Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global
biotechnology company focused on developing and commercializing
novel medicines for rare diseases, today provided its preliminary
and unaudited 2022 revenue, corporate updates, and full-year 2023
outlook.
Corporate Highlights:
- Global revenue in 2022
reached $329 million (preliminary and unaudited) driven by
strong new patient accruals and sustained patient adherence,
representing a year-over-year operational revenue growth measured
at constant exchange rates (CER)1 of 16%. Full-year revenue growth
measured at actual exchange rates was 8% reflecting a negative
currency impact of approximately $26 million, or 8%, in 2022.
Fourth quarter revenue was approximately $88 million (preliminary
and unaudited).
- For the full-year 2023, the
Company anticipates double-digit Galafold revenue growth of 12-17%
at CER. Growth is expected to be driven by continued
underlying demand from both switch and treatment-naïve patients,
geographic expansion, continued diagnosis of new Fabry patients,
and commercial execution across all major markets, including the
U.S., EU, U.K., and Japan.
- Multiple approvals and
launches expected in 2023 for AT-GAA in Late-onset Pompe
disease. In Europe, the Committee for Medicinal Products
for Human Use (CHMP) adopted a positive opinion of Pombiliti™, also
known as cipaglucosidase alfa. A CHMP opinion for miglustat, the
enzyme stabilizer component of AT-GAA is expected in the second
quarter 2023. The regulatory submission process for AT-GAA in the
U.K. was initiated in December 2022, with final approval expected
in the second half of 2023. As previously announced, in the U.S.,
the Food and Drug Administration (FDA) deferred action on AT-GAA.
Amicus remains actively engaged with the Agency on developing a
plan and logistics for the pre-approval inspection and once there
is more clarity, will provide expected approval timing.
- 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.,
France, Germany, and Japan.
- Galafold U.S. intellectual
property estate strengthened following the issuance of 19 new
patents in 2022. Galafold is protected by orphan drug
regulatory exclusivities and a broad U.S. intellectual property
portfolio of 46 orange book-listed patents, including 5 composition
of matter patents, 30 of which provide protection through at least
2038.
- Based on the current
operating plan, the timing of AT-GAA approvals, and through careful
management of expenses, the Company is on track to achieve non-GAAP
profitability2 in the second half
of 2023.
Bradley Campbell, President and Chief Executive
Officer of Amicus Therapeutics, Inc., stated, “In 2022, Amicus
remained steadfast in our mission to transform the lives of people
living with rare diseases with excellent progress made across our
strategic priorities. The Galafold business remained very strong
last year, delivering double-digit operational revenue growth and
finishing the year with over 2,000 patients on Galafold. We
continue to expect robust growth again for 2023 driven by patient
demand across the globe for this precision medicine for Fabry
disease. In Pompe disease, we eagerly anticipate multiple AT-GAA
regulatory approvals in key geographies including the EU, U.K., and
U.S. following completion of the FDA inspection, and look forward
to launching our second commercial product. We remain excited for
the opportunity to offer a new and innovative treatment option, one
that we believe has the potential to be the new standard of care,
to people living with Pompe disease around the world. We believe we
have the opportunity to deliver significant value for our
shareholders as Amicus transforms into a leading global rare
disease biotechnology company with two innovative therapies that
can make a significant impact on the lives of people living with
Fabry disease and Pompe disease.”
Amicus is focused on the following five key
strategic priorities in 2023:
- Sustain double-digit Galafold
revenue growth (12-17% at CER)
- 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
Mr. Campbell will discuss the Amicus corporate
objectives and key milestones in a presentation at the 41st Annual
J.P. Morgan Healthcare Conference on Monday, January 9, 2023, at
2:15 p.m. PT. A live webcast of the presentation can be accessed
through the Investors section of the Amicus Therapeutics corporate
website at http://ir.amicusrx.com/events.cfm, and will be archived
for 90 days.
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 actual exchange rate 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. We define
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, loss on impairment of
assets, restructuring charges, and income taxes.
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 DiseaseFabry
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 TherapeuticsAmicus
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 StatementThis
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, and PMDA, may not grant or may
delay approval for our product candidates; 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,
2021 and the Quarterly Report filed on Form 10-Q for the quarter
ended September 30, 2022. 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
Executive Director, Investor Relations
afaughnan@amicusrx.com(609) 662-3809
Media: Amicus Therapeutics Diana Moore Head of
Global Corporate Communications
dmoore@amicusrx.com(609) 662-5079
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