Continued Global
Galafold®
(Migalastat) Adoption and Expansion – Reaffirms FY18
Revenue Guidance of $75M-$85M at Top End of Range
Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company
focused on discovering, developing and delivering novel medicines
for rare metabolic diseases, today announced financial results for
the first quarter ended March 31, 2018. The Company also summarized
recent program updates and reiterated full-year 2018 revenue and
net cash spend guidance.
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics, Inc. stated, “Since the beginning
of 2018, we have successfully executed across our five key
strategic priorities for the year. We are on track to more than
double our Galafold® revenue from last year, we secured approval of
Galafold in Japan, and we have advanced our Pompe clinical,
manufacturing and regulatory activities. Along with our key value
drivers in Fabry and Pompe, we continue to evaluate other potential
first- or best-in-class technologies that we believe may deliver
significant benefits for many more people living with rare
metabolic diseases. Today, more than ever, we are well positioned
to achieve our vision to impact as many people as possible who are
living with rare metabolic diseases as we continue to build a
leading global rare disease biotech company.”
2018 Key Strategic
Priorities
- 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 AT-GAA (also known as 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
First Quarter 2018 Financial Results and
Full-Year 2018 Financial Guidance
- Total revenue in the first quarter 2018 was $16.7 million, a
year-over-year increase of 300% from total revenue of $4.2 million
in the first quarter of 2018.
- Cash, cash equivalents, and marketable securities totaled
$605.2 million at March 31, 2018, compared to $358.6 million at
December 31, 2017.
- Total operating expenses increased to $70.3 million for the
first quarter 2018 compared to $55.4 million in the first quarter
2017 reflecting increased investment in Pompe clinical and
manufacturing activities as well as Galafold commercial launch and
launch preparations.
- Net cash spend was $48.0 million for the first quarter
2018.
- Net loss was $49.9 million, or $0.28 per share, compared to a
net loss of $55.0 million, or $0.39 per share, for the first
quarter 2017.
“The first quarter of 2018 marked another period
of continued growth for Galafold and our strongest balance sheet in
our Company’s history,” said Bradley L. Campbell, President and COO
of Amicus Therapeutics. “We are confident in our ability to achieve
the high end of our full-year 2018 Galafold revenue guidance given
the current and anticipated increase in patient and physician
adoption; expansion into new countries; and what we are seeing as a
very high rate of compliance and adherence for this oral precision
medicine.”
2018 Financial Guidance
For the full-year 2018 the Company anticipates
total Galafold revenue at the high end of the $75 million to $85
million guidance range. The Galafold revenue guidance includes
anticipated revenue from countries with final pricing and
reimbursement, and does not currently include potential partial
year contributions from Japan or the U.S.
The Company continues to expect full-year 2018
net cash spend between $230 million and $260 million. The current
cash position, including proceeds from the February 2018 equity
offering and expected Galafold revenues, is sufficient to fund
ongoing Fabry and Pompe program operations into at least 2021.
Potential future business development collaborations, pipeline
expansion, and investment in biologics manufacturing capabilities
could impact the Company’s future capital requirements.
Program Highlights
Migalastat for Fabry
DiseaseAmicus is committed to advancing the highest
quality therapies for all people living with Fabry disease.
Migalastat is an oral precision medicine intended to treat Fabry
disease in patients 16 years or older who have amenable genetic
mutations. Regulatory authorities in the European Union, Japan,
Switzerland, Israel, Canada, Australia, and South Korea have
granted full approval for migalastat under the trade name Galafold.
In the U.S., the FDA accepted the Company’s new drug application
(NDA) for migalastat under priority review with a six-month PDUFA
goal date of August 13, 2018.
For people with non-amenable mutations who are
not eligible for migalastat as an oral precision medicine, the
strategy is to advance next-generation therapies such as a novel
Fabry ERT (ATB101) co-formulated with migalastat or other
innovative technologies that continue to be evaluated.
Global Fabry Updates:
- Pricing and reimbursement secured in 18 countries with first
commercial patients treated in multiple new countries in 2018
- Approvals secured in EU, Australia, Canada, Japan, Israel,
South Korea and Switzerland
- Launch team hired and trained in Japan to execute upcoming
launch
- U.S. leadership, including Mike Keavany, to serve as Senior
Vice President of the U.S. Business, and majority of field team now
in place to support planned U.S. launch
Anticipated Milestones:
- Japanese launch (2Q18)
- U.S. FDA regulatory decision (3Q18)
Advanced and Targeted GAA (AT-GAA, also
known as ATB200/AT2221) for Pompe Disease
AT-GAA 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.
During WORLDSymposium™ in February 2018, Amicus presented
updated positive data from an ongoing global Phase 1/2
clinical study (ATB200-02) to evaluate safety, tolerability,
pharmacokinetics (PK), pharmacodynamics (PD), and functional
outcomes of AT-GAA across ambulatory ERT-switch patients (Cohort
1), non-ambulatory ERT-switch patients (Cohort 2), and ERT-naïve
patients (Cohort 3).
The Company is engaged in ongoing collaborative
discussions with U.S. and EU regulators regarding a
registration-directed study for full approval, manufacturing
activities, and the best and fastest pathway forward for this novel
treatment regimen. Amicus is pursuing formal scientific advice from
the European Medicines Agency (EMA). A scientific advice meeting
with the EMA is scheduled in 2Q18. The Company expects to
provide an update on the results from that meeting by the end of
2Q. In the U.S., ongoing interactions on this program include a
Type C meeting to occur in early 3Q18. Amicus expects to provide an
FDA update in 3Q after receipt of written minutes from this
meeting. The Company continues to believe that the evolving
regulatory path will include a series of further iterative
discussions with regulators as the program advances and as
additional data are collected, including data from ongoing studies,
data from new patients being enrolled and the results of a formal
natural history study of Pompe patients receiving current ERT
standard of care.
Amicus also continues to make progress with
clinical and manufacturing activities to support the needs of the
Pompe community.
Pompe Clinical Activities
- All 19 patients for whom extension study data have been
previously reported continue treatment with AT-GAA in the ongoing
Phase 1/2 ATB200-02 clinical study
- Currently enrolling Cohort 4 (up to 10 additional ambulatory
ERT-switch patients) in ATB200-02 study
- Supportive studies underway including a retrospective study
(POM-002) on the natural history of Pompe disease in up to 100
ERT-treated Pompe patients to help provide context for the
ATB200-02 clinical study results; and a prospective observational
study (POM-003) to assess safety and functional outcomes in
patients currently treated with standard of care ERT, and to serve
as a potential run-in for a registration study
Pompe Manufacturing Activities:
- As previously announced, FDA agreed on comparability between
250L scale and 1000L engineering batches, as well as the testing
strategy for demonstrating comparability between 250L scale and
1000L GMP batches
- GMP production runs of ATB200 drug substance and drug product
completed at 1,000L commercial scale
Anticipated Upcoming Pompe Program
Milestones:
- Full enrollment of additional patients in ATB200-02 clinical
study
- Final demonstration of comparability between 1,000L GMP
material and 250L material
- Pompe regulatory updates (2Q18 and 3Q18)
- Release of 1,000L GMP material for initiation of
registration-directed study (2H18)
- 18-month data from ATB200-02 clinical study (4Q18)
Conference Call and
WebcastAmicus Therapeutics will host a conference call and
audio webcast today, May 8, 2018, at 8:30 a.m. ET to discuss first
quarter 2018 financial results and corporate updates. Interested
participants and investors may access the conference call by
dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international),
conference ID: 6678368.
An audio webcast can also be accessed via the
Investors section of the Amicus Therapeutics corporate website at
http://ir.amicusrx.com/, and will be archived for 30 days. Web
participants are encouraged to go to the website 15 minutes prior
to the start of the call to register, download, and install any
necessary software. A telephonic replay of the call will be
available for seven days beginning at 11:30 a.m. ET on May 8, 2018.
Access numbers for this replay are 855-859-2056 (U.S./Canada) and
404-537-3406 (international); conference ID: 6678368.
Non-GAAP Financial MeasuresIn
addition to the United States generally accepted accounting
principles (GAAP) results, this earnings release contains non-GAAP
financial measures that we believe, when considered together with
the GAAP information, provides useful information to investors that
promotes a more complete understanding of our operating results and
financial position for the current period. Management uses these
non-GAAP financial measures internally for planning, forecasting,
evaluating and allocating resources to the Company's
programs.
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-dedicated
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 lead biologics program in the Amicus pipeline is
AT-GAA, 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,
2017 to be filed on March 1, 2018. 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, IRCVice President, Investor Relations
& Corporate Communicationsspellegrino@amicusrx.com (609)
662-5044
Media:Pure CommunicationsJennifer Paganelli
jpaganelli@purecommunications.com (347) 658-8290
FOLD–G
|
TABLE 1 |
Amicus
Therapeutics, Inc.Consolidated Statements of
Operations(in thousands, except share and per share
amounts) |
|
|
Three Months Ended March 31, |
|
|
2018 |
|
|
|
2017 |
|
Revenue: |
|
|
|
Net product sales |
$ |
16,696 |
|
|
$ |
4,169 |
|
Cost of goods sold |
|
2,615 |
|
|
|
775 |
|
Gross Profit |
|
14,081 |
|
|
|
3,394 |
|
Operating
Expenses: |
|
|
|
Research
and development |
|
40,798 |
|
|
|
30,876 |
|
Selling,
general and administrative |
|
27,396 |
|
|
|
19,132 |
|
Changes
in fair value of contingent consideration payable |
|
1,100 |
|
|
|
4,578 |
|
Depreciation |
|
969 |
|
|
|
823 |
|
Total operating
expenses |
|
70,263 |
|
|
|
55,409 |
|
Loss from
operations |
|
(56,182 |
) |
|
|
(52,015 |
) |
Other income
(expense): |
|
|
|
Interest
income |
|
1,737 |
|
|
|
759 |
|
Interest
expense |
|
(4,488 |
) |
|
|
(4,290 |
) |
Change in
fair value of derivatives |
|
4,861 |
|
|
|
- |
|
Other
income |
|
2,764 |
|
|
|
610 |
|
Loss before income
tax |
|
(51,308 |
) |
|
|
(54,936 |
) |
Income tax benefit
(expense) |
|
1,392 |
|
|
|
(56 |
) |
Net loss
attributable to common stockholders |
$ |
(49,916 |
) |
|
$ |
(54,992 |
) |
Net loss attributable
to common stockholders per common share — basic and
diluted |
$ |
(0.28 |
) |
|
$ |
(0.39 |
) |
Weighted‑average common
shares outstanding — basic and diluted |
|
175,977,700 |
|
|
|
142,770,629 |
|
TABLE 2 |
Amicus
Therapeutics, Inc.Consolidated Balance
Sheets (in thousands, except share and per
share amounts) |
|
|
|
|
|
|
|
|
March 31, |
|
|
December 31, |
|
|
2018 |
|
|
2017 |
Assets |
|
|
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
|
|
Cash and
cash equivalents |
|
$ |
114,322 |
|
|
$ |
49,060 |
|
Investments in marketable securities |
|
|
448,198 |
|
|
|
309,502 |
|
Accounts
receivable |
|
|
10,836 |
|
|
|
9,464 |
|
Inventories |
|
|
8,070 |
|
|
|
4,623 |
|
Prepaid
expenses and other current assets |
|
|
10,607 |
|
|
|
19,316 |
|
Total current
assets |
|
|
592,033 |
|
|
|
391,965 |
|
Investments in marketable securities |
|
|
42,673 |
|
|
|
- |
|
Property
and equipment, less accumulated depreciation of $13,461 and $12,515
at March 31, 2018 and December 31, 2017, respectively |
|
|
8,910 |
|
|
|
9,062 |
|
In-process research & development |
|
|
23,000 |
|
|
|
23,000 |
|
Goodwill |
|
|
197,797 |
|
|
|
197,797 |
|
Other
non-current assets |
|
|
5,592 |
|
|
|
5,200 |
|
Total
Assets |
|
$ |
870,005 |
|
|
$ |
627,024 |
|
|
|
|
|
|
Liabilities and
Stockholders’ Equity |
|
|
|
|
Current
liabilities: |
|
|
|
|
Accounts
payable, accrued expenses, and other current liabilities |
|
$ |
43,678 |
|
|
$ |
53,890 |
|
Deferred
reimbursements |
|
|
7,750 |
|
|
|
7,750 |
|
Derivative liability |
|
|
80,577 |
|
|
|
- |
|
Contingent consideration payable |
|
|
8,700 |
|
|
|
8,400 |
|
Total current
liabilities |
|
|
140,705 |
|
|
|
70,040 |
|
Deferred
reimbursements |
|
|
14,156 |
|
|
|
14,156 |
|
Convertible notes |
|
|
166,768 |
|
|
|
164,167 |
|
Contingent consideration payable |
|
|
17,800 |
|
|
|
17,000 |
|
Deferred
income taxes |
|
|
6,465 |
|
|
|
6,465 |
|
Other
non-current liability |
|
|
2,494 |
|
|
|
2,346 |
|
Total
liabilities |
|
|
348,388 |
|
|
|
274,174 |
|
Commitments and
contingencies |
|
|
|
|
Stockholders’
equity: |
|
|
|
|
Common
stock, $0.01 par value, 250,000,000 shares authorized 187,972,218
and 166,989,790 shares issued and outstanding at March 31, 2018 and
December 31, 2017, respectively |
|
|
1,929 |
|
|
|
1,721 |
|
Additional paid-in capital |
|
|
1,621,479 |
|
|
|
1,400,758 |
|
Accumulated other comprehensive loss: |
|
|
|
|
Foreign
currency translation adjustment |
|
|
(3,847 |
) |
|
|
(1,659 |
) |
Unrealized loss on available-for-sale securities |
|
|
(877 |
) |
|
|
(436 |
) |
Warrants
|
|
|
16,076 |
|
|
|
16,076 |
|
Accumulated deficit |
|
|
(1,113,143 |
) |
|
|
(1,063,610 |
) |
Total stockholders’
equity |
|
|
521,617 |
|
|
|
352,850 |
|
Total
Liabilities and Stockholders’ Equity |
|
$ |
870,005 |
|
|
$ |
627,024 |
|
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