UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM
CURRENT REPORT PURSUANT TO
SECTION 13 OR 15(d) OF THE
SECURITIES EXCHANGE ACT OF 1934
Date of Report (Date of
earliest event reported):
(Exact Name of Registrant as Specified in Its Charter)
| (State or Other Jurisdiction of Incorporation) |
(Commission File Number) |
(I.R.S. Employer Identification No.) |
(Address of Principal Executive Offices, and Zip Code)
Registrant’s Telephone 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 | ||
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 Financial Condition.
On May 9, 2024, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the fiscal quarter ended March 31, 2024. A copy of this press release is attached hereto as Exhibit 99.1. The Company will host a conference call and webcast on May 9, 2024 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 9, 2024 | |
| 99.2 | May 9, 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: May 9, 2024 | By: | /s/ Ellen S. Rosenberg |
| Name: | Ellen S. Rosenberg | |
| Title: | Chief Legal Officer and Corporate Secretary | |
Exhibit 99.1
Amicus Therapeutics Announces First Quarter 2024 Financial Results and Corporate Updates
1Q 2024 Total Revenue of $110.4M, a 28% Increase Year-over-Year
Guiding to Full-Year 2024 Total Revenue Growth of 25%-30% at CER
Raising Full-Year 2024 Galafold® Guidance on Continued Strong Demand
Strong Pombiliti® + Opfolda® Launch with Increasing Rate of Commercial Patient Starts
Reiterating Full-Year Non-GAAP Profitability Projected in 2024
Conference Call and Webcast Today at 8:30 a.m. ET
PRINCETON, NJ, May 9, 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 first quarter ended March 31, 2024.
“Amicus delivered a great start to the year across our global business,” said Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc. “In the first quarter, we continued to deliver excellent commercial performance across the business. Once again, we have delivered mid-teen growth in global sales of Galafold, leading us to raise our product guidance for the year. We are also very pleased with the strong commercial launch of Pombiliti and Opfolda, which continues to build momentum with an increasing rate of commercial patient starts in the first months of the year. In 2024, we look to deliver significant total revenue growth of 25% to 30% coupled with continued expense management to deliver full year non-GAAP profitability. With these two therapies, we believe Amicus continues to make a profound difference in the lives of many individuals affected by rare diseases across the globe.”
First Quarter 2024 Financial Highlights:
| · | Total revenue in the first quarter 2024 was $110.4 million, a year-over-year increase of 28% from total revenue of $86.3 million in the first quarter 2023. On a constant currency basis (CER)1, first-quarter 2024 total revenue growth was 28%. |
| (in thousands) | Three Months Ended March 31, | Year-over-Year % Growth | ||||||||||||||
| 2024 | 2023 | Reported | at CER1 | |||||||||||||
| Galafold® | 99,359 | 86,112 | 15 | % | 16 | % | ||||||||||
| Pombiliti® + Opfolda® | 11,044 | 158 | n/a | n/a | ||||||||||||
| Net Product Revenues | $ | 110,403 | $ | 86,270 | 28 | % | 28 | % | ||||||||
| · | Galafold (migalastat) net product sales were $99.4 million in the first quarter 2024, a year-over-year increase of 15%, or 16% at constant exchange rates1. |
| · | Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) net product sales were $11.0 million in the first quarter 2024, a 30% increase from the fourth quarter of 2023. As of the end of April, over 155 patients are on treatment with commercial product or scheduled to be treated. |
| · | Total GAAP operating expenses of $124.6 million for the first quarter 2024 increased by 6% as compared to $117.0 million for the first quarter 2023. Total non-GAAP operating expenses of $85.6 million for the first quarter 2024 increased by 6% as compared to $80.6 million for the first quarter 2023. |
| · | GAAP net loss was $48.4 million, or $0.16 per share, for the first quarter 2024, and was reduced compared to a net loss of $52.9 million, or $0.18 per share, for the first quarter 2023. Non-GAAP net loss was $4.6 million, or $0.02 per share, for the first quarter 2024, and was reduced compared to a net loss of $16.8 million, or $0.06 per share, for first quarter 20232. |
1
| · | Cash, cash equivalents, and marketable securities totaled $239.6 million at March 31, 2024, compared to $286.2 million at December 31, 2023. |
2024 Financial Guidance:
| Updated | Previous | |||||||
| Total Revenue Growth1 | 25% to 30% | n/a | ||||||
| Galafold Revenue Growth1 | 13% to 17% | 11% to 16% | ||||||
| Pombiliti + Opfolda Revenue1 | $62M to $67M | n/a | ||||||
| Non-GAAP Operating Expense3 | $345M to $365M | $345M to $365M |
Amicus is focused on the following key strategic priorities in 2024:
| · | Delivering double-digit Galafold revenue growth | |
| · | Executing multiple successful launches of Pombiliti + Opfolda | |
| · | Advancing ongoing studies to support medical and scientific leadership in Fabry and Pompe diseases | |
| · | Achieving full-year non-GAAP profitability4 |
1 At constant exchange rates (CER). 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 revenue guidance utilizes actual exchange rate as of December 31, 2023.
2 Full reconciliation of GAAP results to the Company’s non-GAAP adjusted measures for the reporting period(s) appear in the tables to this press release.
3 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.
4 Based on projections of Amicus’ non-GAAP Net (Loss) Income under current operating plans, which includes successful Pombiliti + Opfolda launch 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.
Conference Call and Webcast
Amicus Therapeutics will host a conference call and audio webcast today, May 9, 2024, at 8:30 a.m. ET to discuss the first quarter 2024 financial results and corporate updates. Participants and investors interested in accessing the call by phone will need to register using the online registration form. After registering, all phone participants will receive a dial-in number along with a PIN number to access the event.
A live audio webcast and related presentation materials can also be accessed via the Investors section of the Amicus Therapeutics corporate website at ir.amicusrx.com. Web participants are encouraged to register on the website 15 minutes prior to the start of the call. An archived webcast and accompanying slides will be available on the Company's website shortly after the conclusion of the live event.
About Galafold
Galafold® (migalastat) 123 mg capsules is an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) for the treatment of Fabry disease in adults who have amenable galactosidase alpha gene (GLA) variants. In these patients, Galafold works by stabilizing the body’s own dysfunctional enzyme so that it can clear the accumulation of disease substrate. Globally, Amicus Therapeutics estimates that approximately 35 to 50 percent of people living with Fabry disease may have amenable GLA variants, though amenability rates within this range vary by geography. Galafold is approved in more than 40 countries around the world, including the U.S., EU, U.K., and Japan.
U.S. INDICATIONS
AND USAGE
Galafold is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene
(GLA) variant based on in vitro assay data.
This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
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.
2
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.
About Pombiliti + Opfolda
Pombiliti + Opfolda, is a two-component therapy that consists of cipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that’s designed to reduce loss of enzyme activity in the blood.
U.S. INDICATIONS AND USAGE
POMBILITI in combination with OPFOLDA is indicated for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).
SAFETY INFORMATION
HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS: Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, POMBILITI should be discontinued immediately and appropriate medical treatment should be initiated. INFUSION-ASSOCIATED REACTIONS (IARs): If severe IARs occur, immediately discontinue POMBILITI and initiate appropriate medical treatment. RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of serious exacerbation of 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.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information, please visit the company’s website at www.amicusrx.com, and follow on X and LinkedIn.
Non-GAAP Financial Measures
In addition to financial information prepared in accordance with U.S. GAAP, this press release also contains adjusted financial measures that we believe provide investors and management with supplemental information relating to operating performance and trends that facilitate comparisons between periods and with respect to projected information. These adjusted financial measures are non-GAAP measures and should be considered in addition to, but not as a substitute for, the information prepared in accordance with U.S. GAAP. We use these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. We typically exclude certain GAAP items that management does not believe affect our basic operations and that do not meet the GAAP definition of unusual or non-recurring items. Other companies may define these measures in different ways. When we provide our expectation for non-GAAP operating expenses and profitability on a forward-looking basis, a reconciliation of the differences between the non-GAAP expectation and the corresponding GAAP measure generally is not available without unreasonable effort due to potentially high variability, complexity and low visibility as to the items that would be excluded from the GAAP measure in the relevant future period, such as unusual gains or losses. The variability of the excluded items may have a significant, and potentially unpredictable, impact on our future GAAP results.
3
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, and on Form 10-Q for the quarter ended March 31, 2024, to be filed today. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.
CONTACT:
Investors:
Amicus Therapeutics
Andrew Faughnan
Vice President, Investor Relations
afaughnan@amicusrx.com
(609) 662-3809
Media:
Amicus Therapeutics
Diana Moore
Head of Global Corporate Affairs and Communications
dmoore@amicusrx.com
(609) 662-5079
FOLD-G
4
TABLE 1
Amicus Therapeutics, Inc.
Consolidated Statements of Operations
(Unaudited)
(in thousands, except share and per share amounts)
| Three Months Ended March 31, | ||||||||
| 2024 | 2023 | |||||||
| Net product sales | $ | 110,403 | $ | 86,270 | ||||
| Cost of goods sold | 13,567 | 6,942 | ||||||
| Gross profit | 96,836 | 79,328 | ||||||
| Operating expenses: | ||||||||
| Research and development | 28,329 | 41,499 | ||||||
| Selling, general, and administrative | 88,029 | 73,957 | ||||||
| Changes in fair value of contingent consideration payable | — | 251 | ||||||
| Restructuring charges | 6,045 | — | ||||||
| Depreciation and amortization | 2,154 | 1,257 | ||||||
| Total operating expenses | 124,557 | 116,964 | ||||||
| Loss from operations | (27,721 | ) | (37,636 | ) | ||||
| Other expense: | ||||||||
| Interest income | 1,540 | 2,199 | ||||||
| Interest expense | (12,436 | ) | (11,844 | ) | ||||
| Other expense | (4,966 | ) | (5,938 | ) | ||||
| Loss before income tax | (43,583 | ) | (53,219 | ) | ||||
| Income tax (expense) benefit | (4,836 | ) | 287 | |||||
| Net loss attributable to common stockholders | $ | (48,419 | ) | $ | (52,932 | ) | ||
| Net loss attributable to common stockholders per common share — basic and diluted | $ | (0.16 | ) | $ | (0.18 | ) | ||
| Weighted-average common shares outstanding — basic and diluted | 302,903,009 | 291,336,750 | ||||||
5
TABLE 2
Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(Unaudited)
(in thousands, except share and per share amounts)
| March 31, 2024 | December 31, 2023 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 209,761 | $ | 246,994 | ||||
| Investments in marketable securities | 29,842 | 39,206 | ||||||
| Accounts receivable | 76,433 | 87,632 | ||||||
| Inventories | 60,759 | 59,696 | ||||||
| Prepaid expenses and other current assets | 54,444 | 49,533 | ||||||
| Total current assets | 431,239 | 483,061 | ||||||
| Operating lease right-of-use assets, net | 23,003 | 26,312 | ||||||
| Property and equipment, less accumulated depreciation of $26,563 and $25,429 at March 31, 2024 and December 31, 2023, respectively | 32,421 | 31,667 | ||||||
| Intangible assets, less accumulated amortization of $3,328 and $2,510 at March 31, 2024 and December 31, 2023, respectively | 19,672 | 20,490 | ||||||
| Goodwill | 197,797 | 197,797 | ||||||
| Other non-current assets | 17,657 | 18,553 | ||||||
| Total Assets | $ | 721,789 | $ | 777,880 | ||||
| Liabilities and Stockholders’ Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 9,210 | $ | 15,120 | ||||
| Accrued expenses and other current liabilities | 124,622 | 144,245 | ||||||
| Operating lease liabilities | 8,270 | 8,324 | ||||||
| Total current liabilities | 142,102 | 167,689 | ||||||
| Long-term debt | 388,391 | 387,858 | ||||||
| Operating lease liabilities | 47,831 | 48,877 | ||||||
| Other non-current liabilities | 12,771 | 13,282 | ||||||
| Total liabilities | 591,095 | 617,706 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders’ equity: | ||||||||
| Common stock, $0.01 par value, 500,000,000 shares authorized, 296,159,417 and 293,594,209 shares issued and outstanding at March 31, 2024 and December 31, 2023, respectively | 2,922 | 2,918 | ||||||
| Additional paid-in capital | 2,853,550 | 2,836,018 | ||||||
| Accumulated other comprehensive loss: | ||||||||
| Foreign currency translation adjustment | 6,847 | 5,429 | ||||||
| Unrealized loss on available-for-sale securities | (203 | ) | (188 | ) | ||||
| Warrants | 71 | 71 | ||||||
| Accumulated deficit | (2,732,493 | ) | (2,684,074 | ) | ||||
| Total stockholders’ equity | 130,694 | 160,174 | ||||||
| Total Liabilities and Stockholders’ Equity | $ | 721,789 | $ | 777,880 | ||||
6
TABLE 3
Amicus Therapeutics, Inc.
Reconciliation of Non-GAAP Financial Measures
(in thousands)
(Unaudited)
| Three Months Ended March 31, | ||||||||
| 2024 | 2023 | |||||||
| Total operating expenses - as reported GAAP | $ | 124,557 | $ | 116,964 | ||||
| Research and development: | ||||||||
| Stock-based compensation | 4,871 | 8,490 | ||||||
| Selling, general and administrative: | ||||||||
| Stock-based compensation | 25,932 | 26,404 | ||||||
| Restructuring charges | 6,045 | — | ||||||
| Changes in fair value of contingent consideration payable | — | 251 | ||||||
| Depreciation and amortization | 2,154 | 1,257 | ||||||
| Total operating expense adjustments to reported GAAP | 39,002 | 36,402 | ||||||
| Total operating expenses - as adjusted | $ | 85,555 | $ | 80,562 | ||||
7
TABLE 4
Amicus Therapeutics, Inc.
Reconciliation of Non-GAAP Financial Measures
(in thousands, except share and per share amounts)
(Unaudited)
Three Months Ended March 31, | ||||||||
| 2024 | 2023 | |||||||
| GAAP net loss | $ | (48,419 | ) | $ | (52,932 | ) | ||
| Share-based compensation | 30,803 | 34,894 | ||||||
| Changes in fair value of contingent consideration payable | — | 251 | ||||||
| Depreciation and amortization | 2,154 | 1,257 | ||||||
| Restructuring charges | 6,045 | — | ||||||
| Income tax expense (benefit) | 4,836 | (287 | ) | |||||
| Non-GAAP net loss | $ | (4,581 | ) | $ | (16,817 | ) | ||
| Non-GAAP net loss attributable to common stockholders per common share — basic and diluted | $ | (0.02 | ) | $ | (0.06 | ) | ||
| Weighted-average common shares outstanding — basic and diluted | 302,903,009 | 291,336,750 | ||||||
8
 | AT THE FOREFRONT OF THERAPIES FOR RARE DISEASES 1Q24 Results Conference Call & Webcast May 9, 2024 |
| 2 Forward-Looking Statements 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 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, and on Form 10-Q for the quarter ended March 31, 2024, to be filed today 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. Non-GAAP Financial Measures 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 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. |
| A Rare Company A leading biotech company projected to deliver 2024 total revenue growth of 25%-30%1 LEVERAGEABLE GLOBAL COMMERCIAL ORGANIZATION >500 EMPLOYEES in 20+ Countries First Two-Component Therapy for Pompe Disease 2 APPROVED THERAPIES Combined Peak Revenue Potential $1.5B – $2B World Class Clinical Development Capabilities 13-17% FY 2024 Galafold Revenue Growth1 Guiding to Full Year 2024 Non-GAAP Profitability 3 25-30% FY 2024 Total Revenue Growth1 1At CER: Constant Exchange Rates |
| 1 2 3 4 A Transformative Year Ahead for Amicus 1CER: Constant Exchange Rates; 2024 Galafold revenue guidance utilizes actual exchange rates as of December 31, 2023 2024 Strategic Priorities 4 Galafold® revenue growth of 11-16% at CER1, now raised to 13-17% Execute multiple successful launches of Pombiliti® + Opfolda® Advance ongoing studies to support medical and scientific leadership in Fabry and Pompe diseases Achieve non-GAAP profitability for the full year 4 |
| 5 Galafold® (migalastat) Continued Growth Building a leadership position in the treatment of Fabry disease |
| 6 2024 Galafold Success (as of March 31, 2024) 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%) were headache, nasopharyngitis, urinary tract infection, nausea, and pyrexia. For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf. 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. A unique mechanism of action for Fabry patients with amenable variants 35-50% Fabry Patients Amenable to Galafold 40+ Countries with Regulatory Approvals 2,400+ Individuals Treated $99.4M 1Q24 Galafold Revenue 13-17% 2024 Galafold Growth at CER1 >60% Share of Treated Amenable Patients Galafold is the only approved oral treatment option in Fabry disease 1At CER: Constant Exchange Rates |
| 7 FY16 FY17 FY18 FY19 FY20 FY21 FY22 FY23 FY24 $5M $37M $91M $182M $261M $306M $329M Galafold Performance Global mix of switch (~42%) and previously untreated patients (~58%)2 Expect non-linear quarterly growth to continue due to uneven ordering patterns and FX fluctuations Raising FY 2024 Galafold growth guidance to 13%-17% at CER 1At CER: Constant Exchange Rates 2 Data on file Q1 $99M $388M $438M-$454M1 Distribution of Galafold revenue by quarter over previous 5 years: Q1 Q2 Q3 Q4 5 Year Avg. 22% 24% 26% 28% Q1 2024 Galafold reported revenue growth of +16% at CER to $99.4M Q2 $94M Q3 $101M Q4 $107M Q1 $86M |
| 8 Expanding market through uptake in naïve population as well as geographic and label expansion Increasing patient identification through ongoing medical education, screening, and improved diagnostics Driving market share of treated amenable patients through excellent execution Maintaining >90% adherence and compliance through HCP and patient education and support 8 Key Growth Drivers for 2024 Highest patient demand in last four years lays strong foundation for continued double-digit Galafold growth in 2024 8 |
| 9 Pombiliti® (cipaglucosidase alfa-atga) Opfolda® (miglustat) Potential to establish a new standard of care for people living with late-onset Pompe disease + |
| 10 FY23 FY24 Pombiliti + Opfolda Performance Guiding to $62M to $67M in FY 2024 Pombiliti + Opfolda Revenue1 $62M-67M1 Q1 2024 revenue of $11M, up +30% from Q4 2023, provides strong foundation for 2024 Q1 $81M Q1 $11M $11M 1At CER: Constant Exchange Rates |
| 11 Successful Global Launch of Pombiliti + Opfolda Underway Access and Reimbursement Positive interactions with global payors Time through U.S. insurance process accelerating Country-by-country reimbursement process underway Multiple launches expected in 2H 2024 ~155 patients treated with commercial product or scheduled to be treated ~135 patients on treatment Very positive early feedback from real-world experience Double the level of new commercial patients in 2024 vs. 2023 KOL Outreach Successfully engaged with top prescribers in each approved country Existing relationships with HCPs at key treatment centers Ongoing disease education Increasing depth and breadth of prescribers Patient Demand As of end of April 2024 11 Focus in 2024 is on maximizing the number of patients on therapy by year end 11 |
| 12 Regulatory and Clinical Updates Continuing to build the body of evidence and expand commercial access >10 reimbursement dossiers and multiple regulatory submissions throughout 2024 Ongoing clinical studies in children with late-onset Pompe disease (LOPD) and infantile-onset Pompe disease (IOPD) Amicus registry for Pompe disease to continue generating evidence on differentiated MOA and long-term effect Significant presence at WORLDSymposium 2024 with 11 posters and an oral presentation highlighting work in Fabry and Pompe |
| 13 Corporate Outlook Delivering on our mission for patients and shareholders |
| 14 Q1 2024 Select Financial Results Q1 2024 revenue of $110M, up 28% and net loss reduced (in thousands, except per share data) Mar. 31, 2024 Mar. 31, 2023 Product Revenue $110,403 $86,270 Cost of Goods Sold 13,567 6,942 R&D Expense 28,329 41,499 SG&A Expense 88,029 73,957 Changes in Fair Value of Contingent Consideration - 251 Restructuring Charges 6,045 - Depreciation and Amortization 2,154 1,257 Loss from Operations (27,721) (37,636) Interest Income 1,540 2,199 Interest Expense (12,436) (11,844) Other Expense (4,966) (5,938) Income Tax (Expense) Benefit (4,836) 287 Net Loss (48,419) (52,932) Net Loss Per Share (0.16) (0.18) March 31, 2024 weighted-average common shares outstanding: 302,903,009 March 31, 2023 weighted-average common shares outstanding: 291,336,750 |
| 15 Updated Full-Year 2024 Guidance Updated Guidance Previous Guidance Total Revenue Growth1 25% to 30% - Galafold Revenue Growth1 13% to 17% 11% to 16% Pombiliti + Opfolda Revenue1 $62M to $67M - Non-GAAP Operating Expense $345M to $365M $345M to $365M Guiding to full-year 2024 non-GAAP profitability 1At CER: Constant Exchange Rates |
| 16 Accelerating total revenue growth 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 assets, depreciation and amortization, acquisition related income (expense), loss on extinguishment of debt, restructuring charges and income taxes. Clear line of sight to generating positive cashflow Delivering full-year non-GAAP1 profitability Positioned for Significant Value Creation in 2024 Unlocking the value of two unique commercial therapies in sizeable and growing markets |
| 17 Ultimate Measure of Success: Impacting the Lives of People Living with Rare Diseases YE17 2024+ >2,600 Patients* Many Thousands of Patients* YE23 >350 Patients* *Clinical and commercial, all figures approximate |
| Appendix |
| 19 Appendix I |
| 20 Appendix II |
| 21 Environmental, Social, & Governance (ESG) Snapshot Board of Directors Committed to ongoing Board refreshment and diversity of background, gender, skills, and experience: 89% Board Independence 56% Overall Board Diversity Address a rare genetic disease First-in-class or best-in-class Impart meaningful benefit for patients 517 Global Employees 58% % Female Employees Who We Serve Our mission is to drive sustainability with our partners by incorporating environmental and sustainability principles into all our commercial relationships Pledge for a Cure Designate a portion of product revenue back into R&D for that specific disease until there is a cure. 3 Female 1 Veteran Status 1 African American Director Diversity Leverage employee capabilities and expertise to provide a culture that drives performance and ultimately attracts, energizes, and retains critical talent. Employee Recruitment, Engagement, & Retention Amicus is Certified as a Great Place to Work in the U.S., U.K., Italy, Germany, Spain, France, and Japan Career Development Employees say Amicus is a great place to work compared to 57% of employees at a typical U.S.-based company Committed to producing transformative medicines for people living with rare diseases while practicing environmental responsibility and adhering to sustainability best practices in our operations. Environmental Management 0% Amicus-owned Direct Manufacturing and Related Scope 1 and Scope 2 Emissions Diversity, Equity, & Inclusion (DEI) 511 Volunteer hours (U.S.): 37 Amicus-supported community programs: 32 patients /24countries Expanded Access through Feb 2024: Pricing PROMISE Contributions allocated: $1,980,516 U.S. $706,417 Intl. Charitable Giving (as of December 31, 2023) Committed to never raising the annual price of our products more than consumer inflation. Pledge to support a more inclusive culture to impact our employees, our communities, and society. We have embedded DEI into our business units, our Belief Statement, and Mission-Focused Behaviors 90% Programs we invest in have 3 key characteristics: (as of December 31, 2023) (as of March 31, 2024) |
| 22 FX Sensitivity and Galafold Distribution of Quarterly Sales Impact from Foreign Currency Q1 2024: Currency Variances: USD/ Q1 2023 Q1 2024 YoY Variance EUR 1.073 1.086 1.2% GBP 1.215 1.268 4.4% JPY 0.008 0.007 (10.8%) Full-year 2024 Revenue Sensitivity Given the high proportion of Amicus revenue Ex-US (>60%), a change in exchange rates of +/- 5% compared to year-end 2023 rates could lead to a ~$15M move in global reported revenues in 2024. Distribution of Galafold Revenue by Quarter over Past 5 Years: Q1 Q2 Q3 Q4 5 Year Avg. 22% 24% 26% 28% |
| 23 Streamlined Rare Disease Pipeline with Focus on Fabry Disease and Pompe Disease Franchises INDICATION DISCOVERY PRECLINICAL PHASE 1/2 PHASE 3 REGULATORY COMMERCIAL FABRY FRANCHISE Galafold® (migalastat) Fabry Genetic Medicines Next-Generation Chaperone POMPE FRANCHISE Pombiliti® (cipaglucosidase alfa-atga) + Opfolda® (miglustat) Pompe Genetic Medicines OTHER Discovery Programs |
