AMICUS THERAPEUTICS, INC. - Quarter Report: 2025 March (Form 10-Q)

RSUs awarded under the Plan are generally subject to graded vesting and are contingent on an employee's continued service. RSUs are generally subject to forfeiture if employment terminates prior to the release of vesting restrictions. The Company expenses the cost of the RSUs, which is determined to be the fair market value of the shares of common stock underlying the RSUs at the date of grant, ratably over the period during which the vesting restrictions lapse.

As of March 31, 2025, there was $ million of total unrecognized compensation cost related to non-vested RSUs with service-based vesting conditions. These costs are expected to be recognized over a weighted average period of .

Deferred compensation plan liability is recorded as a component of other non-current liabilities on the Company's Consolidated Balance Sheets. The Company did not have any Level 3 assets or liabilities as of March 31, 2025 or December 31, 2024.

Dilutive common stock equivalents would include the dilutive effect of outstanding common stock options and non-vested RSUs. Potentially dilutive common stock equivalents were excluded from the diluted earnings per share denominator for all periods because of their anti-dilutive effect.

The following discussion should be read in conjunction with the unaudited Consolidated Financial Statements and the notes thereto included in this Quarterly Report on Form 10-Q and the audited Consolidated Financial Statements and the notes thereto included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024. Some of the statements we make in this section are forward-looking statements within the meaning of the federal securities laws. For a complete discussion of forward-looking statements, see the section in this Quarterly Report on Form 10-Q entitled “Special Note Regarding Forward-Looking Statements”. Certain risk factors may cause actual results, performance or achievements to differ materially from those expressed or implied by the following discussion. For a discussion of such risk factors, see the section in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 entitled “Risk Factors”.

We are a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases. We seek to deliver the highest quality therapies that have the potential to obsolete current treatments, provide significant benefits to patients, and be first- or best-in-class. 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 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 and Switzerland. 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, the Company entered into an exclusive license agreement with Dimerix Limited ("Dimerix") for the commercialization of Dimerix' Phase 3 drug candidate, DMX-200, for all indications, including Focal Segmental Glomerulosclerosis ("FSGS") in the United States. In exchange for these rights, the Company will pay a $30 million upfront payment. The next potential milestone payment is based on positive data from the Phase 3 trial in FSGS. In total, Dimerix is eligible to receive potential success-based development and regulatory milestone payments of up to $75 million, $35 million on first sale, commercial sales milestone payments of up to $410 million, and tiered royalties from the low-teens to low-twenties percentages of DMX-200 net sales in the U.S. In addition, Dimerix is eligible to receive up to $40 million in milestone payments for potential future indications.

Our strategy is to create, manufacture, test, and deliver the highest quality medicines for people living with rare diseases through internally developed, jointly developed, acquired, or in-licensed products and product candidates. We are leveraging our global capabilities to develop and broaden our franchises in Fabry and Pompe disease, with focused discovery work on next generation therapies and novel technologies.

•Commercial success in Fabry disease. For the three months ended March 31, 2025, Galafold® revenue was $104.2 million of consolidated revenue, which represented an increase of $4.9 million compared the same period in the prior year. We continue to see strong commercial momentum and expansion into additional geographies.

•Commercial and regulatory success in Pompe disease. For the three months ended March 31, 2025, Pombiliti® + Opfolda® revenue was $21.0 million of consolidated revenue. As of March 31, 2025, Pombiliti® + Opfolda® has been approved by the respective regulatory authorities in the E.U., U.S., U.K., Canada, Switzerland and Australia. Additionally, in 2024, we established reimbursement agreements in multiple E.U. countries.

•Pipeline advancement and growth. On April 30, 2025, Amicus licensed exclusive rights to commercialize DMX-200, a small molecule currently in a pivotal Phase 3 study ("ACTION3"), for all indications, including Focal Segmental Glomerulosclerosis ("FSGS") in the United States. We are also continuing to leverage our global capabilities to

Our oral precision medicine, Galafold®, was granted accelerated approval by the FDA in August 2018 for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene ("GLA") variant based on in vitro assay data. Galafold® was approved in the E.U. and U.K. in May 2016 as a first-line therapy for long-term treatment of adults and adolescents, aged 16 years and older, with a confirmed diagnosis of Fabry disease and who have an amenable variant. Marketing authorization approvals as well as approvals for adolescents aged 12 years and older weighing 45 kg or more have been granted in over 40 countries around the world. We plan to continue to launch Galafold® in additional countries upon receipt of marketing authorization.

As an orally administered monotherapy, Galafold® is designed to bind to and stabilize an endogenous alpha-galactosidase A ("alpha-Gal A") enzyme in those patients with genetic variants identified as amenable in a Good Laboratory Practice ("GLP") cell-based amenability assay.

We have leveraged our biologics capabilities to develop Pombiliti® + Opfolda®, a novel two-component treatment paradigm for Pompe disease. Pombiliti® + Opfolda® was approved by the EC, the MHRA, and the FDA in 2023, and the Swissmedic and Australia in 2024 for adult late-onset Pompe disease ("LOPD") patients. Additional regulatory submissions and reimbursement processes with global health authorities are currently underway.

Pombiliti® + Opfolda® consists of a uniquely engineered rhGAA enzyme, cipaglucosidase alfa-atga, with an optimized carbohydrate structure to enhance cellular uptake, administered intravenously in combination with orally administered miglustat. Miglustat binds to and stabilizes the cipaglucosidase alfa-atga in circulation reducing inactivation of rhGAA in circulation to improve the uptake of active enzyme into key disease relevant tissues. Miglustat is not an active ingredient that contributes directly to glycogen reduction.

DMX-200 is a small molecule inhibitor of the chemokine receptor 2 (CCR2) under development in a pivotal Phase 3 study, ACTION3, for the treatment of Focal Segmental Glomerulosclerosis (FSGS) kidney disease. In early 2024, Dimerix reported positive interim results from the ACTION3 trial in FSGS showing DMX-200 was performing better than placebo in reducing proteinuria with no safety concerns to date. An additional blinded interim analysis is planned once the revised primary and secondary endpoints have been pre-specified in the protocol and agreed with the FDA. In a March 2025 Type C meeting, Dimerix successfully aligned with the FDA on proteinuria as an appropriate primary endpoint for traditional marketing approval for DMX-200.

FSGS is a rare, serious kidney disorder characterized by progressive scarring (sclerosis) in parts of the glomeruli—the kidney’s filtering units. This scarring leads to proteinuria, progressive loss of kidney function, and often end-stage renal disease. FSGS is increasingly understood to have an inflammatory component, with monocyte and macrophage activation contributing to glomerular injury. In the United States, more than 40,000 people are estimated to be living with FSGS, including both adults and children. There are no therapies specifically approved for FSGS in the U.S., and management relies on non-specific immunosuppressive and supportive therapies. In patients with progressive or treatment-resistant FSGS, the average time from diagnosis to end-stage kidney disease can be as short as five years. Even among those who undergo kidney transplantation, disease recurrence occurs in up to 60% of cases, underscoring the urgent need for new, disease-modifying treatments.

We are committed to continued innovation for all people living with Fabry or Pompe disease. As part of our long-term commitment, we are also continuing discovery for next-generation genetic medicines for Fabry and Pompe disease.

We will continue to evaluate business development opportunities to build stockholder value and provide us with access to the financial, technical, clinical, commercial resources, and intellectual property necessary to develop and market technologies or products in rare and orphan diseases. We are exploring potential collaborations, alliances, and various other business development opportunities on a regular basis. These opportunities may include business combinations, partnerships, the strategic out-licensing of certain assets, or the acquisition of preclinical-stage, clinical-stage, or marketed products or novel technologies consistent with our corporate strategy to develop and provide therapies to patients living with rare and orphan diseases.


	Three Months Ended March 31,
(in thousands)	2025		2024		Change
Net product sales	$	125,249	$	110,403	$	14,846
Cost of goods sold	11,698		13,567		(1,869)
Gross profit	113,551		96,836		16,715
Operating expenses:
Research and development	27,839		28,329		(490)
Selling, general, and administrative	91,827		88,029		3,798
Restructuring charges	—		6,045		(6,045)
Depreciation and amortization	1,837		2,154		(317)
Other expense:
Interest income	812		1,540		(728)
Interest expense	(11,455)		(12,436)		981
Other income (expense)	550		(4,966)		5,516
Income tax expense	(3,641)		(4,836)		1,195
Net loss attributable to common stockholders	$	(21,686)	$	(48,419)	$	26,733

Net Product Sales. Net product sales increased $14.8 million during the three months ended March 31, 2025 compared to the same period in the prior year. The increase was primarily due to both the continued growth of Galafold® in the U.S. as well as the continued growth of Pombiliti® + Opfolda® in Europe and the U.S., partially offset by a $1.4 million unfavorable 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 decreased by $1.9 million primarily due to inventory write-offs in the prior period.


(in thousands)	Three Months Ended March 31,
Projects	2025		2024
Third party direct project expenses
Galafold® (Fabry Disease)	$	2,925	$	1,815
Pombiliti® + Opfolda® (Pompe Disease)	12,551		11,320
Pre-clinical and other programs	362		617
Total third-party direct project expenses	15,838		13,752
Other project costs
Personnel costs	9,372		11,905
Other costs	2,629		2,672
Total other project costs	12,001		14,577
Total research and development costs	$	27,839	$	28,329

The $0.5 million decrease in research and development costs was primarily driven by a decrease in the number of employees supporting research and development projects, partially offset by increases in third party research and development expense.

Selling, General, and Administrative Expense. Selling, general, and administrative expense increased $3.8 million, primarily driven by personnel costs resulting from an increase in the number of employees and increased facility and technology transfer related expenses in connection with our continuing expansion of manufacturing capabilities.

Income Tax Expense. 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.

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.

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 an aggregate offering gross proceeds of up to $250.0 million. As of March 31, 2025, an aggregate of $164.2 million worth of shares remain available to be issued and sold under the ATM program.

As of March 31, 2025, we had cash, cash equivalents, and marketable securities of $250.6 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 3. Cash, Cash Equivalents, Marketable Securities, and Restricted Cash," in our Notes to Consolidated Financial Statements.

Net cash provided by operations for the three months ended March 31, 2025 was $7.8 million. The components of net cash provided by operations primarily reflect net loss of $21.7 million adjusted for non-cash expenses of $23.4 million, which includes $25.2 million of stock compensation, partially offset by $5.4 million gain in foreign currency remeasurement. This is further driven by a net decrease in operating assets and liabilities of $6.0 million. The changes in operating assets and liabilities were primarily due to a decrease in accounts receivable of $15.3 million and an increase in accounts payable and accrued expenses of $3.6 million, partially offset by an increase in inventory of $8.3 million to support our continued commercial growth, and an increase in prepaid expenses and other current assets of $3.6 million.

Net cash used in operations for the three months ended March 31, 2024 was $29.7 million. The components of net cash used in operations included the net loss for the three months ended March 31, 2024 of $48.4 million and a net decrease in changes in operating assets and liabilities of $22.3 million, offset by $30.8 million of stock compensation and $10.2 million of other non-cash adjustments. The changes in operating assets and liabilities were primarily due to a decrease in accounts payable and accrued expenses of $24.5 million associated with payments for Pombiliti® + Opfolda® launch activities and personnel costs.

Net cash used in investing activities for the three months ended March 31, 2025 was $32.9 million. Our investing activities have consisted primarily of purchases, sales, and maturities of investments and capital expenditures. Net cash used in investing activities reflects $38.8 million for the purchase of marketable securities, partially offset by $6.2 million from the sale and redemption of marketable securities and $0.2 million for capital expenditures.

Net cash provided by investing activities for the three months ended March 31, 2024 was $7.5 million. Our investing activities have consisted primarily of purchases, sales and maturities of investments and capital expenditures. Net cash provided by investing activities reflects $38.9 million from the sale and redemption of marketable securities, partially offset by $29.6 million for the purchase of marketable securities and $1.8 million for capital expenditures.

Net cash used in financing activities for the three months ended March 31, 2025 was $11.7 million. Net cash used in financing activities primarily reflects withholding taxes paid on vested restricted stock units of $11.8 million, partially offset by $0.1 million of proceeds from the exercise of stock options.

Net cash used in financing activities for the three months ended March 31, 2024 was $13.3 million. Net cash used in financing activities primarily reflects the withholding taxes paid on vested restricted stock units of $16.7 million, partially offset by $3.5 million of proceeds from the exercise of stock options,

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:

•any changes in laws, rules or regulations, including the imposition of tariffs, affecting our ability to manufacture, transport, test, develop, or commercialize our products, including Galafold®, Pombiliti® + Opfolda®, or our product candidates;

•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;

We may seek additional funding through public or private financings of debt or equity. Based on our current operating model, which includes expected revenues, we believe that the current cash position is 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.

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. 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 and make changes when necessary. 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.

There were no significant changes during the three months ended March 31, 2025 to the items that we disclosed as our significant accounting policies and estimates described in "—Note 2. Summary of Significant Accounting Policies" to the Company's financial statements as contained in the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2024.

Our market risks, and the way we manage them, are summarized in Part II, Item 7A, "Quantitative and Qualitative Disclosures About Market Risk" of our Annual Report on Form 10-K for the fiscal year ended December 31, 2024. As of March 31, 2025, there have been no material changes to our market risks or to our management of such risks since December 31, 2024.

As of the end of the period covered by this Quarterly Report on Form 10-Q, an evaluation of the effectiveness of our disclosure controls and procedures (pursuant to Rule 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended (the "Exchange Act") was carried out under the supervision of our Principal Executive Officer and Principal Financial Officer, with the participation of our management. Based on that evaluation, the Principal Executive Officer and the Principal Financial Officer concluded that, as of the end of such period, our disclosure controls and procedures are effective in recording, processing, summarizing and reporting, on a timely basis, information required to be disclosed by us in the reports that we file or submit under the Exchange Act and are effective in ensuring that information required to be disclosed by us in the reports that we file or submit under the Exchange Act is accumulated and communicated to our management, including our Principal Executive Officer and Principal Financial Officer, as appropriate, to allow timely decisions regarding required disclosure.

During the fiscal quarter covered by this report, there has been no change in our internal control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act) that occurred during the last fiscal quarter that has materially affected, or is reasonably likely to materially affect, our internal control over financial reporting.

In the fourth quarter of 2022, the Company received Paragraph IV Certification Notice Letters from Teva Pharmaceuticals USA, Inc. ("Teva"), Aurobindo Pharma Limited ("Aurobindo"), and Lupin Limited ("Lupin") in connection with Abbreviated New Drug Applications (“ANDA”) filed with the FDA requesting approval to market generic Galafold®. In November 2022, the Company filed four lawsuits against Teva, Lupin, and Aurobindo in the U.S. District Court for the District of Delaware (the "Court") for infringement of its Orange Book-listed patents. In the fourth quarter of 2023, a stipulation order to stay litigation with respect to Lupin was ordered. Additionally, in the first quarter of 2024, a stipulation was filed with the court and approved by the presiding judge, whereby the parties agreed to accept the Company’s definition of the terms that were in dispute. As such, the scheduled Markman hearing was deemed unneeded and cancelled.

In October 2024, the Company entered into a non-exclusive, non-transferable, royalty-free, fully paid-up license with Teva which will allow Teva to market its generic version of Galafold® in the United States beginning on January 30, 2037, or earlier in certain circumstances. In accordance with the license agreement, a consent judgment and permanent injunction was entered with the Court and all Hatch-Waxman litigation between Amicus and Teva has been terminated. As required by law, Amicus and Teva have submitted the confidential license agreement to the U.S. Federal Trade Commission and the U.S. Department of Justice for review.

The litigation will continue against Aurobindo as the remaining active party, and the litigation stay remains in place for Lupin. The Company has, and will continue to, vigorously enforce its Galafold® intellectual property rights.


Period	Total Number of Shares Purchased (1)	Average Price Paid per Share		Total Number of Shares Purchased as Part of Publicly Announced Plans or Programs	Maximum Number (or Approximate Dollar Value) of Shares That May Yet Be Purchased Under the Plans or Programs
January 1, 2025 through January 31, 2025	728,235	$	9.39	—	—
February 1, 2025 through February 28, 2025	24,411	$	9.54	—	—
March 1, 2025 through March 31, 2025	37,740	$	8.91	—	—
Total	790,386	$	9.37	—	—

______________________________

(1) The dates in this column represent the scheduled expiration date of each director or officer’s Rule 10b5-1 Trading Plan. Each Rule 10b5-1 Trading Plan may terminate earlier than the date provided should all transactions contemplated thereunder occur prior to such date.

(3) Trading under Mr. Clark’s new Rule 10b5-1 Trading Plan may not commence until August 16, 2025, which is after the expiration date of Mr. Clark’s existing Rule 10b5-1 Trading Plan, adopted on August 28, 2024 (the “Existing Plan”), and disclosed in the Company’s Quarterly Report on Form 10-Q for the period ended September 30, 2024.

(4) Mr. Clark’s new Rule 10b5-1 Trading Plan provides for the sale of an indeterminable number of shares of common stock which have been obtained from the vesting of restricted stock unit and performance restricted stock unit awards. The total number of shares of common stock available for sale is dependent on the number of shares that are ultimately sold under the Existing Plan.

ITEM 6. EXHIBITS


Exhibit Number						Description

31.1						Certification of Principal Executive Officer pursuant to Rules 13a-14 and 15d-14 promulgated pursuant to the Securities Exchange Act of 1934, as amended

31.2						Certification of Principal Financial Officer pursuant to Rules 13a-14 and 15d-14 promulgated pursuant to the Securities Exchange Act of 1934, as amended

32.1						Certification of Principal Executive Officer and Principal Financial Officer pursuant to Section 906 of the Sarbanes-Oxley Act of 2002

101.INS						Inline XBRL Instance Document - the instance document does not appear in the Interactive Data File because its XBRL tags are embedded within the Inline XBRL document
101.SCH						Inline XBRL Taxonomy Extension Schema Document
101.CAL						Inline XBRL Taxonomy Extension Calculation Linkbase Document
101.LAB						Inline XBRL Taxonomy Extension Label Linkbase Document
101.PRE						Inline XBRL Taxonomy Extension Presentation Linkbase Document
101.DEF						Inline XBRL Taxonomy Extension Definition Linkbase Document
104						Cover Page Interactive Data File (formatted in Inline XBRL and included in Exhibit 101)


		AMICUS THERAPEUTICS, INC.

Date:	May 1, 2025	By:	/s/ Bradley L. Campbell
			Bradley L. Campbell
			President and Chief Executive Officer
			(Principal Executive Officer)

Date:	May 1, 2025	By:	/s/ Simon Harford
			Simon Harford
			Chief Financial Officer
			(Principal Financial Officer)

AMICUS THERAPEUTICS, INC. - Quarter Report: 2025 March (Form 10-Q)

Similar companies