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 2021 revenue, corporate updates, and full-year 2022
outlook and revenue guidance.
Corporate Highlights:
- Global revenue for
Galafold® (migalastat) in 2021
reached $306 million driven by strong new patient accruals and
sustained patient adherence, representing a year-over-year increase
of 17%.
- AT-GAA regulatory reviews
are underway: In the U.S., the Food and Drug
Administration (FDA) accepted for review the Biologics License
Application (BLA) for cipaglucosidase alfa and the New Drug
Application (NDA) for miglustat, the two components of AT-GAA. The
FDA has set a Prescription Drug User Fee Act (PDUFA) action date of
May 29, 2022 for the NDA and July 29, 2022 for the BLA. In the EU,
the Marketing Authorization Applications (MAA) were submitted and
validated in the fourth quarter by the European Medicines Agency
(EMA).
- AT-GAA launch preparations
are accelerating: Development of global launch plans,
targeted investments in additional personnel, and launch inventory
are fully underway as company believes AT-GAA can rapidly become
the new standard of care treatment regimen for people living with
Pompe disease.
- Pipeline of next generation
genetic medicines to advance through both internal efforts and
creation of R&D focused new company, Caritas
Therapeutics.
- Cash Flow and Balance Sheet
sufficient to achieve self-sustainability and profitability in
2023. Through careful management of expenses, the Company
is on the path to achieve self-sustainability and profitability in
2023 as it executes on the global Galafold expansion and prepares
for AT-GAA global launch.
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics, Inc., stated, “In 2021, Amicus made
great strides for people worldwide living with rare diseases
through the broad execution of our annual strategic priorities.
Despite the resurgence of COVID with Delta and Omicron variants,
the Galafold business remains very strong, and we delivered on our
full year revenue guidance and expect robust growth this year
driven by strong adoption across the globe for our Fabry disease
precision medicine. We are underway with the global regulatory
reviews and launch preparations for AT-GAA in Pompe disease with
high expectations that this novel medicine has the potential to
become the new standard of care in Pompe disease treatment and the
potential to address unmet needs for thousands of Pompe patients in
the years ahead. We see further opportunity ahead to impact the
lives of those living with rare disease through our genetic
medicine business and capabilities. Together, Amicus is in a
stronger position than ever and we remain steadfast on our mission
of transforming the lives of people living with rare,
life-threatening conditions and creating significant value for our
shareholders.”
Bradley Campbell, President and Chief Operating
Officer of Amicus Therapeutics, Inc., stated, “We are looking ahead
to transforming Amicus into a leading global rare disease
biotechnology company led by two innovative therapies that we
believe meaningfully impact the lives of people living with Fabry
and Pompe disease. This year we will be focused on continuing to
bring Galafold to patients around the world and delivering on the
anticipated approval and launch of AT-GAA.”
Amicus is focused on the following five key
strategic priorities in 2022:
- Continued double-digit Galafold
growth (15-20%) with revenue of $350M to $365M
- Secure FDA approval and positive
CHMP opinion for AT-GAA
- Initiate successful, rapid launch
in the U.S. for AT-GAA
- Advance best-in-class next
generation genetic medicines and capabilities
- Maintain strong financial position
on path to profitability
Mr. Crowley and Mr. Campbell will discuss the
Amicus corporate objectives and key milestones in a presentation at
the 40th Annual J.P. Morgan Healthcare Conference on Wednesday,
January 12, 2022, at 3:45 p.m. ET. 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.
Full-Year 2021 Revenue Summary and 2022
Revenue Guidance
Global revenue for Galafold in full-year 2021
was approximately $306 million, preliminary and unaudited,
representing a year-over-year increase of 17% from total revenue of
$260.9 million in 2020. Full-year revenue benefited from a positive
currency impact of approximately $7 million. Fourth quarter
Galafold revenue was approximately $84 million, preliminary and
unaudited.
For the full-year 2022, the Company anticipates
total Galafold revenue of $350 million to $365 million.
Double-digit revenue growth (15-20%) in 2022 is expected to be
driven by continued underlying demand from both switch and naïve
patients, geographic expansion, the continued diagnosis of new
Fabry patients and commercial execution across all major markets,
including the U.S., EU, U.K., and Japan.
The current cash position is sufficient to
achieve self-sustainability and profitability in 2023.
Updates and Anticipated Milestones by
Program
Galafold (migalastat) Oral Precision
Medicine for Fabry Disease
- Sustain double-digit revenue growth in 2022 of $350 million to
$365 million
- Continue geographic expansion
- Registry and other Phase 4 studies ongoing
AT-GAA for Pompe Disease
- U.S. Prescription Drug User Fee Act (PDUFA) action date of May
29, 2022 for the NDA and July 29, 2022 for the BLA
- EU Committee for Medicinal Products for Human Use (CHMP)
opinion expected in late 2022
- Continue to broaden access through early access plans in the
U.K., Germany, Japan, and other countries
- Ongoing supportive studies, including pediatric and extension
studies
Gene Therapy Pipeline
- Advance IND-enabling studies,
manufacturing activities, and regulatory activities for the Fabry
disease gene therapy program towards an anticipated IND in
2023
- Progress preclinical studies,
manufacturing activities, and regulatory activities for the Pompe
disease gene therapy program
- Discontinue CLN6 Batten disease
gene therapy program following review of long-term extension study
data. It was recently determined that any initial stabilization of
disease progression at the two-year time point was not maintained
through the long-term extension study. Amicus plans to further
analyze and share the Phase 1/2 data with key stakeholders in the
CLN6 Batten disease community and work with the community to
support continued research efforts to find better treatments and
cures which are so desperately and urgently needed
- Advance CLN3 Batten disease program
with the higher dose, different promoter, and intra-cisterna magna
(ICM) route of delivery pending further Phase 1/2 clinical data and
pre-clinical data expected in 2022. These data will inform timeline
for commencement of any pivotal clinical study
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 Fabry patients may have
amenable GLA variants, though amenability rates within
this range vary by geography. Galafold is approved in over 40
countries around the world, including the U.S., EU, U.K., Japan and
others.
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 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 metabolic diseases. With extraordinary patient
focus, Amicus Therapeutics is committed to advancing and expanding
a robust pipeline of cutting-edge, first- or best-in-class
medicines for rare metabolic diseases. For more information please
visit the company’s website at www.amicusrx.com, and follow us
on Twitter and LinkedIn.
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 in particular the potential goals,
progress, timing, and results of preclinical studies and clinical
trials, 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 our other product
candidates 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, 2020 and the Quarterly
Report filed on Form 10-Q for the quarter ended September 30, 2021.
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
FaughnanExecutive Director, 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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