1Q23 Revenue Growth of 14% at CER to $86.3M

On Track to Deliver Full-Year 2023 Galafold Revenue Growth of 12%-17% at CER

U.S. FDA Pre-approval Inspection for AT-GAA Complete; Approval Expected 3Q 2023

European Launch of Pombiliti®+Opfolda® Expected 3Q 2023

Non-GAAP Profitability Projected in 2H 2023

Conference Call and Webcast Today at 8:30 a.m. ET

PHILADELPHIA, May 10, 2023 (GLOBE NEWSWIRE) -- 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, 2023.

Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “We had an outstanding start to 2023 across our global business. In Q1, Galafold saw strong operational growth primarily driven by robust patient demand in our major markets. We are pleased with the outcome of the U.S. FDA inspection of the WuXi Biologics manufacturing facility and remain highly confident in the anticipated global approvals of AT-GAA as we move towards launch in the three largest Pompe markets this year. Our strategic focus remains on continuing to grow Galafold, securing regulatory approvals and launching of AT-GAA, and achieving non-GAAP profitability in the second half of 2023. Together, we see these driving value for our stakeholders and advancing our mission of delivering high quality medicines for people living with rare diseases.”

Corporate Highlights:

(in thousands)Three Months Ended
March 31,		 Year over Year
% Growth		 
 2023 2022 As
Reported		 at CER1 
Net Product Revenues$86,270 $78,715 10% 14% 

First Quarter 2023 Financial Results

2023 Financial Guidance

Amicus is focused on the following five key strategic priorities in 2023:

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 2023 Galafold revenue guidance utilizes the actual exchange rates at December 31, 2022.
2 Based on projections of Amicus’ non-GAAP Net Income under current operating plans, which includes successful AT-GAA regulatory approvals and continued Galafold growth. Amicus defines non-GAAP Net Income as GAAP Net 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.

Conference Call and Webcast

Amicus Therapeutics will host a conference call and audio webcast today, May 10, 2023, at 8:30 a.m. ET to discuss the first quarter 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.

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 Fabry patients 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.

U.S. IMPORTANT SAFETY INFORMATION

ADVERSE REACTIONS
The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia.

USE IN SPECIFIC POPULATIONS
There is insufficient clinical data on Galafold use in pregnant women to inform a drug-associated risk for major birth defects and miscarriage. Advise women of the potential risk to a fetus.

It is not known if Galafold is present in human milk. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition.

Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis.

The safety and effectiveness of Galafold have not been established in pediatric patients.

To report Suspected Adverse Reactions, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf.

EU Important Safety Information
Treatment with Galafold should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. Galafold is not recommended for use in patients with a nonamenable mutation.

For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.

About Fabry Disease

Fabry disease is an inherited lysosomal disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which results from mutations in the GLA gene. The primary biological function of alpha-Gal A is to degrade specific lipids in lysosomes, including globotriaosylceramide (referred to here as GL-3 and also known as Gb3). Lipids that can be degraded by the action of alpha-Gal A are called "substrates" of the enzyme. Reduced or absent levels of alpha-Gal A activity lead to the accumulation of GL-3 in the affected tissues, including heart, kidneys, and skin. Accumulation of GL-3 and progressive deterioration of organ function is believed to lead to the morbidity and mortality of Fabry disease. The symptoms can be severe, differ from person to person, and begin at an early age.

About Pompe Disease
Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function, to a more slowly progressive, late-onset form primarily affecting skeletal muscle and progressive respiratory involvement. Late-onset Pompe disease can be severe and debilitating, including progressive muscle weakness throughout the body, particularly the skeletal muscles and muscles controlling breathing, that worsens over time.

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 Twitter 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 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 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, including as they are impacted by COVID-19 related disruption, are based on current information. The potential impact on operations from the COVID-19 pandemic is inherently unknown and cannot be predicted with confidence and may cause actual results and performance to differ materially from the statements in this release, including without limitation, because of the impact on general political and economic conditions, including as a result of efforts by governmental authorities to mitigate COVID-19, such as travel bans, shelter in place orders and third-party business closures and resource allocations, manufacturing and supply chain disruptions and limitations on patient access to commercial or clinical product. In addition to the impact of the COVID-19 pandemic, 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, including the FDA, EMA, MHRA, and PMDA, 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 commercializing Galafold in Europe, Japan, the US and other geographies or AT-GAA if and when approved; 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 and manufacturing. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. Statements regarding corporate financial guidance and financial goals and the attainment of such goals. With respect to statements regarding projections of the Company's revenue 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, 2022, and on Form 10-Q for the quarter ended March 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 Communications

dmoore@amicusrx.com
(609) 662-5079

FOLD-G

TABLE 1

Amicus Therapeutics, Inc.
Consolidated Statements of Operations
(Unaudited)
(in thousands, except share and per share amounts) 
  Three Months Ended March 31,
  2023   2022 
Net product sales$86,270  $78,715 
Cost of goods sold 6,942   7,582 
Gross profit 79,328   71,133 
Operating expenses:   
Research and development 41,499   81,517 
Selling, general, and administrative 73,957   58,116 
Changes in fair value of contingent consideration payable 251      (1,188)
Loss on impairment of assets —   6,616 
Depreciation and amortization 1,257   1,411 
Total operating expenses 116,964   146,472 
Loss from operations (37,636)  (75,339)
Other (expense) income:   
Interest income 2,199   133 
Interest expense (11,844)  (8,147)
Other (expense) income (5,938)  1,902 
Loss before income tax (53,219)  (81,451)
Income tax benefit (expense) 287   (3,809)
Net loss attributable to common stockholders$(52,932) $(85,260)
Net loss attributable to common stockholders per common share — basic and diluted$(0.18) $(0.30)
Weighted-average common shares outstanding — basic and diluted 291,336,750   288,481,741 

TABLE 2

Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(Unaudited)
(in thousands, except share and per share amounts)   
 March 31,
2023		 December 31,
2022
Assets   
Current assets:   
Cash and cash equivalents$160,602  $148,813 
Investments in marketable securities 106,507   144,782 
Accounts receivable 68,178   66,196 
Inventories 27,004   23,816 
Prepaid expenses and other current assets 37,406   40,209 
Total current assets 399,697   423,816 
Operating lease right-of-use assets, net 28,483   29,534 
Property and equipment, less accumulated depreciation of $22,901 and $22,281 at March 31, 2023 and December 31, 2022, respectively 31,406   30,778 
Intangible asset, less accumulated depreciation of $36 and $0 at March 31, 2023 and December 31, 2022, respectively 22,964   23,000 
Goodwill 197,797   197,797 
Other non-current assets 20,172   19,242 
Total Assets$700,519  $724,167 
Liabilities and Stockholders' Equity   
Current liabilities:   
Accounts payable$24,965  $15,413 
Accrued expenses and other current liabilities 92,747   93,636 
Contingent consideration payable 12,668   21,417 
Operating lease liabilities 8,005   8,552 
Total current liabilities 138,385   139,018 
Long-term debt 392,658   391,990 
Operating lease liabilities 51,349   51,578 
Deferred reimbursements 5,906   4,656 
Deferred income taxes —   4,939 
Other non-current liabilities 9,648   8,939 
Total liabilities 597,946     
Commitments and contingencies   
Stockholders' equity:   
Common stock, $0.01 par value, 500,000,000 shares authorized, 283,300,585 and 281,108,273 shares issued and outstanding at March 31, 2023 and December 31, 2022, respectively 2,820   2,815 
Additional paid-in capital 2,691,836   2,664,744 
Accumulated other comprehensive loss:   
Foreign currency translation adjustment (6,543)  (11,989)
Unrealized loss on available-for-sale securities (201)  (116)
Warrants 83   83 
Accumulated deficit (2,585,422)  (2,532,490)
Total stockholders' equity 102,573   123,047 
Total Liabilities and Stockholders' Equity$           700,519  $ 724,167  

TABLE 3

Amicus Therapeutics, Inc.
Reconciliation of Non-GAAP Financial Measures
(in thousands) 
 Three Months Ended March 31,
  2023  2022 
Total operating expenses - as reported GAAP$116,964 $ 146,472  
Research and development:   
Stock-based compensation 8,490  9,365 
Selling, general and administrative:   
Stock-based compensation 26,404  21,286 
Loss on impairment of assets —  6,616 
Changes in fair value of contingent consideration payable  251  (1,188)
Depreciation and amortization 1,257  1,411 
Total operating expense adjustments to reported GAAP 36,402  37,490 
Total operating expenses - as adjusted$80,562 $108,982