Continued Strong Galafold Launch Trends
in Early 2019 - On Track to Achieve FY19 Revenue Guidance of
$160M-$180M
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 full year ended December 31, 2018. The Company also summarized
recent program updates and reiterated its full-year 2019 guidance.
Corporate Highlights for Full-Year 2018
and Year-to-Date 2019
- More than doubled global revenue for Galafold
(migalastat) in 2018. As previously announced, revenue
grew from $36.9 million in full-year 2017 to $91.2 million in
full-year 2018, exceeding the high end of the full-year 2018
guidance range of $80 million to $90 million.
- Strong Launch Trends Continue in Early 2019.
On track to achieve full-year 2019 revenue guidance of $160M-$180M
and 1,000+ patients on Galafold by year-end.
- Updated AT-GAA clinical data in Pompe disease presented
at WORLDSymposium™ in February 2019. As previously
announced, consistent and durable responses continued across key
measures of safety, functional outcomes and biomarkers for both
ERT-naïve and ERT-switch patients treated with AT-GAA for up to 24
months in the ongoing Phase 1/2 clinical study.
- AT-GAA received Breakthrough Therapy Designation (BTD)
in late onset Pompe disease. The BTD was based on clinical
efficacy results from the ongoing ATB200-02 Phase 1/2 clinical
study, and further strengthens the Company’s conviction in the
potential for AT-GAA to become the next standard of
care.
- Enrollment momentum in ongoing pivotal PROPEL study in
Pompe disease and Phase 1/2 study in CLN3 Batten disease.
The PROPEL study is on track to achieve full enrollment by year-end
2019. The first patient remains in the CLN3 Batten disease study
with no serious adverse events after more than two months following
a single administration of AAV9-CLN3 gene therapy. Further
patient dosing is expected in the coming months.
- Robust gene therapy pipeline continues to advance
toward important data milestones. Additional two-year data
from Phase 1/2 study in CLN6 Batten disease expected mid-year.
Preclinical proof-of-concept for Fabry and Pompe gene therapies
anticipated throughout 2019.
- New Global Research and Gene Therapy
Center of Excellence in Philadelphia. This new
headquarters for the global Amicus science organization and the
gene therapy leadership team advances the Company’s commitment to
world-class science.
- Strong financial position to continue executing the
Galafold launch and advance development programs. The
current cash position of approximately $504.2 million at December
31, 2018 is expected to fund ongoing operations into at least
mid-2021.
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics, Inc. stated, “Over the course of
last year and into early 2019 we have made great strides in our
continued evolution as a leading global rare disease biotechnology
company. We are on track and well-capitalized to achieve all of our
2019 key strategic priorities including our global Fabry launch,
Pompe late-stage development program, and gene therapy pipeline.
With a very successful, now global, commercial precision medicine
product in Fabry disease, a late stage program with BTD in late
onset Pompe disease and 14 Gene Therapy programs for rare diseases
in development, including two in the clinic, I believe that we are
now, more than ever before, strongly positioned to achieve our
vision. We endeavor to deliver groundbreaking new medicines and
hopefully one day cures for people living with rare metabolic
diseases and to be one of a handful of leading companies in the
world in this field. Achieving this vision will create enormous
value for people with these life-threatening conditions as well as
our shareholders.”
Full-Year 2018 Financial
Results
- Total revenue in the full-year 2018 was $91.2 million, an
increase from total revenue of $36.9 million in the full-year
2017.
- Cash, cash equivalents, and marketable securities totaled
$504.2 million at December 31, 2018 compared to $358.6 million at
December 31, 2017.
- Total operating expenses decreased to $405.6 million for the
full-year 2018 compared to $472.7 million in the full-year 2017.
Operating expenses reflecting increased investments in the Galafold
launch, Pompe program, and gene therapy pipeline.
- Net cash spend was $189.3 million for the full-year 2018, which
was below full-year 2018 net cash spend guidance of $200 million to
$225 million and reflects careful expense management.
- Net loss was $349.0 million, or $1.88 per share, compared to a
net loss of $284.0 million, or $1.85 per share, for the full-year
2017.
2019 Key Strategic
Priorities
- Nearly double again, annual revenue for Galafold (FY19 guidance
of $160M-$180M in worldwide revenue) with 1,000+ Fabry patients on
Galafold by year end.
- Complete enrollment in pivotal study in Pompe disease and
report additional Phase 2 data.
- Report additional two-year results from Phase 1/2 clinical
study in CLN6 Batten disease and complete enrollment in ongoing
CLN3 Batten disease Phase 1/2 study.
- Establish preclinical proof of concept for Fabry and Pompe gene
therapies.
- Maintain a strong financial position.
2019 Financial Guidance
Amicus recorded $91.2 million in full-year 2018
revenue from commercial sales and reimbursed expanded access
programs for Galafold. For the full-year 2019 the Company
anticipates total Galafold revenue of $160 million to $180 million.
Growth in 2018 was largely driven by EU and other countries outside
the U.S. and Japan. Growth in 2019 is expected to be driven by
continued growth in EU markets, further geographic expansion, and
further success from the first full year of launch in the U.S. and
Japan.
Cash, cash equivalents, and marketable
securities totaled $504.2 million at December 31, 2018. The Company
expects to end 2019 with approximately $300 million in cash on
hand. The current cash position is anticipated to fund ongoing
operations into at least mid- 2021.
Anticipated 2019 Milestones by
Program
Amicus previously announced full-year 2018
program updates as well as anticipated 2019 program milestones in
early January 2019. All anticipated milestones remain on track as
follows:
Galafold (migalastat) Oral Precision
Medicine for Fabry Disease
- On track to meet full-year 2019 revenue guidance range of $160
million to $180 million.
AT-GAA for Pompe Disease
- Initial 6-month data in additional ERT-switch patients (Cohort
4) in Phase 1/2 ATB200-02 clinical study.
- Retrospective natural history study data in approximately 100
ERT-treated Pompe patients.
- Additional supportive studies, including an open-label study in
pediatric patients.
- Full enrollment in Phase 3 PROPEL study.
- Advance agreed upon CMC requirements to support BLA.
Gene Therapy Pipeline:
- Additional two-year data from CLN6 Batten disease Phase 1/2
study.
- Full enrollment of ongoing CLN3 Batten disease Phase 1/2
study.
- Preclinical data for next-generation gene therapies for Fabry,
Pompe and CDD.
- Preclinical work across additional neurologic LSDs.
Conference Call and
WebcastAmicus Therapeutics will host a conference call and
audio webcast today, February 28, 2019 at 8:30 a.m. ET to discuss
the full year 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: 6983148.
A live 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 register on the website 15 minutes
prior to the start of the call. A replay of the call will be
available for seven days beginning at 11:30 a.m. ET on February 28,
2019. Access numbers for this replay are 855-859-2056 (U.S./Canada)
and 404-537-3406 (international); conference ID: 6983148.
About Galafold
Galafold® (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 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 Australia, Canada, European Union, Israel, Japan,
South Korea, Switzerland and the U.S.
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
reactions reported with Galafold (≥10%) were headache,
nasopharyngitis, urinary tract infection, nausea and pyrexia.
USE IN SPECIFIC POPULATIONS There 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 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. 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 on Twitter and
LinkedIn.
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, 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. 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,
2018 filed today. 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
and Corporate Communicationsspellegrino@amicusrx.com (609)
662-5044
Media:Amicus TherapeuticsMarco Winkler
Director, Corporate Communications mwinkler@amicusrx.com(609)
662-2798
FOLD–G
TABLE
1
Amicus
Therapeutics, Inc.Consolidated Statements of
Operations(in thousands, except share and per
share amounts)
|
Years Ended December 31, |
|
2018 |
|
2017 |
|
2016 |
Revenue: |
|
|
|
|
|
Net product sales |
$ |
91,245 |
|
|
$ |
36,930 |
|
|
$ |
4,958 |
|
Cost of goods sold |
14,404 |
|
|
6,236 |
|
|
833 |
|
Gross profit |
76,841 |
|
|
30,694 |
|
|
4,125 |
|
Operating
expenses: |
|
|
|
|
|
Research
and development |
270,902 |
|
|
149,310 |
|
|
104,793 |
|
Selling,
general and administrative |
127,200 |
|
|
88,671 |
|
|
71,151 |
|
Changes
in fair value of contingent consideration payable |
3,300 |
|
|
(234,322 |
) |
|
6,760 |
|
Loss on
impairment of assets |
— |
|
|
465,427 |
|
|
— |
|
Restructuring charges |
— |
|
|
— |
|
|
69 |
|
Depreciation |
4,216 |
|
|
3,593 |
|
|
3,242 |
|
Total operating
expenses |
405,618 |
|
|
472,679 |
|
|
186,015 |
|
Loss from
operations |
(328,777 |
) |
|
(441,985 |
) |
|
(181,890 |
) |
Other income
(expenses): |
|
|
|
|
|
Interest
income |
10,461 |
|
|
4,096 |
|
|
1,602 |
|
Interest
expense |
(22,402 |
) |
|
(17,240 |
) |
|
(5,398 |
) |
Change in
fair value of derivatives |
(2,739 |
) |
|
— |
|
|
— |
|
Loss on
extinguishment of debt |
— |
|
|
— |
|
|
(13,302 |
) |
Other
income (expense) |
(5,632 |
) |
|
6,008 |
|
|
(4,793 |
) |
Loss before income
tax |
(349,089 |
) |
|
(449,121 |
) |
|
(203,781 |
) |
Income tax benefit |
94 |
|
|
165,119 |
|
|
3,739 |
|
Net loss
attributable to common stockholders |
$ |
(348,995 |
) |
|
$ |
(284,002 |
) |
|
$ |
(200,042 |
) |
Net loss attributable
to common stockholders per common share — basic and
diluted |
$ |
(1.88 |
) |
|
$ |
(1.85 |
) |
|
$ |
(1.49 |
) |
Weighted-average common
shares outstanding — basic and diluted |
185,790,021 |
|
|
153,355,144 |
|
|
134,401,588 |
|
TABLE
2
Amicus
Therapeutics, Inc.Consolidated Balance
Sheets(in thousands, except share and per share
amounts)
|
December 31, |
|
2018 |
|
2017 |
Assets |
|
|
|
Current assets: |
|
|
|
Cash and
cash equivalents |
$ |
79,749 |
|
|
$ |
49,060 |
|
Investments in marketable securities |
424,403 |
|
|
309,502 |
|
Accounts
receivable |
21,962 |
|
|
9,464 |
|
Inventories |
8,390 |
|
|
4,623 |
|
Prepaid
expenses and other current assets |
16,592 |
|
|
19,316 |
|
Total current
assets |
551,096 |
|
|
391,965 |
|
Property
and equipment, less accumulated depreciation of $15,671 and $12,515
at December 31, 2018 and 2017, respectively |
11,375 |
|
|
9,062 |
|
In-process research & development |
23,000 |
|
|
23,000 |
|
Goodwill |
197,797 |
|
|
197,797 |
|
Other
non-current assets |
6,683 |
|
|
5,200 |
|
Total
Assets |
$ |
789,951 |
|
|
$ |
627,024 |
|
Liabilities and
Stockholders' Equity |
|
|
|
Current
liabilities: |
|
|
|
Accounts
payable, accrued expenses, and other current liabilities |
$ |
80,625 |
|
|
$ |
53,890 |
|
Deferred
reimbursements |
5,500 |
|
|
7,750 |
|
Contingent consideration payable |
— |
|
|
8,400 |
|
Total
current liabilities |
86,125 |
|
|
70,040 |
|
Deferred
reimbursements |
10,156 |
|
|
14,156 |
|
Convertible notes |
175,006 |
|
|
164,167 |
|
Senior
secured term loan |
146,734 |
|
|
— |
|
Contingent consideration payable |
19,700 |
|
|
17,000 |
|
Deferred
income taxes |
6,465 |
|
|
6,465 |
|
Other
non-current liabilities |
2,853 |
|
|
2,346 |
|
Total Liabilities |
447,039 |
|
|
274,174 |
|
Commitments and
contingencies |
|
|
|
Stockholders'
equity: |
|
|
|
Common stock, $.01 par
value, 500,000,000 shares authorized, 189,383,924 shares issued and
outstanding at December 31, 2018 Common stock, $.01 par value,
250,000,000 shares authorized, 166,989,790 shares issued and
outstanding at December 31, 2017 |
1,942 |
|
|
1,721 |
|
Additional paid-in
capital |
1,740,061 |
|
|
1,400,758 |
|
Accumulated other
comprehensive loss: |
|
|
|
Foreign
currency translation adjustment |
495 |
|
|
(1,659 |
) |
Unrealized loss on available-for securities |
(427 |
) |
|
(436 |
) |
Warrants |
13,063 |
|
|
16,076 |
|
Accumulated
deficit |
(1,412,222 |
) |
|
(1,063,610 |
) |
Total stockholders'
equity |
342,912 |
|
|
352,850 |
|
Total
Liabilities and Stockholders' Equity |
$ |
789,951 |
|
|
$ |
627,024 |
|
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