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 2023 revenue, corporate updates, and full-year 2024
outlook.
In 2023, Amicus met or exceeded its strategic
priorities, highlighted by:
- Sustaining double-digit Galafold
revenue growth
- Securing FDA, EMA, and MHRA
approvals for Pombiliti + Opfolda
- Initiating successful global
launches of Pombiliti + Opfolda
- Advancing next-generation pipeline
programs
- On-track to achieving non-GAAP
profitability in the fourth quarter of 2023
Preliminary and Unaudited 2023
Revenue:
- Total
revenue in 2023 reached ~$399.4 million, representing a
year-over-year increase of 21%, reflecting strong
operational growth measured at constant exchange rates (CER)1 of
20% and a favorable currency impact of approximately $2.7 million,
or 1%. Fourth quarter total revenue was ~$115.1 million.
- Galafold
(migalastat) net product sales in 2023 were ~$387.8
million, representing a year-over-year increase of
18%, or 17% at CER. Fourth quarter Galafold net product
sales were ~$106.6 million.
- Pombiliti (cipaglucosidase
alfa-atga) + Opfolda (miglustat) net product sales in 2023 were
~$11.6 million. The commercial launch of Pombiliti +
Opfolda is successfully underway in the three largest markets with
~120 patients on treatment with commercial product or scheduled to
be treated as of the end of 2023. Fourth quarter Pombiliti +
Opfolda net product sales were ~$8.5 million.
Bradley Campbell, President and Chief Executive
Officer of Amicus Therapeutics, Inc., stated, “Last year was an
incredible year for Amicus highlighted by the continued
double-digit growth of Galafold sales and the global regulatory
approvals of our second commercial therapy, which is off to a
fantastic launch. We expect 2024 to be a truly transformative year
as we continue to drive significant revenue growth by treating an
increasing number of Fabry patients globally, executing on the
global launches of Pombiliti and Opfolda, which we believe has the
potential to become the standard of care in a >$1B market today,
and delivering our first full year of non-GAAP profitability. We
look forward to reporting on our progress throughout this year as
we further our mission for people living with rare diseases.”
Amicus is focused on the following four key
strategic priorities in 2024:
- Delivering double-digit Galafold
revenue growth (11-16% at CER)
- Ensuring the successful global
launches of Pombiliti + Opfolda
- Advancing ongoing studies to
support medical and scientific leadership in Fabry and Pompe
diseases
- Achieving full year non-GAAP
profitability2
Mr. Campbell will discuss the Amicus corporate
objectives and key milestones in a presentation at the 42nd Annual
J.P. Morgan Healthcare Conference on Monday, January 8, 2024, 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 2024 Galafold revenue guidance utilizes actual
exchange rate as of December 31, 2023.2 Non-GAAP Net Income defined
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
GalafoldGalafold® (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 drug reactions reported
with Galafold (≥10 %) are headache, nasopharyngitis, urinary tract
infection, nausea, and pyrexia.
DRUG INTERACTIONS
Avoid co-administration of Galafold with
caffeine at least 2 hours before and 2 hours after taking
Galafold.
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 Therapeutic
IndicationGalafold® (migalastat) is indicated for
long-term treatment of adults and adolescents aged 12 years and
older with a confirmed diagnosis of Fabry disease (α-galactosidase
A deficiency) and who have an amenable mutation.
EU Important Safety
InformationTreatment with Galafold should be initiated and
supervised by specialist physicians experienced in the diagnosis
and treatment of Fabry disease. Galafold is not intended for
concomitant use with enzyme replacement therapy.
The safety and efficacy of Galafold in children
aged less than 12 years have not been established. No data are
available.
Galafold is contraindicated in patients with
hypersensitivity to the active substance or to any of the
excipients listed in the Summary of Product Characteristics
(SmPC).
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 or switched to Galafold.
In case of meaningful clinical deterioration, further clinical
evaluation or discontinuation of treatment with Galafold should be
considered.
Galafold is not indicated for use in patients
with non-amenable mutations.
Galafold is not recommended for use in patients
with severe renal insufficiency, defined as estimated GRF less than
30 mL/min/1.73m2.
Food and caffeine should not be consumed at
least 2 hours before and 2 hours after taking Galafold to give a
minimum 4 hours fast.
Galafold is not recommended in women of
childbearing potential not using contraception. Galafold is not
recommended during pregnancy. It is not known whether Galafold is
secreted in human milk.
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 SmPC.
OVERDOSE: General medical care is recommended in
the case of Galafold overdose.
For complete information please see the EU SmPC available at
https://www.ema.europa.eu/en/medicines/human/EPAR/galafold
About Pombiliti +
OpfoldaPombiliti + 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 USAGEPOMBILITI 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.
EU Important Safety
InformationPombiliti (cipaglucosidase alfa)
Important Safety Information
Posology and Method of
Administration: Pombiliti must be used in combination with
miglustat 65 mg hard capsules. The recommended dose of Pombiliti is
20 mg/kg of body weight every other week. The Pombiliti infusion
should start 1 hour after taking miglustat capsules.
Paediatric population: The safety and efficacy of
Pombiliti in combination with miglustat therapy in paediatric
patients less than 18 years old have not yet been established. No
data are available.
Contraindications:
Life-threatening hypersensitivity to the active substance, or to
any of the excipients. Contraindication to miglustat.
Anaphylaxis and infusion-associated reactions
(IARs): Serious anaphylaxis and IARs have occurred in some
patients during infusion and following infusion with Pombiliti.
Premedication with oral antihistamine, antipyretics, and/or
corticosteroids may be administered to assist with signs and
symptoms related to IARs experienced with prior enzyme replacement
therapy (ERT) treatment. Reduction of the infusion rate, temporary
interruption of the infusion, symptomatic treatment with oral
antihistamine, or antipyretics, and appropriate resuscitation
measures should be considered to manage serious IARs. If
anaphylaxis or severe allergic reactions occur, infusion should be
immediately paused, and appropriate medical treatment should be
initiated. The current medical standards for emergency treatment of
anaphylactic reactions are to be observed and cardiopulmonary
resuscitation equipment should be readily available. The risks and
benefits of re-administering Pombiliti following anaphylaxis or
severe allergic reaction should be carefully considered, and
appropriate resuscitation measures made available. Risk of
acute cardiorespiratory failure in susceptible patients:
Patients with acute underlying respiratory illness or compromised
cardiac and/or respiratory function may be at risk of serious
exacerbation of their cardiac or respiratory compromise during
infusions. Appropriate medical support and monitoring measures
should be readily available during Pombiliti infusion.
Immune complex-related reactions: Immune
complex-related reactions have been reported with other ERTs in
patients who had high IgG antibody titres, including severe
cutaneous reactions and nephrotic syndrome. If immune
complex-related reactions occur, discontinuation of the
administration of Pombiliti should be considered and appropriate
medical treatment should be initiated. The risks and benefits of
re-administering Pombiliti following an immune complex-related
reaction should be reconsidered for each individual patient.
Contraception in females: Reliable contraceptive
measures must be used by women of childbearing potential during
treatment with Pombiliti in combination with miglustat, and for 4
weeks after discontinuing treatment. Pregnancy:
Pombiliti in combination with miglustat therapy is not recommended
during pregnancy. Breast feeding: It is not known
if Pombiliti and miglustat are secreted in human breast milk. A
decision must be made whether to discontinue breast-feeding or to
discontinue/abstain from Pombiliti in combination with miglustat
therapy, taking into account the benefit of breast-feeding for the
child and the benefit of therapy for the woman. Summary of
the safety profile: The most commonly reported adverse
reactions only attributable to Pombiliti were chills (4.0%),
dizziness (2.6%), flushing (2.0%), somnolence (2.0%), chest
discomfort (1.3%), cough, (1.3%), infusion site swelling (1.3%),
and pain (1.3%). Reported serious adverse reactions only
attributable to Pombiliti were urticaria (2.0%), anaphylaxis
(1.3%), pyrexia (0.7%), presyncope (0.7%), dyspnoea (0.7%),
pharyngeal oedema (0.7%), wheezing (0.7%), and hypotension (0.7%).
Refer to SmPC for full list.
Opfolda (miglustat) 65 mg hard capsules
Important Safety Information
Posology and Method of
Administration: Opfolda must be used in combination with
Pombiliti. The recommended dose is to be taken orally every other
week and is based on body weight. Opfolda should be taken
approximately 1 hour but no more than 3 hours before the start of
the Pombiliti infusion. Paediatric population: The
safety and efficacy of Opfolda in combination with Pombiliti
therapy in paediatric patients less than 18 years old have not yet
been established. No data are available.
Contraindications: Hypersensitivity to the active
substance or to any of the excipients. Contraindication to
cipaglucosidase alfa. Food Interaction: Patients
should fast for 2 hours before and 2 hours after taking Opfolda.
Contraception in females: Reliable contraceptive
measures must be used by women of childbearing potential during
treatment with Opfolda in combination with Pombiliti, and for 4
weeks after discontinuing treatment. Pregnancy:
Opfolda crosses the placenta. Opfolda in combination with Pombiliti
therapy is not recommended during pregnancy. Breast
feeding: It is not known if Opfolda and Pombiliti are
secreted in human breast milk. A decision must be made whether to
discontinue breast-feeding or to discontinue/abstain from Opfolda
in combination with Pombiliti therapy, taking into account the
benefit of breastfeeding for the child and the benefit of therapy
for the woman. Summary of the safety profile: The
most commonly reported adverse reaction only attributable to
Opfolda 65 mg was constipation (1.3%). Refer to SmPC for full
list.
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 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 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, 2022, and on Form 10-Q for the
quarter ended September 30, 2023. 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
FaughnanVice President, Investor
Relationsafaughnan@amicusrx.com(609) 662-3809
Media: Amicus Therapeutics Diana Moore Head of
Global Corporate Communicationsdmoore@amicusrx.com(609)
662-5079
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