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 third quarter ended September 30, 2019. The Company also
summarized recent program updates and reiterated its full-year 2019
guidance.
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics, Inc. stated, “The third quarter
represented another consecutive period of significant growth and
adoption for Galafold across all geographies, as well as continued
momentum in our Pompe pivotal study and our gene therapy pipeline.
We are on track to meet or exceed each of our key strategic
priorities for the year as we lay the foundation for our long-term
success. Importantly, we have significantly strengthened our
financial outlook with careful management of our expenses and
investments. With confidence in our base business and overall
financial outlook, we are well capitalized to continue to grow our
revenues, advance our pipeline, and move toward self-sustainability
and profitability as we continue to build Amicus into a leading
global rare disease biotechnology company delivering on our mission
for patients and shareholders.”
Corporate Highlights for 3Q19 and Early
4Q19
- Global revenue for Fabry precision medicine Galafold in
the third quarter of 2019 was $48.8 million and continues to track
toward the upwardly revised full-year 2019 revenue guidance of $170
million to $180 million. Third quarter revenue represented
a year-over-year increase of 137% from total revenue of $20.6
million in the third quarter of 2018, and a quarter over-quarter
increase of 11% from total revenue of $44.1 million in the second
quarter of 2019. As of September 30, 2019,
Galafold represented an estimated 30% of global market share of
treated amenable patients. Global compliance and adherence rates
continue to exceed 90%.
- Financial outlook strengthened with current cash runway
now revised to well into 1H 2022 through major portfolio milestones
and global growth.
- Positive Phase 2 clinical data for AT-GAA in Pompe
disease. Amicus presented initial six-month data in
additional ERT-switch patients (Cohort 4) and full 24-month data
from the first three cohorts in Phase 1/2 ATB200-02 clinical study
at World Muscle Society.
- Pompe Pivotal PROPEL study is expected to over-enroll
(~120 Patients) by YE 2019. Given the strong global
interest among the Pompe patient and physician community for
AT-GAA, which has U.S. Breakthrough Therapy designation, this
global study is now expected to enroll ~120 patients by year-end
2019. Pompe manufacturing also continues to advance with PPQ runs
now initiated at WuXi.
- Positive interim Phase 1/2 clinical data for gene
therapy in CLN6 Batten disease. Initial results as well as
additional supportive data at Child Neurology Society showed
AAV-CLN6 gene therapy has the potential to halt the progression of
a devastating disease that causes loss of brain function and is
fatal in childhood.
- Pompe gene therapy clinical candidate declared to move
into IND-enabling studies. Dose-ranging preclinical
studies are currently underway to build off the initial preclinical
results showing robust uptake and glycogen reduction in multiple
tissues, including brain and spinal cord.
- Robust portfolio of gene therapy programs and
technologies provides foundation for future, including two
clinical-stage programs (CLN6 and CLN3), and eight preclinical gene
therapies.
3Q19 Financial Results
- Total revenue in the third quarter 2019 was $48.8 million, a
year-over-year increase of 137% from total revenue of $20.6 million
in the third quarter of 2018, and a quarter over-quarter increase
of 11% from total revenue of $44.1 million in the second quarter of
2019. On a constant currency basis, third quarter
2019 total revenue was $50.3 million, representing operational
revenue growth measured at constant currency exchange rates of
143%, which was offset by a negative currency impact of $1.3
million, or 6%.
- Cash, cash equivalents, and marketable securities totaled
$514.2 million at September 30, 2019, compared to $504.2 million at
December 31, 2018.
- Total GAAP operating expenses of $100.5 million for the third
quarter of 2019 decreased as compared to $172.5 million in the
third quarter of 2018. The decrease is primarily due to an upfront
payment of $100 million for the Celenex asset acquisition in 2018
partially offset by continued investments in the Galafold launch,
Pompe clinical study program, and our gene therapy pipeline.
- Total non-GAAP operating expenses of $89.7 million for the
third quarter of 2019 increased as compared to $63.0 million in the
third quarter of 2018, reflecting continued investments in the
Galafold launch, Pompe clinical study program, and our gene therapy
pipeline. Full reconciliation of GAAP results to the Company’s
non-GAAP adjusted measures for all reporting periods appear in the
tables to this press release.
- Net loss was $61.8 million, or $0.24 per share, compared to a
net loss of $159.2 million, or $0.84 per share, for the third
quarter 2018.
2019 Financial Guidance
Following the success in the first three
quarters of the year, in addition to the strength in global
Galafold launch metrics across all major geographies, Amicus raised
the lower end of the full-year 2019 Galafold revenue guidance from
$160 to $180 million to $170 to $180 million. The Company
anticipates full-year 2019 non-GAAP operating expense of $410
million to $420 million. A reconciliation of the differences
between the non-GAAP expectation and the corresponding GAAP measure
is not available without unreasonable effort due to high
variability, complexity and low visibility as to the items that
would be excluded from the GAAP measure.
Cash, cash equivalents, and marketable
securities totaled $514.2 million at September 30, 2019. Following
a diligent review of current and outer year operating and capital
expense projections, and robust outlook for Galafold revenue,
Amicus now expects to end 2019 with more than $420 million in cash
on hand and has extended the cash runway projection from 2021 to
well into the first half of 2022.
2019 Key Strategic
Priorities
- Nearly double annual worldwide revenue for Galafold with over
1,000 Fabry patients on Galafold by year end.
- Complete enrollment in pivotal Phase 3 PROPEL clinical 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.
Conference Call and
WebcastAmicus Therapeutics will host a conference
call and audio webcast today, November 11, 2019 at 8:30 a.m. ET to
discuss the third quarter 2019 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: 8654236.
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 November 11,
2019. Access numbers for this replay are 855-859-2056 (U.S./Canada)
and 404-537-3406 (international); conference ID: 8654236.
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 Argentina, 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.
Non-GAAP Financial Measures In
addition to financial information prepared in accordance with U.S.
GAAP, this press release also contains adjusted financial measures
that we believe provide investors and management with supplemental
information relating to operating performance and trends that
facilitate comparisons between periods and with respect to
projected information. These adjusted financial measures are
non-GAAP measures and should be considered in addition to, but not
as a substitute for, the information prepared in accordance with
U.S. GAAP. We typically exclude certain GAAP items that management
does not believe affect our basic operations and that do not meet
the GAAP definition of unusual or non-recurring items. Other
companies may define these measures in different ways. Full
reconciliations of GAAP results to the comparable non-GAAP measures
for the reported periods appear in the financial tables section of
this press release. When we provide our expectation for non-GAAP
operating expenses on a forward-looking basis, a reconciliation of
the differences between the non-GAAP expectation and the
corresponding GAAP measure generally is not available without
unreasonable effort due to potentially high variability, complexity
and low visibility as to the items that would be excluded from the
GAAP measure in the relevant future period, such as unusual gains
or losses. The variability of the excluded items may have a
significant, and potentially unpredictable, impact on our future
GAAP results.
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. 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:Sara Pellegrino,
IRCVice President, Investor Relations spellegrino@amicusrx.com
(609) 662-5044
Media:Christopher Byrne Executive Director,
Corporate Communications cbyrne@amicusrx.com (609) 662-2798
FOLD–G
|
TABLE
1 |
|
Amicus Therapeutics, Inc. |
Consolidated Statements of Operations |
(Unaudited) |
(in thousands, except share and per share
amounts) |
|
|
Three Months Ended September 30, |
|
Nine Months Ended September 30, |
|
|
2019 |
|
2018 |
|
2019 |
|
2018 |
Revenue: |
|
|
|
|
|
|
|
Net product sales |
$ |
48,768 |
|
|
$ |
20,596 |
|
|
$ |
126,944 |
|
|
$ |
58,601 |
|
Cost of goods sold |
5,596 |
|
|
4,310 |
|
|
15,018 |
|
|
10,060 |
|
Gross profit |
43,172 |
|
|
16,286 |
|
|
111,926 |
|
|
48,541 |
|
Operating expenses: |
|
|
|
|
|
|
|
Research and development |
58,892 |
|
|
138,227 |
|
|
194,466 |
|
|
213,685 |
|
Selling, general, and administrative |
39,680 |
|
|
31,867 |
|
|
126,561 |
|
|
88,435 |
|
Changes in fair value of contingent consideration payable |
789 |
|
|
1,300 |
|
|
2,652 |
|
|
2,700 |
|
Depreciation and amortization |
1,116 |
|
|
1,073 |
|
|
3,261 |
|
|
3,015 |
|
Total operating expenses |
100,477 |
|
|
172,467 |
|
|
326,940 |
|
|
307,835 |
|
Loss from operations |
(57,305 |
) |
|
(156,181 |
) |
|
(215,014 |
) |
|
(259,294 |
) |
Other income (expense): |
|
|
|
|
|
|
|
Interest income |
2,752 |
|
|
2,721 |
|
|
7,990 |
|
|
7,371 |
|
Interest expense |
(4,026 |
) |
|
(4,715 |
) |
|
(15,105 |
) |
|
(13,763 |
) |
Loss on exchange of convertible notes |
— |
|
|
— |
|
|
(40,624 |
) |
|
— |
|
Change in fair value of derivatives |
— |
|
|
— |
|
|
— |
|
|
(2,739 |
) |
Other expense |
(3,481 |
) |
|
(1,039 |
) |
|
(3,272 |
) |
|
(3,593 |
) |
Loss before income tax |
(62,060 |
) |
|
(159,214 |
) |
|
(266,025 |
) |
|
(272,018 |
) |
Income tax benefit
(expense) |
251 |
|
|
51 |
|
|
(634 |
) |
|
1,104 |
|
Net loss attributable
to common stockholders |
$ |
(61,809 |
) |
|
$ |
(159,163 |
) |
|
$ |
(266,659 |
) |
|
$ |
(270,914 |
) |
Net loss attributable to
common stockholders per common share — basic and diluted |
$ |
(0.24 |
) |
|
$ |
(0.84 |
) |
|
$ |
(1.13 |
) |
|
$ |
(1.47 |
) |
Weighted-average common shares
outstanding — basic and diluted |
254,674,422 |
|
|
189,162,841 |
|
|
235,527,540 |
|
|
184,606,790 |
|
TABLE
2 |
|
Amicus Therapeutics, Inc. |
Consolidated Balance Sheets |
(Unaudited) |
(in thousands, except share and per share
amounts) |
|
|
September 30, 2019 |
|
December 31, 2018 |
Assets |
|
|
|
Current assets: |
|
|
|
Cash and cash equivalents |
$ |
166,319 |
|
|
$ |
79,749 |
|
Investments in marketable securities |
347,875 |
|
|
424,403 |
|
Accounts receivable |
33,731 |
|
|
21,962 |
|
Inventories |
9,154 |
|
|
8,390 |
|
Prepaid expenses and other current assets |
19,578 |
|
|
16,592 |
|
Total current assets |
576,657 |
|
|
551,096 |
|
Operating lease right-of-use assets, less accumulated amortization
of $2,420 and $0 at September 30, 2019 and December 31, 2018,
respectively |
35,814 |
|
|
— |
|
Property and equipment, less accumulated depreciation of $17,907
and $15,671 at September 30, 2019 and December 31, 2018,
respectively |
34,673 |
|
|
11,375 |
|
In-process research & development |
23,000 |
|
|
23,000 |
|
Goodwill |
197,797 |
|
|
197,797 |
|
Other non-current assets |
14,351 |
|
|
6,683 |
|
Total
Assets |
$ |
882,292 |
|
|
$ |
789,951 |
|
Liabilities and
Stockholders’ Equity |
|
|
|
Current liabilities: |
|
|
|
Accounts payable, accrued expenses, and other current
liabilities |
$ |
81,475 |
|
|
$ |
80,625 |
|
Deferred reimbursements |
5,250 |
|
|
5,500 |
|
Operating lease liabilities |
6,356 |
|
|
— |
|
Total current liabilities |
93,081 |
|
|
86,125 |
|
Deferred reimbursements |
8,906 |
|
|
10,156 |
|
Convertible notes |
2,096 |
|
|
175,006 |
|
Senior secured term loan |
147,164 |
|
|
146,734 |
|
Contingent consideration payable |
22,036 |
|
|
19,700 |
|
Deferred income taxes |
6,465 |
|
|
6,465 |
|
Operating lease liabilities |
49,686 |
|
|
— |
|
Other non-current liabilities |
4,591 |
|
|
2,853 |
|
Total liabilities |
334,025 |
|
|
447,039 |
|
Commitments and
contingencies |
|
|
|
Stockholders’ equity: |
|
|
|
Common stock, $0.01 par value,
500,000,000 shares authorized, 254,772,163 and 189,383,924 shares
issued and outstanding at September 30, 2019 and December 31, 2018,
respectively |
2,591 |
|
|
1,942 |
|
Additional paid-in
capital |
2,210,890 |
|
|
1,740,061 |
|
Accumulated other
comprehensive loss: |
|
|
|
Foreign currency translation adjustment |
1,156 |
|
|
495 |
|
Unrealized gain (loss) on available-for-sale securities |
124 |
|
|
(427 |
) |
Warrants |
12,387 |
|
|
13,063 |
|
Accumulated deficit |
(1,678,881 |
) |
|
(1,412,222 |
) |
Total stockholders’
equity |
548,267 |
|
|
342,912 |
|
Total Liabilities and
Stockholders’ Equity |
$ |
882,292 |
|
|
$ |
789,951 |
|
TABLE
3 |
|
Amicus Therapeutics, Inc. |
Reconciliation of Non-GAAP Financial Measures |
(in thousands) |
|
|
|
Three Months Ended September 30, |
|
|
Nine Months Ended September
30, |
|
|
2019 |
|
2018 |
|
|
2019 |
|
2018 |
|
Total operating expenses - as reported GAAP |
|
$ |
100,477 |
|
$ |
172,467 |
|
|
$ |
326,940 |
|
$ |
307,835 |
|
Research and development: |
|
|
|
|
|
|
|
|
|
|
Share-based compensation |
|
|
3,106 |
|
|
2,905 |
|
|
|
12,090 |
|
|
8,603 |
|
Research and development asset acquisition expense |
|
|
- |
|
|
100,000 |
|
|
|
- |
|
|
100,000 |
|
Selling, general and administrative: |
|
|
|
|
|
|
|
|
|
|
Share-based compensation |
|
|
5,737 |
|
|
4,149 |
|
|
|
19,432 |
|
|
12,270 |
|
Changes in fair value of contingent consideration
payable |
|
|
789 |
|
|
1,300 |
|
|
|
2,652 |
|
|
2,700 |
|
Depreciation and amortization |
|
|
1,116 |
|
|
1,073 |
|
|
|
3,261 |
|
|
3,015 |
|
Total operating expense adjustments to reported
GAAP |
|
|
10,748 |
|
|
109,427 |
|
|
|
37,435 |
|
|
126,588 |
|
Total operating expenses
- as adjusted Non-GAAP |
|
$ |
89,729 |
|
$ |
63,040 |
|
|
$ |
289,505 |
|
$ |
181,247 |
|
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