8-K
AMICUS THERAPEUTICS, INC. (FOLD)
UNITEDSTATES
SECURITIESAND EXCHANGE COMMISSION
WASHINGTON,D.C. 20549
FORM
8-K
CURRENTREPORT PURSUANT TO
SECTION13 OR 15(d) OF THE
SECURITIES EXCHANGE ACT OF 1934
Date of Report (Date of earliest event reported): November 4, 2025
AMICUS THERAPEUTICS, INC.
(Exact Name of Registrant as Specified in Its Charter)
| Delaware | 001-33497 | 71-0869350 |
|---|---|---|
| (State or Other Jurisdiction of Incorporation) | (Commission File Number) | (I.R.S. Employer Identification No.) |
47 Hulfish Street,
Princeton, New Jersey 08542
(Address of PrincipalExecutive Offices, and Zip Code)
609-662-2000
Registrant’sTelephone Number, Including Area Code
(Former Name or Former Address, if Changed Since Last Report.)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
| ¨ | Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
|---|---|
| ¨ | Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
| ¨ | Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
| ¨ | Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading Symbol(s) | Name of each exchange on which registered |
|---|---|---|
| Common Stock Par Value $0.01 | FOLD | NASDAQ |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (17 CFR §230.405) or Rule 12b-2 of the Securities Exchange Act of 1934 (17 CFR §240.12b-2). Emerging growth company ¨
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ¨
Item 2.02 Results of Operations and FinancialCondition.
On November 4, 2025, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the fiscal quarter ended September 30, 2025. A copy of this press release is attached hereto as Exhibit 99.1. The Company will host a conference call and webcast on November 4, 2025 to discuss its third quarter results of operations. A copy of the conference call presentation materials is attached hereto as Exhibit 99.2. Both exhibits are incorporated herein by reference.
In accordance with General Instruction B.2. of Form 8-K, the information in this Current Report on Form 8-K and the Exhibits shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liability of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits:
| Exhibit No. | Description |
|---|---|
| 99.1 | Press Release dated November 4, 2025 |
| 99.2 | November 4, 2025 Conference Call Presentation Materials |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) |
Signature Page
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| AMICUS THERAPEUTICS, INC. | ||
|---|---|---|
| Date: November 4, 2025 | By: | /s/ Ellen S. Rosenberg |
| Name: | Ellen S. Rosenberg | |
| Title: | Chief Legal Officer and Corporate Secretary |
Exhibit 99.1
Amicus Therapeutics Announces Third Quarter2025Financial Results and Corporate Updates
Q3 2025 Total Revenue of $169M, up 17% atCER
Positive Q3 2025 GAAP Net Income of $17M
Cash Position of$264M, a $33M Increase in Q3 2025
Conference Call and Webcast Today at 8:30a.m. ET
PRINCETON, NJ,Nov. 4, 2025 – Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced financial results for the quarter ended September 30, 2025.
“Amicus achieved yet another impressive quarter, delivering revenue growth of 17 percent and reaching the milestone of GAAP profitability. We served more patients than ever before, driven by new Galafold starts and growing adoption for Pombiliti + Opfolda,” said Bradley Campbell, President and Chief Executive Officer, Amicus Therapeutics Inc. “Alongside this commercial momentum, enrollment in the Phase 3 study of DMX-200 remains on track for completion by year-end. Through our continued execution, Amicus is well positioned to achieve our 2025 strategic objectives and deliver sustainable double-digit growth in the years ahead.”
Third Quarter 2025 Financial Highlights:
| · | Total revenues for the third quarter 2025 were $169.1 million, reflecting strong operational<br> growth measured at constant exchange rates (CER)^1^ of 17% and a currency tailwind<br> of ~$4 million. | |||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| (in thousands) | Three Months Ended<br> September 30, | Year over Year %<br> Growth | Nine Months Ended<br> September 30, | Year over Year %<br> Growth | ||||||||||||||||
| --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- |
| 2025 | 2024 | Reported | at CER^1^ | 2025 | 2024 | Reported | at CER^1^ | |||||||||||||
| Galafold^®^ | $ | 138,347 | $ | 120,381 | 15 | % | 12 | % | $ | 371,463 | $ | 330,557 | 12 | % | 10 | % | ||||
| Pombiliti^®^ + Opfolda^®^ | $ | 30,714 | $ | 21,136 | 45 | % | 42 | % | $ | 77,535 | $ | 48,032 | 61 | % | 59 | % | ||||
| Net Product Revenues | $ | 169,061 | $ | 141,517 | 19 | % | 17 | % | $ | 448,998 | $ | 378,589 | 19 | % | 17 | % | ||||
| · | Galafold (migalastat) net product sales for the third quarter 2025 were $138.3 million, representing<br> a year-over-year increase of 15%, or 12% at CER^1^, driven by continued commercial<br> execution in all markets, net new patient starts, and strong compliance. | |||||||||||||||||||
| --- | --- | |||||||||||||||||||
| · | Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) net product sales for the third quarter 2025 were $30.7 million, representing a year-over-year increase of 45%, or 42% at CER^1^,<br> driven by high commercial demand and new launch countries. | |||||||||||||||||||
| --- | --- | |||||||||||||||||||
| · | Total GAAP operating expenses of $115.3 million for the third quarter 2025 increased by 8%<br> as compared to $106.6 million for the third quarter 2024. Total non-GAAP operating expenses^2^<br> were up 15% to $95.4 million for the third quarter 2025 as compared to $82.6 million for<br> the third quarter 2024. | |||||||||||||||||||
| --- | --- | |||||||||||||||||||
| · | GAAP net income of $17.3 million, or $0.06 per share basic and diluted, was achieved in the<br> third quarter 2025, compared to a GAAP net loss of $6.7 million, or $0.02 per share basic<br> and diluted, for the third quarter 2024. Non-GAAP net income^2,3^<br> was $54.2 million, or $0.18 per share basic and $0.17 diluted, for the third quarter 2025,<br> compared to non-GAAP net income of $30.8 million, or $0.10 per share basic and diluted, for<br> the third quarter 2024. | |||||||||||||||||||
| --- | --- | |||||||||||||||||||
| · | Cash, cash equivalents, and marketable securities increased to $263.8 million at September 30,<br> 2025, as compared to $249.9 million at December 31, 2024. | |||||||||||||||||||
| --- | --- |
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Corporate Updates:
| · | Pombiliti + Opfolda pricing and reimbursement agreements recently completed in four countries, including Japan and Belgium. The Company remains on track for up to 10 new launch countries in<br> 2025. |
|---|---|
| · | New 4-year data from the PROPEL open-label extension (OLE) adds to growing body of evidence for Pombiliti + Opfolda**.** As<br> previously announced, in this new analysis presented at the International Congress of Inborn<br> Errors of Metabolism (ICIEM), patients within the ERT-experienced group showed stability<br> or improvement on measures of muscle function, muscle strength, and biomarkers out to 4 years. |
| --- | --- |
| · | During<br> the third quarter, our manufacturing partner, WuXi Biologics, announced that its Dundalk, Ireland facility has received approval from European Medicines Agency (EMA) as a commercial<br> manufacturing site for Pombiliti. |
| --- | --- |
| · | ACTION3 Study of DMX-200 is on track for full enrollment by end of year. DMX-200 is a first in<br> class treatment for Focal Segmental Glomerulosclerosis (FSGS), a rare and fatal kidney disease<br> with no approved therapies and significant market potential. The ongoing Phase 3 study (ACTION3),<br> funded and executed by Dimerix, continues to be on track for full enrollment by end of 2025. |
| --- | --- |
| · | Amicus is focused on delivering significant long-term revenue growth and anticipates surpassing $1 billion in total sales in 2028. The Company anticipates<br> continuing to grow its current commercial business with Galafold and Pombiliti + Opfolda resulting in strong total revenue<br> growth. |
| --- | --- |
2025 Financial Guidance
Amicus reiterates its financial guidance for 2025, as follows:
| Total Revenue Growth**^1^** | 15% to 22% |
|---|---|
| Galafold Revenue Growth**^1^** | 10% to 15% |
| Pombiliti + Opfolda Revenue Growth**^1^** | 50% to 65% |
| Gross Margin | Mid 80% |
| Non-GAAP Operating Expenses**^4^** | $380M to $400M |
| GAAP Net Income | Positive during H2 2025 |
^1^In order to illustrate underlying performance, Amicus discusses its results in terms of constant exchange rate (CER) growth. This represents growth calculated as if the exchange rates had remained unchanged from those used in the comparative period.
^2^Full reconciliation of GAAP results to the Company’s non-GAAP adjusted measures for all reporting periods appear in the tables to this press release.
^3^Amicus defines non-GAAP Net (Loss) Income as GAAP Net (Loss) Income excluding the impact of share-based compensation expense, changes in fair value of contingent consideration, loss on impairment of assets, depreciation and amortization, acquisition related income (expense), loss on extinguishment of debt, restructuring charges and income taxes.
^4^A reconciliation of the differences between the non-GAAP expectation and the corresponding GAAP measure is not available without unreasonable effort due to high variability, complexity, and low visibility as to the items that would be excluded from the GAAP measure.
Conference Call and Webcast
Amicus Therapeutics will host a conference call and audio webcast today, November 4, 2025, at 8:30 a.m. ET to discuss the third quarter 2025 financial results and corporate updates. Participants and investors interested in accessing the call by phone will need to register using the online registration form. After registering, all phone participants will receive a dial-in number along with a personal PIN to access the event.
A live audio webcast and related presentation materials can also be accessed via the Investors section of the Amicus Therapeutics corporate website at ir.amicusrx.com. Web participants are encouraged to register on the website 15 minutes prior to the start of the call. An archived webcast and accompanying slides will be available on the Company's website shortly after the conclusion of the live event.
About Galafold
Galafold^®^ (migalastat) 123 mg capsules is an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) for the treatment of Fabry disease in adults who have amenable galactosidase alpha gene (GLA) variants. In these patients, Galafold works by stabilizing the body’s own dysfunctional enzyme so that it can clear the accumulation of disease substrate. Globally, Amicus Therapeutics estimates that approximately 35 to 50 percent of people living with Fabry disease may have amenable GLA variants, though amenability rates within this range vary by geography. Galafold is approved in more than 40 countries around the world, including the U.S., EU, U.K., and Japan.
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U.S. INDICATIONS AND USAGE
Galafold is indicated 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.
This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
U.S. IMPORTANT SAFETY INFORMATION
**ADVERSE REACTIONS:**The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia. USE IN SPECIFIC POPULATIONS: There is insufficient clinical data on Galafold use in pregnant women to inform a drug-associated risk for major birth defects and miscarriage. Advise women of the potential risk to a fetus. It is not known if Galafold is present in human milk. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition. Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis. The safety and effectiveness of Galafold have not been established in pediatric patients. To report Suspected Adverse Reactions, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. For additional information aboutGalafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf.
About Pombiliti + Opfolda
Pombiliti + Opfolda, is a two-component therapy that consists of cipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that’s designed to reduce loss of enzyme activity in the blood.
U.S. INDICATIONS AND USAGE
POMBILITI in combination with OPFOLDA is indicated for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).
SAFETY INFORMATION
HYPERSENSITIVITYREACTIONS INCLUDING ANAPHYLAXIS: Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readilyavailable. If a severe hypersensitivity reaction occurs, POMBILITI should be discontinued immediately and appropriate medical treatmentshould be initiated. INFUSION-ASSOCIATED REACTIONS (IARs): If severe IARs occur, immediately discontinue POMBILITI and initiate appropriatemedical treatment. RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients susceptible to fluid volume overload, orthose with acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of serious exacerbationof their cardiac or respiratory status during POMBILITI infusion. See PI for complete Boxed Warning. CONTRAINDICATION: POMBILITI in combination with Opfolda is contraindicated in pregnancy. EMBRYO-FETAL TOXICITY: May cause embryo-fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose. Adverse Reactions: Most common adverse reactions ≥ 5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia. Please see full PRESCRIBING INFORMATION, including BOXED WARNING, for POMBILITI(cipaglucosidase alfa-atga) LINK and full PRESCRIBING INFORMATION for OPFOLDA (miglustat) LINK.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information please visit the company’s website at www.amicusrx.com, and follow on LinkedIn.
Non-GAAP Financial Measures
In addition to financial information prepared in accordance with U.S. GAAP, this press release also contains adjusted financial measures that we believe provide investors and management with supplemental information relating to operating performance and trends that facilitate comparisons between periods and with respect to projected information. These adjusted financial measures are non-GAAP measures and should be considered in addition to, but not as a substitute for, the information prepared in accordance with U.S. GAAP. We use these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. We typically exclude certain GAAP items that management does not believe affect our basic operations and that do not meet the GAAP definition of unusual or non-recurring items. Other companies may define these measures in different ways. When we provide our expectation for non-GAAP operating expenses and profitability on a forward-looking basis, a reconciliation of the differences between the non-GAAP expectation and the corresponding GAAP measure generally is not available without unreasonable effort due to potentially high variability, complexity and low visibility as to the items that would be excluded from the GAAP measure in the relevant future period, such as unusual gains or losses. The variability of the excluded items may have a significant, and potentially unpredictable, impact on our future GAAP results.
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Forward Looking Statement
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to the prospects and timing of the potential regulatory and pricing approval of our products, commercialization plans, manufacturing and supply plans, financing plans, the collaboration with Dimerix, and the projected revenues and cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities and pricing and reimbursement authorities, are based on current information. Actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that regulatory authorities may not grant or may delay approval for our product candidates; the potential that required regulatory inspections may be delayed or not be successful and delay or prevent product approval; the potential that we may not be successful in negotiations with pricing and reimbursement authorities; the potential that we may not be successful in commercializing Galafold and/or Pombiliti and Opfolda in Europe, the UK, the US and other geographies; the potential that the Dimerix license agreement for of DMX-200 may not be successful, including without limitation expectations of the timing of the Phase 3 clinical trial evaluating DMX-200; the likelihood of success of such clinical trial; the prospects for FDA approval of DMX-200 for FSGS or other indications; the estimated prevalence of FSGS; the achievement of any milestone and timing of any payments associated with milestones and the success of any efforts to commercialize DMX-200, including any projections of future financial performance or payments; the potential that we may not be able to manufacture or supply sufficient commercial products; and the potential that we will need additional funding to complete the manufacturing and commercialization of our products. With respect to statements regarding corporate financial guidance and financial goals and the expected attainment of such goals and projections of the Company's revenue, GAAP and non-GAAP profitability and cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2024 and our Quarterly Report on Form 10-Q to be filed today. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.
CONTACT:
Investors:
Amicus Therapeutics
Andrew Faughnan
Vice President, Investor Relations
afaughnan@amicusrx.com
(609) 662-3809
Media:
Amicus Therapeutics
Brendan McEvoy
Executive Director, External Communications
bmcevoy@amicusrx.com
(609) 662-5005
FOLD-G
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TABLE 1
Amicus Therapeutics, Inc.
Consolidated Statements of Operations
(Unaudited)
(in thousands, except share and per share amounts)
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 2025 | 2024 | 2025 | 2024 | |||||||||
| Net product sales | $ | 169,061 | $ | 141,517 | $ | 448,998 | $ | 378,589 | ||||
| Cost of goods sold | 19,467 | 13,279 | 46,382 | 38,107 | ||||||||
| Gross profit | 149,594 | 128,238 | 402,616 | 340,482 | ||||||||
| Operating expenses: | ||||||||||||
| Research and development | 23,415 | 26,160 | 112,102 | 79,172 | ||||||||
| Selling, general, and administrative | 90,036 | 75,106 | 266,406 | 236,711 | ||||||||
| Restructuring charges | — | 3,143 | — | 9,188 | ||||||||
| Loss on impairment of assets | — | — | 1,702 | — | ||||||||
| Depreciation and amortization | 1,874 | 2,170 | 5,563 | 6,506 | ||||||||
| Total operating expenses | 115,325 | 106,579 | 385,773 | 331,577 | ||||||||
| Income from operations | 34,269 | 21,659 | 16,843 | 8,905 | ||||||||
| Other expense: | ||||||||||||
| Interest income | 829 | 1,081 | 2,484 | 3,991 | ||||||||
| Interest expense | (11,711 | ) | (12,692 | ) | (34,731 | ) | (37,640 | ) | ||||
| Other income (expense) | 10,887 | (3,263 | ) | 12,452 | (11,946 | ) | ||||||
| Income (loss) before income tax | 34,274 | 6,785 | (2,952 | ) | (36,690 | ) | ||||||
| Income tax expense | (16,968 | ) | (13,514 | ) | (25,848 | ) | (34,155 | ) | ||||
| Net income (loss) attributable to common stockholders | $ | 17,306 | $ | (6,729 | ) | $ | (28,800 | ) | $ | (70,845 | ) | |
| Net income (loss) attributable to common stockholders per common share — basic | $ | 0.06 | $ | (0.02 | ) | $ | (0.09 | ) | $ | (0.23 | ) | |
| Net income (loss) attributable to common stockholders per common share — diluted | 0.06 | (0.02 | ) | (0.09 | ) | (0.23 | ) | |||||
| Weighted-average common shares outstanding — basic | 308,468,423 | 304,690,596 | 308,139,134 | 303,792,479 | ||||||||
| Weighted-average common shares outstanding — diluted | 310,433,494 | 304,690,596 | 308,139,134 | 303,792,479 |
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TABLE 2
Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
(Unaudited)
| December 31, 2024 | |||||
|---|---|---|---|---|---|
| Assets | |||||
| Current assets: | |||||
| Cash and cash equivalents | 190,553 | $ | 213,752 | ||
| Investments in marketable securities | 73,290 | 36,194 | |||
| Accounts receivable | 113,838 | 101,099 | |||
| Inventories | 177,928 | 118,782 | |||
| Prepaid expenses and other current assets | 38,457 | 34,909 | |||
| Total current assets | 594,066 | 504,736 | |||
| Operating lease right-of-use assets, net | 21,549 | 22,278 | |||
| Property and equipment, less accumulated depreciation of 31,271 and 28,775 at September 30, 2025 and December 31, 2024, respectively | 27,759 | 29,383 | |||
| Intangible assets, less accumulated amortization of 8,257 and 5,802 at September 30, 2025 and December 31, 2024, respectively | 14,743 | 17,198 | |||
| Goodwill | 197,797 | 197,797 | |||
| Other non-current assets | 12,897 | 13,641 | |||
| Total Assets | 868,811 | $ | 785,033 | ||
| Liabilities and Stockholders’ Equity | |||||
| Current liabilities: | |||||
| Accounts payable | 19,103 | $ | 12,947 | ||
| Accrued expenses and other current liabilities | 171,165 | 127,300 | |||
| Operating lease liabilities | 8,662 | 8,455 | |||
| Total current liabilities | 198,930 | 148,702 | |||
| Long-term debt | 391,985 | 390,111 | |||
| Operating lease liabilities | 42,174 | 45,078 | |||
| Other non-current liabilities | 5,298 | 7,097 | |||
| Total liabilities | 638,387 | 590,988 | |||
| Commitments and contingencies | |||||
| Stockholders’ equity: | |||||
| Common stock, 0.01 par value, 500,000,000 shares authorized, 308,499,614 and 299,041,653 shares issued and outstanding at September 30, 2025 and December 31, 2024, respectively | 3,017 | 2,944 | |||
| Common stock in treasury, at cost; 7,390 shares as of September 30, 2025 | (71 | ) | — | ||
| Additional paid-in capital | 2,973,625 | 2,926,115 | |||
| Accumulated other comprehensive income (loss): | |||||
| Foreign currency translation adjustment | 22,886 | 5,302 | |||
| Unrealized loss on available-for-sale securities | (53 | ) | (207 | ) | |
| Warrants | — | 71 | |||
| Accumulated deficit | (2,768,980 | ) | (2,740,180 | ) | |
| Total stockholders’ equity | 230,424 | 194,045 | |||
| Total Liabilities and Stockholders’ Equity | 868,811 | $ | 785,033 |
All values are in US Dollars.
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TABLE 3
Amicus Therapeutics, Inc.
Reconciliation of Non-GAAP Financial Measures
(in thousands)
(Unaudited)
| Three Months Ended<br> September 30, | Nine Months Ended<br> September 30, | |||||||
|---|---|---|---|---|---|---|---|---|
| 2025 | 2024 | 2025 | 2024 | |||||
| Total operating expenses - as reported GAAP | $ | 115,325 | $ | 106,579 | $ | 385,773 | $ | 331,577 |
| Research and development: | ||||||||
| Share-based compensation | 2,368 | 4,397 | 8,765 | 12,329 | ||||
| Selling, general and administrative: | ||||||||
| Share-based compensation | 15,721 | 14,291 | 52,055 | 53,359 | ||||
| Loss on impairment of assets | — | — | 1,702 | — | ||||
| Restructuring Charges | — | 3,143 | — | 9,188 | ||||
| Depreciation and amortization | 1,874 | 2,170 | 5,563 | 6,506 | ||||
| Total operating expense adjustments to reported GAAP | 19,963 | 24,002 | 68,085 | 81,382 | ||||
| Total operating expenses - as adjusted | $ | 95,362 | $ | 82,577 | $ | 317,688 | $ | 250,195 |
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TABLE 4
Amicus Therapeutics, Inc.
Reconciliation of Non-GAAP Financial Measures
(in thousands, except share and per share amounts)
(Unaudited)
| Three Months Ended<br> September 30, | Nine Months Ended<br> September 30, | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| 2025 | 2024 | 2025 | 2024 | ||||||||
| GAAP net income (loss) | $ | 17,306 | $ | (6,729 | ) | $ | (28,800 | ) | $ | (70,845 | ) |
| Share-based compensation | 18,089 | 18,688 | 60,820 | 65,688 | |||||||
| Depreciation and amortization | 1,874 | 2,170 | 5,563 | 6,506 | |||||||
| Loss on impairment of assets | — | — | 1,702 | — | |||||||
| Restructuring charges | — | 3,143 | — | 9,188 | |||||||
| Income tax expense | 16,968 | 13,514 | 25,848 | 34,155 | |||||||
| Non-GAAP net income | $ | 54,237 | $ | 30,786 | $ | 65,133 | $ | 44,692 | |||
| Non-GAAP net income attributable to common stockholders per common share — basic | $ | 0.18 | $ | 0.10 | $ | 0.21 | $ | 0.15 | |||
| Non-GAAP net income attributable to common stockholders per common share — diluted | $ | 0.17 | $ | 0.10 | $ | 0.21 | $ | 0.15 | |||
| Weighted-average common shares outstanding — basic | 308,468,423 | 304,690,596 | 308,139,134 | 303,792,479 | |||||||
| Weighted-average common shares outstanding — diluted | 310,433,494 | 304,690,596 | 308,139,134 | 303,792,479 |
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Exhibit 99.2
| AT THE FOREFRONT OF<br>THERAPIES FOR RARE DISEASES<br>Q3 2025 Results<br>Conference Call<br>& Webcast<br>November 4, 2025 |
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| 2<br>Forward-Looking Statements<br>This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to the prospects and timing of the potential<br>regulatory and pricing approval of our products, commercialization plans, manufacturing and supply plans, financing plans, the collaboration with Dimerix, and the projected revenues<br>and cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the<br>forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and<br>uncertainties. For example, statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities and pricing and reimbursement authorities, are<br>based on current information. Actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without<br>limitation: the potential that regulatory authorities may not grant or may delay approval for our product candidates; the potential that required regulatory inspections may be delayed<br>or not be successful and delay or prevent product approval; the potential that we may not be successful in negotiations with pricing and reimbursement authorities; the potential that we<br>may not be successful in commercializing Galafold and/or Pombiliti and Opfolda in Europe, the UK, the US and other geographies; the potential that the Dimerix license agreement for<br>of DMX-200 may not be successful, including without limitation expectations of the timing of the Phase 3 clinical trial evaluating DMX-200; the likelihood of success of such clinical trial;<br>the prospects for FDA approval of DMX-200 for FSGS or other indications; the estimated prevalence of FSGS; the achievement of any milestone and timing of any payments associated<br>with milestones and the success of any efforts to commercialize DMX-200, including any projections of future financial performance or payments; the potential that we may not be able<br>to manufacture or supply sufficient commercial products; and the potential that we will need additional funding to complete the manufacturing and commercialization of our products.<br>With respect to statements regarding corporate financial guidance and financial goals and the expected attainment of such goals and projections of the Company's revenue, GAAP and<br>non-GAAP profitability and cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all<br>forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2024 and our Quarterly Report on Form 10-Q to be<br>filed today. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in<br>their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.<br>Non-GAAP Financial Measures<br>In addition to financial information prepared in accordance with U.S. GAAP, this presentation also contains adjusted financial measures that we believe provide investors and<br>management with supplemental information relating to operating performance and trends that facilitate comparisons between periods and with respect to projected information. These<br>adjusted financial measures are non-GAAP measures and should be considered in addition to, but not as a substitute for, the information prepared in accordance with U.S. GAAP. We<br>typically exclude certain GAAP items that management does not believe affect our basic operations and that do not meet the GAAP definition of unusual or non-recurring items. Other<br>companies may define these measures in different ways. When we provide our expectation for non-GAAP operating expenses on a forward-looking basis, a reconciliation of the<br>differences between the non-GAAP expectation and the corresponding GAAP measure generally is not available without unreasonable effort due to potentially high variability,<br>complexity and low visibility as to the items that would be excluded from the GAAP measure in the relevant future period, such as unusual gains or losses. The variability of the excluded<br>items may have a significant, and potentially unpredictable, impact on our future GAAP results. |
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| A Rare Company<br>3 1 At CER: Constant Exchange Rates<br>$169M<br>Q3 2025 Total Revenue<br>(+17% Growth)1<br>$1B+<br>Total Revenue<br>Expected in FY 2028<br>First Oral<br>Precision<br>Medicine for<br>Fabry Disease<br>10-15%<br>FY 2025<br>Galafold Revenue<br>Growth1<br>Expanded<br>Portfolio with<br>U.S. Licensing of<br>DMX-200<br>Phase 3 Program<br>Leverageable<br>Global<br>Commercial<br>Organization<br>First Two-Component Therapy<br>for Pompe Disease 50-65%<br>FY 2025<br>Pombiliti + Opfolda<br>Revenue Growth1<br>A unique story in biotech with significant revenue growth and profitability |
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| 4<br>Galafold® (migalastat)<br>Continued Growth<br>Building a leadership position<br>in the treatment of Fabry disease |
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| 5<br>Only approved oral treatment in Fabry disease and standard of care for amenable patients<br>2025 Galafold Success (as of September 30, 2025)<br>Galafold is indicated for adults with a confirmed diagnosis of Fabry disease and an amenable variant. The most common adverse reactions reported with Galafold (≥10%)<br>were headache, nasopharyngitis, urinary tract infection, nausea, and pyrexia. For additional information about Galafold, including the full U.S. Prescribing Information, please<br>visit https://amicusrx.com//pi/galafold.pdf. For further important safety information for Galafold, including posology and method of administration, special warnings,<br>drug interactions, and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.<br>A unique mechanism of action for<br>Fabry patients with amenable variants<br>35-50%<br>Fabry Patients<br>Amenable to<br>Galafold 40+<br>Countries with<br>Regulatory<br>Approvals<br>~2,730<br>Individuals<br>Treated1<br>$138.3M<br>Q3 25 Galafold<br>Revenue +15%<br>Q3 25 Reported<br>Sales Growth<br>69%<br>Share of Treated<br>Amenable Patients<br>1 As of YE 2024 |
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| 6<br>FY23 FY24 FY25<br>$388M<br>$458M<br> Quarterly patient starts remain strong: +13% growth<br>YoY in Q3 2025<br> Highest demand quarter in Q3 2025 and best number<br>of starts YTD since launch<br> Global mix of naïve (~65%) and switch (~35%)<br>patients2<br> Expanding market through uptake in naïve<br>population as well as label and geographic expansion<br> Maintaining >90% adherence and compliance<br>through HCP and patient education and support<br>Galafold Performance<br>Reiterating FY 2025 Galafold growth guidance of 10-15% at CER<br>Q1<br>$99M<br>Q2<br>$111M<br>Q3<br>$120M<br>Q4<br>$128M<br>+10-15%1<br>Q3 2025 Galafold reported revenue of $138.3M (+12% at CER)<br>1 CER: Constant Exchange Rates<br>2 Data on file<br>Q1<br>$104M<br>Q2<br>$129M<br>Q3<br>$138M |
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| 7<br>Fabry Market: Significant Remaining Unmet Need<br>7<br>Based on publicly reported sales of Fabry therapies<br>1Applying incidence of multiple newborn screening studies to population estimates in commercial markets:<br>Burton 2017 J Pediatr 2017;190:130-5 ; Mechtler et al., The Lancet, 2011 Dec.; Hwu et al., Hum Mutation, 2009 Jun; Spada et al., Am J Human Genet., 2006 Jul<br>7<br>Research suggests there could be >100k people living with Fabry disease who remain undiagnosed<br>U.S.<br>37%<br>Europe<br>36%<br>ROW<br>27%<br>Treated Population<br>(>12,0001)<br>Diagnosed Untreated<br>(~6,0001)<br>Undiagnosed<br>(Est. >100,0001) |
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| 8<br>New FINDING FABRY website & campaign now live<br>New FINDING FABRY Initiative in the U.S.<br>https://findingfabry.com/ |
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| 9<br>Pombiliti® (cipaglucosidase alfa-atga)<br><br>Opfolda® (miglustat)<br>Potential to establish a new standard of care<br>for people living with late-onset Pompe disease<br>+ |
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| 10<br>FY23 FY24 FY25<br>Pombiliti + Opfolda Performance<br>Q3 2025 Pombiliti + Opfolda reported revenue of $30.7M (+42% at CER)<br>Q1<br>$11M<br>Q3<br>$21M<br>Q4<br>$22M<br>1 CER: Constant Exchange Rates<br>2 Real-World Evidence<br>$70.2M<br>+50-65%1<br>Q2<br>$16M Q1<br>$21M<br> Strong Q3 sales growth YoY<br>– Increasing depth and breadth of prescribers<br>– Doubled number of naïve starts (ex-U.S) in first 9<br>months of 2025 vs FY 2024<br>– Doubled number of avalglucosidase alfa switches<br>in first 9 months of 2025 vs FY 2024<br> H2 2025 growth driven by:<br>– Benefit of new patient starts in existing and newly<br>launched markets<br>– Growing body of RWE2 supporting switch from<br>both alternative therapies<br>Reiterating FY 2025 Pombiliti + Opfolda growth guidance of 50-65% at CER1<br>Q2<br>$25M<br>$11.6M<br>Q3<br>$31M |
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| 11<br>Significant progress made towards expanding access to Pombiliti + Opfolda in 2025<br>Pombiliti + Opfolda Expansion<br> Approved in Australia and Canada in Q1 2025<br> Approved in Japan in June 2025<br>AUSTRALIA CANADA JAPAN<br>Regulatory Reimbursement<br> Now reimbursed in 15 countries with 10 countries added<br>in 2025 providing access to ~2,400 treated patients<br> First commercial patients in 8 new countries YTD<br> Pombiliti + Opfolda selected as preferred treatment<br>for adults with LOPD in the Netherlands<br>SWEDEN ITALY<br>NETHERLANDS PORTGUAL<br>Reimbursement agreements completed in 2025:<br>CZECH BELGIUM<br>REPUBLIC<br>SWITZERLAND JAPAN IRELAND<br>LUXEMBOURG<br>Regulatory approvals in 2025: |
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| 12<br>Growing number of abstracts, manuscripts, and case studies supporting<br>Pombiliti + Opfolda differentiation<br>Pombiliti + Opfolda Body of Evidence<br>Case Studies & Real-World Reports<br>Mechanistic & Translational Insights<br> Long-term Phase 1/2 open-label safety and efficacy study (ATB200-02)<br> 104-week Phase 3 open-label extension study of efficacy and safety (ATB200-07)<br>Clinical Trials & Long-Term Data<br>Comparative & Real-World Data<br> Miglustat: a first-in-class enzyme stabilizer for LOPD<br> Linking mechanism of action to clinical outcomes in LOPD<br> Network meta-analysis comparing the efficacy of cipaglucosidase alfa + miglustat with other ERTs<br> U.K. EAMS1 registry post-baseline outcomes<br> CDMU–UCLH2 cohort analysis comparing cipaglucosidase alfa + miglustat vs. avalglucosidase alfa<br> Case studies supporting the switch from both alternative therapies<br> Case studies of patients switching from high dose, high frequency alglucosidase alfa<br>1 EAMS: Early Access to Medicines Scheme<br>2 Dr. Robin Lachmann - Charles Dent Metabolic Unit, University College London Hospitals NHS Foundation Trust, London, UK |
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| 13<br>Real-world data recently presented by independent researchers<br>Emerging Pombiliti + Opfolda Real-World Evidence<br>cipaglucosidase alfa+<br>miglustat vs.<br>alglucosidase alfa<br>avalglucosidase alfa<br>vs. alglucosidase alfa<br>6MWD Significant<br>increase<br>Non-significant<br>decrease<br>10MWT Significant<br>decrease (faster)<br>Non-significant<br>increase (slower)<br>FVC Non-significant<br>increase<br>Non-significant<br>increase<br>CK Non-significant<br>decrease<br>Significant<br>decrease<br>Summary of adjusted models:<br>1 Lucas Del Pozo et al. Poster presented Dr Robin Lachmann at the International Congress of Inborn Errors of Metabolism (ICIEM); Kyoto, Japan; September 2–6, 2025; Poster P-357 2 Indirect longitudinal comparison of adults with LOPD switching from alglucosidase alfa to cipaglucosidase + miglustat (n = 13) or avalglucosidase alfa (n = 15); regression-adjusted for baseline and fixed effects. |
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| 14<br>DMX-200<br>Potential first-in-class investigational small molecule<br>for the treatment of FSGS in the U.S. |
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| 15<br> Irreversible scarring leads to permanent kidney<br>damage and eventual end-stage renal failure1<br> Symptoms include proteinuria, edema, high<br>cholesterol and blood pressure, low albumin<br>levels<br> Average time from diagnosis to onset of<br>complete kidney failure is typically five to ten<br>years2<br> FSGS kidney damage can lead to dialysis,<br>kidney transplants, or death<br>Focal Segmental<br>Glomerulosclerosis (FSGS)<br>is a rare disease leading to<br>irreversible kidney damage<br>Pathogenic Feedback<br>Loop in FSGS<br>3<br>Fibrosis causes<br>loss of kidney<br>cells (cannot<br>regenerate)<br>2<br>Constant<br>pressure causes<br>inflammation of<br>kidney cells and<br>subsequent<br>scarring/fibrosis<br>1<br>High blood<br>pressure causes<br>hyperfiltration<br>within blood<br>vessels of the<br>kidney<br>Kidney vessels have to work harder<br>under high pressure<br>Existing blood pressure<br>medication targets<br>angiotensin receptor<br>blocker (ARB) lowers<br>blood pressure<br>Less kidney<br>cells cause further<br>hyperfiltration and<br>inflammation<br>As cells die,<br>kidney becomes more<br>“leaky”, and<br>protein spills into the<br>urine (proteinuria)<br>DMX-200<br>specifically blocks kidney<br>inflammatory signaling<br>1 Guruswamy Sangameswaran KD, Baradhi KM. Focal Segmental Glomerulosclerosis (July 2021), online:<br>https://www.ncbi.nlm.nih.gov/books/NBK532272/;<br>2 Kiffel et. Al. Adv Chronic Kidney Dis. (September 2011), online: https://pmc.ncbi.nlm.nih.gov/articles/PMC3709971/pdf/nihms286597.pdf |
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| 16<br>MOA Precision therapy to disrupt the pathogenic monocyte-driven inflammatory feedback<br>loop in the kidney of patients with FSGS<br>Phase 2 Positive efficacy signals and well-tolerated across studies (n=80), including impacts<br>on proteinuria and inflammation in FSGS study<br>ACTION3<br>Phase 3<br>Enrollment well underway (259 of 286 pts to date); Interim analysis (n=72 at 36 wks)<br>showed DMX-200 performing better than placebo in reducing proteinuria1<br>FDA and<br>Project PARASOL Alignment on proteinuria as a primary endpoint for approval<br>ACTION3 Part 2<br>Interim Analysis Expected after planned follow-up meeting with FDA<br>ACTION3 Part 3<br>Final Analysis<br>2-year proteinuria (primary) and eGFR (secondary) data serves as basis for Full<br>Approval (n=286)<br>Dimerix has built a strong body of evidence and made significant clinical and regulatory progress<br>DMX-200 Clinical and Regulatory Progress<br>1 Predictive power statistical model, using industry standard as set by the independent renal biostatistician consultant for Dimerix |
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| 17<br>Corporate Outlook<br>Delivering on our mission for patients<br>and shareholders |
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| 18<br>Q3 2025 Select Financial Results<br>Q3’25 YTD’25<br>(in thousands, except per share data) Sep. 30, 2025 Sep. 30, 2024 Sep. 30, 2025 Sep. 30, 2024<br>Net product sales $ 169,061 $ 141,517 $ 448,998 $ 378,589<br>Cost of goods sold 19,467 13,279 46,382 38,107<br>GAAP operating expenses 115,325 106,579 385,773 331,577<br>Non-GAAP operating expenses 95,362 82,577 317,688 250,195<br>GAAP net income (loss) 17,306 (6,729) (28,800) (70,845)<br>Non-GAAP net income 54,237 30,786 65,133 44,692<br>GAAP net income (loss) per share – basic and<br>diluted $ 0.06 $ (0.02) $ (0.09) $ (0.23)<br>Non-GAAP net income per share – basic $ 0.18 $ 0.10 $ 0.21 $ 0.15<br>Non-GAAP net income per share – diluted $ 0.17 $ 0.10 $ 0.21 $ 0.15<br>QTD September 30, 2025 basic and diluted weighted-average common shares outstanding: 308,468,423 and 310,433,494 respectively; QTD September 30, 2024 basic and diluted weighted-average common shares outstanding: 304,690,596 and 304,690,596 respectively<br>YTD September 30, 2025, basic and diluted weighted-average common shares outstanding: 308,139,134 and 308,139,134 respectively; YTD September 30, 2024, basic and diluted weighted-average common shares outstanding: 303,792,479 and 303,792,479 respectively<br>Second quarter 2025 operating expenses reflect the previously announced $30M upfront payment for the U.S. licensing agreement of DMX-200<br>Q3 2025 revenue of $169M, up 17% at CER and non-GAAP net income of $54.2M |
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| 19<br>FY 2025 Financial Guidance Reiterated<br>FY 2025 Financial Guidance1<br>Total Revenue Growth1 15% to 22%<br>Galafold Revenue Growth1 10% to 15%<br>Pombiliti + Opfolda Revenue Growth1 50% to 65%<br>Gross Margin Mid 80%<br>Non-GAAP Operating Expense $380M to $400M2<br>GAAP Net Income Positive during<br>H2 2025<br>1 Full-Year 2025 guidance is provided at CER (Constant Exchange Rates) using Full-Year 2024 Average Exchange Rates 2 Inclusive of $30M upfront payment for the U.S. licensing agreement of DMX-200<br>FY 2025 Revenue Sensitivity<br>Given the proportion of Amicus revenue ex-US<br>(~60% in 2024), a change in USD exchange<br>rates of +/- 1% compared to 2024 rates could<br>lead to a ~$4M move in Total Reported<br>Revenues in 2025 |
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| 20 1 CER: Constant Exchange Rates<br>2025 Strategic Priorities<br>Deliver total revenue growth of 15-22% at CER1<br>Double-digit Galafold® revenue growth of 10-15% at CER1<br>Pombiliti®+ Opfolda® revenue growth of 50-65% at CER1<br>Advance ongoing studies in Fabry, Pompe and FSGS<br>Deliver positive GAAP net income during H2 2025 |
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| 21<br>A unique story in biotech with significant revenue growth and profitability<br>Two Approved<br>Therapies<br>One Late Stage<br>Phase 3 Program<br>Surpassing $1B<br>in Total Sales<br>in 2028 Double-digit<br>Revenue<br>Growth<br>Self-Sustainable<br>Company and<br>Growing Free<br>Cash Flow<br>A Rare Company<br>Leverageable<br>Rare Disease<br>Infrastructure |
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| Appendix |
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| 23<br>Reconciliation of Non-GAAP Financial Measures |
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| 24<br>Reconciliation of Non-GAAP Financial Measures (Cont’d) |
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| 25<br>Exchange Rates<br>Currency Average Rates<br>FX Rates Q3 2024 Q3 2025 Variance<br>USD/EUR 1.099 1.169 6.4%<br>USD/GBP 1.301 1.349 3.7%<br>USD/JPY 0.007 0.007 0.9% |
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| 26<br>Rare Disease Pipeline<br>1Exclusive rights to commercialize in the United States<br>INDICATION DISCOVERY PRECLINICAL PHASE 1/2 PHASE 3 REGULATORY COMMERCIAL<br>FABRY DISEASE<br>Galafold® (migalastat)<br>Fabry Genetic Medicines<br>POMPE DISEASE<br>Pombiliti® (cipaglucosidase alfa-atga) + Opfolda® (miglustat)<br>Pompe Genetic Medicines<br>RARE KIDNEY DISEASE<br>DMX-200 in Focal Segmental Glomerulosclerosis1<br>DMX-200 in Additional Indications1 |
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