310+ People with Fabry Disease Treated with
Reimbursed Galafold® at YE17
Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company
focused on discovering, developing and delivering novel
cutting-edge medicines for rare metabolic diseases, today provided
its full-year 2018 strategic outlook and financial guidance.
Key 2017 Accomplishments
- Exceeded “Target 300” goal with more than 310 people treated
with reimbursed Galafold™ (migalastat) oral precision medicine for
Fabry disease at year-end 2017. Full-year 2017 Galafold revenue
totaled approximately $36 million.
- Completed global regulatory submissions for migalastat in Japan
(J-NDA), the U.S. (NDA), and other key geographies
- Established important clinical proof-of-concept for novel,
highly differentiated Pompe treatment regimen ATB200/AT2221 on
safety, functional outcomes and key disease biomarkers
- Successfully scaled manufacture of Pompe biologic engineering
batches at commercial scale (1,000L) with capacity plans to ensure
that entire Pompe population can be served as quickly as
possible
- Strengthened balance sheet with total cash, cash equivalents
and marketable securities of $359 million at December 31, 2017 and
cash runway into the second half of 2019
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics, Inc. stated, “During 2017 we
continued to build a leading global rare disease biotech company
while advancing our vision to maximize the impact of our medicines
for people living with rare diseases. We exceeded our EU launch and
regulatory objectives for our Fabry precision medicine Galafold,
and we reported clinical data from our Pompe clinical study that
lays the foundation for a potential new treatment paradigm for this
muscle disease. Throughout 2018 we are poised to create significant
additional value for patients and shareholders across our key
programs in Fabry and Pompe, and through our focused commitment to
advancing and expanding a pipeline of novel medicines for rare
metabolic diseases.”
Amicus is focused on five key strategic
priorities in 2018:
- Double global revenue for Galafold ($75 million - $85
million)
- Secure approvals for migalastat in Japan and the U.S.
- Achieve clinical, manufacturing and regulatory milestones to
advance ATB200/AT2221 toward global regulatory submissions and
approvals as soon as possible
- Develop and expand preclinical pipeline to ensure at least one
new clinical program in 2019
- Maintain a strong balance sheet
Mr. Crowley will discuss Amicus' corporate
objectives and key milestones in a presentation at the 36th Annual
J.P. Morgan Healthcare Conference on Tuesday, January 9, 2018 at
8:00 a.m. PT (11:00 a.m. ET). A live webcast of the presentation
can be accessed through the Investors section of the Amicus
Therapeutics corporate web site at
http://ir.amicusrx.com/events.cfm, and will be archived for 90
days.
Full-Year 2017 Financial Summary and
2018 Guidance
Amicus recorded approximately $36 million in
full-year 2017 revenue from commercial sales and reimbursed
expanded access programs for Galafold. For the full-year 2018 the
Company anticipates total Galafold revenue of $75 million to $85
million.
Cash, cash equivalents, and marketable
securities totaled approximately $359 million at December 31, 2017.
As previously announced, the Company strengthened the balance sheet
during 2017 with a $243.0 million in net proceeds from a follow on
public offering in July 2017. The Company expects full-year 2018
net cash spend of between $230 million and $260 million. The
current cash position is anticipated to fund ongoing operations
into at least the second half of 2019.
Program Highlights
Migalastat for Fabry
DiseaseMigalastat is an oral precision medicine intended
to treat Fabry disease in patients who have amenable genetic
mutations. Regulatory authorities in the European Union,
Switzerland, Israel, Canada, Australia, and South Korea have
granted full approval for migalastat under the trade name Galafold.
The EU approval may serve as the basis for regulatory approvals in
more than two-thirds of the global Fabry market that is outside the
U.S. In the U.S., Amicus submitted a new drug application (NDA) to
the FDA in December 2017 to seek approval of migalastat.
Amicus is committed to advancing the highest
quality therapies for all people living with Fabry disease. For
people with non-amenable mutations who are not eligible for
migalastat as an oral precision medicine, the Company has
established initial preclinical proof-of-concept for a novel Fabry
ERT (ATB101) co-formulated with migalastat as part of our CHART®
platform.
Global Fabry Updates:
- 310+ patients (naïve and ERT-switch) on reimbursed Galafold as
of December 31, 2017
- Total full-year 2017 revenue of $36 million from global
commercial sales and expanded access programs (EAPs)
- Pricing and reimbursement secured in 15 countries
- Approvals secured in EU, Australia, Canada, Israel, South Korea
and Switzerland
- Approvals pending in Japan, U.S. and other key geographies
Anticipated Milestones:
- FDA acceptance of U.S. NDA for filing (1Q18)
- Regulatory decision on Japanese J-NDA (1H18)
- Total full-year 2018 revenue guidance of $75 million to $85
million
- ATB101 co-formulated with migalastat advancing toward the
clinic in 2019
ATB200/AT2221 for Pompe
Disease
ATB200/AT2221 is a novel treatment paradigm
that consists of ATB200, a unique recombinant human acid
alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate
structures, particularly mannose-6 phosphate (M6P), to enhance
uptake, co-administered with AT2221, a pharmacological chaperone.
Throughout 2017, Amicus presented a cascade of positive data
from an ongoing global Phase 1/2 clinical study (ATB200-02) to
evaluate safety, tolerability, pharmacokinetics (PK), and
pharmacodynamics (PD) of ATB200/AT2221 across ambulatory ERT-switch
patients (Cohort 1), non-ambulatory ERT-switch patients (Cohort 2),
and ERT-naïve patients (Cohort 3).
Amicus continues to assemble the highest quality
data package and to scale up manufacturing to meet the needs of the
Pompe community. The Company is in the midst of a series of
collaborative discussions with U.S. and EU regulators
regarding the best and fastest pathway forward for this novel
treatment option, and continues to anticipate a Pompe regulatory
pathway update in the first half of
2018.
Pompe Program Updates:
- Data collection underway in a retrospective natural history
study (POM-002)
- Prospective observational study (POM-003) initiated
- GMP production of ATB200 commenced at the large commercial
scale (1,000 Liters)
- FDA agreement reached on comparability between 250L scale and
1000L engineering batches
- FDA agreement reached on testing strategy for demonstrating
comparability between 250L scale and 1000L GMP batches
Anticipated Upcoming Pompe Program
Milestones:
- Expansion of ongoing ATB200-02 clinical study to include four
to six additional ambulatory ERT-switch patients
- Additional data from ATB200-02 clinical study
at 14th Annual WORLDSymposium™ (February 5-9, 2018)
- Final regulatory agreement on manufacturing comparability
between 1,000L and 250L GMP scale
- Completion and release for clinic of 1,000L GMP commercial
scale material
- Pompe regulatory pathway update (1H18)
- Initiation of larger registration-directed study
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 0–15 years of age
have not yet been established.
- 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.
- 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 Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a global, patient-centric
biotechnology company focused on discovering, developing and
delivering novel high-quality medicines for people living with rare
metabolic diseases. The cornerstone of the Amicus portfolio is
migalastat, an oral precision medicine for people living with Fabry
disease who have amenable genetic mutations. Migalastat is
currently approved under the trade name Galafold™ in the European
Union, with additional approvals granted and pending in several
geographies. The future value driver of the Amicus pipeline is
ATB200/AT2221, a novel, late-stage, potential best-in-class
treatment paradigm for Pompe disease. The Company is committed to
advancing and expanding a robust pipeline of cutting-edge, first-
or best-in-class medicines for rare metabolic diseases.
Forward-Looking StatementsThis
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, 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 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. 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,
2016 and Quarterly Report on Form 10-Q for the quarter ended
September 30, 2017. 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.
CONTACTS:
Investors/Media:Amicus
TherapeuticsSara Pellegrino, IRCSenior Director, Investor
Relationsspellegrino@amicusrx.com (609) 662-5044
Media:Pure CommunicationsJennifer Paganelli
jpaganelli@purecommunications.com (347) 658-8290
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