Amicus Therapeutics Inc.

11/04/2025 | Press release | Distributed by Public on 11/04/2025 15:04

Quarterly Report for Quarter Ending September 30, 2025 (Form 10-Q)

MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS
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".
Overview
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, 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.
Our Strategy
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.
Highlights of our progress include:
•Commercial success in Fabry disease.For the nine months ended September 30, 2025, Galafold®revenue was $371.5 million of consolidated revenue, which represented an increase of $40.9 million compared to 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 nine months ended September 30, 2025, Pombiliti®+ Opfolda®revenue was $77.5 million of consolidated revenue. As of September 30, 2025, Pombiliti®+ Opfolda®has been approved by the respective regulatory authorities in the E.U., U.S., U.K., Canada, Switzerland, Australia, and Japan. Additionally, throughout 2024 and 2025, 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"), in the United States for treatment of FSGS and other indications.
•Financial strength. Total cash, cash equivalents, and marketable securities as of September 30, 2025 was $263.8 million.
Our Commercial Products and Product Candidates
Galafold® (migalastat HCl) for Fabry Disease
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.
Pombiliti®(cipaglucosidase alfa-atga) + Opfolda®(miglustat) for Pompe Disease
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 European Commission ("EC"), the Medicines and Healthcare products Regulatory Agency ("MHRA"), and the FDA in 2023, the Swissmedic and the Therapeutics Good Administration in Australia in 2024, and Health Canada, and Japan's Ministry of Health, Labor and Welfare in 2025 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 recombinant human acid alpha-glucosidase ("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.
In addition, clinical studies are ongoing in pediatric patients for both the LOPD and infantile-onset Pompe disease ("IOPD") populations.
DMX-200 for Focal Segmental Glomerulosclerosis (FSGS)
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 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 leakage of protein into the urine (proteinuria), and progressive loss of kidney function leading to end-stage kidney 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. Management of the disease 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. DMX-200 is the only clinical-stage kidney disease program that
specifically targets monocyte-driven inflammation, a mechanistic approach distinct from all other known therapies in development. Several other agents are in late-stage clinical development for FSGS, targeting alternative pathways including endothelin receptor antagonists and APOL1 inhibitors. Preclinical in vitro data supports potential mechanistic synergy between DMX-200 and endothelin antagonists.
Next Generation Therapies
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.
Strategic Alliances and Arrangements
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 in-licensing or 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.
Consolidated Results of Operations
Three Months Ended September 30, 2025 compared to September 30, 2024
The following table provides selected financial information for the Company:
Three Months Ended September 30,
(in thousands) 2025 2024 Change
Net product sales $ 169,061 $ 141,517 $ 27,544
Cost of goods sold 19,467 13,279 6,188
Gross profit 149,594 128,238 21,356
Operating expenses:
Research and development 23,415 26,160 (2,745)
Selling, general, and administrative 90,036 75,106 14,930
Restructuring charges - 3,143 (3,143)
Depreciation and amortization 1,874 2,170 (296)
Other expense:
Interest income 829 1,081 (252)
Interest expense (11,711) (12,692) 981
Other income (expense) 10,887 (3,263) 14,150
Income tax expense (16,968) (13,514) (3,454)
Net income (loss) attributable to common stockholders $ 17,306 $ (6,729) $ 24,035
Net Product Sales.Net product sales increased $27.5 million during the three months ended September 30, 2025 compared to the same period in the prior year. The increase was due to the continued growth of Galafold®and Pombiliti®+ Opfolda® in Europe and the U.S. and a $3.8 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 $6.2 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.
Research and Development Expense. The following table summarizes our principal development programs and the expenses incurred:
(in thousands) Three Months Ended September 30,
Projects 2025 2024
Third party direct project expenses
Galafold®(Fabry disease)
$ 2,771 $ 2,380
Pombiliti®+ Opfolda®(Pompe disease)
10,145 10,590
Pre-clinical and other programs 413 646
Total third-party direct project expenses 13,329 13,616
Other project costs
Personnel costs 7,467 9,707
Other costs 2,619 2,837
Total other project costs 10,086 12,544
Total research and development costs $ 23,415 $ 26,160
The $2.7 million decrease in research and development costs was primarily driven by lower personnel costs.
Selling, General, and Administrative Expense. Selling, general, and administrative expense increased $14.9 million, primarily driven by higher professional fees to support ongoing legal efforts and increased personnel costs resulting from an increase in headcount to support the supply of our products.
Other Income (Expense). The net change of $14.2 million was primarily related to movement in foreign exchange rates caused by remeasurement of foreign-denominated balances.
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.
Nine Months Ended September 30, 2025 compared to September 30, 2024
The following table provides selected financial information for the Company:
Nine Months Ended September 30, 2025
(in thousands) 2025 2024 Change
Net product sales $ 448,998 $ 378,589 $ 70,409
Cost of goods sold 46,382 38,107 8,275
Gross profit 402,616 340,482 62,134
Operating expenses:
Research and development 112,102 79,172 32,930
Selling, general, and administrative 266,406 236,711 29,695
Restructuring charges - 9,188 (9,188)
Loss on impairment of assets 1,702 - 1,702
Depreciation and amortization 5,563 6,506 (943)
Other expense:
Interest income 2,484 3,991 (1,507)
Interest expense (34,731) (37,640) 2,909
Other income (expense) 12,452 (11,946) 24,398
Income tax expense (25,848) (34,155) 8,307
Net loss attributable to common stockholders $ (28,800) $ (70,845) $ 42,045
Net Product Sales.Net product sales increased $70.4 million during the nine months ended September 30, 2025 compared to the same period in the prior year. The increase was due to the continued growth of Galafold®and Pombiliti®+ Opfolda® in Europe and the U.S. and a $7.7 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 $8.3 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.
Research and Development Expense. The following table summarizes our principal development programs and expenses incurred:
(in thousands) Nine Months Ended September 30, 2025
Projects 2025 2024
Third party direct project expenses
Galafold®(Fabry disease)
$ 9,912 $ 6,420
Pombiliti®+ Opfolda®(Pompe disease)
38,629 31,003
DMX-200 (FSGS kidney disease) 30,000 -
Pre-clinical and other programs 1,238 2,095
Total third-party direct project expenses 79,779 39,518
Other project costs
Personnel costs 24,639 31,287
Other costs 7,684 8,367
Total other project costs 32,323 39,654
Total research and development costs $ 112,102 $ 79,172
The $32.9 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 $29.7 million, primarily driven by $13.6 million of higher personnel costs resulting from an increase in headcount to support the supply of our products, $7.2 million of higher professional fees to support ongoing legal efforts, and $5.7 million of higher expense in connection with our continuing expansion of manufacturing capabilities.
Restructuring Charges. In the first quarter of 2024, restructuring charges were primarily related to an initiative to reduce operating costs by abandoning a lease that was no longer useful in our operations.
Other Income (Expense). The net change of $24.4 million was primarily related to movement in foreign exchange rates caused by remeasurement of foreign-denominated balances.
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.
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.
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 an aggregate offering gross proceeds of up to $250.0 million. As of September 30, 2025, an aggregate of $164.2 million worth of shares remain available to be issued and sold under the ATM program.
Cash Flow Discussion
As of September 30, 2025, we had cash, cash equivalents, and marketable securities of $263.8 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 (Used in) Operating Activities
Net cash provided by operations for the nine months ended September 30, 2025 was $16.9 million. The components of net cash provided by operations primarily reflect net loss of $28.8 million adjusted for non-cash expenses of $78.6 million, which includes $60.8 million of stock compensation and $5.6 million of depreciation expense. This is further driven by a net increase in operating assets and liabilities of $32.9 million. The changes in operating assets and liabilities were primarily driven by an increase in inventory of $67.2 million to support our continued commercial growth and an increase in other non-current assets and liabilities of $4.3 million, partially offset by a $44.6 million increase in accounts payable and accrued expenses.
Net cash used in operations for the nine months ended September 30, 2024 was $30.0 million. The components of net cash used in operations included the net loss for the nine months ended September 30, 2024 of $70.8 million and a net decrease in changes in operating assets and liabilities of $55.2 million, offset by $65.7 million of stock compensation and $30.4 million of other non-cash adjustments. The changes in operating assets and liabilities were primarily due to an increase in inventory of $61.6 millionto support our continued commercial growth and an increase in accounts receivable of $9.1 million, partially offset by a decrease in prepaid expenses and other current assets of $28.2 million anda decrease in accounts payable and accrued expenses of $11.5 millionassociated with timing of payments.
Net Cash Used in (Provided by) Investing Activities
Net cash used in investing activities for the nine months ended September 30, 2025 was $39.9 million. Our investing activities have consisted primarily of purchases, sales, and maturities of investments and capital expenditures. Net cash used in investing activities reflects $71.4 million for the purchase of marketable securities, partially offset by $34.4 million from the sale and redemption of marketable securities and $3.0 million for capital expenditures.
Net cash provided by investing activities for the nine months ended September 30, 2024 was $19.8 million. Our investing activities have consisted primarily of purchases, sales and maturities of investments and capital expenditures. Net cash provided by investing activities reflects $101.9 million from the sale and redemption of marketable securities, partially offset by $78.8 million for the purchase of marketable securities and $3.3 million for capital expenditures.
Net Cash Used in (Provided by) Financing Activities
Net cash used in financing activities for the nine months ended September 30, 2025 was $13.4 million. Net cash used in financing activities primarily reflects withholding taxes paid on vested restricted stock units of $13.9 million.
Net cash provided by financing activities for the nine months ended September 30, 2024 was $4.0 million. Net cash provided by financing activities primarily reflects $19.2 million of proceeds from the issuance of shares in connection with the ATM program offering, net of issuance costs, and $6.1 million of proceeds from the exercise of stock options, partially offset by the withholding taxes paid on vested restricted stock units of $21.2 million.
Funding Requirements
We expect to continue incurring 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 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 future results of preclinical research and subsequent clinical trials for 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 or other trade restrictions, 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 or commercial products, including 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;
•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.
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.
Critical Accounting Policies and Significant Judgments
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 nine months ended September 30, 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.
Recent Accounting Pronouncements
Please refer to "-Note 2. Summary of Significant Accounting Policies" in our Notes to Consolidated Financial Statements.
Amicus Therapeutics Inc. published this content on November 04, 2025, and is solely responsible for the information contained herein. Distributed via Edgar on November 04, 2025 at 21:04 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]