Amicus Therapeutics(FOLD) - 2023 Q3 - Quarterly Report

Special Note Regarding Forward-Looking Statements 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 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] - 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][13] - 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][14] PART I. FINANCIAL INFORMATION Item 1. Consolidated Financial Statements and Notes (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 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 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 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 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] Consolidated Statements of Cash Flows 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 1. Description of Business - 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][38] - Galafold is the first oral monotherapy for Fabry disease, approved in the U.S., E.U., U.K., and Japan[38] - 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] - 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] - 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][39] - Management believes current cash, including expected revenues, is sufficient to fund operations and research programs for at least the next 12 months[40] 2. Summary of Significant Accounting Policies - Unaudited Consolidated Financial Statements are prepared in accordance with U.S. GAAP for interim financial information[41] - The functional currency for most foreign subsidiaries is their local currency, with translation adjustments recorded in accumulated other comprehensive income[44] - 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] - Revenue is recognized when performance obligations are satisfied, typically when pharmacies or distributors obtain control, net of estimated variable consideration[52] 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] - 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][58][59] 3. Intangible Assets - Intangible assets primarily consist of lead enzyme replacement therapy assets from the 2013 Callidus Biopharma acquisition[61] - Following EC approval of Pombiliti in March 2023, these assets began to be amortized over an initial regulatory exclusivity period of 7 years[61] 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 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] - 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] 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 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] 6. Debt 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] - 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] 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 - 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] - As of September 30, 2023, $184.4 million worth of shares remain available under the ATM program[73] - In October 2023, a private placement of 2,467,104 common shares to Blackstone generated $29.8 million in net proceeds[74] 8. Stock-Based Compensation - 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] 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] 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] 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 - 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][81] 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] - 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] 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 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] 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 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 - Amicus Therapeutics is a global biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases[88] - 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][89] Our Strategy - 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] - 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] - Current cash position is believed to be sufficient to fund operations and research programs for at least the next 12 months[91] Our Commercial Products and Product Candidates - 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] - The Galafold U.S. patent portfolio includes 54 Orange Book listed patents, with 38 providing protection through at least 2038[93] - 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] - 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][96][97] Consolidated Results of Operations - Three Months Ended September 30, 2023 compared to September 30, 2022 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] - 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] - 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] Consolidated Results of Operations - Nine Months Ended September 30, 2023 compared to September 30, 2022 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] - 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] - 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] - 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] Liquidity and Capital Resources - The company has historically funded operations through stock offerings, product revenues, debt issuance, and collaborations, having generated operating losses since 2002[105] - As of September 30, 2023, cash, cash equivalents, and marketable securities totaled $280.3 million[107] - 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] - 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] - 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] - Future capital requirements depend on clinical trial costs, manufacturing, regulatory approvals, commercialization, and potential business development[113] - Management believes current cash and expected revenues are sufficient for at least the next 12 months, but may seek additional funding[114] Critical Accounting Policies and Significant Judgments - The preparation of financial statements requires management to make estimates and assumptions affecting reported amounts of assets, liabilities, revenues, and expenses[115] - No significant changes occurred during the nine months ended September 30, 2023, to the previously disclosed significant accounting policies and estimates[116] Recent Accounting Pronouncements - The company has evaluated recent accounting pronouncements and believes none will have a material effect on its Consolidated Financial Statements or related disclosures[60][117] Item 3. Quantitative and Qualitative Disclosures about Market Risk 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] - 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] - 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] - No material changes to market risks or their management have occurred since December 31, 2022, except for the debt reference rate transition[121] Item 4. Controls and Procedures 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] - No material changes in internal control over financial reporting occurred during the fiscal quarter ended September 30, 2023[123] PART II. OTHER INFORMATION Item 1. Legal Proceedings 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] - 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] Item 1A. Risk Factors 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] Item 2. Unregistered Sales of Equity Securities and Use of Proceeds 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] 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] Item 3. Defaults Upon Senior Securities This section reports no defaults upon senior securities during the reported period - No defaults upon senior securities were reported[128] Item 4. Mine Safety Disclosures This section indicates no mine safety disclosures to report - No mine safety disclosures were reported[129] Item 5. Other Information 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][133] - These plans were adopted during an open insider trading window and in accordance with company policies[131] - 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] Item 6. Exhibits 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] - 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] - Inline XBRL documents (Instance, Schema, Calculation, Label, Presentation, Definition Linkbase) are provided for interactive data filing[134][135] SIGNATURES Report Signatures 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]