Measured at Fair Value](index=19&type=section&id=Note%208.%20Assets%20and%20Liabilities%20Measured%20at%20Fair%20Value) The company classifies its financial assets and liabilities within a fair value hierarchy (Level 1, 2, or 3). Cash is Level 1, marketable securities and debt are Level 2, and contingent consideration payable is Level 3. The fair value of contingent consideration payable increased to **$27.3 million** at June 30, 2021, from **$25.8 million** at December 31, 2020, with **$19.8 million** classified as current | Asset/Liability (in thousands) | Fair Value (Jun 30, 2021) | Fair Value (Dec 31, 2020) | Fair Value Hierarchy Level | | :-------------------------------- | :------------------------ | :------------------------ | :------------------------- | | Commercial paper | $147,339 | $217,095 | Level 2 | | Asset-backed securities | $20,680 | $9,438 | Level 2 | | Corporate debt securities | $12,039 | $39,513 | Level 2 | | U.S. government agency bonds | $26,071 | $53,582 | Level 2 | | Money market funds | $4,582 | $4,427 | Level 2 | | Contingent consideration payable | $27,317 | $25,825 | Level 3 | | Deferred compensation plan liability | $4,233 | $4,078 | Level 2 | - Contingent consideration payable, a Level 3 liability, increased to **$27.3 million** at June 30, 2021, with **$19.8 million** expected to be paid within the next twelve months[83](index=83&type=chunk)[85](index=85&type=chunk) [Note 9. Basic and Diluted Net Loss per Common Share](index=21&type=section&id=Note%209.%20Basic%20and%20Diluted%20Net%20Loss%20per%20Common%20Share) The basic and diluted net loss per common share for the three months ended June 30, 2021, was **$(0.19)**, an improvement from **$(0.20)** in the prior year. For the six months, it improved to **$(0.44)** from **$(0.55)**. Potentially dilutive common stock equivalents were excluded from the diluted EPS calculation due to their anti-dilutive effect | Metric | Q2 2021 | Q2 2020 | H1 2021 | H1 2020 | | :-------------------------------- | :------ | :------ | :------ | :------ | | Net loss per common share (basic and diluted) | $(0.19) | $(0.20) | $(0.44) | $(0.55) | | Weighted average common shares outstanding | 266,398,516 | 257,973,329 | 265,384,865 | 257,548,623 | - Potentially dilutive common stock equivalents (options, convertible notes, warrants, RSUs) totaling **22.8 million shares** as of June 30, 2021, were excluded from diluted EPS calculation due to their anti-dilutive effect[87](index=87&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=22&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section provides an overview of the company's business, strategy, and recent progress, including commercial and clinical milestones. It details the consolidated results of operations for the three and six months ended June 30, 2021, compared to the prior year, and discusses the company's liquidity, capital resources, funding requirements, and financial uncertainties related to future payments [Overview and Strategy](index=22&type=section&id=Overview%20and%20Strategy) This section outlines the company's business, strategic objectives, and operational focus - Amicus Therapeutics is a global biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases, including Fabry disease (Galafold), Pompe disease (AT-GAA), and a gene therapy portfolio[89](index=89&type=chunk)[90](index=90&type=chunk)[91](index=91&type=chunk) - The company's strategy is to create first- or best-in-class medicines for rare metabolic diseases through internal development, acquisition, or in-licensing, with a focus on expanding its genomic medicine pipeline[92](index=92&type=chunk) - COVID-19 has not significantly impacted operations, but the company observes increased lag times for Galafold initiation and acknowledges potential future negative impacts on results[93](index=93&type=chunk) [Highlights of Progress](index=23&type=section&id=Highlights%20of%20Progress) This section summarizes key commercial, clinical, and financial achievements during the reporting period - Galafold revenue for the six months ended June 30, 2021, totaled **$77.4 million**, an increase of **$15.1 million** year-over-year, driven by growth in the U.S., Europe, and Japan markets[94](index=94&type=chunk) - Completed last patient, last visit in the global Phase 3 PROPEL study for AT-GAA in December 2020, reporting topline results in February 2021[94](index=94&type=chunk) - AT-GAA received Breakthrough Therapy Designation (FDA), Orphan Drug Designation (Japan), Promising Innovative Medicine (MHRA), and a positive scientific opinion through EAMS (MHRA)[94](index=94&type=chunk) - Presented initial clinical data from Phase 1/2 CLN3 gene therapy study suggesting early signs of disease stabilization and preclinical data for Fabry disease gene therapy candidate (AT-GTX-701) showing improved substrate reduction[94](index=94&type=chunk) - Maintained uninterrupted global supply chains for Galafold and clinical products during the COVID-19 pandemic[94](index=94&type=chunk) - Total cash, cash equivalents, and marketable securities were **$383.1 million** as of June 30, 2021, believed to be sufficient for self-sustainability based on current operating models[94](index=94&type=chunk) [Product Pipeline Details](index=24&type=section&id=Product%20Pipeline%20Details) This section provides detailed updates on the company's various product development programs and clinical trials [Gene Therapy for Fabry Disease](index=24&type=section&id=Gene%20Therapy%20for%20Fabry%20Disease) Amicus is developing a Fabry gene therapy (AT-GTX-701) for patients with non-amenable variants, leveraging a collaboration with Penn. Preclinical data showed the engineered hGLA AAV gene therapy (AT-GTX-701) demonstrated enhanced stability and significantly greater substrate reduction across Fabry disease-relevant tissues compared to wild-type constructs - Developing Fabry gene therapy (AT-GTX-701) for patients with non-amenable variants, showing enhanced stability and superior substrate reduction in preclinical studies[95](index=95&type=chunk) [Novel ERT for Pompe Disease](index=24&type=section&id=Novel%20ERT%20for%20Pompe%20Disease) The AT-GAA program for Pompe disease, consisting of cipaglucosidase alfa and miglustat, completed its Phase 3 PROPEL study. While the primary endpoint (6-minute walk distance in the combined population) did not achieve statistical significance for superiority, AT-GAA showed nominally statistically significant and clinically meaningful superiority in slowing respiratory decline (FVC) and improvements in strength, coordination, and mobility (GSGC) compared to alglucosidase alfa. Switch patients showed significant improvements in 6-minute walk distance and stabilized respiratory function - AT-GAA is a novel ERT for Pompe disease, combining cipaglucosidase alfa (engineered enzyme) with miglustat (enzyme stabilizer)[96](index=96&type=chunk) - Phase 3 PROPEL study for AT-GAA completed, showing that while the primary endpoint (6-minute walk distance in combined population) did not achieve statistical significance for superiority (p=0.072), AT-GAA demonstrated nominally statistically significant superiority in slowing respiratory decline (FVC, p=0.023) and improvements in GSGC (p<0.05)[96](index=96&type=chunk) - In switch patients, AT-GAA treated patients walked **16.9 meters** farther than baseline (vs. **0.0 meters** for alglucosidase alfa, p=0.046) and showed a **0.1%** absolute increase in FVC (vs. **4.0%** decline for alglucosidase alfa, p=0.006)[97](index=97&type=chunk)[98](index=98&type=chunk) [Gene Therapy for Pompe Disease](index=25&type=section&id=Gene%20Therapy%20for%20Pompe%20Disease) Amicus is advancing a next-generation gene therapy for Pompe disease through a collaboration with Penn. Preclinical data for engineered hGAA AAV gene therapy demonstrated more robust and consistent glycogen reduction in key tissues, including neuronal cells, compared to unmodified hGAA AAV gene therapy - Advancing a next-generation Pompe gene therapy; preclinical data shows engineered hGAA AAV gene therapy achieved more robust and consistent glycogen reduction in key tissues, including neuronal cells[101](index=101&type=chunk)[102](index=102&type=chunk) [Gene Therapy for Various Types of Batten Disease](index=25&type=section&id=Gene%20Therapy%20for%20Various%20Types%20of%20Batten%20Disease) The company has two clinical programs (CLN6 and CLN3) and several preclinical programs for Batten disease. The CLN6 Phase 1/2 study showed AT-GTX-501 was generally well tolerated and demonstrated a meaningful effect in slowing disease progression in Hamburg Motor and Language scores. The CLN3 Phase 1/2 study for AT-GTX-502 showed initial safety and potential early signs of disease stabilization - CLN6 Batten disease AAV-CLN6 gene therapy (AT-GTX-501) Phase 1/2 study showed general tolerability and meaningful slowing of disease progression in motor and language scores[106](index=106&type=chunk) - CLN3 Batten disease AAV serotype AT-GTX-502 gene therapy Phase 1/2 study demonstrated initial safety and potential early signs of disease stabilization[107](index=107&type=chunk) [CDKL5 Deficiency Disorder](index=26&type=section&id=CDKL5%20Deficiency%20Disorder) Amicus is researching a potential first-in-class genetic medicine for CDKL5 Deficiency Disorder (CDD), involving an engineered CDKL5 protein delivered as protein replacement or gene therapy, to address severe neurological impairment caused by CDKL5 protein deficiency - Researching a first-in-class genetic medicine for CDKL5 Deficiency Disorder (CDD) using an engineered CDKL5 protein for protein replacement or gene therapy[108](index=108&type=chunk) [Other Preclinical Gene Therapies](index=26&type=section&id=Other%20Preclinical%20Gene%20Therapies) The company has active preclinical development programs for other gene therapies, including MPS IIIB and a next-generation MPS IIIA program, aiming to develop first- or best-in-class AAV gene therapies for these rare pediatric neurological lysosomal storage diseases - Active preclinical gene therapy programs include MPS IIIB and next-generation MPS IIIA, targeting rare pediatric neurological lysosomal storage diseases[109](index=109&type=chunk) [Strategic Alliances and Arrangements](index=26&type=section&id=Strategic%20Alliances%20and%20Arrangements) This section discusses the company's approach to business development and external collaborations - Continuously evaluates business development opportunities (collaborations, partnerships, out-licensing, acquisitions) to build stockholder value and access resources for rare and orphan disease therapies[110](index=110&type=chunk) [Consolidated Results of Operations](index=27&type=section&id=Consolidated%20Results%20of%20Operations) This section analyzes the company's financial performance for the current and prior reporting periods [Three Months Ended June 30, 2021 compared to June 30, 2020](index=27&type=section&id=Three%20Months%20Ended%20June%2030,%202021%20compared%20to%20June%2030,%202020) Net product sales increased by **$15.1 million** (24.2%) to **$77.4 million**, driven by growth in the U.S., Europe, and Japan. Research and development expenses decreased by **$6.6 million** (9.5%) due to timing of clinical and manufacturing costs for the Pompe program and reduced personnel costs. Selling, general, and administrative expenses increased by **$7.6 million** (22.0%) due to higher personnel costs. Interest expense rose by **$4.5 million** due to the Senior Secured Loan. Net loss improved by **$1.3 million** | Metric (in thousands) | Q2 2021 | Q2 2020 | Change | % Change | | :-------------------------------- | :------ | :------ | :------- | :------- | | Net product sales | $77,413 | $62,353 | $15,060 | 24.2% | | Research and development | $63,003 | $69,611 | $(6,608) | -9.5% | | Selling, general, and administrative | $42,276 | $34,657 | $7,619 | 22.0% | | Interest expense | $(8,150) | $(3,635) | $(4,515) | 124.2% | | Net loss attributable to common stockholders | $(51,225) | $(52,492) | $1,267 | -2.4% | - R&D decrease primarily due to timing of clinical research and manufacturing costs for Pompe program and decreased personnel costs[113](index=113&type=chunk) [Six Months Ended June 30, 2021 compared to June 30, 2020](index=28&type=section&id=Six%20Months%20Ended%20June%2030,%202021%20compared%20to%20June%2030,%202020) Net product sales increased by **$20.9 million** (17.0%) to **$143.8 million**, driven by continued growth in key markets. Research and development expenses decreased by **$31.6 million** (19.9%) due to timing of Pompe clinical and manufacturing costs, gene therapy program spend, and personnel cost realignment. Selling, general, and administrative expenses increased by **$14.1 million** (18.9%) due to higher personnel costs. Interest expense rose by **$8.8 million** due to the Senior Secured Loan. Net loss improved by **$24.6 million** | Metric (in thousands) | H1 2021 | H1 2020 | Change | % Change | | :-------------------------------- | :------ | :------ | :------- | :------- | | Net product sales | $143,815 | $122,878 | $20,937 | 17.0% | | Research and development | $127,120 | $158,731 | $(31,611) | -19.9% | | Selling, general, and administrative | $89,002 | $74,872 | $14,130 | 18.9% | | Interest expense | $(16,142) | $(7,364) | $(8,778) | 119.2% | | Net loss attributable to common stockholders | $(116,889) | $(141,440) | $24,551 | -17.4% | - R&D decrease primarily due to timing of clinical research and manufacturing costs for Pompe program, decrease in gene therapy programs, and personnel cost realignment[117](index=117&type=chunk) [Liquidity and Capital Resources](index=29&type=section&id=Liquidity%20and%20Capital%20Resources) This section assesses the company's financial liquidity, capital availability, and future funding needs [Cash Flow Discussion](index=29&type=section&id=Cash%20Flow%20Discussion) This section provides a general overview of the company's cash flow activities, noting that detailed information is available in Note 3 - Cash, cash equivalents, and marketable securities totaled **$383.1 million** as of June 30, 2021[120](index=120&type=chunk) [Net Cash Used in Operating Activities](index=29&type=section&id=Net%20Cash%20Used%20in%20Operating%20Activities) Net cash used in operating activities decreased to **$108.3 million** for the six months ended June 30, 2021, from **$141.5 million** in the prior year. This improvement was primarily due to a reduced net loss and higher stock compensation, partially offset by changes in operating assets and liabilities related to contract manufacturing and research payments | Metric (in thousands) | H1 2021 | H1 2020 | Change | | :-------------------------------- | :------ | :------ | :------- | | Net cash used in operating activities | $(108,277) | $(141,460) | $33,183 | - Decrease in cash used in operations driven by reduced net loss and **$32.1 million** in stock compensation, partially offset by **$32.6 million** decrease from changes in operating assets and liabilities[121](index=121&type=chunk) [Net Cash Provided by Investing Activities](index=29&type=section&id=Net%20Cash%20Provided%20by%20Investing%20Activities) Net cash provided by investing activities decreased to **$112.3 million** for the six months ended June 30, 2021, from **$163.3 million** in the prior year. This was mainly due to a lower net inflow from the sale and purchase of marketable securities | Metric (in thousands) | H1 2021 | H1 2020 | Change | | :-------------------------------- | :------ | :------ | :------- | | Net cash provided by investing activities | $112,278 | $163,258 | $(50,980) | | Sale and redemption of marketable securities | $258,767 | $210,139 | $48,628 | | Purchases of marketable securities | $(145,255) | $(45,005) | $(100,250) | [Net Cash Provided by Financing Activities](index=30&type=section&id=Net%20Cash%20Provided%20by%20Financing%20Activities) Net cash provided by financing activities significantly increased to **$11.1 million** for the six months ended June 30, 2021, from **$2.6 million** in the prior year. This was primarily driven by **$19.2 million** from warrant exercises and **$6.7 million** from stock option exercises, partially offset by **$14.4 million** in employee withholding taxes for restricted stock unit vesting | Metric (in thousands) | H1 2021 | H1 2020 | Change | | :-------------------------------- | :------ | :------ | :------- | | Net cash provided by financing activities | $11,064 | $2,599 | $8,465 | | Proceeds from warrants exercised | $19,230 | $0 | $19,230 | | Proceeds from stock options exercised, net | $6,661 | $10,734 | $(4,073) | | Payments of employee withholding taxes | $(14,438) | $(8,097) | $(6,341) | [Funding Requirements](index=30&type=section&id=Funding%20Requirements) The company expects continued operating losses due to significant R&D expenses, particularly for clinical trials. Future capital needs depend on the progress of drug candidates (AT-GAA, CLN6, CLN3), manufacturing costs, regulatory outcomes, commercialization efforts, and potential acquisitions. The current cash position is deemed sufficient for self-sustainability, but additional funding may be sought - Expects continued operating losses due to significant R&D expenses, especially for clinical trials[127](index=127&type=chunk) - Future capital requirements are influenced by clinical trial progress, manufacturing costs, regulatory approvals, commercialization, and business development activities[127](index=127&type=chunk) - Current cash position, including expected revenues, is believed to be sufficient for self-sustainability, but additional funding may be sought[128](index=128&type=chunk) [Financial Uncertainties Related to Potential Future Payments](index=31&type=section&id=Financial%20Uncertainties%20Related%20to%20Potential%20Future%20Payments) The company has various potential future payment obligations, including development and regulatory milestones, sales-based milestones, and royalties, stemming from agreements with Celenex, Nationwide Children's, University of Pennsylvania, and GlaxoSmithKline. These payments could total significant amounts upon achievement of specified milestones - Potential future payments include up to **$10 million** in development milestones, **$220 million** in regulatory approval milestones, and **$75 million** in tiered sales milestones related to the Celenex acquisition[129](index=129&type=chunk) - Nationwide Children's is eligible for up to **$7.8 million** in development and sales-based milestones per product[130](index=130&type=chunk) - University of Pennsylvania is eligible for up to **$88.0 million** per indication in development and commercial milestones, plus royalties and **$10.0 million** annually for discovery research[131](index=131&type=chunk) - GlaxoSmithKline is eligible for post-approval and sales-based milestones up to **$40 million**, plus tiered mid-teens royalties in eight major markets outside the U.S. for Galafold[132](index=132&type=chunk) [Critical Accounting Policies and Significant Judgments](index=31&type=section&id=Critical%20Accounting%20Policies%20and%20Significant%20Judgments) The company's financial statements rely on estimates and assumptions in accordance with U.S. GAAP. There were no significant changes to critical accounting policies or estimates during the six months ended June 30, 2021, as previously disclosed in the 2020 Annual Report on Form 10-K - Financial statements require estimates and assumptions, which are evaluated on an ongoing basis[133](index=133&type=chunk) - No significant changes to critical accounting policies or estimates during the six months ended June 30, 2021[134](index=134&type=chunk) [Recent Accounting Pronouncements](index=31&type=section&id=Recent%20Accounting%20Pronouncements) The company refers to Note 2 for details on recent accounting pronouncements - Refer to Note 2 for details on recent accounting pronouncements[135](index=135&type=chunk) [Item 3. Quantitative and Qualitative Disclosures about Market Risk](index=32&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) The company's primary market risk exposure is to interest rate changes on its cash, cash equivalents, and marketable securities, which are high-quality, short-term instruments with minimal risk. A **1%** change in interest rates would impact the investment portfolio by **$0.7 million**. Variable rate debt (Senior Secured Term Loan) also exposes the company to interest rate risk, with a **1%** change impacting interest expense by **$1.0 million**. The company does not currently hedge these risks and expects no material impact from the LIBOR phase-out. Foreign exchange risk is also present but not hedged, with no material impact expected from a **10%** change in rates - Primary market risk is interest rate changes on cash, cash equivalents, and marketable securities; a **1%** change would impact the investment portfolio by **$0.7 million**[136](index=136&type=chunk) - Variable rate debt (Senior Secured Term Loan) exposes the company to interest rate risk; a **1%** change in the average interest rate would result in a **$1.0 million** change in interest expense[137](index=137&type=chunk) - No current hedging activities for interest rate or foreign currency exchange rate risks; LIBOR phase-out not expected to have a material impact[136](index=136&type=chunk)[138](index=138&type=chunk)[139](index=139&type=chunk) [Item 4. Controls and Procedures](index=32&type=section&id=Item%204.%20Controls%20and%20Procedures) As of June 30, 2021, the Principal Executive Officer and Principal Financial Officer concluded that the company's disclosure controls and procedures were effective. There were no material changes in internal control over financial reporting during the quarter - Disclosure controls and procedures were evaluated and deemed effective as of June 30, 2021[141](index=141&type=chunk) - No material changes in internal control over financial reporting occurred during the fiscal quarter[142](index=142&type=chunk) [PART II. OTHER INFORMATION](index=33&type=section&id=PART%20II.%20OTHER%20INFORMATION) This section covers additional disclosures not included in the financial statements [Item 1. Legal Proceedings](index=33&type=section&id=Item%201.%20Legal%20Proceedings) On July 13, 2021, Teva Pharmaceuticals filed a lawsuit against the company alleging violations of the CREATES ACT, seeking monetary damages. The company believes the lawsuit is without merit and intends to vigorously defend itself - Teva Pharmaceuticals filed a lawsuit on July 13, 2021, alleging CREATES ACT violations, seeking monetary damages[143](index=143&type=chunk) - The company believes the lawsuit is without merit and will vigorously defend itself[143](index=143&type=chunk) [Item 1A. Risk Factors](index=33&type=section&id=Item%201A.%20Risk%20Factors) There have been no material changes to the risk factors previously disclosed in the Annual Report on Form 10-K for the fiscal year ended December 31, 2020 - No material changes to risk factors since the Annual Report on Form 10-K for December 31, 2020[144](index=144&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=33&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) There were no unregistered sales of equity securities. The company purchased 55,830 shares of common stock during the three months ended June 30, 2021, at an average price of **$9.84** per share, primarily for employee withholding taxes related to restricted stock awards - No unregistered sales of equity securities[145](index=145&type=chunk) | Period | Total Shares Purchased | Average Price Paid per Share | | :-------------------------------- | :--------------------- | :--------------------------- | | April 1, 2021 through April 30, 2021 | 5,406 | $10.02 | | May 1, 2021 through May 31, 2021 | 13,013 | $9.40 | | June 1, 2021 through June 30, 2021 | 37,411 | $10.16 | | Total (Q2 2021) | 55,830 | $9.84 | - Shares purchased were primarily for employee withholding taxes related to restricted stock awards[146](index=146&type=chunk) [Item 3. Defaults Upon Senior Securities](index=33&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) No defaults upon senior securities - No defaults upon senior securities[147](index=147&type=chunk) [Item 4. Mine Safety Disclosures](index=33&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) No mine safety disclosures - No mine safety disclosures[148](index=148&type=chunk) [Item 5. Other Information](index=33&type=section&id=Item%205.%20Other%20Information) No other information to disclose - No other information to disclose[149](index=149&type=chunk) [Item 6. Exhibits](index=34&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the Form 10-Q, including certifications from the Principal Executive Officer and Principal Financial Officer, and Inline XBRL documents - Includes certifications (31.1, 31.2, 32.1) and Inline XBRL documents (101.INS, 101.SCH, 101.CAL, 101.LAB, 101.PRE, 101.DEF, 104)[150](index=150&type=chunk) [SIGNATURES](index=35&type=section&id=SIGNATURES) The report was signed by John F. Crowley, Chairman and Chief Executive Officer, and Daphne Quimi, Chief Financial Officer, on August 5, 2021 - Report signed by John F. Crowley (Chairman and CEO) and Daphne Quimi (CFO) on August 5, 2021[155](index=155&type=chunk)

Revenue and Sales Performance - Galafold (migalastat) generated $66.4 million in revenue for the three months ended March 31, 2021, an increase of $5.9 million compared to the same period in the prior year[86]. - Net product sales increased by $5.9 million to $66.4 million for the three months ended March 31, 2021, compared to $60.5 million in the same period of 2020, driven by growth in the U.S., Europe, and Japan markets[104]. Clinical Trials and Research - The Phase 3 PROPEL study of AT-GAA for Pompe disease included 123 patients across 62 sites in 24 countries, with topline results reported in February 2021[89]. - Patients treated with AT-GAA walked an average of 21 meters farther at 52 weeks compared to 7 meters for those treated with alglucosidase alfa, although statistical significance was not achieved (p=0.072)[89]. - AT-GAA demonstrated a 0.9% absolute decline in percent-predicted Forced Vital Capacity (FVC) compared to a 4.0% decline in the alglucosidase alfa arm, achieving statistical significance (p=0.023)[90]. - The company is conducting clinical programs for CLN6 and CLN3 Batten disease, with positive interim results indicating treatment was well tolerated and showed meaningful effects in slowing disease progression[99][100]. - The engineered hGAA AAV gene therapy demonstrated better targeting and clearance of glycogen storage in Pompe mice compared to unmodified hGAA AAV, indicating potential for effective treatment[96]. Financial Position and Cash Flow - Total cash, cash equivalents, and marketable securities as of March 31, 2021, was $417.4 million, sufficient to fund operations and ongoing research programs[86]. - The company reported a net loss attributable to common stockholders of $65.7 million for the three months ended March 31, 2021, an improvement of $23.3 million compared to a net loss of $88.9 million in the same period of 2020[104]. - Net cash used in operating activities was $72.4 million for the three months ended March 31, 2021, compared to $107.9 million for the same period in 2020[110][111]. - Net cash provided by investing activities was $86.6 million for the three months ended March 31, 2021, reflecting $163.7 million from the sale and redemption of marketable securities[112]. - Net cash provided by financing activities was $8.8 million for the three months ended March 31, 2021, primarily from the exercise of outstanding warrants and stock options[114]. Regulatory Designations and Approvals - The FDA granted Breakthrough Therapy designation to AT-GAA for late-onset Pompe disease, and orphan drug designation was received in Japan[86]. - Galafold is approved in over 40 countries, including the U.S., E.U., U.K., and Japan, with plans for further launches in 2021[86]. Strategic Alliances and Collaborations - The company is exploring strategic alliances and business development opportunities to enhance stockholder value and access necessary resources for developing therapies for rare diseases[103]. - The company has agreed to pay up to an additional $10 million in milestone payments and $220 million for regulatory approval milestones related to the acquisition of Celenex[118]. - Under the exclusive license agreement with Nationwide Children's, the company is obligated to pay up to $7.8 million in development and sales-based milestones for each product[119]. - The company has a research collaboration agreement with Penn, which allows for milestone payments up to $88 million per indication and annual funding of $10 million for discovery research[120]. - The collaboration agreement with GlaxoSmithKline includes potential post-approval and sales-based milestones up to $40 million, along with tiered royalties in the mid-teens across eight major markets outside the U.S.[121]. Debt and Financial Risks - The company holds a $400 million Senior Secured Term Loan due 2026, with an annual average variable interest rate of 7.5%[126]. - A hypothetical 100 basis point change in the average interest rate on the company's variable rate debt would result in a $1 million change in interest expense[126]. - The company faces foreign exchange risk from transactions in currencies other than U.S. dollars, but does not currently engage in foreign currency hedging activities[128]. - There have been no material changes in the company's financial instrument portfolio or market risk exposures since the fiscal year ended December 31, 2020[129]. Operational Outlook - The company expects to incur operational losses due to ongoing research and development expenses, particularly for clinical trials of drug candidates such as AT-GAA, CLN6, and CLN3[116]. - The company anticipates an increase in cash used in operations over the next several quarters and years without additional funding, despite generating revenue from product sales[117]. - Research and development expenses decreased by $25.0 million to $64.1 million for the three months ended March 31, 2021, primarily due to the timing of clinical research and manufacturing costs associated with the Pompe program[105]. - The company has maintained operations and supply chains during the COVID-19 pandemic, ensuring uninterrupted access to medicines[85].

N-140N-233N-390N-470N-652N-882N-925 rhGAA – kif + Endo H IHC : LC3 II B. 0 1 2 3 4 0 20 40 60 80 100 120 Time (Hours) Normalized GAA Activity(%) Stability in buffer (pH 7.4) 0 1 2 3 4 0 20 40 60 80 100 120 Cipaglucosidase alfa Cipaglucosidase alfa + 17 µM miglustat Cipaglucosidase alfa + 170 µM miglustat Stability in human blood Time (Hours) Normalized GAA Activity(%) Enhancing Delivery of Acid Alpha-Glucosidase (GAA) to Skeletal Muscle in Pompe Disease (PD): Key Challenges and Attributes of AT-GAA Nithya S ...

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, 2020 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 UNITED STATES SECURITIES AND EXCHANGE COMMISSION For the transition period from to Commission file number 001-33497 Amicus Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) (State or Other Jurisdiction of (I.R.S. Employ ...

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, 2020 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 ...