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UNITED
STATES
SECURITIES
AND EXCHANGE COMMISSION
WASHINGTON,
D.C. 20549
FORM
8-K
CURRENT
REPORT PURSUANT TO
SECTION
13 OR 15(d) OF THE
SECURITIES EXCHANGE ACT OF 1934
Date of Report (Date of
earliest event reported): May 9, 2024
AMICUS THERAPEUTICS, INC.
(Exact
Name of Registrant as Specified in Its Charter)
| Delaware |
|
001-33497 |
|
71-0869350 |
(State or Other Jurisdiction
of Incorporation) |
|
(Commission
File Number) |
|
(I.R.S. Employer
Identification No.) |
47 Hulfish Street,
Princeton, New Jersey 08542
(Address of Principal
Executive Offices, and Zip Code)
609-662-2000
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 |
| Common Stock Par Value $0.01 |
|
FOLD |
|
Nasdaq |
Indicate by check mark whether the registrant is an emerging growth
company as defined in Rule 405 of the Securities Act of 1933 (17 CFR §230.405) or Rule 12b-2 of the Securities Exchange Act of 1934
(17 CFR §240.12b-2). Emerging growth company ¨
If an emerging growth company, indicate
by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial
accounting standards provided pursuant to Section 13(a) of the Exchange Act. ¨
Item 2.02 Results of Operations and 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:
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. |
| · | 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.
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.
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
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 | |
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 | |
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 | |
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 | |
Exhibit 99.2
 | 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 |
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