Amicus Therapeutics Inc.

02/20/2026 | Press release | Distributed by Public on 02/20/2026 15:05

Annual Report for Fiscal Year Ending December 31, 2025 (Form 10-K)

MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS
Overview
We are a leading, global biotechnology company with a clear and compelling mission to develop and deliver transformative medicines for people living with rare diseases. With extraordinary patient focus, we strive to redefine expectations in rare disease. Our two marketed therapies are Galafold®, the first oral monotherapy for people living with Fabry disease who have amenable genetic variants, and Pombiliti®+ Opfolda®, a novel two-component treatment designed to improve uptake of active enzyme into key disease relevant tissues for adults living with late-onset Pompe disease.
Galafold®(also referred to as "migalastat") is approved in over 40 countries around the world, including the United States ("U.S."), European Union ("E.U."), United Kingdom ("U.K."), and Japan. Additionally, Galafold®has been granted orphan drug designation in the U.S., E.U., U.K., Japan, and several other countries.
Pombiliti®+ Opfolda®(also referred to as "cipaglucosidase alfa-atga/miglustat") is approved in the U.S., the E.U., the U.K., Canada, Australia, Switzerland, and Japan. Multiple regulatory submissions and reimbursement processes with global health authorities are currently underway. Additionally, Pombiliti®+ Opfolda®has been granted orphan drug designation or status in the U.S., U.K., Switzerland and Japan and data exclusivity in the E.U.
On April 30, 2025, we entered into an exclusive license agreement with Dimerix Bioscience Pty Limited ("Dimerix") for the United States commercialization rights of Dimerix' Phase 3 drug candidate, DMX-200 for treatment of Focal Segmental Glomerulosclerosis ("FSGS") and other indications. In exchange for these rights, we paid Dimerix an upfront payment of $30 million. We will be obligated to pay Dimerix for certain success-based development and regulatory milestones for FSGS of up to a maximum aggregate amount of $75 million, regulatory milestones for other indications of up to a maximum aggregate amount of $40 million, commercial milestones of up to a maximum aggregate amount of $445 million, and tiered royalties of DMX-200 net sales in the U.S. ranging from the low-teens to low-twenties. Dimerix will continue to fund and execute the ACTION3 study, and we will be responsible for submission and maintenance of the regulatory dossier in the United States, as well as all costs of commercialization activities. Additionally, we will have the exclusive rights to develop DMX-200 in other future indications in the United States. Amicus and Dimerix have formed a Joint Steering Committee to align the development and commercialization of DMX-200 in FSGS in U.S.
On December 19, 2025, we entered into a Merger Agreement with BioMarin and its wholly owned acquisition subsidiary. Under the terms of the Merger Agreement, and in accordance with the DGCL, if the Merger is completed, BioMarin's wholly owned acquisition subsidiary will merge with and into Amicus, and Amicus will continue as the surviving corporation as a wholly owned subsidiary of BioMarin. In addition, all shares of our common stock outstanding immediately prior to closing (other than Excluded Shares or Dissenting Shares (as defined in the Merger Agreement)) will be cancelled and converted into the right to receive $14.50 per share in cash, without interest and subject to any applicable withholding of taxes. Following completion of the Merger, shares of our common stock will no longer be publicly listed. Refer to "- Note 15. Merger Agreement," in our Notes to Consolidated Financial Statements for additional information.
-75-
Consolidated Results of Operations
The following discussion should be read in conjunction with the Consolidated Financial Statements and related notes included elsewhere in this report. The following section generally discusses 2025 and 2024 items and year-to-year comparisons between 2025 and 2024. Discussions of 2023 items and year-to-year comparisons between 2024 and 2023 that are not included in this Form 10-K can be found in Part II, Item 7 of the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2024, which comparisons are hereby incorporated by reference.
Year Ended December 31, 2025 Compared to Year Ended December 31, 2024
The following table provides selected financial information of our operations:
Years ended December 31,
(in thousands) 2025 2024 Change
Net product sales $ 634,210 $ 528,295 $ 105,915
Cost of goods sold 72,929 52,943 19,986
Gross Profit 561,281 475,352 85,929
Operating expenses:
Research and development 135,843 109,362 26,481
Selling, general, and administrative 383,487 323,379 60,108
Loss on impairment of assets 1,702 - 1,702
Restructuring charges - 9,188 (9,188)
Depreciation and amortization 7,460 8,547 (1,087)
Other (expense) income:
Interest income 3,317 5,407 (2,090)
Interest expense (46,159) (49,598) 3,439
Other expense 10,244 (9,441) 19,685
Income tax expense (27,301) (27,350) 49
Net loss attributable to common stockholders $ (27,110) $ (56,106) $ 28,996
Net Product Sales. Net product sales increased $105.9 million during the year ended December 31, 2025 compared to the prior year. The increase was primarily due to both the continued growth of Galafold®and Pombiliti®+ Opfolda®in Europe and the U.S. and a $13.5 million favorable impact of foreign currency exchange.
Cost of Goods Sold. Cost of goods sold includes manufacturing costs as well as royalties associated with net product sales. Cost of goods sold increased by $20.0 million primarily related to the increase in net product sales. A portion of Pombiliti® inventory was expensed as research and development costs prior to regulatory approval and as such, the cost of goods sold and related gross margins are not necessarily indicative of future costs of goods sold and gross margin.
-76-
Research and Development Expense. The following table summarizes our principal product development programs for each product candidate in development, and the out-of-pocket, third-party expenses incurred with respect to each product candidate:
(in thousands) Years ended December 31,
Projects 2025 2024
Third-party direct project expenses
Galafold®(Fabry Disease)
$ 11,229 $ 9,298
Pombiliti®+ Opfolda®(Pompe Disease)
48,553 45,215
DMX-200 (FSGS kidney disease) 30,000 -
Pre-clinical and other programs 1,518 2,437
Total third-party direct project expenses 91,300 56,950
Other project costs 1
Personnel costs 34,373 41,405
Other costs 10,170 11,007
Total other project costs 44,543 52,412
Total research and development costs $ 135,843 $ 109,362
The $26.5 million increase in research and development costs was primarily driven by the $30.0 million upfront license payment to Dimerix.
Selling, General, and Administrative Expense. Selling, general, and administrative expense increased $60.1 million, primarily driven by higher personnel costs and additional legal and professional fees in connection with our intellectual property litigation and the signing of the Merger Agreement with BioMarin.
Restructuring Charges. Restructuring charges in the prior year were primarily related to an initiative to reduce operating costs by abandoning a lease that we no longer believed was useful in our operations.
Other (Expense) Income. The $19.7 million variance was primarily related to the movement in foreign exchange rates caused by remeasurement of foreign-denominated balances.
Income Tax Expense. The income tax expense for the year ended December 31, 2025 was $27.3 million. We are subject to income taxes in various jurisdictions. Our tax liabilities are largely dependent on the mix of pre-tax earnings among the many jurisdictions in which we operate and differences in the timing of the recognition of such earnings under the relevant accounting standards and tax rules.
-77-
Critical Accounting Policies and Significant Judgments and Estimates
The discussion and analysis of our financial condition and results of operations are based on our financial statements, which we have prepared in accordance with U.S. generally accepted accounting principles ("U.S. GAAP"). The preparation of these financial statements requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements, as well as the reported revenues and expenses during the reporting periods. On an ongoing basis, we evaluate our estimates and judgments, including those described in greater detail below. We base our estimates on historical experience and on various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions. We believe that the following discussion represents our critical accounting policies.
Revenue Recognition
Our net product sales consist of sales of Galafold® for the treatment of Fabry disease and Pombiliti®+ Opfolda® for the treatment of Pompe disease. We have recorded revenue on sales where our products are available either on a commercial basis or through a reimbursed early access program. Orders for our products are generally received from distributors and pharmacies with the ultimate payor often a government authority.
We recognize revenue when our performance obligations with our customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of our products. The transaction price is determined based on fixed consideration in our customer contracts net of estimates for variable consideration. Variable consideration, which primarily consists of discounts and rebates due to foreign and U.S. government programs, is estimated based on contractual arrangements or statutory obligations, which may vary by product and payer and is recorded in the same period the related sales occur.
Estimation requires evaluation of our actual historical experience, customer mix, current contractual and statutory obligations, and inventory channel levels. We evaluate our customer mix to estimate which sales will be subject to which revenue dilutive items and consider changes to government program guidelines or contractual obligations that would impact the actual rebate or discount and/or our estimates of which sales qualify for such rebate or discount. We recognize revenue to the extent that it is probable that a significant revenue reversal will not occur in a future period. These estimates may differ from actual consideration received. We evaluate these estimates each reporting period to reflect known changes.
Liquidity and Capital Resources
As a result of our significant research and development expenditures, as well as expenditures to build a commercial organization to support the launch of Galafold® and Pombiliti®+ Opfolda®, we have not been profitable and have generated operating losses since we were incorporated in 2002. We have historically funded our operations through stock offerings, product revenues, debt issuance, collaborations, and other financing arrangements. The Merger Agreement with BioMarin includes restrictions on the conduct of our business prior to the completion of the Merger or termination of the Merger Agreement. These limitations include, among other things, restrictions on our ability to incur additional indebtedness, issue additional shares of our common stock outside of existing equity plans and enter into or amend certain contracts.
Sources of Liquidity
In November 2022, we entered into a Sales Agreement with Goldman Sachs & Co. LLC to create an at-the-market equity program ("ATM program"), pursuant to which we may offer to sell shares of our common stock having aggregate offering gross proceeds of up to $250.0 million. During the year ended December 31, 2025, there were no shares issued and sold through the ATM program. As of December 31, 2025, an aggregate of $164.2 million worth of shares remain available to be issued and sold under the ATM program. Under the Merger Agreement, we are prohibited from offering to sell any additional shares of our common stock under the ATM program.
-78-
In October 2023, we entered into the Senior Secured Term Loan due 2029. This transaction resulted in net proceeds of $387.4 million, after deducting fees and expenses. There were no warrants or equity conversion features associated with the Senior Secured Term Loan due 2029. Simultaneously, we also entered into a securities purchase agreement with funds managed by Blackstone, for the private placement of an aggregate of 2,467,104 shares of our common stock, at a purchase price of $12.16 per share. Proceeds from the private placement, net of offering costs, were $29.8 million. We used proceeds from the Senior Secured Term Loan due 2029 and the private placement to prepay the Senior Secured Term Loan due 2026, inclusive of the outstanding principal amount, accrued interest and prepayment premium.
Cash Flow Discussion
As of December 31, 2025, we had cash, cash equivalents, and marketable securities of $293.5 million. We invest cash in excess of our immediate requirements in regard to liquidity and capital preservation in a variety of interest-bearing instruments, including obligations of U.S. government agencies and money market accounts. Wherever possible, we seek to minimize the potential effects of concentration and degrees of risk. Although we maintain cash balances with financial institutions in excess of insured limits, we do not anticipate any losses with respect to such cash balances. For more details on the cash, cash equivalents, and marketable securities, refer to "- Note 4. Cash, Cash Equivalents, Marketable Securities, and Restricted Cash," in our Notes to Consolidated Financial Statements.
Net Cash Provided by (Used in) Operating Activities
Net cash provided by operations for the year ended December 31, 2025 was $33.1 million. The components of net cash provided by operations primarily reflect net loss of $27.1 millionadjusted for non-cash expenses of $99.9 million, which includes $87.4 millionof stock compensation and $7.5 millionof depreciation expense. This is further driven by a net increase in operating assets and liabilities of $39.6 million. The changes in operating assets and liabilities were primarily driven by an increase in inventory of $111.5 millionto support our continued commercial growth, partially offset by an $82.8 million increase in accounts payable and accrued expenses.
Net cash used in operations for the year ended December 31, 2024 was $33.9 million. The components of net cash used in operations included the net loss for the year ended December 31, 2024 of $56.1 million and the net change in operating assets and liabilities of $98.9 million partially offset by $84.9 million of stock compensation and $36.3 million of other non-cash adjustments. The change in operating assets was primarily due to an increase in inventory of $73.7 million to support our continued commercial growth, an increase in accounts receivable of $19.5 million, and a decrease in accounts payable and accrued expenses of $16.5 million associated with timing of payments, partially offset by a decrease in prepaid expenses and other current assets by $14.0 million.
Net Cash Used in Investing Activities
Net cash used in investing activities for the year ended December 31, 2025 was $46.4 million. Our investing activities have consisted primarily of purchases, sales, and maturities of investments and capital expenditures. Net cash used in investing activities reflects $94.4 million for the purchase of marketable securities and $3.3 million for capital expenditures, partially offset by $51.3 million for the sale and redemption of marketable securities.
Net cash used in investing activities for the year ended December 31, 2024 was $0.6 million. Our investing activities have consisted primarily of purchases, sales, and maturities of investments and capital expenditures. Net cash used in investing activities reflects $114.8 million for the purchase of marketable securities and $3.6 million for capital expenditures, partially offset by $117.8 million for the sale and redemption of marketable securities.
Net Cash Provided by Financing Activities
Net cash provided by financing activities for the year ended December 31, 2025 was $0.6 million. Net cash provided by financing activities primarily reflects $19.2 million in proceeds from the exercise of stock options, partially offset by $18.3 million for payments of employee withholding taxes related to restricted stock unit vesting.
-79-
Net cash provided by financing activities for the year ended December 31, 2024 was $5.1 million. Net cash provided by financing activities primarily reflects $19.6 million of net proceeds from the issuance of shares in connection with our ATM program and $7.7 million in proceeds from the exercise of stock options, partially offset by $22.0 million for payments of employee withholding taxes related to restricted stock unit vesting.
Funding Requirements
We expect to continue to incur significant costs in the foreseeable future primarily due to research and development expenses, including expenses related to conducting clinical trials. Our future capital requirements will depend on a number of factors, including:
•the pending transaction with BioMarin;
•the scope, progress, results and costs of clinical trials for our drug candidates;
•the cost of manufacturing drug supply for our commercial, clinical and preclinical studies, including the cost of manufacturing Pombiliti®(also referred to as "ATB200" or "cipaglucosidase alfa");
•the results of preclinical research and clinical trials for DMX-200 and other pipeline candidates we may identify from time to time, including our ability to obtain regulatory approvals and commercialize such therapies;
•the costs, timing, and outcome of regulatory review of our product candidates;
•any changes in regulatory standards relating to the review of our product candidates;
•any changes in laws, rules or regulations, including the imposition of tariffs, most favored nation requirements, or other trade restrictions or requirements, affecting our ability to manufacture, transport, test, develop, or commercialize our products, including Galafold®, Pombiliti®+ Opfolda®, or our product candidates;
•the costs of commercialization activities, including product marketing, sales, and distribution;
•the emergence of competing technologies and other adverse market developments;
•the estimates regarding the potential market opportunity for our products and product candidates;
•our ability to successfully commercialize Galafold®(also referred to as "migalastat HCl");
•our ability to successfully commercialize Pombiliti®+ Opfolda®in the E.U., U.K., Japan, and U.S., and elsewhere, if regulatory applications are approved;
•our ability to manufacture or supply sufficient clinical trial or commercial products, including DMX-200, Galafold® and Pombiliti®+ Opfolda®;
•our ability to obtain reimbursement for Galafold® and Pombiliti®+ Opfolda®;
•our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of Galafold®and Pombiliti®+ Opfolda®;
•our ability to obtain market acceptance of Galafold® and Pombiliti®+ Opfolda®or any other product developed or acquired that has received regulatory approval;
•the costs of preparing, filing, and prosecuting patent applications and maintaining, enforcing, and defending intellectual property-related claims, including Hatch-Waxman litigation;
•the impact of litigation that has been or may be brought against us or of litigation that we are pursuing or may pursue against others, including Hatch-Waxman litigation;
•the extent to which we acquire or invest in businesses, products, and technologies;
•our ability to successfully integrate acquired products and technologies into our business, or successfully divest or license existing products and technologies from our business, including the possibility that the expected benefits of the transactions will not be fully realized by us or may take longer to realize than expected;
•our ability to establish licensing agreements, collaborations, partnerships or other similar arrangements and to obtain milestone, royalty, or other economic benefits from any such collaborators;
-80-
•the costs associated with, and our ability to comply with, emerging sustainability standards, including climate reporting requirements at the local, state and national levels, especially abroad;
•our ability to successfully protect our information technology systems and maintain our global operations and supply chain without interruption;
•our ability to accurately forecast revenue, operating expenditures, or other metrics impacting profitability;
•fluctuations in foreign currency exchange rates; and
•changes in accounting standards.
If the Merger is not consummated, we may seek additional funding through public or private financings of debt or equity. Based on our current operating model and excluding any impact of the pending consummation of the Merger, we believe that the current cash position and expected revenues are sufficient to fund our operations and ongoing research programs for at least the next 12 months. Potential impacts of business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact our long-term capital requirements.
Contractual Obligations and Commitments
As of December 31, 2025, remaining maturities, including expected interest payments through maturity, on our Senior Secured Term Loan due 2029 were $498.1 million. Refer to "- Note 11. Debt," to the Consolidated Financial Statements for more information.
We are lessees to various operating leases for facilities and equipment. As of December 31, 2025, our undiscounted cash liabilities for operating leases were $72.0 million, with maturities ranging up through fiscal 2034. Refer to "- Note 12. Leases," to the Consolidated Financial Statements for more information.
On December 19, 2025, the Company entered into the Merger Agreement with BioMarin. Under the terms of the Merger Agreement, the Company may be required to pay BioMarin a termination fee of $175 million if the Merger Agreement is terminated under specific circumstances described in the Merger Agreement.
We have a number of binding minimum purchase and manufacturing commitments due to our third-party manufacturers. As of December 31, 2025, these purchase and manufacturing obligations totaled $218.8 million, of which $195.7 million and $23.1 million are expected in 2026 and 2027, respectively. Contracts for which our commitment is variable, based on volumes, with no fixed minimum quantities, and contracts that can be canceled without payment penalties have been excluded. These purchase obligations are in addition to amounts recorded on our December 31, 2025 Consolidated Balance Sheets.
We have no off-balance sheet arrangements as of December 31, 2025 and 2024.
Recent Accounting Pronouncements
Please refer to "- Note 2. Summary of Significant Accounting Policies," in our Notes to the Consolidated Financial Statements.
Amicus Therapeutics Inc. published this content on February 20, 2026, and is solely responsible for the information contained herein. Distributed via EDGAR on February 20, 2026 at 21:05 UTC. If you believe the information included in the content is inaccurate or outdated and requires editing or removal, please contact us at [email protected]