2Q18 Net Product Sales of $21.3M Driven
by Galafold®
(Migalastat) Expansion
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 second quarter ended June 30, 2018. The Company also summarized
recent program updates, raised its full-year 2018 revenue guidance
and updated its net cash spend guidance for the year.
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics, Inc. stated, “In the first half of
2018, we have made tremendous progress advancing Galafold,
expanding access to patients with amenable mutations across the
world. Given the continued strong momentum of the Galafold launch,
the early commercial launch in Japan, and the pending accelerated
approval in the U.S., we are raising our 2018 revenue guidance to
$80 million to $90 million. We have also continued to advance our
Pompe clinical, manufacturing and regulatory activities and look
forward to providing an update later this quarter on the pivotal
study design and best and fastest regulatory path in the United
States for this important program. Additionally, we are also
extraordinarily focused on assembling a portfolio of technologies,
programs and partnerships in the gene therapy space. Our goal
beginning in the second half of this year is a bold one: to build
one of the most robust gene therapy pipelines in the field of rare,
metabolic disorders. 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.”
Second Quarter 2018 Financial Results
and Full-Year 2018 Financial Guidance
- Total revenue in the second quarter 2018 was $21.3 million, a
year-over-year increase of 198% from total revenue of $7.2 million
in the second quarter of 2017.
- Cash, cash equivalents, and marketable securities totaled
$552.8 million at June 30, 2018, compared to $358.6 million at
December 31, 2017.
- Total operating expenses increased to $65.1 million for the
second quarter 2018 compared to $53.2 million in the second quarter
2017, reflecting an increased investment in Pompe clinical and
manufacturing activities as well as Galafold commercial launch and
launch preparations.
- Net cash spend was $51.0 million for the second quarter 2018.
Net loss was $61.8 million, or $0.33 per share, for the second
quarter 2018 compared to a net loss of $48.1 million, or $0.34 per
share, for the second quarter 2017.
“The second quarter of 2018 marked another
period of continued growth for Galafold,” said Bradley L. Campbell,
President and COO of Amicus Therapeutics. “We are raising our
full-year 2018 Galafold global revenue guidance given the current
and anticipated increase in patient and physician adoption in our
existing markets, the ongoing launch in Japan, and our anticipated
PDUFA date and launch in the U.S. this quarter. And we’re
pleased to announce that we’ve hired 100% of our US launch team
comprised of passionate professionals with significant rare disease
experience.”
2018 Financial Guidance
For the full-year 2018 the Company is increasing its total
Galafold revenue guidance to $80 million to $90 million from the
previous range of $75 million to $85 million. This reflects global
revenue from all expected 2018 commercial markets.
Based on the increase in expected 2018 revenue
and operating expenses that are trending favorably to budget, the
Company is updating its full-year 2018 net cash spend to $220 to
$250 million from the previous range of $230 to $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, the Company’s first therapy, is an oral precision
medicine. Regulatory authorities in Australia, Canada, EU, Israel,
Japan, South Korea and Switzerland have granted full approval for
migalastat under the trade name Galafold to treat Fabry disease in
patients 16 years or older who have amenable genetic mutations. In
the U.S., the FDA accepted the Company’s new drug application (NDA)
for migalastat under Subpart H 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 19 countries with first
commercial patients treated in multiple new countries in 2018
- Approvals secured in Australia, Canada, EU, Israel, Japan,
South Korea and Switzerland
- Commercial launch initiated in Japan during the second
quarter
- U.S. leadership and field team now in place to support planned
U.S. launch
Anticipated Milestones:
- U.S. FDA regulatory decision (3Q18)
AT-GAA 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 to
stabilize ATB200 while in the circulation to deliver active
therapeutic enzyme.
The Company is engaged in collaborative
discussions with U.S. and EU regulators regarding a
number of key topics including a registration-directed study for
full approval, manufacturing activities, and the best and fastest
pathway forward for this novel treatment regimen. The Company
believes that the evolving regulatory paths in both the
U.S. and EU will include a series of iterative discussions
with regulators as the program advances and as additional data are
collected, including data from existing patients in ongoing
studies, data from new patients being enrolled into ongoing
studies, and the results of a formal retrospective natural history
study of Pompe patients receiving current standard of care ERT.
Amicus expects to provide an FDA update in the third quarter 2018
after receipt of written minutes from a scheduled Type C
meeting.
Manufacturing activities to support the needs of
the Pompe community are also ongoing.
Pompe Manufacturing Updates:
- Release of 1,000L GMP material for use in the planned pivotal
study.
- Feedback from German regulatory authorities (BfArM) indicating
general agreement with the manufacturing strategy for ATB200,
including the strategy to demonstrate comparability of drug
substance and drug product between the 1,000 liter scale and the
250 liter scale.
Anticipated Pompe Program Milestones in
2H18:
- Pompe US regulatory update (3Q18).
- Completion of a retrospective natural history study in ~100
ERT-treated Pompe patients.
- 18-month data from ATB200-02 clinical study to be presented at
the 23rd International Congress of the World Muscle Society
(4Q18).
- Commence pivotal study in 2H18.
Conference Call and
WebcastAmicus Therapeutics will host a conference call and
audio webcast today, August 7, 2018, at 8:30 a.m. ET to discuss the
second 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: 5887047.
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 August 7,
2018. Access numbers for this replay are 855-859-2056 (U.S./Canada)
and 404-537-3406 (international); conference ID: 5887047.
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-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
Galafold, an oral precision medicine for people living with Fabry
disease who have amenable genetic mutations. The lead biologics
program in the Amicus pipeline is AT-GAA, a novel, late-stage
treatment for Pompe disease. Amicus 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 as well as our Quarterly Report on Form 10-Q for the quarter
ended June 30, 2018 to be filed August 7, 2018 with the Securities
and Exchange Commission. 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
TherapeuticsAndrew FaughnanAssociate Director, Investor
Relationsafaughnan@amicusrx.com (609) 662-3809
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 June 30, |
|
Six Months Ended June 30, |
|
2018 |
|
2017 |
|
2018 |
|
2017 |
Revenue: |
|
|
|
|
|
|
|
Net product sales |
$ |
21,309 |
|
|
$ |
7,158 |
|
|
$ |
38,005 |
|
|
$ |
11,327 |
|
Cost of goods sold |
3,135 |
|
|
1,061 |
|
|
5,750 |
|
|
1,836 |
|
Gross Profit |
18,174 |
|
|
6,097 |
|
|
32,255 |
|
|
9,491 |
|
Operating
Expenses: |
|
|
|
|
|
|
|
Research
and development |
34,660 |
|
|
31,985 |
|
|
75,458 |
|
|
62,861 |
|
Selling,
general and administrative |
29,172 |
|
|
19,311 |
|
|
56,568 |
|
|
38,443 |
|
Changes
in fair value of contingent consideration payable |
300 |
|
|
1,050 |
|
|
1,400 |
|
|
5,628 |
|
Depreciation |
973 |
|
|
812 |
|
|
1,942 |
|
|
1,636 |
|
Total operating
expenses |
65,105 |
|
|
53,158 |
|
|
135,368 |
|
|
108,568 |
|
Loss from
operations |
(46,931 |
) |
|
(47,061 |
) |
|
(103,113 |
) |
|
(99,077 |
) |
Other income
(expense): |
|
|
|
|
|
|
|
Interest
income |
2,913 |
|
|
753 |
|
|
4,650 |
|
|
1,512 |
|
Interest
expense |
(4,560 |
) |
|
(4,179 |
) |
|
(9,048 |
) |
|
(8,469 |
) |
Change in
fair value of derivatives |
(7,600 |
) |
|
— |
|
|
(2,739 |
) |
|
— |
|
Other
(expense) income |
(5,316 |
) |
|
2,400 |
|
|
(2,554 |
) |
|
3,010 |
|
Loss before income
tax |
(61,494 |
) |
|
(48,087 |
) |
|
(112,804 |
) |
|
(103,024 |
) |
Income tax benefit
(expense) |
(339 |
) |
|
(49 |
) |
|
1,053 |
|
|
(105 |
) |
Net loss attributable
to common stockholders |
$ |
(61,833 |
) |
|
$ |
(48,136 |
) |
|
$ |
(111,751 |
) |
|
$ |
(103,129 |
) |
Net loss attributable
to common stockholders per common share — basic and
diluted |
$ |
(0.33 |
) |
|
$ |
(0.34 |
) |
|
$ |
(0.61 |
) |
|
$ |
(0.72 |
) |
Weighted-average common
shares outstanding — basic and diluted |
188,621,423 |
|
|
143,000,718 |
|
|
182,303,128 |
|
|
142,886,614 |
|
TABLE 2 |
Amicus
Therapeutics, Inc. Consolidated Balance
Sheets (in thousands, except share and per
share amounts) |
|
|
|
|
|
June 30, |
|
December 31, |
|
2018 |
|
2017 |
Assets |
|
|
|
Current assets: |
|
|
|
Cash and
cash equivalents |
$ |
73,311 |
|
|
$ |
49,060 |
|
Investments in marketable securities |
465,641 |
|
|
309,502 |
|
Accounts
receivable |
15,077 |
|
|
9,464 |
|
Inventories |
7,769 |
|
|
4,623 |
|
Prepaid
expenses and other current assets |
14,432 |
|
|
19,316 |
|
Total current
assets |
576,230 |
|
|
391,965 |
|
Investments in marketable securities |
13,836 |
|
|
|
— |
|
Property
and equipment, less accumulated depreciation of $14,415 and $12,515
at June 30, 2018 and December 31, 2017, respectively |
9,111 |
|
|
9,062 |
|
In-process research & development |
23,000 |
|
|
23,000 |
|
Goodwill |
197,797 |
|
|
197,797 |
|
Other
non-current assets |
5,915 |
|
|
5,200 |
|
Total
Assets |
$ |
825,889 |
|
|
$ |
627,024 |
|
Liabilities and
Stockholders’ Equity |
|
|
|
Current
liabilities: |
|
|
|
Accounts
payable, accrued expenses, and other current liabilities |
$ |
41,613 |
|
|
$ |
53,890 |
|
Deferred
reimbursements |
2,750 |
|
|
7,750 |
|
Contingent consideration payable |
8,600 |
|
|
8,400 |
|
Total current
liabilities |
52,963 |
|
|
70,040 |
|
Deferred
reimbursements |
14,156 |
|
|
14,156 |
|
Convertible notes |
169,440 |
|
|
164,167 |
|
Contingent consideration payable |
18,200 |
|
|
17,000 |
|
Deferred
income taxes |
6,465 |
|
|
6,465 |
|
Other
non-current liability |
2,770 |
|
|
2,346 |
|
Total liabilities |
263,994 |
|
|
274,174 |
|
Commitments and
contingencies |
|
|
|
Stockholders’
equity: |
|
|
|
Common stock, $0.01 par
value, 500,000,000 and 250,000,000 shares authorized, 189,053,214
and 166,989,790 shares issued and outstanding at June 30, 2018 and
December 31, 2017, respectively |
1,939 |
|
|
1,721 |
|
Additional paid-in
capital |
1,723,865 |
|
|
1,400,758 |
|
Accumulated other
comprehensive loss: |
|
|
|
Foreign
currency translation adjustment |
(1,539 |
) |
|
(1,659 |
) |
Unrealized gain on available-for-sale securities |
(455 |
) |
|
(436 |
) |
Warrants |
13,063 |
|
|
16,076 |
|
Accumulated
deficit |
(1,174,978 |
) |
|
(1,063,610 |
) |
Total stockholders’
equity |
561,895 |
|
|
352,850 |
|
Total
Liabilities and Stockholders’ Equity |
$ |
825,889 |
|
|
$ |
627,024 |
|
Amicus Therapeutics (NASDAQ:FOLD)
Historical Stock Chart
From Apr 2024 to May 2024
Amicus Therapeutics (NASDAQ:FOLD)
Historical Stock Chart
From May 2023 to May 2024