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 28, 2024
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 Operations and FinancialCondition
On February 28, 2024, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the fiscal year ended December 31, 2023. A copy of this press release is attached hereto as Exhibit 99.1. The Company will host a conference call and webcast on February 28, 2024 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 28, 2024 |
| 99.2 | February 28, 2024 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 28, 2024 | By: | /s/<br> Ellen S. Rosenberg |
| Name: | Ellen S. Rosenberg | |
| Title: | Chief Legal Officer and Corporate Secretary |
Exhibit 99.1
Amicus TherapeuticsAnnounces Full-Year 2023 Financial Results and Corporate Updates
2023 TotalRevenue of $399.4M, a 21% Increase Year-over-Year
Strong PatientDemand Continues for Pombiliti^™^ + Opfolda^™^ in the U.S., U.K., and Germany
Projecting2024 Galafold^®^ Revenue Growth of 11-16% at CER
AnticipatingFull-Year Non-GAAP Profitability in 2024
ConferenceCall and Webcast Today at 8:30 a.m. ET
PRINCETON, NJ,Feb. 28, 2024 – 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, 2023.
“In 2023, Amicus made tremendous progress across all our strategic priorities,” said Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc. “We strengthened our leadership position in Fabry and Pompe disease globally and achieved our goal of non-GAAP profitability in the fourth quarter. Patient demand for Galafold exceeded our expectations and grew at the highest rate seen in the last four years, and we continue to be excited by the long-term growth potential of this important medicine. We also successfully launched our second commercial therapy, Pombiliti + Opfolda, in the three largest Pompe disease markets. In 2024, we will continue to drive significant top line revenue growth supported by sustained double-digit Galafold performance and the successful ongoing global commercial launch of Pombiliti + Opfolda, putting us on track for our first full year of non-GAAP profitability. Amicus is at a major inflection point and strongly positioned to continue to advance our mission of delivering groundbreaking new medicines to thousands of people living with rare diseases and creating value for our shareholders.”
Corporate Highlights:
| · | Total revenues for the full-year 2023 were $399.4 million, up 21%, reflecting operational growth<br> measured at constant exchange rates (CER)^1^ of 20% and favorable currency impact<br> of $2.7 million or 1%. Fourth quarter total revenues were $115.1 million, up 31%, or 27%<br> at CER. | |||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| (in thousands) | Three Months Ended<br> December 31, | Year over Year % <br> Growth | Twelve Months Ended<br> <br>December 31, | Year over Year %<br> <br>Growth | ||||||||||||||||
| --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- |
| 2023 | 2022 | Reported | at CER^1^ | 2023 | 2022 | Reported | at CER^1^ | |||||||||||||
| Galafold^®^ | 106,600 | 87,989 | 21 | % | 18 | % | 387,777 | 329,046 | 18 | % | 17 | % | ||||||||
| Pombiliti^™^+ Opfolda^™^ | 8,482 | 107 | n/a | n/a | 11,579 | 187 | n/a | n/a | ||||||||||||
| Net Product Revenues | $ | 115,082 | $ | 88,096 | 31 | % | 27 | % | $ | 399,356 | $ | 329,233 | 21 | % | 20 | % | ||||
| · | Galafold (migalastat) net product sales for the full-year 2023 were $387.8 million, representing<br> a year-over-year increase of 18%, or 17% at CER. Fourth quarter net product sales were $106.6<br> million. At the end of 2023, there were >2,400 people living with Fabry disease on Galafold<br> following a year of increased demand. | |||||||||||||||||||
| --- | --- | |||||||||||||||||||
| · | Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) net product sales for the full-year 2023 were $11.6 million. Fourth quarter net product sales were $8.5 million. The commercial<br> launch of Pombiliti + Opfolda is underway in the three largest markets with 120 patients<br> on treatment with commercial product or scheduled to be treated as of early January and continued strong patient demand. | |||||||||||||||||||
| --- | --- | |||||||||||||||||||
| · | Eleven posters and an oral presentation highlighting Amicus’ development programs in Fabry disease and Pompe disease presented at the 20th Annual WORLDSymposium™.<br> Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) honored with the 2024 New Treatment<br> Award, which recognizes important achievements in advancing new treatments approved for lysosomal<br> diseases. | |||||||||||||||||||
| --- | --- | |||||||||||||||||||
| · | On a GAAP basis, net loss in the fourth quarter of 2023 was $33.8 million. The Company achieved non-GAAP profitability^3^ in the fourth quarter of 2023 of $2.6 million. | |||||||||||||||||||
| --- | --- |
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Full-Year 2023 Financial Results
| · | Total<br> revenue in the full-year 2023 was $399.4 million, a year-over-year increase of 21% from total<br> revenue of $329.2 million in the full-year 2022. On a constant currency basis, full-year<br> 2023 total revenue growth was 20%. Reported revenue had a favorable currency impact of approximately<br> $2.7 million, or 1%. |
|---|---|
| · | Total<br> GAAP operating expenses of $439.2 million for the full-year 2023 decreased by 13% as compared<br> to $502.8 million for the full-year 2022. |
| --- | --- |
| · | Total<br> non-GAAP operating expenses of $341.6 million for the full-year 2023 decreased by 17% as<br> compared to $413.2 million for the full-year 2022. |
| --- | --- |
| · | GAAP<br> net loss was $151.6 million, or $0.51 per share, for the full-year 2023, and was reduced<br> compared to a net loss of $236.6 million, or $0.82 per share, for the full-year 2022. |
| --- | --- |
| · | Non-GAAP<br> net loss was $38.5 million, or $0.13 per share, for the full-year 2023, and was reduced compared<br> to a net loss of $152.5 million, or $0.53 per share, for the full-year 2022. |
| --- | --- |
| · | Cash,<br> cash equivalents, and marketable securities totaled $286.2 million at December 31, 2023,<br> compared to $293.6 million at December 31, 2022. |
| --- | --- |
2024 FinancialGuidance
| · | For<br> the full-year 2024, the Company anticipates total Galafold revenue growth between 11% and<br> 16% at CER^1^driven by continued underlying demand from both switch and treatment-naïve<br> patients, geographic expansion, label extensions, the continued diagnosis of new Fabry patients,<br> and commercial execution across all major markets, including the U.S., EU, U.K., and Japan. |
|---|---|
| · | Non-GAAP<br> operating expense guidance for the full-year 2024 is $345 million to $365 million, driven<br> by disciplined expense management offset by continued investment in Galafold, Pombiliti +<br> Opfolda clinical studies, as well as global launch activities^4^. |
| --- | --- |
Amicusis focused on the following key strategic priorities in 2024:
| · | Delivering<br> double-digit Galafold revenue growth (11-16% at CER) |
|---|---|
| · | Executing<br> multiple successful launches of Pombiliti + Opfolda |
| --- | --- |
| · | Advancing<br> ongoing studies to support medical and scientific leadership in Fabry and Pompe diseases |
| --- | --- |
| · | Achieving<br> full year non-GAAP profitability^2^ |
| --- | --- |
^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. Full-year 2024 Galafold revenue guidance utilizes actual exchange rate as of December 31, 2023.
^2^Based on projections of Amicus’ non-GAAP Net (Loss) Income under current operating plans, which includes successful Pombiliti + Opfolda launches and continued Galafold growth. 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.
^3^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.
^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 28, 2024, at 8:30 a.m. ET to discuss the full-year 2023 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.
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U.S. INDICATIONSAND 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. IMPORTANTSAFETY INFORMATION
ADVERSEREACTIONS
The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia.
USE IN SPECIFICPOPULATIONS
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 the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf.
About Pombiliti+ Opfolda
Pombiliti + Opfolda, is a two-component therapy that consists of cipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that’s designed to reduce loss of enzyme activity in the blood.
U.S. INDICATIONSAND USAGE
POMBILITI in combination with OPFOLDA is indicated for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).
SAFETY INFORMATION
HYPERSENSITIVITYREACTIONS INCLUDING ANAPHYLAXIS: Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readilyavailable. If a severe hypersensitivity reaction occurs, POMBILITI should be discontinued immediately and appropriate medical treatmentshould be initiated. INFUSION-ASSOCIATED REACTIONS (IARs): If severe IARs occur, immediately discontinue POMBILITI and initiate appropriatemedical treatment. RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients susceptible to fluid volume overload, orthose with acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of serious exacerbationof their cardiac or respiratory status during POMBILITI infusion. See PI for complete Boxed Warning. CONTRAINDICATION: POMBILITI in combination with Opfolda is contraindicated in pregnancy. EMBRYO-FETAL TOXICITY: May cause embryo-fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose. Adverse Reactions: Most common adverse reactions ≥ 5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia. Please see full PRESCRIBING INFORMATION, including BOXED WARNING, for POMBILITI (cipaglucosidase alfa-atga) LINK and full PRESCRIBING INFORMATION for OPFOLDA (miglustat) LINK.
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About AmicusTherapeutics
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-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.
Forward Looking 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, 2023 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
FOLD-G
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TABLE 1
Amicus Therapeutics, Inc.
ConsolidatedStatements of Operations
(in thousands,except share and per share amounts)
| Years Ended December 31, | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| 2023 | 2022 | 2021 | |||||||
| Net product sales | $ | 399,356 | $ | 329,233 | $ | 305,514 | |||
| Cost of goods sold | 37,326 | 38,599 | 34,466 | ||||||
| Gross profit | 362,030 | 290,634 | 271,048 | ||||||
| Operating expenses: | |||||||||
| Research and development | 152,381 | 276,677 | 272,049 | ||||||
| Selling, general, and administrative | 275,270 | 213,041 | 192,710 | ||||||
| Changes in fair value of contingent consideration payable | 2,583 | 1,078 | 6,514 | ||||||
| Loss on impairment of assets | 1,134 | 6,616 | — | ||||||
| Depreciation and amortization | 7,873 | 5,342 | 6,209 | ||||||
| Total operating expenses | 439,241 | 502,754 | 477,482 | ||||||
| Loss from operations | (77,211 | ) | (212,120 | ) | (206,434 | ) | |||
| Other (expense) income: | |||||||||
| Interest income | 7,078 | 3,024 | 509 | ||||||
| Interest expense | (50,149 | ) | (37,119 | ) | (32,471 | ) | |||
| Loss on extinguishment of debt | (13,933 | ) | — | (257 | ) | ||||
| Other (expense) income | (15,886 | ) | 4,176 | (2,901 | ) | ||||
| Loss before income tax | (150,101 | ) | (242,039 | ) | (241,554 | ) | |||
| Income tax (expense) benefit | (1,483 | ) | 5,471 | (8,906 | ) | ||||
| Net loss attributable to common stockholders | $ | (151,584 | ) | $ | (236,568 | ) | $ | (250,460 | ) |
| Net loss attributable to common stockholders per common share — basic and diluted | $ | (0.51 | ) | $ | (0.82 | ) | $ | (0.92 | ) |
| Weighted-average common shares outstanding — basic and diluted | 295,164,515 | 289,057,198 | 271,421,986 |
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TABLE 2
Amicus Therapeutics, Inc.
ConsolidatedBalance Sheets
(in thousands,except share and per share amounts)
| 2022 | |||||
| Assets | |||||
| Current assets: | |||||
| Cash and cash equivalents | 246,994 | $ | 148,813 | ||
| Investments in marketable securities | 39,206 | 144,782 | |||
| Accounts receivable | 87,632 | 66,196 | |||
| Inventories | 59,696 | 23,816 | |||
| Prepaid expenses and other current assets | 49,533 | 40,209 | |||
| Total current assets | 483,061 | 423,816 | |||
| Operating lease right-of-use assets, net | 26,312 | 29,534 | |||
| Property and equipment, less accumulated depreciation of 25,429 and 22,281 at December 31, 2023 and 2022, respectively | 31,667 | 30,778 | |||
| Intangible assets, less accumulated amortization of 2,510 and 0 at December 31, 2023 and December 31, 2022, respectively | 20,490 | 23,000 | |||
| Goodwill | 197,797 | 197,797 | |||
| Other non-current assets | 18,553 | 19,242 | |||
| Total Assets | 777,880 | $ | 724,167 | ||
| Liabilities and Stockholders' Equity | |||||
| Current liabilities: | |||||
| Accounts payable | 15,120 | $ | 15,413 | ||
| Accrued expenses and other current liabilities | 144,245 | 93,636 | |||
| Contingent consideration payable | — | 21,417 | |||
| Operating lease liabilities | 8,324 | 8,552 | |||
| Total current liabilities | 167,689 | 139,018 | |||
| Long-term debt | 387,858 | 391,990 | |||
| Operating lease liabilities | 48,877 | 51,578 | |||
| Other non-current liabilities | 13,282 | 18,534 | |||
| Total liabilities | 617,706 | 601,120 | |||
| Commitments and contingencies | |||||
| Stockholders' equity: | |||||
| Common stock, 0.01 par value, 500,000,000 shares authorized, 293,594,209 and 281,108,273 shares issued and outstanding at December 31, 2023 and 2022, respectively | 2,918 | 2,815 | |||
| Additional paid-in capital | 2,836,018 | 2,664,744 | |||
| Accumulated other comprehensive gain (loss): | |||||
| Foreign currency translation adjustment | 5,429 | (11,989 | ) | ||
| Unrealized loss on available-for-sale securities | (188 | ) | (116 | ) | |
| Warrants | 71 | 83 | |||
| Accumulated deficit | (2,684,074 | ) | (2,532,490 | ) | |
| Total stockholders' equity | 160,174 | 123,047 | |||
| Total Liabilities and Stockholders' Equity | 777,880 | $ | 724,167 |
All values are in US Dollars.
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TABLE 3
Amicus Therapeutics, Inc.
Reconciliationof Non-GAAP Financial Measures
(in thousands)
(Unaudited)
| Years Ended December 31, | ||||||
|---|---|---|---|---|---|---|
| 2023 | 2022 | 2021 | ||||
| Total GAAP operating expenses | $ | 439,241 | $ | 502,754 | $ | 477,482 |
| Research and development: | ||||||
| Share-based compensation | 21,469 | 25,089 | 17,340 | |||
| Selling, general and administrative: | ||||||
| Share-based compensation | 64,608 | 51,423 | 40,498 | |||
| Loss on impairment of assets | 1,134 | 6,616 | — | |||
| Changes in fair value of contingent consideration payable | 2,583 | 1,078 | 6,514 | |||
| Depreciation and amortization | 7,873 | 5,342 | 6,209 | |||
| Total Non-GAAP operating expense adjustments | 97,667 | 89,548 | 70,561 | |||
| Total Non-GAAP operating expenses | $ | 341,574 | $ | 413,206 | $ | 406,921 |
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TABLE 4
Amicus Therapeutics, Inc.
Reconciliationof Non-GAAP Financial Measures
(in thousands,except share and per share amounts)
(Unaudited)
| Three Months Ended<br> <br>December 31, | Years Ended<br> <br>December 31, | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 2023 | 2022 | 2023 | 2022 | |||||||||
| GAAP net loss | $ | (33,843 | ) | $ | (55,865 | ) | $ | (151,584 | ) | $ | (236,568 | ) |
| Share-based compensation | 18,095 | 18,626 | 86,077 | 76,512 | ||||||||
| Loss on impairment of assets | — | — | 1,134 | 6,616 | ||||||||
| Changes in fair value of contingent consideration payable | — | 1,584 | 2,583 | 1,078 | ||||||||
| Depreciation and amortization | 2,182 | 1,311 | 7,873 | 5,342 | ||||||||
| Loss on extinguishment of debt | 13,933 | — | 13,933 | — | ||||||||
| Income tax expense (benefit) | 2,183 | (14,214 | ) | 1,483 | (5,471 | ) | ||||||
| Non-GAAP net income (loss) | $ | 2,550 | $ | (48,558 | ) | $ | (38,501 | ) | $ | (152,491 | ) | |
| Non-GAAP net income (loss) attributable to common stockholders per common share — basic and diluted | $ | 0.01 | $ | (0.17 | ) | $ | (0.13 | ) | $ | (0.53 | ) | |
| Weighted-average common shares outstanding — basic and diluted | 300,648,503 | 289,602,648 | 295,164,515 | 289,057,198 |
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Exhibit 99.2
| AT THE FOREFRONT OF<br>THERAPIES FOR RARE DISEASES<br>FY23 Results<br>Conference Call<br> & Webcast<br>February 28, 2024 |
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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 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 press release 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<br>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<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, 2023 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<br>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<br>circumstances after the date hereof.<br>Non-GAAP Financial Measures<br>In addition to financial information prepared in accordance with U.S. GAAP, this presentation also contains adjusted financial measures that we believe provide investors and<br>management with supplemental information relating to operating performance and trends that facilitate comparisons between periods and with respect to projected information. These<br>adjusted financial measures are non-GAAP measures and should be considered in addition to, but not as a substitute for, the information prepared in accordance with U.S. GAAP. We<br>typically exclude certain GAAP items that management does not believe affect our basic operations and that do not meet the GAAP definition of unusual or non-recurring items. Other<br>companies may define these measures in different ways. When we provide our expectation for non-GAAP operating expenses on a forward-looking basis, a reconciliation of the<br>differences between the non-GAAP expectation and the corresponding GAAP measure generally is not available without unreasonable effort due to potentially high variability,<br>complexity and low visibility as to the items that would be excluded from the GAAP measure in the relevant future period, such as unusual gains or losses. The variability of the excluded<br>items may have a significant, and potentially unpredictable, impact on our future GAAP results. |
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| 3<br>A leading biotech company with >$500M of sales projected in 2024<br>A Rare Company<br>LEVERAGEABLE<br>GLOBAL<br>COMMERCIAL<br>ORGANIZATION<br> >500 EMPLOYEES<br>in 20+ Countries<br>First Two-Component Therapy<br>for Pompe Disease<br>2<br>APPROVED<br>THERAPIES<br>Combined Peak<br>Revenue Potential<br>$1.5B – $2B<br>Non-GAAP<br>PROFITABILITY<br>Q4 2023<br>ACHIEVED<br>World Class<br>Clinical<br>Development<br>Capabilities<br>$399M<br>in 2023 Revenue<br>21%<br>Increase Year-Over-Year<br>Expect Full Year<br>2024<br>Non-GAAP<br>Profitability |
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| 1<br>2<br>3<br>4<br>A Transformative<br>Year Ahead for<br>Amicus<br>1CER: Constant Exchange Rates; 2024 Galafold revenue guidance utilizes actual exchange rate as of December 31, 2023<br>2024<br>Strategic<br>Priorities<br>4<br>Drive double-digit Galafold® revenue growth of 11-16% at CER1<br>Execute multiple successful launches of Pombiliti + Opfolda<br>Advance ongoing studies to support medical and scientific<br>leadership in Fabry and Pompe diseases<br>Achieve non-GAAP profitability for the full year |
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| 5<br>Galafold® (migalastat)<br>Continued Growth<br>Building a leadership position in the<br>treatment of Fabry disease |
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| 6<br>Galafold is the only approved oral treatment option in Fabry disease<br>2023 Galafold Success (as of December 31, 2023)<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://www.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>2,400+<br>Individuals<br>Treated<br>$388M<br>2023 Galafold<br>Revenue<br>17%<br>YoY Growth at CER1<br>60-65%<br>Share of Treated<br>Amenable Patients<br>1CER: Constant Exchange Rates |
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| 7<br>Galafold YTD reported revenue growth of +18% to $388M<br>FY16 FY17 FY18 FY19 FY20 FY21 FY22 FY23 FY24<br>$5M<br>$37M<br>$91M<br>$182M<br>$261M<br>$306M<br>$329M<br>Galafold Performance<br> Global mix of switch (~43%) and previously<br>untreated patients (~57%)2<br> Expect non-linear quarterly growth to continue<br>due to uneven ordering patterns<br>and FX fluctuations<br>FY24 revenue growth guidance to 11% to 16% at CER<br>1 At constant exchange rate (CER)<br>2 Data on file<br>Q1<br>$86M<br>Q2<br>$94M<br>Q3<br>$101M<br>Q4<br>$107M<br>$388M<br>$430M-450M1<br>Distribution of Galafold revenue<br>by quarter over previous 5 years:<br>Q1 Q2 Q3 Q4<br>5 Year Avg. 22% 24% 26% 28% |
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| 8<br>Key Growth Drivers for 2024<br>Building off a strong year with highest patient demand seen in last four years to lay the<br>groundwork for continued double-digit Galafold growth in 2024<br> Increasing patient identification through ongoing medical<br>education, screening, and improved diagnostics<br> Driving market share of treated amenable patients<br>through excellent execution<br> Expanding market through uptake in naïve population<br>as well as geographic and label expansion<br> Maintaining >90% adherence and compliance through HCP<br>and patient education and support<br>8 |
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| 9<br>Pombiliti (cipaglucosidase alfa-atga)<br>Opfolda (miglustat)<br>Potential to establish a new standard of care<br>for people living with late-onset Pompe disease<br>+ |
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| 10<br>Successful Global Launch of Pombiliti + Opfolda Underway<br>FY 2023 revenue of $11.6M ($8.5M in Q4 2023) provides strong foundation for 2024<br>Access and<br>Reimbursement<br>Positive interactions<br>with US, UK, and EU payors<br>Focus on broad patient<br>access<br>Country-by-country<br>reimbursement process<br>underway<br>Multiple launches expected<br>in 2H 2024<br>~120 patients treated with<br>commercial product or<br>scheduled to be treated<br>~105 patients from clinical<br>trials and early access<br>~15 new patients from<br>competitor ERTs or naïve<br>Very positive early feedback<br>from real-world experience<br>KOL Outreach<br>Successfully engaged with<br>top prescribers in each<br>approved country<br>Existing relationships with<br>HCPs at key treatment<br>centers<br>Ongoing disease education<br>Patient Demand<br>As of early January 2024<br>10<br>Focus in 2024 is on maximizing the number of patients on therapy by year end |
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| 11<br>Regulatory and Clinical Updates<br>Continuing to build the body of evidence and expand commercial access<br> >10 reimbursement dossiers and multiple<br>regulatory submissions throughout 2024<br> Ongoing clinical studies in children with late-onset<br>Pompe disease (LOPD) and infantile-onset Pompe<br>disease (IOPD)<br> Amicus registry for Pompe disease to continue<br>generating evidence on differentiated MOA and<br>long-term effect<br> Significant presence at WORLDSymposium 2024<br>with 11 posters and an oral presentation<br>highlighting work in Fabry and Pompe |
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| 12<br>Corporate Outlook<br>Delivering on our mission for patients<br>and shareholders |
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| 13<br>FY 2023 Select Financial Results<br>2023 revenue of $399.4M, up 20% at CER, and net loss significantly reduced<br>(in thousands, except per share data) Dec. 31, 2023<br>Dec. 31, 2022<br>Product Revenue $399,356 $329,233<br>Cost of Goods Sold 37,326 38,599<br>R&D Expense 152,381 276,677<br>SG&A Expense 275,270 213,041<br>Changes in Fair Value of Contingent Consideration 2,583 1,078<br>Loss on Impairment of Assets 1,134 6,616<br>Depreciation and Amortization 7,873 5,342<br>Loss from Operations (77,211) (212,120)<br>Interest Income 7,078 3,024<br>Interest Expense (50,149) (37,119)<br>Loss on Extinguishment of Debt (13,933) ―<br>Other (Expense) Income (15,886) 4,176<br>Income Tax (Expense) Benefit (1,483) 5,471<br>Net Loss (151,584) (236,568)<br>Net Loss Per Share (0.51) (0.82)<br>2023 weighted-average common shares outstanding: 295,164,515<br>2022 weighted-average common shares outstanding: 289,057,198 |
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| Financial Outlook and Path to Profitability<br>Clear strategy to build our business, advance our portfolio, and achieve profitability<br>14<br>Sustain Revenue<br>Growth<br>Deliver on<br>Financial Goals<br>Successfully Launch<br>Pombiliti + Opfolda<br>$399M FY23 revenue,<br>+21% YoY growth<br> >$500M in total<br>revenue in FY24<br>Galafold and<br>Pombiliti + Opfolda<br>expected to drive<br>strong double-digit<br>growth long term<br>Focused on disciplined<br>expense management<br>Achieve FY24<br>non-GAAP profitability1<br>1 Based on projections of Amicus non-GAAP Net (Loss) Income under current operating plans. We define non-GAAP Net (Loss) Income as GAAP Net (Loss) Income excluding the impact of share-based compensation expense, changes in fair value of contingent<br>consideration, depreciation and amortization, acquisition related income (expense), loss on extinguishment of debt, loss on impairment of assets, restructuring charges, and income taxes.<br>FY24 non-GAAP operating<br>expense guidance of<br>$345M-$365M |
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| 15<br>Accelerating<br>total revenue<br>growth<br>Positioned for Significant Value Creation in 2024<br>Unlocking the value of two unique commercial therapies in sizeable and growing markets<br>1 Non-GAAP Net (Loss) Income defined as GAAP Net (Loss) Income excluding the impact of stock-based compensation expense, changes in fair value of contingent consideration, loss on impairment of<br>assets, depreciation and amortization, acquisition related income (expense), loss on extinguishment of debt, restructuring charges and income taxes.<br>Clear line of<br>sight to<br>generating<br>positive<br>cashflow<br>Delivering<br>full-year<br>non-GAAP1<br>profitability |
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| Appendix |
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| 17<br>Appendix I |
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| 18<br>Appendix II |
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| 19<br>Environmental, Social, & Governance (ESG) Snapshot<br>Board of Directors<br>Committed to ongoing Board refreshment and diversity of<br>background, gender, skills, and experience:<br>80% Board<br>Independence<br>60% Overall Board<br>Diversity<br>Address a rare genetic disease<br>First-in-class or best-in-class<br>Impart meaningful benefit for patients<br>517<br>Global Employees<br>58%<br>% Female Employees<br>Who We Serve Our mission is to drive<br>sustainability with<br>our partners by<br>incorporating<br>environmental and<br>sustainability principles<br>into all our commercial<br>relationships<br>Pledge for a Cure<br>Designate a portion of product revenue back into<br>R&D for that specific disease until there is a cure.<br>Programs we<br>invest in<br>have 3 key<br>characteristics<br>3 Female<br>2 Veteran Status<br>1 African American<br>Director Diversity<br>Leverage employee capabilities and expertise to provide a<br>culture that drives performance and ultimately attracts,<br>energizes, and retains critical talent.<br>Employee Recruitment,<br>Engagement, & Retention<br>Pulse surveys reveal employees feel high personal<br>satisfaction in their job, are proud of their work<br>and what they contribute to the community<br>Career Development<br>Reimagined performance management process to<br>measure the what and the how, rewarding those who<br>role-model our Mission-focused Behaviors.<br>Committed to producing<br>transformative medicines for<br>people living with rare diseases<br>while practicing environmental<br>responsibility and adhering to<br>sustainability best practices in our<br>operations.<br>Environmental<br>Management<br>0% Amicus-owned Direct Manufacturing<br>and Related GHG Emissions<br>Diversity, Equity, &<br>Inclusion (DEI)<br>580<br>Volunteer<br>hours (U.S.):<br>22<br>Amicus-supported<br>community programs:<br>32 patients /24countries<br>Expanded Access through Jan 2024:<br>Pricing PROMISE<br>Contributions allocated:<br>$2,288,998 U.S.<br>$954,349 Intl.<br>Charitable Giving<br>Committed to never raising the annual price of<br>our products more than consumer inflation.<br>Pledge to support a more inclusive culture to impact<br>our employees, our communities, and society.<br>Goal of maintaining gender diversity and<br>increasing overall diversity throughout<br>our global workforce. |
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| 20<br>FX Sensitivity and Galafold Distribution of Quarterly Sales<br>Impact from Foreign Currency Q4 2023:<br>Currency Variances:<br>USD/<br>Q4 2022 Q4 2023 YoY Variance<br>EUR 1.021 1.076 5.4%<br>GBP 1.174 1.241 5.7%<br>JPY 0.007 0.007 (4.4%)<br>Full-year 2024 Revenue Sensitivity<br>Given the high proportion of Amicus revenue Ex-US (~60%), a change in exchange rates of<br>+/- 5% compared to year-end 2023 rates could lead to a $15M move in global reported<br>revenues in 2024.<br>Distribution of Galafold Revenue<br>by Quarter over Past 5 Years:<br>Q1 Q2 Q3 Q4<br>5 Year Avg. 22% 24% 26% 28% |
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| 21<br>Streamlined Rare Disease Pipeline with Focus on Fabry Disease and<br>Pompe Disease Franchises<br>INDICATION DISCOVERY PRECLINICAL PHASE 1/2 PHASE 3 REGULATORY COMMERCIAL<br>FABRY FRANCHISE<br>Galafold® (migalastat)<br>Fabry Genetic Medicines<br>Next-Generation Chaperone<br>POMPE FRANCHISE<br>Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat)<br>Pompe Genetic Medicines<br>OTHER<br>Discovery Programs |
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| Thank you |
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