Amicus Therapeutics (Nasdaq: FOLD), a global, patient-dedicated
biotechnology company focused on discovering, developing and
delivering novel medicines for rare diseases, today announced
financial results for the first quarter ended March 31, 2020. The
Company also summarized recent program updates and reiterated its
full-year 2020 guidance.
Revenue
- Global revenue for Galafold® (migalastat) in the first
quarter of 2020 was $60.5M and continues to track toward full-year
2020 revenue guidance of $250 million to $260 million.
First quarter revenue represented a year-over-year increase of 78%
from total revenue of $34.0 million in the first quarter of
2019. On a constant currency basis, first quarter
2020 total revenue was $61.4 million, representing operational
revenue growth measured at constant currency exchange rates of 81%,
which was offset by a negative currency impact of $0.9 million, or
3%.
- First quarter revenue represents the continued
performance across the global business, including new patient
starts from switch and naïve patients throughout the quarter in all
major regions, including those hardest hit by COVID-19.
Performance driven largely by strong patient demand with a minimal
contribution from ordering patterns as healthcare organizations in
some countries adjusted to the impact of the COVID-19 virus. Global
compliance and adherence rates continue to exceed 90%.
Supply Chain
- Global supply chain for Galafold
maintains continuous supply during the COVID-19
pandemic. With sufficient inventory on hand and product
effectively moving down the supply chain into individual countries,
Amicus continues to fulfill the needs of Fabry patients without
interruption. The company expects to be able to continuously supply
this oral precision medicine in all global geographies for any and
all people living with Fabry disease who have an amenable variant
throughout the current COVID-19 pandemic and beyond.
- Manufacturing and supply of AT-GAA remain intact
globally. Amicus maintains focus on providing a continuous
supply of AT-GAA to all patients and continues to coordinate drug
delivery per a site-by-site basis in order to address the needs of
each patient. The Company has a high degree of confidence that
there is ample supply of AT-GAA to support all ongoing clinical
studies in the Pompe program, including the Phase 3 PROPEL study,
which continues to track towards completing on schedule.
Research and Development
- Global Phase 3 PROPEL clinical study of AT-GAA in
late-onset Pompe disease remains on track for top line data in
1H21. To date, 97% out of the 2,250 planned infusions for
the ongoing PROPEL study have been completed on schedule. Process
performance qualification (PPQ) runs have been successfully
completed for the drug substance with key strategic partner, WuXi
Biologics, and will serve as the foundation for the Chemistry,
Manufacturing, and Control (CMC) module for a biologics license
application (BLA) submission. The Company plans to initiate a
rolling BLA for AT-GAA in the second half of 2020, completing final
submission in the first half of 2021.
- Expanded Access Program for infantile-onset Pompe
patients underway. Amicus has initiated an expanded access
program for its investigational medicine AT-GAA for young children
living with infantile-onset Pompe disease (IOPD).
- Plan to advance regulatory discussions to finalize
clinical and regulatory path in both CLN6 and CLN3 this
year. Additional data in the CLN6 Phase 1/2 study, as well
as, initial CLN3 Phase 1/2 data expected later this year.
- The U.S. Food and Drug Administration (FDA) has granted
Fast Track Designation to the CLN3 Batten disease gene therapy,
AT-GTX-502, for the treatment of pediatric patients less than 18
years of age. The Fast Track program facilitates the
development and accelerates the review of new drugs for serious
conditions, which have the potential to address unmet medical
needs. A drug development program with Fast Track designation is
afforded greater access to the FDA for the purpose of expediting
the drug's development, review and potential approval.
- Additional preclinical gene therapy data expected
across multiple programs in industry leading gene therapy
portfolio. As previously announced, one oral presentation
and two posters accepted at the American Society of Gene & Cell
Therapy 23rd Annual Meeting being held virtually on May 12–15.
Financial Strength
- Cash runway now extended to well into 2H2022.
Amicus continues to carefully manage expenses and investments,
while executing on the Galafold launch and advancing development
programs. Extension of the cash runway will be achieved
through continued careful expense management, prioritization of
very early stage research programs and more measured capital
expenditures.
- First quarter non-GAAP operating expense on track for
full-year guidance. First quarter operating expenses
driven by the timing of charges related to payments of the process
performance qualification runs of AT-GAA and initiation of the tech
transfer to our gene therapy contract manufacturing partners.
Non-GAAP operating expense to decline throughout 2020 on path to
achieving the 2020 non-GAAP operating expense guidance of $410
million to $420 million.
2020 Key Strategic
Priorities
- Achieve $250 million to $260 million of global product revenue
for Galafold
- Complete Pompe Phase 3 PROPEL study, enroll pediatric studies
and advance manufacturing to support 2021 BLA and MAA
- Advance clinical development, manufacturing and regulatory
discussions for CLN6 and CLN3 Batten programs
- Progress Pompe gene therapy towards Investigational New Drug
(IND) application and disclose up to two additional IND
candidates
- Maintain strong financial position
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics, Inc. stated, “During the first
quarter, we have made tremendous progress advancing our mission for
patients. Following success in the first quarter, we are on track
and well-capitalized to achieve our 2020 key strategic priorities
including our global Fabry launch, Pompe late-stage development
program, and advancing our industry-leading gene therapy pipeline.
In addition, we have taken steps to prioritize our spend and now
see our cash runway lasting well into the second half of 2022.
Through these efforts, we remain strongly positioned to achieve our
vision of delivering groundbreaking new medicines and hopefully,
one day, cures for people living with rare diseases.”
First Quarter 2020 Financial
Results
- Total revenue in the first quarter 2020 was $60.5 million, a
year-over-year increase of 78% from total revenue of $34.0 million
in the first quarter of 2019. On a constant currency basis, first
quarter 2020 total revenue was $61.4 million, representing
operational revenue growth measured at constant currency exchange
rates of 81%, which was offset by a negative currency impact of
$0.9 million, or 3%.
- Cash, cash equivalents, and marketable securities totaled
$338.9 million at March 31, 2020, compared to $452.7 million at
December 31, 2019.
- Total GAAP operating expenses of $132.0 million for the first
quarter 2020 increased as compared to $111.3 million for the first
quarter 2019, reflecting continued investments in the Galafold
launch, Pompe program, and gene therapy pipeline.
- Total non-GAAP operating expenses of $116.7 million for the
first quarter of 2020 increased as compared to $96.2 million in the
first quarter of 2019, reflecting continued investments in the
Galafold launch, Pompe clinical study program, and gene therapy
pipeline. First quarter non-GAAP operating expense on track for
full-year guidance. Non-GAAP operating expense to decline
throughout 2020 on path to achieving the 2020 expense guidance of
$410 million to $420 million.1
- Net loss was $88.9 million, or $0.35 per share, compared to a
net loss of $120.3 million, or $0.56 per share, for the first
quarter 2019.
1 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.
2020 Financial Guidance
- For the full-year 2020, the Company anticipates total Galafold
revenue of $250 million to $260 million based on the average
exchange rates for 2019.
- Non-GAAP operating expense guidance for the full-year 2020 is
$410 million to $420 million, driven by continued investment in the
global Galafold launch, AT-GAA clinical studies, and advancing our
gene therapy pipeline.2
- Cash, cash equivalents, and marketable securities totaled
$338.9 million at March 31, 2020. The current cash position is
anticipated to fund ongoing operations now well into 2H2022.
2 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.
Anticipated 2020 Milestones by
Program
Galafold (migalastat) Oral Precision
Medicine for Fabry Disease
- On track to meet full-year 2020 revenue guidance range of $250
million to $260 million
- Continued geographic expansion
- Registry and other Phase 4 supportive studies underway
AT-GAA for Pompe Disease
- Plans to initiate a Rolling Biologics License Application (BLA)
for AT-GAA in 2020, with addition of complete clinical results for
PROPEL in 1H2021 to support full approval
- Retrospective natural history study data in approximately 100
enzyme replacement therapy treated Pompe patients
- Additional supportive studies, including an open-label study in
12- to <18-year-old patients
Gene Therapy Portfolio
- Report further safety and efficacy data in the CLN6 Batten
disease Phase 1/2 study and advance regulatory discussions to
finalize clinical and regulatory path
- Report initial data from the CLN3 Batten disease Phase 1/2
study and advance regulatory discussions to finalize clinical and
regulatory path
- Continue IND-enabling toxicology work in Pompe disease and
progress towards IND
- Additional preclinical data expected across multiple programs
with disclosure of up to two additional IND candidates
- Manufacturing advancements across portfolio
Conference Call and Webcast
Amicus Therapeutics will host a conference call
and audio webcast today, May 7, 2020 at 8:30 a.m. ET to discuss the
first quarter 2020 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: 1287896.
A live audio webcast and related presentation
materials can also be accessed via the Investors section of the
Amicus Therapeutics corporate website at ir.amicusrx.com. 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 May 7, 2020.
Access numbers for this replay are 855-859-2056 (U.S./Canada) and
404-537-3406 (international); conference ID: 1287896.
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 over 40 countries around the world, including the U.S.,
EU, Japan and others.
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 MeasuresIn
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, including as they are impacted by COVID-19 related
disruption, are based on current information. The potential
impact on operations from the COVID-19 pandemic is inherently
unknown and cannot be predicted with confidence and may cause
actual results and performance to differ materially from the
statements in this release, including without limitation, because
of the impact on general political and economic conditions,
including as a result of efforts by governmental authorities to
mitigate COVID-19, such as travel bans, shelter in place orders and
third-party business closures and resource allocations,
manufacturing and supply chain disruptions and limitations on
patient access to commercial or clinical product. In addition to
the impact of the COVID-19 pandemic, 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. Statements regarding corporate financial guidance
and financial goals and the attainment of such goals. 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, 2019 and the Quarterly Report filed on Form 10-Q
to be 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:
Amicus TherapeuticsAndrew FaughnanDirector,
Investor Relationsafaughnan@amicusrx.com (609) 662-3809
FOLD–G
TABLE 1
Amicus
Therapeutics, Inc.Consolidated Statements of
Operations(Unaudited)(in
thousands, except share and per share amounts)
|
Three Months Ended March 31, |
|
2020 |
|
2019 |
Net product sales |
$ |
60,525 |
|
|
$ |
34,046 |
|
Cost of goods sold |
6,552 |
|
|
4,055 |
|
Gross profit |
53,973 |
|
|
29,991 |
|
Operating expenses: |
|
|
|
Research and development |
89,120 |
|
|
64,593 |
|
Selling, general, and administrative |
40,215 |
|
|
44,303 |
|
Changes in fair value of contingent consideration payable |
931 |
|
|
1,383 |
|
Depreciation and amortization |
1,764 |
|
|
991 |
|
Total operating expenses |
132,030 |
|
|
111,270 |
|
Loss from operations |
(78,057 |
) |
|
(81,279 |
) |
Other income (expense): |
|
|
|
Interest income |
1,515 |
|
|
2,639 |
|
Interest expense |
(3,729 |
) |
|
(6,454 |
) |
Loss on exchange of convertible notes |
— |
|
|
(36,123 |
) |
Other (expense) income |
(8,316 |
) |
|
1,086 |
|
Loss before income tax |
(88,587 |
) |
|
(120,131 |
) |
Income tax expense |
(361 |
) |
|
(168 |
) |
Net loss attributable
to common stockholders |
$ |
(88,948 |
) |
|
$ |
(120,299 |
) |
Net loss attributable to
common stockholders per common share — basic and diluted |
$ |
(0.35 |
) |
|
$ |
(0.56 |
) |
Weighted-average common shares outstanding — basic and
diluted |
256,968,248 |
|
|
213,519,287 |
|
TABLE 2
Amicus
Therapeutics, Inc.Consolidated Balance
Sheets(Unaudited)(in thousands,
except share and per share amounts)
|
March 31,2020 |
|
December 31,2019 |
Assets |
|
|
|
Current assets: |
|
|
|
Cash and cash equivalents |
$ |
123,231 |
|
|
$ |
142,837 |
|
Investments in marketable securities |
215,642 |
|
|
309,903 |
|
Accounts receivable |
40,555 |
|
|
33,284 |
|
Inventories |
12,831 |
|
|
14,041 |
|
Prepaid expenses and other current assets |
15,690 |
|
|
20,008 |
|
Total current assets |
407,949 |
|
|
520,073 |
|
Operating lease right-of-use assets, less accumulated amortization
of $6,260 and $5,342 at March 31, 2020 and December 31, 2019,
respectively |
32,501 |
|
|
33,315 |
|
Property and equipment, less accumulated depreciation of $19,260
and $17,604 at March 31, 2020 and December 31, 2019,
respectively |
47,688 |
|
|
47,705 |
|
In-process research & development |
23,000 |
|
|
23,000 |
|
Goodwill |
197,797 |
|
|
197,797 |
|
Other non-current assets |
29,414 |
|
|
28,317 |
|
Total
Assets |
$ |
738,349 |
|
|
$ |
850,207 |
|
Liabilities and
Stockholders’ Equity |
|
|
|
Current liabilities: |
|
|
|
Accounts payable |
$ |
16,239 |
|
|
$ |
21,722 |
|
Accrued expenses and other current liabilities |
66,722 |
|
|
98,651 |
|
Deferred reimbursements |
1,250 |
|
|
1,250 |
|
Operating lease liabilities |
7,503 |
|
|
7,189 |
|
Total current liabilities |
91,714 |
|
|
128,812 |
|
Deferred reimbursements |
8,906 |
|
|
8,906 |
|
Convertible notes |
2,167 |
|
|
2,131 |
|
Senior secured term loan |
147,569 |
|
|
147,374 |
|
Contingent consideration payable |
23,612 |
|
|
22,681 |
|
Deferred income taxes |
5,051 |
|
|
5,051 |
|
Operating lease liabilities |
52,522 |
|
|
53,531 |
|
Other non-current liabilities |
4,214 |
|
|
5,296 |
|
Total liabilities |
335,755 |
|
|
373,782 |
|
Commitments and
contingencies |
|
|
|
Stockholders’ equity: |
|
|
|
Common stock, $0.01 par value,
500,000,000 shares authorized, 257,449,955 and 255,417,869 shares
issued and outstanding at March 31, 2020 and December 31, 2019,
respectively |
2,607 |
|
|
2,598 |
|
Additional paid-in
capital |
2,238,346 |
|
|
2,227,225 |
|
Accumulated other
comprehensive loss: |
|
|
|
Foreign currency translation adjustment |
6,981 |
|
|
2,785 |
|
Unrealized (loss) gain on available-for-sale securities |
(169 |
) |
|
40 |
|
Warrants |
12,387 |
|
|
12,387 |
|
Accumulated deficit |
(1,857,558 |
) |
|
(1,768,610 |
) |
Total stockholders’
equity |
402,594 |
|
|
476,425 |
|
Total Liabilities and
Stockholders’ Equity |
$ |
738,349 |
|
|
$ |
850,207 |
|
TABLE 3
Amicus
Therapeutics, Inc.Reconciliation of Non-GAAP
Financial Measures(in thousands)
|
Three Months Ended March
31, |
|
2020 |
|
2019 |
Total operating expenses - as reported GAAP |
$ |
132,030 |
|
$ |
111,270 |
|
Research and development: |
|
|
|
|
Share-based compensation |
|
5,253 |
|
|
5,032 |
|
Selling, general and administrative: |
|
|
|
|
Share-based compensation |
|
7,343 |
|
|
7,712 |
|
Changes in fair value of contingent consideration payable |
|
931 |
|
|
1,383 |
|
Depreciation and amortization |
|
1,764 |
|
|
991 |
|
Total operating expense adjustments to reported
GAAP |
|
15,291 |
|
|
15,118 |
|
Total operating expenses
- as adjusted |
$ |
116,739 |
|
$ |
96,152 |
|
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