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): February 19, 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, NJ 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 Operationsand Financial Condition
On February 19, 2025, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the fiscal year ended December 31, 2024. A copy of this press release is attached hereto as Exhibit 99.1. The Company will host a conference call and webcast on February 19, 2025 to discuss its full year 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 February 19, 2025 |
| 99.2 | February 19, 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: February 19, 2025 | By: | /s/ Ellen S. Rosenberg |
| Name: | Ellen S. Rosenberg | |
| Title: | Chief Legal Officer and Corporate Secretary |
Exhibit99.1
AmicusTherapeutics Announces Full-Year 2024Financial Results and Corporate Updates
2024Total Revenue of $528.3M, a 33% Increase Year-over-Year
Projecting2025 Total Revenue Growth of 17-24% at CER
AnticipateAchieving Positive GAAP Net Income During H2 2025
ConferenceCall and Webcast Today at 8:30 a.m. ET
PRINCETON,NJ, Feb. 19, 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 full-year ended December 31, 2024.
“We closed 2024 beating expectations with exceptional revenue growth of 33 percent. With our portfolio and global rare disease capabilities, we have a clear path to deliver continued revenue growth and accelerating profitability in 2025 and the years ahead. We have firmly established Amicus as a unique biotechnology company: two growing medicines with long runways, a leverageable infrastructure with patients at the center, and the financial strength to continue to build the business and our leadership in rare diseases,” said Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc.
CorporateHighlights:
| · | Total revenues for the full-year 2024 were $528.3 million, reflecting operational growth measured<br> at constant exchange rates (CER)^1^ of 33% and a small currency impact of $1.1 million<br> or 1%. Fourth quarter total revenues were $149.7 million, up 30% as reported and at CER.<br> For the full year 2025, the Company provides total revenue growth guidance of +17% to +24%<br> on a constant currency basis (CER)^1^. | |||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| (in thousands) | Three Months Ended<br><br> December 31, | Year over Year %<br> <br>Growth | Twelve Months Ended<br> <br>December 31, | Year over Year %<br> <br>Growth | ||||||||||||||||
| --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- |
| 2024 | 2023 | Reported | at CER^1^ | 2024 | 2023 | Reported | at CER^1^ | |||||||||||||
| Galafold^®^ | 127,497 | 106,601 | 20 | % | 20 | % | 458,054 | 387,777 | 18 | % | 19 | % | ||||||||
| Pombiliti^®^+ Opfolda^®^ | 22,209 | 8,481 | 162 | % | 161 | % | 70,241 | 11,579 | 507 | % | 504 | % | ||||||||
| Net Product Revenues | 149,706 | 115,082 | 30 | % | 30 | % | $ | 528,295 | $ | 399,356 | 32 | % | 33 | % | ||||||
| · | Galafold (migalastat) net product sales for the full-year 2024 were $458.1 million, representing<br> a year-over-year increase of 18%, or 19% at CER. Fourth quarter net product sales were $127.5<br> million. At the end of 2024, there were ~2,730 people living with Fabry disease on Galafold<br> following a year of increased demand. For the full year 2025, the Company provides revenue<br> growth guidance for Galafold of +10% to +15% on a constant currency basis (CER)^1^. | |||||||||||||||||||
| --- | --- | |||||||||||||||||||
| · | Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) net product sales for the full-year 2024 were $70.2 million. Fourth quarter net product sales were $22.2 million. Following a<br> successful commercial launch in the U.S., Germany, Austria, Spain and the U.K., there were<br> ~220 patients treated or scheduled with commercial product as of the end of 2024. For the<br> full year 2025, the Company provides revenue growth guidance for Pombiliti + Opfolda of +65%<br> to +85% on a constant currency basis (CER)^1^. | |||||||||||||||||||
| --- | --- | |||||||||||||||||||
| · | Multiple Pombiliti + Opfolda pricing and reimbursement agreements recently achieved. Agreements<br> completed in late 2024 and early 2025 include Italy, Sweden, Switzerland, Czech Republic<br> and most recently the Netherlands. First commercial patients from these countries are anticipated<br> to begin treatment over the first half of 2025. Additionally, Pombiliti + Opfolda received<br> regulatory approval in Australia and the Company anticipates new regulatory decisions in<br> Canada and Japan in 2025 as well as additional reimbursement agreements throughout the year. | |||||||||||||||||||
| --- | --- |
1
| · | Amicus is focused on delivering significant long-term revenue growth and anticipates surpassing $1 billion in total sales in 2028. The Company anticipates continuing to grow its current<br> commercial business with Galafold and Pombiliti + Opfolda resulting in strong total revenue<br> growth. |
|---|---|
| · | Total GAAP operating expenses of $450.5 million for the full year 2024 increased by 2.6% as<br> compared to $439.2 million for the full year 2023. **Total non-GAAP operating expenses^2^**were up 1.8% to $347.8 million for the full year 2024 as compared to $341.6 million<br> for the full year 2023. For the full year 2025, the Company provides non-GAAP operating expense^4^guidance range of $350 million to $370 million. |
| --- | --- |
| · | GAAP net loss was $56.1 million, or $0.18 per share basic and diluted, for the full year 2024,<br> and was reduced compared to a net loss of $151.6 million, or $0.51 per share basic and diluted,<br> for the full year 2023. GAAP net income was $14.7 million, or $0.05 per share basic<br> and diluted, for the fourth quarter 2024, compared to a net loss of $33.8 million, or $0.11<br> per share basic and diluted, for the fourth quarter 2023. The company anticipates achieving<br> positive GAAP net income during H2 2025. |
| --- | --- |
| · | Non-GAAP net income^2,3^ was $73.9 million, or $0.24 per share, for the full year 2024,<br> compared to a non-GAAP net loss of $38.5 million, or $0.13 per share basic and diluted, for<br> the full year 2023. Non-GAAP net income was $29.2 million, or $0.10 per share basic and $0.09<br> per share diluted, for the fourth quarter 2024, compared to a net income of $2.6 million,<br> or $0.01 per share basic and diluted, for the fourth quarter 2023. |
| --- | --- |
| · | Cash, cash equivalents, and marketable securities totaled $249.9 million at December 31,<br> 2024, compared to $249.8 million at September 30, 2024 and $286.2 million at December 31,<br> 2023. |
| --- | --- |
2025Financial Guidance
To advance our strategy and expand our leadership in Fabry and Pompe disease, our financial guidance for 2025 is as follows:
| Total Revenue Growth^1^ | 17% to 24% |
|---|---|
| Galafold Revenue Growth^1^ | 10% to 15% |
| Pombiliti + Opfolda Growth^1^ | 65% to 85% |
| Gross Margin | Mid 80% |
| Non-GAAP Operating Expenses^4^ | $350M to $370M |
| 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.
ConferenceCall and Webcast
Amicus Therapeutics will host a conference call and audio webcast today, February 19, 2025, at 8:30 a.m. ET to discuss the full-year 2024 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.
AboutGalafold
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.
2
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
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 aboutGalafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf.
AboutPombiliti + 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).
SAFETYINFORMATION
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.
AboutAmicus 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.
3
Non-GAAPFinancial 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.
ForwardLooking Statement
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, manufacturing and supply plans, financing plans, 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, with respect to 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 results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; 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 preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; the potential that we may not be able to manufacture or supply sufficient clinical or commercial products; and the potential that we will need additional funding to complete all of our studies, 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 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
Diana Moore
Head of Global Corporate Affairs and Communications
dmoore@amicusrx.com
(609) 662-5079
4
FOLD-G
TABLE1
AmicusTherapeutics, Inc.
ConsolidatedStatements of Operations
(inthousands, except share and per share amounts)
| Years<br> ended December 31, | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| 2024 | 2023 | 2022 | |||||||
| Net product sales | $ | 528,295 | $ | 399,356 | $ | 329,233 | |||
| Cost of goods sold | 52,943 | 37,326 | 38,599 | ||||||
| Gross profit | 475,352 | 362,030 | 290,634 | ||||||
| Operating expenses: | |||||||||
| Research and development | 109,362 | 152,381 | 276,677 | ||||||
| Selling, general, and administrative | 323,379 | 275,270 | 213,041 | ||||||
| Changes in fair value of contingent<br> consideration payable | — | 2,583 | 1,078 | ||||||
| Loss on impairment of assets | — | 1,134 | 6,616 | ||||||
| Restructuring charges | 9,188 | — | — | ||||||
| Depreciation and<br> amortization | 8,547 | 7,873 | 5,342 | ||||||
| Total operating expenses | 450,476 | 439,241 | 502,754 | ||||||
| Income (loss) from operations | 24,876 | (77,211 | ) | (212,120 | ) | ||||
| Other (expense) income: | |||||||||
| Interest income | 5,407 | 7,078 | 3,024 | ||||||
| Interest expense | (49,598 | ) | (50,149 | ) | (37,119 | ) | |||
| Loss on extinguishment of debt | — | (13,933 | ) | — | |||||
| Other (expense)<br> income | (9,441 | ) | (15,886 | ) | 4,176 | ||||
| Loss before income tax | (28,756 | ) | (150,101 | ) | (242,039 | ) | |||
| Income tax (expense)<br> benefit | (27,350 | ) | (1,483 | ) | 5,471 | ||||
| Net loss attributable<br> to common stockholders | $ | (56,106 | ) | $ | (151,584 | ) | $ | (236,568 | ) |
| Net loss attributable to common stockholders<br> per common share — basic and diluted | $ | (0.18 | ) | $ | (0.51 | ) | $ | (0.82 | ) |
| Weighted-average common shares outstanding —<br> basic and diluted | 304,380,502 | 295,164,515 | 289,057,198 |
5
TABLE2
AmicusTherapeutics, Inc.
ConsolidatedBalance Sheets
(inthousands, except share and per share amounts)
| 2023 | |||||
| Assets | |||||
| Current assets: | |||||
| Cash and cash equivalents | 213,752 | $ | 246,994 | ||
| Investments in marketable securities | 36,194 | 39,206 | |||
| Accounts receivable | 101,099 | 87,632 | |||
| Inventories | 118,782 | 59,696 | |||
| Prepaid expenses<br> and other current assets | 34,909 | 49,533 | |||
| Total current assets | 504,736 | 483,061 | |||
| Operating lease right-of-use assets,<br> net | 22,278 | 26,312 | |||
| Property and equipment, less accumulated<br> depreciation of 28,775 and 25,429 at December 31, 2024 and 2023, respectively | 29,383 | 31,667 | |||
| Intangible assets, less accumulated<br> amortization of 5,802 and 2,510 at December 31, 2024 and December 31, 2023, respectively | 17,198 | 20,490 | |||
| Goodwill | 197,797 | 197,797 | |||
| Other non-current<br> assets | 13,641 | 18,553 | |||
| Total Assets | 785,033 | $ | 777,880 | ||
| Liabilities and Stockholders' Equity | |||||
| Current liabilities: | |||||
| Accounts payable | 12,947 | $ | 15,120 | ||
| Accrued expenses and other current<br> liabilities | 127,300 | 144,245 | |||
| Operating lease<br> liabilities | 8,455 | 8,324 | |||
| Total current liabilities | 148,702 | 167,689 | |||
| Long-term debt | 390,111 | 387,858 | |||
| Operating lease liabilities | 45,078 | 48,877 | |||
| Other non-current<br> liabilities | 7,097 | 13,282 | |||
| Total liabilities | 590,988 | 617,706 | |||
| Commitments and contingencies | |||||
| Stockholders' equity: | |||||
| Common stock, 0.01 par value, 500,000,000<br> shares authorized, 299,041,653 and 293,594,209 shares issued and outstanding at December 31, 2024 and 2023, respectively | 2,944 | 2,918 | |||
| Additional paid-in capital | 2,926,115 | 2,836,018 | |||
| Accumulated other comprehensive gain (loss): | |||||
| Foreign currency translation adjustment | 5,302 | 5,429 | |||
| Unrealized loss on available-for-sale<br> securities | (207 | ) | (188 | ) | |
| Warrants | 71 | 71 | |||
| Accumulated deficit | (2,740,180 | ) | (2,684,074 | ) | |
| Total stockholders'<br> equity | 194,045 | 160,174 | |||
| Total Liabilities<br> and Stockholders' Equity | 785,033 | $ | 777,880 |
All values are in US Dollars.
6
TABLE3
AmicusTherapeutics, Inc.
Reconciliationof Non-GAAP Financial Measures
(inthousands)
(Unaudited)
| Years Ended December 31, | ||||||
|---|---|---|---|---|---|---|
| 2024 | 2023 | 2022 | ||||
| Total GAAP operating expenses | $ | 450,476 | $ | 439,241 | $ | 502,754 |
| Research and development: | ||||||
| Share-based compensation | 15,969 | 21,469 | 25,089 | |||
| Selling, general and administrative: | ||||||
| Share-based compensation | 68,936 | 64,608 | 51,423 | |||
| Loss on impairment of assets | — | 1,134 | 6,616 | |||
| Restructuring charge | 9,188 | — | — | |||
| Changes in fair value of contingent consideration payable | — | 2,583 | 1,078 | |||
| Depreciation and amortization | 8,547 | 7,873 | 5,342 | |||
| Total Non-GAAP operating expense adjustments | 102,640 | 97,667 | 89,548 | |||
| Total Non-GAAP operating expenses | $ | 347,836 | $ | 341,574 | $ | 413,206 |
7
TABLE4
AmicusTherapeutics, Inc.
Reconciliationof Non-GAAP Financial Measures
(inthousands, except share and per share amounts)
(Unaudited)
| Three Months Ended<br> <br>December 31, | Years Ended<br> <br>December 31, | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 2024 | 2023 | 2024 | 2023 | |||||||||
| GAAP net income (loss) | $ | 14,739 | $ | (33,843 | ) | $ | (56,106 | ) | $ | (151,584 | ) | |
| Share-based compensation | 19,217 | 18,095 | 84,905 | 86,077 | ||||||||
| Loss on impairment of assets | — | — | — | 1,134 | ||||||||
| Changes in fair value of contingent consideration payable | — | — | — | 2,583 | ||||||||
| Depreciation and amortization | 2,041 | 2,182 | 8,547 | 7,873 | ||||||||
| Loss on extinguishment of debt | — | 13,933 | — | 13,933 | ||||||||
| Restructuring charges | — | — | 9,188 | — | ||||||||
| Income tax (benefit) expense | (6,805 | ) | 2,183 | 27,350 | 1,483 | |||||||
| Non-GAAP net income (loss) | $ | 29,192 | $ | 2,550 | $ | 73,884 | $ | (38,501 | ) | |||
| Non-GAAP net<br> income (loss) attributable to common stockholders per common share — basic | $ | 0.10 | $ | 0.01 | $ | 0.24 | $ | (0.13 | ) | |||
| Non-GAAP net<br> income (loss) attributable to common stockholders per common share — diluted | $ | 0.09 | $ | 0.01 | $ | 0.24 | $ | (0.13 | ) | |||
| Weighted-average common shares outstanding — basic | 306,136,125 | 300,648,503 | 304,380,502 | 295,164,515 | ||||||||
| Weighted-average common shares outstanding — diluted | 310,146,355 | 306,787,370 | 308,463,764 | 295,164,515 |
8
Exhibit 99.1
| AT THE FOREFRONT OF<br>THERAPIES FOR RARE DISEASES<br>FY24 Results<br>Conference Call<br>& Webcast<br>February 19, 2025 |
|---|
| 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 preclinical and clinical development of our<br>product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates,<br>commercialization plans, manufacturing and supply plans, financing plans, and the projected revenues and cash position for the Company. The inclusion of forward-looking statements<br>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 presentation may turn out to be wrong and<br>can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress,<br>timing, and outcomes of discussions with regulatory authorities and pricing and reimbursement authorities, are based on current information. Actual results may differ materially from<br>those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate<br>that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities may not grant or<br>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<br>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<br>Pombiliti and Opfolda in Europe, the UK, the US and other geographies; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other<br>safety issues; the potential that we may not be able to manufacture or supply sufficient clinical or commercial products; and the potential that we will need additional funding to<br>complete all of our studies, the manufacturing, and commercialization of our products. With respect to statements regarding corporate financial guidance and financial goals and the<br>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<br>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<br>year ended December 31, 2024 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<br>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. |
| --- |
| A Rare Company<br>A unique story in biotech with significant revenue growth and profitability<br>3 1 At CER: Constant Exchange Rates<br>$528M<br>2024 Total Revenue<br>(+33% Growth)1<br>$1B+<br>Total Revenues<br>Expected in 2028<br>65-85%<br>FY 2025<br>Pombiliti+Opfolda<br>Revenue Growth1<br>First Oral<br>Precision<br>Medicine for<br>Fabry Disease<br>First Two-Component Therapy 10-15% for Pompe Disease<br>FY 2025<br>Galafold Revenue<br>Growth1<br>FY 2024<br>Non-GAAP<br>Profitability<br>Achieved<br>Leverageable<br>Global<br>Commercial<br>Organization |
| --- |
| 4<br>2024 Results<br>1 Full-Year 2024 guidance provided at CER (Constant Exchange Rates) using Full-Year 2023 Average Exchange Rates<br>We closed 2024 with exceptional growth of 33% and strong momentum heading into 2025<br>FY 2024 Guidance Results<br>Total Revenue Growth1 30% to 32% 33%<br>Galafold Revenue Growth1 16% to 18% 19%<br>Pombiliti + Opfolda Revenue1 $69M to $71M $70.2M<br>Non-GAAP Operating Expense $340M to $350M $347.8M<br>Non-GAAP Net Income Non-GAAP Profitable $73.9M |
| --- |
| 5 1 CER: Constant Exchange Rates<br>2025 Strategic Priorities<br>Deliver total revenue growth of 17-24% at CER1<br>Double-digit Galafold® revenue growth of 10-15% at CER1<br>Pombiliti®+ Opfolda® revenue growth of 65-85% at CER1<br>Advance ongoing studies to broaden labels and strengthen<br>scientific leadership in Fabry and Pompe diseases<br>Deliver positive GAAP net income during H2 2025 |
| --- |
| 6<br>Galafold® (migalastat)<br>Continued Growth<br>Building a leadership position<br>in the treatment of Fabry disease |
| --- |
| 7<br>Only approved oral treatment in Fabry disease and standard of care for amenable patients<br>2024 Galafold Success (as of December 31, 2024)<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>$458.1M<br>FY24 Galafold<br>Revenue<br>+19%<br>FY24 Galafold<br>Growth at CER1<br>65%<br>Share of Treated<br>Amenable Patients<br>1 CER: Constant Exchange Rates<br>2 As of YE 2024 |
| --- |
| 8<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> Global mix of naïve (~60%) and switch<br>(~40%) patients2<br> Expanding market through uptake in<br>naïve population as well as geographic<br>and label expansion<br> Maintaining >90% adherence and<br>compliance through HCP and patient<br>education and support<br> Expect non-linear quarterly growth<br>to continue due to uneven ordering<br>patterns and FX fluctuations<br>Galafold Performance<br>Strong FY 2025 Galafold growth guidance of 10-15% at CER1<br>Q1<br>$99M<br>Q2<br>$111M<br>Q3<br>$120M<br>Q4<br>$128M<br>+10-15%1<br>FY 2024 Galafold reported revenue of $458.1M (+19% growth at CER)<br>1 CER: Constant Exchange Rates<br>2 Data on file |
| --- |
| 9<br>Multiple initiatives leveraging AI and family screening to improve diagnosis of Fabry disease<br>Improving Diagnosis of Fabry Disease<br> 580K+ medical records screened<br> 100 people with highest risk of Fabry identified<br> Outreach ongoing to offer genetic testing<br> Initial findings from Fabry pilot programs in U.K.<br>– Minority and low-income groups significantly under-represented<br>– >90% of diagnosed Fabry population white<br>– ~85% from the least deprived areas<br> Initiative already identified low-income families who otherwise<br>wouldn’t have been diagnosed<br>Collaboration using AI to diagnose Fabry Collaboration for change in health disparity<br>Additional initiatives in several countries ongoing leveraging AI and/or targeted screening |
| --- |
| 10<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>+ |
| --- |
| 11<br>Pombiliti + Opfolda Performance<br>Successful first full year of launch in the U.S., Germany, Austria, Spain and the U.K.<br>FY23 FY24<br>U.S.<br>Ex-U.S. 43%<br>57%<br>Annual Revenue 2024 Geographic Mix<br>Q1<br>$11M<br>Q2<br>$16M<br>Q3<br>$21M<br>Q4<br>$22M<br>$70.2M<br>$11.6M<br>1 CER: Constant Exchange Rates |
| --- |
| 12<br>~220 individuals treated or scheduled provides strong foundation for 2025<br>Pombiliti + Opfolda Global Launch Metrics<br>• ~220 patients have been treated or scheduled to<br>be treated with commercial product<br>– ~209 treated patients<br>– ~25 new prescriptions in Q4<br>• All eligible clinical trial patients from launched<br>markets on commercial therapy by end of 1H24<br>• New commercial patients time through U.S.<br>insurance process optimized to <30 days<br>• Patients starting Pombiliti + Opfolda at<br>proportional rate to the respective market share<br>120<br>220<br>4Q23 1Q24 2Q24 3Q24 4Q24<br>Global Launch: Individuals Treated or Scheduled<br>~ |
| --- |
| 13<br>Pombiliti + Opfolda Growth Drivers<br>Amicus focused on key drivers of growth in 2025<br> Increasing number of net new patients<br> Increasing depth and breadth of prescribers<br> Expect to launch in up to 10 new countries<br>throughout 2025<br> Continuing to drive differentiation through<br>evidence generation and real-world evidence<br> Anticipate 90%+ compliance and adherence<br>1 At CER: Constant Exchange Rates<br>$12M<br>$70M<br>FY23 FY24 FY25<br>Pombiliti + Opfolda Revenue<br>+65-85%1 |
| --- |
| 14<br>New reimbursement agreements completed in:<br>Regulatory approvals anticipated in 2025:<br>3 new regulatory approvals and up to 10 new launch countries in 2025<br>Geographic Expansion<br> Recently approved in Australia<br> Regulatory approvals in Canada and Japan also<br>anticipated in 2025<br>ITALY CZECH REPUBLIC SWITZERLAND<br>CANADA<br>SWEDEN<br>AUSTRALIA JAPAN<br>Combined ~150-200 people 18+ living with LOPD<br>and being treated with a Pompe therapy<br>Regulatory<br>Reimbursement<br> In 2025, expect to launch in up to 10 new countries,<br>including 5 recent agreements<br>>650 LOPD patients 18+ in those 10 countries<br> First commercial patients from those new launch countries<br>anticipated over H1 2025<br> Anticipate >20 individuals switching from clinical trials<br>or early access programs in new countries in 2025<br>Combined ~325-375 people 18+ living with LOPD<br>and being treated with a Pompe therapy<br>NETHERLANDS |
| --- |
| 15<br>Continuing to build the body of evidence to support planned label expansion<br>Ongoing Clinical Studies<br>Clinical study in children with late-onset<br>Pompe disease (LOPD)<br>Clinical study in children with<br>infantile-onset Pompe disease (IOPD)<br>Amicus registry adding to evidence on<br>differentiated MOA and long-term effect |
| --- |
| 16<br>Strong presence at WORLDSymposium demonstrating leadership in Fabry and Pompe diseases<br>WORLDSymposiumTM 2025<br>• 2 platform and 20 poster presentations<br>• Expanding our real-world evidence of<br>Galafold with high utility<br>• Further evidence supporting use of<br>Pombiliti + Opfolda<br>• Significant conference focus on improving<br>diagnosis and benefits of early treatment |
| --- |
| 17<br>Corporate Outlook<br>Delivering on our mission for patients<br>and shareholders |
| --- |
| 18<br>FY 2024 Select Financial Results<br>Q4 Full Year<br>(in thousands, except per share data) Dec. 31, 2024 Dec. 31, 2023 Dec. 31, 2024 Dec. 31, 2023<br>Net product sales $ 149,706 $ 115,082 $ 528,295 $ 399,356<br>Cost of goods sold 14,836 11,324 52,943 37,326<br>GAAP operating expenses 118,900 107,450 450,476 439,241<br>Non-GAAP operating expenses 97,641 87,173 347,836 341,574<br>GAAP net income (loss) 14,739 (33,843) (56,106) (151,584)<br>Non-GAAP net income (loss) 29,192 2,550 73,884 (38,501)<br>GAAP net income (loss) per share – basic and<br>diluted $ 0.05 $ (0.11) $ (0.18) $ (0.51)<br>Non-GAAP net income (loss) per share - basic $ 0.10 $ 0.01 $ 0.24 $ (0.13)<br>Non-GAAP net income (loss per share - diluted $ 0.09 $ 0.01 $ 0.24 $ (0.13)<br>QTD December 31, 2024 basic weighted-average common shares outstanding: 306,136,125; QTD December 31, 2023 weighted-average common shares outstanding: 300,648,503<br>YTD December 31, 2024, basic weighted-average common shares outstanding: 304,380,502; YTD December 31, 2023, weighted-average common shares outstanding: 295,164,515<br>FY 2024 revenue of $528M, up 32% and non-GAAP net income of $73.9M |
| --- |
| 19<br>Full-Year 2025 Financial Guidance<br>FY 2025 Financial Guidance1 2025<br>Total Revenue Growth1 17% to 24%<br>Galafold Revenue Growth1 10% to 15%<br>Pombiliti + Opfolda Revenue Growth1 65% to 85%<br>Gross Margin Mid 80%<br>Non-GAAP Operating Expense $350M to $370M<br>GAAP Net Income Positive during 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 year-end 2024<br>rates could lead to a ~$4M move in Total<br>Reported Revenues in 2025 |
| --- |
| 20<br>Distribution of Galafold Quarterly Sales<br>Distribution of Galafold® Revenue by Quarter over Past 5 Years<br>Q1 Q2 Q3 Q4<br>5 Year Avg. 22% 24% 26% 28% |
| --- |
| 21<br>A unique story in biotech with significant revenue growth and profitability<br>Two Approved<br>Therapies<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 |
| --- |
| Appendix |
| --- |
| 23<br>Reconciliation of Non-GAAP Financial Measures |
| --- |
| 24<br>Reconciliation of Non-GAAP Financial Measures (Cont’d) |
| --- |
| 25<br>Exchange Rates<br>Q4 2024 Currency Average Rates<br>FX Rates Q4 2023 Q4 2024 Variance<br>USD/EUR 1.076 1.067 (0.8%)<br>USD/GBP 1.241 1.282 3.3%<br>USD/JPY 0.007 0.007 (3.0%)<br>Year-End 2024 vs. Full-Year 2024 Currency Average Rates1<br>FX Rates Year-End 2024 Full-Year 2024 Variance<br>USD/EUR 1.041 1.082 (3.8%)<br>USD/GBP 1.255 1.278 (1.8%)<br>USD/JPY 0.006 0.007 (3.5%)<br>1 The variance between Year-End 2024 and Full-Year 2024 USD exchange rates of ~4% would translate into a<br>negative impact of ~$15M on Total Revenue in 2025 if rates were to remain at Year-End 2024 level |
| --- |