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, 2019. The Company also summarized
recent program updates and reiterated its full-year 2019 guidance.
Corporate Highlights for 1Q19 and Early
2Q19
- Global revenue for Galafold (migalastat) in the first
quarter of 2019 was $34.0M, in line with management expectations,
and continues to track toward full-year 2019 revenue guidance of
$160M-$180M and 1,000+ patients by year-end. The first
quarter revenue represented a year-over-year increase of 104% from
total revenue of $16.7 million in the first quarter of
2018. Patient adoption and wide prescriber experience
continues globally for Galafold. Highest number since
launch of net new patients added in 1Q19 (150+) with higher than
anticipated prescriptions in EU, Japan and US, and continued global
compliance and adherence rates exceeding 90%. Over 200 patient
referral forms (PRFs) written now by over 90 physicians in the
United States, with continued strong reimbursement from payors,
since the launch in August 2018.
- New intellectual property
issued for Galafold. The new patent covers a method of
treatment and provides additional protection through 2038.
- Positive Initial Proof-of-Concept for Preclinical Pompe
Gene Therapy Data. Positive preclinical results showed
robust uptake and glycogen reduction in multiple tissues, including
brain and spinal cord. Additional preclinical studies underway to
select a clinical candidate in 2019. This program is significantly
ahead of schedule reflecting a strong collaboration with the
University of Pennsylvania.
- Enrollment momentum in ongoing pivotal PROPEL study in
Pompe disease. The PROPEL study is now underway at 24
participating sites and is on track to achieve full enrollment of
~100 patients by year-end 2019. Significant enthusiasm from Pompe
patient and physician community continues.
- Additional ERT-Switch Patients (Cohort 4) Fully
Enrolled in Pompe Phase 1/2 Clinical Study: Initial
6-month data in Cohort 4, and complete 24-month data in Cohorts
1-3, anticipated in 2H19.
- Additional data from Phase 1/2 CLN6 Batten disease
study anticipated 3Q19. A total of 12 patients have
received a single administration of AAV9-CLN6 gene therapy
(exposure ranging from ~5 to 39 months). The top-line data are
expected to include two-year results in approximately half the
patients in 3Q19 with more detail at a scientific congress later in
2019.
- Low dose cohort fully enrolled in Phase 1/2 study in
CLN3 Batten disease. A total of three patients were dosed
in the low dose cohort in the CLN3 Batten disease study with no
serious adverse events to date following a single administration of
AAV9-CLN3 gene therapy. The high dose cohort is expected to
commence in the coming months.
- Robust gene therapy pipeline continues to advance
toward important data milestones. Additional preclinical
proof-of-concept data in Pompe, initial preclinical data in Fabry,
and further advancement of neurologic lysosomal disorder
preclinical programs, expected throughout 2019.
- Strong financial position to continue executing the
Galafold launch, advance development programs and build leadership
position in gene therapy science, development and manufacturing
capabilities and capacity. The current cash position of
approximately $438.3 million at March 31, 2019 is expected to fund
ongoing operations into at least mid-2021.
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics, Inc. stated, “During the first
quarter and into the second quarter, we have made tremendous
progress in executing across our five key strategic priorities for
2019. With the success of the global Galafold launch and long-term
opportunity in Fabry, our leadership position in Pompe disease with
a late-stage Phase 3 program and now positive preclinical data in
gene therapy, and a rapidly advancing gene therapy pipeline that
includes two clinical-stage programs in Batten disease, we are
marching closer toward our vision to treat 5,000+ patients and
achieve $1B+ in revenue by 2023. As we continue to build a leading
global biotechnology company in rare metabolic diseases,
specifically within the field of human genomic medicine, we remain
more focused than ever on developing groundbreaking new medicines
and hopefully one day cures, and delivering them to people living
with these life-threatening conditions as quickly as possible.”
First Quarter 2019 Financial
Results
- Total revenue in the first quarter 2019 was $34.0 million, a
year-over-year increase of 104% from total revenue of $16.7 million
in the first quarter of 2018. This includes year-over-year
operational revenue growth measured at constant currency exchange
rates of 114.9%, offset by negative currency impact of $1.8M, or
11.0%.
- Cash, cash equivalents, and marketable securities totaled
$438.3 million at March 31, 2019, compared to $504.6 million at
December 31, 2018.
- Total operating expenses of $111.3 million for the first
quarter of 2019 increased as compared to $70.3 million in the first
quarter of 2018, reflecting continued investments in the Galafold
launch, Pompe program, and gene therapy pipeline.
- Net loss was $120.3 million, or $0.56 per share, compared to a
net loss of $49.9 million, or $0.28 per share, for the first
quarter 2018.
2019 Key Strategic
Priorities
- Nearly double 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
For the full-year 2019 the Company anticipates
total Galafold revenue of $160 million to $180 million. Growth in
2019 is expected to be driven by continued growth in EU markets,
further geographic expansion, and successful adoption in the first
full year of launch in the U.S. and Japan. Consistent with Galafold
adoption trends and ordering patterns seen in previous years,
Amicus expects higher revenue growth in the second and fourth
quarters of 2019.
Cash, cash equivalents, and marketable
securities totaled $438.3 million at March 31, 2019. 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
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.
- Additional preclinical data including next-generation gene
therapies for Fabry and Pompe
- Selection of Pompe AAV gene therapy clinical candidate to move
into IND-enabling studies
Conference Call and
WebcastAmicus Therapeutics will host a conference call and
audio webcast today, May 8, 2019 at 8:30 a.m. ET to discuss the
first 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: 8358807.
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 May 8, 2019.
Access numbers for this replay are 855-859-2056 (U.S./Canada) and
404-537-3406 (international); conference ID: 8358807.
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.
Non-GAAP Financial
MeasuresOperational revenue growth excluding the impact of
foreign currency exchange rates is a non-GAAP financial measure and
should not be considered a replacement for, and should be read
together with, the most comparable GAAP financial
measure.
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/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(Unaudited)(in
thousands, except share and per share amounts)
|
Three Months Ended March 31, |
|
2019 |
|
2018 |
Revenue: |
|
|
|
Net product sales |
$ |
34,046 |
|
|
$ |
16,696 |
|
Cost of goods sold |
4,055 |
|
|
2,615 |
|
Gross profit |
29,991 |
|
|
14,081 |
|
Operating expenses: |
|
|
|
Research and development |
64,593 |
|
|
40,798 |
|
Selling, general and administrative |
44,303 |
|
|
27,396 |
|
Changes in fair value of contingent consideration payable |
1,383 |
|
|
1,100 |
|
Depreciation and amortization |
991 |
|
|
969 |
|
Total operating expenses |
111,270 |
|
|
70,263 |
|
Loss from operations |
(81,279 |
) |
|
(56,182 |
) |
Other income (expense): |
|
|
|
Interest income |
2,639 |
|
|
1,737 |
|
Interest expense |
(6,454 |
) |
|
(4,488 |
) |
Loss on exchange of convertible notes |
(36,123 |
) |
|
— |
|
Change in fair value of derivatives |
— |
|
|
4,861 |
|
Other income |
1,086 |
|
|
2,764 |
|
Loss before income tax |
(120,131 |
) |
|
(51,308 |
) |
Income tax (expense)
benefit |
(168 |
) |
|
1,392 |
|
Net loss attributable
to common stockholders |
$ |
(120,299 |
) |
|
$ |
(49,916 |
) |
Net loss attributable to
common stockholders per common share — basic and diluted |
$ |
(0.56 |
) |
|
$ |
(0.28 |
) |
Weighted-average common shares
outstanding — basic and diluted |
|
213,519,287 |
|
|
|
175,977,700 |
|
TABLE 2
Amicus
Therapeutics, Inc.Consolidated Balance
Sheets(Unaudited)(in thousands,
except share and per share amounts)
|
March 31, 2019 |
|
December 31, 2018 |
Assets |
|
|
|
Current assets: |
|
|
|
Cash and cash equivalents |
$ |
96,349 |
|
|
$ |
79,749 |
|
Investments in marketable securities |
341,978 |
|
|
424,403 |
|
Accounts receivable |
22,960 |
|
|
21,962 |
|
Inventories |
8,167 |
|
|
8,390 |
|
Prepaid expenses and other current assets |
13,592 |
|
|
16,592 |
|
Total current assets |
483,046 |
|
|
551,096 |
|
Operating and finance lease right-of-use assets, less accumulated
amortization of $2,111 and $0 at March 31, 2019 and December 31,
2018, respectively |
36,308 |
|
|
— |
|
Property and equipment, less accumulated depreciation of $15,794
and $15,671 at March 31, 2019 and December 31, 2018,
respectively |
13,286 |
|
|
11,375 |
|
In-process research & development |
23,000 |
|
|
23,000 |
|
Goodwill |
197,797 |
|
|
197,797 |
|
Other non-current assets |
11,265 |
|
|
6,683 |
|
Total
Assets |
$ |
764,702 |
|
|
$ |
789,951 |
|
Liabilities and
Stockholders’ Equity |
|
|
|
Current liabilities: |
|
|
|
Accounts payable, accrued expenses, and other current
liabilities |
$ |
60,596 |
|
|
$ |
80,625 |
|
Deferred reimbursements |
2,750 |
|
|
5,500 |
|
Operating and finance lease liabilities |
2,523 |
|
|
— |
|
Total current liabilities |
65,869 |
|
|
86,125 |
|
Deferred reimbursements |
11,406 |
|
|
10,156 |
|
Convertible notes |
22,052 |
|
|
175,006 |
|
Senior secured term loan |
146,766 |
|
|
146,734 |
|
Contingent consideration payable |
20,767 |
|
|
19,700 |
|
Deferred income taxes |
|
6,465 |
|
|
6,465 |
|
Operating and finance lease liabilities |
36,100 |
|
|
— |
|
Other non-current liabilities |
|
3,609 |
|
|
|
2,853 |
|
Total liabilities |
313,034 |
|
|
447,039 |
|
Commitments and
contingencies |
|
|
|
Stockholders’ equity: |
|
|
|
Common stock, $0.01 par value,
500,000,000 shares authorized, 230,180,714 and 189,383,924 shares
issued and outstanding at March 31, 2019 and December 31, 2018,
respectively |
2,347 |
|
|
1,942 |
|
Additional paid-in
capital |
1,970,607 |
|
|
1,740,061 |
|
Accumulated other
comprehensive loss: |
|
|
|
Foreign currency translation adjustment |
(1,309 |
) |
|
495 |
|
Unrealized gain (loss) on available-for-sale securities |
157 |
|
|
(427 |
) |
Warrants |
12,387 |
|
|
13,063 |
|
Accumulated deficit |
(1,532,521 |
) |
|
(1,412,222 |
) |
Total stockholders’
equity |
451,668 |
|
|
342,912 |
|
Total Liabilities and
Stockholders’ Equity |
$ |
764,702 |
|
|
$ |
789,951 |
|
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