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 a year-over-year increase of 10% from total revenue of
$78.7 million in the first quarter of 2022. First quarter
operational revenue growth measured at constant exchange rates
(CER)1 was 14%.
(in thousands) |
Three Months Ended March 31, |
|
Year over Year % Growth |
|
|
2023 |
|
2022 |
|
As Reported |
|
at CER1 |
|
Net Product Revenues |
$86,270 |
|
$78,715 |
|
10% |
|
14% |
|
- For the
full-year 2023, the Company anticipates double-digit Galafold
revenue growth of 12-17% at
CER1. Growth is expected
to be driven by continued underlying demand from both switch and
treatment-naïve patients, geographic expansion, label extensions,
continued diagnosis of new Fabry patients, and commercial execution
across all major markets, including 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
comments and observations received at the close of the FDA
inspection are all addressable and continues to expect regulatory
approval of AT-GAA in the U.S. in the third quarter of 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 for Pombiliti®
(cipaglucosidase alfa), used in combination with miglustat for
adults with late-onset Pompe disease. In April, the Committee for
Medicinal Products for Human Use (CHMP) adopted a positive opinion
of Opfolda® (miglustat), the enzyme stabilizer component. Full
approval of Pombiliti + Opfolda is anticipated in the third quarter
of 2023. In the U.K., the regulatory submission process was
initiated in December 2022, with final approval expected in the
third quarter of 2023.
- Strategic
expansion of Supply and Manufacturing Services Agreement with WuXi
Biologics. The long-term agreement further strengthens
relationship with WuXi and secures long-term capacity at the 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. In the U.K., under the Early
Access to Medicines Scheme (EAMS), multiple physicians have
requested access from each of the leading Pompe centers in the
country. Many patients with Pompe disease are participating in
additional expanded access programs in the U.S., Germany, 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.
intellectual property portfolio of 49 orange book-listed patents,
including 8 composition 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 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 profitability2
in the second half of 2023.
First Quarter 2023 Financial
Results
- Total revenue in the first quarter
2023 was $86.3 million, a year-over-year increase of 10% from total
revenue of $78.7 million in the first quarter 2022. On a constant
currency basis, first quarter 2023 total revenue growth was 14%.
Compared to the first quarter 2022, reported revenue was offset by
a negative currency impact of $3.8 million, or 4%.
- Cash, cash equivalents, and
marketable securities totaled $267.1 million at March 31, 2023,
compared to $293.6 million at December 31, 2022.
- Total GAAP operating expenses of
$117.0 million for the first quarter 2023 decreased as compared to
$146.5 million for the first quarter 2022.
- Total non-GAAP operating expenses
of $80.6 million for the first quarter 2023 decreased as compared
to $109.0 million for the first quarter 2022, primarily reflecting
decreased program spend.3
- Net loss was $52.9 million, or
$0.18 per share in the first quarter 2023, and was reduced compared
to a net loss of $85.3 million, or $0.30 per share, for the first
quarter 2022.
2023 Financial Guidance
- For the full-year 2023, the Company
anticipates total Galafold revenue growth between 12 and 17% at
CER1 driven by continued underlying demand from both switch and
treatment-naïve patients, geographic expansion, label extensions,
the continued diagnosis of new Fabry patients, and commercial
execution across all major markets, including the U.S., EU, U.K.,
and Japan.
- Non-GAAP operating expense guidance
for the full-year 2023 is $340 million to $360 million, driven by
prudent expense management offset by continued investment in
Galafold, AT-GAA clinical studies and pre-launch activities, in
addition to certain non-recurring costs for manufacturing to
support the global launch of AT-GAA4.
- The Company is on-track to achieve
non-GAAP profitability2 in the second half of 2023.
Amicus is focused on the following five key strategic
priorities in 2023:
- Sustain double-digit Galafold
revenue growth (12-17% at CER1)
- Secure FDA, EMA, and MHRA approvals
for AT-GAA
- Initiate successful global launches
of AT-GAA
- Advance next generation pipeline
programs (Fabry GTx, Fabry Next-Generation Chaperone, Pompe
GTx)
- Maintain 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.
Conference Call and
Webcast
Amicus Therapeutics will host a conference call
and audio webcast today, May 10, 2023, at 8:30 a.m. ET to discuss
the first quarter 2023 financial results and corporate updates.
Participants and investors interested in accessing the call by
phone will need to register using the online registration form.
After registering, all phone participants will receive a dial-in
number along with a personal PIN number to access the event.
A live audio webcast and related presentation
materials can also be accessed via the Investors section of the
Amicus Therapeutics corporate website at ir.amicusrx.com. Web
participants are encouraged to register on the website 15 minutes
prior to the start of the call. An archived webcast and
accompanying slides will be available on the Company's website
shortly after the conclusion of the live event.
About
Galafold Galafold® (migalastat)
123 mg capsules is an oral pharmacological chaperone of
alpha-Galactosidase A (alpha-Gal A) for the treatment of Fabry
disease in adults who have amenable galactosidase alpha gene
(GLA) variants. In these patients, Galafold works by
stabilizing the body’s own dysfunctional enzyme so that it can
clear the accumulation of disease substrate. Globally, Amicus
Therapeutics estimates that approximately 35 to 50 percent of Fabry
patients may have amenable GLA variants, though
amenability rates within this range vary by geography. Galafold is
approved in more than 40 countries around the world, including the
U.S., EU, U.K., and Japan.
U.S. INDICATIONS AND USAGEGalafold 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 REACTIONSThe most common adverse
reactions reported with Galafold (≥10%) were headache,
nasopharyngitis, urinary tract infection, nausea and pyrexia.
USE IN SPECIFIC POPULATIONSThere is
insufficient clinical data on Galafold use in pregnant women to
inform a drug-associated risk for major birth defects and
miscarriage. Advise women of the potential risk to a fetus.
It is not known if Galafold is present in human milk. Therefore,
the developmental and health benefits of breastfeeding should be
considered along with the mother’s clinical need for Galafold and
any potential adverse effects on the breastfed child from Galafold
or from the underlying maternal condition.
Galafold is not recommended for use in patients with severe
renal impairment or end-stage renal disease requiring dialysis.
The safety and effectiveness of Galafold have not been
established in pediatric patients.
To report Suspected Adverse Reactions, contact Amicus
Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088
or www.fda.gov/medwatch.
For additional information about Galafold, including the full
U.S. Prescribing Information, please
visit https://www.amicusrx.com/pi/Galafold.pdf.
EU Important Safety
InformationTreatment 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 is not intended for concomitant use with enzyme
replacement therapy.
- Galafold is not recommended for use in patients with Fabry
disease who have severe renal impairment (<30 mL/min/1.73 m2).
The safety and efficacy of Galafold in children less than 12 years
of age have not yet been established. No data are available.
- No dosage adjustments are required in patients with hepatic
impairment or in the elderly population.
- There is very limited experience with the use of this medicine
in pregnant women. If you are pregnant, think you may be pregnant,
or are planning to have a baby, do not take this medicine until you
have checked with your doctor, pharmacist, or nurse.
- While taking Galafold, effective birth control should be used.
It is not known whether Galafold is excreted in human milk.
- Contraindications to Galafold include hypersensitivity to the
active substance or to any of the excipients listed in the
PRESCRIBING INFORMATION.
- Galafold 123 mg capsules are not for children (≥12 years)
weighing less than 45 kg.
- It is advised to periodically monitor renal function,
echocardiographic parameters and biochemical markers (every 6
months) in patients initiated on Galafold or switched to
Galafold.
- OVERDOSE: General medical care is recommended in the case of
Galafold overdose.
- The most common adverse reaction reported was headache, which
was experienced by approximately 10% of patients who received
Galafold. For a complete list of adverse reactions, please review
the SUMMARY OF PRODUCT CHARACTERISTICS.
- Call 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 Pompe DiseasePompe
disease is an inherited lysosomal disorder caused by deficiency of
the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels
of GAA lead to accumulation of glycogen in cells, which is believed
to result in the clinical manifestations of Pompe disease. Pompe
disease ranges from a rapidly fatal infantile form with significant
impacts to heart function, to a more slowly progressive, late-onset
form primarily affecting skeletal muscle and progressive
respiratory involvement. Late-onset Pompe disease can be severe and
debilitating, including progressive muscle weakness throughout the
body, particularly the skeletal muscles and muscles controlling
breathing, that worsens over time.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated
biotechnology company focused on discovering, developing and
delivering novel high-quality medicines for people living with rare
diseases. With extraordinary patient focus, Amicus Therapeutics is
committed to advancing and expanding a pipeline of cutting-edge,
first- or best-in-class medicines for rare diseases. For more
information please visit the company’s website at www.amicusrx.com,
and follow on Twitter and LinkedIn.
Non-GAAP Financial Measures In
addition to financial information prepared in accordance with U.S.
GAAP, this press release also contains adjusted financial measures
that we believe provide investors and management with supplemental
information relating to operating performance and trends that
facilitate comparisons between periods and with respect to
projected information. These adjusted financial measures are
non-GAAP measures and should be considered in addition to, but not
as a substitute for, the information prepared in accordance with
U.S. GAAP. We typically exclude certain GAAP items that management
does not believe affect our basic operations and that do not meet
the GAAP definition of unusual or non-recurring items. Other
companies may define these measures in different ways. When we
provide our expectation for non-GAAP operating expenses on a
forward-looking basis, a reconciliation of the differences between
the non-GAAP expectation and the corresponding GAAP measure
generally is not available without unreasonable effort due to
potentially high variability, complexity and low visibility as to
the items that would be excluded from the GAAP measure in the
relevant future period, such as unusual gains or losses. The
variability of the excluded items may have a significant, and
potentially unpredictable, impact on our future GAAP results.
Forward Looking Statement
This press release contains "forward-looking
statements" within the meaning of the Private Securities Litigation
Reform Act of 1995 relating to preclinical and clinical development
of our product candidates, the timing and reporting of results from
preclinical studies and clinical trials, the prospects and timing
of the potential regulatory approval of our product candidates,
commercialization plans, manufacturing and supply plans, financing
plans, and the projected revenues and cash position for the
Company. The inclusion of forward-looking statements should not be
regarded as a representation by us that any of our plans will be
achieved. Any or all of the forward-looking statements in this
press release may turn out to be wrong and can be affected by
inaccurate assumptions we might make or by known or unknown risks
and uncertainties. For example, with respect to statements
regarding the goals, progress, timing, and outcomes of discussions
with regulatory authorities, including as they are impacted by
COVID-19 related disruption, are based on current information. The
potential impact on operations from the COVID-19 pandemic is
inherently unknown and cannot be predicted with confidence and may
cause actual results and performance to differ materially from the
statements in this release, including without limitation, because
of the impact on general political and economic conditions,
including as a result of efforts by governmental authorities to
mitigate COVID-19, such as travel bans, shelter in place orders and
third-party business closures and resource allocations,
manufacturing and supply chain disruptions and limitations on
patient access to commercial or clinical product. In addition to
the impact of the COVID-19 pandemic, actual results may differ
materially from those set forth in this release due to the risks
and uncertainties inherent in our business, including, without
limitation: the potential that results of clinical or preclinical
studies indicate that the product candidates are unsafe or
ineffective; the potential that it may be difficult to enroll
patients in our clinical trials; the potential that regulatory
authorities, including the FDA, EMA, MHRA, and PMDA, may not grant
or may delay approval for our product candidates; the potential
that required regulatory inspections may be delayed or not be
successful and delay or prevent product approval; the potential
that we may not be successful in commercializing Galafold in
Europe, Japan, the US and other geographies or AT-GAA if and when
approved; the potential that preclinical and clinical studies could
be delayed because we identify serious side effects or other safety
issues; the potential that we may not be able to manufacture or
supply sufficient clinical or commercial products; and the
potential that we will need additional funding to complete all of
our studies and manufacturing. Further, the results of earlier
preclinical studies and/or clinical trials may not be predictive of
future results. Statements regarding corporate financial guidance
and financial goals and the attainment of such goals. With respect
to statements regarding projections of the Company's revenue and
cash position, actual results may differ based on market factors
and the Company's ability to execute its operational and budget
plans. In addition, all forward-looking statements are subject to
other risks detailed in our Annual Report on Form 10-K for the year
ended December 31, 2022, and on Form 10-Q for the quarter ended
March 31, 2023, to be filed today. You are cautioned not to place
undue reliance on these forward-looking statements, which speak
only as of the date hereof. All forward-looking statements are
qualified in their entirety by this cautionary statement, and we
undertake no obligation to revise or update this news release to
reflect events or circumstances after the date hereof.
CONTACT:
Investors: Amicus Therapeutics Andrew
Faughnan
Vice President, Investor Relations
afaughnan@amicusrx.com(609) 662-3809
Media: Amicus Therapeutics Diana Moore Head of
Global Corporate Communications
dmoore@amicusrx.com(609) 662-5079
FOLD-G
TABLE 1
Amicus Therapeutics, Inc.Consolidated
Statements of
Operations(Unaudited)(in
thousands, except share and per share amounts) |
|
Three Months Ended March 31, |
|
|
2023 |
|
|
|
2022 |
|
Net product sales |
$ |
86,270 |
|
|
$ |
78,715 |
|
Cost of goods sold |
|
6,942 |
|
|
|
7,582 |
|
Gross profit |
|
79,328 |
|
|
|
71,133 |
|
Operating expenses: |
|
|
|
Research and development |
|
41,499 |
|
|
|
81,517 |
|
Selling, general, and administrative |
|
73,957 |
|
|
|
58,116 |
|
Changes in fair value of contingent consideration payable |
|
251 |
|
|
|
(1,188 |
) |
Loss on impairment of assets |
|
— |
|
|
|
6,616 |
|
Depreciation and amortization |
|
1,257 |
|
|
|
1,411 |
|
Total operating expenses |
|
116,964 |
|
|
|
146,472 |
|
Loss from operations |
|
(37,636 |
) |
|
|
(75,339 |
) |
Other (expense) income: |
|
|
|
Interest income |
|
2,199 |
|
|
|
133 |
|
Interest expense |
|
(11,844 |
) |
|
|
(8,147 |
) |
Other (expense) income |
|
(5,938 |
) |
|
|
1,902 |
|
Loss before income tax |
|
(53,219 |
) |
|
|
(81,451 |
) |
Income tax benefit
(expense) |
|
287 |
|
|
|
(3,809 |
) |
Net loss attributable
to common stockholders |
$ |
(52,932 |
) |
|
$ |
(85,260 |
) |
Net loss attributable to
common stockholders per common share — basic and diluted |
$ |
(0.18 |
) |
|
$ |
(0.30 |
) |
Weighted-average common shares
outstanding — basic and diluted |
|
291,336,750 |
|
|
|
288,481,741 |
|
TABLE 2
Amicus Therapeutics, Inc.Consolidated
Balance Sheets(Unaudited)(in
thousands, except share and per share
amounts) |
|
March 31,2023 |
|
December 31, 2022 |
Assets |
|
|
|
Current assets: |
|
|
|
Cash and cash equivalents |
$ |
160,602 |
|
|
$ |
148,813 |
|
Investments in marketable securities |
|
106,507 |
|
|
|
144,782 |
|
Accounts receivable |
|
68,178 |
|
|
|
66,196 |
|
Inventories |
|
27,004 |
|
|
|
23,816 |
|
Prepaid expenses and other current assets |
|
37,406 |
|
|
|
40,209 |
|
Total current assets |
|
399,697 |
|
|
|
423,816 |
|
Operating lease right-of-use assets, net |
|
28,483 |
|
|
|
29,534 |
|
Property and equipment, less accumulated depreciation of $22,901
and $22,281 at March 31, 2023 and December 31, 2022,
respectively |
|
31,406 |
|
|
|
30,778 |
|
Intangible asset, less accumulated depreciation of $36 and $0 at
March 31, 2023 and December 31, 2022, respectively |
|
22,964 |
|
|
|
23,000 |
|
Goodwill |
|
197,797 |
|
|
|
197,797 |
|
Other non-current assets |
|
20,172 |
|
|
|
19,242 |
|
Total
Assets |
$ |
700,519 |
|
|
$ |
724,167 |
|
Liabilities and
Stockholders' Equity |
|
|
|
Current liabilities: |
|
|
|
Accounts payable |
$ |
24,965 |
|
|
$ |
15,413 |
|
Accrued expenses and other current liabilities |
|
92,747 |
|
|
|
93,636 |
|
Contingent consideration payable |
|
12,668 |
|
|
|
21,417 |
|
Operating lease liabilities |
|
8,005 |
|
|
|
8,552 |
|
Total current liabilities |
|
138,385 |
|
|
|
139,018 |
|
Long-term debt |
|
392,658 |
|
|
|
391,990 |
|
Operating lease liabilities |
|
51,349 |
|
|
|
51,578 |
|
Deferred reimbursements |
|
5,906 |
|
|
|
4,656 |
|
Deferred income taxes |
|
— |
|
|
|
4,939 |
|
Other non-current liabilities |
|
9,648 |
|
|
|
8,939 |
|
Total liabilities |
|
597,946 |
|
|
|
|
|
Commitments and
contingencies |
|
|
|
Stockholders' equity: |
|
|
|
Common stock, $0.01 par value,
500,000,000 shares authorized, 283,300,585 and 281,108,273 shares
issued and outstanding at March 31, 2023 and December 31, 2022,
respectively |
|
2,820 |
|
|
|
2,815 |
|
Additional paid-in
capital |
|
2,691,836 |
|
|
|
2,664,744 |
|
Accumulated other
comprehensive loss: |
|
|
|
Foreign currency translation adjustment |
|
(6,543 |
) |
|
|
(11,989 |
) |
Unrealized loss on available-for-sale securities |
|
(201 |
) |
|
|
(116 |
) |
Warrants |
|
83 |
|
|
|
83 |
|
Accumulated deficit |
|
(2,585,422 |
) |
|
|
(2,532,490 |
) |
Total stockholders'
equity |
|
102,573 |
|
|
|
123,047 |
|
Total Liabilities and
Stockholders' Equity |
$ |
700,519 |
|
|
$ |
724,167 |
|
TABLE 3
Amicus
Therapeutics, Inc.Reconciliation of Non-GAAP
Financial Measures(in
thousands) |
|
Three Months Ended March 31, |
|
|
2023 |
|
|
2022 |
|
Total operating expenses
- as reported GAAP |
$ |
116,964 |
|
$ |
146,472 |
|
Research and development: |
|
|
|
Stock-based compensation |
|
8,490 |
|
|
9,365 |
|
Selling, general and administrative: |
|
|
|
Stock-based compensation |
|
26,404 |
|
|
21,286 |
|
Loss on impairment of assets |
|
— |
|
|
6,616 |
|
Changes in fair value of contingent consideration
payable |
|
251 |
|
|
(1,188 |
) |
Depreciation and amortization |
|
1,257 |
|
|
1,411 |
|
Total operating expense
adjustments to reported GAAP |
|
36,402 |
|
|
37,490 |
|
Total operating expenses - as adjusted |
$ |
80,562 |
|
$ |
108,982 |
|
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