8-K
AMICUS THERAPEUTICS, INC. (FOLD)
UNITEDSTATES
SECURITIESAND EXCHANGE COMMISSION
WASHINGTON,D.C. 20549
FORM
8-K
CURRENTREPORT PURSUANT TO
SECTION13 OR 15(d) OF THE
SECURITIES EXCHANGE ACT OF 1934
Date of Report (Date of earliest event reported): May 10, 2023
AMICUS THERAPEUTICS, INC.
(Exact Name of Registrant as Specified in Its Charter)
| Delaware | 001-33497 | 71-0869350 |
|---|---|---|
| (State or Other Jurisdiction<br><br> <br>of Incorporation) | (Commission<br><br> <br>File Number) | (I.R.S. Employer<br><br> <br>Identification No.) |
3675 Market Street,
Philadelphia, PA 19104
(Address of PrincipalExecutive Offices, and Zip Code)
215-921-7600
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<br> of each class | Trading<br> Symbol(s) | Name<br> of each exchange on which registered |
|---|---|---|
| Common<br> Stock Par Value $0.01 | FOLD | NASDAQ |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (17 CFR §230.405) or Rule 12b-2 of the Securities Exchange Act of 1934 (17 CFR §240.12b-2). Emerging growth company ¨
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ¨
Item 2.02 Results of Operations and FinancialCondition.
On May 10, 2023, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the fiscal quarter ended March 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 May 10, 2023 to discuss its first quarter results of operations. A copy of the conference call presentation materials is attached hereto as Exhibit 99.2. Both exhibits are incorporated herein by reference.
In accordance with General Instruction B.2. of Form 8-K, the information in this Current Report on Form 8-K and the Exhibits shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liability of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits:
| Exhibit No. | Description |
|---|---|
| 99.1 | Press Release dated May 10, 2023 |
| 99.2 | May 10, 2023 Conference Call Presentation Materials |
| 104 | Cover Page Interactive Data File (embedded within<br> the Inline XBRL document) |
Signature Page
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| AMICUS THERAPEUTICS, INC. | ||
|---|---|---|
| Date: May 10, 2023 | By: | /s/ Ellen S. Rosenberg |
| Name: | Ellen S. Rosenberg | |
| Title: | Chief Legal Officer and Corporate Secretary |
Exhibit 99.1
Amicus TherapeuticsAnnounces First Quarter 2023 Financial Results and Corporate Updates
1Q23Revenue Growth of 14% at CER to $86.3M
OnTrack to Deliver Full-Year 2023 Galafold Revenue Growth of 12%-17% at CER
U.S. FDA Pre-approvalInspection for AT-GAA Complete; Approval Expected 3Q 2023
European Launchof Pombiliti^®^+Opfolda^®^ Expected 3Q 2023
Non-GAAP ProfitabilityProjected in 2H 2023
ConferenceCall and Webcast Today at 8:30 a.m. ET
PHILADELPHIA,PA, May. 10, 2023 – 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:
| · | Global revenue in the first quarter 2023 was $86.3 million. First quarter revenue represented<br> a year-over-year increase of 10% from total revenue of $78.7 million in the first quarter<br> of 2022. First quarter operational revenue growth measured at constant exchange rates (CER)^1^was 14%. | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| (in thousands) | Three Months Ended <br><br>March 31, | Year over Year<br> <br>% Growth | ||||||||
| --- | --- | --- | --- | --- | --- | --- | --- | --- | --- | --- |
| 2023 | 2022 | As <br><br>Reported | at CER^1^ | |||||||
| Net Product Revenues | $ | 86,270 | $ | 78,715 | 10 | % | 14 | % | ||
| · | For the full-year 2023, the Company anticipates double-digit Galafold revenue growth of 12-17% at CER^1^. Growth is expected to be driven by continued underlying demand from<br> both switch and treatment-naïve patients, geographic expansion, label extensions, continued<br> diagnosis of new Fabry patients, and commercial execution across all major markets, including<br> the U.S., EU, U.K., and Japan. | |||||||||
| --- | --- | |||||||||
| · | The U.S. Food and Drug Administration (FDA) has very recently completed the required pre-approval inspection of the WuXi Biologics manufacturing site in China. The Company believes the<br> comments and observations received at the close of the FDA inspection are all addressable<br> and continues to expect regulatory approval of AT-GAA in the U.S. in the third quarter of<br> 2023. | |||||||||
| --- | --- | |||||||||
| · | The EU and U.K. AT-GAA regulatory reviews remain on-track with marketing authorization expected in 3Q 2023. In the European Union (EU), the European Commission (EC) granted approval<br> for Pombiliti^®^ (cipaglucosidase alfa), used in combination with miglustat<br> for adults with late-onset Pompe disease. In April, the Committee for Medicinal Products<br> for Human Use (CHMP) adopted a positive opinion of Opfolda^®^ (miglustat), the<br> enzyme stabilizer component. Full approval of Pombiliti + Opfolda is anticipated in the third<br> quarter of 2023. In the U.K., the regulatory submission process was initiated in December 2022,<br> with final approval expected in the third quarter of 2023. | |||||||||
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| · | Strategic expansion of Supply and Manufacturing Services Agreement with WuXi Biologics. The long-term<br> agreement further strengthens relationship with WuXi and secures long-term capacity at the<br> new state-of-the-art manufacturing facility of WuXi in Dundalk, Ireland. |
|---|---|
| · | Expanded access programs continue to meet the growing demand for AT-GAA across multiple countries.<br> In the U.K., under the Early Access to Medicines Scheme (EAMS), multiple physicians have<br> requested access from each of the leading Pompe centers in the country. Many patients with<br> Pompe disease are participating in additional expanded access programs in the U.S., Germany,<br> France, and Japan. |
| --- | --- |
| · | Galafold U.S. intellectual property estate strengthened following the issuance of multiple new patents in 2023. Galafold is protected by orphan drug regulatory exclusivities and a broad U.S.<br> intellectual property portfolio of 49 orange book-listed patents, including 8 composition<br> of matter patents, 33 of which provide protection through at least 2038. |
| --- | --- |
| · | Full-year 2023 non-GAAP operating expense guidance of $340 million to $360 million, driven by prudent<br> expense management while investing in AT-GAA manufacturing and pre-launch activities. |
| --- | --- |
| · | Based on the current operating plan, the timing of AT-GAA approvals, and through prudent management of expenses, the Company is on-track to achieve non-GAAP profitability^2^ in the second half of 2023. |
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First Quarter2023 Financial Results
| · | Total<br> revenue in the first quarter 2023 was $86.3 million, a year-over-year increase of 10% from<br> total revenue of $78.7 million in the first quarter 2022. On a constant currency basis, first<br> quarter 2023 total revenue growth was 14%. Compared to the first quarter 2022, reported revenue<br> was offset by a negative currency impact of $3.8 million, or 4%. |
|---|---|
| · | Cash,<br> cash equivalents, and marketable securities totaled $267.1 million at March 31, 2023,<br> compared to $293.6 million at December 31, 2022. |
| · | Total<br> GAAP operating expenses of $117.0 million for the first quarter 2023 decreased as compared<br> to $146.5 million for the first quarter 2022. |
| · | Total<br> non-GAAP operating expenses of $80.6 million for the first quarter 2023 decreased as compared<br> to $109.0 million for the first quarter 2022, primarily reflecting decreased program spend.^3^ |
| · | Net<br> loss was $52.9 million, or $0.18 per share in the first quarter 2023, and was reduced compared<br> to a net loss of $85.3 million, or $0.30 per share, for the first quarter 2022. |
2023 FinancialGuidance
| · | For<br> the full-year 2023, the Company anticipates total Galafold revenue growth between 12 and<br> 17% 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 2023 is $340 million to $360 million, driven<br> by prudent expense management offset by continued investment in Galafold, AT-GAA clinical<br> studies and pre-launch activities, in addition to certain non-recurring costs for manufacturing<br> to support the global launch of AT-GAA^4^. |
| · | The<br> Company is on-track to achieve non-GAAP profitability^2^ in the second half of 2023. |
Amicusis focused on the following five key strategic priorities in 2023:
| · | Sustain<br> double-digit Galafold revenue growth (12-17% at CER^1^) |
|---|---|
| · | Secure<br> FDA, EMA, and MHRA approvals for AT-GAA |
| · | Initiate<br> successful global launches of AT-GAA |
| · | Advance<br> next generation pipeline programs (Fabry GTx, Fabry Next-Generation Chaperone, Pompe GTx) |
| · | Maintain<br> strong financial position on path to profitability |
^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.
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ConferenceCall 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.
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 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. 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.
EU ImportantSafety 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.
| · | Galafold<br> is not intended for concomitant use with enzyme replacement therapy. |
|---|---|
| · | Galafold<br> is not recommended for use in patients with Fabry disease who have severe renal impairment<br> (<30 mL/min/1.73 m2). The safety and efficacy of Galafold in children less than 12 years<br> of age have not yet been established. No data are available. |
| · | No<br> dosage adjustments are required in patients with hepatic impairment or in the elderly population. |
| · | There<br> is very limited experience with the use of this medicine in pregnant women. If you are pregnant,<br> think you may be pregnant, or are planning to have a baby, do not take this medicine until<br> you have checked with your doctor, pharmacist, or nurse. |
| · | While<br> taking Galafold, effective birth control should be used. It is not known whether Galafold<br> is excreted in human milk. |
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| · | Contraindications<br> to Galafold include hypersensitivity to the active substance or to any of the excipients<br> listed in the PRESCRIBING INFORMATION. |
|---|---|
| · | Galafold<br> 123 mg capsules are not for children (≥12 years) weighing less than 45 kg. |
| · | It<br> is advised to periodically monitor renal function, echocardiographic parameters and biochemical<br> markers (every 6 months) in patients initiated on Galafold or switched to Galafold. |
| · | OVERDOSE:<br> General medical care is recommended in the case of Galafold overdose. |
| · | The<br> most common adverse reaction reported was headache, which was experienced by approximately<br> 10% of patients who received Galafold. For a complete list of adverse reactions, please review<br> the SUMMARY OF PRODUCT CHARACTERISTICS. |
| · | Call<br> your doctor for medical advice about side effects. |
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 PompeDisease
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 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 Twitter 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 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.
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Forward Looking Statement
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to 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
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TABLE 1
Amicus Therapeutics, Inc.
ConsolidatedStatements 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 |
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TABLE 2
Amicus Therapeutics, Inc.
ConsolidatedBalance Sheets
(Unaudited)
(in thousands,except share and per share amounts)
| December 31,<br><br> 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 | 601,120 | |||
| 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 |
All values are in US Dollars.
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TABLE 3
Amicus Therapeutics, Inc.
Reconciliationof 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 |
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Exhibit 99.2
| AT THE FOREFRONT OF<br>THERAPIES FOR RARE DISEASES<br>1Q23 Results<br>Conference Call &<br>Webcast<br>May 10, 2023 |
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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 product<br>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 should<br>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 can be affected<br>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<br>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<br>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,<br>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<br>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,<br>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<br>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<br>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<br>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<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 complete<br>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<br>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<br>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<br>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<br>or update this newsrelease to reflect events or circumstances after the date hereof.<br>Non-GAAP Financial Measures<br>In addition to financial information prepared in accordance with U.S. GAAP, this presentation also contains adjusted financial measures that we believe provide investors and management with<br>supplemental information relating to operating performance and trends that facilitate comparisons between periods and with respect to projected information. These adjusted financial<br>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<br>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<br>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<br>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<br>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<br>unpredictable, impact on our future GAAP results. |
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| 3<br>A Rare Company<br>Patient-dedicated, rare disease biotechnology company with sustained double-digit revenue<br>growth, a global commercial infrastructure, and late-stage development capabilities<br>GLOBAL<br>COMMERCIAL<br>ORGANIZATION<br>EMPLOYEES<br>in 20 Countries<br>GALAFOLD<br> &<br>POMBILITI +<br>OPFOLDA<br>Gene Therapy<br>Platform<br>Leveraging<br>Experience in Protein<br>Engineering<br> & Glycobiology<br>12-17%<br>FY23 Galafold<br>Revenue Growth<br>at CER<br>Non-GAAP<br>PROFITABILITY<br>expected in<br>2H 2023<br>Cumulative $1.5B-<br>$2B Peak Potential<br>AT-GAA<br>Under Global<br>Regulatory Reviews for<br>Pompe Disease<br>$267M<br>Cash<br>as of 3/31/23<br>World-class<br>Clinical<br>Development<br>Capabilities |
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| 2023<br>Strategic<br>Priorities<br>Sustain double-digit Galafold revenue growth of 12-17% at CER1<br>Secure FDA, EMA, and MHRA approvals for AT-GAA<br>Initiate successful global launches of AT-GAA<br>Advance best-in-class, next-generation Fabry and Pompe pipeline<br>programs and capabilities<br>Maintain strong financial position on path to profitability<br>4<br>1<br>2<br>3<br>4<br>5<br>1CER: Constant Exchange Rates; 2023 Galafold revenue guidance utilizes actual exchange rate as of December 31, 2022 |
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| 5<br>Galafold® (migalastat) Continued Growth<br>Building a leadership position in the<br>treatment of Fabry disease |
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| 6<br>2023 Galafold Success (as of March 31, 2023)<br>Building on Galafold’s success and leveraging leadership position to drive continued growth<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>Galafold is the first and only approved oral<br>treatment option with a unique mechanism of<br>action for Fabry patients with amenable variants<br>350<br>Amenable<br>Variants in U.S.<br>Label 40+<br>Countries with<br>Regulatory<br>Approvals<br>49<br>Orange Book<br>Listed Patents<br>$86.1M<br>1Q23 Galafold<br>Revenue<br>12-17%<br>2023 Galafold<br>Operational Growth<br>at CER<br>1,386<br>Amenable<br>Mutations in<br>EU Label |
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| 7<br>Galafold Performance<br>Year-over-year reported revenue growth of +10% to $86.1M – Strong operational growth of<br>+14% at CER<br> >55% share of treated amenable patients<br> Global mix of switch (~45%) and previously<br>untreated patients (~55%)1<br> Compliance and adherence over 90%+<br>$79M $86M<br>+14% -4%<br>1Q22 Operational<br>Growth<br>FX Impact 1Q23<br>Year-over-Year Sales Growth<br>1Data on file |
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| 8<br>Galafold Quarterly Trends<br>Galafold quarterly growth remains strong with Q1 revenue of $86.1M<br>$82M<br>$79M<br>$81M $82M<br>$88M $86M<br>4Q21 1Q22 2Q22 3Q22 4Q22 1Q23<br>Quarterly Galafold Sales<br> Expect non-linear quarterly growth to continue due<br>to uneven ordering patterns and FX fluctuations<br>Distribution of Galafold Revenue<br>by Quarter in Past 5 years:<br>Q1 Q2 Q3 Q4<br>5 Year Avg. 22% 24% 26% 28% |
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| 9<br>Galafold Global Commercial Momentum (as of March 31, 2023)<br>Strong patient demand and performance against key metrics lay the foundation for continued<br>double-digit growth in 2023<br>9<br>Continued penetration into existing markets<br>Further uptake in diagnosed untreated population<br>Continued geographic expansion and label extensions<br>Maintaining compliance and adherence<br>Driving reimbursement and access<br>Sustained Growth in 2023 Driven by: |
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| 10<br>Pombiliti + Opfolda Launch Preparations<br>Experienced and passionate rare disease commercial and medical organization<br>ready to support second successful product launch<br>Scientific<br>Exchange<br>Educational and promotional materials developed<br>to help facilitate outreach at launch<br>Payors<br>KOLs<br>Commitment to patient access<br>Expansion of Amicus Assist in U.S.<br>Existing relationships with HCPs at key treatment centers<br>and hospitals to leverage upon launch<br>Training and launch readiness established across commercial<br>and medical teams<br>Tier 1 countries trained and prepared for launch<br>Engaging with payors to demonstrate value<br>Payors<br>International distribution system with product moving<br>through the channel<br>Robust clinical data and continued disease education through<br>publications and medical congresses<br>Focus within the first 90 days on converting clinical trial<br>and expanded access patients<br>Supply<br>Chain<br>Payors<br>Access Scientific<br>Exchange<br>Marketing<br>Team<br>Initial<br>Focus |
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| 11<br>AT-GAA<br>(cipaglucosidase alfa) + (miglustat)<br>Potential to establish a new standard of care<br>for people living with Pompe disease |
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| 12<br>Deficiency of GAA leading to lysosomal<br>glycogen accumulation and cellular<br>dysfunction<br>Age of onset ranges from<br>infancy to adulthood<br>Symptoms include muscle weakness,<br>respiratory failure, and cardiomyopathy<br>Respiratory and cardiac<br>failure are leading causes of<br>morbidity and mortality<br>Estimated incidence of ~1:28,000;<br>Significant underdiagnosis<br>~$1.2B+ global Pompe<br>ERT sales1<br>Majority of patients on current<br>standard of care decline after<br>~2 years<br>Pompe Disease Overview<br>1Based on 12 months ended December 31, 2022. Source: Sanofi Press Release<br>Pompe is a severe and fatal neuromuscular disease caused by the deficiency of lysosomal enzyme GAA |
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| 13<br>13 1Data on file<br>AT-GAA: Global Regulatory Status<br>Expect regulatory approvals and launch into the three largest Pompe markets in 2023<br> Pombiliti® (cipaglucosidase alfa) EC approval granted in March 2023<br> Opfolda® (miglustat) positive CHMP opinion in April 2023<br>EC approval expected in 3Q 2023<br> U.S. FDA pre-approval inspection complete<br> FDA approval expected 3Q 2023<br> U.K. MAA submitted via recognition procedure based on CHMP<br>opinion<br> Anticipating 3Q 2023 MHRA approval<br>13 |
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| 14<br>AT-GAA: Ongoing Clinical Studies and Expanded Access Mechanisms<br> Ongoing clinical studies in children and adolescents1 with<br>LOPD as well as in Infantile-Onset Pompe Disease (IOPD)<br> Multiple expanded access mechanisms in place, including in<br>the U.S., U.K., Germany, France, Japan, and others<br> ~200 people living with Pompe disease are now on AT-GAA<br>across extension studies and expanded access programs<br> ~75 centers worldwide currently participating in clinical trials<br>and access programs<br>Advancing science though ongoing clinical studies and providing expanded access<br>through multiple mechanisms<br>1 Children and adolescents aged 0 to <18 years old |
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| 15<br>Corporate Outlook<br>Delivering on our mission for patients and<br>shareholders |
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| 16<br>2023 Select Financial Results<br>2023 revenue of $86.3M and growth rate of 14% at CER from global sales<br>(in thousands, except per share data) Mar. 31, 2023 Mar. 31, 2022<br>Product Revenue $86,270 $78,715<br>Cost of Goods Sold 6,942 7,582<br>R&D Expense 41,499 81,517<br>SG&A Expense 73,957 58,116<br>Changes in Fair Value of Contingent Consideration 251 (1,188)<br>Loss on Impairment of Assets — 6,616<br>Depreciation and Amortization 1,257 1,411<br>Loss from Operations (37,636) (75,339)<br>Income Tax Benefit (Expense) 287 (3,809)<br>Net Loss (52,932) (85,260)<br>Net Loss Per Share (0.18) (0.30) |
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| Financial Outlook and Path to Profitability<br>Clear strategy to build our business, advance our portfolio, and achieve profitability<br>17<br>Sustain Revenue<br>Growth<br>Deliver on<br>Financial Goals<br>Secure Approvals<br>of AT-GAA<br>$86.3M 1Q 2023 revenue,<br>+14% YoY<br>operational growth<br>2023 Galafold revenue<br>growth guidance of<br>+12-17% YoY at CER<br>Galafold and<br>AT-GAA expected to<br>drive strong double-digit growth long term<br>Focused on prudent<br>expense management<br>Achieve profitability1<br>in 2H 2023<br>1Based on projections of Amicus non-GAAP Net Income under current operating plans, which includes successful AT-GAA regulatory approvals and continued Galafold growth. We define non-GAAP Net Income as GAAP Net<br>Income excluding the impact of share-based compensation expense, changes in fair value of contingent consideration, depreciation and amortization, acquisition related income (expense), loss on extinguishment of debt, loss on<br>impairment of assets, restructuring charges, and income taxes.<br>2023 non-GAAP operating<br>expense guidance of<br>$340M-$360M |
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| 18<br>Positioned for Significant Value Growth<br>Focused on execution and driving sustainable double-digit revenue growth on path to profitability<br>Continue to<br>bring Galafold®<br>to as many<br>patients as<br>possible, sustain<br>double-digit<br>operational<br>revenue growth<br>Successful<br>launch of<br>AT-GAA for<br>people living<br>with Pompe<br>disease<br>Advance<br>next-generation<br>gene therapies<br>in Fabry and<br>Pompe diseases<br>Fully leverage<br>global<br>capabilities and<br>infrastructure as<br>a leader in rare<br>diseases<br>Achieve<br>non-GAAP<br>profitability<br>in 2H 20231<br>1 Based on projections of Amicus non-GAAP Net Income under current operating plans, which includes successful AT-GAA regulatory approvals and continued Galafold growth. Non-GAAP Net Income defined as GAAP Net Income excluding the impact of share-based compensation expense, changes in fair<br>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. |
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| Appendix |
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| 20<br>Appendix |
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| 21<br>2022 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<br>patients<br>484<br>Global Employees<br>57%<br>% Female Employees<br>Who We Serve Our mission is to<br>drive sustainability<br>with our partners<br>by incorporating<br>environmental and<br>sustainability<br>principles into all<br>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>% Hiring Slate Diversity 97%<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 patients<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 and<br>Related GHG Emissions<br>Diversity, Equity, &<br>Inclusion (DEI)<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.<br>580<br>Volunteer hours<br>(U.S.):<br>22<br>Amicus supported<br>community programs:<br>79 patients /19countries<br>Expanded Access through Feb 2023:<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 our<br>products more than consumer inflation. |
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| 22<br>FX Sensitivity and Galafold Distribution of Quarterly Sales<br>Impact from Foreign Currency Q1 2023<br>Currency Variances:<br>USD/<br>Q1 2022 Q1 2023 YoY Variance<br>EUR 1.122 1.073 (4.4%)<br>GBP 1.342 1.215 (9.5%)<br>JPY 0.009 0.008 (12.2%)<br>Full Year 2023 Revenue Sensitivity<br>Given the high proportion of Amicus revenue Ex-US, a change in exchange rates of +/- 5%<br>compared to year end 2022 rates could lead to a $11M-$12M change in global reported<br>revenues in 2023.<br>Distribution of Galafold Revenue<br>by Quarter in Past 5 years:<br>Q1 Q2 Q3 Q4<br>5 Year Avg. 22% 24% 26% 28% |
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| 23<br>INDICATION DISCOVERY PRECLINICAL PHASE 1/2 PHASE 3 REGULATORY COMMERCIAL<br>FABRY FRANCHISE<br>Galafold®(migalastat)<br>Fabry Gene Therapy<br>Next-Generation Chaperone<br>POMPE FRANCHISE<br>AT-GAA (cipaglucosidase alfa + miglustat)<br>Pompe Gene Therapy<br>OTHER<br>CLN3 Batten Disease Gene Therapy<br>Next-Generation Research Programs<br>Amicus Pipeline<br>Streamlined rare disease pipeline with focus on Fabry disease and Pompe disease franchises<br>ODD<br>BTD ODD<br>ODD - Orphan Drug Designation BTD - Breakthrough Therapy Designation |
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| Thank you |
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