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): May 1, 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 May 1, 2025, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the fiscal quarter ended March 31, 2025. A copy of this press release is attached hereto as Exhibit 99.1. The Company will host a conference call and webcast on May 1, 2025 to discuss its second 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 May 1, 2025 |
| 99.2 | May 1, 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: May 1, 2025 | By: | /s/ Ellen S. Rosenberg |
| Name: | Ellen S. Rosenberg | |
| Title: | Chief Legal Officer and Corporate Secretary |
Exhibit 99.1
Amicus Therapeutics Announces First Quarter2025Financial Results and Corporate Updates
1Q 2025 Total Revenue of $125.2M, a 15% IncreaseYear-over-Year at CER
Expanding Portfoliothrough In-Licensing of DMX-200 Phase 3 Program for Rare Kidney Disease with Significant Market Potential in the U.S.
Maintaining 2025Guidance for Galafold, Reflecting Strong Underlying Demand
Updating 2025 Pombiliti+ Opfolda Guidance with New Patient Starts Accelerating in 2H
Adjusting 2025 Total Revenue Growth Guidanceto 15-22% at CER
Reiterating GAAPProfitability During H2 2025
Conference Call and Webcast Today at 8:30a.m. ET
PRINCETON,NJ, May 1, 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 first quarter ended March 31, 2025.
“Amicus delivered another consecutive quarter of significant double-digit revenue growth. Looking forward, the underlying patient demand we observed in Q1 will drive robust growth for Galafold, and we continue to expect accelerating Pombiliti + Opfolda sales as the year progresses, driven by patient starts from new launch markets as well as anticipated acceleration in U.S. switches. While some unexpected factors impacted revenue in the quarter, the key performance indicators for both products are very strong and we remain on-track to achieve GAAP profitability during the second half of 2025 and to deliver double-digit revenue growth this year and beyond,” said Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc.
Mr. Campbell continued “We are also thrilled to have announced the in-licensing of the U.S. commercial rights to Dimerix’ DMX-200, a first-in-class treatment in Phase 3 development for people living with FSGS, a rare and potentially fatal kidney disease. This aligns perfectly with our strategy to leverage our rare disease commercial infrastructure and brings a third program with blockbuster market potential to our portfolio. Amicus is well positioned to create substantial value for shareholders and to deliver on our mission for patients, and we very much look forward to working with Dimerix to bring this much needed therapy to people living with FSGS in the United States.”
First Quarter 2025 Financial Highlights
| · | Total revenues for the first quarter 2025were $125.2 million, reflecting operational growth measured at constant exchange rates (CER)^1^ of 15% and a currency headwind<br>of $1.4 million or 1%. | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| Three Months Ended<br><br> March 31, | Year over Year %<br> <br>Growth | |||||||||
| --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- |
| (in thousands) | 2025 | 2024 | Reported | at CER^1^ | ||||||
| Galafold^®^ | 104,244 | 99,359 | 5 | % | 6 | % | ||||
| Pombiliti^®^ + Opfolda^®^ | 21,005 | 11,044 | 90 | % | 92 | % | ||||
| Net Product Revenues | $ | 125,249 | $ | 110,403 | 13 | % | 15 | % | ||
| · | Galafold^®^ (migalastat)net product sales for the first quarter 2025 were $104.2 million, representing a year-over-year increase of 5%, or 6% at CER^1^.<br>Strong patient demand of 14% in the quarter was partially offset by order timing and the ongoing impact of the higher than anticipated<br>VPAG (Voluntary Scheme for Branded Medicines Pricing and Access) rebate in the U.K. Given the significant underlying demand, the Company<br>anticipates Galafold revenue to accelerate in the second quarter and is reiterating its full year 2025 revenue growth guidance of +10-15%<br>at CER. | |||||||||
| --- | --- |
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| · | Pombiliti (cipaglucosidase alfa-atga) + Opfolda(miglustat) net product sales for the first quarter 2025 were $21.0 million, representing a year-over-year increase of 90%, or 92%<br>at CER^1^. First quarter sales reflected the timing of patient starts in new launch countries and the ongoing impact of the higher<br>than anticipated VPAG rebate in the U.K. The Company now expects the benefit of patient starts in new launch markets to be more weighted<br>towards the second half of the year. Taken together with an anticipated acceleration in U.S. switches, the Company is therefore adjusting<br>its 2025 revenue growth guidance range for Pombiliti + Opfolda to +50-65% at CER. |
|---|---|
| · | Total GAAP operating expenses of $121.5<br>million for the first quarter 2025 decreased by 2.5% as compared to $124.6 million for the first quarter 2024. Total non-GAAP operatingexpenses^2^ were up 10.4% to $94.5 million for the first quarter 2025 as compared to $85.6 million for the first quarter<br>2024. |
| --- | --- |
| · | GAAP net loss was $21.7 million, or $0.07<br>loss per share basic and diluted, for the first quarter 2025, compared to a net loss of $48.4 million, or $0.16 per share basic and diluted,<br>for the first quarter 2024. Non-GAAP net income^2,3^ was $9.0 million, or $0.03 per share basic and diluted, for the first<br>quarter 2025, compared to a non-GAAP net loss of $4.6 million, or $0.02 per share basic and diluted, for the first quarter 2024. |
| --- | --- |
| · | Cash, cash equivalents, and marketable securities<br>totaled $250.6 million at March 31, 2025, representing a slight increase as compared to $249.9 million at December 31, 2024. |
| --- | --- |
Corporate Updates:
| · | Pombiliti + Opfolda selected as preferredtreatment for adults living with late-onset Pompe disease in the Netherlands. The five-year agreement will enable broad and sustained<br>access to Pombiliti + Opfolda for adults living with late-onset Pompe disease in the Netherlands currently receiving enzyme replacement<br>therapy or naïve to treatment. First commercial patients are expected to begin treatment in 2Q 2025. The Netherlands has the highest<br>prevalence of Pompe disease in Europe with over 150 individuals living with the condition. |
|---|---|
| · | Pombiliti + Opfolda regulatory approval grantedin Canada and Australia for adult LOPD patients. The Company continues to anticipate a regulatory decision in Japan this year as well<br>as additional reimbursement agreements throughout the year. The Company also remains on track for up to 10 new launch countries in 2025,<br>which include more than 650 individuals living with LOPD. |
| --- | --- |
| · | Commercial manufacturing and supply servicesagreement reached with Sharp Sterile to manufacture Pombiliti drug product in the U.S. This agreement is another important step in<br>further diversifying the supply chain for Pombiliti. |
| --- | --- |
| · | Entered into exclusive U.S. licensing agreementwith Dimerix. As announced separately, Amicus has licensed exclusive rights for the U.S. commercialization of Dimerix’ Phase<br>3 program, DMX-200, a first in class treatment for FSGS, a rare and fatal kidney disease with no approved therapies and significant market<br>potential. |
| --- | --- |
| · | Amicus is focused on delivering significant long-term revenue growth andanticipates surpassing $1 billion in total sales in 2028. The Company anticipates continuing to grow its current commercial business<br>with Galafold and Pombiliti + Opfolda resulting in strong total revenue growth. |
| --- | --- |
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2025 Financial Guidance
Amicus’ updated financial guidance for 2025, which includes the upfront license payment and all other anticipated operating expenses of the licensing of DMX-200 in the U.S., is as follows:
| Previous | Updated | |||
|---|---|---|---|---|
| Total Revenue Growth^1^ | 17% to 24% | → | 15% to 22% | |
| Galafold Revenue Growth^1^ | 10% to 15% | → | 10% to 15% | |
| Pombiliti + Opfolda Growth^1^ | 65% to 85% | → | 50% to 65% | |
| Gross Margin | Mid 80% | → | Mid 80% | |
| Non-GAAP Operating Expenses^4^ | $350M to $370M | → | $380M to $400M | (Incl. $30M Upfront License Payment) |
| GAAP Net Income | Positive during <br> 2H 2025 | → | Positive during <br> 2H 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, May 1, 2025, at 8:30 a.m. ET to discuss the first 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 number 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.
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.
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U.S. IMPORTANT SAFETY INFORMATION
ADVERSEREACTIONS: 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 about Galafold, including thefull 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).
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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-FETALTOXICITY: 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. AdverseReactions: Most common adverse reactions ≥ 5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia. Pleasesee full PRESCRIBING INFORMATION, including BOXED WARNING, for POMBILITI (cipaglucosidase alfa-atga) LINK and full PRESCRIBINGINFORMATION 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 X and 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 collaboration and license agreement for of DMX-200 may not be successful, including without limitation expectations of the timing of 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, 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.
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CONTACT:
Investors:
Amicus Therapeutics
Andrew Faughnan
Vice President, Investor Relations
afaughnan@amicusrx.com
(609) 662-3809
Media:
Amicus Therapeutics
Diana Moore
Head of Global Corporate Affairs and Communications
dmoore@amicusrx.com
(609) 662-5079
FOLD-G
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TABLE 1
AmicusTherapeutics, Inc.
ConsolidatedStatements of Operations
(Unaudited)
(in thousands, except share and per share amounts)
| Three Months Ended March 31, | ||||||
|---|---|---|---|---|---|---|
| 2025 | 2024 | |||||
| Net product sales | $ | 125,249 | $ | 110,403 | ||
| Cost of goods sold | 11,698 | 13,567 | ||||
| Gross profit | 113,551 | 96,836 | ||||
| Operating expenses: | ||||||
| Research and development | 27,839 | 28,329 | ||||
| Selling, general, and administrative | 91,827 | 88,029 | ||||
| Restructuring charges | — | 6,045 | ||||
| Depreciation and amortization | 1,837 | 2,154 | ||||
| Total operating expenses | 121,503 | 124,557 | ||||
| Loss from operations | (7,952 | ) | (27,721 | ) | ||
| Other expense: | ||||||
| Interest income | 812 | 1,540 | ||||
| Interest expense | (11,455 | ) | (12,436 | ) | ||
| Other income (expense) | 550 | (4,966 | ) | |||
| Loss before income tax | (18,045 | ) | (43,583 | ) | ||
| Income tax expense | (3,641 | ) | (4,836 | ) | ||
| Net loss attributable to common stockholders | $ | (21,686 | ) | $ | (48,419 | ) |
| Net loss attributable to common stockholders per common share — basic and diluted | $ | (0.07 | ) | $ | (0.16 | ) |
| Weighted-average common shares outstanding — basic and diluted | 307,689,207 | 302,903,009 |
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TABLE 2
AmicusTherapeutics, Inc.
ConsolidatedBalance Sheets
(in thousands, except share and per share amounts)
| December 31,<br><br> 2024 | |||||
|---|---|---|---|---|---|
| Assets | |||||
| Current assets: | |||||
| Cash and cash equivalents | 181,657 | $ | 213,752 | ||
| Investments in marketable securities | 68,916 | 36,194 | |||
| Accounts receivable | 88,323 | 101,099 | |||
| Inventories | 132,412 | 118,782 | |||
| Prepaid expenses and other current assets | 39,491 | 34,909 | |||
| Total current assets | 510,799 | 504,736 | |||
| Operating lease right-of-use assets, net | 22,138 | 22,278 | |||
| Property and equipment, less accumulated depreciation of 29,842 and 28,775 at March 31, 2025 and December 31, 2024, respectively | 28,718 | 29,383 | |||
| Intangible assets, less accumulated amortization of 6,611 and 5,802 at March 31, 2025 and December 31, 2024, respectively | 16,389 | 17,198 | |||
| Goodwill | 197,797 | 197,797 | |||
| Other non-current assets | 13,998 | 13,641 | |||
| Total Assets | 789,839 | $ | 785,033 | ||
| Liabilities and Stockholders’ Equity | |||||
| Current liabilities: | |||||
| Accounts payable | 14,451 | $ | 12,947 | ||
| Accrued expenses and other current liabilities | 129,877 | 127,300 | |||
| Operating lease liabilities | 8,562 | 8,455 | |||
| Total current liabilities | 152,890 | 148,702 | |||
| Long-term debt | 390,708 | 390,111 | |||
| Operating lease liabilities | 44,196 | 45,078 | |||
| Other non-current liabilities | 8,487 | 7,097 | |||
| Total liabilities | 596,281 | 590,988 | |||
| Commitments and contingencies | |||||
| Stockholders’ equity: | |||||
| Common stock, 0.01 par value, 500,000,000 shares authorized, 307,923,069 and 299,041,653 shares issued and outstanding at March 31, 2025 and December 31, 2024, respectively | 3,016 | 2,944 | |||
| Common stock in treasury, at cost; 7,390 shares as of March 31, 2025 | (71 | ) | — | ||
| Additional paid-in capital | 2,939,673 | 2,926,115 | |||
| Accumulated other comprehensive income (loss): | |||||
| Foreign currency translation adjustment | 12,941 | 5,302 | |||
| Unrealized loss on available-for-sale securities | (135 | ) | (207 | ) | |
| Warrants | — | 71 | |||
| Accumulated deficit | (2,761,866 | ) | (2,740,180 | ) | |
| Total stockholders’ equity | 193,558 | 194,045 | |||
| Total Liabilities and Stockholders’ Equity | 789,839 | $ | 785,033 |
All values are in US Dollars.
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TABLE 3
AmicusTherapeutics, Inc.
Reconciliationof Non-GAAP Financial Measures
(inthousands)
(Unaudited)
| Three Months Ended March 31, | ||||
|---|---|---|---|---|
| 2025 | 2024 | |||
| Total GAAP operating expenses | $ | 121,503 | $ | 124,557 |
| Research and development: | ||||
| Share-based compensation | 4,004 | 4,871 | ||
| Selling, general and administrative: | ||||
| Share-based compensation | 21,168 | 25,932 | ||
| Restructuring charge | — | 6,045 | ||
| Depreciation and amortization | 1,837 | 2,154 | ||
| Total Non-GAAP operating expense adjustments | 27,009 | 39,002 | ||
| Total Non-GAAP operating expenses | $ | 94,494 | $ | 85,555 |
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TABLE 4
AmicusTherapeutics, Inc.
Reconciliationof Non-GAAP Financial Measures
(inthousands, except share and per share amounts)
(Unaudited)
| Three Months Ended<br> <br>March 31, | ||||||
|---|---|---|---|---|---|---|
| 2025 | 2024 | |||||
| GAAP net loss | $ | (21,686 | ) | $ | (48,419 | ) |
| Share-based compensation | 25,172 | 30,803 | ||||
| Depreciation and amortization | 1,837 | 2,154 | ||||
| Restructuring charges | — | 6,045 | ||||
| Income tax expense | 3,641 | 4,836 | ||||
| Non-GAAP net income (loss) | $ | 8,963 | $ | (4,581 | ) | |
| Non-GAAP net income (loss) attributable to common stockholders per common share — basic and diluted | $ | 0.03 | $ | (0.02 | ) | |
| Weighted-average common shares outstanding — basic | 307,689,207 | 302,903,009 | ||||
| Weighted-average common shares outstanding — diluted | 309,654,136 | 302,903,009 |
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Exhibit 99.2
| AT THE FOREFRONT OF<br>THERAPIES FOR RARE DISEASES<br>1Q25 Results<br>Conference Call<br>& Webcast<br>May 1, 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 collaboration and<br>license agreement for of DMX-200 may not be successful, including without limitation expectations of the timing of Phase 3 clinical trial evaluating DMX-200; the likelihood of success of<br>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<br>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<br>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<br>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<br>the Company's revenue, 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<br>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<br>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<br>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<br>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>$125M<br>1Q 2025 Total Revenue<br>(+15% 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 March 31, 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>Treated2<br>$104.2M<br>1Q25 Galafold<br>Revenue +14%<br>1Q25 Galafold<br>Patient Demand<br>Growth1<br>69%<br>Share of Treated<br>Amenable Patients<br>1 Data on file<br>2 As of YE 2024 |
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| 6<br>FY16 FY17 FY18 FY19 FY20 FY21 FY22 FY23 FY24 FY25<br>$5M<br>$37M<br>$91M<br>$182M<br>$261M<br>$306M<br>$329M<br>$388M<br>$458M<br> Quarterly patient starts remains strong: +14%<br>growth in 1Q YoY2<br> 1Q revenue impacted by ordering patterns and<br>greater than anticipated U.K. rebate<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<br>expansion<br> Maintaining >90% adherence and compliance<br>through HCP and patient education and support<br>Galafold Performance<br>Revenue growth expected to accelerate throughout the year<br>and FY 2025 Galafold growth guidance of 10-15% at CER1 reiterated<br>Q1<br>$99M<br>Q2<br>$111M<br>Q3<br>$120M<br>Q4<br>$128M<br>+10-15%1<br>1Q 2025 Galafold reported revenue of $104.2M (+6% growth at CER)<br>1 CER: Constant Exchange Rates<br>2 Data on file<br>Q1<br>$104M |
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| 7<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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| 8<br>FY23 FY24 FY25<br>Pombiliti + Opfolda Performance<br>1Q 2025 Pombiliti + Opfolda reported revenue of $21.0M (+92% at CER)<br>1Q<br>$11M<br>3Q<br>$21M<br>4Q<br>$22M<br>$11.6M<br>1 CER: Constant Exchange Rates<br>2 Real-World Evidence<br>$70.2M<br>+50-65%1<br>2Q<br>$16M 1Q<br>$21M<br> Strong 1Q sales growth YoY, particularly in the U.S.<br>– Number of net new patients continues to grow<br>– Increasing depth and breadth of prescribers<br> 1Q 2025 revenue reflected timing of patient starts in<br>new launch countries and higher than anticipated<br>VPAG rebate in the U.K.<br> Ramp in new patient starts expected to be weighted<br>to 2H 2025 due to:<br>– Multiple new launch markets coming online<br>– Anticipated acceleration in U.S. switches<br>– Growing body of RWE2 supporting switch from both<br>alternative therapies<br>Updating FY 2025 Pombiliti + Opfolda growth guidance to 50-65% at CER1<br>1Q<br>$21M |
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| 9<br>Regulatory approvals anticipated in 2025:<br>3 regulatory approvals and up to 10 new launch countries in 2025<br>Pombiliti + Opfolda Expansion<br> Recently approved in Australia and Canada<br> Regulatory approval in Japan anticipated in 2025 AUSTRALIA CANADA JAPAN<br>Combined ~150-200 people 18+ living with LOPD<br>and being treated with a Pompe therapy<br>Regulatory<br>Reimbursement<br> Expect to launch in up to 10 new countries this year<br>>650 LOPD patients 18+ in those 10 countries<br> First commercial patients from all 6 newly reimbursed<br>countries anticipated no later than 2Q 2025<br> Pombiliti + Opfolda selected as preferred treatment<br>for adults with LOPD in the Netherlands<br> 5-year agreement enables broad and sustained access<br>for adults with LOPD currently on ERT<br>New reimbursement agreements completed in:<br>SWEDEN SWITZERLAND CZECH REPUBLIC<br>ITALY<br>Combined ~325-375 people 18+ living with LOPD<br>and being treated with a Pompe therapy<br>NETHERLANDS PORTGUAL |
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| 10<br>Supply Chain<br> New commercial manufacturing and supply services<br>agreement in place with Sharp Sterile Manufacturing<br> Pombiliti drug product manufacturing capacity<br>to be further expanded to a U.S. site in Lee, MA<br> Diversifying our global supply chain for Pombiliti<br>Limited Tariff Impact Diversifying Supply Chain<br> Anticipate no material impact to P&L or business<br>operations in 2025 of any of the proposed tariffs:<br> Well managed through careful expense management<br>and supply chain planning<br> Majority of revenue outside the U.S.<br> 2025 U.S. sales inventory already within the U.S.<br>Ensuring access to our two medicines through supply chain planning |
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| 11<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>1 EAMS: Early Access to Medicines Scheme<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> Case studies supporting the switch from both alternative therapies<br> Case studies of patients switching from high dose, high frequency alglucosidase alfa |
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| 12<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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| 13<br>Deal adds significant value to Amicus today and aligns with Amicus strategy to leverage<br>our rare disease commercial infrastructure with promising late-stage program<br>Exclusive U.S. License Agreement with Dimerix<br> Focal Segmental Glomerulosclerosis (FSGS) is a rare and fatal kidney disease1<br>that affects >40,000 people in the U.S., with no approved treatments<br> DMX-200 is a Phase 3 program, with Orphan Drug designation,<br>in development for a disease with blockbuster market potential in the U.S.<br> ACTION3 study is well underway with positive interim analysis2<br>and agreement with FDA on proteinuria as primary endpoint<br> Structured in-licensing deal with modest upfront and downstream<br>success-based milestones and tiered royalties<br> Opportunity to pursue additional indications<br>1<br>2<br>3<br>4<br>5<br>1 Guruswamy Sangameswaran KD, Baradhi KM. Focal Segmental Glomerulosclerosis (July 2021), online: https://www.ncbi.nlm.nih.gov/books/NBK532272/; 2 Predictive power statistical model, using industry standard as set by the independent renal biostatistician consultant for Dimerix |
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| 14<br>DMX-200 Transaction Summary<br>No change to Amicus GAAP profitability guidance during 2H 2025<br>License agreement is heavily weighted to mutually beneficial success-based milestones<br>Upfront investment of $30M paid with cash on hand<br>Tiered royalties at the low teens to low twenties<br>Up to $560M in potential success-based milestone payments<br> Up to $75M in development & regulatory milestones until FDA approval of DMX-200 in FSGS<br> $35M on first sale<br> Up to $410M in commercial sales milestones<br> Up to $40M in milestones for potential future indications |
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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:<br>https://pmc.ncbi.nlm.nih.gov/articles/PMC3709971/pdf/nihms286597.pdf |
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| 16<br>Dimerix has built a strong body of evidence and made significant<br>clinical and regulatory progress with DMX-200 in FSGS<br>1 Predictive power statistical model, using industry standard as set by the independent renal biostatistician consultant for Dimerix<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 (185 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) |
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| 17<br>FDA aligned on proteinuria endpoint for approval; 185 patients already randomized/dosed to date<br>DMX-200 Phase 3 Clinical Trial in FSGS<br>A randomised, double-blind, multi-center, placebo-controlled study of renal outcomes of DMX-200 in patients with FSGS receiving an ARB<br>ARB + DMX-200<br>ARB + placebo<br>Part 2:<br>analysis outcome<br>Total of 286 patients<br>@ 104 weeks<br>(uPCR/eGFR)<br>Phase 3 Trial Timeline<br>Part 3:<br>final analysis<br>Background<br>• Patients recruited, then screened<br>and stabilised on background<br>medications<br>• Patients randomized to receive<br>drug or placebo<br>• DXB remains blinded at all times<br>during study<br>72 patients @ 35 weeks<br>Successful analysis outcome<br>(using statistical measure)<br>(% change in uPCR)<br>Open Label Extension<br>DMX-200<br>ACTION3 Study End<br>blinded interim data collection following<br>additional analysis of the PARASOL data<br>Dimerix will continue to fund and execute the ACTION3 study |
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| 18<br>Corporate Outlook<br>Delivering on our mission for patients<br>and shareholders |
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| 19<br>Q1 2025 Select Financial Results<br>Q1’25<br>(in thousands, except per share data) Mar. 31, 2025 Mar. 31, 2024<br>Net product sales $ 125,249 $ 110,403<br>Cost of goods sold 11,698 13,567<br>GAAP operating expenses 121,503 124,557<br>Non-GAAP operating expenses 94,494 85,555<br>GAAP net loss (21,686) (48,419)<br>Non-GAAP net income (loss) 8,963 (4,581)<br>GAAP net loss per share – basic and diluted $ (0.07) $ (0.16)<br>Non-GAAP net income (loss) per share – basic and diluted $ 0.03 $ (0.02)<br>March 31, 2025, basic and diluted weighted-average common shares outstanding: 307,689,207 and 309,654,136, respectively.<br>March 31, 2024, basic and diluted weighted-average common shares outstanding: 302,903,009, respectively.<br>Q1 2025 revenue of $125M, up 15% at CER and non-GAAP net income of $9.0M |
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| 20<br>FY 2025 Financial Guidance<br>FY 2025 Financial Guidance1 Previous Guidance Updated Guidance<br>Total Revenue Growth1 17% to 24% 15% to 22%<br>Galafold Revenue Growth1 10% to 15% 10% to 15%<br>Pombiliti + Opfolda Revenue Growth1 65% to 85% 50% to 65%<br>Gross Margin Mid 80% Mid 80%<br>Non-GAAP Operating Expense $350M to $370M $380M to $400M<br>inclusive of $30M upfront fee<br>GAAP Net Income Positive during<br>H2 2025<br>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<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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| 21 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 now FSGS<br>Deliver positive GAAP net income during H2 2025 |
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| 22<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>Leverageable<br>Rare Disease<br>Infrastructure<br>A Rare Company |
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| Appendix |
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| 24<br>Reconciliation of Non-GAAP Financial Measures |
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| 25<br>Reconciliation of Non-GAAP Financial Measures (Cont’d) |
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| 26<br>Exchange Rates<br>Currency Average Rates<br>FX Rates Q1 2024 Q1 2025 Variance<br>USD/EUR 1.086 1.052 (3.1%)<br>USD/GBP 1.268 1.259 (0.7%)<br>USD/JPY 0.007 0.007 (2.7%) |
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| Rare Disease Pipeline<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<br>1 Exclusive rights to commercialize in the United States |
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